Parents fight for Quebec to fund spinal muscular atrophy treatment

The parents of a child with spinal muscular atrophy are fighting for the Quebec government to fund a new drug they say has improved their daughter's quality of life.

The drug, Spinraza, costs $118,000 per dose and a minimum of three doses are needed yearly. Seven are needed in the first year of treatment.

It was approved by the U.S. Food and Drug Administration last December and seven-year-old Jessika Parry, fromNotre-Dame-de-l'le-Perrot, has been taking it. Her parents say she has greatly benefited from doses of the new drug.

"She's stabilized. She's started to gain movement in muscles that she hasn't been able to use for a long time," Jessika's mother, Eva de Blois said.

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Benefits not proven, says government

Quebec's national institute of health and social services excellence (INESSS) decided against the province funding the drug, through the Rgie de l'assurance maladie du Qubec, because the benefits of it havenot been proven.

"In this case, INESSS concludes that the drug does not meet the criterion of therapeutic value, because it has not been demonstrated," spokesperson for the Ministry of Health and Social Services, Catherine W. Audetsaid.

As part of its decision, the INESSS stated that the duration of the study intoSpinraza was insufficient for evaluating the maintenance and improvement of motor functioning in people with spinal muscular atrophy.

Parry's treatment has until now been covered by Biogen, the pharmaceutical company responsible for Spinraza. She was one of the children chosen to receive it for free through an early access program.

"If we lose that now, we'll start regressing again, and it will be the worst feelingto watch Jessika lose everything that she's gained especially now that she understands what she's losing," de Blois said.

A petition has been launched to pressure the government to reverse its decision and fund the treatment.

The petition has gathered more than 6,000 signatures so far.

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