Boston Globe – A Few Notes

photo-3Hi 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

Request for Collaborative Approach – An Open Letter To Flemming Ornskov

vickers-quote---fowlerDear Mr. Ornskov and Mr. Vickers,

I am writing to request an opportunity for you to review information we have gathered to support an Individual, emergency use, expanded access application for Jack Fowler.  I am very confident that the document we have prepared will present a very compelling case to allow access to your investigational drug SHP-609 outside of the clinical trial setting.  After consultations with the FDA and some of the most experienced and powerful people throughout the pharmaceutical world, I truly believe that allowing this application to move forward to the FDA can and will work to meet the the best interests of the patient, the Hunter community as a whole, and Shire plc.  We are firm in our belief that the information we have gathered to support this application enhances the clinical trial process, which in turn is a direct benefit to Shire.


If you are interested in working collaboratively to meet the needs of the entire patient community that you hope to represent, please indicate in writing if you will accept our document that lays out our position.  I would very much like to receive your feedback and counter arguments against supporting this application, with hopes that we can work together to come to a resolution.  Perhaps an open and honest discussion could follow as we continue to find the best way to meet the needs of this patient while supporting and enhancing the clinical trial that is currently underway.


I look forward to hearing from you with a response within the next 24 hours.


Very Sincerely,


Andrew McFadyen

The Isaac Foundation
“Love, Laughter, and Hope”


Meeting With Shire – Update

flemmingHi Everyone,

It’s with a heavy heart, and with anger and frustration, that I update you on the meeting that took place this morning between the Fowler family, The Isaac Foundation, and Shire Pharmaceuticals.  The meeting was organized after Shire’s decision to deny Jack Fowler the life saving treatment he immediately requires.

This meeting was supposed to take place at the Fowler home, 45 minutes outside of the city of Chicago.  It was set to take place at 1:30 p.m.  However, a few days before the meeting, Shire’s CEO Flemming Ornskov abruptly changed the location of the meeting to the Chicago airport, and changed the time to an unseemly 7:30 am.  The Fowler family struggled to put child care in place.  And when you are dealing with a special-needs child, that is no easy task.

We made it to the meeting on time, and brought Jack in with us to meet with the Shire team.  Present members for Shire were CEO Flemming Ornskov and Head of Research and Development, Phil Vickers.

The Isaac Foundation began the meeting by thanking Shire for taking the time to meet with the Fowler family.  We expressed that the purpose of the meeting from our point of view was to discover how we could work collaboratively with Shire Pharmaceuticals so that we can find the best treatment options for Jack Fowler and in a timely fashion.  We expressed that if Shire was present to simply reiterate their position from December and deny Jack the treatment he needs, then the meeting would need to come to an abrupt end.  I made very clear to Mr. Ornskov one very simple fact – an immoral decision is still immoral, even if it’s delivered while looking us in the eye and said to our face.

Flemming looked at us and said “We are not changing our decision.  I guess this meeting is over.”  With that, the hopes of the Fowler family were dashed, and our hope to work together with Shire to save this little boy was ended.   We left a large print out of the 32,000 signatures that were signed in the online petition, as well as letters of support from a high percentage of the families currently participating in the clinical trial of the drug that Jack desperately needs.

As we were leaving, Jack walked around the table and gave Flemming a hug.  Close to tears, Jack’s mom said “If he could talk, he would be asking you to Be Brave, like your motto says, and save him.”  It was one of the most heartbreaking moments of my life – watching a sweet little boy who doesn’t have any idea what is going on give a hug to the man who just gave him a certain death sentence.  I told Flemming that I had a hard time understanding how he can go home and look his children in the face after that moment.  He just looked at us and said “It was nice to meet the family.”

The facts of this case remain the same, and are very clear.  Jack Fowler needs access to a drug that will save his life.  He easily qualifies for individual use access through the FDA’s Expanded Use guidelines.  It is those guidelines that decide whether any investigational drug is safe for use outside the bounds of a clinical trial.  The question of whether enough safety data exists to proceed or not doesn’t rest with Shire Pharmaceuticals, nor does it rest with any pharmaceutical undergoing the same process.  It rests with the FDA first and foremost, and it rests with the physician in charge of treating the patient.  All Shire has to do is begin the application process on Jack’s behalf and leave the decision to the FDA.  When I stated this very clearly to Mr. Ornskov and asked if he would submit the application, he flatly refused.

There are things in this world that many people would be better off not knowing.  What lurks in the minds of pharmaceutical decision makers should be at the top of everyone’s list.  To have the ability to provide help and support, to be able to save the life of a precious little boy, and then choose not to, shows a callous disregard toward life.  That callous disregard is a painful reminder of what Big Pharma is after – money, product, fortune, and fame.  Don’t ever be fooled that the patient comes first.  As Flemming so plainly stated before I gave my introduction – “We don’t work with patients.  We don’t work with families.  We are in the business of developing product.”

With that, there’s nothing left to be said.

Our press release goes out early this week.  Stay tuned on how you can help us #SaveJack.  We will never quit when the life of a child hangs in the balance.

Thank you for your ongoing and tremendous support.

With Love,

The Isaac Foundation