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Politico Pro — Inflation’s hit to the FDA

Politico Pro — Inflation’s hit to the FDA

FOCUS ON CELL, GENE THERAPIES — Hiring personnel with expertise in cell and gene therapies remains a challenge at the FDA, but senior officials said Monday they’re optimistic about the progress they’ve made so far.

CBER’s Office of Therapeutic Products has done well in hiring the allotted personnel under the prescription drug user fee program, which was reauthorized in 2022, Dr. Peter Marks, head of the FDA’s Center for Biologics Evaluation and Research, said during a Friends of Cancer Research event in Washington.

“Right now, it’s not a bad time because there are … a fair number of people that are skilled in [chemistry, manufacturing and controls] that are out there that we’ve been able to hire — physicians that are earlier on in their career who we’ve been able to hire — so I’m pretty encouraged with the people who we brought on board recently,” Marks said. “It’s exciting because we have a diversity of experience and expertise that will serve us well for these products.”

Making the therapy: A major challenge for cell and gene therapies is the manufacturing process. The therapies are generated using the patient’s cells, known as autologous therapies, or a donor’s cells, known as allogeneic therapies.

Allogeneic treatments can be made in large batches for “off-the-shelf” access, while autologous therapies are personalized for the donor patient.

Marks said the FDA is neutral on centralized manufacturing versus decentralized manufacturing.

“There are advantages potentially to decentralized manufacturing for certain products — particularly ones that are made fewer times — but we are just there to support this process forward just as long as the ultimate product, whether centralized or decentralized, and manufacturer is at the same high quality,” he said.

Covid-19 lessons: The agency has launched a pilot program for rare disease therapy development in which makers of prospective cell and gene therapies have more frequent communication with FDA staff to help with clinical data generation. The pilot seeks to emulate Covid vaccine makers’ access to regulators when they worked to rapidly test and deploy shots to control the pandemic.

“If you get rid of a lot of 30-day or 75-day waits, development can occur much more rapidly,” Marks said, referring to how long it typically takes to schedule a meeting with the agency. “We hypothesize that, for other products, that kind of communication could help, particularly ones for serious diseases, cancers.”

If the pilot demonstrates that those interactions can slash development timelines, Marks said, it could help make the case for the FDA to hire more personnel. The agency currently employs more than 18,000 people.

“Even a 10 or 20 percent reduction in time of development can really have an impact on patient lives when you’re dealing with a life-threatening illness,” he said.