Today, the House Energy and Commerce Subcommittee on Health will be hosting a markup session on the comprehensive legislative package to reauthorize the human drug user fee programs, including the Prescription Drug User Fee Act (PDUFA VII) and the Biosimilar User Fee Act (BsUFA III). In February, PhRMA had the opportunity to testify before the subcommittee on how both programs have played, and will continue to play, a significant role in sustaining the U.S. Food and Drug Administration’s (FDA) ability to keep pace with the number of innovative drugs, biologics and biosimilars entering the regulatory review pipeline. In March, PhRMA had an additional opportunity to testify on potential legislative “riders” including those relating to clinical trial diversity, accelerated approval and rare disease drug development. The recent release of the legislative text and today’s subcommittee markup are important milestones in Congress’ review and approval of these critical programs.
PDUFA was first enacted in 1992 as a bipartisan solution to increase the efficiency and timeliness of new medicine review at the FDA in response to the AIDS epidemic, which left patients waiting for years for an under-staffed FDA to review and approve new treatments. To address this issue, the FDA, Congress and the biopharmaceutical industry worked together to establish the PDUFA program to help ensure the FDA was appropriately resourced to support the regulatory review process for new medicines. Under PDUFA, biopharmaceutical companies pay two different user fees to supplement Congressional appropriations. These fees are used to strengthen the agency’s ability to maintain high standards for patient safety and scientific regulatory review and make critical investments toward modernizing the drug evaluation process and improving staffing capabilities.
Prior to PDUFA’s first authorization in 1992, it often took the FDA more than two years to review applications for new medicines. Now, the median review time for a new medicine is 10 months for standard applications and 8 months for priority review applications. BsUFA was first enacted in 2012 to provide the FDA with the necessary resources and staffing specifically to support a biosimilar approval pathway and promote greater consistency, certainty and predictability in the review of biosimilar products. Since then, the FDA has approved 35 new biosimilars, including two interchangeable biosimilar products.
Through PDUFA and BsUFA, the United States leads the world in the introduction of novel medicines and the FDA’s human drug review program is the global gold standard for regulatory review and approval. In 2021, 76% of novel drugs were first approved in the United States before any other country.
Looking ahead, it is important to recognize that timely reauthorization of PDUFA and BsUFA before the September 30th deadline will be crucial for the continued success of the FDA’s human drug review program. The PDUFA VII goals letter expands upon the most recent iteration of the user fee agreements with a renewed focus on strengthening review fundamentals, enhancing accountability and transparency and advancing innovation for patients.
Specifically, PDUFA VII will target:
- 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 FDA’s reviewing, tracking and communicating of post-market safety information.
- Strengthening scientific dialogue and advancing innovation: Expand opportunities for sponsors to obtain the 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 the 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 incorporate 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.
Similarly, BsUFA III initiatives will build on the success of the program and help increase timely access to safe and effective biosimilar products. In helping provide FDA with the resources needed to enhance the development and review of biosimilars, BsUFA III will, in turn, help increase competition in the marketplace to the benefit of patients. Key provisions outlined in the BsUFA III Performance Goals Letter include:
- Advancing development of interchangeable biosimilar products, including additional guidance to sponsors, and piloting a regulatory science program with demonstration projects focused on advancing the development of interchangeable biosimilar products and improving the efficiency of biosimilar product development.
- Committing to timelines for review of certain application supplements, including those seeking to update safety labeling to reflect changes to the reference product labeling, to provide enhanced consistency and predictability of review timelines.
- Enhancing manufacturing inspection-related communications and modernizing facility assessment approaches.
- Modernizing FDA’s information technology infrastructure and supporting adoption of cloud-based technologies.
- Enabling timely interactions between sponsors and the FDA during biosimilar development and review, including the establishment of a new meeting type for rapid, targeted feedback.
As Congress makes efforts to move these reauthorization packages forward, it is important that the integrity of the reauthorization of these critical programs not be undermined by the inclusion of extraneous policies that would slow the reauthorization process and jeopardize FDA’s timely review and approval of critical new medicines.
For the United States to maintain its leadership in biopharmaceutical research and development and regulatory review, reauthorization of PDUFA and BsUFA is critical. New discoveries in drug development, including platforms for vaccines and cell and gene therapies, deliver the promise of treating debilitating diseases such as cancer, diabetes and many rare disorders. Fulfilling this promise depends on a modern regulatory framework supported by PDUFA and BsUFA that helps ensure patients have timely access to lifesaving medicines.
To learn more about PDUFA, visit PhRMA.org/PDUFA and to learn more about BSUFA, visit PhRMA.org/Biosimilars.