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1259 Results

Closing the Evidence Gap for Drugs in Children — Measures to Strengthen the Pediatric Research Equity Act

Liu ITT, Bourgeois F - New England Journal of Medicine

  • Commentary & Opinion
| April 2026
  • Innovation Incentives and Competition
  • Regulation and Clinical Evidence
Talk bubble graphics representing commentary and opinion.
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Anticompetitive Mergers — Pharmaceutical Buyouts as a Strategy for Maintaining Market Dominance

Tu SS, King J - New England Journal of Medicine

  • Commentary & Opinion
| April 2026
  • Innovation Incentives and Competition
Talk bubble graphics representing commentary and opinion.
Pharmaceutical “killer acquisitions,” in which incumbent firms buy out smaller competitors to shelve or delay their products, pose a threat to competition and innovation that current antitrust frameworks under the Hart-Scott-Rodino Act are poorly equipped to catch. The authors recommend coordinated legislative and regulatory reforms to better protect competition and patient interests, including lowering notification thresholds for concentrated industries, accounting for intangible assets like patents in merger valuations, and applying heightened scrutiny to acquisitions involving overlapping products.
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Skinny Labels at the Supreme Court—Generic Competition, Patent Inducement, and Affordable Medicines

Tu SS, Tessema FA, Kesselheim AS - JAMA

  • Commentary & Opinion
| April 2026
  • Innovation Incentives and Competition
  • Price, Value, and Access
Talk bubble graphics representing commentary and opinion.
The skinny label pathway, which allows generic manufacturers to enter the market by omitting patented indications from their labels, is under threat following Federal Circuit decisions in GlaxoSmithKline v. Teva and Amarin v. Hikma, which expanded induced infringement liability to include truthful, FDA-aligned statements and contributed to a sharp drop in skinny labeled generic entries. With Amarin now before the Supreme Court, the authors recommend ways the Court can clarify the skinny label safe harbor, warning that failure to do so risks undermining a cornerstone of the Hatch-Waxman Act and prolonging brand-name drug monopolies.
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Out-of-Pocket Spending for Insulin by Medicare Beneficiaries After Monthly Caps

Hong D, Cantos K, Morgenstern D, Dasgupta S, Kesselheim AS, Song Z, Tong AY, Chadaram R, Potter D, Patorno E, Avorn J, Rome BN - JAMA Internal Medicine

  • Research Publications
| April 2026
  • Price, Value, and Access
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In an interrupted time-series analysis of 4.8 million Medicare beneficiaries with type 2 diabetes from 2019-2023, implementation of a $35 monthly insulin out-of-pocket cap was associated with substantial declines in insulin out-of-pocket spending, modest increases in insulin use, and decreases in patient blood glucose levels. These findings suggest that federal cost-sharing caps can meaningfully improve patient access and adherence to essential medicines like insulin for patients with chronic conditions.
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Industry Payments and Prescribing of Brand-Name Multiple Sclerosis Medications in Medicare

Patel AN, Kesselheim AS, Rome BN - Neurology

  • Research Publications
| April 2026
  • Innovation Incentives and Competition
  • Price, Value, and Access
  • Regulation and Clinical Evidence
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In a cross-sectional study of clinicians who prescribed two multiple sclerosis drugs to Medicare patients, those who received payments from the brand-name drug manufacturers were more likely to prescribe the brand-name version rather than available generic equivalents. This suggests that these industry payments may be undermining generic competition and leading to higher costs for patients and the health care system.
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Global Access to Commercial CAR T Cells: A Cross-Sectional Study of Health Technology Assessment in the G20

Ge AY, Feldman WB, Kaiser MF, Rejeski K, Iacoboni G, Narula G, Chan JY, Dickinson M, Kesselheim AS, Cliff ERS - Blood

  • Research Publications
| April 2026
  • Price, Value, and Access
  • Regulation and Clinical Evidence
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Fewer than half of FDA-approved CAR T-cell therapy indications are reimbursed by health systems in the G20 countries, despite their strong clinical benefits. Many national health technology assessments cited high costs, limited evidence maturity, logistical challenges, and uncertainty about long-term outcomes as barriers to coverage, highlighting the need for new strategies and policy interventions to expand global patient access to CAR T therapies.
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Inflation Reduction Act Changes to Part D Plan Design: Lower Premiums, Higher Deductibles, and Some Smaller Formularies

Anderson DM, McEnany M, Petry SE, Anderson KE - Health Affairs

  • Research Publications
| April 2026
  • Price, Value, and Access
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National Prescribing Trends of Direct-Acting Antivirals for Hepatitis C

Kishore S, Hayden M, Johnson M, Kesselheim AS, Rome BN - JAMA

  • Research Publications
| March 2026
  • Price, Value, and Access
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National prescribing of hepatitis C direct-acting antivirals peaked at 185,677 treatment courses in 2015 but has since fallen to about 69,000 annually, far below the level needed to achieve national elimination targets. Despite shifts toward treatment among Medicaid patients, younger adults, and rural populations, overall treatment volume remains insufficient to reduce national prevalence. These findings highlight the need for expanded screening, rapid treatment models, and policy reforms to accelerate treatment in the US.
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Relevant Industry Payments to 2023–2024 Voting Members of the Advisory Committee on Immunization Practices

Alpern JD, Kesselheim AS - Journal of General Internal Medicine

  • Research Publications
| March 2026
  • Regulation and Clinical Evidence
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Among 23 physician members who voted on the ACIP between 2023 and 2024, only 5 (22%) received industry payments relevant to a vote during their tenure, of which 93% were research payments rather than personal compensation. These findings refute claims that the former ACIP was plagued with industry conflicts of interest, and the authors argue that future policy decisions impacting the committee should be data-driven.
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Brand Manufacturer Coupons and Pharmaceutical Product Hopping

Gibbons JB, DiStefano M, Anderson GF, Kesselheim AS, Socal M - JAMA Network Open

  • Research Publications
| March 2026
  • Innovation Incentives and Competition
  • Price, Value, and Access
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In an analysis of over 380,000 prescription claims for 47 biologic products, manufacturer coupon use for biologic line extension products increased by 19% relative to comparators as the originator products approached 12 years after market approval when market exclusivity protections began to expire. Among products with older originators, coupons for product hops offset nearly 40% more of patient cost-sharing than comparators, suggesting manufacturers may use coupon programs strategically to facilitate product hopping and dampen biosimilar competition.
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Fluticasone- vs Budesonide-Based Dual Therapy for COPD

Feldman WB, Ambati VL, Suissa S, Kesselheim AS, Avorn J, Schneeweiss S, Wang SV - JAMA Network Open

  • Research Publications
| March 2026
  • Regulation and Clinical Evidence
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In a large cohort study of new ICS-LABA users with COPD, the once-daily dry powder inhaler fluticasone furoate-vilanterol was associated with a 9% lower risk of moderate or severe COPD exacerbations compared with budesonide-formoterol and a 6% lower risk compared with fluticasone propionate-salmeterol, with no differences in pneumonia risk. These findings suggest that dry powder fluticasone-containing inhalers offer similar or slightly improved clinical outcomes for patients with COPD over metered-dose formulations, with substantially less greenhouse gas emissions.
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Cost-Related Medication Nonadherence After the Inflation Reduction Act

Marinacci LX, Mein S, Rome BN, Wadhera RK - JAMA Internal Medicine

  • Research Publications
| March 2026
  • Price, Value, and Access
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The Inflation Reduction Act’s 2024 prescription changes to Medicare Part D were associated with a 4.9% reduction in cost-related medication nonadherence among Medicare beneficiaries relative to privately insured beneficiaries, with an even larger decline (7.8%) among those with multiple chronic conditions. However, no corresponding improvement was observed in broader measures of health care-related financial strain, suggesting that additional policy interventions may be needed to address patient challenges with health care costs.
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Bundling Drugs: The Risks Of Combining Copay Assistance

Daval CJR, Bloomfield D, Rome BN, Kesselheim AS - Health Affairs Forefront

  • Commentary & Opinion
| March 2026
  • Innovation Incentives and Competition
  • Price, Value, and Access
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Eli Lilly’s program linking copay discounts for tirzepatide (Zepbound) to prescriptions for ixekizumab (Taltz) could constitute anticompetitive behavior that harms competition in unrelated markets without producing meaningful price competition. Th authors call on the FTC to evaluate such arrangements and urge Congress to regulate copay assistance programs in commercial insurance.
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The Case Against Six-Month Limits on Buprenorphine Telehealth Prescribing

Klein DE, Bullen M, Zhu DT, Kesselheim AS - Annals of Internal Medicine

  • Commentary & Opinion
| March 2026
  • Price, Value, and Access
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The Drug Enforcement Agency’s (DEA) new rule requiring in-person visits after six months of telehealth-initiated buprenorphine treatment lacks clinical or public health justification and could disrupt care for patients with opioid use disorder. The authors contend that the rule’s seven exceptions are largely impractical for outpatient addiction settings and urge for the removal of the arbitrary time limit.
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Scope and Characteristics of Patients Filling Medications Through Specialty Pharmacies by Ownership Type

Anderson KE, McEnany M, Mattingly TJ - Health Affairs Scholar

  • Research Publications
| March 2026
  • Price, Value, and Access
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Using Colorado all-payer claims data, the authors found that while only 0.8% of insured patients filled medications at specialty pharmacies, these users were older, more often female, more socially vulnerable, and had significantly higher prescription use than other patients. Vertically integrated specialty pharmacies accounted for most fills, and their users exhibited higher social vulnerability and more complex medication needs, suggesting that legislation banning PBM or insurer ownership of specialty pharmacies could disproportionately disrupt patient access.
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Research Submission to Inform Medicare Drug Price Negotiation, IPAY 2028

Martin MJ, Hwang TJ, Tibau A, Kesselheim AS - Centers for Medicare and Medicaid Services (CMS)

  • Policy Interactions
| March 2026
  • Price, Value, and Access
  • Regulation and Clinical Evidence
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PORTAL submits summaries of peer-reviewed research to inform CMS’s assessment of 15 drugs selected for Medicare price negotiation in Initial Price Applicability Year (IPAY) 2028, including Anoro Ellipta, Biktarvy, Botox, Cimzia, Cosentyx, Entyvio, Erleada, Kisqali, Lenvima, Orencia, Rexulti, Tradjenta, Trulicity, Verzenio, and Xeljanz.
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List Price Reductions Among Brand-Name ICS-LABA Inhalers in 2024 Were Associated With Increased Generic Uptake

Han J, Wang SV, Kesselheim AS, Avorn J, Feldman WB - Health Affairs

  • Research Publications
| March 2026
  • Price, Value, and Access
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Following the American Rescue Plan Act’s removal of the Medicaid rebate cap in January 2024, brand-name ICS-LABA manufacturers reduced list prices by 21-50%, and generic ICS-LABA use immediately increased by 23%, driven by gains in commercial plans and Medicare. The authors conclude that list these price reductions likely limited brand-name manufacturers’ ability to offer large rebates to PBMs, disrupting financial incentives that had previously suppressed generic uptake.
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Clinical Benefit and the Trump Administration’s Cancer Medicine Reforms

Hwang TJ, Vokinger KN - Lancet Oncology

  • Commentary & Opinion
| March 2026
  • Price, Value, and Access
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CMS should adopt validated value frameworks like the ESMO-MCBS in Medicare drug price negotiation to target the deepest discounts at low-benefit drugs, align international reference pricing baskets with comparably large markets to avoid delayed access abroad, and pass savings from these programs on to patients through reduced cost-sharing.
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New Medicare Payment Policy on Drug Shortages

Hwang TJ, Choueiri TK, Vokinger KN - JAMA Health Forum

  • Commentary & Opinion
| February 2026
  • Price, Value, and Access
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CMS’s new Medicare add-on payment for hospitals to maintain 6-month buffer stocks of essential medicines is an important step but is unlikely to meaningfully reduce drug shortages given its narrow hospital eligibility and the limited reimbursement (about 11% of costs). The authors argue that more durable solutions, such as quality-based purchasing and reform of inflation rebates for generics are needed to stabilize the US drug supply.
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The Growing Success of Medicare Drug-Price Negotiation

Rome BN, Kesselheim AS - New England Journal of Medicine

  • Commentary & Opinion
| February 2026
  • Price, Value, and Access
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The second round of Medicare drug price negotiations achieved average price reductions of 44% across 15 drugs, double the savings of the first round. These added savings may reflect both improvements in CMS’ negotiating strategy as well as the negotiation of more low-rebate drugs, such as cancer therapies, for which the savings potential is greater. Despite legal and political challenges, the authors conclude the program is delivering substantial savings and should be expanded to further improve drug affordability in Medicare.
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Comments on Proposed Rule Implementing the Guarding US Medicare Against Rising Drug Costs (GUARD) Model

Hwang TJ, Venkateswaran S, Cai CL, Krishnan A, Martin MJ, Kesselheim AS - Centers for Medicare and Medicaid Services (CMS)

  • Policy Interactions
| February 2026
  • Price, Value, and Access
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The commenters support GUARD Model’s aim of lowering Medicare Part D prescription drug spending and recommen that CMS narrow the reference country basket, use median pricing for international benchmarks, and aggregate international pricing data across dosage forms and strengths to identify the lowest available price.
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Comments on Proposed Rule Implementing the Global Benchmark for Efficient Drug Pricing (GLOBE) Model

Hwang TJ, Venkateswaran S, Cai CL, Krishnan A, Martin MJ, Kesselheim AS - Centers for Medicare and Medicaid Services (CMS)

  • Policy Interactions
| February 2026
  • Price, Value, and Access
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The commenters support the GLOBE Model’s goal of reducing Medicare Part B drug costs through international reference pricing and recommends that CMS use a narrower basket of economically comparable reference countries and define the benchmark using the median of country-level prices rather than the proposed approach.
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Brief of 76 Scholars of Law, Business, Economics, and Medicine in Hikma v. Amarin (Merits)

Carrier MA, Duan C, Tu SS, Kesselheim AS - US Supreme Court

  • Policy Interactions
| February 2026
  • Innovation Incentives and Competition
  • Price, Value, and Access
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Filed in support of Hikma, the amici outline that patent inducement liability has long required specific, unambiguous, and affirmative conduct, and that the Federal Circuit erred by allowing inducement claims based on speculative inferences from generic drug labels, AB-rating designations, and general marketing statements. Such expansion of patent inducement undermines the “skinny label” pathway for generic drugs, distorts innovation incentives toward low-value method-of-use patents, and threatens patient access to affordable medicines.
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Law, Politics, and Expert Panels at the US Food and Drug Administration

Daval CJ, Kesselheim AS - JAMA Health Forum

  • Commentary & Opinion
| February 2026
  • Regulation and Clinical Evidence
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The FDA’s recent shift from formal advisory committees to ad hoc “expert panels” risks undermining the agency’s scientific independence by soliciting advice that appears aligned with predetermined policy positions. The authors argue this approach raises legal concerns under the Federal Advisory Committee Act and reflects a troubling prioritization of political signaling over evidence-based decisionmaking.
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Ensuring Integrity of the Medicaid Drug Rebate Program

Abraham PA, Kannarkat JT, Rome BN, Darrow JJ - Therapeutic Innovation & Regulatory Science

  • Commentary & Opinion
| February 2026
  • Price, Value, and Access
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Although the Medicaid Drug Rebate Program has generated substantial savings, persistent drug misclassification and ambiguous price-reporting rules have led to billions in unpaid rebates. The authors conclude that CMS’s 2024 final rule improves program oversight, but stronger enforcement and auditing practices are needed to safeguard the integrity of the program.
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Risk of Neutropenia-Related Hospitalisation Among Clozapine Initiators

Sarpatwari A, Mahesri M, Lii J, Lee SB, Zakoul H, Toyserkani GA, Zhou EH, LaCivita C, Dal Pan GJ, Bykov K, Moran L, Avorn J, Kesselheim AS, Huybrechts KF - BMJ Mental Health

  • Research Publications
| February 2026
  • Regulation and Clinical Evidence
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Clozapine initiators had a markedly higher relative risk of neutropenia-related hospitalization than olanzapine initiators, especially within the first 6 months after initiation, though absolute risks were very low. The findings help contextualize FDA’s 2025 decision to remove the clozapine risk evaluation and mitigation strategy (REMS) while reinforcing the importance of continued monitoring for neutropenia in patients taking clozapine.
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Conflicts of Interest in United States Food and Drug Administration Advisory Committees: A Systematic Literature Review

Gentilini A, Raymakers AJN, Rand LZ - Value in Health

  • Research Publications
| February 2026
  • Regulation and Clinical Evidence
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Across 18 studies, financial conflicts of interest were common among FDA advisory committee participants, affecting 15% to over 70% of meetings for committee members and about one quarter of public speakers. While evidence linking member conflicts to committee voting outcomes was mixed, public speakers with financial conflicts were consistently 3 to 6 times more likely to provide testimony in favor of the approval of the drug under review.
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Priority Without Progress: The FDA’s Neglected Tropical Disease Voucher Program After 18 Years

Goh M, Outterson K, Kesselheim AS - Health Affairs Scholar

  • Research Publications
| February 2026
  • Innovation Incentives and Competition
  • Regulation and Clinical Evidence
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Of the 14 priority review vouchers awarded by the FDA for neglected tropical disease (NTD) products since 2007, most went to existing therapies already widely used outside the US, with FDA approval often occurring years after use in regions in which the NTDs were prevalent. These findings suggest that the NTD voucher program largely rewards regulatory submissions rather than innovation. Policymakers should abandon the voucher or implement reforms that tie voucher eligibility to equitable pricing and demonstrable gains in patient access.
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Physician Awareness of FDA’s Relaxation of REMS-Required Laboratory Testing Requirements and Changes in Prescribing Practices During the COVID-19 Pandemic

Hwang CS, Lu Z, Russo M, Zakoul H, Toyserkani GA, Zhou EH, LaCivita C, Dal Pan GJ, Kesselheim AS, Sarpatwari A - Drug Safety

  • Research Publications
| January 2026
  • Regulation and Clinical Evidence
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About half of surveyed physicians were aware of the FDA’s temporary policy during the COVID-19 pandemic that allowed clinicians to use medical judgment in place of certain REMS-required laboratory tests, and far fewer physicians reported changing their testing practices in response, often citing the perceived clinical importance of continued monitoring. Targeted, reinforced FDA communication may be needed to ensure clinicians receive and act on temporary policy changes moving forward.
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The 25th Anniversary of a Nearly Unknown Health Policy Turning Point

Avorn J - New England Journal of Medicine

  • Commentary & Opinion
| January 2026
  • Price, Value, and Access
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A grassroots campaign by clinicians in the late 1990s nearly succeeded in mandating universal health care coverage in Massachusetts, laying the foundation for later reforms like “Romneycare” and the Affordable Care Act. Citizen-driven efforts can spark major policy changes, even with limited resources, offering lessons for today’s ongoing challenges in expanding access to affordable health care.
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Tumultuous Development of Venetoclax in t(11;14) Multiple Myeloma

Kaiser MF, Cliff ERS - Journal of Clinical Oncology

  • Research Publications
| January 2026
  • Innovation Incentives and Competition
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Venetoclax (Venclexta) demonstrated early, biologically coherent activity in t(11;14) myeloma, but an all‑comer phase 3 strategy produced safety concerns and regulatory freezes that ultimately blocked access even for likely responders. Drug development and regulatory pathways must better accommodate strong biological signals in small subgroups to avoid repeating failures that strand effective therapies.
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Physician Antibiotic Prescribing Preferences in Community Acquired Pneumonia and Complicated Urinary Tract Infections: A National Discrete Choice Experiment

Hong D, Kesselheim AS, Metlay JP, Powers JH, Morlock R, Feldman WB - Open Forum Infectious Diseases

  • Research Publications
| January 2026
  • Price, Value, and Access
  • Regulation and Clinical Evidence
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In a discrete choice experiment, physicians were found to prioritize rapid symptom improvement and dosing convenience over side-effect risks and future antibiotic resistance when selecting antibiotics to treat community-acquired pneumonia and complicated urinary tract infections, with variation by specialty and practice setting. These prescribing preferences can inform antibiotic stewardship efforts and guide development of antibiotic trials with clinically meaningful endpoints.
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Physician Perspectives on Real-World Evidence for Prescribing to Older Patients

Rand LZG, Wang J, Woloshin S, Wang SV, Darrow JJ, Kesselheim AS - Journal of Pharmacy Practice

  • Research Publications
| January 2026
  • Regulation and Clinical Evidence
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Interviewed physicians saw real-world evidence (RWE) as valuable for filling evidence gaps when prescribing medications to older adults, but expressed concerns about data quality, confounding, and interpretation of such information, emphasizing reliance on trusted sources such as professional guidelines. Closer collaboration between RWE researchers and guideline developers is necessary to ensure actionable, clinically meaningful dissemination of RWE.
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Out-Of-Pocket Spending for Biologic Drugs After Biosimilar Competition for Medicare Patients

Riegler JS, Kesselheim AS, Rome BN - JAMA Network Open

  • Research Publications
| January 2026
  • Innovation Incentives and Competition
  • Price, Value, and Access
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Among nearly 275,000 Medicare Advantage patients who used one of seven clinician‑administered biologics, average annual out-of-pocket spending fell by about $94 within four years of biosimilar competition, with savings concentrated among patients with coinsurance or deductibles. These findings contrast reports of inconsistent out-of-pocket savings for patients with private insurance, suggesting that Medicare’s reimbursement for biologics based on average sales prices allows biosimilar competition to translate more directly into patient savings.
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Generic Liraglutide—Overlooked but Not Forgotten

Gondi S, Kesselheim AS, Rome BN - New England Journal of Medicine

  • Commentary & Opinion
| January 2026
  • Innovation Incentives and Competition
  • Price, Value, and Access
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The arrival of generic liraglutide offers a lower-cost option for treating diabetes and obesity, which could help expand access and reduce financial strain on health systems grappling with skyrocketing GLP-1 drug spending. Insurers, clinicians, and patients should consider generic liraglutide, noting its proven effectiveness and safety, even though newer GLP-1 medications are more convenient and effective.
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The Curious Constraints on Clofazimine

Goh M, Eisenkraft Klein D, Kesselheim AS - Journal of Antimicrobial Chemotherapy

  • Research Publications
| January 2026
  • Price, Value, and Access
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Despite decades of safe use, access to clofazimine, a WHO-recommended treatment for leprosy and emerging therapy for drug-resistant tuberculos,remains limited in the US and other high income countries due to reliance on a single manufacturer and complex expanded access pathways. Streamlining regulatory and procurement processes, as well as expanded public manufacturing capcity, can help ensure equitable supply of this essential medicine.
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Combination-Only Approvals as a Barrier to Affordable HIV Care

Tu SS, Bonis T, Sinha MS, Slade DH - JAMA

  • Commentary & Opinion
| January 2026
  • Innovation Incentives and Competition
  • Regulation and Clinical Evidence
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“Combination-only” drug approvals, in which new HIV medications are approved only as part of multi-drug combinations and not as standalone products, are keeping HIV treatment costs high by blocking generic competition and preventing cost-saving regimen changes. To expand affordable access to HIV care, the FDA should require that new drugs used in combinations also be approved as monotherapies
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Tertiary Patents on Drugs Approved By the FDA

Teng TW, Tu SS, Mooney H, Bendicksen L, Gabriele SME, Wouters OJ, Feldman WB - JAMA Health Forum

  • Research Publications
| January 2026
  • Innovation Incentives and Competition
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In a review of over 300 drug-device combinations approved by the FDA from 1986 to 2023, tertiary patents on the delivery device made up more than half of the patents listed for these products.These tertiary patents extended market exclusivity periods by a median of 7.5 years, despite nearly 60% of these patents not mentioning the drug’s active ingredient. Policymakers and regulators should improve oversight of tertiary patents to mitigate such improper Orange Book listings and to ensure timely generic competition.
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Changes in Medication Use During Medicaid Continuous Enrollment and Unwinding

Rome BN, Han J, McIntyre A, Kesselheim AS, Sommers BD - JAMA Health Forum

  • Research Publications
| January 2026
  • Price, Value, and Access
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Expanded Medicaid enrollment during the COVID-19 continuous coverage period and the subsequent unwinding were associated with corresponding changes in use of prescription medications for chronic diseases, suggesting that these Medicaid policies affected patients’ access to necessary health care. States that implemented protective policies during unwinding experienced smaller decreases in medication use, highlighting the value of efforts to maintain patient access to medications during coverage transitions.
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Using Prescription Medication Utilization Trends to Evaluate Health System Resilience and Inform Future Emergency Preparedness

Dardouri M, Wright G, Saseen JJ, Nair KV, Anderson KE - Journal of Managed Care & Specialty Pharmacy

  • Research Publications
| January 2026
  • Price, Value, and Access
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In an analysis of prescription medication trends in Colorado during the COVID-19 pandemic, the authors found an immediate drop of 3.3 prescription fills per 100 insured adults, with retail pharmacy prescriptions rebounding over time due to telehealth and mail-order options while physician-administered therapies saw sustained decreases. These findings highlight importance of maintaining telehealth flexibilities and ensuring safe access to in-person treatments to build a more resilient health system for future public health emergencies.
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Stigma, Scarcity and the Price of Legitimacy: Chronic Pain Advocacy and the Politics of Pharmaceutical Partnerships in Canada

Eisenkraft Klein D, Hawkins B, Grundy Q, Schwartz R - Sociology of Health & Illness

  • Research Publications
| January 2026
  • Price, Value, and Access
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Drawing on interviews with 22 Canadian chronic pain advocates, this study shows pharmaceutical funding was widely viewed as a pragmatic response to stigma, weak public funding, and institutional neglect rather than as simple ethical capture. The authors conclude that these structural constraints entrenched pharmaceuticalized approaches to pain advocacy, even as industry ties later became a source of reputational liability.
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Characteristics of Speakers at US FDA Advisory Committee Meetings on Drug Approval

Rand LZG, Mooney H, Hong D, Eisenkraft Klein D, Kesselheim AS, Rome BN - JAMA Internal Medicine

  • Research Publications
| January 2026
  • Regulation and Clinical Evidence
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During the 161 FDA advisory committee meetings held from 2015 to 2023, patients and family members made up nearly half of all speakers during public comment periods. 82% of speakers supported drug approval, and speakers reporting conflicts of interest (COI) were 11% more likely to favor approval. These findings highlight the need for COI disclosure and broader stakeholder input at these committee meetings.
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The Pharmaceutical Industry’s Quiet Win in the One Big Beautiful Bill Act

Bucha S, Martin MJ, Feldman WB - Annals of Internal Medicine

  • Commentary & Opinion
| December 2025
  • Price, Value, and Access
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The One Big Beautiful Bill Act benefits the pharmaceutical industry by limiting Medicare’s ability to negotiate drug prices, expanding tax breaks, and shrinking Medicaid coverage. These policy changes are expected to raise health care costs, reduce patient access, and weaken recent drug pricing reforms, prioritizing industry profits over public health and affordability.
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Estimating Racial and Ethnic Disparities in Substance Use Disorder Treatment and Harm Reduction Services: Findings From a Survey of People Who Use Drugs

Gibbons JB, Bandara S, Cook BL, Byrne L, Sugarman OK, Kerins L, Pugh T, Hulsey EG, Heller D, Minna S, Saloner B - Health Services Research

  • Research Publications
| December 2025
  • Price, Value, and Access
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In a survey of 1,651 people who use drugs, Black, Hispanic, and American Indian/Alaska Native respondents reported substantially lower use of harm reduction and substance use disorder (SUD) treatment services than White respondents. The authors conclude that conventional adjustment methods in such studies can mask true inequities and recommend Institute of Medicine-aligned approaches to more accurately measure and address health disparities.
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Patient Perceptions of and Experiences with Risk Evaluation and Mitigation Strategies

Sarpatwari A, Lee SB, Zakoul H, Tekle W, Freedman A, Belitkar S, Toyserkani GA, LaCivita C, Zhou EH, Oswell KH, Dal Pan GJ, Kesselheim AS - Clinical Pharmacology & Therapeutics

  • Research Publications
| January 2026
  • Regulation and Clinical Evidence
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In interviews with patients and caregivers using seven drugs subject to FDA risk evaluation and mitigation strategy (REMS) programs, participants were found to be generally knowledgeable about REMS-related drug risks, but some encountered challenges finding prescribers, navigating testing requirements, and managing the administrative burdens of these programs. REMS programs are effective in educating patients about drug risks, but better integration of REMS into clinical workflows can improve medication access and adherence.
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Outcomes of Nononcology Accelerated Approvals (Reply)

Liu ITT, Kesselheim AS, Cliff ERS - JAMA

  • Commentary & Opinion
| December 2025
  • Regulation and Clinical Evidence
Talk bubble graphics representing commentary and opinion.
Surrogate measures used for accelerated approval are, by definition, not fully validated and may lack strong evidence linking them to clinical benefit. The authors highlight recent cases where reliance on such surrogates led to regulatory withdrawals or ongoing uncertainty, and emphasize that while accelerated approval can speed access to new treatments, it also requires careful follow-up and realistic communication about risks.
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Serial Patent Litigation: an Emerging Strategy to Delay Entry of Generic Competition

Bonis T, Kesselheim AS, Tu SS - Health Affairs Scholar

  • Commentary & Opinion
| December 2025
  • Innovation Incentives and Competition
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Brand-name drug manufacturers are increasingly filing repeated, nearly identical lawsuits, known as “serial patent litigation,” to delay the entry of generic competitors and keep drug prices high. This strategy raises costs for generic manufacturers, discourages competition, and ultimately limits patient access to affordable medicines, highlighting the need for legal reforms to protect timely generic entry.
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The Strength and Importance of Government-Funded Patents for Approved Drugs (Reply)

Gabriele SME, Martin MJ, Kesselheim AS, Tu SS - Nature Biotechnology

  • Commentary & Opinion
| December 2025
  • Innovation Incentives and Competition
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In response to a letter about their original analysis, the authors clarify that the Bayh–Dole Act does allow the government to exercise march-in rights when patented, government-funded drugs are not made available on reasonable terms, including cases of excessive pricing. TApplying these rights aligns with Congress’s original intent to ensure publicly funded medicines serve the public interest.
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Discovering a Transformative Cancer Drug: The Case of Ibrutinib

Bendicksen L, King L, Cliff ERS, Kesselheim AS - Drug Discovery Today

  • Research Publications
| December 2025
  • Innovation Incentives and Competition
  • Regulation and Clinical Evidence
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Ibrutinib, originally developed as a tool compound for autoimmune research, was repurposed into a breakthrough cancer treatment. This analysis documents how despite relatively low development costs, ibrutinib’s success was shaped by flexible regulatory approaches, public funding, and the willingness to redefine clinical trial criteria, ultimately leading to over $50 billion in sales and transforming treatment for B-cell cancers while raising affordability and access concerns.
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Constructing Opioid Legitimacy: The Canadian Pain Task Force’s Framing of the Overdose Crisis

Eisenkraft Klein D, Grundy Q, Hawkins B, Schwartz R - Journal of Law, Medicine & Ethics

  • Research Publications
| Winter 2025
  • Price, Value, and Access
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Analysis of Canadian Pain Task Force reports, consultations, and interviews shows the overdose crisis was primarily framed as a problem of illicit supply and stigma rather than prescription opioid use. This framing shaped access‑oriented policy recommendations and risks sidelining prescription‑related harms in opioid policymaking.
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Prescription Drugs Subject to a Risk Evaluation and Mitigation Strategy: Patient Perspectives on Risk Communication and the Value of Educational Materials

Chimonas S, Cosenza C, Kesselheim AS, Toyserkani GA, Oswell KH, LaCivita C, Dal Pan GJ, Sarpatwari A - Drug Safety

  • Research Publications
| November 2025
  • Regulation and Clinical Evidence
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In a survey of patients and caregivers with experience navigating FDA risk evaluation and mitigation strategy (REMS) programs, most respondents reported feeling well-informed about the safety risks of their medications, yet their understanding of REMS requirements the value of REMS educational materials varied. These findings highlight the need for clearer, more tailored educational resources and greater provider involvement to improve patient knowledge and adherence to REMS programs.
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Op-Ed: The Trump Administration Is Turning Drug Review Into Make-A-Deal

Carpenter D, Hwang TJ, Kesselheim AS - STAT

  • Commentary & Opinion
| November 2025
  • Innovation Incentives and Competition
  • Regulation and Clinical Evidence
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The FDA’s new National Priority Voucher program replaces science-based drug review with deal-making and political bargaining. Vouchers are awarded based on vague “national health priorities” and private negotiations, rather than clear, evidence-based criteria. This approach risks corruption, undermines transparency, and could harm public trust in the FDA.
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US Spending on High-Revenue Rare Disease Drugs in 2022

Mooney H, Kesselheim AS, Rome BN - American Journal of Managed Care

  • Research Publications
| November 2025
  • Price, Value, and Access
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Nearly one in five of the top 100 highest-revenue prescription drugs in the US were approved only for rare diseases, with spending on these 19 rare disease drugs totaling $45.1 billion, or 7.5% of US prescription drug spending. Excluding rare disease drugs from federal and state drug price policies limits potential savings and patient impact of efforts improve prescription drug affordability.
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Mapping Intellectual Property Abuses in the Pharmaceutical Field

Tu SS, Rutschman AS - JAMA Health Forum

  • Commentary & Opinion
| November 2025
  • Innovation Incentives and Competition
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This viewpoint provides an overview of how brand-name pharmaceutical manufacturers exploit patents, trade secrets, copyrights, and trademarks to block competition and keep drug prices high, often at the expense of patient access and public health. Stronger patent examination and other legal remedies can curb these intellectual property abuses.
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Enhancing Formulary Accessibility: a Step Toward Better Care

Agbafe VC, Kesselheim AS, Barbieri JS - Journal of General Internal Medicine

  • Commentary & Opinion
| November 2025
  • Price, Value, and Access
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Making drug formularies more accessible via federal legislation to mandate formulary transparency and integretaton with electronic health records would help clinicians align prescriptions with coverage, reduce administrative burdens, and improve patient adherence.
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Inhaler-Related Greenhouse Gas Emissions in the US: A Serial Cross-Sectional Analysis

Feldman WB, Han J, Raymakers AJN, Furie GL, Chesebro BB - JAMA

  • Research Publications
| November 2025
  • Regulation and Clinical Evidence
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Between 2014 and 2024, the greenhouse gas emissions of asthma and COPD metered-dose inhalers in the US accounted for 98% of the estimated 24.9 million metric tons of CO₂-equivalent emissions during this period, equivalent to 530,000 gas-powered cars on the road each year. Policymakers and regulators should incentivize a shift toward dry powder and soft mist inhalers while facilitating market entry of affordable low-emission metered-dose products.
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Optimizing Oncology Drug Development: Systematic Review of 22 Years of Myeloma Randomized Controlled Trials

Mainou M, Alsadhan M, Tsapa K, Visram A, Mian H, Popat R, Mai EK, Chakraborty R, Al Hadidi S, Mohan M, Szabo A, Van Oekelen O, Cliff ERS, Mohyuddin GR - Journal of the National Cancer Institute

  • Research Publications
| November 2025
  • Regulation and Clinical Evidence
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Across 123 randomized myeloma trials, studies were far less likely to succeed when overall survival was the primary endpoint and when enrolled populations were older. Endpoint selection and trial population design strongly influence apparent trial success and should be explicitly weighed by sponsors and regulators.
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Patient and Population Impacts of Multigene Panel and Pembrolizumab Coverage in Metastatic Melanoma

Weymann D, Krebs E, Pollard S, McPhail M, Bosdet I, Yip S, Weppler AM, Karsan A, Anderson H, Bubela T, Law MR, Kesselheim AS, Regier DA - Journal of the National Cancer Institute

  • Research Publications
| November 2025
  • Price, Value, and Access
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Expanding coverage for multi-gene panel testing and pembrolizumab in metastatic melanoma patients in British Columbia led to a 111-day increase in two-year patient survival and a mean per-patient cost increase of $53,963. However, these individual gains did not translate into improved population-level mortality, while overall healthcare system costs rose significantly.
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Pricing Trends and Overlapping Indications of Checkpoint Inhibitors for Cancer Treatment

Paul JM, Mitchell AP, Kesselheim AS, Rome BN - JCO Oncology Practice

  • Research Publications
| November 2025
  • Price, Value, and Access
  • Regulation and Clinical Evidence
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Despite the introduction of 11 FDA-approved checkpoint inhibitors for the treatment of cancer between 2015 and 2024, prices for this drug class decreased only slightly, in part because nearly half of approved indications were unique to a single drug, limiting direct competition. Expanding price negotiations and incentivizing comparative effectiveness research could help address persistent high prices for these therapies.
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Therapeutic Value and Regulatory Characteristics of Breakthrough Therapy Drugs in the USA and China (2021-2023): A Retrospective Cohort Study

Mao X, Yin H, Bai X, Kesselheim AS, Li G - Drug Discovery Today

  • Research Publications
| November 2025
  • Regulation and Clinical Evidence
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In this comparison of drugs granted breakthrough therapy designation (BTD) by the US FDA and China’s NMPA, BTD drugs were found in both markets to more often receive accelerated or conditional approval based on limited evidence. Many of these products were also classified as offering only minor or insufficient clinical benefit at market entry. Regulators in both countries should grant BTD more cautiously and emphasize robust, evidence-based evaluation of therapeutic value to ensure this expedited pathway delivers meaningful clinical benefits.
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Real-World Evidence in Drug Approvals at the European Medicines Agency

Bachinger M, Jankowski MA, Kesselheim AS, Krüger N - JAMA Network Open

  • Research Publications
| November 2025
  • Regulation and Clinical Evidence
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Real-world evidence (RWE) was included in fewer than 10% of European Medicines Agency (EMA) drug approval documents from 2020 to 2023, with inconsistent reporting in how RWE was defined and presented. in drug approval decisions from 2020 to 2023. Standardized assessment tools are needed to better incorporate RWE in regulatory decision-making.
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Recent Changes in Discretionary Denials of Drug Patent Challenges

Tu SS, Rai AK, Kesselheim AS - Health Affairs Scholar

  • Research Publications
| November 2025
  • Innovation Incentives and Competition
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USPTO policy changes that sharply limit administrative patent challenges for drug patents, such as discretionary denials based on “settled expectations,” make it harder to challenge weak patents, potentially delaying generic and biosimilar competition. Congress should clarify and restrict discretionary denials to preserve effective patent oversight and timely access to affordable medicines.
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US Government Spending on New Drugs with Incomplete and Postponed Mandatory Pediatric Trials

Liu ITT, Kesselheim AS - JAMA Pediatrics

  • Research Publications
| November 2025
  • Price, Value, and Access
  • Regulation and Clinical Evidence
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Among new drugs approved between 2015 and 2022 that were required to conduct pediatric studies under the Pediatric Research Equity Act (PREA), 38% had their pediatric trials delayed by more than a year, with the US government spending $27 billion on these products through Medicare and Medicaid in the interim. Current enforcement mechanisms to improve compliance with PREA requirements are weak and stricter financial penalties should be used to encourage timely completion of studies.
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Halting Hyaluronidase Hopping to Maintain the Integrity of Medicare Drug Price Negotiation

Kim J, Kesselheim AS, Rome BN - Journal of Managed Care & Specialty Pharmacy

  • Commentary & Opinion
| October 2025
  • Price, Value, and Access
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A loophole in Medicare drug price negotiation may enable manufacturers of certain intravenously infused biologic drugs to delay negotiation eligibility by launching subcutaneous versions of their products that contain hyaluronidase. CMS should prevent these reformulated versions from delaying price negotiation by bundling reformulated products with their originals to ensure the price negotiation is meeting its intended policy goals.
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Glucagon-Like Peptide-1 Receptor Agonists and Pay-Per-Click Direct-To-Consumer Advertising

Eisenkraft Klein D, Zenone M, Kesselheim AS - JAMA Network Open

  • Research Publications
| October 2025
  • Price, Value, and Access
  • Regulation and Clinical Evidence
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Over two years, $7.5 million was spent on pay-per-click (PPC) direct-to-consumer online advertising to promote semaglutide (Ozempic) in the US, with substantial spending targeting weight-loss keywords and competitor drug names, even though Ozempic is not FDA-approved for weight loss. Given that PPC advertising can influence consumer behavior, the FDA should issue guidelines on appropriate use of this advertising strategy or review PPC content to prevent misleading drug promotion.
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Flaws in the FDA’s New Priority Voucher Program

Carpenter D, Hwang TJ, Kesselheim AS - New England Journal of Medicine

  • Commentary & Opinion
| October 2025
  • Innovation Incentives and Competition
  • Regulation and Clinical Evidence
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The FDA’s new Commissioner’s National Priority Voucher (CNPV) program is flawed, with broad and opaque eligibility criteria that risk politicizing drug approvals and undermining public trust. The program may have little impact on overall drug development timelines, while imposing burdens on agency staff and opening the door to conflicts of interest. The FDA should implement strong safeguards on the CNPV program to ensure transparency, fairness, and regulatory integrity.
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Biosimilar Interchangeability and Substitution in the US: What Comes Next?

Rome BN, Bhaskar A, Kesselheim AS - American Journal of Managed Care

  • Commentary & Opinion
| October 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
  • Regulation and Clinical Evidence
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FDA regulatory changes that allow more biosimilars to be deemed interchangeable with their reference product are important steps to improve access to biosimilars. However, other policy changes are needed to boost biosimilar competition in the US, including adjusting state laws to more easily permit automatic pharmacist substitution of biosimilars, reforms to pharmacy benefit manager practices, and better clinician and patient education.
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Testimony: Public Funding of R&D and Fair Drug Prices Are Essential to Support Innovation in Biotechnology

Kesselheim AS - 119th Congress, Senate Committee on Health, Education, Labor, and Pensions

  • Policy Interactions
| October 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
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Kesselheim details how NIH funding has underpinned virtually all major biotechnology innovations and warns that the Trump administration’s proposed 40% NIH budget cut threatens future innovation. He also calls on Congress to eliminate the 4-year “biologic bonus” delay in Medicare drug price negotiation, reverse the expansion of the rare disease carve-out, and take action to address patent thickets to ensure that patients can access breakthrough innovations at fair prices. Read his written testimony.
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Testimony: Reforming the 340B Drug Pricing Program and Ensuring Access to Affordable Medications

Feldman WB - 119th Congress, Senate Committee on Health, Education, Labor, and Pensions

  • Policy Interactions
| October 2025
  • Price, Value, and Access
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Feldman expresses support for reforming the 340B program—which has grown to over $65 billion in annual drug purchases—through improved transparency, stronger HRSA oversight, limits on child sites and contract pharmacies, clearer community benefit standards, and other measures. However, he emphasizes that 340B reform alone will not lower US drug prices and should be paired with other reforms, including protecting and expanding Medicare drug price negotiation, combating patent thickets, and preserving Medicaid and ACA coverage for vulnerable patients. Read his written testimony.
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Op-Ed: Corporate Support Cannot Make Up for Threats to the NIH Budget

Avorn J - STAT

  • Commentary & Opinion
| October 2025
  • Innovation Incentives and Competition
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Phamaceutical industry funding cannot substitute for robust public investment in biomedical research, especially from the NIH. Industry support is often limited to product-driven, patentable research and can restrict open scientific communication, whereas publicly funded research enables broad dissemination of basic scientific research.
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Role of Health Workers and Representative Health Worker Organisations in LMIC Pharmaceutical Policy: A Scoping Review Protocol

Eisenkraft Klein D, Noor MN, Eiwanger L, Linton J, Ellis U, Sriram V - BMJ Open

  • Research Publications
| October 2025
  • Price, Value, and Access
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This scoping review protocol outlines a systematic assessment of how health workers and their organizations have shaped pharmaceutical policy in low- and middle-income countries (LMICs).
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Revenue Differences Between Top-Selling Small-Molecule Drugs and Biologics in Medicare

Vogel M, Feldman WB, Cowan Z, Rome BN, Chandra A, Kesselheim AS, Wouters OJ - JAMA Health Forum

  • Research Publications
| October 2025
  • Price, Value, and Access
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Among the top-selling small-molecule drugs and biologics eligible for Medicare drug price negotiation from 2012 to 2022, biologics generated substantially higher cumulative revenues than small-molecule drugs, totaling $4.9 billion more over 9 years and $7.9 billion more over 13 years. These findings suggest the longer exemption period for biologics from price negotiation is unecessary.
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Pharmaceutical Access in Brazil: Challenges and Opportunities

Ruas CM, Portela R, De Assis Acurcio F, Alvares-Teodoro J, Guerra Júnior AA, Kesselheim AS - Globalization and Health

  • Research Publications
| October 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
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In this review of pharmaceutical access in Brazil, the authors note that the Brazilian pharmaceutical market is highly concentrated, with low investment in R&D and heavy reliance on imported active ingredients. High drug prices and drug shortages also contribute to inequities in patient access. Prioritizing R&D investment, reducing dependency on international markets, and other reforms to drug pricing and patent standards can help alleviate these access challenges.
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Trends in Medicare Spending on Drugs for Pulmonary Arterial Hypertension, 2012-2022

Shih JA, Lee CC, Feldman WB - Chest

  • Research Publications
| October 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
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Annual estimated net Medicare spending on drugs to treat pulmonary arterial hypertension (PAH) more than doubled from $1.2 billion in 2012 to $2.8 billion in 2022, driven both by increased use and costs of the medications. By 2022, despite more generic availability, brand-name drugs still accounted for nearly 9 in every 10 dollars spent in Medicare on PAH medications.
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Regulatory and Clinical Outcomes of Nononcology Accelerated Approvals

Liu ITT, Reynolds G, Kesselheim AS, Cliff ERS - JAMA

  • Research Publications
| October 2025
  • Regulation and Clinical Evidence
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In an analysis of 50 non-oncology drugs granted FDA accelerated approval from 2013 to 2024, 38% were converted to full approval at a median 3 years later, but over half of these conversions relied on surrogate endpoints rather than direct clinical outcomes. The study raises concerns about the clinical utility of some accelerated approvals and calls for more robust confirmatory trials to ensure patient benefit.
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Brief of Professors of Law, Medicine, and Public Health in Vanda v. United States

Feldman WB, Kesselheim AS, Tu SS - US Court of Appeals for the Federal Circuit

  • Policy Interactions
| October 2025
  • Regulation and Clinical Evidence
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Vanda mischaracterizes Ruckelshaus v. Monsanto by treating it as establishing a per se taking whenever FDA uses or discloses trade secret information, when in fact Monsanto requires a statutory promise of secrecy, absent from the Food, Drug, & Cosmetic Act, for any such takings claim to succeed. The amici warn that accepting Vanda’s broad interpretation would obstruct generic drug competition under Hatch-Waxman and chill FDA’s ability to communicate essential public health information such as adverse event data, manufacturing inspection reports, clinical trial protocols, and drug shortage information.
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PORTAL Post | October 2025

  • PORTAL Post
| October 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
  • Regulation and Clinical Evidence
A graphic icon of a megaphone as a symbol for the PORTAL Post email newsletter.
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Isotretinoin Risk Evaluation and Mitigation Strategy and Pregnancy Incidence

Zhu Y, Anand P, Sarpatwari A, Hernández-Díaz S, Bykov K, Kesselheim AS, Avorn J, Vine SM, Adomi M, DiCesare EC, Mahesri M, Zakoul H, Mogun H, Toyserkani GA, LaCivita C, Zhou EH, Dal Pan GJ, Huybrechts KF - JAMA Internal Medicine

  • Research Publications
| October 2025
  • Regulation and Clinical Evidence
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Initiation of isotretinoin under the iPLEDGE risk evaluation and mitigation strategy (REMS) program was associated with a 70%–80% lower pregnancy incidence than topical retinoids across publicly and commercially insured patients, though differences persisted by insurance type and race/ethnicity. Despite these reductions, fetal exposre to isotretinoin still occurred, highlighting the need for strategies to improve adherence to REMS requirements.
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Changes in Medicare Part D Plan Designs After the Inflation Reduction Act

Cai CL, Bhaskar A, Kesselheim AS, Rome BN - JAMA Internal Medicine

  • Research Publications
| October 2025
  • Price, Value, and Access
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After the Inflation Reduction Act (IRA), Medicare Part D plans increased deductibles and relied more heavily on coinsurance for brand-name drugs. For the most common medications, average monthly out-of-pocket costs in Medicare Advantage plans ranged from $46 to $55 between 2019 and 2024 and then increased to $73 in 2025. In standalone plans, mean monthly costs increased from $62 to $108 from 2019 to 2025. While the IRA capped annual out-of-pocket spending at $2,000, these plan design changes may result in higher costs for beneficiaries who do not reach the cap, suggesting a need for further policy action.
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Assessing Outcomes Emerging After Conversion to Regular Approval for Cancer Drug Indications Granted Accelerated Approval, 1992-2021

Tibau Martorell A, Romano A, Liu ITT, Han J, Cliff ERS, Kesselheim AS - Journal of the National Cancer Institute

  • Research Publications
| October 2025
  • Regulation and Clinical Evidence
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Among all cancer drug indications granted accelerated approval by the FDA between 1992 and 2021 that were later converted to full approval, only 32% of confirmatory trials used to support full approval showed overall survival benefit and fewer than half (47%) met the ESMO-MCBS threshold for substantial benefit. These findings highlight the need for stronger approval standards for cancer therapies granted accelerated approval and the value of scales like ESMO-MCBS better guide treatment value for patients and clinicians.
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Whose Burden, Whose Benefit? Revisiting Ethical Trade-Offs in the WHO Guidelines on Scaling Up Mass Azithromycin Administration

Goh M, Viens AM, Karim SA, Kesselheim AS, Outterson K - PLOS Medicine

  • Research Publications
| September 2025
  • Price, Value, and Access
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The authors critique the World Health Organization’s (WHO) cautious approach to mass azithromycin administration (MDAA) to reduce childhood mortality in high-burden, low-resource settings due to concerns about antimicrobial resistance (AMR). This approach insufficiently addresses justice and equity considerations, and revised WHO guidelines should place greater emphasis on these ethical considerations, expanding eligibility for MDAA based on context-specific criteria and community input.
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Prices and Spending for Phosphodiesterase 4 Inhibitors in Pulmonary and Dermatologic Disease

Perry NJ, Barbieri JS, Rome BN - JAMA Dermatology

  • Research Publications
| September 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
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In this analysis of US spending on two phosphodiesterase 4 inhibitors, roflumilast and apremilast, the authors found that apremilast was priced 10-times higher than roflumilast, and this gap widened after roflumilast faced generic competition starting in 2022. From 2014 to 2024, apremilast spending totaled $16 billion, which could have been reduced by 84% ($13.5 billion) had roflumilast been substituted. These findings raise concerns about incentives for companies to develop new higher-cost medications rather than repurposing existing ones for new indications.
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Brief of Economists and Scholars of Health Policy in National Infusion Center Association v. Kennedy (5th Circuit)

Frank RG, Scott Morton FM, Kesselheim AS, Anderson GF, Conti RM, Cutler DM, Hoadley J - US Court of Appeals for the Fifth Circuit

  • Policy Interactions
| September 2025
  • Price, Value, and Access
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The amici express support for the Medicare drug price negotiation program and rebut assertions that the program will harm biopharmaceutical innovation. They argue that patents, exclusivity periods, and insurance coverage distort the drug market far from free-market conditions, that continued strong R&D investment and clinical trial activity since the Act’s passage undermine industry claims of innovation harm, and that the program’s structure mirrors fair negotiation processes long used by other federal purchasers.
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FDA Regulatory Pathways for Expedited Drug Development and Approval

Kesselheim AS - Clinical Advances in Hematology & Oncology

  • Commentary & Opinion
| September 2025
  • Regulation and Clinical Evidence
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While expedited FDA approval pathways can speed patient access to promising new cancer therapies, they also increase the risk of approving drugs with uncertain benefits or safety issues. To mitigate these risks, FDA should provide clearer communication about the nature of expedited approvals, rapidly update drug labels when new evidence emerges, and take action to ensure sponsors complete confirmatory trials in a timely manner.
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Modeling the Budgetary Impacts of Sickle Cell Disease Gene Therapies on State Medicaid Programs

Harvey JP, Raymakers AJN, Rand LZG, Goshua G, Kesselheim AS, Pandya A - Journal of General Internal Medicine

  • Research Publications
| September 2025
  • Price, Value, and Access
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New gene therapies for sickle cell disease could increase state Medicaid budgets by an estimated $837.5 million over five years, highliging the need for novel policy interventions and payment mechanisms, such as the CMS Cell and Gene Therapy Access Model, to control costs while maintaining patient access.
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Overlapping Method of Use Patents to Prevent Generic Entry

Park J, Kesselheim AS, Tu SS - Journal of Law, Medicine & Ethics

  • Commentary & Opinion
| Fall 2025
  • Innovation Incentives and Competition
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Brand-name pharmaceutical companies increasingly use overlapping patents and FDA use codes to delay the generic entry. This study uses two recent cases, icosapent ethyl (Vascepa) and sacubitril-valsartan (Entresto), to show how these tactics can obstruct the skinny labeling pathway for generic products. To restore balance between exclusivity and competition in the US pharmaceutical market, reforms to use codes and the legal standards for patent ingringement are necessary.
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Breakthrough Therapy Designations in China and the United States

Mao X, Li Y, Kesselheim AS, Ba Y, Li G - Nature Reviews Drug Discovery

  • Research Publications
| September 2025
  • Regulation and Clinical Evidence
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Since 2020, the Chinese NMPA received nearly twice as many breakthrough therapy designation (BTD) applications for drugs as the FDA, but granted a lower percentage than the US regulator. Most BTDs in China were for domestically developed drugs, and oncology led BTD approvals in both countries. As use of the BTD pathway continues, both countries should create clear, rigorous evaluation criteria to ensure the pathway prioritizes truly promising new therapies.
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Medicare Part D Savings Under the Manufacturer Discount Program vs Coverage Gap Discounts

Rome BN, Garrett KR, Maini L - JAMA Network Open

  • Research Publications
| September 2025
  • Price, Value, and Access
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When comparing the Coverage Gap Discount (CGD) and the Manufacturer Discount Program (MDP) in Medicare Part D, Medicare would have received $34 billion from drug makers under the MDP, compared to $17 billion under the CGD based on 2022 data. Discounts in the MDP also increased substantially for high-cost specialty drugs, highlighting how this policy change will affect Medicare Part D spending going forward.
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Bridging the Fair Share Gap for Antibacterial Innovation: an Observational Analysis of Antibacterial Revenues in the G7 and EU27

Goh M, McEnany M, Freeman R, Newton M, Kesselheim AS, Outterson K - eClinicalMedicine

  • Research Publications
| September 2025
  • Price, Value, and Access
  • Regulation and Clinical Evidence
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Among high-income countries, the G7 and EU countries fall short of revenue targets needed to sustain research for new antibiotic development, with the UK and Italy as notable exceptions. Revenue guarantee incentives could help bridge gaps in antibiotic investment in wealthier countries amid a growing threat of antimicrobial resistance.
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Physician Perspectives on Pharmaceutical Promotion

Mooney H, Austad KE, Campbell EG, Avorn J, Lu Z, Kesselheim AS - JAMA Health Forum

  • Research Publications
| September 2025
  • Regulation and Clinical Evidence
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Physicians surveyed in 2011 and 2024 increasingly recognized the value of the materials they receive from drug companies, but remained concerned about the impact on public trust in medicine. To address these concerns, professional societies, payers, and regulators should collaborate to create unbiased educational resources for clinicians on the efficacy and safety of drugs.
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Lowering Drug Prices in the US-Look Within, Not Abroad

Gondi S, Papanicolas I, Rome BN - JAMA

  • Commentary & Opinion
| September 2025
  • Price, Value, and Access
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The authors outline the limitations of benchmarking US drug prices to those abroad via international reference pricing, recommending instead that the US adopt transparent, evidence-based health technology assessment and negotiation processes to address high drug costs.
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Trends in Time to Withdrawal and Full Approval of Accelerated Approval Cancer Drug Indications (1992-2024)

Jenei K, Hahn G, Kesselheim AS, Tibau Martorell A - Journal of Cancer Policy

  • Research Publications
| September 2025
  • Regulation and Clinical Evidence
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The median time from FDA accelerated approval to full approval for cancer drugs decreased from 4.3 years in 1992 to 2.3 years in 2025, with a reduction in median withdrawal time from 9.5 to 3.2 years. The proportion of confirmatory studies underway at approval rose from 63% to 85% during this period. These findings may reflect improved regulatory oversight or more flexible approval, highlighting the need for timely, clinically meaningful confirmatory evidence to ensure real patient benefit and reduce uncertainty.
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Pediatric Exclusivity-Associated Revenues and Labeling Changes, 2013-2023

Liu ITT, Raymakers AJN, Sarpatwari A, Kesselheim AS - Journal of Pediatrics

  • Research Publications
| September 2025
  • Innovation Incentives and Competition
  • Regulation and Clinical Evidence
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The Best Pharmaceuticals for Children Act (BPCA) provides a 6-month exclusivity for manufacturers that conduct additional pediatric studies of their products. 97% of pediatric exclusivities granted over a 10-year period resulted in new pediatric efficacy data added to the drug label. However, this data was often generated a decade after market entry, generating a median excess revenue of $134 million per exclusivity, suggesting optimization of BPCA requirements may be necessary to facilitate timely evidence generation.
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National Analysis of the Requirements and Implementation of State Prescription Drug Price Transparency Laws

Rahim H, Kesselheim AS - Milbank Quarterly

  • Research Publications
| September 2025
  • Price, Value, and Access
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In this review of the design and implementation of prescription drug price transparency laws across 21 US states, the authors find that while these laws have improved public access to pricing data and informed state-level policymaking, there is limited evidence that such policies have curbed drug prices or altered manufacturer behavior. Stronger enforcement mechanisms and data quality standards may enhance the impact of these transparency efforts.
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Fireproofing the FDA: Power, Politics, and Public Health

Daval CJR, Deshmukh A, Sinha M - University of Utah Quinney College of Law

  • Presentations & Panels
| September 2025
  • Regulation and Clinical Evidence
A graphic of a person sharing research while giving a presentation.
Joseph Daval, Anjali Deshmukh, and Michael Sinha joined other FDA scholars and journalists for a University of Utah Quinney College of Law symposium on how to rebuild and improve the FDA amid a period of political interference and upheaval.
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The Advisory Committee on Immunization Practices-Legal Roles, Challenges, and Guardrails

Daval CJR, Kesselheim AS - JAMA

  • Commentary & Opinion
| August 2025
  • Price, Value, and Access
  • Regulation and Clinical Evidence
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The authors discuss the legal foundations of the CDC’s Advisory Committee on Immunization Practices (ACIP), a body that makes vaccine recommendations that have been statutorily embedded in numerous federal programs. They discuss the legality of disbanding ACIP, removing committee members, and the HHS Secretary to override ACIP decisions, highlighting the need for Congressional action to protect the committee’s independence and maintain a mechanism for evidence-based vaccine policy deliberation.
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Advance Market Commitments and Their Role in Public Innovation

Nagar SN, Cacodcar A, Kesselheim AS - Journal of Law, Medicine & Ethics

  • Commentary & Opinion
| Summer 2025
  • Innovation Incentives and Competition
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Advance market commitments (AMCs) have potential as an alternative funding mechanism for drug development, as seen with the Gavi pneumococcal vaccine initiative and Operation Warp Speed for COVID-19 vaccines. However, AMCs’ effectiveness is maximized when combined with robust public research funding and complementary policy tools.
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Health Professional Students’ Knowledge About FDA Drug and Medical Device Regulation

Sangha KK, Redberg RF, Kesselheim AS, Woloshin S, Dhruva SS - Academic Medicine

  • Research Publications
| August 2025
  • Regulation and Clinical Evidence
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A survey of final-year medical, pharmacy, and nurse practitioner students at the University of California, San Francisco identified significant gaps in students’ understanding of FDA drug and device regulation, as well as the quality of clinical trial evidence. Most students reported only basic or cursory teaching on these topics and struggled with key concepts, highlighting the need for stronger education on regulatory processes to better prepare future clinicians.
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Estimating Costs of Market Exclusivity Extensions for 4 Top-Selling Prescription Drugs in the US

Hong D, Tu SS, Beall RF, Russo M, Rome BN, Kesselheim AS, Sarpatwari A - JAMA Health Forum

  • Research Publications
| August 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
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For four top-selling drugs with US market exclusivity beyond key patent expirations, this extended exclusivity was estimated to delay generic competition by 7 to 13 months, resulting in an estimated $3.5 billion in excess US spending on these products over two years in the absence of the exclusivity extension. These findings underscore how timely generic entry could yield substantial savings and the need for policy reforms to limit exclusivity extensions for top-selling products.
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Drug Versioning and Legal Accountability (Reply)

Tu SS, Bonis T - JAMA

  • Commentary & Opinion
| August 2025
  • Regulation and Clinical Evidence
Talk bubble graphics representing commentary and opinion.
In response to comments on their prior analysis, the authors clarify that legal accountability for delayed drug releases should apply only when companies knowingly withhold safer alternatives, arguing that such liability is necessary to protect public health and prevent such practices from becoming industry standard.
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Benefits and Limitations of Direct-To-Consumer Pharmacies in the US

Lalani HS, Kesselheim AS, Rome BN - JAMA Network Open

  • Commentary & Opinion
| August 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
Talk bubble graphics representing commentary and opinion.
Commenting on an analysis of the prices of generic drugs for neurologic conditions available via a direct-to-consumer pharmacy, the authors emphasize that while direct-to-consumer pharmacies can offer lower prices for some generic drugs compared to traditional insurance, broader reforms are needed to improve drug affordability and access in the US.
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Combating Pharmaceutical Patent Thickets in the Trump Administration

Tu SS, Kesselheim AS, Feldman WB - Health Affairs Forefront

  • Commentary & Opinion
| August 2025
  • Innovation Incentives and Competition
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The authors provide an overview of how pharmaceutical companies use patent thickets to delay generic and biosimilar competition and highlight bipartisan federal legislation like the ETHIC Act that could achieving meaningful patent reform.
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The Limits of Physician-Level Interventions for Cost-Related Nonadherence: A Case for Structural Reform

Eisenkraft Klein D, Kesselheim AS, Rand LZG - American Journal of Bioethics

  • Commentary & Opinion
| August 2025
  • Price, Value, and Access
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Commenting on an article discussing clinicians’ responsibility to mitigate cost-related nonadherence, the authors contend that while physicians do have a role in helping patients navigate high drug costs, lasting solutions require systemic reforms in pharmaceutical pricing, regulation, and patent law, rather than relying solely on individual clinicians.
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The Puzzle of Biologics Manufacturing Platform Patents (Reply)

Jefferson OA, Price WN, Tu SS, Vishnubhakat S, Rai AK - Nature Biotechnology

  • Commentary & Opinion
| August 2025
  • Innovation Incentives and Competition
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The authors respond to Amgen’s critique of the authors’ prior analysis of biologic platforms patents, reemphasizing how post-approval manufacturing patents and opaque litigation practices can delay biosimilar entry even after regulatory approval.
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Health Technology Assessment, Again: A Transparent, Evidence-Based Approach for CMS Drug Price Negotiations

Ollendorf DA, Rand LZG, Blonda A, Mooney H, Phillips M, Basu A - Health Affairs

  • Research Publications
| August 2025
  • Price, Value, and Access
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CMS should adopt a formal health technology assessment (HTA) framework for Medicare drug price negotiations that integrates comparative value assessment, multicriteria decision analysis, and independent advisory committee deliberation to ensure fair pricing and public trust in the negotiation process.
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Expansion of 340B Disproportionate Share Hospitals in the United States From 2010 to 2022

Liu BY, Russo M, Kesselheim AS, Knox RP, Sarpatwari A, Feldman WB - Health Services Research

  • Research Publications
| August 2025
  • Price, Value, and Access
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Between 2010 and 2022, new child sites and contract pharmacies associated with 340B disproportionate share hospitals were increasingly established in wealthier areas, with the Social Deprivation Index (SDI) for these sites decreasing from a median of 59 and 72, respectively, in 2010 to 53 and 53 by 2022. This shift towards serving more affluent communities raises questions about the alignment of the 340B program’s expansion with its original mission.
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Differences Between Trial Populations and Approved Label Populations of New Drugs in the United States and Europe (2012 to 2023): A Cross-Sectional Study

Vokinger KN, Serra-Burriel M, Glaus CEG, Welti L, Ross JS, Kesselheim AS - Annals of Internal Medicine

  • Research Publications
| August 2025
  • Regulation and Clinical Evidence
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In a review of 263 drugs approved in the US, EU, and Switzerland from 2012 to 2023, regulatory agencies were found to often approve drugs for use in populations broader than those studied in pivotal trials, particularly in the US. Labels were most commonly broadened by “patient fitness” (e.g., health status criteria like comorbidities, lab tests, and performance scores), meaning that drugs were often approved for patients who would not have qualified for the trials that supported their approval. These findings underscore the need for post-approval studies to ensure safety and efficacy in patient populations excluded from trials.
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Community Pharmacy Turnover and Context of Openings and Closings By Ownership Type

Mattingly II TJ, Sahu M, Anderson KE - JAMA Health Forum

  • Research Publications
| August 2025
  • Price, Value, and Access
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In this analyis of the rate of community pharmacy openings and closures in the US, market turnover was 86.8% between 2010 and 2023, corresponding to an annual turnover rate of 6.2%. Independent pharmacies opened and closed more frequently than chain pharmacies. These market shifts reflect the further need for research into the patient impact of pharmacy closures in the US.
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Insurer Exits After the Inflation Reduction Act Part D Redesign

Cai CL, Kesselheim AS, Bhaskar A, Rome BN - JAMA

  • Research Publications
| July 2025
  • Price, Value, and Access
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Following changes in Medicare Part D plan design under the Inflation Reduction Act, 7.5% of beneficiaries (2.9 million people) lost their Part D insurer in 2024, a sharp rise from prior years, with higher insurer exit rates among standalone and enhanced plans. These findings suggest a need for additional policies to ensure adequate competition and affordability in the Part D marketplace.
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Prescription Drug Method-Of-Use Patent Protection, 1991-2018

Bloomfield D, Teng TW, Kesselheim AS, Tu SS - Journal of General Internal Medicine

  • Research Publications
| July 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
  • Regulation and Clinical Evidence
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Method-of-use patents have increasingly extended brand-name drugs’ market exclusivity in the US, with the median additional protection rising from 3.2 years in 1991 to 5.4 years in 2018. This shift has resulted in a total median patent protection of nearly 15 years after FDA approval for many products, emphasizing the need for the preservation of the “skinny-labeling” pathway and other strategies to enable timely generic competition.
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Share of Sales Subject to Medicare Inflation Rebates and Price Increases of Top-Selling Drugs

Egilman AC, Kesselheim AS, Rome BN - Health Services Research

  • Research Publications
| July 2025
  • Price, Value, and Access
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Top-selling drugs that were the most affected by Medicare inflationary rebates in 2023 due to a higher share of Medicare sales did not have larger year-over-year price increases than drugs less affected by the rebates. This suggests that although the rebates will save money for Medicare, there may not be spillover effects of lower prices for other payers; additional measures, such as extending inflation rebates to commercial insurance, may be needed to prevent manufacturers from raising prices on existing brand-name drugs.
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Revenue, Patents, and New Generic Competition for Prescription Drugs in the USA

Tu SS, Kesselheim AS, Ross JS, Gupta R - Journal of General Internal Medicine

  • Research Publications
| July 2025
  • Innovation Incentives and Competition
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Among 178 brand-name drugs approved in the US between 2000 and 2012, those with higher revenue were more likely to face generic competition, while those with more patents had fewer generic competitors. Specifically, every $100 million increase in average brand-name revenue was associated with an 8% increase in the likelihood of generic approval, while each additional patent reduced that likelihood by 7%.
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Impostor Syndrome: Fraudulent Participants in Qualitative Research Can Skew Results

Rand LZG, McGraw SA, Wang J, Darrow JJ, Kesselheim AS - Journal of Law, Medicine & Ethics

  • Commentary & Opinion
| Summer 2025
  • Regulation and Clinical Evidence
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The authors discuss how fraudulent participants in qualitative health research can meaningfully distort study findings, emphasizing the need for robust screening and reporting strategies to protect data validity.
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Trump’s Ongoing Tariff Chaos Will Make Medications Less Affordable and Harder to Find

Kesselheim AS, Avorn J - Health Affairs Forefront

  • Commentary & Opinion
| July 2025
  • Price, Value, and Access
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Proposed tariffs on imported pharmaceuticals will likely raise medication costs and worsen drug shortages, while ignoring the root causes of high drug prices in the US and risking harms to patient access.
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The Strength and Importance of Government-Funded Patents for Approved Drugs

Gabriele SME, Martin MJ, Kesselheim AS, Tu SS - Nature Biotechnology

  • Research Publications
| July 2025
  • Innovation Incentives and Competition
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In this examination of the role of US federal funding in late-stage drug development, 2.5% of small molecule drug patents listed in the FDA Orange Book were found to cite government funding. However, these government-funded patents were more likely to be primary patents and to receive patent term extensions, indicating their strength and value. Recognizing the public’s investment in drug discovery should both inform efforts to address high drug prices and reemphasize the importance of robust federal funding for biomedical research.
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Frequency and Nature of Generic “Design Around” of Brand‐name Patents in the United States

Freilich J, Kesselheim AS - Clinical Pharmacology & Therapeutics

  • Research Publications
| July 2025
  • Innovation Incentives and Competition
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In 153 patent litigation cases regarding generic manufactuers’ efforts to “design around” brand-name drug patents, 82% of cases involved changes to the physical product or packaging, with 48% altering the drug’s formulation, 12% changing the active ingredient, and 10% modifying the manufacturing process. These design-around strategies can help lower drug prices and increase market competition.
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Evolving Recommendations for Patient Populations Among Oncology Medicines: A Quantitative and Qualitative Analysis

Hogervorst MA, Vreman RA, Oduol TA, Mantel‐Teeuwisse AK, Goettsch WG, Kesselheim AS - Clinical Pharmacology & Therapeutics

  • Research Publications
| July 2025
  • Regulation and Clinical Evidence
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Across five countries they find that approved patient populations for oncology drugs are adjusted an average of 10 times throughout a drug’s market lifecycle. Drug developers made the most adjustments, often expanding the patient population, while regulators and HTA organizations often imposed restrictions. How patient populations were described was also inconsistent, highlighting the need for a standardized patient population reporting framework.
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Design Patents: A Potential Threat to Drug Competition

Freilich J, Kesselheim AS - Applied Health Economics and Health Policy

  • Research Publications
| July 2025
  • Innovation Incentives and Competition
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Drug design patents, which protect the ornamental appearance of drugs and delivery devices, are increasingly prevalent, are often easier and quicker to obtain than utility patents, and may complicate generic and biosimilar competition. To mitigate these impacts, USPTO should avoid granting design patents for drugs and the FDA should prohibit listing such patents in the Orange Book.
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Data Manipulation Within the US Federal Government

Freilich J, Kesselheim AS - Lancet

  • Research Publications
| July 2025
  • Regulation and Clinical Evidence
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The authors document the widespread, unlogged changes to federal health datasets, most notably the replacement of “gender” with “sex,” and raise concerns about data integrity, transparency, and the reliability of the research and policy decision-making that depend on these resources.
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Examining Pricing and Availability for Neglected Tropical Disease Therapies in the United States

Goh M, Outterson K, Kesselheim AS - Open Forum Infectious Diseases

  • Research Publications
| June 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
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Many essential therapies for neglected tropical diseases (NTDs) remain expensive and vulnerable to shortage in the US, in part due to few manufacturers and supply chain fragility. Policy interventions such as public manufacturing and supply chain resilience efforts may help improve access to affordable, reliable treatment for these diseases
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Congress Should Remove the Rare Disease Carve-Out From Medicare Drug Price Negotiation, Not Expand It

Mooney H, Kesselheim AS, Rome BN - Health Affairs Forefront

  • Commentary & Opinion
| June 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
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An expansion of the orphan drug exemption in Medicare drug price negotiation would shield high-revenue drugs from negotiation, reduce savings, and create new perverse incentives for drug manufacturers to avoid negotiation eligibility. The rare disease exemption should instead be removed to enable greater savings and improve affordability of rare disease drugs for Medicare patients.
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Disappearing Data at the U.S. Federal Government

Freilich J, Price WN, Kesselheim AS - New England Journal of Medicine

  • Research Publications
| June 2025
  • Regulation and Clinical Evidence
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The authors discuss the alarming trend of disappearing health datasets from U.S. federal health agencies. Between January 21 and February 11, 2025, the CDC alone removed 203 datasets, undermining scientific research, public health efforts, and policy-making. As government datasets disappear, stakeholders are forced to turn to private sources, increasing costs and potentially reducing research quality. Action is needed to ensure the government continues to collect and publish high-quality data.
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Comments on Initial Guidance for the Medicare Drug Price Negotiation Program, IPAY 2028

Kim J, Kesselheim AS, Rome BN - Centers for Medicare and Medicaid Services (CMS)

  • Policy Interactions
| June 2025
  • Price, Value, and Access
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The commenters support CMS’s proposed clarification that fixed-combination drugs containing therapeutically inactive ingredients (such as hyaluronidase used for subcutaneous delivery) should be aggregated with the original formulations for price negotiation purposes, preventing manufacturers from evading negotiation through strategic reformulations. They also recommend that CMS close a related loophole by aggregating spending only across formulations without generic or biosimilar competition when selecting drugs for negotiation.
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What Can We Learn From Patient Input at FDA Advisory Committee Meetings?

Rand LZG, Eisenkraft Klein D - Journal of Law, Medicine & Ethics

  • Commentary & Opinion
| Summer 2025
  • Regulation and Clinical Evidence
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Commenting on an analysis of FDA advisory committee decisions, the authors explore the complex role of patient testimony in regulatory deliberations. While patient input can offer valuable insights into lived experience and unmet needs, tensions exist between experiential narratives and evidence-based decision-making, especially in data-limited contexts. Clearer frameworks are needed to ensure that patient engagement enhances transparency and legitimacy without compromising scientific rigor.
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Medicare Spending and Use of Subcutaneous Biologic Formulations with Hyaluronidase

Kim J, Kesselheim AS, Cliff ERS, Rome BN - The Oncologist

  • Research Publications
| June 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
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Medicare spending on biologic drugs reformulated with hyaluronidase for subcutaneous administration totaled $10.3 billion in 2022. While these formulations may offer clinical advantages—such as fewer infusion reactions and reduced delivery time for patients—the timing of their introduction raises concerns about “hyaluronidase hopping” as a strategy to delay biosimilar competition and Medicare drug price negotiation. Policy safeguards are needed to ensure that such reformulations can still provide clinical value without undermining cost-containment efforts.
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Use and Cost of First-Line Biologic Medications to Treat Plaque Psoriasis in the US

Rome BN, Han J, Mooney H, Kesselheim AS - JAMA Dermatology

  • Research Publications
| June 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
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The average annual treatment cost for first-line biologic treatments for plaque psoriasis in the US more than doubled from 2007 to 2021. Costs in 2021 would have been 44% lower if patients had initiated the lowest-cost medication within each class, highlighting the potential savings from using lower-cost psoriasis therapies and better aligning prices with a drug’s clinical benefits.
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Predictors of Withdrawal of Anticancer Drug Indications Granted Accelerated Approval: a Retrospective Cohort Study

Tibau Martorell A, Cliff ERS, Romano A, Borrell M, Molto C, Kesselheim AS - eClinicalMedicine

  • Research Publications
| June 2025
  • Regulation and Clinical Evidence
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Among 167 cancer indications granted FDA accelerated approval from 1992 to 2022, 19% were withdrawn and 61% were converted to regular approval, with drugs demonstrating low clinical benefit as measured using the ESMO-MCBS scale more likely to be withdrawn. Drugs with breakthrough therapy designations or genome-targeted mechanisms had lower withdrawal rates. These findings underscore the value of benefit assessment frameworks to inform regulatory and clinical decision-making.
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Changes in Oncology Medication Use After Withdrawal of Accelerated Approval

Hwang CS, Kesselheim AS, Kelkar AH, Cliff ERS, Rome BN - JAMA Oncology

  • Research Publications
| June 2025
  • Regulation and Clinical Evidence
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For four oncology drugs granted accelerated approval, the release of negative confirmatory trial results prompted a decrease in use, and few patients were initiating treatment by the time of the drug’s indication was withdrawn. However, the time between trial results publication and regulatory withdrawal should be shortened to minimize patient exposure to ineffective treatments.
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Association Between Industry Payments and Prescription of Inhaled Medications

Nadeem H, Donovan LM, Feemster LC, Au DH, Feldman WB, Duan KI - American Journal of Respiratory and Critical Care Medicine

  • Research Publications
| June 2025
  • Price, Value, and Access
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Inhaler prescribers who received payments from inhaler manufacturers in 2018 were found to be more likely to prescribe inhalers from the same manufacturer in 2019, with larger payments associated with increased prescribing.
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Op-Ed: The Trouble with ‘Do Your Own Research’ for Drugs

Avorn J - MedPage Today

  • Commentary & Opinion
| May 2025
  • Price, Value, and Access
  • Regulation and Clinical Evidence
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The author critiques the notion that individual patients or physicians can independently evaluate drug efficacy, highlighting the complexity of clinical trial data and a lack of public access to the full evidence manufacturers submit for regulatory review. Bypassing FDA oversight risks exposing patients to ineffective or harmful treatments, while raising questions about who would be expected to pay for such therapies.
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Brief of 30 Scholars of Law, Economics, and Medicine in Hikma v. Amarin (Cert Petition)

Feldman WB, Kesselheim AS, Rome BN, Tu SS - US Supreme Court

  • Policy Interactions
| May 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
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Filed in support of Hikma’s cert petition, the amici claim that the Federal Circuit erred in holding that a generic manufacturer could face induced infringement liability based on truthful product equivalence statements, contending that this decision conflicts with the common law foundations of inducement doctrine and threatens to extend patent liability indefinitely through successive new-use patents. The ruling’s reach extends well beyond pharmaceuticals to any industry relying on equivalence or compatibility claims, and impeding such statements would stifle free-market competition and harm consumers by sowing confusion about equivalent products.
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The Comparative Effectiveness and Safety of Fluticasone-Salmeterol Via Metered-Dose Versus Dry Powder Inhalers for COPD: A New User Cohort Study

Demkowicz BJ, Rader K, Wang SV, Kesselheim AS, Feldman WB - PLOS Medicine

  • Research Publications
| May 2025
  • Regulation and Clinical Evidence
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The authors compared the effectiveness and safety of fluticasone-salmeterol delivered via metered-dose versus dry powder inhalers among patients with COPD and found similar outcomes in the two treatment groups. Given that metered-dose inhalers are associated with 20x the greenhosue gas emissions of dry powder versions, these findings highlight the value of efforts to transition to dry powder inhalers.
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Testimony: Improving Residency Selection and Addressing Threats to Science and the US Health Care System

Feldman WB - 119th Congress, House Committee on the Judiciary, Subcommittee on the Administrative State, Regulatory Reform, and Antitrust

  • Policy Interactions
| May 2025
  • Innovation Incentives and Competition
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Feldman argues that eliminating the residency match’s antitrust exemption would not solve physician shortages or low resident wages, and instead recommends increasing CMS funding for residency slots, streamlining licensing for international medical graduates, and supporting resident unionization. More broadly, he discusses threats to US medicine from the Trump administration’s proposed 37% NIH cut and 50% NSF cut, FDA layoffs that have already delayed drug reviews, and Medicaid cuts that would leave over 8 million uninsured. Read his written testimony.
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Costs of Extending the Small Molecule Exemption Period in Medicare Drug Price Negotiation

Cai CL, Kesselheim AS, Rome BN - Health Affairs Forefront

  • Research Publications
| May 2025
  • Price, Value, and Access
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The authors evaluate the impact of extending the waiting period for Medicare drug price negotiation from 9 to 13 years for small molecule drugs, finding that delaying negotiation eligibility could increase Medicare spending by billions of dollars annually, undermining cost-saving goals and delaying access to lower prices for key medications.
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Drug Versioning and Legal Accountability for Preventable Product Harms

Tu SS, Bonis T - JAMA

  • Commentary & Opinion
| May 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
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The authors highlight a new form of “product hopping” called “drug versioning,” where pharmaceutical companies delay releasing improved drug versions to maximize profits on existing ones, using the alleged postponed launch of tenofovir alafenamide fumarate for HIV as a case study. This legal challenge highlights debates over corporate responsibility, and holding companies accountable for such behavior could deter unethical practices and ensure timely access to safer medications.
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Strategies to Advance Drug Repurposing for Rare Diseases

Hong D, Kesselheim AS - JAMA Network Open

  • Commentary & Opinion
| May 2025
  • Innovation Incentives and Competition
  • Regulation and Clinical Evidence
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The authors comment on a discussion of the role of rare disease nonprofit organizations in drug repurposing. Policy reforms—such as increased public funding for repurposing studies, expanded FDA authority to update drug labels based on nonprofit-submitted data, and better use of real-world evidence—are essential to ensure that repurposed rare disease therapies are effective and affordable.
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Brief of Economists and Scholars of Health Policy in Teva v. Kennedy

Frank RG, Scott Morton FM, Kesselheim AS, Anderson GF, Conti RM, Cutler DM, Hoadley J - US District Court for the District of Columbia

  • Policy Interactions
| May 2025
  • Price, Value, and Access
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The amici express support the Medicare drug price negotiation program and specifically defend CMS’s practice of aggregating products sharing the same active ingredient (including Austedo and Austedo XR) as a statutorily mandated response to “product hopping.” They argue that Teva’s proposed interpretation would create a systemic loophole enabling manufacturers to endlessly reset the negotiation clock through minor reformulation. They also reiterate that the negotiation program restores bargaining equity in Medicare, mirrors other federal drug pricing mechanisms, and poses minimal risk to genuine innovation given that targeted drugs have already generated billions in surplus revenue.
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Sotorasib’s Accelerated Approval: Wrong Dose and Indication

Ratain MJ, Kesselheim AS - JAMA Oncology

  • Commentary & Opinion
| May 2025
  • Regulation and Clinical Evidence
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The atuhors critique the FDA’s accelerated approval of sotorasib for non-small cell lung cancer, highlighting issues with the approved dose and indication. Confirmatory trials revealed no clear benefit of the higher dose over a lower dose, and reforms should be made to ensure drug labels reflect accurate dosing information. CMS should also consider covering lower doses if clinical trials show them to be equally effective.
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Opioid Settlements in Canada: Avoiding U.S. Pitfalls, Embracing Best Practices

Eisenkraft Klein D, Minhee C - International Journal on Drug Policy

  • Commentary & Opinion
| May 2025
  • Price, Value, and Access
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Drawing on the early US opioid settlement experience, the authors argue that Canada’s opioid settlement funds risk dilution without binding safeguards. They recommend mandating dedicated remediation spending, public reporting with outcome metrics, investment in a broad range of harm‑reduction services, and community‑led allocation to ensure settlements meaningfully reduce overdose harms rather than replicate US missteps.
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Certification Marks for Supporting Generic Drug Quality

Lindgren L, Kesselheim AS, Tu SS - Journal of Law, Medicine & Ethics

  • Commentary & Opinion
| Spring 2025
  • Innovation Incentives and Competition
  • Regulation and Clinical Evidence
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Certification marks, similar to USDA Organic or Energy Star labels, have the potential to improve the perception and oversight of generic drug quality. Independent certification marks could be a cost-effective way to effectively communicate drug quality information to purchasers, incentivize manufacturers to exceed baseline quality standards, and improve consumer confidence in the generic drug supply.
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Brief of Economists and Scholars of Health Policy in National Infusion Center Association v. Kennedy

Frank RG, Scott Morton FM, Kesselheim AS, Anderson GF, Conti RM, Cutler DM, Hoadley J - US District Court for the Western District of Texas

  • Policy Interactions
| April 2025
  • Price, Value, and Access
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The amici express support for the Medicare drug price negotiation program and rebut assertions that the program will harm biopharmaceutical innovation. They argue that patents, exclusivity periods, and insurance coverage distort the drug market far from free-market conditions, that continued strong R&D investment and oncology trial activity since the Act’s passage undermine industry claims of innovation harm, and that the program’s structure mirrors fair negotiation processes long used by other federal purchasers.
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Op-Ed: It’s Not Hard to See Through RFK Jr.’s ‘Radical Transparency’ Pledge

Lurie P, Kesselheim AS, Sharfstein JM - Washington Post

  • Commentary & Opinion
| April 2025
  • Regulation and Clinical Evidence
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The authors discuss Department of Health and Human Services (HHS) Secretary Robert F. Kennedy, Jr.’s claim that he would bring “radical transparency” to the Department, and how recent actions at the FDA, NIH, CDC, and other HHS agencies—including major staff reductions, communications restrictions, limiting public comment opportunities, and disbanding advisory committees—suggest a much more agenda-driven reality.
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Testimony: NIH Funding, Drug Price Negotiation, and Biosimilars: Three Factors Essential for Patients and Pharmaceutical Innovation

Kesselheim AS - 119th Congress, House Committee on Ways and Means, Subcommittee on Health

  • Policy Interactions
| April 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
An icon of a bill representing government policy.
Kesselheim identifies three pillars essential to pharmaceutical innovation and patient access: restoring NIH research funding paused by the Trump administration, preserving and extending Medicare drug price negotiation, and fostering timely biosimilar competition for high-cost biologics. He rebuts constitutional and innovation-based challenges to the Inflation Reduct Act, noting that the first 10 negotiated drugs saw 38-79% price reductions while still generating substantial manufacturer revenue. Read his written testimony.
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Patent Thickets and Product Hops: Challenges and Opportunities for Legislative Reform

Feldman WB - Journal of Law, Medicine & Ethics

  • Commentary & Opinion
| Spring 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
Talk bubble graphics representing commentary and opinion.
The author provides an overview of how brand-name drug manufacturers use patent thickets and product hops to delay generic competition, and discusses policy reforms that may mitigate these tactics and improve competition.
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Engaging Pharmacists in Strategies for Navigating Prescription Drug Costs (Reply)

Lalani HS, Hwang CS, Rome BN - JAMA

  • Commentary & Opinion
| April 2025
  • Price, Value, and Access
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The authors discuss the root causes of high drug prices and high out-of-pocket costs for patients and emphasize how patients and clinicians can use the seven strategies outlined in their prior research to navigate high prescription drug costs.
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Therapeutic Benefit of Top-Selling Oncology Drugs in Medicare

Wang S, Egilman AC, Hahn G, Kesselheim AS - JAMA Network Open

  • Research Publications
| April 2025
  • Price, Value, and Access
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Of the 50 top-selling oncology drugs in Medicare in 2022, four-fifths were determined by French and German health technology assessment (HTA) bodies to provide high added benefit over alternatives. However, drugs found to provide low or no added benefit still accounted for $6.7 billion in post-rebate Medicare spending. These findings emphasize the role of value assessment in Medicare drug price negotiations and underscore the need for a national US HTA agency to facilitate cost-effective health care spending.
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Will I Feel Better? Raising the Bar for Quality of Life in Oncology

Tibau Martorell A, Romano A, Kesselheim AS - Nature Reviews Clinical Oncology

  • Research Publications
| April 2025
  • Regulation and Clinical Evidence
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Applying the ESMO-Magnitude of Clinical Benefit Scale (ESMO-MCBS) quality-of-life (QoL) checklist to oncology indications approved by the FDA and EMA between 2020 and 2023, nearly a quarter of drugs showed some QoL improvement, but only 13% met checklist requirements, highlighting gaps in QoL outcomes in pivotal trials.
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Glucose-Lowering Medications and Risk of Chronic Obstructive Pulmonary Disease Exacerbations in Patients with Type 2 Diabetes

Ray A, Paik JM, Wexler DJ, Sreedhara SK, Bykov K, Feldman WB, Patorno E - JAMA Internal Medicine

  • Research Publications
| April 2025
  • Regulation and Clinical Evidence
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In a comparative effectiveness analysis of diabetes medications among nearly 400,000 patients with COPD, GLP-1 receptor agonists and SGLT-2 inhibitors were both associated with lower risks of moderate or severe COPD exacerbations compared to DPP-4 inhibitors.
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Frequency of First Generic Drugs Approved Through “Skinny Labeling,” 2021 to 2023

Ziaks TJ, Akanegbu CM, Egilman AC, Kesselheim AS - Journal of Managed Care & Specialty Pharmacy

  • Research Publications
| April 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
  • Regulation and Clinical Evidence
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The proportion of first generic drugs approved through “skinny labeling” decreased annually from 56% in 2021 to 20% in 2023, potentially due to increased legal risks following the 2021 decision in GSK v. Teva. Higher-selling brand-name drugs more frequently faced competition from skinny label generics, emphasizing the pathway’s importance in promoting cost-reducing generic competition for patients and payors.
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Comparative Bleeding Risk in Older Patients with HIV and Atrial Fibrillation Receiving Oral Anticoagulants

Quinlan CM, Avorn J, Kesselheim AS, Singer DE, Zhang Y, Cervone A, Lin KJ - JAMA Internal Medicine

  • Research Publications
| April 2025
  • Regulation and Clinical Evidence
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Among older adults with HIV and atrial fibrillation, warfarin and rivaroxaban were associated with higher rates of major bleeding versus apixaban, particularly among patients treated with antiretroviral therapy. These findings suggest apixaban may have a more favorable safety profile for this high-risk population.
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Factors in Time to Full Approval or Withdrawal for Anticancer Medicines Granted Accelerated Approval By the FDA

Tibau Martorell A, Hwang TJ, Romano A, Borrell M, Gich I, Molto C, Kesselheim AS - JAMA Network Open

  • Research Publications
| March 2025
  • Regulation and Clinical Evidence
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Priority review designation, absence of boxed warnings, initiation of confirmatory trials before approval, and ratings of intermediate or high clinical benefit were associated with shorter conversion times from FDA accelerated approval to full approval among 102 cancer drug indications. These findings emphasize the importance of rigorous efficacy and safety evaluations and timely trial initiation to support the benefits of accelerated approval drugs.
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Brief of 12 Narcolepsy Patients, Public Interest Organizations, Medical Professionals, and Professors of Law and Medicine in Jazz v. Avadel

Kesselheim AS, Tu SS - US Court of Appeals for the District of Columbia

  • Policy Interactions
| March 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
  • Regulation and Clinical Evidence
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The amici argue that Avadel’s once-nightly sodium oxybate product Lumryz offers substantial real-world benefits over Jazz’s twice-nightly Xyrem and Xywav products. Additionally, they contend that the Orphan Drug Act’s “same drug” phrase should be interpreted, consistent with FDA’s clinical superiority exception, to exclude clinically superior products from an incumbent’s exclusivity, because overly broad exclusivity scope harms competition and ultimately deprives patients of innovative treatments.
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Trends in Fills, Spending, and Prices of Doxepin for Insomnia

Cai CL, Kesselheim AS, Rome BN - JAMA

  • Research Publications
| March 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
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Doxepin was originally developed in the 1960s as an antidepressant and has been off patent for decades, but low doses of doxepin have been found to be effective as a first line treatment for insomnia. The authors detail how this repurposing has sustained high prices despite the availability of generics. Liquid doxepin should be considered as a potentially cheaper alternative, alongside broader reforms to address market failures in generic drug repurposing.
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Active Postmarketing Device Surveillance as a Legislative Priority

Feldman WB, Kesselheim AS - BMJ

  • Commentary & Opinion
| March 2025
  • Regulation and Clinical Evidence
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Passive safety surveillance of medical devices, relying on stakeholder reporting of problems, is fraught with limitations, whereas active safety surveillance, in which the FDA conducts routine post-marketing studies using real-world evidence, can provide a more complete picture of potential safety concerns. The authors make a case for better tracking of devices in medical claims and more funding for the FDA to conduct postmarketing surveillance studies.
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Comments on Draft Guidance, “Accelerated Approval and Considerations for Determining Whether a Confirmatory Trial Is Underway”

Tibau Martorell A, Martin MJ, Kesselheim AS - Food and Drug Administration (FDA)

  • Policy Interactions
| March 2025
  • Regulation and Clinical Evidence
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The commenters support the FDA’s efforts to prevent delays in confirmatory trials for drugs granted accelerated approval and recommend that the agency require expedited timelines for drugs with serious safety signals, strengthen transparency around trial progress, and apply lessons from research showing that boxed warnings and early trial initiation are key factors in timely conversion to full approval.
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Real-World Evidence Is a Vital Tool for Informing Treatment Strategies in Chronic Obstructive Pulmonary Disease

Feldman WB, Wang SV, Kesselheim AS - BMJ

  • Commentary & Opinion
| March 2025
  • Regulation and Clinical Evidence
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Real-world evidence, including longitudinal healthcare database studies, can provide valuable insights on clinical questions in COPD, including through the investigation of intraclass differences between COPD therapies and other subtle clinical differences that may impact routine clinical care.
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The Puzzle of Biologics Manufacturing Platform Patents

Jefferson OA, Price WN, Tu SS, Vishnubhakat S, Rai AK - Nature Biotechnology

  • Research Publications
| March 2025
  • Innovation Incentives and Competition
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Biologic originator compaies strategically use manufacturing platform patents to delay or block biosimilar competition. Many such patents are filed long after FDA approval and may be invalid or unused, yet are still widely asserted in litigation, often without scrutiny of prior use by the USPTO. Improved FDA-USPTO coordination and caps on post-approval patent assertions can promote fairer competition and transparency.
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Research Submission to Inform Medicare Drug Price Negotiation, IPAY 2027

Martin MJ, Kesselheim AS, Rome BN - Centers for Medicare and Medicaid Services (CMS)

  • Policy Interactions
| March 2025
  • Price, Value, and Access
  • Regulation and Clinical Evidence
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PORTAL submits summaries of peer-reviewed research to inform CMS’s evidence-based assessment of drugs selected for Medicare price negotiation in Initial Price Applicability Year (IPAY) 2027, including Trelegy Ellipta, Breo Ellipta, Ozempic/Rybelsus/Wegovy, Pomalyst, Xtandi, and Calquence.
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Primary Medication Adherence in Medicare Beneficiaries Prescribed Sacubitril-Valsartan or Renin-Angiotensin System Blockers for Heart Failure with Reduced Ejection Fraction

Hwang CS, Desai RJ, Kesselheim AS, Levin R, Rome BN - American Heart Journal

  • Research Publications
| March 2025
  • Price, Value, and Access
  • Regulation and Clinical Evidence
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No difference in medication adherence was observed among patients newly prescribed a costly brand-name heart failure medication, sacubitril-valsartan (Entresto), compared to those prescribed an inexpensive angiotensin-converting enzyme inhibitor (ACE-I) or angiotensin-II-receptor blocker (ARB). Adherence was high among patients switching from an ACE-I or ARB to sacubitril-valsartan, suggesting that this switch was not associated with interruptions in renin-angiotensin blockade.
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Out-Of-Pocket Savings Under the Inhaler Price Cap

Mein SA, Kludze AK, Feldman WB, Tale AP, Stevens JP, Wadhera RK - JAMA Internal Medicine

  • Research Publications
| March 2025
  • Price, Value, and Access
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Three of the four major inhaler manufacturers in the US announced voluntary $35 out-of-pocket price caps for inhalers in 2024. Using data from the Medical Expenditure Survey Panel approximately 40% of patients receiving an inhaler for COPD or asthma during the study period would have benefited from the price cap, with median annual out-of-pocket savings of $76.
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National Survey of Factors Associated with Physician Antibiotic Prescribing Preferences

Hong D, Kesselheim AS, Morlock R, Metlay JP, Powers JH, Feldman WB - Journal of General Internal Medicine

  • Research Publications
| March 2025
  • Innovation Incentives and Competition
  • Regulation and Clinical Evidence
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Among a group of 130 surveyed physicians, the majority reported prioritizing an antiobiotic’s clinical outcomes, including treatment efficacy and risk of side effects, over economic or public health-related factors like patient out-of-pocket costs or antibiotic resistance risk when making prescribing decisions. Understanding this prioritization can aid the development of policy interventions to incentivize antibiotic development.
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First-In-Class Drugs Experienced Different Regulatory Treatment in the US and Europe

Han J, Kesselheim AS - Health Affairs

  • Research Publications
| March 2025
  • Regulation and Clinical Evidence
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In this review of FDA and EMA approval data for nearly 200 first-in-class drugs between 2013 and 2023, the FDA was found to offer significantly greater regulatory flexibility, with 50% of first-in-class drugs approved based on pivotal trials lacking clinical endpoints and 30% lacking blinding. The FDA also grants more expedited review designations than the EMA. These findings reveal the challenge for regulators in balancing accelerated access to novel drugs with thorough regulatory scrutiny.
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Changes in Biologic Drug Revenues After Administrative Patent Challenges

Raymakers AJN, Van de Wiele VL, Kesselheim AS, Tu SS - Health Affairs

  • Research Publications
| March 2025
  • Innovation Incentives and Competition
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Since 2012, 70-74% of biologic patents challenged via inter partes review (IPR) by biosimilar or alternative product manufacturers were invalidated, and these challenges were associated with reduced revenue for reference biologics. Policymakers should not restrict access to IPR, as it is a valuable pathway to facilitate biosimilar entry.
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An Empirical Investigation Into Concerns Over Quality-Adjusted Life-Years: A Review of Cost-Effectiveness Analyses in Oncology

Raymakers AJN, Rand LZG, Feldman WB, Kesselheim AS - Journal of Cancer Policy

  • Research Publications
| March 2025
  • Price, Value, and Access
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A common argument put forth by those who wish to restrict the use of quality-adjusted life-years (QALYs) is that cost-effectiveness analyses (CEAs) using QALYs identify sub-groups of patients for whom a new treatment is not considered cost-effective, thereby limiting access. In this analysis, the authors identified all US-based cancer CEAs and found no substantive evidence that this analytical framework was used in the way suggested. These findings are further evidence that bans on QALY use lack empirical justification.
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Using Trademarks to Ensure Completion of Postapproval Trials

Hwang CS, Tu SS - JAMA Health Forum

  • Commentary & Opinion
| February 2025
  • Innovation Incentives and Competition
  • Regulation and Clinical Evidence
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The FDA should use trademarks to ensure the timely completion of postapproval confirmatory trials for drugs granted accelerated approval . Manufacturer use of a drug’s trademarked brand or generic name should be restricted until trials confirm clinical benefit, instead requiring investigational names in promotional materials to communicate the drug’s clinical uncertainty.
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The Moral Injury of Inhaler Prescribing

Feldman WB, Furie GL - New England Journal of Medicine

  • Commentary & Opinion
| February 2025
  • Price, Value, and Access
  • Regulation and Clinical Evidence
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The authors discuss the dilemmas in treating patients with chronic lung disease, including the tension between the environmental impacts of existing metered-dose inhalers, limited access to dry powder inhalers, and the affordability challenges patients may face from the high cost of new, low-carbon alternatives. They call for structural reform to address this tension to ensure patients can access, effective, affordable, and environmentally stable therapies.
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Brief of Law Scholars in Novartis v. Kennedy

Kesselheim AS, Sarpatwari A, Tu SS - US Court of Appeals for the Third Circuit

  • Policy Interactions
| February 2025
  • Price, Value, and Access
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The amici argue that the Medicare drug price negotiation program does not effectuate an unconstitutional taking under the Fifth Amendment, because the federal government routinely negotiates prices as a market participant and because the pharmaceutical industry is precisely the kind of highly regulated, government-privileged sector in which price regulation has long been held constitutional. They further warn that accepting Novartis’s per se takings theory would destabilize the negotiated discount structures underlying Medicare, Medicaid, the 340B program, and the Veterans Health Administration, and could even invite takings challenges to obligations like EMTALA’s emergency care mandate.
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Brief of 14 Professors of Medicine and Law in Teva v. Amneal (Rehearing En Banc)

Feldman WB, Kesselheim AS, Tu SS, Avorn J, Rome BN - US Court of Appeals for the Federal Circuit

  • Policy Interactions
| February 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
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Filed in opposition to Teva’s petition for rehearing en banc, the amici argues that Teva’s predictions that the panel’s decision will dramatically shrink the Orange Book and unleash a flood of antitrust litigation, are unfounded. Device-only patents represent only about 5% of the roughly 10,000 patents listed in the Orange Book over the past 40 years, and prior challenges to improperly listed patents have been limited. The authors contend that the panel’s ruling aligns with the Hatch-Waxman Act’s goals and FDA’s own understanding of listable patents, and that delisting improperly listed device-only patents will benefit patients by removing barriers to lower-cost generic competition for important therapies like inhalers.
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Out of Pocket Getting Out of Hand—Reducing the Financial Toxicity of Rapidly Approved Drugs

Ubel PA, Grouls A, Kesselheim AS - New England Journal of Medicine

  • Commentary & Opinion
| February 2025
  • Price, Value, and Access
  • Regulation and Clinical Evidence
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Patients increasingly face financial toxicity from the high cost of drugs with uncertain clinical benefits. Various strategies can protect patients from financial harm while these drugs undergo further review, including enhancing drug labeling for accelerated approval products, establishing a drug price review board to recommend value-based prices, and capping out-of-pocket costs until post-approval studies are completed.
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Health Care Spending After Initiating Sacubitril-Valsartan vs Renin-Angiotensin System Blockers for Heart Failure Treatment

Hwang CS, Desai RJ, Kesselheim AS, Levin R, K Sreedhara S, Rome BN - JAMA Health Forum

  • Research Publications
| February 2025
  • Price, Value, and Access
  • Regulation and Clinical Evidence
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Among Medicare patients initiating sacubitril-valsartan (Entresto) versus ACE-I/ARBs for heart failure with reduced ejection fraction, those initiating sacubitril-valsartan had similar total health care spending compared to those starting on ACE-I/ARBs, with sacubitril-valsartan’s high cost offset by lower inpatient and outpatient spending. However, initiating sacubitril-valsartan was associated with higher out-of-pocket costs, which may negatively impact patient adherence and access.
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Physician Experiences with and Perspectives on Clozapine Prescribing

Sarpatwari A, Lu Z, Russo M, Zakoul H, Lee SB, Toyserkani GA, Zhou EH, LaCivita C, Hyatt Hawkins Shaw K, Dal Pan GJ, Kesselheim AS - JAMA Network Open

  • Research Publications
| February 2025
  • Regulation and Clinical Evidence
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Clozapine, an effective therapy for treatment-resistant schizophrenia, had a risk evaluation and mitigation strategy (REMS) program in place to manage the risk of agranulocytosis. In a survey of nearly 200 physicians, two-thirds viewed the REMS program positively, though some physicians called for more educational materials and raised concerns about delays in patient access due to required laboratory testing.
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Predicting Patent Challenges for Small-Molecule Drugs: A Cross-Sectional Study

Memedovich A, Steele B, Orr T, Chaudhry S, Tadrous M, Kesselheim AS, Hollis A, Beall RF - PLOS Medicine

  • Research Publications
| February 2025
  • Innovation Incentives and Competition
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The authors used supervised machine learning techniques to predict Paragraph IV patent challenges within a branded drug’s first year of eligibility, finding that drugs with large market sizes are more likely to face patent challenges in the first year, while anti-infective drugs and those with fast track designations were less likely to be challenged, demonstrating inconsistencies in generic entry across the prescription drug market.
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Regulatory Flexibilities Balancing Unmet Needs, Benefits and Risks in the Approvals of Imported Cancer Drugs in China: a Cohort Study From 2012 to 2021

Mao X, Xu J, Liu X, Kong S, Li Y, Bai X, Yang J, Kesselheim AS, Li G - Lancet Regional Health - Western Pacific

  • Research Publications
| February 2025
  • Regulation and Clinical Evidence
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Among new oncology drugs approved in China between 2012 and 2021, most pivotal trials supporting approval were early phase, single-arm studies with response rate as the primary endpoint and few Chinese trial participants. This reliance on limited clinical evidence reveals a balance between addressing unmet medical needs and maintaining regulatory standards.
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Physician-Patient Communication About Novel Drugs and High-Risk Medical Devices

Dhruva SS, Kesselheim AS, Woloshin S, Ji RZ, Lu Z, Darrow JJ, Redberg RF - Medical Decision Making

  • Research Publications
| February 2025
  • Regulation and Clinical Evidence
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In a survey of 500+ physicians, half reported that they “usually” tell patients when a drug or device was approved based on a surrogate measure or when the FDA required a post-approval trial to further evaluate a product’s safety or effectiveness. Physician communication with patients about the quality of evidence supporting the new drugs and devices they prescribe is critical important.
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Government Funding for the Development of Enzalutamide

Gyawali B, Jung EH, Mooney H, Avorn J, Kesselheim AS - JAMA Oncology

  • Research Publications
| February 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
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The US government contributed at least $74 million to the development of prostate cancer drug enzalutamide (Xtandi), most of which was invested in the early research stages when there is often the greatest risk. Given the treatment’s high cost, the government should act to ensure US taxpayers can access Xtandi and other drugs with substantial government investment at a fair price.
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Financial Challenges of Being on Long-Term, High-Cost Medications

Elshiekh C, Rudà R, Cliff ERS, Gany F, Budhu JA - Neuro-Oncology Practice

  • Research Publications
| February 2025
  • Price, Value, and Access
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Long‑term use of high‑cost cancer therapies imposes substantial financial toxicity on patients, particularly in health systems without price negotiation or cost‑effectiveness review. Expanding health technology assessment, price negotiation, and patient access protections are essential as indefinite targeted therapies become more common.
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AI Devices in Neurology-Moving From Diagnosis to Prognosis

Hillis JM, Cliff ERS, Vokinger KN - JAMA Neurology

  • Commentary & Opinion
| February 2025
  • Regulation and Clinical Evidence
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The FDA’s first authorization of an AI prognostic device for dementia progression marks a shift toward lifecycle regulation with heavy reliance on post‑market evidence. The authors emphasize the need for rigorous enforcement of post‑approval studies and caution clinicians against over‑interpreting probabilistic predictions in clinical decision‑making.
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Op-Ed: How to Protect HHS, FDA, NIH, and Other Health Agencies From Political Interference

Bhaskar A, Kesselheim AS, Carpenter D - STAT

  • Commentary & Opinion
| January 2025
  • Regulation and Clinical Evidence
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Political interference in the FDA and other health agencies during the 2020 election and COVID-19 pandemic severely damaged public trust in public health institutions. Amid new risks to scientific independence, Congress should act to ensure that public health decisions at HHS, FDA, NIH, and CDC are guided by evidence rather than politics.
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A Regulatory Roadmap for Repurposing: Comparing Pathways for Making Repurposed Drugs Available in the EU, UK, and US

Scholte M, Bendicksen L, Grimm SE, Abu-Zahra T, Pauly B, Joore MA, Kesselheim AS - Journal of Law, Medicine & Ethics

  • Research Publications
| Winter 2025
  • Innovation Incentives and Competition
  • Regulation and Clinical Evidence
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The authors outline existing pathways in the US, EU, and UK for repurposed drugs to receive regulatory approval for new uses. A repurposed drug’s status as a new drug, off-patent drug, or shelved drug (drugs abandoned during the research phase) alter the regulatory standards required to obtain approval for new uses, alongside other barriers academic or nonprofit institutions may face in trying to repurpose drugs.
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Comments on Draft Guidance, “Expedited Program for Serious Conditions-Accelerated Approval of Drugs and Biologics”

Tibau Martorell A, Martin MJ, Kesselheim AS - Food and Drug Administration (FDA)

  • Policy Interactions
| January 2025
  • Regulation and Clinical Evidence
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The commenters offer recommendations to strengthen the accelerated approval pathway, including reforms to address delays in confirmatory trial completion, overreliance on unvalidated surrogate measures, and extended timelines for withdrawal of indications when clinical benefit is not confirmed.
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Brief of Law Scholars in Boehringer Ingelheim v. Kennedy

Kesselheim AS, Sarpatwari A, Tu SS - US Court of Appeals for the Second Circuit

  • Policy Interactions
| January 2025
  • Price, Value, and Access
An icon of a bill representing government policy.
The amici support affirmance of the district court decisions in the challenges to the Medicare drug price negotiation program brought by Boehringer Ingelheim. They argue that the government routinely negotiates contract prices for goods including drugs without implicating the Takings Clause, that Congress has broad authority to directly regulate prices in the heavily-subsidized and highly-regulated pharmaceutical industry, and that ruling for Boehringer would destabilize Medicare, Medicaid, and Veterans Administration drug programs that depend on negotiated discounts.
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Characteristics of Trials Preceding FDA Approval of Novel Psychiatric Drugs

Ahn-Horst RY, Turner EH, Kesselheim AS - JAMA Network Open

  • Research Publications
| January 2025
  • Regulation and Clinical Evidence
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Among 16 novel psychiatric drugs approved by the FDA between 2013 and 2024, most products targeted common mechanisms of action and were approved based on multiple positive phase 3 trials. However, three drugs were found to have been approved despite having fewer than half of the supporting trials being positive, underscoring inconsistencies in FDA approval standards that suggest the need for clearer regulatory guidelines for psychiatric drug approvals.
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The Role of Importation in Remediating U.S. Generic Drug Shortages

Bollyky TJ, Nagar SN, Searchinger C, Kesselheim AS - New England Journal of Medicine

  • Commentary & Opinion
| January 2025
  • Price, Value, and Access
  • Regulation and Clinical Evidence
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Regulatory delays and manufacturing challenges have contributed to persistent shortages of generic drugs, particularly injectable products, and the FDA should expedite temporary importation of drugs in shortage from well-regulated foreign markets to enhance supply chain resilience to mitigate drug shortages.
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Comments on Draft Guidance, “Expedited Program for Serious Condions-Accelerated Approval of Drugs and Biologics”

Ratain MJ, Kesselheim AS - Food and Drug Administration (FDA)

  • Policy Interactions
| January 2025
  • Regulation and Clinical Evidence
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The FDA should clarify how it will evaluate confirmatory trials conducted in populations different from the original accelerated approval population. The commenters also urge the agency to require confirmation of appropriate dosing as part of the accelerated approval process to ensure accurate benefit-risk assessments.
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Academic Detailing Interventions and Evidence-Based Prescribing: A Systematic Review

Rome BN, Dancel E, Chaitoff A, Trombetta D, Roy S, Fanikos P, Germain J, Avorn J - JAMA Network Open

  • Research Publications
| January 2025
  • Regulation and Clinical Evidence
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In this systemic review of 118 studies, academic detailing (interactive educational outreach to clinicians) was shown to increase evidence-based prescribing in a variety of clinical settings. Among 36 studies deemed to have the lowest risk of bias, 25 (69%) showed significant improvements in at least one prescribing behavior, and the median proportion of patients who used the targeted medications changing by 4.0% in the intended direction.
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Accelerating Biosimilar Market Access: The Case for Allowing Earlier Standing

Tu SS, Goode R, Turner M, Van de Wiele VL - Journal of Law and the Biosciences

  • Research Publications
| January 2025
  • Innovation Incentives and Competition
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Biosimilar manufacturers should be permitted to initiate patent litigation at the start of Phase 3 clinical trials rather than after FDA approval. This reform could expedite market access, reduce costs, and align biosimilar patent processes with those of small-molecule generics.
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Leveraging Old Hepatitis C Therapies

Tu SS, Kottilil S, Mattingly II TJ - New England Journal of Medicine

  • Commentary & Opinion
| January 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
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The authors highlight strategies to improve access to high-cost hepatitis C treatments in the US, including leveraging older direct-acting antivirals (DAAs), acquiring patents for generic production, or negotiating better prices for current therapies.
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Use of Means-Plus-Function Claiming to Evade the Enablement Requirement

Bonis T, Tu SS - Texas Intellectual Property Law Journal

  • Research Publications
| January 2025
  • Innovation Incentives and Competition
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The authors argue that “means-plus-function” type patent claims should not be allowed to bypass the enablement requirement.  Courts should simply apply the Amgen v. Sanofi standard to all claims, including means-plus-function claims, to prevent overly broad patents.
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Revising EU Pharmaceutical Legislation: Will It Foster Drug Repurposing?

Scholte M, Grimm SE, Pauly B, Verbeeck F, Pasmooij AMG, Bouma B, Van Duijn-Wiersma J, Guney E, Kesselheim AS, Schmidt HHHW, Joore MA - Drug Discovery Today

  • Commentary & Opinion
| January 2025
  • Innovation Incentives and Competition
  • Regulation and Clinical Evidence
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Drug repurposing provisions included in EU pharmaceutical legislation have the potential to improve access to affordable treatments, but additional regulatory support, fair data protection periods, and improved awareness strategies are needed tomake repurposed drugs more accessible, affordable, and sustainable.
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Pediatric Exclusivity Revenues for Cancer Drugs

Sarpatwari A, Bendicksen L, Hawkins DS, Gore L, Bourgeois FT - JAMA Pediatrics

  • Research Publications
| January 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
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When comparing the revenues generated by 6-month pediatric exclusivities to the cost of clinical trials required to obtain these exclusivities for a set of four oncology drugs, the average manufacturer revenue generated by the added exclusivity ($309 million) exceeded the average investment cost required to conduct the underlying trials ($156 million).
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Comparative Effectiveness and Safety of Single Inhaler Triple Therapies for Chronic Obstructive Pulmonary Disease: New User Cohort Study

Feldman WB, Suissa S, Kesselheim AS, Avorn J, Russo M, Schneeweiss SG, Wang SV - BMJ

  • Research Publications
| December 2024
  • Regulation and Clinical Evidence
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Two single-inhaler triple therapies for chronic obstructive pulmonary disease (COPD) are available in the US: Breztri Aerosphere, a twice daily metered-dose inhaler, and Trelegy Ellipta, a once daily dry powder inhaler. Breztri Aerosphere was associated with a 9% higher risk of moderate or severe COPD exacerbations and an identical risk of pneumonia hospitalizations compared to Trelegy Ellipta among patients with COPD treated in routine clinical practice.
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Differential Legal Protections for Biologics vs Small-Molecule Drugs in the US

Wouters OJ, Vogel M, Feldman WB, Beall RF, Kesselheim AS, Tu SS - JAMA

  • Research Publications
| December 2024
  • Innovation Incentives and Competition
  • Price, Value, and Access
  • Regulation and Clinical Evidence
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The authors explore whether there is empirical justification for the greater legal protections given to biologics over small-molecule drugs in the US. Across development times, clinical trial success rates, research and development costs, patent protection, market exclusivity periods, revenues, and treatment costs, no no evidence supporting differential treatment was found, suggesting that US law overly rewards the development of biologics relative to small-molecule drugs.
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Op-Ed: Lessons from Humira on How to Tackle Unjust Extensions of Drug Monopolies with Policy

Bonis T, Tu SS - BioSpace

  • Commentary & Opinion
| December 2024
  • Innovation Incentives and Competition
Talk bubble graphics representing commentary and opinion.
Using Humira as a case study, the authors highlight how drugmakers extend monopolies by building patent thickets of obvious, low‑value patents with terminal disclaimers that delay generic and biosimilar competition and keep prices high. Congress should curb these practices by limiting enforcement of patents tied by terminal disclaimers, promoting competition without undermining genuine pharmaceutical innovation.
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Characteristics of Academic Inventors on Government-Linked US Drug Patents

Martin MJ, Gabriele SME, Kesselheim AS, Tu SS - JAMA

  • Research Publications
| December 2024
  • Innovation Incentives and Competition
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Among academic inventors listed on small molecule drug patents tied to US federal funding, most were senior researchers at major research institutions, many of whom had robust grant portfolios and prior industry experience. By contrast, early- and mid-career researchers were less represented, presenting an opportunity for NIH to expand its role in technology transfer by offering patent-related training and financing to these investigators.
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Use of Public Research and Manufacturing Enterprises to Lower Prescription Drug Prices and Increase Innovation

Moss A, Brown D, Tu SS - Journal of Law, Medicine & Ethics

  • Commentary & Opinion
| Winter 2024
  • Price, Value, and Access
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The authors discuss the opportunities and challenges associated with establishing public research and manufacturing enterprises (PRME) to expand the government’s role in the development, manufacturing, and distribution of new drugs. Moving beyond investments in early-stage research could improve access to and affordability of novel products while maximizing public welfare, though the financial and political capital required to launch such a venture could be substantial.
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Will the New Administration’s FDA Be a Threat or an Opportunity for the Public’s Health?

Kesselheim AS, Sharfstein JM - Health Affairs Forefront

  • Commentary & Opinion
| December 2024
  • Regulation and Clinical Evidence
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The authors outline the threat some proposed changes to the FDA pose to public health while outlining alternative reforms that would benefit the public interest by decreasing industry influence at the FDA, improving transparency, addressing prescription drug affordability, and strengthening the agency’s scientific integrity.
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Use, Spending, and Prices of Adalimumab Following Biosimilar Competition

Rome BN, Bhaskar A, Kesselheim AS - JAMA Health Forum

  • Research Publications
| December 2024
  • Innovation Incentives and Competition
  • Price, Value, and Access
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Following the introduction of the first adalimumab (Humira) biosimilar to the US market, biosimilars made up 40%, the biologic’s list price increased by 8%. This indicates that Humira’s manufacturer likely offered sizeable rebates to health plans and PBMs to retain formulary placement.
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Prescription Drug Price Measures to Inform State Upper Payment Limits

Martin MJ, Mooney H, Kesselheim AS, Rome BN - National Academy for State Health Policy (NASHP)

  • Policy Interactions
| December 2024
  • Price, Value, and Access
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This memo provides an overview of the many prescription drug price measures that state PDABs may encounter when setting upper payment limits, organized by supply chain transactions, reimbursement rates, and purchaser-specific prices. It also compares these measures across federal programs and presents a price comparison for nine drugs selected in the first round of Medicare price negotiation.
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Brief of Law Scholars in Novo Nordisk v. Kennedy

Kesselheim AS, Sarpatwari A, Tu SS - US Court of Appeals for the Third Circuit

  • Policy Interactions
| December 2024
  • Price, Value, and Access
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The amici argue that the Medicare drug price negotiation program is constitutional because the federal government has long-settled authority to negotiate prices for goods it purchases and to regulate prices within industries, particularly those (like pharmaceuticals) that benefit from extensive government-granted privileges such as patents, regulatory exclusivities, and research subsidies. They warn that accepting Novo Nordisk’s Fifth Amendment takings theory would destabilize core federal health programs, including Medicaid, the 340B program, the Veterans Health Administration, and EMTALA, all of which rely on similar statutory pricing and discount mechanisms to function.
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Clearing Dense Drug-Patent Thickets

Chao B, Whalen R, Kesselheim AS, Tu SS - New England Journal of Medicine

  • Research Publications
| December 2024
  • Innovation Incentives and Competition
Simple graphic of magnifying glass examining papers to signify reviewing research publications.
Two proposed patent reforms, the USPTO terminal disclaimer rule and a bipartisan bill to address patent thickets, could significantly reduce patent thickets on biologic and small molecule products. For two products, adalimumab (Humira), and lenalidomide (Revlimid), the USPTO rule would reduce each product’s thicket by 43% and 70%, respectively, while the legislationw would making only 25 of 105 Humira patents and 12 of 30 Revlimid patents enforceable.
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