Brad's Story

Brad's fight to obtain treatment in BC for adult-onset Pompe Disease

2008		May 27, 2010		Full page article in the Sun - Feb 16, 2010 Brad's interview on CHBC News and Global - Jan 27, 2010 (Unfortunately CHBC only keeps these interviews avaiable for 30 days.) Brad's now on Facebook
Update: (August 7) Brad just had his sixth enzyme replacement therapy. For the first time he was able to lay back in a recliner during the whole time without the use of breathing assistance. Previously he had to sit up. This is a big improvement and an obvious sign that his blood oxygen content is increasing. Way to go Brad. It just occured to me that this was at the beginning of the injection and so was a full two weeks since the last one. This means that his blood oxygen content is continually improving between infusions, and is not just a reaction of the "infusion of the moment". Update: (July 8) Brad has now had four enzyme replacement therapies at the Penticton Regional Hospital. Before beginning these therapies his baseline blood oxygen content was about 94%, dangerously low. Normal is 98% to 100%. On June 24th, two weeks after his second therapy, his blood oxygen content had increased to 95+%. On July 8th, two weeks after his third therapy his doctor informed him that his blood oxygen content had increased to 98+%. There can be no doubt that these therapies are helping Brad. Why aren't all Pompe disease patients in Canada receiving these therapies? Update: (June 15) As you can see, we have moved Brad's story onto it's own page. We will follow his story closely and post any new developments as his treatment progresses. We will also report on news coming from the Pompe community's ongoing efforts to have this treatment funded by all provinces and territories in Canada as well as research into both Infant and Adult-onset, or Late-onset, Pompe Disease. Update: (May 20) Mission accomplished! Today Brad received a call from the Coordinator at the Penticton Regional Hospital to tell him that his first infusion date will be Thursday, May 27, 2010. This has been a tough time not only for Brad and our family and friends, but also for the BC Ministry of Health and Pharamacare. This is a very expensive therapy and we want to thank Pharmacare and the BC Ministry of Health for their compassion throughout and for doing the right thing. We also want to thank Genzyme Canada for their compassion, paticipation and support. Finally, we want to thank everyone who flooded the BC Ministry of Health with all those letters and emails on Brad's behalf and the many doctors and professionals from across Canada and the US for showing their support. This has been a team effort by all. Now, as soon as Brad is settled into his therapy, which will be every two weeks for the rest of his life or until a cure is found, our efforts will be directed toward getting this therapy for Pompe disease supported in every province and territory across Canada. Then it's on to the other degenerative muscle diseases included in this group. If a therapy is available then it should be provided regardless the cost. Update: (April 14) As promised, the Assistant Deputy Minister called Brad today. He said that they needed a few more days. He will call Brad again on April 19th. I've updated the countdown below. It should be noted that Brad is losing an average of 2% of his lung capacity each month this process takes. Update: (March 30) As you can see from the countdown below, the British Columbia Ministry of Health has asked for more time to decide on funding Brad's Myozyme therapy. It should be noted that during the time since Brad's doctor first requested funding, Brad's lung capacity has dropped from 60% of normal to 50% of normal. Why, after Health Canada approved Myozyme therapy on August 14th 2006, and both Alberta and Ontario have completed their assessment and are funding it, does BC have to do their own assessment again, at considerable cost to the taxpayers of BC and great personal harm to my son? Pompe disease is progressive and devastating. It is a fatal disease. Why is this happening in BC?

Press Releases
(Most recent at the top)

Subject: PRESS RELEASE: Penticton paddling community teams up to raise funds for Muscular Dystrophy When local steers-person and much loved coach Brad Crittenden stepped down from steering before last year's dragon boat season it had nothing to do with a schedule packed full of coaching for one team and steering for another. "I made the decision to stop steering because I felt that my disease had progressed to the point where I didn't have the strength to safely steer a dragon boat anymore", said Crittenden. Brad has a rare disease, called Pompe Disease, which falls under the Muscular Dystrophy Canada "umbrella" of diseases that affect the muscles and nervous system. Steering a dragon boat takes balance, skill and strength to control the 15 m long, 400 kg boat, paddled by 20 paddlers in a sprint against up to 8 other crews at a time. Brad's condition worsened over the past year, to the point where he even needs assistance to breathe when sleeping. A Facebook page was set up, along with a letter-writing campaign to help Brad get the drugs he needed to slow the progress of the disease. There is no cure. The local dragon boating community wanted to do something to help raise awareness, and funds. Crittenden never wanted to be the focus of the fundraising, "It's not just about me. There are a lot of people in similar situations. Some of the donations that we raise will be directly accessible locally but all of it will support people that aren't as fortunate as us". The Okanagan Super Sprints have been held for the past two years in early July. This year's event, on July 3, 2010, has been turned into a fundraiser for Muscular Dystrophy, with existing teams challenged to raise funds in their own communities. In addition, local groups are being asked to form teams to race as part of the fundraiser. The Penticton Paddling Center has donated use of the boats for training the newly formed teams, plus local coaches have donated their time to ensure the teams are trained and ready to race. "This is a great team builder for any business or group", said Don Mulhall, local paddling organizer. "Not only is it an exciting sport that anyone can do, it's fun, and we're raising funds and awareness for Muscular Dystrophy Canada. For more information about how you can get involved, or to register, call the Penticton Paddling Center at 1-866 658-6333, or go to www.pentictondragonboat.com. To learn more about Pompe disease, visit www.pompe.com or www.muscle.ca

Correspondence with the BC Ministry of Health
(Most recent at the top)

Second Reply: Ministry of Health Services Response - 824402: March 8, 2010

Mr. John Crittenden

Dear Mr. Crittenden:

Thank you for your email of February 11, 2010, in response to my previous email regarding PharmaCare coverage of alglucosidase alfa (Myozyme®) for your son Brad.

As noted in my email from January 26, 2010, the Ministry of Health Services anticipates making the alglucosidase alfa coverage decision for Brad in March 2010 taking into consideration the recommendation from the Advisory Committee (the Committee).

In response to your comment regarding the Common Drug Review recommendation for alglucosidase alfa, this is one of the pieces of clinical information considered by the Committee. Other information considered by the Committee includes the natural disease history, clinical efficacy, clinical safety, ethics, economics and the application of this information to ensure that recommendations developed are both clinically appropriate and safe.

Once the Ministry makes a coverage decision for Brad, this will be communicated to your son through his physician.

Sincerely,
Bob Nakagawa, B.Sc. (Pharm.), ACPR, FCSHP
Assistant Deputy Minister
Pharmaceutical Services

Fourth letter: March 7, 2010:

Hon. Kevin Falcon
BC Minister of Health Services
British Columbia Liberal Party

CC: Bob Nakagawa, B.Sc. (Pharm.), ACPR, FCSHP
Assistant Deputy Minister
Pharmaceutical Services

Dear Mr Falcon,

It has been brought to my attention that treatment for "other" lysosomal storage diseases, for instance Gaucher's, Fabry's, MPS I, MPS II, are funded in BC. Why should adult-onset Pompe be different? I realize that the drug is currently under review by the Committee and a recommendation is anticipated in March, however, this has been pending since October 2009.

While you know that Pompe is a progressive disease and Brad's condition is worsening daily, and while you know that he has already lost the use of one diaphragm, you make this man wait month after month for a reply. Why is that?

I am once again requesting that you please make the drug immediately available, pending the recommendation from the Committee.

Respectfully,
John Crittenden

Third letter: To Ministry of Health Services Response - 821213: February 11, 2010

Bob Nakagawa, B.Sc. (Pharm.), ACPR, FCSHP
Assistant Deputy Minister
Pharmaceutical Services

Dear Bob:

Thank you for your reply to my e-mail of January 26, 2010. Thank you also for explaining the process in BC.

One month ago the Ministry of Health told me that a decision would take months. The spokesperson stressed the "months". That was the point where I decided I needed to get involved on my son's behalf. Brad simply does not have "months". I won't go over the details again but time is now critical for Brad.

The CDR recommendation that you refer to was released in June of 2007. It recommended listing of Myozyme for patients with infantile-onset Pompe disease who meet certain criteria. It reserved recommendation for patients with adult-onset Pompe disease citing insufficient evidence for evaluation at that time. That was three and a half [two and a half] years ago. Since then, more information has become available and it was on this basis that Ontario decided to include late-onset Pompe disease as eligible for funding as long as certain clinical criteria were achieved. Brad's doctor, Dr Sirrs, assures us that Brad would meet those criteria if he were in Ontario.

I understand a process of this kind takes time. However, I simply cannot fathom BC refusing treatment for my son while that same treatment is available in Alberta and Ontario. Can you? That being the case, there should be no problem for the Minister to approve funding on a compassionate basis while the application goes through the process at Pharmacare. Given my son's circumstances, it is simply the right thing to do.

Sincerely,
John Crittenden

First Reply: Ministry of Health Services Response - 821213: February 10, 2010

Mr. John Crittenden

Thank you for your e-mail of January 26, 2010, regarding coverage of alglucosidase alfa (Myozyme) for your son Brad, who has been diagnosed with Pompe disease. Honourable Kevin Falcon, Minister of Health Services has asked me to respond on his behalf.

I would first like to recognize the considerable concern for your son's condition. I appreciate the challenges this must create for your son and your family.

I appreciate your comments about the coverage provided by Alberta and Ontario for this drug. I understand that these decisions were made for individual patients, following the process for dealing with these rare diseases in those provinces. In British Columbia (BC), requests for coverage for alglucosidase alfa (Myozyme) and other similar enzyme replacement drugs are considered through a review process specific for coverage for drugs for rare diseases.

The BC process includes an arms-length independent Advisory Committee (Committee). The Committee membership includes specialized physicians who treat rare diseases in children and adults, experts in medical genetics and biochemical disease, pharmacists, health administrators, a health economist, and an ethicist. The Committee is responsible for evaluating patient-specific funding requests and providing recommendations to the Ministry of Health Services (the Ministry) for consideration in coming to a decision.

Your son's case was introduced to the Committee in October 2009. The Committee initiated the process of gathering the necessary information in order to develop their recommendation. This process does take time to complete as information is gathered and thoroughly assessed around various considerations, including an evaluation of the available evidence, the natural disease history, clinical efficacy, clinical safety, ethics, economics and the application of this information to your son's case to ensure that the recommendation that is developed is both clinically appropriate and safe.

One of the pieces of clinical information that the Committee will consider is the report from the Common Drug Review (CDR). In 2007, the CDR recommended coverage be provided for cases of infant-onset Pompe where diagnosis is made up to 12 months of age, as there is clinical evidence that the drug may improve survival in this patient group. The CDR did not recommend funding for other forms of Pompe as there is little evidence of the effectiveness and safety of this drug in treating them. More information on the CDR review can be obtained at: http://www.cadth.ca/media/cdr/complete/cdr_complete_Myozyme_June-14-2007_e.pdf.

The Ministry will consider the Committee recommendation in making its decision. This is anticipated to be in March of this year.

I appreciate the opportunity to respond to your concerns.

Sincerely,
Bob Nakagawa, B.Sc. (Pharm.), ACPR, FCSHP
Assistant Deputy Minister
Pharmaceutical Services

Second letter: February 10, 2010: TO THE HONOURABLE KEVIN FALCON, BC MINSTER OF HEALTH:

Dear Kevin,

This is my second letter to you and the reason is that I want to clearly identify the life and death situation my son faces so that I know that you know the seriousness of this.

• Brad has adult-onset Pompe disease which will take his life if not treated immediately.
• Myozyme therapy for Pompe has been approved by Health Canada and adult-onset Pompe therapy is funded in Alberta and Ontario. It is now saving the lives of adult-onset Pompe patients in both provinces but has not been made available to Brad, who lives in British Columbia, because BC has not funded it. We have been told that Brad is the only adult-onset Pompe patient in BC that would benefit from this therapy.

Brad's condition is deteriorating and time is of the essence. He has already lost one diaphragm. If he loses the other diaphragm he will face the rest of a short life bedridden, on a respirator, with a feeding tube, and die a disgusting and cruel death.

Brad's doctor has applied for funding for a two year trial for Brad which would provide the treatment he needs. That application is tied up in Pharmacare and I was told by a person at the Ministry of Health that it will be months before a decision is made. By then it will be too late to save Brad.

You have the power to do something about this. What would you do if Brad were your son?

Please don't let this happen. Approve treatment on a compassionate basis. There is not a person in this province that would fault you. Quite the contrary. It is the right thing to do.

Sincerely,
John Crittenden

Attachment: Brad's request for Myozyme report (PDF)
Medical report: Pompe LOTS Early Absract Laforet (PDF)
Medical report: Survey2-AusPatientsOnMyozyme-July2009-FINAL (2) (PDF)
Medical report: Pompe respiratory Munich Khan.pdf (PDF)

First letter: January 26, 2010: TO THE HONOURABLE KEVIN FALCON, BC MINSTER OF HEALTH:

Dear Kevin,

My son Brad has Pompe disease. Brad's doctor, Dr Sandra Sirrs, Medical Director, Adult Metabolic Diseases Clinic, VGH, has applied for funding for a two-year trial that could save Brad's life. It is now tied up in Pharmacare and the Ministry of Health tell me it will be many months before we get an answer.

If Brad lived in Alberta or Ontario he would be receiving Myozyme, the only treatment available for Pompe disease. Because Brad lives in British Columbia he's not on Myozyme. He has lost one diaphragm already, and he's one step away from a wheelchair and a respirator. Why is this being allowed to happen? At a time when Alberta and Ontario have already done their assessment and have approved funding for Myozyme, BC is doing it a third time, and Brad's health worsens in the meantime. This make no sense.

I understand that this may not be your fault, in fact you may not even know about Brad or the application for funding yet. It's the "System". But you are in a position to deal with it.

One last thing. Is it not a waste of taxpayer's money to be repeating an assessment that has already been paid for and approved in Alberta and Ontario? You are the only one who has the authority to do anything about this. We are asking you to facilitate this immediately and help my son.

Sincerely,
John Crittenden

Attachment: Brad's request for Myozyme report (PDF)

If anyone reading this would like to send an email of support to the Minister on behalf of Brad this is his email address. If you happen to be an adult-onset Pompe sufferer who has benefited from Myozyme that would be critical first-hand information to tell the Minister as well.

Email: Honourable Kevin Falcon, BC Minister of Health

Brad is now on Facebook. His page was put up a few days ago by Trevor Pare and is being maintained by him and his mom, Linda. Thank you Trevor and Linda. After a few days Brad's Group has grown to 274 members and counting. The more the better. Let's join and ask our friends and family to join. Ask them to ask their friends and family to join. As soon as we have therapy for Brad it's on to the rest of Canada. Already approved by Health Canada, this therapy needs to be funded by every province in Canada and supported by the federal government.

A FURTHER OBSERVATION: Myozyme enzyme replacement therapy is a treatment that is today helping several Adult-onset Pompe patients in Alberta and Ontario while research for a cure continues. There are incredibly dedicated and talented researchers in Canada and around the world who are working hard at finding that cure. Compared to long term care if Myozyme is denied, the cost of enzyme replacement therapy is a small price to pay until a cure is avaliable. And it's the right thing to do.