April 29, 2016

Your FDA at Work

I look forward to the day I can stop posting these stories and stop complaining. But this tale of the FDA blocking treatment for Duchenne Muscular Dystrophy hits too close to home. I saw a video of a mother with two sons. Both had Duchenne. One was in the trial and had to watch his brother deteriorate because he could not get this treatment. You'd think the FDA would at least let Mom decide which kid she wants to let the government kill.

No, measured speech will not be employed in this post. I have been on trials for nine years and have seen how resource-intensive they are. Big Pharma has spent a ton of dough on me: some to develop and test product, but as much or more to satisfy a government which keeps the compound that helps me away from the others in the infusion room. At least they're not my brothers.

A small company like Sarepta can't summon the resources to produce unlimited drug doses at government whim, but set that aside. There aren't enough patients for such a double-blind trial. An estimated 12,000 boys in the U.S. suffer from Duchenne, but only 13% have the mutation amenable to eteplirsen (more iterations are on the way, unless FDA prevails). Many patients have deteriorated too far to be eligible or live too far from a city with a trial.

FDA's demands also violate ethical standards of medicine. Eteplirsen requires a weekly muscle infusion, with biopsies that are exhausting and risky for someone with a degenerative neuromuscular disorder. The agency would ask kids to be lab rats and lose the ability to walk or catch a ball while receiving intensive injections of what might be sugar water.


Well, those 12,000 boys can just wither and die I guess. I'm sure a new study will help the next generation of patients. Oh. Wait.
FDA has suggested no alternative path to approval.

JS Mill weeps. If you cannot read the story, let me know and I will smuggle through the paywall.

UPDATE: Here is the story I mentioned of the mother with two sons.

Pharmaceuticals Posted by John Kranz at April 29, 2016 10:30 AM

Your words are far less outraged than you would be entitled to use.

Unlike Rick Perry, I would have no problem at all naming three Federal agencies that I would take a chainsaw too, and I wouldn't need to trade any draft choices to make the FDA a first-round pick (hey, by the way, Paxton Lynch? Great choice, especially under the tutelage of the right QB coach.). If we were a free society, this mother - and for that matter, you - would be able to walk into a pharmacy, point, and say "our doctor says we're going to try that one." A willing buyer and a willing seller shouldn't need to deal with a nosy government coming between them.

With respect to your personal stake in this, I will yield the floor to you. Don't apologize for making a full-throated protest at this government's unfitness to deal with the circumstances.

Posted by: Keith Arnold at April 29, 2016 12:47 PM

Double-blind trials suck. I read an article on cancer immunotherapy studies last week, however, where they give all of the study participants the trial drug. There are no sacrificial placebo patients. I wonder why the double standard?

"I Survived Stage IV Melanoma: How Immunotherapy Saved My Life"
http://www.health.com/mind-body/immunotherapy-treatment
'Health' magazine, May 2016 issue

Posted by: johngalt at April 29, 2016 1:06 PM

Re: Paxton Lynch - In my circles we're pretty happy with the pick. Brock who? Don't know anyone by that name. ;)

Posted by: johngalt at April 29, 2016 1:54 PM

For better or worse, double-blind is the government standard. There are a few oddities. I received routine and expensive MRIs -- but my neurologist was not able to see them. Mmmkay.

When "shopping" for trials, I was very careful to select studies where full placebo was not an option. I was deteriorating quickly and didn't want three years of sugar pills.

My first trial was a combination on two drugs; some got both, some one, some the other. They go through these elaborate double-blinding but both compounds have huge and notable side effects; you knew what you were on in a few weeks, and if you chatted with your Doctor, so did he or she. I gave myself a shot every day for three years that I was 91.753% sure was nothing. Your blog brother is truly dedicated to science.

My second (and successful) one was a Phase II where they were trying different dosages. A bag would show up with my initials on it and they'd insert (IV, not suppository...) it without knowledge of the dose. That probably was a decent variable. [SPOILER ALERT:] That stopped progression of symptoms for all intents and purposes and I am on a continuation trial where I take a known dose and they just make sure I am still alive and have not "grown an extra teat" as my wife tells the story.

Since that time, I have listened to several of Russ Roberts's EconTalk podcasts. If you go by the numbers, the placebo control group in many trials does quite well. That is counter-intuitive and depressing to someone who celebrates innovation as I do. But in many trials it is not a death sentence to draw the sugar card.

I'll call this study different. There is a minimal chance of "just getting better" and I take their point on the stress and potential harm of treatment.

Posted by: jk at April 29, 2016 2:15 PM | What do you think? [4]