Limitations in Clinical Trials Leading to Anticancer Drug Approvals by the US Food and Drug Administration.

JAMA Internal Medicine
Talal HilalVinay Prasad

Abstract

While there have been multiple assessments of clinical trials leading to anticancer drug approvals by the US Food and Drug Administration (FDA), the cumulative percentage of approvals based on trials with a limitation remains uncertain. To assess the percentage of clinical trials with limitations in 4 domains-lack of randomization, lack of significant overall survival advantage, inappropriate use of crossover, and use of suboptimal control arms-that led to FDA approvals from June 30, 2014, to July 31, 2019. This observational analysis included all anticancer drug indications approved by the FDA from June 30, 2014, through July 31, 2019. All indications were investigated, and each clinical trial was evaluated for design, enrollment period, primary end points, and presence of a limitation in the domains of interest. The standard-of-care therapy was determined by evaluating the literature and published guidelines 1 year prior to the start of clinical trial enrollment. Crossover was examined and evaluated for optimal use. The percentage of approvals based on clinical trials with any or all limitations of interest was then calculated. Estimated percentage of clinical trials with limitations of interest that led to an anticancer drug...Continue Reading

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Citations

Aug 25, 2020·Nutrition and Cancer·Diorge Jônatas MarmittMárcia Inês Goettert
Jan 20, 2021·PharmacoEconomics·Adam J N RaymakersStuart J Peacock
Jul 4, 2020·Emerging Microbes & Infections·Peng XuJian Wang
Nov 17, 2020·Journal of European CME·Reinhard GriebenowRobert Schäfer
Mar 27, 2021·The Lancet. Haematology·Ghulam Rehman MohyuddinVinay Prasad
Aug 18, 2021·British Journal of Cancer·John-John B SchnogAshley J Duits
Aug 22, 2021·Journal of Controlled Release : Official Journal of the Controlled Release Society·Monika NehraAjeet Kaushik
Sep 24, 2021·BMJ Open·Francesco PerroneMaria Carmela Piccirillo
Jan 4, 2022·Japanese Journal of Clinical Oncology·Yasushi GotoYuichiro Ohe

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