FDA accelerated approval pathway lacks transparency, influential pricing body warns

ICER report calls for stricter evidence standards, value-based pricing links and tougher enforcement of confirmatory trial requirements

The US Food and Drug Administration should do more to improve transparency when granting accelerated approvals for new drugs, according to a report published on Thursday by the Institute for Clinical and Economic Review, one of the most influential bodies in American drug pricing policy.

The accelerated approval pathway, designed to speed up authorisation of treatments for serious conditions where no alternatives exist, allows the FDA to approve drugs based on surrogate endpoints — markers that predict clinical benefit without directly measuring it — rather than waiting for full evidence of patient outcomes. Companies whose drugs receive accelerated approval are still required to conduct confirmatory trials to verify the anticipated benefit.

The pathway has come under increasing scrutiny in recent years. The controversy surrounding Biogen's Alzheimer's drug Aduhelm, which received accelerated approval in 2021 despite the absence of clear evidence of benefit to patients, proved a watershed moment. The company ultimately abandoned the treatment three years later. More recent recipients of accelerated approval include Denali Therapeutics' rare disease treatment Avlayah and Rocket Pharmaceuticals' gene therapy Kresladi, approved for a childhood disorder.

Sarah Emond, chief executive of ICER, told Reuters that the pathway has become a flashpoint for competing concerns within the industry and among regulators. Some argue that increasingly demanding evidence standards are suppressing the number of drugs being approved; others contend that too many treatments are receiving approval on thin evidence without adequate confirmatory follow-up.

The FDA moved last year to address some of these tensions, issuing new guidance to clarify its expectations around clinical evidence — including developing surrogate endpoints that are "reasonably likely" to predict genuine clinical benefit.

ICER's report goes further, recommending a range of structural reforms. These include strengthening the criteria for selecting surrogate endpoints, making advisory committee review mandatory for accelerated approval decisions, and improving the transparency of the FDA's decision-making process.

The report also proposes the introduction of a scoring system for clinical trial and evidence quality, which it argues could allow the agency to monitor the pathway's performance over time and serve as an early warning mechanism for trials at risk of delay or failure to generate the data required for conversion to full approval.

On pricing, ICER recommended that coverage of accelerated approval drugs be tied to value-based pricing, with prices updated as further evidence emerges, and that patient cost-sharing be limited in cases where no alternative treatments are available.

The report further calls for stronger enforcement of requirements to complete confirmatory trials and for focused re-reviews by drug class or disease area, with priority given to those where evidence uncertainty is greatest.

The National Pharmaceutical Council, a health policy research organisation whose members include Eli Lilly, Biogen and Johnson & Johnson, pushed back against the more critical elements of the assessment. Despite acknowledging the pathway's challenges, it said research showed it was "largely working as intended" and that accelerated approvals continued to deliver value to patients, with overall benefits outweighing the risks.