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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