RE:RE:RE:RE:RE:RE:RE:RE:RE:RE:RE:Type C Meeting TimingsRe:
Considerations for Discussion of a New Surrogate Endpoint(s) at a Type C PDUFA Meeting Request" - Prescription Drug User Fee Act (PDUFA) was enacted in 1992
- Leading up to PDUFA reauthorization, FDA and stakeholders negotiate goals
- PDUFA outlines the “user fees” drug sponsors must pay for FDA activities related to prescription drug reviews
- FDA agrees to user fee “goals” for review timelines and additional goals or programs (e.g., patient-focused drug development meetings)
Accelerated Approval/Subpart H –
Unlike Fast Track, Priority Review, Breakthrough, and RMAT which reduce FDA’s review time, Accelerated Approval shortens the Sponsor’s clinical development time – For serious conditions with a meaningful advantage over available therapy – Permits the use of a “surrogate endpoint” that is likely to predict clinical benefit (e.g., viral load, tumor shrinkage) – Sponsors required to conduct post-marketing studies to confirm benefit (i.e., confirmatory trials) – FDCA § 506(c); 21 CFR 314.500 3
Type C meeting for discussion of novel surrogate endpoints
https://www.fda.gov/drugs/development-resources/surrogate-endpoint-resources-drug-and-biologic-development
Ongoing discussions between FDA and sponsors are an important part of any drug development program. For a new biomarker intended as a surrogate endpoint, early consultation gives the division an opportunity to provide input and guidance at the beginning of the process of biomarker development, and to work collaboratively with the sponsor over the course of development.
Based on our PDUFA VI Commitment letter.4 , we intend to utilize the type C meeting process to engage with sponsors who would like to employ a biomarker as a surrogate endpoint that has not been used previously as the primary basis for product approval in the proposed context of use. This meeting will apply to new surrogate endpoints in support of either accelerated or traditional approval.
The purpose of this meeting is to discuss the feasibility of the surrogate as a primary efficacy endpoint, identify any gaps in knowledge that may exist, and discuss how those gaps could be addressed. Depending on the level of evidence provided, the outcome of this meeting may be that the sponsor needs to conduct further investigations to address issues raised by the Agency before the surrogate endpoint can be used as the primary basis for product approval.
This meeting is expected to occur when the sponsor has preliminary clinical study results that show that the proposed biomarker responds to the candidate drug at doses that appear to be generally tolerable. To qualify for this consultation, requests for this Type C meeting should be accompanied by the complete meeting background package.4