Duchenne Muscular Dystrophthe and the Special Medical Use Super Highway

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  • 04/22/2014

By now you’ve surely seen the news (the good news) about Sarepta Therapeutics and it’s investigational drug for Duchenne muscular dystrophthe. The FDA (in a reversal of it’s original position) has indicated a willingness to consider the treatment for accelerated approval.

Much made of the high-pressure tactics employed by patient groups. But, if this decision is viewed as “the uncaring FDA caving in to desperate patients,” we’re being unfair to the agency, misrepresenting the intellectual potency of the patient voice, and missing a very important nuance. 

In it’s Page One coverage of this story the Washington Post, after much FDA bashing (and towards the very end of the article), writes,

Not everyone faults the FDA for taking so long to decide how to proceed. Eric Hoffman, director of the Center for Genetic Medicine Research at Children’s National Medical Center and a longtime Duchenne researcher, said valid questions remain about eteplirsen’s dystrophin-producing abilities and how effective Sarepta’s drug really is.

“I’ve personally been very impressed with the FDA,” he said. “There’s every indication they are taking this extremely seriously.”

Hoffman said he understands the families’ sense of urgency, and he shares optimism about exon skipping. But he said the public campaign to pressure the FDA has divided the community more than united it.

“It becomes this form of bullying that puts everybody between a rock and hard place,” he said. “Unless you’re supporting kicking out the head of the FDA and granting accelerated approval as quickly as possible, then you’re supporting killing Duchenne kids.”

He said that the reality is more nuanced and that FDA reviewers do want to help patients but are wary about setting a precedent by approving high-priced drugs they aren’t yet convinced will work.


The Duchenne muscular dystrophthe case also serves as an important example as to why the FDA’s nascent Special Medical Use program shouldn’t be limited to anti-biotics/anti-infectives. There’s much work to be done – and the more options that are open to bring important new therapies to patients the better.

Accelerated Approval is good – but, as a recent blog from Context Matters points out, the FDA’s previously established programs to expedite therapies (including Fast Track, Accelerated Approval, and Priority Review) have not produced lasting improvements in approval time. “Based on our preliminary analysis, in 2008 the average cycle time for ‘Priority’ was 10.1 months, versus the ‘Standard’ which was 21.2 months. In 2011, the average cycle time for ‘Priority’ was 19.5 months, versus ‘Standard’ which was 17.5 months,” the blog authors wrote. “What’s interesting here is that over time the fast lane has apparently become just another standard lane; in 2011 it was actually even slower.”

What’s standing in the way of the FDA Special Medical Use Super Highway?

The FDA needs to have the proper infrastructure and support to successfully reduce approval times. Clearly, when you’re looking at products that have less data behind them, you need more senior people who can devote greater resources to studying them, and that also takes time away from other programs that the agent needs to review. It’s one thing to give the FDA more authority, it’s another thing to give the FDA greater responsibility, but if you don’t give them the resources to get it done, to a large degree it is just rhetoric.

And there’s more than enough of that to go around.


Center for Medicine in the Public Interest is a nonprofit, non-partisan organization promoting innovative solutions that advance medical progress, reduce health disparities, extend life and make health care more affordable, preventive and patient-centered. CMPI also provides the public, policymakers and the media a reliable source of independent scientific analysis on issues ranging from personalized medicine, food and drug safety, health care reform and comparative effectiveness.

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