Introduction
The FDA’s draft guidance Expedited Program for Serious Conditions—Accelerated Approval of Drugs and Biologics proposes an accelerated approval pathway, which would expedite access to promising treatments, particularly for serious conditions with unmet medical needs. However, in some instances, the FDA may propose to withdraw a product’s approval under this pathway if data do not sufficiently support the clinical benefit or if other safety and effectiveness concerns arise. The process for challenging such a proposal through an appeal is well-defined and offers sponsors a structured opportunity to present their case.
Here’s an overview of the key steps in the proposed accelerated approval withdrawal process:
Submitting a Written Appeal
The sponsor can submit a written appeal if the FDA proposes withdrawing approval for a product under accelerated approval. This appeal should present the sponsor’s objections to the withdrawal proposal and may include new data, information, or evidence supporting the case. Any data or analyses referenced must be directly related to the discussions or materials presented in advisory committee meetings unless they were not available or foreseeable at the time. If a sponsor provides additional data, they must explain why they did not include it earlier and why it is essential for the appeal.
Center’s Response to the Appeal
Once the appeal is submitted, the Center has an opportunity to respond. The Commissioner or designee may request that the Center specifically address certain aspects of the appeal. This request ensures the decision is made based on the most comprehensive understanding of all available evidence.
Scheduling a Meeting with the Commissioner/Designee
Following the submission of the written appeal and any responses from the Center, the sponsor may request a meeting with the Commissioner or designee. This meeting is not a decisional one, but it provides the sponsor with a platform to present their case in person.
Consulting an Advisory Committee
If the sponsor requests it, the FDA may convene an advisory committee to assess whether withdrawal criteria are met. However, a second meeting is generally unnecessary if an advisory committee has already evaluated the issue. This ensures the process avoids unnecessary delays while allowing for an independent, expert review when needed.
Final Decision by the Commissioner/Designee
After reviewing the appeal, any responses, and the results of any advisory committee consultations, the Commissioner or designee will make a final decision. The Commissioner or designee will decide based on the evidence and public comments, and the rationale behind the withdrawal or retention of approval will be outlined. If the decision is to withdraw approval, the FDA will also provide a summary of public comments and any other relevant responses.
Communication and Implementation
The FDA will publish the final decision on its website and take the necessary steps to implement it. If the sponsor waived their right to appeal, the FDA could finalize the withdrawal decision more swiftly. Regardless, the goal is to maintain transparency throughout the process, allowing the public to understand the reasons behind the decision.
Conclusion
The proposed process for appealing a proposal to withdraw accelerated approval aims to balance fairness for sponsors with the FDA’s responsibility to ensure that only safe and effective products remain available to patients. The FDA is looking to ensure they withdraw approvals with the utmost transparency and accountability by allowing for comprehensive appeals, expert consultations, and public input.
