FDA's Woodcock, Marks and Pazdur Talk Sustainability and New Approaches

Posted 14 November 2017 By Zachary Brennan

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Three of the US Food and Drug Administration's (FDA) top experts sat together on a panel at the third Biopharma Congress in Washington, D.C. on Tuesday, discussing everything from where the agency is headed in the next several years, how the costly clinical trial system is not sustainable and how they interview prospective FDA employees.

Janet Woodcock, director of the Center for Drug Evaluation and Research (CDER), reiterated a claim she made back in September that the clinical trial system is "broken" and not sustainable because of the expensive ways in which trials are run. She noted that pre-approval trials often do not answer the appropriate questions and those answers do not come in the postmarket trials. She also repeated the need for companies to commit to master protocols.

When asked what she's concerned with moving forward, she elaborated on that earlier position, "For medical products in the US, the elephant in the room is the willingness of society to pay for the products. And that elephant is taking up most of the air in this room."

Peter Marks, director of FDA's Center for Biologics Evaluation and Research, said that companies often "don't have the courage to fail," particularly in later phases, though data is meticulously collected monitored, scrubbed and put in data vaults, though often that work is not entirely necessary and only later used if a company fails on a primary endpoint.

Marks also offered an update on the new regenerative medicine advanced therapy (RMAT) designation, noting that as of last Friday, the agency has received 34 designation requests, acted on 31 requests and granted 11 RMAT designations.

Richard Pazdur, director of FDA's Oncology Center of Excellence, discussed the way in which cancer drug development has progressed from the 1980s and 1990s, when companies "basically sent people to the rainforest and the bottom of the sea and used a roulette wheel to try to figure out what works," to the more precise and targeted approaches used now.

Oncology drug developers are now presenting their entire drug pipelines to the agency, he added, which is "important for our reviewers to hear," as they can look at the directions companies are moving in rather than just see the different applications submitted for approval. He also noted the importance for reviewers in understanding the selection criteria companies use to move molecules forward.

In terms of hiring new reviewers for OCE, Pazdur said part of the process is asking applicants: "How do you see yourself – as a drug regulator or an oncologist? We hope they would say oncologist because that's what we're after … we need people to keep up with the science."

Woodcock, Marks and Pazdur also touched on the fact that the agency is shifting from a regulator divided by its drug, biologic and medical device applications, to one more focused on the disease areas for which applications are submitted.

Marks and Woodcock also agreed that they dislike the term "regulatory science," noting that it's basically a catch-all term that does not explain the type of translational science their departments do.

Peter Stein, deputy director of Office of New Drugs, also noted in an earlier panel that a reorganization is in the works, particularly in terms of how reviews are conducted, how patient perspectives are used, and other considerations that will be announced in the near future.

And on the topic of patient voices, Wilson Bryan, director of FDA's Office of Tissues and Advanced Therapies, noted an example – presumably with Amgen's melanoma treatment Imlygic (talimogene laherparepvec) – when a patient's story during an advisory committee meeting helped push the agency to fully approve the treatment, rather than offer accelerated approval. Bryan said the woman described the disappearance of lesions on her father "influenced heavily our decision to grant a regular approval."

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Categories: Biologics and biotechnology, Drugs, News, US, CBER, CDER

Tags: Pazdur, Marks, Woodcock, oncology center of excellence

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