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