Below is a note I sent to Shire CEO Flemming Ornskov, requesting information we’ve been waiting on for the past week. Outlined is our rationale why Jack should have access to expanded use of IT ERT. After being very open with communication while they were deciding Jack’s fate, Shire has decided not to provide us with any requested information after they decided to deny Jack a fair chance at life.
I am posting this here because we have been inundated with requests for updates from families throughout the US and Canada. I will do my best to ensure all information is made readily available. I appreciate the time everyone has taken to contact us and the help and support you’ve offered.
We are speaking with the Shire team tomorrow evening. I’ll post any news as it becomes available. In the meantime, send Jack your words of Hope, and send Shire a message telling them you support Treatment For Jack HERE.
Saturday, December 14, 2013
Dear Mr. Ornskov,
On Monday of last week, I requested the criteria that Shire uses to determine which candidates, if any, qualify for pre-approval access to an investigational drug. I have made two other requests for the information in the subsequent days. As of yet, we have not received that criteria. It would be helpful for my organization, as well as Dr. Barbara Burton, to be able to review those criteria before we speak with your team on Monday, December 16th. I am writing, once again, to request that those criteria be made available. Successful companies that develop therapies which can potentially and dramatically enhance the quality of life for individuals traditionally have pre-set conditions with which patients qualify for EAP use and I am confident that Jack meets the criteria your company has created.
In addition, I firmly believe that Jack meets the criteria set forth by the FDA for approval for any expanded use of your therapy. As I have previously stated, I understand your concern about the number of children you currently have safety data on (15). In the rare disease world, this is a high number. With the promising results thus far, it is our opinion that this treatment would easily qualify under the FDA’s Breakthrough Therapy designation and could be fast tracked in short order. Providing access to Jack has the potential to bolster that case even further and should be considered so that all sufferers of Hunter Syndrome can be treated sooner.
The facts of this case are clear: we have a child who is in desperate need of treatment. We have an available treatment that has the potential to save his life. That child qualifies under the FDA’s criteria set forth for pre-approval access to an investigational drug. We have a physician who is anxious to treat Jack as soon as possible, and we have an independent review board that we are confident will approve such treatment.
It is clear that this decision comes down to a question of values. Specifically, will Shire live up to its stated objectives of “keeping the patient at the heart of everything we do”? Will you choose to “Be Brave”, as your responsibility creedo dictates you aim to be for your patients? Or will you abandon your responsibility to help those you can when they are most in need?
The MPS community is shocked, angered, and disheartened by the news released last week regarding treatment for Jack. Many participants currently enrolled in your clinical trial are organizing a boycott because they feel abandoned and controlled by a pharmaceutical company clearly protecting their own best interests. I’ve been contacted by numerous families throughout Canada and the United States questioning whether you believe in your study – whether you believe in the treatment you have under development. Denial of access to treatment for Jack shows many that you don’t, in fact, believe in its potential. If you did, action would be taken immediately to save the life of a child that only you can save.
Please pass along your EAP criteria as soon as possible so that we may review it prior to Monday’s meeting. If you would like to speak in person regarding that criteria or to update me on when I can expect it, please don’t hesitate to contact me anytime at 613.328.9136. If no such criteria exists within Shire, we are happy to work with you to help bring such a policy to fruition. Creating life-altering treatments comes with a responsibility to make it available to those most in need, and we would welcome an opportunity to work collaboratively to ensure those needs are met.
The Isaac Foundation
“Love, Laughter, and Hope”