The Prescription Drug User Fee Act (PDUFA) plays a foundational role in America’s biopharmaceutical innovation system. By permitting the Food and Drug Administration (FDA) to collect user fees from industry, PDUFA helps ensure the agency is adequately staffed with high-quality personnel and has appropriate workflow and project-management frameworks in place to make accurate and timely determinations regarding the safety and efficacy of applications for new human drug approvals. Moreover, PDUFA plays an important role in fostering innovation, particularly by ensuring that the latest advances in regulatory science are incorporated into the drug-approval process, including by creating pathways for the inclusion of real-world evidence and patient perspectives in the drug-evaluation process.
Created by Congress on a bipartisan basis and launched in 1992, PDUFA has since played a transformational role in turning the FDA into the world’s leading drug-regulatory agency and in helping to ensure that safe, effective medicines get to U.S. patients faster. As Congress considers reauthorizing PDUFA for the fifth time, lawmakers should recognize the foundational role it plays in underpinning America’s biomedical innovation system and improving patient outcomes.
This report begins by introducing PDUFA and by detailing the critical role PDUFA plays in America’s medical-discovery system, documenting the benefits that PDUFA has produced since its inception. It shows how PDUFA has played a key role in accelerating timely patient access to new medicines and in providing a key pillar of a broader supportive environment for life-sciences innovation in the United States. The report then examines several key elements of PDUFA that will come before Congress and the Trump administration in 2017 as part of the law’s fifth reauthorization and explains how the continued improvements in “PDUFA VI,” as it is known, are positioned to make important contributions to America’s life-sciences innovation capacity. Finally, the report addresses some of the specific criticisms that have been leveled against PDUFA and addresses the consequences of a failure to reauthorize the law in a timely manner.
Considering the totality of evidence, the report concludes that PDUFA plays an instrumental and effective role in America’s life-sciences innovation system, and that Congress should promptly reauthorize it.