Looks like the judge sided with the Gunvalson's. He's ordered that PTC Therapeutics make PTC124 available to them...
NY Times Article

For those not following - here's a history of articles:
Original lawsuit article: http://www.nytimes.com/2008/07/17/business/17dystrophy.html?pagewan...
Follow-up article on case:
http://www.nytimes.com/2008/08/20/business/20dystrophy.html?ref=hea...

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Been thinking on this a bit...
I can understand fighting the case in court, to dissuade every parent out there (including myself) from saying - "Give it to my son, too". But why appeal it, it seems to me, from reading the articles that the Gunvalson's argued their case on more points than just "Compassionate Use". So why not just add them into the 2a extension study?
I wish there was someone who truly knew all the implications for everyone involved. How does this affect the future of this one boy? Will his part of the trial be different? How does this affect all boys involved in the trial? What about other non-ambulatory boys? How will it affect PTC financially and ultimately will it affect getting this drug FDA approved for all? How will if affect Project Catalyst and how they run future trials? Will it change the information PTC shares with PPMD & families in the future? How will it affect other clinical trials, in all disorders???

I feel like there is so much else involved in this, than just this one boy. . . but, what do I know anyway?!?!?

Ang ;~)
Angela said:
...I feel like there is so much else involved in this, than just this one boy. . .

Yes... lots of unknowns... hopefully it all works out in the end...
Our local news was there to interview Cheri yesterday, here is the link if anyone is interested. For those that are not lucky enough to know Cheri and all she has done for our boys and DMD research this is a great time to learn more about her!
http://www.kare11.com/news/news_article.aspx?storyid=522424&cat...
We all can appreciate what the Gunvalson's are going through - we all want a miracle, and now! But I do worry how this will impact the future of the current study, and if it will distract PTC's research, or divert funds from research into litigation.

I was disappointed my own son couldn't participate in the 2b study b/c of his age. I'd argue "better he participate early when he still has the most muscle function" rather than to wait until he gets worse. But I'd rather see a pure, controlled, rigorous study completed that will give the FDA what they are looking for, in the most timely fashion, to potentially approve the drug for widespread consumption (by boys of all ages).

What if Jacob develops complications that ambulatory boys in the study don't? Will that hold up drug approval? What if he improves, but not enough per the FDA?

I think "compassionate use" is a valid argument, and if the Gunvalsons had based their claim just on that point I wouldn't have a problem with it.

But, I don't think they should have used their cozy relationship with the company as part of their claim. It taints the "compassionate use" argument and makes you feel that they ingratiated themselves, but are disgruntled they didn't get preferential treatment in return, even though the company supposedly "promised." They never thought to say "Really, we can have it when the time comes? Great, then put that in writing." Does a sleepover at a PTC employee's house count as a binding agreement in court? People and companies can "promise" lots of things, but if you don't have it in writing...

I just hope the study and the research aren't derailed by any of this controversy...
I hope this does not affect Diane Goetz job. She has always been very pleasant to talk to. She has been great to communicate with. Hope this does not change the company's policy on being open and talking to us.
Angela said:
Will his part of the trial be different? How does this affect all boys involved in the trial?

According to the FDA rep @ conference, if someone is accepted for Compassionate use, then that patient(s) would be monitored differently. They would basically have their own trial.

But this isn't compassionate use at all, so who knows how it will be handled. I wonder if the FDA can step in and over-rule the judge in this case?
Hi

Its Mark here from the UK - new to the forum. I think as long as this is treated as a seperate study - rather than being incorporated into any double blind random testing - they should be able to isolate any anomalies in the data arising from the inclusion of this additional patient.

Whether or not this is compassionate, do we know if it can be treated as a trial of its own? Would the FDA agree to this, given the circumstances. I don't see that the family would be concerned with that - in fact, they might they are not getting a placebo.
They will get THE drug if granted approval. That's why ptc said it's such a danger. People won't want to participate in the study (and maybe get placebo) if they can sue for the actual drug.

Marks33 said:
Hi

Its Mark here from the UK - new to the forum. I think as long as this is treated as a seperate study - rather than being incorporated into any double blind random testing - they should be able to isolate any anomalies in the data arising from the inclusion of this additional patient.

Whether or not this is compassionate, do we know if it can be treated as a trial of its own? Would the FDA agree to this, given the circumstances. I don't see that the family would be concerned with that - in fact, they might they are not getting a placebo.
On what basis would others be able to not participate in the trial and sue? On the basis that there is a boy who has been able to bypass the trial and definitely receive the drug. But presumably no-one else can claim the special circumstances of this family which, rightly or wrongly, are behind their case.

Forgive my ignorance, but in the USA, when a drug is at 2B stage, can a company give out a drug like this outside of a trial? Would the FDA permit this. If not, perhaps this is something which an appeal court would overrule.
I just found this article and I thought it would be an interesting addition to this discussion:

One Disease, Two Legal Positions in 3rd Circuit Experimental Drug Case
Zach Lowe
The American Lawyer
December 18, 2008

Jacob Gunvalson is a 17-year-old boy with a terminal genetic disease called Duchenne muscular dystrophy, but he was not allowed to participate in a drug company's trial of an experimental drug that holds some promise as a possible cure.

Nicolas Fuca is a 13-year-old boy with the same disease, and he, too, was not allowed into the same company's experimental trials because he cannot walk --a key standard for participation.

Two boys, two very similar tragic situations. But their families were on different sides of a landmark case in the 3rd U.S Circuit Court of Appeals that helps set the standard -- for now -- that families should not be able to use litigation to force drug companies into providing experimental drugs before trials prove their safety.

The decision was a painful defeat for the Gunvalsons and their counsel at Saiber in New Jersey (home state of the drug company in the case, PTC Therapeutics) and Blackwell Burke, a boutique in Minnesota (the Gunvalsons home state). A federal district court had ruled in August that the PTC and the Food and Drug Administration should start the process of getting Jacob the experimental drugs. Judge William Martini of federal district court ruled Jacob deserved access to the drugs because PTC "promised" his family that Jacob could get them outside the trial process, for which he was ineligible because of a heart condition and kidney problems.

The company and its lawyers (led by John Hutchinson and John Lavelle at Sidley Austin) appealed to the 3rd Circuit, arguing that courts should not be able to force a company to provide drugs to patients before they are sure about their safety.

That's where Nicolas Fuca came into the case. The Fucas agreed with PTC, and they got Jones Day (and partner Mark Herrmann, co-author of the Drug and Device Law blog) to write an amicus brief explaining why the family of one boy with Duchenne would side against the family of another.

There are two reasons, it turns out. The first is that the Fucas fear that litigation like the Gunvalson case will discourage patients from enrolling in clinical trials and therefore limit a drug company's ability to do solid research on potential products. Why? Because if a patient's family can sue to get access to an experimental drug before a trial is over, more patients will go to court instead of enrolling in a trial in which they might get a placebo and not the actual drug. Second, punishing PTC for statements it made to Gunvalson's family (statements the 3rd Circuit found to be vague and not definite promises) would discourage drug companies from openly communicating with patients.

Hutchinson and Lavelle declined comment on the case, citing PTC's restrictions. A PTC spokeswoman told the Am Law Daily the company would not permit the lawyers to talk to the media. Dechert partner Robert Heim, who also represented PTC, did not return calls seeking comment.

Saiber's Marc Wolin, who argued the case for the Gunvalsons, also declined comment.
Recent development - decision was reversed on appeal:

http://druganddevicelaw.blogspot.com/2008/12/gunvalson-v-ptc-therap...

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