In the span of a single year, Biogen went from winning accelerated approval for its Alzheimer’s drug, Aduhelm, to all but abandoning the controversial medicine. Now, Congress is weighing changes to the FDA program that triggered the entire episode.
The proposals are designed to give the FDA more control over the accelerated approval program, The Wall Street Journal reports. The approval pathway is designed to speed up therapies for patients with few or no other options, but drugs approved under the program have to go through a postmarketing trial to confirm its benefits.
The agency would have more say in confirmatory trials—those that are designed to confirm a drug works—and to revoke marketing licenses for ineffective medicines, the newspaper says. Specifically, the agency would be able to require that confirmatory trials are underway before granting accelerated approvals, and it would have more say in how those studies are conducted.
For drugs that don’t prove their merit, the FDA wouldn’t need to complete as many steps to pull them from the market, WSJ adds. Instead of revoking approvals itself, the agency has recently put the onus on drugmakers to do so.
The House of Representatives this week passed a bill containing the reform proposals, WSJ reports.
Accelerated approval reform has taken center stage in the wake of the Aduhelm debacle. Last June, the agency approved the drug based on the surrogate endpoint of reducing amyloid beta plaque in the brain. At the time, the agency said a positive effect on the endpoint was “reasonably likely to predict a clinical benefit to patients.”
It's safe to say not everyone was convinced. A controversy ensued, and in a high-profile decision earlier this year, the Centers for Medicare & Medicaid Services restricted coverage to patients in approved clinical trials. The decision amounted to a severe throttling of the rollout.
Pricing is another topic at hand with accelerated approvals. Critics have said the program allows pharmaceutical companies to price their unproven products at will before demonstrating efficacy. Since confirmatory trials can take years to complete, companies can rake in serious sales on unproven drugs, critics have argued.
But proponents of the program say it allows promising treatments to reach patients earlier than would be possible under traditional approval pathways.