As I work away and prep for Saturday’s meeting between the Fowler Family, The Isaac Foundation, and Shire’s CEO Flemming Ornskov and Head of Research and Development Phil Vickers, I was directed to this youtube video featuring Phil and a few other panelists. While the discussion is interesting, it’s the end quotation from Mr. Vickers that struck me as ironic and, as it turns out, disingenuous based on Shire’s recent decision to deny treatment to Jack Fowler.
In the video, Mr. Vickers states:
We provide Hope to patients and their families. And that’s a very sobering thing. And I would say, the best times of parts of my jobs are meeting those families and some of those patients and seeing the impact we can have on their lives. And some of my worst discussions are with those same families where, there’s somebody that – you know you have to have specific enrollment criteria so you can have a successful study. It’s very tough when somebody has a child that’s outside those enrollment criteria, that may have advanced so far that the chances of them getting benefit are very, very low, and you cannot compromise your study, and you have to say that to a parent. It’s a very, very tough thing to do. And sometimes you can’t even provide therapy, in some cases, outside of the clinical trial because you’ve got no evidence of safety and you’ve got no evidence of clinical benefits, and that’s a very, very tough discussion to have.
We are proud of our early access programs. Those are very important. And at that time, you do have a sense…more than a sense…you know the safety and the efficacy issues…
Phil Vickers – Rare disease clinical trials with Shire, Novartis and more at World Orphan Drug Congress USA 2013
Thus far, I’ve not seen the Hope that Shire says they provide patients. I’ve not seen them live up to their motto of “being as brave as those we treat.” I’ve seen only a callous disregard for the life of a little boy they know they can save, and I’ve seen them ignore the telephone calls and the emails they have received, by the hundreds and thousands, asking for an explanation of their decision.
Mr. Vickers says that he is “proud of our early access programs.” In reality, Shire doesn’t have one. We requested the criteria that patients must meet in order to participate in any early access program with the company. They didn’t have criteria. In fact, they only started working on one once Jack’s case brought to light the glaring problems that a company faces without such a policy and criteria.
In the video, Mr. Vickers expresses that you have to know the safety and efficacy issues involved before opening up any compassionate or expanded access program. In this case, we do know! The trial has been going on for over 3 years and no adverse affects have been reported. We have letters from over 1/3 of the trail participants expressing their support for Jack and describing how incredible the treatment has been for their children. It’s been a lifesaver, and Jack needs his life saved. He qualifies to be saved under the FDA’s guidelines for Compassionate Use (read here). In most other pharmaceutical companies, Jack would qualify under their program as well.
I hold out hope that Saturday’s meeting brings a change of heart by Shire Pharmaceuticals. Everything is in place to save Jack. We have a physician eager to treat him, we have a site lined up and preliminary review board approval. We have funding to ensure everything is done safely and in Jack’s best interest. We have the support of thousands and thousands of people throughout the world – indeed, over 31,000 people signed THIS PETITION demanding that Shire #SaveJack! We’ve had extensive media interest in this story, and we’ve held off taking this public through such a media campaign until we’ve learned the outcome of Saturday’s meeting.
In essence – we have everything we need to Save Jack – except Shire. And without Shire leading the way, without Shire fulfilling it’s stated creedo of “being as brave as the people we treat,” Jack will die.
I’ll update as soon as we know more. For now, here’s some thoughts directly from Phil Vickers himself. The quotes from above begin at 32 mins and last until the end of the clip.
Thanks for the continued support.
The Isaac Foundation