RARE Daily

Bill Seeks to Tighten Rules Around Accelerated Approval

March 8, 2022

Energy and Commerce Committee Chairman Frank Pallone, Jr. has introduced legislation intended to ensure drugs that receive accelerated approval provide clinical benefits.

“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. “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. Patients deserve to know that the drugs they are taking are safe and effective.”

Among other things, the Accelerated Approval Integrity Act would streamline the process for taking products off the market if no clinical benefit is proven in a timely manner.

The U.S. Food and Drug Administration instituted the Accelerated Approval Program to allow for earlier approval of drugs that treat serious conditions and fill an unmet medical need based on a surrogate endpoint. A surrogate endpoint is a marker, such as a laboratory measurement, a radiographic image, physical sign, or other measure that is thought to predict clinical benefit, but is not itself a measure of clinical benefit.

The introduction of the legislation follows the controversial accelerated approval of Biogen’s Alzheimer’s drug Aduhelm. The drug won accelerated approval despite overwhelming consensus by an advisory committee that the drug lacked clinical evidence of efficacy to justify approval.

After receiving accelerated approval, manufacturers are required to conduct studies to confirm the anticipated clinical benefit. If the study does not show that the drug provides clinical benefit, FDA has procedures in place that could lead to removing the drug from the market. Many critics have argued the existing tools are cumbersome, resource-intensive, and seldom used.

The Accelerated Approval Integrity Act would give the FDA additional authority to ensure products that receive accelerated approval are providing a clinical benefit to patients in a timely manner. It would codify requirements for manufacturers to conduct post-approval studies on drugs that receive accelerated approval, require manufacturers to enter into an agreement with FDA on how the studies will be conducted before the agency can grant accelerated approval, and allow the FDA to require studies to be underway at the time of approval.

In addition to outlining expedited procedures for withdrawing approval, the bill specifies additional instances in which an accelerated approval can be withdrawn including if a manufacturer fails to achieve agreed upon enrollment targets, milestones, or timely study completion. It would also automatically expire accelerated approval status one year after post-approval studies are scheduled to be complete, and in no case later than five years after approval, unless the post marketing study has been completed and has verified the clinical benefit, or the Secretary of Health and Human Services determines that adequate progress has been made. In that case, those studies can continue.

Author: Rare Daily Staff

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