The FDA should strengthen the evidence needed to approve drugs under the accelerated pathway and increase transparency around regulatory decisions, according to a report (PDF) from a prominent industry and government watchdog.
In the same report, the Institute for Clinical and Economic Review (ICER) also recommends that advisory committees review medicines scheduled for accelerated approval and that pathways more clearly explain the benefits and risks associated with the use of approved medicines within that scope.
“The goal of the accelerated approval pathway has always been to give patients access to innovative treatments faster,” Sara Emond, CEO of ICER, said in the release. “We can point to several success stories where patients have benefited from rapid access to innovative medicines. However, there are also many examples of regulatory inconsistencies, delays in the development of confirmatory trial data, and access restrictions that limit patient use.”
Emond added that patients, manufacturers, payers and purchasers are “frustrated.”
Additionally, ICER said the FDA needs to strengthen its oversight of confirmatory testing and enforcement of postmarket requirements, adding that it must also establish procedures for revoking accelerated approvals without confirmatory evidence.
In its report, ICER cited Sarepta Therapeutics and its Duchenne muscular dystrophy (DMD) drug Exondys 51 as an example of a potential deficiency in the pathway. According to ICER, the drug received early approval in 2016, but Sarepta continued to market the drug and “has since been unable to submit confirmatory data for the drug.”
The FDA established the expedited approval pathway in 1992 in response to the AIDS epidemic and the need to quickly provide medicines for life-threatening diseases with no alternative treatments.
The 2021 accelerated approval of Biogen’s failed Alzheimer’s disease drug Aduhhelm (which was discontinued by the company after less than three years due to lack of efficacy) shined a harsh light on the accelerated pathway.
In January 2025, the Department of Health and Human Services (HHS) Office of Inspector General (OIG) released a report concluding that the Adhelm debacle was a general outlier.
The report looked at early approvals for 24 drugs and found only two other nods raising questions. They are Sarepta’s Exondis and Cobis Pharma’s preterm drug Makena. The OIG found that the FDA approved all three drugs despite concerns raised by its own reviewers and independent advisory committees.
