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As healthcare leaders and policy experts come together for the annual Food and Drug Administration (FDA) CMS Summit in Washington this month, much of the focus will be on how quickly both agencies are evolving to meet the needs of patients. By most accounts, 2017 is gearing up to be a year in which the voice of the individual patient will be front and center in historically bureaucratic and highly-technical environments. 

Case in point is the discussion surrounding the first-ever FDA-approved drug to treat patients with Duchenne muscular dystrophy (DMD) — Eteplirsen, now known as Exondys 51.

The FDA made its decision in September, but there is a lingering debate as to whether the clinical trials used were sufficient for approval. Despite numerous DMD experts weighing in (not to mention patients and families who were directly part of the trials), the evaluation of the medicine came about at a unique time in biomedical innovation.
Many of the breakthroughs that will eventually lead to treatments that turn today’s fatal diseases into chronic and manageable ones do not closely align with FDA’s expectations for large, randomized, placebo controlled trials. In short, new innovation further reinforces the need for evolution of our nation’s medical regulatory systems.

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