Four-year-old Natalie Essex loves to walk but it’s a challenge.
Thanks to a drug called Spinraza, she is regaining the use of the muscles she lost as a result of spinal muscular atrophy (SMA).
Spinraza might be a miracle drug, but it comes with a hefty price tag, costing about $100,000 per dose.
Natalie and all SMA patients need one dose every four months.
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Natalie is on a clinical trial so her family’s costs are covered, but the drug isn’t funded in B.C. If it does get approval, not all SMA patients will be given access to it.
Medications to treat rare diseases have made headlines in recent weeks, mostly due to their sky-high price tag. It’s not uncommon for new drugs to cost tens, if not hundreds of thousands of dollars per dose.
UBC student Shantee Anaquod, who is suffering from a rare auto-immune disease, recently received approval for an expensive drug treatment.
The 23-year-old became the first patient to get Soliris as a publicly-funded treatment in this province.
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Researchers who study the pharmaceutical industry say drug company pricing has no basis in reality.
“They will allude to the high cost of drug development but that actually doesn’t necessarily have anything to do with the actual prices they’re paying, in part because they never fully disclose what they did spend on research and development,” said Steve Morgan, a health economist and professor with UBC’s School of Population and Public Health.
“At the end of the day, the cost of research and development is not necessarily the determinant of the price of the drug.”
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The B.C. government says it has been asking pharmaceutical companies to justify the prices they charge but to little effect.
“They’ve now found kind of a profit pipeline for themselves,” Health Minister Adrian Dix said. “But what we need and what they have to understand, because it’s in their interest too, is we need a sustainable way going forward so that people aren’t caught in this position at the end.”
For the Essex family, none of that matters. All they want universal access to is a drug that has more than proven its worth.