#SaveJack, #BeBrave

Posted Friday January 17, 2014 by Melissa

I’ll never feel okay about kids dying. Never. And I’ll surely never feel okay about my best friend’s son dying.

But in the whole convoluted process that is drug discovery, clinical trials, compassionate access, and drug approvals, swirled in with advocacy for our kids on a day to day basis, I don’t think that anyone would argue that I always have the best interest of all our kids in mind. All of them. I have never looked at the idea of giving Jack compassionate access as one that endangers the use of this drug for everyone, and I doubt any other families in the trial have either (having spoken specifically about this issue with a number of them, I can honestly speak here), but Shire does and I find that difficult to swallow.

I advocate in the most effective way and means I know how, using my legal and strategy background to do so, to gain access to lifesaving treatments for every child, as soon as it is possible. For Jack, every other piece was in place, so here we are.

Below is another great blog by Andrew McFadyen, on behalf of Jack Fowler and the Fowler family, for compassionate access to IT Idursulfase.


On October 11, 1988, ACT UP had one of its most successful demonstrations (both in terms of size and in terms of national media coverage) when it successfully shut down the FDA for a day. (Source: Wikipedia)

On some level, I get that Shire is afraid of adverse events, but aren’t I more so as a parent of a child receiving the drug? And wouldn’t the Fowlers be more so, as Jack’s parents? So if we are all willing to move forward, shouldn’t they be also?

What it boils down to is this. A fear that if there were an adverse event, outside of the group that meets strict criteria for the clinical trial, that it could endanger the FDA’s approval of the drug.

But until pharmaceutical companies focusing on rare, progressive, and fatal childhood diseases stand up to their fear of the FDA’s actions in the event of adverse events, and believe in the communities they are serving, we will forever be watching kids die for the many, many years that drugs run through validation after validation of clinical trials, even when companies find a drug that truly saves children who are dying. My gut wrenches every day, knowing the funerals I will attend in the next four to five years while we await the Phase II/III trial and drug approval.

Don’t they realize that if the FDA even contemplated placing a hold on continuing the drug for my child and others that I would be chained to their doors? The AIDS Act-Up demonstrations would be relived if this drug were pulled. We love our children.that.much. Hunger strike, chaining, arrest. Don’t you love your children that much also? Was spending a week at the FDA last week not enough to show that I would sacrifice everything to make sure this drug gets to all our kids, even if it’s just a delay, much less if it were an actual clinical hold because of an adverse event?


Ultragenyx compassionate use dosing, even during a Phase I/II trial, for MPS VII or Sly Syndrome

And other companies are saving lives in such circumstances, that is what I struggle with. I continually admire Dr. Emil Kakkis, who often leads the way for our MPS and rare disease communities, in both his innovation and his leadership. Ultragenyx, of which Dr. Kakkis is the CEO, has an ERT for Sly Syndrome / MPS VII, an even rarer disease than Hunter Syndrome / MPS II, and they are giving compassionate access here in the U.S. even while their Phase I/II (they are in an even earlier step than Shire is now) is going on in the UK.

Secondarily, there is a fear of wide open doors of patients seeking compassionate access if it were given to Jack. Really? Wow, patients who need a drug so they won’t die who actually want access to it? So the response is, let’s not give it to anyone.

Realistically, however, compassionate access through an individual IND or emergency IND is by its nature and by FDA regulation an INDIVIDUAL inquiry (see FDA guidance). The FDA is required to evaluate each patient and their medical and other circumstances individually. If a patient qualifies for a current clinical trial, they are not even eligible. If the medical circumstances are deemed too risky, they are not eligible. If the FDA deems that it would endanger successful enrollment of the trial, they are not eligible. The FDA makes these determinations. Shire is preventing them from doing so and giving Jack a fair shot at access to this drug. The spurious argument about other patients is irrelevant in the context of what is required to be an individual inquiry.

Entire article

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