USFDA Oncology Drug Approvals from 2014- 2025: Comparing non-immunotherapy and immunotherapy approvals, their endpoints, and their efficacy.
- 1Columbia University
Abstract
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.
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BibTeX
@misc{bapodra2026usfda,
title = {{USFDA Oncology Drug Approvals from 2014- 2025: Comparing non-immunotherapy and immunotherapy approvals, their endpoints, and their efficacy.}},
author = {N. Bapodra and K. G. Morse and S. E. Bates and T. Fojo},
howpublished = {medRxiv},
year = {2026},
month = oct,
doi = {10.64898/2026.10.21.26366185},
url = {https://www.medrxiv.org/content/10.64898/2026.10.21.26366185v1},
note = {CC BY-ND},
}
RIS
TY - UNPB
TI - USFDA Oncology Drug Approvals from 2014- 2025: Comparing non-immunotherapy and immunotherapy approvals, their endpoints, and their efficacy.
AU - N. Bapodra
AU - K. G. Morse
AU - S. E. Bates
AU - T. Fojo
PY - 2026
DA - 2026/10/21
PB - medRxiv
DO - 10.64898/2026.10.21.26366185
UR - https://www.medrxiv.org/content/10.64898/2026.10.21.26366185v1
AB - 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.
ER -