Why it’s time to turn the page on the FDA drug approval process
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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.

No one would argue that the FDA needs to be thorough in ensuring that drugs are both safe and effective before they become available to patients. Our drug review-and-approval system is the best in the world in protecting the public from potential harm and it should remain so.

Eteplirsen was given the go ahead as part of the FDA’s accelerated approval process, which dates back to 1992 and is designed to get safe and effective medicines to patients when no other options exist. It’s made a world of difference in approving treatments for cancer, HIV/AIDS and many other diseases that don’t yet have a cure. In 2012, the Food and Drug Administration Safety Innovation Act (FDASIA) greatly expanded the scope of accelerated approval.

Unfortunately, the internal disagreement over the first DMD treatment has carried over into the public arena, placing leaders at the FDA in a difficult position at a time when it’s critical that we focus on the future of treating rare disease. Both sides have valid points and it’s worth noting that scrutiny in any review process is healthy. However, as FDA Commissioner Dr. Robert Califf noted in a memo released in conjunction with the agency’s approval, “the science is not in dispute beyond the usual types of disagreement that occur when experts review clinical evidence from different perspectives.”
Above all, it’s important to realize that illnesses for which there are no available treatments can simply not be viewed through the same prism as those diseases that have experienced more successful drug development. DMD is a genetic disorder that primarily affects young boys, usually showing initial symptoms between the ages of three and five. Until an approved treatment came on the scene, there was no treatment and many experts predicted we could be years (if not decades) away from getting to patients.

Second, when it comes to combating rare diseases, policymakers need to recognize the reality of numbers, both in clinical trial execution and risk assessment. Populating clinical trials is not an easy process under any circumstances, but it is far more difficult when dealing with a disease that affects just a few thousand boys. When testing and evaluating drugs for diseases affecting a small population, decisions must reflect the different scope of the playing field.

The same applies to how the FDA deals with risk. For the DMD treatment, testing revealed no potential threats whatsoever to patient safety, so this was never an issue. For other rare disease treatments, the agency must recognize that an adverse event will have a much smaller impact on population health than a regulatory error concerning a more widely used medication. Realizing this, the question becomes, is the affected patient population willing to assume this risk in order to have swifter access to a potentially effective therapy? 

Finally, we must consider the patient voice. In the case of DMD, families made it clear that they wanted access to this medication, fully understanding the fully reasonable limitations in clinical testing that are inherent in an any accelerated approval. It is entirely appropriate and morally correct for the FDA to give significant weight to the positions of those, and their loved ones, regarding accessibility of a medicine that may change their lives and futures.

How the FDA deals with these issues in the future will have far-reaching ramifications. Americans afflicted with rare diseases shouldn’t face more daunting disadvantages simply because of their limited population size. Nor should they be subjected to a public airing of differences months after approval.

In the case of Eteplirsen, the process worked. It’s time to turn the page and focus on how we build on it.

Kenneth Thorpe, Ph.D., is a Robert W. Woodruff Professor and Chair of the Department of Health Policy & Management in the Rollins School of Public Health at Emory University, Atlanta, Georgia. Dr. Thorpe was Deputy Assistant Secretary for Health Policy in the U.S. Department of Health and Human Services from 1993 to 1995.

The views expressed by contributors are their own and not the views of The Hill.