Weekly Policy Update: Accelerated Approvals Reform Bill Introduced

CBSA is committed to keeping our community updated on issues that impact life sciences. Our Policy + Advocacy team is reviewing newly proposed legislation that could directly affect life sciences companies. This educational piece provides an overview of the current bill. CBSA is reviewing the proposed bill now and will evaluate whether to take a formal position at the appropriate time, in consultation with our Policy Committee and advocacy partners at the federal level.

Earlier this week, the House Energy and Commerce Committee Chair Rep. Frank Pallone (D-NJ) introduced the Accelerated Approval Integrity Act. According to Rep. Pallone’s announcement, it 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.” This issue was also a recent topic of debate in the Senate during the confirmation hearing for FDA Commissioner Robert Califf, M.D earlier this year. Dr. Califf had remarked that the FDA must ensure drug makers granted accelerated approval conduct timely confirmatory trials to demonstrate the value of their medications.

In a press release announcing the introduction of the Act, Rep. Pallone stated that “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.” Proposed changes to the process are detailed below:

  • Codifying requirements for manufacturers to conduct post-approval studies on drugs that receive accelerated approval;
  • Requiring manufacturers to enter into an agreement with the 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 the 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 the FDA, an opportunity for public comment, and may include the 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.

The FDA’s Oncology Center of Excellence, which reviews the majority of accelerated approvals that are processed by the agency, began holding advisory committee meetings last spring on the issue. Earlier this year, the organization noted that half of all accelerated approval indications have confirmed benefit in a median of 3 years. The remainder of those that have not yet confirmed benefit have been those granted accelerated approval status in the last few years. Less than 10% of these indications have been withdrawn either due to failed trials or because the trials weren’t conducted.

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