As FDA Readies for PDUFA VII, Rare Disease, Cell and Gene Therapies in the Mix

The Food and Drug Administration said it will recommend the creation of a pilot program to support the development of efficacy endpoints in rare disease clinical trials as part of a list proposals the agency will make for the reauthorization of the Prescription Drug User Fee Act.

The set of proposals relating to PDUFA VII came in a listing in the Federal Register announcing a virtual public meeting to discuss recommendations for the legislation.

PDUFA authorizes the FDA to collect user fees from industry to support the review of applications for new drugs and biologics in a timely manner. As part of FDA’s negotiated agreement with industry during each reauthorization, the agency agrees to certain performance and procedural goals and other commitments that apply to aspects of the human drug review program.

The current legislative authority for PDUFA expires in September 2022. At that time, new legislation will be required for FDA to continue collecting prescription drug user fees in future fiscal years. The first PDUFA legislation passed in 1992. The reauthorization of PDUFA the agency is working on will be the seventh incarnation of the legislation.

The proposed recommendations to Congress for PDUFA reauthorization follow a series of discussions between the FDA, industry, and stakeholders.

As part of the recommendations, the FDA is proposing a pilot program for supporting efficacy endpoint development for drugs that treat rare diseases by offering additional engagement opportunities with the agency to sponsors of development programs that meet specific criteria. The agency said that lack of regulatory precedent, small trial populations, and limited understanding of natural history associated with rare diseases creates unique challenges when determining the appropriate efficacy endpoints for clinical trials intended to evaluate the effectiveness of rare disease therapies.

The FDA also is proposing a new pilot program around real-world evidence to improve the quality and acceptability of real-world evidence-based approaches in support of new intended labeling claims, including approval of new indications of approved medical products or to satisfy post-approval study requirements.

And the agency said to ensure new cell and gene therapy products are developed and available to patients in a timely manner, it is proposing to strengthen staff capacity and capability to meet the increasing challenges and demands in this area. It said increasing staff capacity will overcome existing resource limitations, allowing staff to spend additional time on meetings and submission reviews including those with breakthrough or regenerative medicine advanced therapy designations, expand stakeholder outreach, invest in new policy and guidance, and facilitate development and use of regulatory tools and scientific technologies.

The public meeting will be held on September 28, 2021, from 9 a.m. to 2 p.m. Eastern Time, and will be held by webcast only. Submit either electronic or written comments on this public meeting by October 28, 2021. Registration to attend the virtual meeting and other information can be found at https://pdufavii-reauthorization.eventbrite.com. Electronic comments must be submitted on or before October 28, 2021. The https://www.regulations.gov electronic filing system will accept comments until 11:59 p.m. Eastern Time at the end of October 28, 2021.

Author: Rare Daily Staff