Shire
Request for Information – Shire plc
Hi Everyone,
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.
Andrew McFadyen
The Isaac Foundation
“Love, Laughter, and Hope”
www.theisaacfoundation.com
Treatment Denied!
Hi Everyone,
Stunningly, Shire PLC has decided against providing the life-sustaining treatment that Jack Fowler desperately needs. Jack needs immediate access to the Intrathecal ERT that is currently undergoing a clinical trial; a trial that is showing incredibly promising results. The Isaac Foundation made contact with Shire CEO Flemming Ornskov and their Head of Research and Development Phil Vickers over the course of the past week and we presented our case and our plea for them to save Jack’s life. Sadly, and shockingly, Shire has made the decision not to help.
The case that was presented to Shire was very comprehensive, and the argument for providing treatment for Jack remains incredibly strong. Jack requires Intrathecal ERT – essentially a similar treatment that MPS children receive, except this is directly administered into the brain. The clinical trial that Shire is currently undergoing is showing promise, with positive results reported from many families that are currently part of the trial. Thus far, this trial has been going on for 3 years, without any adverse reactions or safety concerns. In short – there is ample evidence to show that it is safe, and ample evidence to show that it works.
We have requested that Jack be provided compassionate access to this treatment because of the results that have been returned thus far. Results are showing that this treatment works. But what is compassionate access? As the FDA aptly describes, “Expanded access, sometimes called ‘compassionate use,’ is the use of an investigational drug outside of a clinical trial to treat a patient with a serious or immediately life-threatening disease or condition who has no comparable or satisfactory alternative treatment options.
FDA regulations allow access to investigational drugs for treatment purposes on a case-by-case basis for an individual patient, or for intermediate-size groups of patients with similar treatment needs who otherwise do not qualify to participate in a clinical trial. They also permit expanded access for large groups of patients who do not have other treatment options available, once more is known about the safety and potential effectiveness of a drug from ongoing or completed clinical trials.”1
Jack is clearly battling a life-threatening condition. He clearly needs access to the treatment that Shire has developed as his health and cognitive abilities are rapidly deteriorating. And he needs it now, or he faces certain death. The case for expanded access has never been more clear.
We made it known to Shire that we have a location where Jack can receive his treatment. Funding has been made available to ensure all baseline studies are completed and to ensure on-going evaluations and safety monitoring are put in place. We have a physician who has agreed to treat Jack, and we are confident that any Independent Review Board will approve that treatment. FDA guidelines allow for expanded access for such a therapy. Once again, we know it’s safe, and we know it works. The only roadblock to providing Jack with his fair chance at life is Shire. To many, this is inhumane and unacceptable.
In truth, the decision to deny Jack treatment came down on Monday of this week. Since that decision was made, I have been trying to get information as to the rationale behind the denial. Important questions have been left unanswered. What were the criteria used to deny this child the treatment he needs? What are the criteria used to allow or deny any child suffering from this disease access to the drug on a compassionate basis? I’ve been struggling to receive answers, and I have put those very questions in front of both CEO Flemming Ornskov and Phil Vickers. I’ve asked for the answers in writing, and have been having a very hard time receiving a prompt response. In my eyes, a company taking away all hope from a child should at least be able to provide an answer as to why, and in a timely fashion.
We have already been contacted by numerous families throughout the MPS community who are shocked, angered, and outraged by this decision. Many families have told me that they feel this is another example of a large pharmaceutical company deciding the fate of their children. Some families have also threatened to boycott the remainder of the clinical trial, a step that could have all sorts of repercussions on the study as it currently exists.
Plans are being made for a teleconference between Shire plc, the Fowler Family, and The Isaac Foundation. This call won’t take place until Monday, and we will keep you informed of news as it occurs. For now, please drop over to our message board for Jack and leave a note for Jack’s family (PLEASE VISIT HERE). As you can well imagine, they are devastated beyond words. Jamie Fowler, Jack’s mom, has updated her site to let her friends and family know the devastating news. Please visit her site here to read and comment.
Thank you for your continued support for our kids. Sadly, until we find a cure these fights for life will remain. And we will never back down when a child’s life is in danger, especially when we know there is something out there that can help.
With Love, and with thanks,
A.
1. http://www.fda.gov/ForConsumers/ByAudience/ForPatientAdvocates/AccesstoInvestigationalDrugs/ucm176098.htm
Jack’s Story
Hi Everyone,
Late last week, I received a note from Jamie Fowler, a fellow MPS parent who lives in the US. The email was entitled “Help!” and, needless to say, left me heartbroken. It was a plea for help, for support, a last ditch effort to anyone listening to help save her son, Jack. Jack suffers from MPS II and has been bravely fighting the disease as best he can. Of late, the disease has been relentlessly attacking Jack’s brain, and he has been regressing at a rapid pace.
To any parent, to any decent human being, this is heartbreaking. Please read Jamie’s email below, and then continue reading to see how you can help Jack receive the treatment he so desperately needs.
From Jack’s Mom
Another sleepless night…
Tonight I found Jason on the kitchen floor sobbing. I’m in bed ready to tackle sleep and trying to contain my emotions, then I hear the sound of my solid rock husband Releasing. He had found pictures of our spunky Jack that he hadn’t seen for awhile. Jack getting into the cabinets, cooking and mixing. Jack playing the flute and drums. Jack playing in his fire station set. Jack exploring in the sandbox. Jack dancing and singing show tunes. And the memories go on. It’s incredibly challenging to share and exposure you ladies to what I’m dealing with at my end. You both go to bed knowing your sons have a chance at life, which lightens me to know, but inevitably I end up going to bed crying from watching my son lose another skill set, a step closer to death. I share bits and pieces but the reality is I struggle every moment of every single day with fear. Fear of this final phase of regressing, losing ALL his skills. Boy do I feel alone. Yesterday Jack stared at me with spoon in his hand, no idea what to do with it. His favorite entertainment, the iPad, he is no longer able to operate it. The list goes on and on. I put on a smile for him and Juliet and “pretend” tomorrow will be better only to find tomorrow is always worse.
He stared at me last weekend while sitting in the bath, boy do I miss him being feisty -yelling and splashing me, now he sits and stares as if he wants to do or say something to me but just can’t. It’s way beyond heartbreaking.But back to Jason in the kitchen. After a long hard cry we discussed our sorrow and how we must cope while moving through this horrific phase with no easy solution for help. And then we both reminded each other our promise we made on Diagnosis Day that we would do ANYTHING and EVERYTHING to make Jack happy and comfortable. Jason feels failure and I just don’t want to ever look back on this time with what ifs.
Each day is another day Jack is gone.
We know there is drug. We know it is safe. We know Jack needs it!
With a heavy and light (yes both),
Jamie.
We have an incredible team that is committed to moving forward swiftly on behalf of Jack. We will be doing everything we can to ensure that Jack gets the treatment that he needs. What is that treatment? Currently, Shire is undertaking a clinical trial where they direct the treatment MPS II sufferers need directly into the brain. The results, so far, have been incredibly positive. Unfortunately, Jack doesn’t qualify for the trial because he has high Intracranial Pressure in his brain – Pressure that would be corrected with the treatment. A sadly ironic situation, but one that can be overcome.
Currently, we have a site where Jack can receive the treatment. We have a physician that can administer the treatment, and we’re confident that the Independent Review Board will approve administering it to Jack. The only thing we need is access to the drug on a compassionate basis. Simply put, without that access, Jack will die.
And I won’t sit back and allow that to happen.
We’ll need our team over the coming weeks and months. I hope to work with Shire to develop a plan to collaboratively work together to help Jack. I’ll drop by here and on our social media pages to update things as they progress.
Hang in there with us, Jack. We’re doing everything we can to help.
With Love,
A.
Jack's Story
Hi Everyone,
Late last week, I received a note from Jamie Fowler, a fellow MPS parent who lives in the US. The email was entitled “Help!” and, needless to say, left me heartbroken. It was a plea for help, for support, a last ditch effort to anyone listening to help save her son, Jack. Jack suffers from MPS II and has been bravely fighting the disease as best he can. Of late, the disease has been relentlessly attacking Jack’s brain, and he has been regressing at a rapid pace.
To any parent, to any decent human being, this is heartbreaking. Please read Jamie’s email below, and then continue reading to see how you can help Jack receive the treatment he so desperately needs.
From Jack’s Mom
Another sleepless night…
Tonight I found Jason on the kitchen floor sobbing. I’m in bed ready to tackle sleep and trying to contain my emotions, then I hear the sound of my solid rock husband Releasing. He had found pictures of our spunky Jack that he hadn’t seen for awhile. Jack getting into the cabinets, cooking and mixing. Jack playing the flute and drums. Jack playing in his fire station set. Jack exploring in the sandbox. Jack dancing and singing show tunes. And the memories go on. It’s incredibly challenging to share and exposure you ladies to what I’m dealing with at my end. You both go to bed knowing your sons have a chance at life, which lightens me to know, but inevitably I end up going to bed crying from watching my son lose another skill set, a step closer to death. I share bits and pieces but the reality is I struggle every moment of every single day with fear. Fear of this final phase of regressing, losing ALL his skills. Boy do I feel alone. Yesterday Jack stared at me with spoon in his hand, no idea what to do with it. His favorite entertainment, the iPad, he is no longer able to operate it. The list goes on and on. I put on a smile for him and Juliet and “pretend” tomorrow will be better only to find tomorrow is always worse.
He stared at me last weekend while sitting in the bath, boy do I miss him being feisty -yelling and splashing me, now he sits and stares as if he wants to do or say something to me but just can’t. It’s way beyond heartbreaking.But back to Jason in the kitchen. After a long hard cry we discussed our sorrow and how we must cope while moving through this horrific phase with no easy solution for help. And then we both reminded each other our promise we made on Diagnosis Day that we would do ANYTHING and EVERYTHING to make Jack happy and comfortable. Jason feels failure and I just don’t want to ever look back on this time with what ifs.
Each day is another day Jack is gone.
We know there is drug. We know it is safe. We know Jack needs it!
With a heavy and light (yes both),
Jamie.
We have an incredible team that is committed to moving forward swiftly on behalf of Jack. We will be doing everything we can to ensure that Jack gets the treatment that he needs. What is that treatment? Currently, Shire is undertaking a clinical trial where they direct the treatment MPS II sufferers need directly into the brain. The results, so far, have been incredibly positive. Unfortunately, Jack doesn’t qualify for the trial because he has high Intracranial Pressure in his brain – Pressure that would be corrected with the treatment. A sadly ironic situation, but one that can be overcome.
Currently, we have a site where Jack can receive the treatment. We have a physician that can administer the treatment, and we’re confident that the Independent Review Board will approve administering it to Jack. The only thing we need is access to the drug on a compassionate basis. Simply put, without that access, Jack will die.
And I won’t sit back and allow that to happen.
We’ll need our team over the coming weeks and months. I hope to work with Shire to develop a plan to collaboratively work together to help Jack. I’ll drop by here and on our social media pages to update things as they progress.
Hang in there with us, Jack. We’re doing everything we can to help.
With Love,
A.