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.

Read More

Recent Work in Regulation & Clinical Evidence

Talk bubble graphics representing commentary and opinion.

Healing Our De-Commissioned FDA

Kesselheim AS, Avorn J - Health Affairs Forefront

  • Regulation and Clinical Evidence
Simple graphic of magnifying glass examining papers to signify reviewing research publications.

Post-Approval Pediatric Use of Drugs Granted Waivers from Pediatric Testing

Liu ITT, Savage TJ, Tsacogianis TN, Kesselheim AS, Sarpatwari A - Clinical Pharmacology & Therapeutics

  • Regulation and Clinical Evidence
Talk bubble graphics representing commentary and opinion.

Clinical Implementation of Multi-Cancer Early Detection Tests: Can We Find a Path Forward?

Tibau A, Kesselheim AS - Nature Reviews Clinical Oncology

  • Regulation and Clinical Evidence