Spinal muscular dystrophy: give access to treatment quickly

The context

• A boy had spinal muscular dystrophy, a condition that leads to wasting of muscles needed for walking or eating, for example. The illness can be treated with a medication called Spinraza. 
• In 2018, the government announced that Spinraza would be available free of charge for certain people. The boy fitted into one of the eligible categories.
• In the spring of 2019, the boy’s physician prescribed the medication and sent a request to the regional hospital.
• A year later, the boy had still not been treated.
• The institution responsible for the services provided by the hospital explained that procedures had to be reviewed, which takes time. Several specialties, such as anaesthesia and rehabilitation, had to be coordinated. The care providers concerned also had to be trained.
• Then COVID-19 struck, which meant further delays.

The complaint

The boy’s mother became impatient. As she saw it, if the hospital could not provide Spinraza quickly, then her son should be referred to another hospital. She complained to the Protecteur du citoyen.

What the investigation showed

• The government announcement imposed numerous changes on the institutions concerned (CISSSs and CIUSSSs). They had to review service organization, especially to be able to provide Spinraza treatments safely.
• In some cases, it took a very long time to put the new procedures in place. Meanwhile, people with the illness saw their treatments postponed more than once.

The outcome

The Protecteur du citoyen intervened with the institution responsible for the services provided by the hospital concerned. The institution pledged to finish organizing its services as soon as possible. The boy’s treatments finally began in late summer 2020.

This case was taken from our 2020-2021 Annual Report.