A federal judge has ordered PTC Therapeutics to supply an experimental drug to a 16-year-old Minnesota boy who is terminally ill with a rare form of muscular dystrophy. However, the decision offers no immediate relief to Jacob Gunvalson, because the teenager can't begin taking the drug immediately, due to federal regulations, theAssociated Press reports.
PTC Therapeutics plans to appeal the ruling by US District Judge William Martini, who also denied the drugmaker's request to put his decision on hold while it was appealed. The boy's family contended that PTC led them to believe he could participate in a clinical trial of the drug that is being investigated as a possible treatment, but that the drugmaker went back on its word.
For its part, PTC maintained no promises were made, and argued that allowing the teen to join the clinical trial would not be safe and would set a bad precedent that could hurt researchers.
"It's clear to me that if the plaintiff, Jacob, was denied this relief, he would suffer irreparable harm," Martini ruled today, while the teenager sat in a wheelchair next to one of his lawyers. "His condition has already deteriorated significantly in the past year."
The teenager suffers from Duchenne muscular dystrophy, a genetic, degenerative disease that mostly affects young boys. Typically, those who suffer from it die in their 20s because of weakness in their heart and lung muscles. There is no known cure, but the Gunvalsons believe the experimental drug holds hope, according to the AP.
The dispute centers around a clinical trial of PTC124 that included a 28-day preliminary phase in 2005 and a 96-week phase that is about to begin. Gunvalson's parents, John and Cheri, claim that PTC employees - including senior vp Claudia Hirawat, who once hosted them at her house overnight - assured them their son would have access to the drug even though the medication he was taking at the time excluded him from taking part in the preliminary trial.
Later, they discovered that Jacob could not participate in the 96-week trial because he hadn't participated in the preliminary trial. The judge said that while he has conflicting accounts of what the family was told, he found the company was particularly close to the family. "They had a special relationship that this court considers more than typical," Martini said.
PTC president and CEO Stuart Peltz denied that, asserting, "In fact, on the very night Mrs. Gunvalson and her son were staying at the home of a PTC employee, another patient's parent was staying with her as well...In contrast to big pharmaceutical concerns, it is quite natural for our team to form close relationships with patients and other members of the rare disease community," Peltz said in a statement that described the company as a small startup. It has no products being marketed.
"Our relationship with the parents of patients should be taken as evidence of our commitment to the community, rather than as evidence of some promise of special treatment," Peltz said.
PTC lawyer John G. Hutchinson, in arguing for a stay of the ruling, questioned the urgency of the matter. The family was told in January that Jacob would not get the drug but did not sue until July, he said.
Peltz said allowing individual exceptions while developing drugs is counterproductive and could delay getting approvals for the drugs. "If we do, there is serious risk no patients will want to participate in our current clinical trials, in which they might receive a placebo rather than PTC124," he said.
The boy's mother, Cheri Gunvalson, helped persuade Congress to significantly boost spending to find a cure for the disease. In 2001, she teamed with the late Sen. Paul Wellstone, D-Minn., for passage of the MD CARE Act, which increased federal funding to study the disease. The legislation established several "centers of excellence" - since renamed "Wellstone Centers" - for muscular dystrophy research.
Source: Associated Press
10 Comments
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.
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.
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.
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?
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.
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.
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.
"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...
I would assume one thing about the FDA: It is a failure.
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.)