Regulation and Clinical Evidence

PORTAL researchers study how the FDA ensures medical products are safe and effective, and what additional evidence patients and doctors need to make informed clinical decisions.

Regulation & Clinical Evidence

Clinical Benefit and Regulatory Outcomes of Cancer Drugs Receiving Accelerated Approval

Liu ITT, Kesselheim AS, Cliff ERS | JAMA (2024)

Among a 5-year cohort of cancer drugs approved by the FDA under accelerated approval, only half of approvals demonstrated improvements in overall survival or quality of life in confirmatory trials, with 60% of conversions to full approval relying on clinical endpoints.

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Recent Work in Regulation & Clinical Evidence

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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

  • Innovation Incentives and Competition
  • Regulation and Clinical Evidence
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Industry Payments and Prescribing of Brand-Name Multiple Sclerosis Medications in Medicare

Patel AN, Kesselheim AS, Rome BN - Neurology

  • Innovation Incentives and Competition
  • Price, Value, and Access
  • Regulation and Clinical Evidence
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

  • Price, Value, and Access
  • Regulation and Clinical Evidence
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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