Many can cause great suffering, physical and/or mental disability, and premature death. Innovative medicines continue to be developed for more rare disorders, but not all new drugs are launched in Canada. Even when approved here, the drugs aren’t necessarily accessible by all sufferers, thanks to the red tape of our health care gatekeepers.

Take the example of spinal muscular atrophy (SMA), a motor neuron disorder. Motor neurons are cells in the brain and spinal cord that signal our muscles allowing us to move, speak, breathe and swallow. They require a particular protein to work. SMA is caused by a problem with a gene that makes this protein. When the gene can’t make enough protein for motor neurons to work properly, they can't send signals from the brain to tell muscles to move. Muscles that don’t move wither.

SMA has five forms depending on the age of onset. These categories are important for our gatekeepers: drug regulators and payers. The types are 0, I, II, III and IV. Types 0 to III develop in childhood and range between the very severe and often fatal Type 0 to the mildest form, Type III, which can develop as early as 18 months or as late as adolescence. Type I, the most common form, begins to affect infants between birth and six months and is also severe. Type IV is the adult form, which usually begins after age 35 and slowly gets worse.

SMA places a significant burden on sufferers and there’s no cure. However, three treatment innovations (Spinraza, Zolgensma and Evrysdi) have been approved by Health Canada as safe and effective.

Spinraza is approved for Types I to III, without age limit, but Canada’s Drug Agency (CDA), which advises government drug plans, recommends Spinraza only be given to children of 12 or younger. Another example of dissonance between our health regulator and CDA. Zolgensma – also approved for Types I to III – should be administered before six months of age for greatest benefit as a one-time infusion. Evrysdi has been shown to be superior to Spinraza and is approved for SMA (types undefined) in patients aged two months and older, although CDA recommends use be restricted to patients under 25 years. Since CDA is funded and governed by federal, provincial and territorial governments, their drug plans mostly follow its recommendations.

Age limits placed on use of these three drugs means adults with SMA are denied coverage. Interviews with three adult sufferers demonstrate the frustration and distress caused by this form of age discrimination.

The first interviewee has a mild form of SMA diagnosed at the age of two (before medications were available) and managed to be a farmer, but recently became confined to a wheelchair, which limits his involvement in the farm. He’s not eligible for any SMA therapy and cannot live the life he used to enjoy. Evrysdi might give him the mobility he wants. He will only know if he gets the chance for treatment.

The second SMA sufferer is a woman for whom being able to do things for herself has always been extremely important and is scared of losing her independence as her disease progresses. She’s angry knowing approved treatments for SMA are available in Canada but not accessible by all sufferers, including her. She was 27 when Evrysdi was approved, just outside CDA’s recommended age limit of 25. How much does our health care system value her independence?

The third individual was diagnosed with SMA at 15 months and, as the disorder progressed as he has aged into adulthood, has slowly lost the ability to do things that give him joy and independence. His physical mobility is now confined to his left thumb. He is afraid that even this limited movement may soon be lost, which Evrysdi could prevent. He too will only know if he gets treatment.

These three human beings are victims of Canada’s health care gatekeepers. How much will taxpayers have to pay for the care of those rendered helpless by the ravages of this neurodegenerative condition due to the choices of our gatekeepers?

Five years ago, in their 2019 federal budget, the Liberals committed to introducing a strategy for rare disorders and promised $1.5 billion over three years in their 2021 election platform to fund rare disorder drugs. Compared with the millions of dollars spent on domestic vaccine manufacturing for COVID-19 (without a single dose for any Canadian) and the billions of dollars Ottawa is providing for dental care and pharmacare, $500 million per year is literally peanuts. It’s less than 0.15% of Canada’s overall annual spending on health of over $344 billion. Even so, not a single Canadian patient has so far benefited from this funding.

The federal government announced in April 2024 that the time for signing agreements with provinces for money from the strategy would run until April 2027. British Columbia signed an agreement in July 2024 covering two drugs for disorders impacting fewer than one in 200,000 Canadians.

Government drug plans will undoubtedly adhere to CDA recommendations when funding new rare disorder medicines, which means access for adult patients is denied when CDA advises limiting use to Canadians below 25. SMA isn’t the only disorder impacted by such restrictions.

It's not surprising Canadians with rare disorders feel left out in the cold by their governments. Ottawa’s plan for national pharmacare focused on medicines for common conditions isn’t going to help them. Canadians with rare disorders of all ages need real action to help them access new medicines that can relieve their suffering and extend their lives. No one should be left behind. 

Nigel Rawson is a senior fellow with the Macdonald-Laurier Institute, as is John Adams, co-founder and CEO of Canadian PKU and Allied Disorders Inc.