SynthesisThe oncologist2026
Pivotal studies of pharmacotherapies approved by the US FDA for cancer treatment: a meta-analysis.
Synthesis in The oncologist, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
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Corrections and comments
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Authors and funding
5 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundThis study aimed to examine the number of FDA-approved cancer pharmacotherapies and analyse pivotal study characteristics over time, including sample sizes.
methodsWe developed a web scraper to collate a cohort of FDA-approved cancer pharmacotherapies from 1953 until 31 December 2024. For each pharmacotherapy, details of pivotal studies leading to approval were recorded, including protocol and final sample size, study design, therapy type, and quality assessment using Cochrane Risk of Bias tools. We used regression analyses and discretization to identify trends in sample size. Type I error was set at 0.05.(Study protocol pre-registration: https://doi.org/10.17605/OSF.IO/KVA23).
resultsWe identified 255 pharmacotherapies, supported by 306 pivotal studies; 125 (49%) were targeted pharmacotherapies, 61 (24%) chemotherapies, 47 (18%) immunotherapies, 21 (8%) hormonal therapies, and 1 (0.4%) other. The median sample size was 290 (IQR = 427); sample sizes increased in the 1990s (median = 407) and remained stable thereafter. Stratified analysis demonstrated smaller sample sizes for phase 1 and 2 studies before 1980, with no change in phase 3 studies. For 165 studies reporting protocol sample sizes, studies from 2020-2024 (median = 147.5) were smaller than those from 2010-2019 (median = 320).
conclusionsThe increase in sample sizes during the 1990s may reflect new policies and legislation. Subsequent stability in sample size could be due to modern trial designs (eg, basket/umbrella studies, surrogate endpoints) that require smaller sample sizes. The recent decrease in protocol sample sizes may herald a similar decline for future studies but requires post-market surveillance to verify credibility.
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