Today, the U.S. Food and Drug Administration (FDA or Agency) released its performance goals letter for the seventh installment of the Prescription Drug User Fee Act (PDUFA).
PDUFA was first enacted in 1992 as a bipartisan solution to the then long FDA regulatory review timelines for new drugs and biologics. To this end, PDUFA allows the Agency to collect fees from biopharmaceutical companies to augment Congressional appropriations to increase FDA’s staffing and other resources to speed and enhance review process to help ensure patients have timely access to life-saving treatments. The PDUFA program helps provide efficiency and predictability of regulatory review while supporting continued independent rigor by the science-based Agency.
The PDUFA program has been critical in supporting the FDA’s ability to fulfill its central mission – to help protect and advance the public health. Thanks in part to PDUFA, the U.S. now leads the world in delivering new medicines to patients, and the FDA’s human drug review program is the global gold standard for regulatory review and approval. Before PDUFA, it often took FDA more than two years to review new medicines. Now, the median review time for a new medicine is 10 months for standard applications and 8 months for priority review applications.
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.
In adapting to and meeting the challenges presented by the COVID-19 pandemic, FDA and the biopharmaceutical industry are utilizing novel approaches to clinical trials and facility inspections to support continued innovation and inform efficient regulatory decision-making. PDUFA VII includes commitments that advance COVID-19 lessons learned, such as increased use of digital technologies and alternative tools to assess manufacturing facilities.
The PDUFA VII agreement will have a lasting and meaningful impact on the biopharmaceutical industry’s ability to develop innovative, safe and effective medicines for patients in a timely manner.
The timely reauthorization of PDUFA will allow FDA and biopharmaceutical companies to build on the incredible strides made during the previous PDUFA cycle. New developments in medical and fundamental science – including immunotherapies and cell and gene therapies – hold the promise of treating debilitating diseases such as cancer, diabetes and many rare disorders. Fulfilling this promise depends on a modern regulatory framework that PDUFA facilitates and that can serve patients by providing timely, science-based regulatory decisions. It is critical that Congress reauthorizes PDUFA, and supports the independent work of FDA, before its expiration in September 2022.
To learn more about PDUFA VII, click here.
Richard Moscicki, M.D. Dr. Moscicki serves as executive vice president, Science and Regulatory Advocacy and chief medical officer at PhRMA. He joined the organization in 2017 after serving as the Deputy Center Director for Science Operations for the U.S. Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) since 2013. While at FDA, Dr. Moscicki brought executive direction of Center operations and leadership in overseeing the development, implementation, and direction of CDER’s programs. Previous positions include serving as Chief Medical Officer at Genzyme Corporation from 1992 to 2011, where he was responsible for worldwide global regulatory and pharmacovigilance matters, as well as all aspects of clinical research and medical affairs for the company. He served as the senior vice president and head of Clinical Development at Sanofi-Genzyme from 2011-2013.