Monday, August 12, 2013
Today we met with our local MLA to ask him for his support. He got SO fired up about the subject when we told him our story and the meeting went much longer than we'd anticipated! It was so encouraging. I walked away with a few more ideas about how we can reach the public with our message and better communicate the value of funding for Soliris for each and every aHUS patient. I have so much more work to do since meeting with him but I'm excited to do it for all the patients I met at the conference, and for all the patients I know are out there struggling!
Saturday, August 3, 2013
After Soliris
Remember these pictures?
We'll call them the Before Soliris pictures.
We'll call them the Before Soliris pictures.
Well, now his treatment looks like this:
That's our couch. In our home. He sits there for about an hour and watches his TV or plays on his phone. The nurse arrives and administers Soliris through an IV and then she packs up and leaves. And he goes on about his business, whatever it may be on that day. He doesn't have the hour and a half drive into Vancouver. He doesn't have to take enough Benedryl to take down a horse, knocking him unconscious for hours and causing the foggy brain he used to have for days afterwards. He doesn't have life-threatening allergic reactions (yes, despite the Benedryl!) or come home and go to bed because he's feeling so miserable, missing whatever family event we'd planned or time spent with his kids.
And here it is. His life-saving medicine. We are so grateful to the drug company for making sure our lives are so much easier and that his access to Soliris won't be cut off. They do this for people all around the world, you know. Give them their million dollar medicine. For free. It kinda makes it hard to demonize the drug company when you think about all the millions of dollars they've donated to the average joe every single year.
On Monday Brian and I have a meeting with our local MLA. It should be interesting! We'll tell our story and try to see if we can get a commitment from him to support us in a practical way. The rest of the week we'll try to carry on as normal, but we'll also be expecting more media interest. Interviewing is my least favorite thing to do but it's so necessary to raise public awareness. I just hope that this time I feel REALLY good about the interview and that the key points come across loud and clear. I may have to practice on my kids. They'll love that.
Friday, August 2, 2013
aHUS Q & A
With the recent articles and interviews now out, I'm interested to see the public comments on each of the stories. And I realize that there are so many misconceptions floating around out there. So it's time to address some of them here!
Q: Why $750,000 per year per patient for Soliris?
A: $750,000 is a figure thrown out by the media recently. I'm not really sure where they got that number from but it sounds astronomical, doesn't it? (To be fair, a few newspapers have reported it as $450,000 a year. Not much better.) Well, the number is wrong. Basically the dosage is different for every patient. It depends on weight, age, and severity of the disease.
Q: Why is it so expensive?
A: If you consider the millions of dollars the drug company spent in research, pinpointing exactly what they want the drug to do, paying for clinical trials and marketing the drug, and paying to have the drug developed every month to give it to the consumer, the cost doesn't seem so high. Soliris' producer, Alexion, invested nearly $1 billion in research and development over 15 years before approval of Soliris, its first product, says Jennifer Devine, a spokeswoman for Alexion.1 In the case of colorectal cancer, just as an example, you have approximately 140,000 new cases a year in the US. Each of those 140,000 people will cost the medical system a certain amount of money in pharmaceuticals. It adds up. The dollar amount the government spends on treatment for them is divided up by the amount of patients. In Canada we have so FEW aHUS patients to cover the cost of the drug, so effectively the cost per person will be higher. But the total cost? No. And yes, they DO want to make some money on top. After all that time and effort, wouldn't you?
Q: How on earth do you pronounce your last name??
A: Thanks. An easy one. Phonetically it's pronounced "Chep-Ke-Ma".
Q: We need to think about the needs of the many, not the few. Shouldn't you just accept that this is life and stop expecting the Canadian government to foot the bill for your medical expenses?
A: (Yes, this question has been asked and personally I think it's a good one) Brian is 39. Yes he'd love to see his children grow up but, for the most part, he's okay with what is happening to his body. It's not fair, but it's life. We're not fighting for funding for HIS sake, though. He is one of the lucky ones. He gets Soliris on a compassionate basis from the drug company, free of charge. We have been told that they will not cut him off. But in April we met children that have aHUS and they aren't so lucky. One of these children is an adorable little blonde girl that captured our hearts. Does she deserve this? Who in their right minds could tell her "Sorry, but it's just not worth our while to keep you alive." We're doing this for her. And for the energetic 11 year old boy we met. And the beautiful preteen girl that is just starting to dream about life outside of her medical issues.
Q: Seeing as this disease is genetic, we think you should consider making sure you don't procreate and when your children become adults they should think about being tested before procreating.
A: Ha ha ha! Oh wait. You're serious. We have very serious discussions with our children about this disease, as you can imagine. One thing we've decided to do is give our children the gift of choice. When THEY choose, they will get tested. Until then we are doing everything in our power to avoid triggers that will cause the activation of the disease in them. This may even be unnecessary seeing as they only have a 50% chance of the gene mutation, and WITH the gene mutation present they have a 50% chance of developing aHUS. Which is why it's so rare. As far as people who have this disease continuing to procreate? We all have our opinions but, ultimately, it's a personal decision.
1. http://www.ottawacitizen.com/health/Rare+condition+could+leave+year+drug+orphan/8738395/story.html
Q: Why $750,000 per year per patient for Soliris?
A: $750,000 is a figure thrown out by the media recently. I'm not really sure where they got that number from but it sounds astronomical, doesn't it? (To be fair, a few newspapers have reported it as $450,000 a year. Not much better.) Well, the number is wrong. Basically the dosage is different for every patient. It depends on weight, age, and severity of the disease.
Q: Why is it so expensive?
A: If you consider the millions of dollars the drug company spent in research, pinpointing exactly what they want the drug to do, paying for clinical trials and marketing the drug, and paying to have the drug developed every month to give it to the consumer, the cost doesn't seem so high. Soliris' producer, Alexion, invested nearly $1 billion in research and development over 15 years before approval of Soliris, its first product, says Jennifer Devine, a spokeswoman for Alexion.1 In the case of colorectal cancer, just as an example, you have approximately 140,000 new cases a year in the US. Each of those 140,000 people will cost the medical system a certain amount of money in pharmaceuticals. It adds up. The dollar amount the government spends on treatment for them is divided up by the amount of patients. In Canada we have so FEW aHUS patients to cover the cost of the drug, so effectively the cost per person will be higher. But the total cost? No. And yes, they DO want to make some money on top. After all that time and effort, wouldn't you?
Q: How on earth do you pronounce your last name??
A: Thanks. An easy one. Phonetically it's pronounced "Chep-Ke-Ma".
Q: We need to think about the needs of the many, not the few. Shouldn't you just accept that this is life and stop expecting the Canadian government to foot the bill for your medical expenses?
A: (Yes, this question has been asked and personally I think it's a good one) Brian is 39. Yes he'd love to see his children grow up but, for the most part, he's okay with what is happening to his body. It's not fair, but it's life. We're not fighting for funding for HIS sake, though. He is one of the lucky ones. He gets Soliris on a compassionate basis from the drug company, free of charge. We have been told that they will not cut him off. But in April we met children that have aHUS and they aren't so lucky. One of these children is an adorable little blonde girl that captured our hearts. Does she deserve this? Who in their right minds could tell her "Sorry, but it's just not worth our while to keep you alive." We're doing this for her. And for the energetic 11 year old boy we met. And the beautiful preteen girl that is just starting to dream about life outside of her medical issues.
Q: Seeing as this disease is genetic, we think you should consider making sure you don't procreate and when your children become adults they should think about being tested before procreating.
A: Ha ha ha! Oh wait. You're serious. We have very serious discussions with our children about this disease, as you can imagine. One thing we've decided to do is give our children the gift of choice. When THEY choose, they will get tested. Until then we are doing everything in our power to avoid triggers that will cause the activation of the disease in them. This may even be unnecessary seeing as they only have a 50% chance of the gene mutation, and WITH the gene mutation present they have a 50% chance of developing aHUS. Which is why it's so rare. As far as people who have this disease continuing to procreate? We all have our opinions but, ultimately, it's a personal decision.
1. http://www.ottawacitizen.com/health/Rare+condition+could+leave+year+drug+orphan/8738395/story.html
Welcome
Welcome to our blog! This blog is about our journey through Brian's Atypical Hemolytic Uremic Syndrome diagnosis and our plea for public funding for his only medical source of hope, a drug called Soliris. A year ago he started on Soliris on a compassionate basis by the drug company, Alexion. We're so thankful to them for changing the course of our lives. What once was a dark, bleak future is now one filled with the hope and promise any young family could wish for. Thank you for your interest in our lives!
Thursday, August 1, 2013
Brian
Tjepkema holds a package of Soliris, a drug he needs to treat a rare
genetic disorder known as atypical Hemolytic Uremic Syndrome at his
Abbotsford home on Thursday. Photograph by: Ric Ernst
, PNG photo
It’s one of the most expensive drugs in the world, but without it, an Abbotsford father says he wouldn’t be able to live. Brian Tjepkema has atypical hemolytic uremic syndrome, also known as
aHUS. He was diagnosed in 2010 with the ultra-rare disorder in which the
immune system attacks the body, damaging vital organs and often
resulting in kidney failure.
“I couldn’t function,” he told The Province. “I couldn’t be the dad I should be to my kids.”
Thanks to an innovative new drug, Soliris, the father-of-two no longer has to deal with the extreme fatigue, headaches, weakness and debilitating nausea. Before Tjepkema started the treatment, he underwent four-hour weekly plasma exchange treatments— where his blood was removed from his body, the plasma separated and replaced with donor plasma— which caused severe allergic reactions. When Tjepkema’s doctor heard about Soliris, he contacted the drug maker. Soliris has put many aHUS patients in remission and improved their kidney function to the point that they can stop other treatments like dialysis and plasma exchange. But now he fears others won’t have access to the drug he gets for free from the drug maker on a compassionate basis.
While it varies for each patient, the drug costs about $450,000 per patient per year, making it one of the most expensive treatments in the world, according to the Canadian Organization for Rare Disorders.
Health Canada approved Soliris for the treatment of aHUS in March, but earlier this week, the Canadian Agency for Drugs and Technologies in Health released a common drug review recommending the provinces and territories not fund the drug.
The review stated the benefits of Soliris were not adequately established as tests did not include a randomized control group. Common drug reviews aims to compare drugs with competitors, determine whether a drug provides value for money, and give advice on whether it is cost-effective for provincial plans to cover.
But Durhane Wong-Rieger, president of the Canadian Organization for Rare Disorders, said ultra-rare diseases, by their very nature, cannot be subjected to the same review framework as other types of drugs.
The disease affects an estimated five in every one million people, according to Dr. Christoph Licht.
As a lead investigator in the international clinical trial for Soliris, Licht said he felt it was unethical to only give the drug to some patients. Control groups are often not included in drug trials for such rare and aggressive diseases, he said.
“I don’t see a way to have done it differently or to ever do it differently,” he said.
While the recommendation to not fund Soliris for aHUS was disappointing, Wong-Rieger said it was not surprising.
In 2009, the review also recommended Soliris not be funded for the treatment of paroxysmal nocturnal hemoglobinuria, or PNH, another ultra-rare and life-threatening disorder. In 2011, B.C. Pharmacare eventually agreed to fund Soliris for the treatment of that disorder despite the negative review.
That gives hope to Tjepkema for others, like his cousin, who haven’t been able to get Soliris for free from the drug maker. Tjepkema said his cousin, who has the same genetic disorder, is in kidney failure and on dialysis. His cousin was previously misdiagnosed, a common problem with the incredibly rare syndrome, and underwent a kidney transplant.Because he isn’t on Soliris, his new kidney is also failing, but he can’t go on the transplant wait list again until he gets the drug.
“I couldn’t function,” he told The Province. “I couldn’t be the dad I should be to my kids.”
Thanks to an innovative new drug, Soliris, the father-of-two no longer has to deal with the extreme fatigue, headaches, weakness and debilitating nausea. Before Tjepkema started the treatment, he underwent four-hour weekly plasma exchange treatments— where his blood was removed from his body, the plasma separated and replaced with donor plasma— which caused severe allergic reactions. When Tjepkema’s doctor heard about Soliris, he contacted the drug maker. Soliris has put many aHUS patients in remission and improved their kidney function to the point that they can stop other treatments like dialysis and plasma exchange. But now he fears others won’t have access to the drug he gets for free from the drug maker on a compassionate basis.
While it varies for each patient, the drug costs about $450,000 per patient per year, making it one of the most expensive treatments in the world, according to the Canadian Organization for Rare Disorders.
Health Canada approved Soliris for the treatment of aHUS in March, but earlier this week, the Canadian Agency for Drugs and Technologies in Health released a common drug review recommending the provinces and territories not fund the drug.
The review stated the benefits of Soliris were not adequately established as tests did not include a randomized control group. Common drug reviews aims to compare drugs with competitors, determine whether a drug provides value for money, and give advice on whether it is cost-effective for provincial plans to cover.
But Durhane Wong-Rieger, president of the Canadian Organization for Rare Disorders, said ultra-rare diseases, by their very nature, cannot be subjected to the same review framework as other types of drugs.
The disease affects an estimated five in every one million people, according to Dr. Christoph Licht.
As a lead investigator in the international clinical trial for Soliris, Licht said he felt it was unethical to only give the drug to some patients. Control groups are often not included in drug trials for such rare and aggressive diseases, he said.
“I don’t see a way to have done it differently or to ever do it differently,” he said.
While the recommendation to not fund Soliris for aHUS was disappointing, Wong-Rieger said it was not surprising.
In 2009, the review also recommended Soliris not be funded for the treatment of paroxysmal nocturnal hemoglobinuria, or PNH, another ultra-rare and life-threatening disorder. In 2011, B.C. Pharmacare eventually agreed to fund Soliris for the treatment of that disorder despite the negative review.
That gives hope to Tjepkema for others, like his cousin, who haven’t been able to get Soliris for free from the drug maker. Tjepkema said his cousin, who has the same genetic disorder, is in kidney failure and on dialysis. His cousin was previously misdiagnosed, a common problem with the incredibly rare syndrome, and underwent a kidney transplant.Because he isn’t on Soliris, his new kidney is also failing, but he can’t go on the transplant wait list again until he gets the drug.
With the relief Soliris has provided him, Tjepkema said he owes it to others in need to ensure they can get the drug, too.
“It’s so critical that we get the government to start funding Soliris,” he said. “However long it takes, we’ll keep at it.”
The B.C. Ministry of Health was unable to respond to a request for comment before going to press.
tfletcher@theprovince.com
twitter.com/thandifletcher
“It’s so critical that we get the government to start funding Soliris,” he said. “However long it takes, we’ll keep at it.”
The B.C. Ministry of Health was unable to respond to a request for comment before going to press.
tfletcher@theprovince.com
twitter.com/thandifletcher
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