Pazdur, Marks and Woodcock Discuss FDA Changes, What’s Coming

  • June 18, 2019

Center for Drug Evaluation and Research (CDER) Director Woodcock said that the difference between years past, when FDA was viewed negatively, and now is that there has been a sort of scientific revolution, with biology being harnessed in novel ways and investments at NIH paying off.

Oncology Center of Excellence Director Pazdur also noted the tremendous growth in understanding diseases and in the immunology of cancer.  “One part of the piece in oncology is that our staff has embraced these changes,” he said, noting they’re “not just bean counters for survival data” but have gotten involved in the science of oncology.  The staff are partners in the process rather than just ticking boxes, he said.

And Marks, director of the Center for Biologics Evaluation and Research, explained how the idea of screening libraries of compounds has gone out the window, while nowadays it’s more about rational drug development – “and gene therapy is the epitome of that.”

On the topic of gene therapy, Marks noted there are now 700 investigational new drug applications before FDA.  However, compare that with cell therapies, he said, noting a gray market of unproven therapies, with people hawking snake oils that are not even well-manufactured and in some cases do not even contain live cells. He also said the use of unapproved stem cell therapies around the country is a surrogate marker for how Right to Try may play out.  FDA does not usually learn about what is going on until something really bad happens, Marks added.

On the prospects of what’s to come at FDA, Woodcock noted the real-time review pilot in the Oncology Center of Excellence, which reviews parts of a submission before the entire application is submitted, is “foreshadowing something bigger.”  But she also explained how the speed of reviews does not really influence the results  – “What has changed is the cost of these drugs, and it’s changed the debate because of value.”

Nowadays and moving into the future, there’s a societal benefit and harm discussion that implicitly invokes “trade-offs in value – that’s the new debate we’re having  – too fast and too slow is really not the issue.”

For Marks, the biggest problem moving forward for gene therapies is the quantum leap needed in manufacturing and to apply the therapies to not just rare diseases.  “We need to do the kind of transformation Steve Jobs did for digital” with manufacturing gene therapies.  He also noted FDA is “very lenient” about some of its manufacturing standards for getting gene therapies into Phase 1 trials, but scaling up the manufacturing is what’s difficult.

Moving into the future legislatively, Woodcock said she’s interested in legislation on generic drug labeling.  “We have to clean up the armamentarium so it doesn’t get out of date,” she said.  She further noted FDA is working with Congress on over-the-counter (OTC) drug reforms, which is the basis for the Office of New Drug (OND) modernization.  “If we don’t get the OTC reform, we’ll have to pull back on the OND reforms,” she said.

REFERENCE:  RAPS Regulatory Focus; 14 NOV 2018; Zachary Brennan

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