The Accelerated Approval Integrity Act Improves FDA’s Ability to Ensure Accelerated Approval Products Provide a Clinical Benefit to Patients
Energy and Commerce Committee Chairman Frank Pallone, Jr. (D-NJ) today introduced the Accelerated Approval Integrity Act, which would improve the Food and Drug Administration’s (FDA) ability to ensure that drugs that receive accelerated approval are providing a proven clinical benefit to patients.
“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. I’m introducing the Accelerated Approval Integrity Act to protect patients and provide FDA with the authority it needs to ensure approved drugs provide a clinical benefit. The bill would also streamline the process for taking products off the market if no clinical benefit is proven in a timely manner.”
FDA 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. 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. However, as multiple witnesses at a February Health Subcommittee hearing testified, these existing tools are cumbersome, resource-intensive, and seldom used.
The Accelerated Approval Integrity Act will provide FDA with additional authority to ensure products that receive accelerated approval are providing a clinical benefit to patients in a timely manner, including:
- Codifying requirements for manufacturers to conduct post-approval studies on drugs that receive accelerated approval;
- Requiring manufacturers to enter into an agreement with FDA on how the studies will be conducted before the agency can grant accelerated approval, which may include details like enrollment targets, milestones, and study design;
- Allowing FDA to require studies to be underway at the time of approval;
- Requiring more frequent updates on post-approval studies, including updates on enrollment targets, milestones, and study design;
- Outlining expedited procedures for withdrawing approval, which would include due notice and opportunity for a written appeal to FDA, an opportunity for public comment, and may include FDA convening and consulting an advisory committee;
- Specifying 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;
- Automatically expiring 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 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;
- Requiring additional information on accelerated approval drugs’ labels; and
- Making failure to submit reports or act with due diligence on post-approval studies prohibited acts subject to penalties.
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