It is with great anger and frustration that I inform you that Shire Pharmaceuticals will not provide Jack Fowler with the life-sustaining treatment he so desperately needs. The Isaac Foundation, as well as the Fowler family, spoke via teleconference this evening to pour over Jack’s specifics, our request for expanded access use of intrathecal (IT) ERT, and their decision to deny Jack access to their treatment.
Shire made it very clear from the beginning of our conversation that they would not alter their decision, nor would they take any information we provided into consideration which shows, in my opinion, a callous disregard for the life of a child that they have the opportunity to save.
They provided us with the rationale behind the decision they made last Monday. This rationale is quite weak, and we are left wondering if Shire has taken the appropriate facts into consideration surrounding Jack’s plight.
Shire’s first reason for denying Jack access to treatment lies with their current clinical trial of the therapy. Rightly so, Shire wants to ensure this trial continues without the possibility of anything damaging their results. They agreed that having Jack receive the treatment on an expanded use basis will do nothing to affect those results in a negative fashion. However, they did stress that they were worried opening up a compassionate program (or expanded use program) could lead to many families trying to access the treatment. Shire’s reasoning, though flawed, is that families would prefer their children receive treatment through a compassionate program rather than through participation in a clinical trial. And while their reasoning seems valid on the surface, it does lack merit.
In essence, children receiving treatment in a clinical trial setting run the risk of receiving a placebo instead of the actual drug. This is common practice so that a true and accurate study of the effects of therapy can be observed. Their argument is that opening up an expanded use program would diminish their available pool of patients who would like to participate in a clinical trial because children accessing the therapy on an expanded use program would be guaranteed to receive the drug - no placebo needed because the observations are collected outside the guidelines of the clinical trial. But here is where their argument doesn’t hold - children requiring access to the treatment on a compassionate basis already do not qualify for a clinical trial. Children needing treatment in order to immediately save their lives already score below the clinical benchmarks set out by the parameters of the trial. Thus, no numbers would be reduced from their available pool, and the clinical trial won’t be stalled due to lack of enrolment. Again - those that require immediate compassionate access don’t qualify to participate in the trial in the first place. Additionally, there are very few children presenting with immediate life-threatening symptoms like Jack, so their trial will not be at risk.
Currently, there is stringent criteria in place to select who qualifies to participate in Shire’s clinical trial. It would be very easy to create similar conditions on who can access the treatment on an expanded access basis. Indeed, countless pharmaceutical companies embrace the idea of expanded access programs. Valuable safety data is collected while patients are treated outside the boundaries of the clinical trial, and that information is vital to ensuring success of a medication moving forward. This is especially true for pharmaceutical companies dealing with rare cancers, and diseases where no readily available treatment options are available. Those companies embrace the data collected during such programs, and Shire should be doing the same for the benefit of all of our kids.
Shire’s second argument was that there hasn’t been enough safety data collected to warrant an expanded use program for this treatment. Again, on the surface this seems logical, but the reality is that ample safety data is available for them to create a compassionate program immediately. Phase I/II of their clinical trial has been taking place for over 3 years. This means they have 3 years worth of data on how the treatment works, how it behaves in the bodies of the children suffering from this disease, the impact it has on patients, etc. During the course of the Phase I/II trial, they have not had to report any adverse events - no safety issues that would have halted the trial. Nothing to indicate that this isn’t safe for expanded use. Indeed - Shire is set to begin the next Phase of the trial, with an emphasis on patients in Europe and a commitment to move forward in the US as soon as possible. This suggests that Shire is willing to risk moving forward with the treatment on those enrolled in the trial, but they are not willing to risk it for Jack, who is already dying. If they have enough safety information to move toward Phase II/III, there is ample safety information to allow access under an expanded use program.
Additionally, the FDA allows for expanded use of a non-approved investigational drug when “the potential benefit justifies the potential risks of the treatment use and those potential risks are not unreasonable in the context of the disease or the condition to be treated.” In short, the FDA will approve expanded use if the patient will die without treatment. Shire is worried about the risks, but Jack faces certain death without treatment. In our mind, some Hope is better than none at all.
Shire made it very clear - this is their final decision. I asked specifically about their stated goals of “Being As Brave As The People They Help”. How can they conceivably refuse to create an expanded use program and continue to promote themselves as “being as brave” as those they help? They didn’t have an answer. When asked how Jack should be cared for moving forward, they replied that those decisions needed to be made by Jack’s physician. Well, Jack’s physician has made the decision - she’s decided that Jack needs this treatment immediately. This sent the question back to Shire for an answer - will they provide Jack’s physician with the medication she needs in order to treat Jack? Will they allow her to save his life? The answer, from Shire, remains no.
Shire can’t talk about “being brave” here. Jack is the brave one. Jack’s parents, Jamie and Jason, are the brave ones. They are staring death in the face and fighting like hell. Shire isn’t being brave. They are running from their commitment to serve the most vulnerable. They are saying no to saving a life. Jamie and Jason won’t take no for an answer. They won’t run from their responsibility to Jack. And unlike Shire, they will continue to “Be Brave” and fight for what they believe in. We stand with them.
I’ll update more as soon as news becomes available. Please contact me directly at email@example.com if you are able to assist in our efforts moving forward. In the meantime, please comment below with your message to the Fowlers and to Shire.