December 31, 2016

FDA Reform

Gotta be optimistic on New Year's Eve!

The WSJ Ed Page suggests that a Trump Administration might reform the FDA -- at least as far as slanted drug committees which provide a platform for bureaucrats without balance rom developers.

This was the process used to dismember a compound for Duchenne Muscular Dystrophy (yes, one I have bored you with several times before). A look into how bad this actually was is startling.

"Do the clinical results of the single historically-controlled study (Study 201/202) provide substantial evidence (i.e., evidence from adequate and well-controlled studies or evidence from a single highly persuasive adequate and well-controlled study that is accompanied by independent findings that substantiate efficacy) that eteplirsen is effective for the treatment of DMD?"

Are you still reading? The obfuscation led one committee member who voted no, Bruce Ovbiagele, to deliver this insight: "Based on all that I heard, the drug definitely works, but the question was framed differently." Imagine hearing this if you are the parent of a Duchenne boy.

I've suggested admittedly extreme extreme proposals for revamping the FDA, and I'd still love to see them. But, in the real world, all that is necessary is to change the bias from "we must find the reason to deny this" to "we must accept this unless we find something contrary." That would take us 85% of the way there.

On the plus side, this will likely be my last FDA post of 2016.

Trump Revolution Posted by John Kranz at December 31, 2016 11:43 AM

If President Trump or his administration actually do make this change, I will expect to hear it here first! ;)

Fingers crossed.

Hey, any word yet whether President Trump intends to continue that awesome "petition the White House" thingy that Obama invented?

Posted by: johngalt at January 3, 2017 2:23 PM | What do you think? [1]