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PDUFA VII is critical for future biopharmaceutical innovation and for patients

The PDUFA VII goals letter expands upon the most recent iteration of the user fee agreements which expire in September of 2022 with a renewed focus on strengthening review fundamentals, enhancing accountability and transparency and advancing innovation for patients.

Key areas of the PDUFA VII goals letter include:

  • Enhancing patient-centric drug review and safety monitoring: Advance the incorporation of patient-centric data, including patient preference information, in drug development and regulatory reviews and support the Agency’s reviewing, tracking and communicating of post-market safety information.
  • Strengthening scientific dialogue and advancing innovation: Expand opportunities for sponsors to obtain FDA’s regulatory feedback and clarity throughout the drug development process.
  • Supporting the next wave of advanced biological therapies: Strengthen the Agency’s staff capacity and capability to support the development and review of cell and gene therapies.
  • Modernizing regulatory evidence generation and drug development tools: Advance the use of real-world evidence in regulatory decision making, facilitate further use of complex adaptive and other novel clinical trial designs, and advance consistency and predictability around the use of modeling and simulations.
  • Advancing digital technologies and information technology (IT) infrastructure: Facilitate adoption of innovative digital health technologies and modernize FDA’s data and IT capacity and capabilities, including adoption of cloud-based technologies.
  • Enhancing innovation in manufacturing and product quality reviews: Facilitate the use of innovative manufacturing technologies for both products in development and those commercially available and incorporates best practices from COVID-19 lessons learned for the use of alternative tools to assess manufacturing facilities.
  • Enhancing FDA hiring, retention and financial management: Build upon PDUFA VI efforts to improve accountability and transparency and modernize financial and staff resource management.

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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 passed the first PDUFA and now, nearly 30 years later, the average approval time for a new medicine is just 10 months, and over the last five years, approximately 75% of novel drugs 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. To help make the review process more efficient and predictable, biopharmaceutical companies pay two different user fees under PDUFA:

  • Application fee: Fee due when a sponsor submits a New Drug Application (NDA) or Biologics License Application (BLA).
  • Program fee: Annual fee for most approved prescription drug products without an approved generic.

The latest reauthorization of PDUFA (PDUFA VI) was signed into law on August 18, 2017 and took effect on October 1, 2017. It expires September 30, 2022.

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PDUFA VI Is Accomplishing:

PDUFA VI is strengthening the FDA’s human drug review program and helping the Agency remain the global “gold-standard” of medicine review through:

  • Innovative Clinical Trial Designs: Innovation in the design and analysis of clinical trials can allow for more efficient drug development. PDUFA VI is enhancing drug development by establishing processes to facilitate appropriate use of innovative clinical trial designs and novel statistical methods which helps to enhance their review and acceptance in the drug review process.
  • Patient-Focused Drug Development: PDUFA VI is boosting the FDA’s expertise and capacity to advance the science of patient input and to incorporate patient perspectives in the drug development and review processes. Patients, family members and caregivers can provide their unique and valuable perspectives on their disease condition and available treatment options. These perspectives can help inform evaluations of a medicine’s benefits and risks, provide additional context for FDA’s regulatory decision-making, and help researchers better identify clinical outcomes that matter to patients and their caregivers.
  • Real-World Evidence (RWE): By employing the data uncovered in the “real-world” practice of medicine – whether through electronic health records, payer administrative claims, or patient registries – the FDA can harness valuable information about the safety and efficacy of a medicine in a broad population. PDUFA VI is providing additional resources to the FDA to help develop a better understanding of how RWE can be used in regulatory decision-making.
  • Breakthrough Therapies and Medicines for Rare Diseases: PDUFA VI is building on the success of the Breakthrough Therapy Program by investing dedicated resources to prioritize the development and availability of breakthrough medicines for patients with serious and life-threatening diseases. PDUFA VI also helps FDA to advance the development and approval of medicines for rare diseases, including pediatric rare diseases.
  • Post-market Safety: PDUFA VI is providing significant resources to enhance the FDA’s ability to review, track, and communicate important post-market safety information, including investments to expand the Sentinel System’s capabilities and enhance the communication process with stakeholders on the use of Sentinel data.

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Biopharmaceutical Response to Covid-19

PDUFA is also helping FDA fulfill its central mission of protecting the public health as the Agency responds to the COVID-19 pandemic. Since the start of the pandemic, FDA has been working with biopharmaceutical companies to accelerate the development of safe and effective COVID-19 therapeutics and vaccines. The Agency has provided timely recommendations, regulatory guidance, and technical assistance to developers of potential treatments and vaccines for COVID-19, including on the use of novel approaches to clinical trials and drug review. FDA has also provided clarity to biopharmaceutical sponsors on how to continue to advance the development of new medicines during the current pandemic. PDUFA has helped support this engagement by ensuring FDA’s human drug review program has the infrastructure, scientific expertise and experience with cutting-edge drug development approaches needed to support continued innovation and inform efficient regulatory decision-making.

(Website updated as of August 24, 2021)