Statement from FDA Commissioner Scott Gottlieb, M.D., on new agency efforts to advance the patient voice in medical product development and FDA regulatory decision-making

June 13, 2018

Over the past decade, advances in our understanding of the basic biology of serious and life-threatening diseases has led to the development and FDA approval of targeted treatments for patients with a specific molecular sub-type. These more targeted treatments often address unmet medical needs. They can represent significant improvements over the prior standard of care.

This medical progress is the result of scientific advancement. But it’s also a consequence of a bipartisan consensus that has supported investments in medical research and a modern regulatory process to advance safe and effective therapies.

For instance, Congress codified the FDA’s early and intensive engagement with sponsors through the Breakthrough Therapy designation. This pathway helps advance treatments that demonstrate significant clinical benefits over the current standard of care. This designation was a recognition that as the science for drug development advanced and enabled more targeted approaches to the treatment of unmet medical needs, the FDA needed the ability to work more closely with sponsors to ensure that trial designs and endpoints were appropriate for the type of therapy and the disease state under consideration.

The benefits of these more efficient approaches can be seen in the treatment of diseases like cancer; where the FDA has modernized the trial designs and endpoints we accept to demonstrate efficacy for some subtypes of tumors after numerous consultations with stakeholders. This includes the support of the FDA’s Oncologic Drugs Advisory Committee, as well input from a diverse group of experts, researchers and especially the feedback we receive from patients and their families.

The FDA has the full statement

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