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Regulatory Focus — Top FDA officials discuss PDUFA hiring, rapid communication, and international collaboration efforts

Regulatory Focus — Top FDA officials discuss PDUFA hiring, rapid communication, and international collaboration efforts

Peter Marks, director of the US Food and Drug Administration’s (FDA) Center for Biologics Evaluation and Research (CBER), said his center is doing a “pretty good job” of filling positions under the current Prescription Drug User Fee Act (PDUFA VII) agreement at a meeting sponsored by the Friends for Cancer Research (FOCR) and Parker Institute for Cancer Immunotherapy (PICI) in Washington, DC on Monday.

Marks was joined by FDA Principal Deputy Commissioner Namandjé Bumpus for a keynote discussion moderated by former Washington Post reporter Laurie McGinley.

PDUFA VII includes specific provisions to scale up CBER’s cell and gene therapy product review staff to accommodate the significant increase in applications in recent years. “Hopefully we will continue to staff up as need be,” Marks said, adding that the agency has “made really good progress” in this area. “Hopefully we will continue to staff up as needed,” Marks said.

Marks added that CBER has done a “pretty good job” of hiring staff with the necessary experience, such as skilled chemistry manufacturing and controls (CMC) reviewers, as well as physicians who are just starting out in their careers. “I am pretty encouraged with the people we have been onboarding lately and it is exciting because we have a diversity of experience that we need,” he said.

Bumpus added that having the right reviewers in place to accommodate the range and complexity of applications “requires a range of expertise, it is the science, and the manufacturing, and people need to understand that … my job as principal deputy is to support the center directors and the work they are doing. I try to ensure that I can reduce or minimize any barriers they may have in doing that.”

Cell and gene therapies

Marks also noted that cell and gene therapies are “branching out” from hematologic diseases into treating solid tumors made possible from the “revolution” of CRISPR-Cas9 gene editing technologies. The agency also plans to apply the learnings from two pilot programs in reviewing new applications for these products, he said.

When asked to comment on what changes they see on the horizon Marks said that “we are in a place now where there is a subtle change, we started out, in terms of chimeric antigen receptor (CAR) T-cells – it was like one of those kids soccer pile ups – everyone was making CD19 CAR-T cells and they were all autologous based with a couple of outliers in the allogeneic space.”

“Now we are starting to see a branching out of this space where we are seeing much more complicated … constructs moving into the allogeneic space from autologous and branching out into other areas. We’re moving from the kind of core hematologic malignances, where we started out in acute lymphoid leukemia and non-Hodgkin’s lymphoma moving into multiple myeloma, but now starting to see the movement in, hopefully making a dent in, solid tumors made possible from the revolutions of gene editing that came with the introduction of CRISPR.”

Bumpus noted that FDA is also starting to see more applications from sponsors of autoimmune diseases and expects to see more applications involving AI.

Learnings from COVID-19, pilots

Bumpus and Marks were also added to address any learnings from the COVID-19 pandemic in its reviews of new cell and gene therapies.

Both agreed that one learning was the importance of working with other regulatory bodies, and this lesson can be applied to the cell and gene therapies space.

Marks said that “we became close with our regulatory colleagues in Europe and Japan and Canada and even Brazil, we got to know each other more and realize there are differences in our regulatory requirement some of which were a barrier to entry.” As the result of these collaborations, regulators were able to break down some of these barriers.

FDA is also working with the World Health Organization (WHO) to develop a harmonized regulatory framework for cell and gene therapies and to get buy in not just from high-income countries but low- and medium-income counties as well.

Marks said WHO published a white paper on this area of converging regulations for cell and gene therapies. (RELATED: WHO charts path to convergence on cell and gene therapiesRegulatory Focus 14 January 2022).,-rapid-comm