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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Deeming Doesn’t Deliver: Reliance on Foreign Regulators Targets the Wrong Stage of Canadian Drug Access

Eisenkraft Klein D, Kesselheim AS - Health Policy

  • Price, Value, and Access
  • Regulation and Clinical Evidence
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Using Regression Discontinuity in Time to Strengthen Real-World Evidence: A Case Study in Lung Cancer

Chen NC, Zemplenyi AT, Adamson B, Kaizer AM, O’Bryant CL, McQueen RB, Anderson KE - Medical Decision Making

  • Regulation and Clinical Evidence
Using a real-world cohort of 1,975 patients with advanced non-small-cell lung cancer, the authors applied regression discontinuity in time (RDiT) to estimate the comparative effectiveness of pembrolizumab versus docetaxel while addressing unmeasured confounding from historical controls. RDiT produced survival estimates more closely aligned with long-term clinical trial data than traditional propensity score methods, suggesting it may be a valuable tool for generating credible real-world evidence to inform regulatory, coverage, and drug pricing decisions.
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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