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Ford government to fund Spinraza, world’s most expensive drug - with age caps

Last Updated Jun 12, 2019 at 6:19 pm EDT

The Ford government today announced it will be covering the cost of the drug Spinraza for most – but not all – patients with Spinal Muscular Atrophy.

SMA is a rare disease that slowly destroys all the muscles in the body. It is the leading genetic cause of death in infants. OHIP will now cover Spinraza for patients with types 1, 2 and 3 – which are the most severe types of SMA – up until the age of 18.

In a statement Health Minister Christine Elliott writes, “Coverage for Spinraza is being expanded from the existing type 1 spinal muscular atrophy (SMA) patients to also include type 2 and 3 children and adolescents. Additionally, other patients with type 2 and 3 SMA may be considered on a case-by-case basis.”

That means people like Tori Lacey, who was profiled on CityNews earlier this year, will not automatically be covered because she is 21. However she, and others in her situation, will be able to apply for coverage. Senior government sources could not immediately say what qualifications would need to be met for those over 18 to get coverage.

Spinraza is covered in more than 40 countries around the world with no caps on ages or types. Quebec has also promised to also cover it for all. Senior government sources could not say why Ontario is implementing age restrictions or how many SMA patients are over the age of 18 – or if a younger patient receiving the drug will still be covered after they turn 18.

Spinraza is called a miracle drug and is the only hope on the market for people with SMA, but it was initially released with a massive price tag – US$750,000 in the first year and US$375,000 every year after that for life. But the impact on lives is immense, young children who could not sit up or walk have been documented taking their first steps.

Sophia Gall, another child CityNews has profiled, was able to ride a bike for the first time in her life a week after receiving a shot of Spinraza. Her parents were told their daughter, who was 3 at the time, would be in a wheelchair within a year if she didn’t get the drug. They are among the fortunate who have had coverage for the drug under work benefits. They say Sophia is getting stronger every day and can now swim and ride a horse.

Health Canada approved Spinraza two years ago but then a lengthy process kicked in to determine coverage. The Canadian Agency for Drugs and Technologies in Health (CADTH), an independent body that determines if provinces should cover the costs of new drugs, recommended only patients with the most severe form of SMA, type 1, should be able to have it covered, providing they are under the age of 12 and were diagnosed by the time they were seven months old.

The Ford government says Ontario has far exceeded those recommendations and the Executive Officer of Ontario Public Drug Programs has been in negotiations with Biogen to bring the cost of Spinraza down for several months. The company told CityNews in February it has lowered the cost of the drug “significantly.”

Biogen estimates there are 500 people in Canada living with SMA while senior government sources estimate 170 live in Ontario.

Physicians who treat SMA patients are being notified of the government’s decision today. According to the senior government sources, patients with SMA – even those who are over the age of 18 and need to apply for it – should be able to get access to the drug “in short order.”

Why is Spinraza not covered for those over 18?

  • Ontario understands that people of different ages are affected by spinal muscular atrophy (SMA) and the Ministry believes expansion in coverage is a positive step in the right direction. Although broad coverage for all ages is not available at this time, the Ministry is committed to working with the manufacturer to continue the dialogue on further expanding Spinraza coverage for SMA patients of all ages.
  • Symptomatic Type 2 and 3 patients under the age of 18 regardless of ever achieving the ability to walk independently will be considered on a case by case basis.
  • Other patients who do not meet the expanded funding criteria may be considered in exceptional cases.
  • SMA patients are encouraged to work with their clinician to submit an application for funding through the Exceptional Access Program (EAP).

How many SMA patients are there over the age of 18 in Ontario?

  • It is difficult to know exactly how many patients there are but estimates suggest that there are approximately 170 people in Ontario who have spinal muscular atrophy (SMA). It is not known this time how many might be eligible for treatment or seek coverage.

What are the criteria that a patient over 18 must meet to qualify for coverage?

  • Patients such as those 18 and over who do not meet the expanded funding criteria may be considered in exceptional cases.
  • Each individual application would be reviewed to consider individual details of a patient and their disease state, and include consideration for the unique individual clinical and circumstantial information provided regarding the specific patient.

Is there an age cap for type 1 coverage?

  • There is no specified age cap for Type 1 patients. In general, type 1 SMA is a severe form of the disease and most individuals present early in life and life expectancy is typically short.

What happens to a patient who is receiving Spinraza when they turn 18? Will they have to apply for it or will they continue to receive it?

  • Patients will be required to undergo an assessment when they apply for coverage. Ongoing assessment will be required in order to monitor for improvement over time, and patients must meet renewal criteria for continuation of treatment.
  • Patients are encouraged to work with their physicians.

What is the estimated cost to cover Spinraza for all patients in Ontario?

  • Cost will depend on how many patients are eligible and seek treatment, and the number of doses each individual patient requires.
  • Confidential pricing terms have been negotiated with the manufacturer to provide improved value.

How much was the province able to reduce the cost of the drug?

  • Pricing terms with negotiations between the province and the manufacturer are confidential.