CDA recommends coverage for Skyclarys, but only at a lower cost
Price must fall by 97% for cost to add only $50K per year gained of good health
A committee of Canada’s Drug Agency (CDA) has recommended that insurers should cover the cost of Skyclarys (omaveloxolone) for Friedreich’s ataxia (FA), but only under certain conditions, including a substantial reduction in the drug’s cost.
“The CDA recommendation represents an important step toward changing that reality by making treatment more accessible and offering patients and families much-needed choice in managing their condition,” Stacey Lintern, CEO of Muscular Dystrophy Canada, said in a press release from Biogen, which markets Skyclarys.
Eric Tse, general manager of Biogen Canada, said the recommendation “represents much-needed progress in addressing a longstanding unmet need for Canadians living with Friedreich’s ataxia.”
“Biogen is proud to be at the forefront of innovation in rare and serious neurodegenerative diseases, and remains committed to working collaboratively with healthcare systems and decision makers across Canada to support timely and equitable access to a treatment that will set the new standard of care in the management of FA,” Tse said.
Skyclarys is an oral therapy that was the first one approved for FA. It was approved this year in Canada for FA patients ages 16 and older, and has been approved in the U.S. and Europe for the same indication.
As part of its recommendation, the CDA’s Canadian Drug Expert Committee reviewed data from the MOXIe Phase 2 clinical trial (NCT02255435). In an open-label extension phase, patients given Skyclarys over nearly three years had slower disease progression than was observed in the natural history of the disease.
“For individuals affected by this progressive genetic disease, even a modest slowing in disease progression can have a profound impact on their lives. Friedreich ataxia is a life-limiting condition that has been marked by a lack of treatment options and longstanding unmet needs in Canada,” Lintern said.
Conditions of CDA’s recommendations
The CDA recommendations suggest Skyclarys should only be reimbursed if the cost comes down considerably. The therapy’s submitted list price is $364.30 (about $264 USD) per capsule, which works out to nearly $346,933 (about $251,000 USD) per year.
At the therapy’s current list price, adding Skyclarys to standard-of-care therapy would lead to an increase in cost of more than $1.5 million (about $1.1 million USD) for each gained year of good health, the CDA estimates. In order for the cost of Skyclarys to add only $50,000 (about $36,000 USD) per year gained of good health — a cutoff for what patients are willing to pay — its price would need to come down by 97%.
In the MOXIe trial, neurological symptom severity was measured with the modified Friedreich Ataxia Rating Scale (mFARS).
Skyclarys should also be reimbursed for patients who have a confirmed genetic diagnosis of FA and have mFARS scores between 20 and 80, which was the population in the MOXIe trial. The CDA also recommends that Skyclarys should only be reimbursed if the patient is under the care of a clinician experienced in treating FA and its related conditions.
“We are encouraged by the CDA’s recognition of Skyclarys as vital therapy for the Friedreich ataxia community, representing critical progress for this life-altering disease in urgent need of treatment options. In regions like Quebec and Eastern Canada, where more people are affected by FA, the need for timely access to new treatment options is especially urgent,” said François-Olivier Théberge, general manager of Ataxia Canada.
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