CBER lays out volume-staff mismatch in PDUFA VII meetings

Regulatory NewsRegulatory News | 19 January 2021 |  By 

During an 11 December public stakeholder meeting, FDA met with representatives of patient and consumer advocacy groups as the Prescription Drug User Fee Act (PDUFA) VII reauthorization process continues.
The meeting, held virtually, began with a review of the cell and gene therapy review programs overseen by FDA’s Center for Biologics Evaluation and Research (CBER). Wilson Bryan, MD, of CBER’s Office of Tissues and Advanced Therapies (OTAT), told attendees at the virtual meeting that OTAT saw the number of investigational new drug applications nearly double from 223 in 2016 to 453 in 2019. During the same period, OTAT’s investigational device exemption applications followed a similar trajectory, rising from 142 to 277.
The number of regenerative medicine advanced therapy (RMAT) designation requests from the first such request in January 2017 has now reached a cumulative total of about 160, and OTAT meetings with sponsors have also continued to grow, hitting 384 in 2020. CBER also issued six final guidances addressing gene therapy in 2020, as well as one in draft form.
CBER’s associate director for review management, Christopher Joneckis, PhD reviewed resource needs and planned modernization of the FDA branch. As detailed by Bryan, Joneckis pointed out the “exponential growth” in the cell and gene therapy pipeline, with no signs the trand will abate.
As increasingly complex technologies and combination products come before CBER, staff are working long hours to meet PDUFA deadlines – and risking burnout, said Joneckis.
Accordingly, goals for FDA in PDUFA VII center on looking ahead to plan for increased growth and innovation in cell and gene therapies. Specifically, Joneckis called for not just upsizing, but rebalancing staffing in CBER’s Cell and Gene Therapy (CGT) program.
Stakeholders must be engaged in the process of developing timely and clear guidance documents that keep pace with rapidly evolving science; this, said Joneckis, is another area that requires up-staffing. Similarly, regulatory science work conducted by CBER must be robust and forward-thinking to stay on track with industry innovation.
Speaking more broadly, Joneckis also called for revamped information technology to support high volumes of complex data. Currently, the agency runs 35 systems on almost as many different technology platforms, and a modernization effort is long overdue, he said. Some steps that began in 2019 continue, but acceleration is needed and can only happen with an infusion of funding in PDUFA VII, according to Joneckis.


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