Trial reportBMC cancer2021
Food and Drug Administration approvals in phase 3 Cancer clinical trials.
Trial report in BMC cancer, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers, 1 of them a synthesis that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
16 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Correlation between surrogate endpoints and overall survival in unresectable hepatocellular carcinoma patients treated with immune checkpoint inhibitors: a systematic review and meta-analysis.Scientific reports · 2024Pooled it
- Bayesian interim analysis and efficiency of phase III randomized trials.British journal of cancer · 2025Article
- Real-World Data and Causal Machine Learning to Enhance Drug Development.Therapeutic innovation & regulatory science · 2025Review
- Survival-inferred fragility of statistical significance in phase III oncology trials.NPJ precision oncology · 2025Article
- Incomplete Toxicity Reporting and Use of Toxicity-Minimizing Language in Phase III Oncology Trials.JCO oncology practice · 2025Article
- Health-related quality of life outcomes in randomized controlled trials in metastatic hormone-sensitive prostate cancer: a systematic review.EClinicalMedicine · 2024Review
- Off-Protocol Radiation Therapy in Phase 3 Metastatic Solid Tumor Trials.International journal of radiation oncology, biology, physics · 2024Article
- Towards replicability and sustainability in cancer research.BJC reports · 2024Review
- Increasing Power in Phase III Oncology Trials With Multivariable Regression: An Empirical Assessment of 535 Primary End Point Analyses.JCO clinical cancer informatics · 2024Article
- Secondary Endpoint Utilization and Publication Rate among Phase III Oncology Trials.Cancer research communications · 2024Article
- Towards Treatment Effect Interpretability: A Bayesian Re-analysis of 194,129 Patient Outcomes Across 230 Oncology Trials.medRxiv : the preprint server for health sciences · 2024Article
- Differential Treatment Effects of Subgroup Analyses in Phase 3 Oncology Trials From 2004 to 2020.JAMA network open · 2024Article
- Article
- Challenges, Complexities, and Considerations in the Design and Interpretation of Late-Phase Oncology Trials.Seminars in radiation oncology · 2023Review
- Assessment of Surrogate End Point Trends in Clinical Trials to Approve Oncology Drugs From 2001 to 2020 in Japan.JAMA network open · 2023Article
- Selection of endpoints in breast cancer clinical trials: a qualitative study of key trial stakeholders.American journal of cancer research · 2022Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
14 authors.
Funding
Abstract
backgroundPhase 3 oncologic randomized clinical trials (RCTs) can lead to Food and Drug Administration (FDA) approvals. In this study, we aim to identify trial-related factors associated with trials leading to subsequent FDA drug approvals.
methodsWe performed a database query through the ClinicalTrials.gov registry to search for oncologic phase 3 RCTs on February 2020. We screened all trials for therapeutic, cancer-specific, phase 3, randomized, multi-arm trials. We then identified whether a trial was used for subsequent FDA drug approval through screening of FDA approval announcements.
resultsIn total, 790 trials were included in our study, with 225 trials (28.4%) generating data that were subsequently used for FDA approvals. Of the 225 FDA approvals identified, 65 (28.9%) were based on trials assessing overall survival (OS) as a primary endpoint (PEP), two (0.9%) were based on trials with a quality of life (QoL) PEP, and 158 approvals (70.2%) were based on trials with other PEP (P = 0.01). FDA approvals were more common among industry funded-trials (219, 97.3%; P < 0.001), and less common among trials sponsored by national cooperative groups (21, 9.3%; P < 0.001). Finally, increased pre-hoc power and meeting patients' accrual target were associated with FDA approvals (P < 0.001).
conclusionsThe majority of FDA approvals are based on data generated from trials analyzing surrogate primary endpoints and trials receiving industry funding. Additional studies are required to understand the complexity of FDA approvals.
Indexed as
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What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.