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House Bill Strengthens FDA’s Power Over Fast Drug Approvals

March 7, 2022, 10:06 PM

The FDA could more easily pull drugs that show no clinical benefit off the market under a bill that aims to revamp the approval process used for Biogen Inc.’s Alzheimer’s drug.

The measure, introduced Monday by House Energy and Commerce Committee Chairman Frank Pallone Jr. (D-N.J.) would make changes to the accelerated approval process, a Food and Drug Administration regulatory mechanism designed to speed up access to game-changing medications for patients with unmet medical needs.

The FDA has faced criticism that it’s speeding up approvals without certainty that the drug will show a definitive clinical benefit down the line—particularly after it approved Biogen’s Aduhelm against the recommendation of its scientific advisers. Under Pallone’s bill, accelerated approval would automatically expire after a certain period of time for companies that haven’t met postmarket clinical benefit requirements, with exceptions.

“The Accelerated Approval Program allows patients to access innovative new drugs while the manufacturer and FDA conduct additional studies to confirm the product’s clinical benefit,” Pallone said in a statement.

“However, under the current system, some products have been allowed to stay on the market for far too long without clinical trials that demonstrate a real clinical benefit for patients,” he said. “Patients deserve to know that the drugs they are taking are safe and effective.”

The bill comes as the House Energy and Commerce Committee weighs how to renew the Prescription Drug User Fee Act, must-pass legislation that allows the FDA to collect fees from brand-name drugmakers to help fund agency operations. The committee is expected to announce a notice for a second PDUFA hearing later this week, a House aide said.

Lawmakers could decide to tack on provisions of Pallone’s bill to PDUFA, which Congress must pass by Sept. 30. An aide for Pallone said he would like to see improvements to the accelerated approval program this year.

Postmarket Studies

Accelerated approval allows the FDA to approve drugs under what’s known as surrogate endpoints, which are laboratory measurements that can stand in for a clinical endpoint—a direct measurement of whether a patient feels or functions better or lives longer. For example, tumor shrinkage is a surrogate endpoint for survival, which usually requires waiting five years to see if the drug worked.

Clinical endpoints offer a stronger indicator of the drug’s benefit than surrogate endpoints but can take much longer to demonstrate. Companies using surrogate endpoints are supposed to conduct follow-up studies to confirm whether the drug offers a clinical benefit. But a British Medical Journal investigation found nearly half the drugs approved under accelerated approval never had a confirmatory trial done.

Pallone’s bill aims to beef up the FDA’s authority over requiring postmarket studies.

Drugmakers and the FDA would have to agree how to conduct postmarket studies before the agency could grant an accelerated approval under the measure. It also outlines expedited procedures for withdrawing approval and allows the FDA to pull approval if the drug company fails to complete its studies in a timely manner or meet its agreed-upon enrollment targets and milestones.

Accelerated approval status would expire automatically one year after studies demonstrating clinical benefit are scheduled to be done, or five years after the FDA grants accelerated approval, unless the drug company has met its post-approval requirements. But the Health and Human Services secretary would be able to intervene and determine that adequate progress has been made to keep the drug on the market.

—With assistance from Alex Ruoff

To contact the reporter on this story: Jeannie Baumann in Washington at jbaumann@bloombergindustry.com

To contact the editors responsible for this story: Alexis Kramer at akramer@bloomberglaw.com; Karl Hardy at khardy@bloomberglaw.com