B.C. family raising funds for baby daughter's $3-million treatment that could save her life
VANCOUVER -- A B.C. family is raising funds for a multi-million dollar treatment that could save their two-month-old daughter's life.
Lucy Van Doormaal was born on April 1 at BC Children's Hospital with a rare neuromuscular disorder called Spinal Muscular Atrophy 1, or SMA1.
If the disorder is left untreated, Lucy may not live longer than a year. But, when Lucy was 10 weeks old, she completed an antibody test that revealed she could be a candidate for a ground-breaking gene therapy.
A single dose of the therapy, however, costs $3 million -- estimated to be the most expensive drug in the world -- and is not covered by Canadian health care.
"It sounds pretty cruel that there could be something that could possibly save her life and we don't have access to it and can't imagine how we would be able to pay for that," Laura Van Doormaal, Lucy's mother, said.
B.C.'s health ministry says it currently covers one treatment for SMA called Spinraza. The other treatment the Van Doormaals are considering, called Zolgensma, was submitted to the Common Drug Review on June 25.
Spinraza costs about $750,000 for the first year and about $350,000 after that, said Dr. Durhane Wong-Rieger of the Canadian Organization for Rare Disorders.
That makes the eye-popping cost of Zolgensma actually cheaper than a decade of similar treatment on Spinraza, she said.
"At this point we're not saying that the B.C. government is doing anything wrong. Typically governments do not fund for drugs that haven't been approved," Wong-Rieger said.
"But we're asking them to do the right thing and be compassionate. At the end of the day it will pay off many times over for the government and also for the baby."
Zolgensma was submitted to the Common Drug Review on June 25.
"We must allow this independent expert review to take place without interference. These reviews are thorough and do take some time," B.C.'s health ministry said in an emailed statement.
"B.C. will continue to work proactively and collaboratively with all organizations, other governments and agencies within the national drug review process to ensure that British Columbians get better access to more affordable prescription drugs."
In some cases, the ministry said, requests can be approved for PharmaCare coverage in exceptional circumstances and under pre-defined criteria.
However, as Zolgensma hasn't been reviewed, criteria for the drug hasn't been established yet.
The Van Doormaals have started an online fundraising campaign to gather donations for Lucy's treatment. As of Friday afternoon, the campaign had so far raised more than $200,000.