ASGCT Submits Comments in Response to FDA’s PDUFA VIII Reauthorization Public Meeting

ASGCT recently submitted comments to FDA’s docket on Reauthorization of the Prescription Drug User Fee Act (PDUFA) following the public meeting that kicked off the process. PDUFA is the law that authorizes FDA to collect user fees from companies, which support FDA operations alongside congressional appropriations. In return, FDA agrees to specific commitments like review timeline goals, guidance documents to issue, and new regulatory pilot programs. The current PDUFA VII agreement, which expires September 2027, has enabled significant progress in the Agency’s review capacity for cell and gene therapy (CGT) products. Looking to PDUFA VIII reauthorization for fiscal years 2028-2032, ASGCT’s comments reiterated that FDA’s review capacity needs to be further strengthened and encouraged FDA to modernize regulatory frameworks that support patient access to transformative therapies.  

Specifically, ASGCT advocated for continued investment in the Center for Biologics Evaluation and Research (CBER) Office of Therapeutic Products (OTP) to support CGT reviews - emphasizing meeting infrastructure, regulatory science, and policy development. PDUFA VII has meaningfully improved the review process at CBER regarding timeliness and consistency of regulatory reviews. New meeting types such as Type D and INitial Targeted Engagement for Regulatory Advice on CBER/CDER ProducTs (INTERACT) provide earlier and more targeted feedback, helping sponsors navigate novel scientific and regulatory questions. 

Additionally, ASGCT believes that timely, science-based guidance benefits patients and innovators alike by providing clear regulatory expectations; in particular, updating guidance on Chemistry, Manufacturing, and Controls (CMC) and Good Manufacturing Practices (GMPs) for platform technologies is a high priority for the field.   

Manufacturing CGT products is a well-known bottleneck to patient access. In PDUFA VII, FDA focused on modernized manufacturing methods and innovative technologies, allowing the field to pursue streamlined development and reduce redundancy in CMC processes. ASGCT’s comments encouraged FDA to prioritize and build upon these areas in PDUFA VIII. The Society also recommended PDUFA VIII recognize and provide additional resources for FDA’s new platform and advanced manufacturing technology (AMT) designations.  

ASGCT expressed support for the continuance and expansion of several pilot programs created under PDUFA VII: 

• The Split Real-Time Application Review (STAR) pilot was modeled after the Oncology Center of Excellence’s Real-Time Oncology Review (RTOR) program with the goal to improve review efficiency. ASGCT suggested the pilot be further evaluated for application beyond efficacy supplements.  

• The Rare Disease Endpoint Advancement (RDEA) pilot facilitates the identification and creation of novel clinical endpoints for rare diseases.  The Society’s comments recommended that FDA explore opportunities to improve visibility of the pilot and attract applications.  

• The CMC Development Readiness Pilot (CDRP) is a valuable tool to enable earlier discussions of manufacturing strategies in accelerated development contexts. ASGCT supports making this program permanent and leveraging it to modernize CMC expectations.  

Finally, ASGCT requested FDA consider opportunities to deepen their collaboration with international regulatory agencies such as the European Medicines Agency (EMA), UK Medicines and Healthcare products Regulatory Agency (MHRA), Health Canada, and Japanese Pharmaceuticals and Medical Devices Agency (PMDA). Exploring mutual recognition agreements could reduce the duplicative requirements between jurisdictions, accelerate global processes, and ease regulatory burden without compromising safety or quality.  

ASGCT commends FDA leadership for implementing commitments under PDUFA VII, and the Society will continue to support FDA’s ongoing efforts to modernize the regulatory framework for CGTs. 

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