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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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.

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.       

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

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.  

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

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.

Sarepta didn't even apply for accelerated approval, and the FDA hasn't said no.  They've asked for more information.  

Most people think it's working, including me.  It doesn't matter what anyone thinks.  It matters what you can prove to the satisfaction of the FDA. 

Update

Signatures needed by March 29, 2014

78,737

Total signatures on this petition

21,263

keep it up

Signatures needed by March 29, 2014

75,859

Total signatures on this petition

24,141

If anyone knows someone on tv  twitter and any media please attempt to contact them.  let's get the signatures we need. 

Signatures needed by March 29, 2014

73,859

Total signatures on this petition

26,141

Signatures needed by March 29, 2014

71,587

Total signatures on this petition

28,413

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

Signatures needed by March 29, 2014

69,408

Total signatures on this petition

30,592

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