@article{912037a89acb47ea922632646737690b,
title = "Food and Drug Administration approvals in phase 3 Cancer clinical trials",
abstract = "Background: Phase 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. Methods: We 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. Results: In 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{\textquoteright} accrual target were associated with FDA approvals (P < 0.001). Conclusions: The 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.",
keywords = "Clinical trials, FDA, Industry, Oncology, Primary endpoint, Surrogate endpoint",
author = "{Abi Jaoude}, Joseph and Ramez Kouzy and Marc Ghabach and Roshal Patel and Dario Pasalic and Elie Ghossain and Miller, {Austin B.} and Lin, {Timothy A.} and Vivek Verma and Fuller, {C. David} and Vivek Subbiah and Minsky, {Bruce D.} and Ludmir, {Ethan B.} and Taniguchi, {Cullen M.}",
note = "Funding Information: Dr. Fuller received/receives funding and salary support unrelated to this project during the period of study execution from: the National Institutes of Health (NIH) National Institute of Biomedical Imaging and Bioengineering (NIBIB) Research Education Programs for Residents and Clinical Fellows Grant (R25EB025787–01); the National Institute for Dental and Craniofacial Research Establishing Outcome Measures Award (1R01DE025248/R56DE025248) and Academic Industrial Partnership Grant (R01DE028290); NCI Early Phase Clinical Trials in Imaging and Image-Guided Interventions Program (1R01CA218148); an NIH/NCI Cancer Center Support Grant (CCSG) Pilot Research Program Award from the UT MD Anderson CCSG Radiation Oncology and Cancer Imaging Program (P30CA016672); an NIH/NCI Head and Neck Specialized Programs of Research Excellence (SPORE) Developmental Research Program Award (P50 CA097007); NIH Big Data to Knowledge (BD2K) Program of the National Cancer Institute (NCI) Early Stage Development of Technologies in Biomedical Computing, Informatics, and Big Data Science Award (1R01CA2148250; National Science Foundation (NSF), Division of Mathematical Sciences, Joint NIH/NSF Initiative on Quantitative Approaches to Biomedical Big Data (QuBBD) Grant (NSF 1557679); NSF Division of Civil, Mechanical, and Manufacturing Innovation (CMMI) grant (NSF 1933369); and the Sabin Family Foundation. Direct infrastructure support is provided to Dr. Fuller by the multidisciplinary Stiefel Oropharyngeal Research Fund of the University of Texas MD Anderson Cancer Center Charles and Daneen Stiefel Center for Head and Neck Cancer and the Cancer Center Support Grant (P30CA016672) and the MD Anderson Program in Image-guided Cancer Therapy. Dr. Fuller has received direct industry grant support, honoraria, and travel funding from Elekta AB unrelated to this project. Dr. Taniguchi is supported by funding from NIH under award R01CA227517-01A1, Cancer Prevention & Research Institute of Texas (CPRIT) grant RR140012, V Foundation (V2015–22), the Kimmel Foundation, Sabin Family Foundation Fellowship, and the McNair Foundation. All other authors report no financial disclosures or conflicts of interests related to this work. Funding Information: This work was supported by NIH grant P30 CA016672. Publisher Copyright: {\textcopyright} 2021, The Author(s).",
year = "2021",
month = dec,
doi = "10.1186/s12885-021-08457-5",
language = "English (US)",
volume = "21",
journal = "BMC cancer",
issn = "1471-2407",
publisher = "BioMed Central",
number = "1",
}