The FDA’s Accelerated Approval Process is Working

It goes without saying that the passage of the 21st Century Cures Act by an overwhelming bipartisan majority vote will help speed up the delivery of innovative treatments to patients. There’s now an opportunity to take a step back and assess how we can build on this bipartisan achievement.

Anyone who pays attention to the debate over federal approval of newly-developed biopharmaceutical therapies knows that there has been a yearslong tug-of-war between those who want to get new innovations to patients more swiftly and opponents who believe speeding the drug approval process will pose threats to patient safety.

As officials from the U.S. Food and Drug Administration and the Centers for Medicare and Medicaid reflect on their recent annual summit, there should be agreement that one significant solution to this debate already exists in the form of the FDA’s accelerated approval process. Policymakers should, in fact, be focusing on ways to bring greater uniformity to the way in which this program is utilized, thereby bringing hope to a greater number of Americans struggling with serious illnesses, including autoimmune diseases that have few or no treatments.

The FDA launched the accelerated approval process in 1992, primarily as a response to public demand for medications to address the growing HIV/AIDS crisis. The criteria was clear as to which new drugs would be placed on the speedier road toward regulatory approval. They had to address unmet medical needs and would have to demonstrate indicators that offered the promise of clinical progress. These medications are then subject to post-market studies to affirm their efficacy and safety.

The latest example showing how this process acts in the public’s best interest was seen in the FDA’s approval of eteplirsen, the first-ever FDA-approved drug focused for a rare subset of Duchenne muscular dystrophy. Duchenne is a devastating illness that overall affects between 9,000 and 12,000 boys in the United States. The disease severely weakens their muscles and affects their heart and respiratory functions, leaving them confined to a wheelchair by their teens and invariably shortening their lives.

For autoimmune diseases, the implications of how accelerated approval was applied in the eteplirsen case (as well as many others) cannot be understated. We need to take incremental steps prior to achieving the breakthroughs that will someday lead to outright cures. To reiterate, improved consistency in applying the accelerated approval process for a wide range of disease categories must be a priority.

In the FDA advisory committee debate over eteplirsen, it was made clear that drugs must show a meaningful level of efficacy before accelerated approval could be considered. Even then, the FDA requires confirmatory trials to verify the anticipated clinical benefit, with patient access as a key part of the post-approval environment. As Janet Woodcock, Director of the Center for Drug Evaluation and Research at the FDA, noted in an interview with CNBC this month, the agency is constantly keeping in mind the need to balance patient access with safety and efficacy. “This is part of the overall way we’ve been doing business for a long time,” said Woodcock.

Without question, the use of accelerated approval would benefit autoimmune diseases such as Behcet’s, Lupus, pemphigus, pemphigoid, myasthenia gravis, vasculitis and many others — all of which are awaiting new treatments.

In developing future policy, the process for approving a treatment for boys with Duchenne muscular dystrophy should serve as an illuminating pathway. Where there is unmet medical need, particularly for those facing a short and difficult life because of their illness, expediting innovative treatments is simply the right thing to do. This shouldn’t be considered debatable, but rather a model for others to follow.


Virginia T. Ladd is President and Executive Director of the American Autoimmune Related Diseases Association. 

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