Analysis of FDA's Accelerated Approval Program Performance December 1992–December 2021

Author:

Beakes-Read GinnyORCID,Neisser Madison,Frey Patrick,Guarducci Mara

Abstract

AbstractThe accelerated approval pathway has been criticized recently for employing lower regulatory standards than traditional drug approval, undue delays in withdrawing approvals of drugs for which studies have not confirmed clinical benefit, and confirmatory trials not being pursued with due diligence. This article examines the status of confirmatory studies of drugs approved under the US Food and Drug Administration’s (FDA’s) accelerated approval program between December 1992 and December 2021. It includes background on the program and provides broader context about the program’s performance to date over its 30-year history. Our analysis demonstrates that the accelerated approval program has been largely successful, with half of accelerated approvals converted to traditional approval in a median time of 3.2 years. Furthermore, recent FDA actions show that the agency is appropriately managing the program when a drug approved under accelerated approval fails to confirm a clinical benefit. Any proposed changes to the program should be based on cumulative experience with the program, rather than outliers.

Publisher

Springer Science and Business Media LLC

Subject

Pharmacology (medical),Public Health, Environmental and Occupational Health,Pharmacology, Toxicology and Pharmaceutics (miscellaneous)

Reference16 articles.

1. Federal Food, Drug, and Cosmetic Act section 506(c)(1)(A); 21 U.S.C. § 356(c)(1)(A).

2. Federal Food, Drug, and Cosmetic Act section 505(d), 21 U.S.C. §§ 355(d) & Federal Food, Drug, and Cosmetic Act section 506(c)(1)(A), 21 U.S.C. § 356(c)(1)(A). See also FDA Guidance for Industry, “Expedited Programs for Serious Conditions – Drugs and Biologics” (2014), at 19 (“Drugs granted accelerated approval must meet the same statutory standards for safety and effectiveness as those granted traditional approval.”). See also U.S. FDA Draft Guidance, Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products (December, 2019), Section II.A.

3. Guidance for Industry, “Expedited Programs for Serious Conditions – Drugs and Biologics” (2014), at 17. (“For purposes of accelerated approval, a surrogate endpoint is a marker, such as a laboratory measurement, radiographic image, physical sign, or other measure, that is thought to predict clinical benefit, but is not itself a measure of clinical benefit.”)

4. FDA Guidance for Industry, “Expedited Programs for Serious Conditions – Drugs and Biologics” (2014).

5. FDA Postmarket Requirements and Commitments Page https://www.accessdata.fda.gov/scripts/cder/pmc/index.cfm.

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