The importance of timely PDUFA and BsUFA reauthorization

Today, PhRMA, along with the U.S. Food and Drug Administration (FDA) and other industry trade associations, will be participating in a congressional hearing with the House Committee on Energy and Commerce Subcommittee on Health discussing the user fee programs which are under consideration by policymakers for reauthorization this year. This follows the U.S. Department of Health and Human Services’ transmittal of the proposed Prescription Drug User Fee Act (PDUFA) VII and Biosimilar User Fee Act (BsUFA) III packages to Congress last month. The beginning of the legislative phase marks an important milestone in the reauthorization process of these two user fee programs.

For our part, we will be speaking to PDUFA VII and BsUFA III. Both programs play an important role in allowing the FDA to keep pace with the number of innovative drugs, biologics and biosimilars entering the regulatory review pipeline. PDUFA and BsUFA must be reauthorized before they expire on September 30^th this year to ensure continuity in the human drug review program and that the new enhancements to FDA’s regulatory review capabilities as outlined in the user fee agreements are implemented as planned and protect against any disruptions to these critical programs.

PDUFA’s timely reauthorization is critical for continued success of the human drug review program

PDUFA was first enacted in 1992 as a bipartisan solution to increase the efficiency and timeliness of new medicine review at 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, 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.

PDUFA allows the Agency to collect fees from biopharmaceutical companies to augment Congressional appropriations. These funds help to increase FDA’s staffing and strengthen the Agency’s ability to independently review human drug applications. Besides helping to ensure patients have timely access to innovative and lifesaving treatments, the PDUFA program provides biopharmaceutical companies with greater regulatory predictability, which fosters industry investment in research and development of new therapies.

In large part because of PDUFA and the biopharmaceutical ecosystem it fosters, the U.S. 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. 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. Since PDUFA’s passage, the FDA has provided timely patient access to more than 1,700 new medicines and biologics including treatments for cancer, rare diseases, cardiovascular, neurological and infectious diseases.

The PDUFA VII Performance Goals Letter expands upon the most recent iteration of the user fee agreement with a renewed focus on strengthening review fundamentals, enhancing accountability and transparency and advancing innovation for patients. In August of last year, the FDA held a public stakeholder meeting to discuss the proposed PDUFA VII Performance Goals Letter. The overarching PDUFA VII efforts are aimed at modernizing the U.S. regulatory and medicine development paradigm and addressing new areas such as advancing digital health technologies, supporting the next wave of advanced biological therapies such as cell and gene therapies and enhancing innovation in manufacturing and drug quality review.

PDUFA VII also includes commitments that advance COVID-19 lessons learned and enhance our future preparedness and ability to keep pace with scientific advancements, including novel approaches to facility inspections and the broader use of digital technologies to enable conduct of remote or decentralized clinical trials. Importantly, such trials can help enable more diverse patient populations to participate in clinical research.

BsUFA’s timely reauthorization is critical for continued development of biosimilar and interchangeable products

BsUFA was first enacted in 2012, to help provide FDA with resources and staffing specifically to support the biosimilar approval pathway and promote greater consistency, certainty and predictability in the review of biosimilar and interchangeable biosimilar products. BsUFA has resulted in 33 FDA-approved biosimilar products to date, including two interchangeable biosimilars.

The proposed BsUFA III initiatives will build on the success of the program and help increase timely access to safe and effective biosimilar and interchangeable 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.

America’s biopharmaceutical research and development ecosystem is the best in the world. It is imperative that FDA remains equipped to help us deliver new treatments and cures to meet patients. PDUFA VII and BsUFA III will help ensure that patients have timely access to lifesaving medicines, while maintaining the United States' global leadership in biopharmaceutical innovation.

It is critical that Congress reauthorizes the PDUFA and BsUFA programs before their expiration later this year. PhRMA looks forward to working with Congress, the administration, the FDA and other stakeholders to ensure the timely reauthorization of the user fee programs.

If you would like to learn more about PDUFA, click here and learn more on BsUFA here. To read the PhRMA testimony, visit here.