For more than two decades, the Prescription Drug User Fee Act (PDUFA) has played a critical role in underpinning America’s leadership in biopharmaceutical innovation. As Congress works toward a bipartisan reauthorization of PDUFA for the fifth time, a significant focus is on incorporating the latest in regulatory data science and taking account of patients’ perspectives to ensure the Food and Drug Administration (FDA) has the resources it needs to expeditiously evaluate the safety and efficacy of innovative new medicines that hold the key to the next wave of treatments and cures. On 6/6, ITIF hosted an event to bring together Congress, policy experts, industry leaders, and patient advocates to discuss why a timely reauthorization of PDUFA is critical to the future of biomedical innovation.
ITIF vice president for global innovation policy Stephen Ezell opened the event by reminding the audience of the context of the original PDUFA bill in 1992. In the 1980s and 1990s, with the HIV/AIDS epidemic at its peak, pharmaceutical companies began a surge of research into new drugs. However, the FDA lacked sufficient resources to evaluate applications for new drugs in a timely manner. The 1992 PDUFA bill and its reauthorizations, Ezell said, have “truly been instrumental in equipping the FDA with the resources it needs to make safe, timely, and accurate determinations with regard to the safety and efficacy of new drug applications.” Quantifying just how helpful PDUFA has been, the median time the FDA takes to make a determination on a new application has fallen by approximately two-thirds since then, Ezell said. He further noted that PDUFA has been instrumental to the United States’s global leadership in life-sciences research: In 1992, 10 percent of new drugs were introduced to Americans first; today, some 60 percent are. Such statistics, Ezell argued, are proof that “PDUFA really represents the best of the public and private sectors working in concert.”
Former Representative Ron Klink, senior policy advisor at Nelson Mullins, echoed many of Ezell’s observations. The FDA was overwhelmed by applications in the 1980s and early 1990s, Klink said, which meant Americans were watching family members suffering and even perishing from conditions treatable with drugs available in Europe. Since PDUFA was passed, however, Klink noted that 20 of 23 applications for new oncology drugs were approved more quickly in the United States than in Europe. In addition to the FDA and patients, Klink also pointed out that pharmaceutical companies, which were initially wary of any new tax, are now broadly supportive of PDUFA reauthorization because they see the benefits of getting new drugs to the market as quickly as possible. This support from and collaboration between industry and the FDA, Klink said, is why “a lot of the hard work is done, and now Congress must not put a lot of ’Christmas tree ornaments’ [i.e., ancillary riders] and just make sure [Congress passes] it as clean as possible.”
Susan Winckler, chief risk management officer at Leavitt Partners, was the FDA chief of staff during the 2007 PDUFA reauthorization. That year’s reauthorization, Winckler said, caused some “angst” because controversies pushed the vote closer to the sunset date. She argued that instead of repeating a political fight, a healthier, “constant interaction” between FDA and industry should be modeled in other policy areas. “User fees generally improve the culture of the entire agency,” she said, “because it drives this idea that industry and the regulator are working together to bring cures to patients.” Winckler concluded, “The [PDUFA] user fees, and particularly the reauthorization process, drives that interaction… [The FDA] now has the resources… to then interact with the regulated industry.” To maintain those lines of cooperation and prevent unnecessary controversy, Winckler urged all stakeholders to differentiate between which issues need to be dealt with in statutory language and which are better ironed out through day-to-day communication.
Cynthia Bens, the Alliance for Aging Research’s vice president of public policy, praised the progress the FDA has made on incorporating patient perspectives into drug development. The most recent PDUFA, she argued, has been invaluable to this effort by providing patient advocacy groups the opportunity “on an annual basis to workshop” with FDA, industry, and academia, which helps facilitate large-scale, long-term clinical trials on new Alzheimer’s drugs. “The culture at FDA… has changed a lot over the last ten years,” Bens said, “but what we feel we need to do as advocates is provide FDA [with the] resources they need not only to review the drugs when they come in but support all these other activities that they’re already doing to work with the community.” The emphasis on quantifying risks and benefits to patients, she argued, is crucial to ensuring that Alzheimer’s research keeps the patient at the forefront.
Keysha Brooks-Coley, senior director of Advocacy Strategic Alliances at the American Cancer Society Cancer Action Network (ACS-CAN), stated “we strongly agree [PDUFA] has been successful” since 1992. Patients, she said, “rely on FDA to ensure safe and effective treatments reach those who are in need, and as we have heard here today, the PDUFA program really has allowed [the FDA] to make sure that individuals who need access to innovative treatment are able to receive it in a timely fashion.” The current proposal to reauthorize PDUFA includes some new additions, and Brooks-Coley stated the ACS-CAN has “been encouraged by a number of provisions,” including “the incorporation of rare disease staff in the product review teams, increased resources dedicated to biomarker development and validation… the use of real-world evidence, increasing efforts to better hire and maintain well-qualified staff at FDA,” and more. The Patient Representative Program formalized in the last PDUFA reauthorization is crucial, she said, because “making sure that the patient perspective is included throughout the drug development process from the very beginning all the way through until it’s brought to market is very important.” The focus on patient perspectives resonated with Bens and other panel members who urged FDA to use PDUFA as an opportunity to incorporate input from patients into the drug-development process.
Representative Diana DeGette (D-CO), finished the event by sharing some insights on the state-of-play of PDUFA reauthorization and the future of FDA-industry-Congressional cooperation in the life sciences. “Congress,” Rep. DeGette said, “has been acutely aware for quite some time that we need to reauthorize PDUFA.” She urged Congress to complete the reauthorization before the August recess, because a delay risks FDA layoffs and could grind the drug approval process to a halt. Rep. DeGette also echoed Brooks-Coley by touting the 21st Century Cures Act, which she cosponsored, for helping “set the stage [for PDUFA] ... by leveraging the patient perspective to fight disease, the use of real world evidence in making approval determinations, and also the [increased use of] biomarker qualification.” Congress has always reauthorized PDUFA with overwhelmingly bipartisan votes, Rep. DeGette noted, and she urged her colleagues to do so again by rejecting the Trump administration’s controversial proposal to recalibrate fees so that “industry [would] carry the entire load of the FDA’s budget.” Such poison pills, she argued, would create unnecessary partisan rancor over a “must-pass piece of legislation.”
The advances in U.S. pharmacology and the globally competitive edge the U.S. pharmaceutical sector has maintained in recent years owe much to PDUFA. This panel underscored the depth of support for reauthorization from industry, patient advocates, and Congress. Not only must Congress reauthorize PDUFA, but legislators should also take this opportunity to ensure that the patient perspectives are heard during every stage of the drug development process to keep the focus on delivering cures to those who need them. PDUFA is an example of a policy that works, and Congress must not break something that isn’t broken.