A Surrey family whose infant son was born with a rare neurodegenerative disorder announced this weekend they’ve raised enough money to pay for his treatment.
Baby Aryan was born with Spinal Muscular Atrophy, a gene defect that would have eventually killed him.
The only treatment that can stop it is a gene therapy called Zolgensma — currently the most expensive drug in the world.
Through an online fundraiser, Aryan’s family raised almost $2.8 million, which is enough to import the drug from the US. It’s not yet approved for use in Canada.
“We can’t wait to see what the future holds for Aryan and we can’t wait for him to get Zolgensma,” his family wrote in a GoFundMe update on Saturday.
“From the bottom of hearts, we want to thank everyone who has supported Aryan in his fight. Without all of you, nothing was possible. We’re speechless, a simple thank you is not enough.”
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The announcement comes the same weekend that a Vancouver family whose daughter has the same disorder also reached their fundraising goal. Baby Lucy is set to get her dose of Zolgensma in September.
Why are rare disease treatments so expensive?
Dr. Millan Patel, co-founder and chief medical officer of Vancouver-based Rare Disease Foundation, said that unfortunately, the current pharmaceutical research and development system is costly to navigate, which can translate to prohibitively expensive drugs for patients with rare diseases.
When the whole process of researching, developing, and running clinical tests on a new drug to bring it to market can take up to 15 years, pharmaceutical companies face pressure to recoup their costs in the first two years before their patents run out and generic versions of the drug enter the market, Patel explained.
With rare diseases, pharmaceutical companies often set the unit price very high because there’s such a small market for the product.
“As a funder, if you’re in charge of the pharma budget, are you going to treat a hundred kids with a rare disease or take the same amount of money and treat 40,000 people with mental health disorders? That’s the decision funders have to make,” he said.
A rare disease is defined as an illness or disorder that affects less than one in 2,000 people. Although on their own rare diseases are uncommon, collectively they affect many Canadians, Patel said. There are over 7,000 rare diseases, and one in 12 Canadians has one, he said.
His foundation tries to jumpstart research into cures for rare diseases, and looks at other drugs that are already approved to see if they can be repurposed for rare disease treatment.
“The ethics of putting $200 billion a year of drug development money into common diseases, many of which are lifestyle diseases, and abandoning the children who have no choice through no fault of their own — is it a good moral choice to make as a society?”