Comments on: Judge: Drugmaker Must Provide Experimental Med

By: HF

HF — Thu, 21 Aug 2008 22:49:52 +0000

Actually, the boy CAN get treatment outside of the clinical trial. His physician (not PTC)would need to file an Emergency Use IND (often called Single Patient IND in the oncology world). With this comes the same responsibilities that come with any IND: (1)everything is reviewed and approved by an Institutional Review Board, and (2) adverse events, clinical updates, and annual reports are filed with FDA.

For products that have shown great clinical promise, there is the Treatment IND, which can open up the drug/biologic to larger groups of people while the licensing application is under review. There are qualifications on this, of course. The disease has to be a serious or immediately life-threatening disease or condition that has no satisfactory approved therapy. But many thousands of AIDS sufferers got access to antiretrovirals that way. “Compassionate Use” INDs are Treatment INDs.

So there ARE avenues for patients to get access to experimental treatments when all other options have been exhausted. And the FDA isn’t likely to say no, as long as you’ve got yourself a decent case. You just have to have the company willing to give the drug to your physician. (Oh, and have a physician who’s willing to do the IND filing and follow-up. No small potatoes.)

By: Joan

Joan — Thu, 21 Aug 2008 20:11:45 +0000

I would assume one thing about the FDA: It is a failure.

By: Nathan

Nathan — Thu, 21 Aug 2008 16:38:35 +0000

“I would assume any FDA review would take into account the circumstances around this high-profile data point if it were indeed problematic.”

Given all the recent scruteny and accusations, I wouldn’t assume anything about the FDA…

By: Question

Question — Thu, 21 Aug 2008 16:11:18 +0000

Of course, if there were an adverse event, maybe it SHOULD jeopardize the future of the drug. ;-)

Honestly, given the circumstances, I would assume any FDA review would take into account the circumstances around this high-profile data point if it were indeed problematic.

I do agree that it does seem a bad precedent to allow the courts to mandate entry into a trial.

It seems to me that there should be some other avenue for patients to get access to experimental treatments when all other options have been exhausted.

By: Nathan

Nathan — Thu, 21 Aug 2008 15:44:05 +0000

1) I don’t believe he can under current regulations. Can anyone else comment on this?
2) Even if he could recieve the drug “outside of the clinical trial”, an adverse event would still be REQUIRED to be reported and could jeapardize the future approval of the drug.

By: Christopher

Christopher — Thu, 21 Aug 2008 15:40:20 +0000

Good point. I’m a little rusty on qualifying criteria for compassionate use but that may have been an alternative. Who pays under that method is in question, and of course the company still must move its regulatory program ahead so that, as Nathan says, others might benefit in the future.

Tough position and motives from both sides are understandable, but this sets an unfortunate precedent.

By: Question

Question — Thu, 21 Aug 2008 14:33:16 +0000

In this case, if he gets access to the drug does that mean he is included in the clinical trial data pool?

Can’t he get access to the treatment without being enrolled in the trial?

By: Nathan

Nathan — Thu, 21 Aug 2008 01:52:09 +0000

FPME - that just isn’t true. What if the child dies soon after taking the drug? What happens? Is it the drug or is it the complicated situation facing this individual child who is already on other medication? An adverse event with this child could put the drug in jeopardy and will thereby put the lives of future children in jeopardy.

Science has to happen in a particular sequence. We CAN’T understand the mechanism of unexpected events unless the experiments take place in a carefully controlled environment.

By: Former pharma Marketing Exec

Former pharma Marketing Exec — Wed, 20 Aug 2008 23:10:18 +0000

Nathan,

In instances such as this where this boy is in terminal end stage disease, this disease is very severe with no known cures available and this drug being the only option left, then this boy has every right to access this drug.

This is exactly what the Abigail Alliance is all about.

Your claims that this could in any way impede further clinical development of this drug is completely false. This case along with all other participants can only serve to help science understand this drug and how it works.

PTC sounds like a great company - hats off to them in trying to work closely with the patient community and being compassionate about this debilitating disease. This disease strikes between 400 to 600 male babies born in the US each year. This is an orphan disease and this company should be warmly welcomed in this disease segment. I hear they are poised ot do some good things in Cancer too! I like the way they focus on RNA.

By: Nathan

Nathan — Wed, 20 Aug 2008 20:48:48 +0000

If upheld, this sets a horrible precedent. If you want to participate in a particular clinical trial, just sue.

While I feel bad for this boy, in the end his quest to take this drug may ultimately result in the delay of it’s approval for the treatment of others with his same condition. Early (phase I and II) clinical trial participants are chosen to be relatively homogeneous so that the results will reach statistical significance even with a small number of patients enrolled. By forcing his way in, he could possibly tip the results such that additional clinical trials would be required to reach satisfactory statistical significance. His small battle may be won, but it is an overall setback for the treatment of Duchenne muscular dystrophy.