Hi Everyone,
Jack Fowler’s case was presented in the Boston Globe today. And while it’s nice to get some exposure, I truly feel the editor at the Globe should be held accountable for the biased and single-sided view the article portrayed.
Nowhere in the article was the FDA’s position put forward – a position we have been open and transparent about presenting since day 1. That position is simple – access to investigational medicines outside of the clinical trial setting have NO BEARING on clinical trial results. In fact, there are cases where expanded use have helped to speed up the process. Indeed, the FDA guidelines say the following:
“There are examples in which FDA has made use of adverse events information from expanded access use in the safety assessment of a drug. There are a small number of cases in which an important adverse event was first identified during expanded access use and those adverse events were in clouded in product labelling. This is not a negative from a public health perspective – the sooner important adverse events are identified the better. Even from the sponsor’s viewpoint, early discovery of a rare adverse event is, on the whole, a benefit. Although adverse events first identified during expanded use of a certain drugs have been included in the drugs’ approval product labelling, we are unaware of any cases in which adverse event information obtained from expanded access use has resulted in denial of approval for a product.”
As I have previously noted, the FDA has been in contact with The Isaac Foundation regarding expanded access, and I have assurances that any adverse events that happen outside of the clinical trial setting will not hinder the clinical trial for any reason. Why the Boston Globe editor did not see fit to ensure that information was presented is beyond me, is unfortunate, and leaves an incomplete picture of the story as a whole.
In addition, no quotes were sought from families who have children currently participating in the trial. The trial is currently in Phase II/III and has lasted over 4 years. Families are reporting incredible results in their children – their lives have been saved. The treatment works, and the reversal of devastating symptoms these children have been experience is proof positive. Moreover, the treatment is safe! Over 4 years of clinical trial has seen NO ADVERSE EVENTS REPORTED. None. Zero. A few words from families who have experience dealing with this treatment would have shed light on the trial, the effectiveness of the treatment, and the safety of administering that treatment to our kids.
Finally, with all due respect to Dr. Muenzer, I have to object to his comment that “compassionate use will destroy a trial.” That statement flies in the face of all evidence the FDA has provided us, and flies in the face of the entire purpose of compassionate use for our kids. Expanded use guidelines were clarified by the FDA a few short years ago, and they were clarified so that physicians and pharmaceutical companies alike could fully understand that compassionate use does not affect trials. Their guidelines ensure that expanded access is provided as a treatment option for patients, not a data collection option for principal investigators or pharmaceutical companies. In short – any expanded use will not impact the trial. Not by any stretch. And to further this point, I emailed Janet Woodcock at the FDA for a direct statement on the matter. She promptly replied and said “…in our collective knowledge here at CDER, adverse events occurring during the development program have not delayed the programs. In one case, we know the drug development was actually accelerated.” One would have expected this information would be available to anyone who asked, including Shire Pharmaceuticals.
It is beyond me why the Boston Globe would neglect to present these viewpoints in their story. Journalism is supposed to present an objective viewpoint on a trending topic. It’s purview is to educate the public and allow for open discussion. For that discussion to take place – for any fair and open discussion to take place – all the viewpoints need to be laid out. Sadly, important information was precluded from the story, and I hope this entry helps clarify the matter once again.
Finally – Phil Vickers – never in this world would it be “ethically wrong” to try to save a child’s life. EVER.
On another note, I emailed Shire CEO Flemming Ornskov yesterday to ask, once again, for a collaborative solution to this matter. I am hopeful that Flemming and his team will agree to sit down directly with the FDA, Dr. Muenzer, and Dr. Burton to work through this matter, set a proper policy and guidelines for access to this investigational drug, and work to do what is best for our children. ALL of our children.
With thanks for your continued support,
The Isaac Foundation
