The FDA Accelerated Approval Program: Data Transparency for Public Health

Abstract

The Accelerated Approval Program (AAP) of the US Food and Drug Administration (FDA) authorizes earlier approval of drugs based on surrogate endpoints to fulfill unmet medical need. Compared to the standard approval process, drugs approved through the AAP have greater uncertainty in clinical benefits due to the reliance on surrogate endpoints and lack of initial confirmatory studies, which raises concerns regarding the rigor and transparency of the program. While the Consolidated Appropriations Act enacted on December 29, 2022 requires the FDA to start reporting its postmarket analysis and decision making publicly, it is neither sufficient nor comprehensive to inform the public of the existing public health risks and benefits of the accelerated approved drugs when the preliminary clinical trial data remains confidential. The lack of data transparency and inherent uncertainty surrounding accelerated approved drugs prevents patients from making fully informed choices and puts public health at greater risk. Thus, we propose that the FDA proactively release the de-identified clinical trial data upon accelerated approval. Disclosure of de-identified clinical trial data would strengthen independent, public health-prioritized data interpretation and analysis, which allows physicians and patients to make better informed decisions about their medical treatment.