PDUFA has helped the FDA fulfill its central mission – to help protect and advance the public health – by allowing the Agency to keep pace with the number and complexity of innovative drugs and biologics entering the review pipeline. In large part because of PDUFA, the United States now leads the world in the introduction of new medicines, and the FDA’s human drug review program is the global gold standard for regulatory review and approval. Since 1992 when the program was enacted, PDFUA has provided more timely access to more than 1,700 new drugs and biologics including treatments for cancer, rare diseases, cardiovascular, neurological and infectious diseases.
PDUFA continues to play a critical role in strengthening the FDA’s ability to review human drug applications. The program also provides biopharmaceutical companies with greater regulatory predictability, which fosters industry investment in research and development. At each five-year reauthorization of PDUFA, FDA and the biopharmaceutical industry have the opportunity to advance initiatives that further support innovation and enhance the regulatory review process.
The PDUFA VII goals letter expands upon the most recent iteration of the user fee agreements which expire in September of 2022 with a renewed focus on strengthening review fundamentals, enhancing accountability and transparency and advancing innovation for patients.
Key areas of the PDUFA VII goals letter include:
- Enhancing patient-centric drug review and safety monitoring: Advance the incorporation of patient-centric data, including patient preference information, in drug development and regulatory reviews and support the Agency’s reviewing, tracking and communicating of post-market safety information.
- Strengthening scientific dialogue and advancing innovation: Expand opportunities for sponsors to obtain FDA’s regulatory feedback and clarity throughout the drug development process.
- Supporting the next wave of advanced biological therapies: Strengthen the Agency’s staff capacity and capability to support the development and review of cell and gene therapies.
- Modernizing regulatory evidence generation and drug development tools: Advance the use of real-world evidence in regulatory decision making, facilitate further use of complex adaptive and other novel clinical trial designs, and advance consistency and predictability around the use of modeling and simulations.
- Advancing digital technologies and information technology (IT) infrastructure: Facilitate adoption of innovative digital health technologies and modernize FDA’s data and IT capacity and capabilities, including adoption of cloud-based technologies.
- Enhancing innovation in manufacturing and product quality reviews: Facilitate the use of innovative manufacturing technologies for both products in development and those commercially available and incorporates best practices from COVID-19 lessons learned for the use of alternative tools to assess manufacturing facilities.
- Enhancing FDA hiring, retention and financial management: Build upon PDUFA VI efforts to improve accountability and transparency and modernize financial and staff resource management.
PhRMA looks forward to working with Congress, the administration, patient and medical provider groups, the FDA and other stakeholders to ensure timely reauthorization of this important program as we continue working together to help patients live longer, healthier lives.
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