Without confirmation of a drug’s benefit, “I can’t look my patient in the eye and say I think this drug is going to help you live longer,” said Sekeres, who is chair of the American Society of Hematology’s communications committee and a former chair of the FDA’s oncology advisory committee.

The National Organization for Rare Diseases, which is generally supportive of the accelerated approval program, views the current controversy as jeopardizing access, particularly when insurers push back as they have with Aduhelm. In Oregon, meanwhile, the state’s Medicaid program has requested permission to deny coverage for accelerated approval products in some circumstances.

“I think that there’s a lot that can be done with post-market confirmatory trials and transparency around them,” said Heidi Ross, NORD’s vice president of policy and regulatory affairs.

The group is recommending more frequent reporting from drugmakers and for the FDA to make these records publicly accessible, Ross said.

The agency could have new tools to prevent “dangling” approvals beginning in October, if Congress votes to include accelerated approval reform in the PDUFA reauthorization legislation, which is renewed every five years.

The Pharmaceutical Research and Manufacturers of America, a powerful industry lobby, argues changes aren’t necessary.

“America’s biopharmaceutical research companies are committed to supplying patients with safe, effective and high-quality innovative therapies and accelerated approvals must meet the same standards of safety and efficacy for approval as all other medicines,” a spokesperson wrote in an email.

The House of Representatives draft bill, which would serve as a PDUFA reauthorization document, would allow the FDA to require drugmakers begin confirmatory trials before an accelerated approval is granted. The agency would also gain strengthened authority to have a drug withdrawn from the market if a company doesn’t follow through or if the trial fails.

The bill doesn’t take more drastic steps included in legislation authored by the Energy and Commerce Committee’s chairperson, Frank Pallone, D-N.J., that would have made accelerated approvals automatically expire after five years. Pallone’s position as the committee’s chair could mean the idea remains a point of negotiation, however.

To Michael Carome, director of non-profit consumer advocacy organization Public Citizen’s Health Research Group, a firm deadline is necessary. “Often these studies are slow getting off the ground [and] they miss their planned deadlines for completion, if they ever get completed at all,” he said.

Other groups aren’t as supportive. The American Society of Clinical Oncology, for instance, is opposed, as cancer drug trials are typically based on events like disease progression or death rather than how long a patient is on treatment. “I wouldn’t like it to be based on when a study is scheduled [to report],” said Julie Gralow, ASCO’s chief medical officer.

The PDUFA reauthorization draft would also clarify the FDA’s process for withdrawing an accelerated approval based on negative clinical data or poor trial execution, including specifying how the agency should notify drugmakers, open a public comment period and give companies an opportunity to appeal.

Strengthened powers to order withdrawal of drugs could be an important step forward, Carome said. He noted the example of Makena, a drug designed to prevent premature birth that won accelerated approval in 2011. While a confirmatory trial failed to prove it works, the medicine remains on the market three years later.