Which is Worse: Not Having a Treatment, or Having One That We Can’t Get?
April 24, 2025
Jeremy Bray and Susi Vander Wyk have been fighting for treatment access for Canadian adults with SMA. Here’s the most recent chapter of their long story.
“At 29 years old, my physical mobility is almost entirely confined to my left thumb. But with that one thumb, I can operate my wheelchair, my phone, and my computer, and I can hold down a good job.” That’s the day-to-day reality for Jeremy Bray, a Manitoba man living with spinal muscular atrophy (SMA), a rare genetic disease that causes progressive degeneration of motor neurons. “But I know it could all disappear quickly,” he says. “If I lose function in my left thumb, I stand to lose my livelihood, my purpose, and my hope.”9
“If I lose function in my left thumb, I stand to lose my livelihood, my purpose and my hope.”
Jeremy Bray
Manitoba man living with SMA
Fortunately, there are Health Canada-approved treatment options that can slow functional decline and improve quality of life in SMA. The bad news: these medications are costly, and most provinces – including Manitoba – don’t reimburse them for adult patients. That’s why Jeremy and other patient advocates like Susi Vander Wyk of Cure SMA Canada have been lobbying for wider access to these therapies – and along the way, showing how real-world evidence can fill data gaps in rare diseases.
Most studies of the available SMA treatments have focused on pediatric populations, so there’s a lack of formal clinical trial evidence in adults. As a result, CDA-AMC has recommended funding these therapies only for younger patients, and all provinces but one have established age-based cut-offs for reimbursement (either 18 years or 25 years, depending on the medication), with limited case-by-case exceptions.9
Quebec and its health technology assessment agency INESSS have taken a different approach. Canadian real-world studies have shown that SMA therapies can benefit adults;46 based on this data and other studies, INESSS recommended funding SMA therapies for all patients who meet clinical criteria, including adults.9 While that’s great news for patients in Quebec, advocates say it highlights how funding bodies in other jurisdictions could do better.
“Which is worse: not having a treatment, or having one that we can’t get?” asks Susi Vander Wyk.47 "We as a country cannot possibly accept healthcare based on age and postal code – it’s the same disease and same life-saving treatment, no matter where an SMA patient lives, and their lives all have equal value.”48
“We cannot accept healthcare based on age and postal code – it’s the same disease and same life-saving treatment, and patients’ lives all have equal value.”
Susi Vander Wyk
Executive Director, Cure SMA Canada
In Manitoba, Jeremy and his family have been lobbying the provincial government to revisit the reimbursement conditions for SMA treatments in adults – with urgency for Jeremy himself. "I hope the province commits to an emergency measure that provides me with treatment as soon as possible,” he says.49 Fortunately, Provincial Health Minister Uzoma Asagwara has committed to looking at additional evidence that could speed that process along, including real-world cases.49 Along with following clinical guidelines and recognized best practices, the province has asked CDA-AMC to conduct an "urgent review" and reconsider its recommendation opposing treatment for adults of a certain age, and has asked if it’s possible to “look at cases in the real-world context and see if there are new recommendations that should be issued?’”49 At time of writing, there has been no update from CDA-AMC on this file.
Susi agrees that diseases like SMA require more flexible approaches to data evaluation and treatment funding decisions. “Rare diseases cannot be painted with the same brush as non-rare diseases; real-world evidence must be admissible as valuable data,” she says. “This information is readily available, Canada’s experienced clinicians support access, and yet patients with a progressive disease are being made to wait. It’s devastating to watch.”50 She’s calling on review and funding bodies to step up and improve their processes. “We look forward to a future when all SMA patients are able to access treatment in a timely manner, so the effects of this condition are halted, and offer hope for the future.”51
References
9. “Why not me?”: Many SMA patients left without access to disease-modifying medicines due to age, geography. Patient Voice. August 16, 2024. https://www.patientvoice.io/many-sma-patients-left-without-access-to-disease-modifying-medicines-due-to-age-geography
46. Côté I, et al. Can J Neurol Sci. 2025 Jan;52(1):119-128. https://pubmed.ncbi.nlm.nih.gov/38532567/
47. B.C. charity fights feds for access to life-saving meds. Langley Advance Times. July 5, 2018. Available at: https://www.langleyadvancetimes.com/community/b-c-charity-fights-feds-for-access-to-life-saving-meds-2511107.
48. CADTH Ruling on SPINRAZA™ (nusinersen) Extinguishes Hope for Adults Needing Treatment for Spinal Muscular Atrophy (SMA). Biogen Canada. August 30, 2022. https://www.newswire.ca/news-releases/cadth-ruling-on-spinraza-tm-nusinersen-extinguishes-hope-for-adults-needing-treatment-for-spinal-muscular-atrophy-sma--817302578.html.
49. Man with degenerative disease pleads for Manitoba to cover treatment while it asks drug agency to review. CBC News. January 21, 2025. https://www.cbc.ca/news/canada/manitoba/jeremy-bray-spinal-muscular-atrophy-treatment-meeting-health-minister-1.7437794.
50. Continued Lack of Treatment Access for Adults with SMA Is Causing Hardship. Health Insight. https://www.healthinsight.ca/advocacy/advocacy-archive/continued-lack-of-treatment-access-for-adults-with-sma-is-causing-hardship/.
51. Evrysdi® (risdiplam) Receives CADTH Reimbursement Recommendation for Some Patients with Spinal Muscular Atrophy. Roche Canada. August 30, 2021. https://www.newswire.ca/news-releases/evrysdi-r-risdiplam-receives-cadth-reimbursement-recommendation-for-some-patients-with-spinal-muscular-atrophy-873584378.html.