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Alliance Learns About CBER Priorities for 2020 and Beyond

January 25, 2020

At the Alliance’s request, Dr. Peter Marks, Director of FDA’s Center for Biologics Evaluation and Research, met with Alliance members this week to discuss opportunities, challenges and priorities at CBER in 2020 and beyond. The Alliance is particularly interested in understanding the level of resources (budget, personnel, infrastructure) the agency (and each Center) needs in order to fulfill its responsibilities.

The major point of discussion was the urgent operational and staffing challenges brought about by explosive growth in development of gene therapy products. CBER currently has 900 active INDs for gene therapy products and that number is growing quickly. In 2017, there were 106 new IND applications for gene therapy products. That rose to 206 in 2018 and 243 in 2019. This situation was totally unexpected, so CBER’s budget (BA appropriations and user fees) was never sized to deal with the review and ongoing management of so many INDs, a substantial number of which involve complex, cutting-edge science.

Apart from the gene therapy situation, Dr. Marks spoke about each of CBER’s seven priorities for 2020. These are:

  • Develop a regulatory program for individualized (bespoke) therapies
  • Cell therapy compliance (enforcement discretion ends November 2020)
  • Address gene therapy human resources and infrastructure needs
  • Advance pathogen reduction research
  • Revise blood donor deferral criteria
  • Advance influenza vaccine manufacturing
  • Foster global regulatory convergence for cell and gene therapies

What struck me about this list was the extraordinary diversity of CBER activities. Additionally, Dr. Marks spoke of the challenge of addressing emerging and re-emerging infectious diseases — an issue as immediate as this week’s headlines about a new strain of highly transmissible influenza that has started in China.

Dr. Marks pointed to how resource shortfalls have impacted CBER’s successful INTERACT program. This allows companies to receive regulatory advice from the agency (on issues such as study design or the adequacy of safety studies before first-in-man trials) even earlier than their pre-IND meeting. Ideally, INTERACT meetings should be scheduled within 30 to 60 days because their goal is to discuss problems that are blocking the company’s development plan and delaying potential new therapies for patients with unmet medical needs. Instead, such meetings are taking 3 to 4 months to schedule.

CBER would also benefit if FDA could develop and implement a comprehensive IT system that was fully integrated. There are about 30 different IT systems within FDA and they are largely siloed from each other. This particularly hinders CBER’s ability to handle cutting-edge science and review applications that are significantly more complex than even a few years ago. It also undercuts the agency’s effort to integrate information for digital health systems, adverse events reports and real world evidence.

Also discussed was the difficulty in recruiting a scientifically trained workforce. There are marketplace issues (companies can pay more), but also the inability to quickly identify, recruit, and hire talented people who want to work at FDA. In industry, the hiring process is often compressed to less than 2 months, where for CBER (and presumably other centers), 6 to 10 months is closer to the norm. Further, in the case of new reviewers, it is often 1 to 2 years before they are fully trained. (We have heard similar numbers from CDER.)

My editorializing: CBER is doing a great job with handling a diverse and rapidly expanding set of responsibilities. More resources would provide incredible value to the public in terms of public health, product safety, and the advance of new therapies and cures.

Editorial note: The Analysis and Commentary section is written by Steven Grossman, Deputy Executive Director of the Alliance for a Stronger FDA.

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