What CBER Director Dr. Marks Said to the Alliance

What CBER Director Dr. Marks Said to the Alliance

On May 10, 2022, the Alliance for a Stronger FDA, hosted a webinar with Dr. Peter Marks, Director of FDA’s Center for Biologics Evaluation and Research.

Following the Alliance’s traditional ‘self-interview’ format, Dr. Marks began with a discussion of eight big-picture priorities: 1) COVID 19 response, 2) backlog of applications, 3) gene therapies, 4) intermediate pathways for cellular products, 5) blood donor policies, 6) regulatory science, 7) modernizing IT and business processes, and 8) workforce recruitment, training, and retention.

Regarding the ongoing response to and recovery from COVID-19, Marks noted that CBER has important obligations with additional EUAs, pediatric booster evaluations and transitioning from EUAs to traditional approvals. Dr. Marks noted that even simple statements on pediatric vaccines are often very complicated.
Turning to the ongoing work of CBER, he noted that a priority for the Center is to address the backlog of approvals consistent with PDUFA.

Looking forward and leaning into future challenges and opportunities is also a priority. In this realm, Dr. Marks noted that gene therapies are a nascent and “exciting field” and that he wants to guide the agency to be able to give feedback in a timely manner. He also talked about creating ‘intermediate pathways’ for certain cellular products so they can be brought forward to patients.

Regarding blood donor policies, he cited a blood donation study currently underway which will explore gender neutrality.

On regulatory science, Dr. Marks articulated his desire to elevate work in this area, including applied sciences as they relate to manufacturing. He noted that CBER has 80 principal investigators/reviewers in this space and could use more.

Rounding out the eight big-picture priorities, Dr. Marks highlighted his commitment to modernizing business processes and IT to serve public health and ongoing critical efforts to recruit, train and retrain a workforces suited to the evolving challenges that face the Center.

When posed the question of what he would do more of if additional resources were available, Marks cited three priorities: 1) hire more vaccine and gene therapy reviewers, 2) accelerate IT improvements, and 3) advance safety surveillance systems including AI and language processing, especially as they apply to adverse event reporting. Dr. Marks noted that there are 60 different immunization information systems across the United States, which makes leveraging data a challenge. He noted that advances in this area may require legislative help.

Regarding hiring priorities, he noted how important it is to have expertise and experience that matches sponsor submissions. Covid-19 also shattered previous assumptions that center personnel would all be in the DC /Maryland/Virginia area. Going forward, some of the workforce will come into the office on a regular basis, some will be hybrid, and some will be primarily remote. This flexibility is key as the center works to attract and retain talent. Dr. Marks also noted that some center employees deferred retirement during Covid-19, and there could be a looming swell of retirements.

As for other likely permanent changes due to the pandemic, Marks noted that FDA guidance development became more efficient and agile – a trend that will hopefully carry over. He noted that throughout Warp Speed, parallel and consistent communication would often result in morning questions from vaccine developers followed by afternoon and evening responses from agency experts. Dr. Marks would like to explore a pilot on this approach for future priority products.

Regarding new technologies such as AI, block chain and continuous manufacturing, Dr. Marks noted that the center has considerable expertise but will need ongoing professional development to keep up. He cited the example of CRISPR technology as a quickly advancing field. He noted that grants and contracts might be a means to pilot immature technologies and explore continuous manufacturing.

Post-market safety surveillance is also top of mind at CBER, noting that vaccine rollouts were accompanied by near real-time safety systems. With the right technology partners, it may be possible to get near real-time results on a broader basis. Dr. Marks also spoke highly of the FDA’s Sentinel Initiative as it relates to biologics.

Dr. Marks also addressed a perception that current EUA applications are taking longer than those earlier in the pandemic. He noted that the Center is receiving more applications that must be measured against more variants and variables in the population.

Editorial Note: The week’s Analysis and Commentary section was written by the Alliance’s Executive Director, Steven Grossman.