Treatment Denied!

Treatment-DeniedHi 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,