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Bipartisan collaboration on the Hill has proven scant in the opening weeks of the Trump era, but the opportunity to reauthorize the Prescription Drug User Fee Act (PDUFA) provides a welcome occasion for bipartisan comity while extending legislation that has played a foundational role in underpinning U.S. life-sciences leadership and helping bring safe, innovative cures to patients faster.
Originally chartered by Congress in 1992, PDUFA authorizes the Food and Drug Administration (FDA) to collect user fees associated with applications from the biopharmaceutical industry for regulatory approval of new human drug submissions. The initial legislation called for Congress to reauthorize PDUFA every five years, which Congress has now done on five occasions on a strong bipartisan basis. With PDUFA’s current authorization set to expire in September 2017, Congress gets the reauthorization ball rolling this week for PDUFA VI with hearings scheduled in Senate Health, Education, Labor and Pensions Committee and the House Energy and Commerce Committee, starting what will hopefully be a smooth and timely reauthorization process.
As ITIF recently documented, PDUFA has succeeded as Congress intended in enabling a safe, timely, and efficient process of drug evaluation while applying best practices in regulatory science that have contributed to making America the world’s leader in biomedical innovation. Since Congress launched PDUFA in 1992, the median time for the FDA to make a determination regarding approving a new medicine has fallen by more than half, from over 20 months to under 10. Moreover, by providing greater certainty, stability, and predictability in the drug approval determination process, as well as by providing gateway reviews enabling innovators to better understand and meet FDA clinical trial requirements, PDUFA has contributed to more innovative new drugs being launched first on the U.S. market. In fact, in 1992, only 10 percent of new drugs were first launched in the United States, but today that figure stands in excess of 60 percent.
And this combination—faster FDA determinations and more first launches—explains why innovative new drugs tend to reach patients faster in the United States than in Europe or elsewhere in the world. As a recent study found, “the FDA consistently took less time than the European Medicines Agency to review a new oncology medicine,” one reason explaining why “new anticancer medicines are typically available in the United States before they are in Europe.” PDUFA has also played a key role, alongside robust public-private investment in life-sciences research and strong intellectual property (IP) protections, in making America the world’s most fertile environment for life-sciences innovation. For instance, between 1997 and 2012, more than half of the IP related to the world’s new medicines was conceived in America, while, in the 2000s, U.S. biopharmaceutical companies introduced more new chemical entities than companies from the next five nations combined.
PDUFA VI seeks to build on prior iterations with several important new provisions. The PDUFA VI commitments letter, which represents the product of FDA discussions with industry and other stakeholders, supports the development and application of 21st-century regulatory science approaches to drug development, including creating a pathway for greater use of real-world evidence (i.e., evidence outside of randomized clinical trials) in regulatory decision-making, supporting innovative clinical-trial approaches, and enhancing biomarker-qualification pathways. PDUFA VI also seeks to create mechanisms to better incorporate patient perspectives in the drug development and review process, to support research into rare diseases and breakthrough therapies, and to enhance post-market safety monitoring of approved medicines. Most importantly, PDUFA VI aims to facilitate workforce and workflow planning to help ensure the FDA remains equipped with the medical, scientific, and administrative capacity it needs to keep pace with biomedical innovation.
A failure to reauthorize PDUFA in a timely manner would have far more dire consequences than just longer wait times for drug reviews and determinations: It could lead to the FDA issuing reduction-in-force notices informing FDA employees whose positions are funded by PDUFA-collected fees that they are at risk of termination and would harm patients as drugs take longer to reach market and fewer enter development. And while other issues have been in the news recently such as drug prices or importation of medicines, it’s important that Congress not let discussion of these issues sidetrack focus from the merits of PDUFA and the need to reauthorize it in a timely manner. PDUFA has produced tremendous benefits for medical innovation, and now gives the 115th Congress an opportunity for an early bipartisan legislative victory.