Sept. 10, 2021 – The Food and Drug Administration (FDA) recently issued a commitment letter regarding the next iteration of the Prescription Drug User Fee Act (PDUFA VII) that spells out its review timeframes, performance goals and modernization priorities for fiscal years (FYs) 2023-2027. Among these priorities are enhancements related to expediting drug development, expanding decision tools to support drug development and review, modernizing the drug safety system, leveraging innovative technologies, and increasing the Center for Biologics Evaluation and Research’s (CBER’s) capacity to review and approve cell and gene therapy products.

The expedited drug development section of the document covers several hot-button issues for the agency:

• Communication between the FDA and sponsors during drug development;
• Continued support of breakthrough therapies;
• Early consults on the use of new surrogate endpoints;
• Advanced development of drugs for rare diseases;
• Advanced development of combination products; and
• Advanced use of real-world evidence in regulatory decision-making.

“As with past PDUFA rounds, PDUFA VII will represent a strategic document as much as an outline of user fees,” said Coalition for Healthcare Communication Executive Director Jon Bigelow. “The emphasis in this round on modernizing infrastructure technology, refining relevant clinical endpoints for drugs for rare diseases, and making better use of real-world evidence is right in line with the priorities of Acting Commissioner Janet Woodcock and should be positive for the biopharma industry.”

The agency has developed two proposed pilot programs to address the unique issues posed by therapies for rare diseases. One pilot program, “Split Real Time Application Review (STAR),” has the stated goal of “shortening the time from the date of a complete submission to the action date, in order to allow earlier patient access to therapies that address an unmet medical need.” The STAR pilot program will apply to efficacy supplements across all therapeutic areas and review disciplines that meet specific criteria. Accepted STAR applications will be submitted in a “split” two-part fashion (with the components submitted approximately two months apart), the document states.

The “Rare Disease Endpoint Advancement (RDEA) Pilot Program” will address the lack of regulatory precedent, small trial populations, and/or limited understanding of disease natural history associated with rare diseases and which pose unique challenges when determining the appropriate efficacy endpoint(s) for clinical trials intended to evaluate the effectiveness of rare disease therapies.

The RDEA pilot program seeks to advance rare disease drug development programs by providing a mechanism for sponsors to collaborate with the FDA throughout the efficacy endpoint development process. An endpoint, or endpoints, will be considered eligible for proposal submission to RDEA if certain criteria are met.

The FDA also has committed to improving FDA hiring and retention of review staff and raising its goalposts for using technology and bioinformatics to increase transparency and support drug development and review, according to the document, which “represents the product of FDA’s discussions with the regulated industry and public stakeholders, as mandated by Congress.”

A virtual public meeting to discuss proposed recommendations for the reauthorization of PDUFA VII will be held Sept. 28.