AI Insight
This study analyzed 143 immunotherapy and 211 non-immunotherapy cancer drug approvals by the FDA from 2014 to 2025, finding that while immunotherapies initially received faster approvals, approval timelines have converged to approximately 2,000-2,300 days. Most approvals now rely on surrogate endpoints rather than overall survival, but immunotherapies show better translation of surrogate benefits to actual survival outcomes (99% vs 60% for non-immunotherapies). The European Medicines Agency generally applies more conservative approval standards than the FDA.
Why it matters
These findings reveal important differences in how immunotherapies and traditional cancer drugs perform in clinical trials and regulatory evaluation, which could inform future drug development strategies and regulatory policies. The substantial gap in how well surrogate endpoints predict actual survival benefits raises concerns about the reliability of these measures for non-immunotherapy approvals.
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⚠️ Preprint – Noch nicht peer-reviewed
Dieser Artikel wurde noch nicht von unabhängigen Experten begutachtet. Die Ergebnisse sind vorläufig und sollten mit Vorsicht interpretiert werden.
In the United States the Food and Drug Administration (FDA), and in Europe, the European Medicines Agency (EMA) are responsible for regulatory approvals of anti-cancer therapeutics. We examined regulatory approvals by both agencies in the period between 2014 and 2025 and compared immunotherapy and non-immunotherapy approvals for the treatment of cancer. We asked whether the approvals were similar in terms of trial size, endpoints, and development time. We present the data based on the FDA approvals given that the US agency has approved some therapies either rejected by or yet to be approved by the EMA. Beginning with the approval of pembrolizumab for melanoma in 2014 through early 2025 we identified 143 immunotherapy and 211 non-immunotherapy approvals. In the first few years the time to approvals of immunotherapies was markedly less but over time the duration has approached that of non-immunotherapies – with durations of 2079 and 2357 days in 2024/2025 due largely to a median trial size of 461 and 698 participants. Surrogate endpoints have overwhelmed overall survival as the primary endpoint for approval, and we find that with non-immunotherapies only a median of 60% of the benefit predicted from progression/disease/recurrence/event-free survivals translates to overall survival, significantly less than the 99% of the surrogate benefit translated to overall survival for immunotherapies. While the FDA and EMA approval portfolios are very similar, the EMA is on the whole more skeptical/conservative in its approvals. The last decade has seen substantial advances, but much remains to be accomplished against a formidable foe. The size and duration of trials need to be addressed and meaningfully reduced and the validity of surrogates defended.