“User fees help provide the FDA with the resources necessary to make accurate, timely determinations about the safety and efficacy of new drugs,” said Stephen J. Ezell, ITIF’s vice president for global innovation policy and the report’s author.
“The user-fee system,” Ezell said, “fosters innovation by ensuring the FDA has the resources and skilled personnel needed to incorporate the latest advances in regulatory science into the drug-approval process.”
“This may at first glance seem like a mundane, bureaucratic process, but it is actually a key factor in why America has emerged in recent decades as the world’s leader in life sciences,” he said. “It is a commonsense policy that Congress should reauthorize with further enhancements to bolster the FDA’s ability to speed cures and monitor safety.”
Since PDUFA passed in 1992 — authorizing the FDA to collect fees from the biopharmaceutical industry as part of applications for regulatory approval of new human drug submissions — the median approval time has fallen to under 10 months, meaning that safe, innovative drugs get to patients faster.
The report also explains that this stability provides the predictability innovators need to invest in new cures.
In addition to benefiting patients by helping speed cures, PDUFA has also played a supporting role in establishing the United States as the world’s leader in life-sciences innovation, an important sector to the U.S. economy that supports over 4.4 million total jobs directly and throughout its supply chain, with exports of $47 billion in 2015, an amount that has tripled since 2003.
As Congress considers reauthorizing this law for the fifth time since 1992, the report urges lawmakers to update the law to accomplish several goals:
- Support the development and application of 21st-century regulatory science approaches to drug development, including creating a pathway for greater use of real-world evidence, supporting innovative clinical-trial approaches, and enhancing biomarker-qualification pathways.
- Direct more attention to patient-focused drug development, including greater effort to incorporate patient perspectives in the drug development and review process, including making greater use of patient-reported outcomes.
- Focus more on supporting rare diseases and breakthrough therapies, including continued application-fee waivers and advanced reviews for medicines that can treat rare diseases, as well as prioritizing the development of breakthrough medicines for patients with life-threatening diseases.
- Enhance post-market safety monitoring of approved drugs, including additional resources for the FDA’s Sentinel system to monitor drugs that have already entered the patient population.
Enhance FDA workflow and workforce planning, including financial management and hiring and workforce enhancements.
Ezell dismissed arguments suggesting PDUFA has caused the FDA to view the biopharmaceutical industry too much as a “partner” or source of revenue, rather than as an industry over which it has regulatory oversight.
“PDUFA isn’t about getting to yes faster. It’s about making a determination in a timely manner about the safety and efficacy of a drug and whether or not it should receive marketing approval,” he said.
The report points out that less than 12 percent of the candidate medicines that make it into Phase I clinical trials are approved by the FDA—half the rate of a decade ago.
“The Prescription Drug User Fee Act has played an instrumental role in ensuring the FDA has the necessary personnel and resources at its disposal to make safe, effective, and timely determinations regarding drug applications,” said Ezell,
“Policymakers should reauthorize PDUFA in a timely manner to ensure that America’s biopharmaceutical industry can continue to innovate and develop new treatments to help patients live healthier lives.”
The Information Technology and Innovation Foundation (ITIF) is an independent, nonpartisan research and educational institute focusing on the intersection of technological innovation and public policy.
Recognized as one of the world’s leading science and technology think tanks, ITIF’s mission is to formulate and promote policy solutions that accelerate innovation and boost productivity to spur growth, opportunity, and progress.