PDUFA
The Prescription Drug User Fee Act: Promoting the timely availability of safe and effective medicines to patients
The Prescription Drug User Fee Act (PDUFA) has helped the U.S. Food and Drug Administration (FDA or Agency) fulfill its central mission – to protect and advance public health – by allowing the Agency to keep pace with the number and complexity of innovative medicines entering the review pipeline. In large part because of PDUFA, the United States leads the world in the introduction of innovative new medicines, and the FDA’s human drug review program is the global gold standard for regulatory review and approval.
Since 1992 when the program was first enacted, PDUFA has helped enable timely access to more than 1,000 novel new drugs and biologics including treatments for cancer, rare diseases, cardiovascular, neurological and infectious diseases.
PDUFA continues to play a critical role in strengthening the FDA’s ability to review human drug applications. The program also provides biopharmaceutical companies with greater regulatory predictability, which fosters industry investment in research and development. At each five-year reauthorization of PDUFA, FDA and the biopharmaceutical industry can advance initiatives that further support innovation and enhance the regulatory review process and that are captured in the PDUFA goals letter.
The Prescription Drug User Fee Act (PDFUA VIII)
PDUFA VII
History of PDUFA
The PDUFA user fee program was first created in response to a bottleneck of new medicine approvals that left patients waiting for years for an under-staffed and under-funded FDA to review new drug applications. Before PDUFA, it often took the FDA more than two years to review new medicines, and more than 70% of medicines were first approved outside of the United States.
In 1992, Congress, recognizing these challenges, passed the first PDUFA and now, 30 years later, the average approval time for a new medicine is 10 months for standard applications and eight months for priority review applications, and in 2023 alone, approximately 64% of novel drugs reviewed by the Center for Drug Evaluation and Research, were approved in the United States before any other country. PDUFA has played an essential role in strengthening FDA’s ability to support innovation while maintaining the Agency’s high standards for scientific rigor and patient safety.
History of PDUFA
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PDUFA VII supports the development and timely regulatory review of innovative medicines to the benefit of patients
PDUFA VII was signed into law on September 30, 2022. The associated PDUFA VII goals letter was negotiated by the FDA, with input from patient advocacy groups and industry stakeholders and builds upon previous iterations with a renewed focus on strengthening FDA’s critical capabilities, improving efficiencies in drug review and driving innovation for patients. The PDUFA VII goals letter addresses new areas such as advancing digital health technologies, enhancing product quality reviews and facilitating increased utilization of innovative manufacturing technologies. The current user fee authorization expires September 2027.
Key areas of the PDUFA VII goals letter include:
PDUFA is Accomplishing
areas of PDUFA VII
Related Resources
Advancing Analytical Methodologies for Unmeasured Confounding in Real-World Evidence
The opportunity to use large amounts of real-world data (RWD) to evaluate the benefits and risks of therapeutic interventions has long been recognized by stakeholders across the health care ecosystem including regulators, academics, providers and the biopharmaceutical industry.
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FDA
Report
The Prescription Drug User Fee Act Fact Sheet
For nearly 30 years, the Prescription Drug User Fee Act (PDUFA) has played a critical role in strengthening the U.S. Food and Drug Administration’s (FDA) ability to help ensure the availability of safe and effective medicines. First created in response to a bottleneck in the drug approval system that left patients waiting for years for an under-staffed and under-funded FDA to review new drug applications, PDUFA has helped meet urgent patient needs for more timely review and approval of life-saving medicines.
Because of PDUFA, the United States now leads the world in the introduction of new medicines, and the FDA human drug review program is the global gold standard for regulatory review and approval.
Read more about this important legislation with this fact sheet from PhRMA.
PDUFA Fact Sheet
Fact Sheet
Preventing and fighting disease: A look at the biopharmaceutical progress made for patients in 2024
Every day, biopharmaceutical researchers in our industry are focused on developing the medicines that patients need to prevent and treat disease. This means taking a comprehensive approach to health care, with a focus on prevention, screening and early intervention, health and wellness and building on our scientific knowledge to develop the next generation of treatments and cures.
2024 was an important year for patients as 59 novel treatments and vaccines were approved by the U.S. Food and Drug Administration (FDA). Some of the key milestones for patients and biopharmaceutical researchers in 2024 included:
- Major advances in cancer care. Last year marked significant progress in the fight against cancer with at least 15 new approvals, including drugs for small cell lung, gastric and breast cancers. The American Cancer Society recently reported that the cancer mortality rate declined by 34% from 1991 to 2022 in the U.S., due in part to better treatments.
- Progress toward alleviating the burden of chronic disease. Among the FDA’s approvals was one that targets a novel pathway in the treatment of hypertension, providing a new treatment in those whose disease is not adequately controlled with available therapies. Hypertension affects roughly half of the U.S. population and is often referred to as the “silent killer” because it often does not have any symptoms and can lead to heart attacks and stroke.
- New cell and gene therapies to address rare diseases. Seven novel cell and gene therapies were approved in 2024 including to treat leukemia, melanoma, and rare neurological and blood disorders. Importantly, cell and gene therapies fight diseases at their source – the cellular genetic level – through one-time administration, resulting in long-term, potentially curative benefits.
- Expanded options for those with neurological and mental health conditions. Several new medicines for neurological conditions were approved by the FDA, including the first novel medicine approval for schizophrenia in decades and a new Alzheimer’s drug that slows the progression of this devastating disease and could allow for fewer infusions and lower treatment costs.
- Better protection against infectious disease threats. Vaccines have long been a crucial tool in preventing deadly diseases like measles and polio, which once caused widespread illness and death. Vaccine innovation continued, as 2024 brought new options to protect at-risk populations, including a new pneumococcal vaccine that targets the majority of serotypes that affect older adults.
The U.S. continues to lead the world in the development of new medicines, thanks in large part to the independence of our FDA and the robust programs it has in place to efficiently review and approve new drugs—such as the Prescription Drug User Fee Act (PDUFA VII) and accelerated approval pathway.
As we reflect on the progress made in 2024, we’re proud of the work biopharmaceutical manufacturers are doing to address unmet medical needs and bring life-changing treatments and vaccines to patients and improving Americans' health.
Mike Ybarra
Scientist in laboratory in pharmaceutical factory
3 Things That Support Robust Biopharmaceutical Research and Development
Over the past year, America’s biopharmaceutical companies have played a critical role in the global response to COVID-19. The unprecedented speed of developing new treatments and vaccines is due in large part to the productivity of the United States’ biomedical research ecosystem, which is sustained by a policy framework designed to support and advance the innovation of new medicines.
Here is a closer look at three key pillars of the biopharmaceutical ecosystem that are central to the success of America’s research and development.
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Research and Development
Fact Sheet
The Prescription Drug User Fee Act: Ensuring the Timely Availability of Safe and Effective Medicines for Patients
The Prescription Drug User Fee Act (PDUFA) was first enacted to help speed the FDA’s review of drug applications so that all Americans could receive timely access to safe and effective medicines. Today, PDUFA plays an important part in positioning the United States as a global leader in biopharmaceutical innovation.
FDA,PDUFA
Fact Sheet
Website Updated as of March 2023