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Whitehouse petition: Urge the FDA to Say YES to Accelerated Approval for children with Duchenne.

WE PETITION THE OBAMA ADMINISTRATION TO:

Urge the FDA to Say YES to Accelerated Approval for safe, effective therapies for children with Duchenne.

We urge the Food and Drug Administration (FDA) to use the Accelerated Approval pathway for approval and access to safe, effective therapies for Duchenne Muscular Dystrophy - the leading genetic killer of children that impacts 1 out of every 3,500 boys born in the United States. It's time for the FDA to Say Yes and make this the first generation of Duchenne survivors.

https://petitions.whitehouse.gov/petition/urge-fda-say-yes-accelera...

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Permalink Reply by Keith Van Houten on March 11, 2014 at 5:55pm

Drugs are approved by the FDA on the basis of clinical benefit. If the biomarker dystrophin is approved as a surrogate endpoint, it will allow faster studies, under the accelerated approval pathway. Because, you'd be able to get an interim approval based on the surrogate, without waiting to see clinical benefit. You still have to show clinical benefit in a confirmatory trial to get full approval. I can't see how surrogate endpoints have anything to do with the size of trials. Trial size is related to what's required to show statistical significance of the results.

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Permalink Reply by Tommy Paine on March 11, 2014 at 6:01pm

But you are saying that the medicine would get to people sooner, rather than later, with accelerated approval, right? with the condition that the FDA could yank it if the positive Phase II results somehow reversed in the required ongoing confirmatory trial? Thanks, Keith.

Keith Van Houten said:

Drugs approved under accelerated approval with a dystrophin surrogate endpoint would still require a confirmatory trial with a clinical endpoint, like the 6MWT, to receive full FDA approval. If no clinical efficacy is shown in the confirmatory trial, the drug would be removed from the market.

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Permalink Reply by Jason Darienzo on March 11, 2014 at 6:03pm

But if they use 6mwt to approve dystrophin biomarker logically they both are equal in showing a clinical benefit.

To bad logic and government don't work together

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Permalink Reply by Keith Van Houten on March 11, 2014 at 6:23pm

Yes, that's true, Tommy. If the drug gets accelerated approval, it's able to be sold to the public while the confirmatory trial is running.

Whether the general public can access it during that period depends on whether the company can physically produce enough drug to sell it to anyone that isn't participating in the confirmatory trial. There might not be enough to go around if a company decides not to invest in enough manufacturing capacity for everyone that wants it, before they know for certain if they're going to be able to sell it permanently.

It also depends on whether insurance would cover it. There seems to be precedent that it would, although I asked my insurance company about it, and they said no. But, I'm not certain they knew what I was even talking about. Also, past precedent might not mean anything when it comes to a drug that will probably cost $400,000 a year.

Cautious optimism is the key.

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Permalink Reply by Keith Van Houten on March 11, 2014 at 6:26pm

You don't know that the biomarker indicates clinical benefit until you run the large scale confirmatory trial with the clinical endpoint.

Jason Darienzo said:

But if they use 6mwt to approve dystrophin biomarker logically they both are equal in showing a clinical benefit.

To bad logic and government don't work together

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Permalink Reply by Jason Darienzo on March 11, 2014 at 7:16pm

Sarepta tried with phase 2 data and it shows a clinical benefit? If someone like Dr. Louis M. Kunkel who discovered the gene in 1987 thinks its working, I side with him over, the FDA's limited experience with DMD.