The Institute for Clinical and Economic Review (ICER) called on Apr. 16 for increased transparency in the U.S. Food and Drug Administration’s accelerated approval program, citing concerns from stakeholders about the agency's decision-making process.
The issue is important because the FDA’s accelerated approval pathway allows some drugs to reach patients faster based on early evidence, but questions remain about how decisions are made and whether confirmatory studies are enforced.
According to ICER, "Greater transparency in FDA decision-making was 'perhaps the most common theme in interviews and discussions' conducted by the Institute for Clinical and Economic Review (ICER) in the preparation of its report, entitled: 'Strengthening the FDA’s Accelerated Approval Pathway: Progress and Unfinished Business.'" The report highlights that since its implementation in 1992, the pathway has led to more than 200 new drugs being brought to market as of November 2024. However, debates have intensified over recent approvals such as Biogen’s Alzheimer’s drug Aduhelm and Sarepta's Elevidys due to safety concerns and questions over supporting evidence.
ICER CEO Sarah Emond said that opinions differ on whether current standards are too strict or too lenient. A BioSpace poll found that 34% of respondents believe the FDA is “too inflexible,” while 23% say it is “too flexible.” Nineteen percent felt there was an appropriate balance, with another 24% unsure.
The watchdog group recommended several improvements including better selection of surrogate endpoints—measures used as substitutes for clinical outcomes—and a requirement for advisory committees during reviews. "There continues to be strong demand for better mechanisms to distinguish promising surrogate endpoints from those more likely to fail, and to incorporate outcomes that most matter to patients," ICER noted.
In addition, ICER urged making rationales behind regulatory decisions public: "To work as intended, the AA pathway requires trust among stakeholders... But instances of lack of transparency and accountability in the agency’s processes and decisions regarding AA approvals and confirmatory trials are at the center of some stakeholders’ concerns." The organization also suggested regulations ensuring publicity, relevance, appeals processes for access-limiting decisions, and oversight enforcement.
While analysts told BioSpace that new policies like real-time publication of complete response letters have improved accountability at FDA, they also pointed out ongoing gaps such as redacted information or private meeting minutes limiting outside review.
