US accelerated drug approval process needs reform, report shows

The US accelerated drug approval process helps provide faster access to innovative therapies but suffers from inconsistency and a lack of transparency, according to a new report by the Institute for Clinical and Economic Review (ICER). This was reported by Reuters.

According to the analysis, patients, manufacturers, and payers are often “disappointed” by the weaknesses of the program, which allows drugs for serious conditions to be approved more quickly based on intermediate or surrogate endpoints, provided that additional studies are subsequently conducted to confirm efficacy and safety.

“The goal of the accelerated pathway has always been for patients to receive innovative treatments faster,” said Sarah Emond, President and CEO of ICER.
“We can point to a number of success stories where patients have benefited from faster access to transformative therapies,” she added.

Success stories include Novartis’ chronic myeloid leukemia drug Gleevec (imatinib), Merck & Co.’s cancer immunotherapy Keytruda (pembrolizumab), and antiretroviral therapies for HIV.

At the same time, some accelerated approvals have sparked serious controversy. These include Biogen and Eisai’s Alzheimer’s drug Aduhelm, which was withdrawn from the market due to doubts about its effectiveness, and Sarepta Therapeutics’ gene therapy Elevidys for Duchenne muscular dystrophy, whose use was restricted due to the risk of liver toxicity.

The report proposes a number of measures for improvement, including:

• stricter selection of the surrogate endpoints used
• wider application of randomized clinical trials
• introduction of special warnings on the labels of such drugs
• stricter oversight for the conduct of mandatory confirmatory studies

“There are many instances of regulatory inconsistency, delayed confirmatory data, and access restrictions that limit benefits to patients,” Emond emphasized.
“The proposed policies aim to build on the program’s successes and address remaining challenges so that patients have access to affordable and effective therapies.”

The report does not address the Food and Drug Administration’s new priority review scheme, which allows for decisions within weeks but is already drawing criticism for a lack of transparency and potential political influence on drug selection.

The analysis also comes amid new FDA regulatory ideas for personalized therapies that could be approved based on a “plausible mechanism,” even with a limited number of patients—an approach that is also sparking debate within the medical community.