America relies on the U.S. Food & Drug Administration (FDA) to keep pace with the scientific advancements happening every day in labs across the country. That’s why the recently enacted Prescription Drug User Fee Act (PDUFA VII) and Biosimilar User Fee Act (BsUFA III) are both great news for biopharmaceutical innovation and for patients. With these programs reauthorized for another five years, the FDA can continue doing its vital work reviewing medicines.
First enacted in the 1990s, PDUFA has supplemented funding for the FDA, so it has sufficient staff and resources to review new medicines. The program has made a real difference for patients. It used to take the FDA more than two years to review new medicines, meaning most were launched outside of the United States before they received regulatory approval here. For example, during the HIV/AIDS epidemic, patients were left waiting years for an under-staffed FDA to review and approve new treatments for the disease. But last year, approximately 76% of new medicines were first approved in the United States thanks in part to the program, which helps provide timely patient access to safe and effective medicines.
PDUFA VII will ensure the FDA’s staffing, resourcing and infrastructure are robust and able to support efficient drug review and approval. The PDUFA VII goals letter was negotiated among the FDA and industry stakeholders with input from patient advocacy groups, and it expands upon the previous iteration with a renewed focus on strengthening FDA’s critical capabilities, improving efficiencies in drug review and driving innovation for patients. PDUFA VII addresses new areas such as advancing digital health technologies, enhancing product quality reviews and facilitating increased utilization of innovative manufacturing technologies.
The reauthorization of PDUFA also notably advances the next wave of biological therapies such as cell and gene therapies by substantially strengthening FDA staff capacity and capability to address growing workload. As we start to see the promise of real-world evidence (RWE), PDUFA VII will establish a new pilot program to advance the use of RWE for regulatory decision-making, including for approval of new indications or to satisfy postmarketing study requirements. The agency will also be holding public workshops on RWE and issuing additional guidance documents. Further, PDUFA VII will continue to incorporate patient voice in drug development and use of patient preference information to support regulatory decision making.
Similarly, since it was first enacted in 2012, BsUFA has provided the FDA with the necessary resources and staffing to review biosimilar and interchangeable products. BsUFA III helps support greater consistency and regulatory predictability for potential treatments in the biosimilar pipeline. In the years since BsUFA was enacted, the FDA has approved 39 new biosimilars, including three 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 bring new treatment options and increase competition in the marketplace to the benefit of patients.
The signing of this legislation is a win for patients, biopharmaceutical innovation and regulatory predictability, and our industry is grateful for Congress’ hard work to get these programs reauthorized for another five years.
Topics: FDA, PDUFA, Biologics and Biosimilars