Industry lauds PDUFA VII focus on innovation, transparency

Regulatory NewsRegulatory News | 26 August 2021 |  By 

Trade associations in the pharmaceutical and biotechnology fields have weighed in on the performance goals FDA has outlined for the upcoming reauthorization of the Prescription Drug User Fee Act (PDUFA VII), taking a generally favorable view of planned initiatives and areas of focus.
“The PDUFA VII agreement released today is an important step forward to continue to support patient safety, maintain the FDA’s high standards of independent regulatory review and promote timely access to safe and effective medicines for patients,” said the chief executive officer of Pharmaceutical Research and Manufacturers of America (PhRMA), Stephen Ubl, in a statement released earlier this week. “It is also an opportunity to advance innovative research and development and review approaches to prepare for the future of drug development, including leveraging applicable lessons learned from the COVID-19 pandemic,” he added.
PhRMA highlighted several performance goals from the 71-page document as being of key significance for industry. The trade association lauded FDA’s ongoing emphasis on adopting a patient-centric approach to drug review both during the development process and in post-marketing safety surveillance. An increased use of real-world evidence also signals that the agency is “modernizing regulatory evidence generation and drug development tools.”
Also, priorities in the commitment letter acknowledge that rare and ultra-rare disease development can benefit from innovation in trial design and conduct; the burgeoning field of cell and gene therapy also requires increased agency resources to meet the increased pace of submissions. (RELATED: PDUFA VII commitment letter: RWE, rare diseases see renewed attention, Regulatory Focus 23 August 2021)

PDUFA VII performance goals include a plan for the Center for Biologics Evaluation and Research (CBER) to have a modernization roadmap completed by the end of FY 2022, with an eye to adopting new data management tools and technologies. New draft guidances are also planned to support sponsors in the development of novel approaches in the realm of cell and gene therapy.
The performance goals sketch out new meeting types and other mechanisms by which sponsors can achieve earlier and more interactive communication with FDA staff. The pandemic, noted PhRMA, has also provided lessons in flexibility where manufacturing and product review processes are concerned, and FDA plans to incorporate some of these going forward.
FDA plans to move more resources and activities to cloud-based platforms and increase use of digital health technology-generated data. The agency will also continue modernizing its IT capacity and capabilities, and, in parallel, will work to boost retention, increase hiring, and continue to work on financial management matters.
Targeted hiring within FDA’s human drug review centers include an ambitious aim of hiring 132 new CBER and 77 new staff members in the Center for Drug Evaluation and Research (CDER) in FY 2023.
The Biotechnology Innovation Organization (BIO) also released a statement this week, concurring with many of PhRMA’s PDUFA VII performance goal highlights.
“BIO is supportive of the newly-released PDUFA VII commitment letter. We believe it achieves our goals of strengthening scientific dialogue between FDA and industry as well as promoting innovation and a patient-centric review process,” said BIO’s chief scientific officer E. Cartier Esham in the statement.



© 2023 Regulatory Affairs Professionals Society.

Discover more of what matters to you