Champion of Change
July 16, 2021
In conversation with SMA patient and advocate Catherine Boivin
Spinal muscular atrophy (SMA) hardly defines Catherine Boivin’s life. With a Master’s degree in theatre design from Concordia University, the 41-year-old Montreal resident has worked as a designer for the stage and film and as a creative researcher for the Cirque du Soleil Entertainment Group. At the same time, Boivin has embraced the role of SMA patient advocate and is working with her fellow advocates to ensure all Canadians with SMA have an equal shot at life-changing therapies. In this candid exchange, Boivin shares her perspectives on SMA, access, and advocacy.
Can you tell us how you came to be an SMA advocate?
After my diagnosis of SMA at 18 months, my parents were told I would probably need to be institutionalized and not survive past early childhood. Well, here I am! In my young adulthood I devoted myself to my studies and career, without intending to become a spokesperson for my disease. But when [novel SMA medication] Spinraza came along and the PMPRB reforms in Canada came to my attention, I realized that people with SMA and other rare diseases were about to face some big challenges and felt called to use my research and communication skills to make a difference.
How was your experience getting access to Spinraza?
It’s been an interesting ride. In January 2019 the drug finally got listed for adults in Quebec only, based on a managed approval process, though we’re still fighting for fair access across the province. I myself had to wait almost a year after listing to get access, due to the complexity of my case and hospital-level delays. Since I began taking the medication I’ve taken it upon myself to document all the changes in my health, whether good or bad. My neurologist submits these records—which are in effect patient-reported outcomes measures—to the government along with the required annual evaluations, hoping they’ll continue to support my treatment. But at present there’s no guarantee or clear process.
How has the drug worked for you?
It’s working. Before starting on the drug I had very low energy levels and felt I was in decline. It was getting harder and harder to put in a full workday. My right arm, which is my better arm, was getting weaker and I had chronic shoulder pain. Routine activities like brushing my teeth—and even eating—were becoming difficult. All this has improved with Spinraza. My shoulder pain has disappeared. While I can’t say I feel 20 years younger, I no longer feel super-weak.
What was your reaction to the CADTH and INESSS recommendations for Spinraza?
(Note: In 2019, CADTH gave a positive recommendation to Spinraza for a narrow subset of pediatric patients38. After an initial negative recommendation, INESSS issued a “listing with conditions” recommendation in 2018.39) I found it hard to believe this was happening in Canada, while small countries in Europe were already enabling their patients to access the drug. One of the sticking points was a lack of long-term efficacy data to support cost-effectiveness, but this requirement doesn’t make sense for rapidly progressing conditions with few or no other treatment options. I would love to see assessors work with manufacturers on a longitudinal strategy for data collection.
In many other countries, patients have been able to access Spinraza through OBAs or similar agreements. Should this approach be considered in Canada?
The short answer is yes, though I don’t think it would work for every rare disease drug. It all depends on having measurable outcomes and agreeing on which ones to use. An OBA scheme would be workable for Spinraza as it’s a drug you have to take for life and there are many ways to assess progress. There’s a caveat, though: existing measures of progress were designed for young children and don’t capture the spectrum of improvements in adults, especially non-ambulatory ones like me. For example, one of the measures involves testing a certain reflex—which disappears naturally after age 1. In non-ambulatory adults it would make more sense to measure something like improvement in writing speed and endurance.
How do you see the patient’s role in designing or participating in OBA-type agreements?
Patients know best which outcomes make the greatest difference to their quality of life, so they must be part of the process. Industry should also be consulting with patient organizations to ensure their clinical trials include meaningful outcomes.
To play devil’s advocate, might extra consideration for patient-centric measures lead to indiscriminate support for drugs with little clinical benefit?
We have to remember that no patient wants to stay on a drug that doesn’t work. Spinraza is a complex therapy that requires lumbar punctures and regular visits to the hospital. I have heard of adults who decided to stop Spinraza because it didn’t give them enough benefit or caused other problems. If a patient wants to continue, there’s a good reason for it.
“Patients should be part of the OBA creation process because they know best which outcomes mean the most to them. We should also remember that no patient wants to stay on a drug that isn’t helping.”
Is there anything that industry could be doing to support better access?
Industry is already supporting some great projects, like the newborn SMA screening initiative. It’s a good example of the power of building partnerships and relationships, and such public-private collaborations help defray government costs. There’s always room for more collaboration all around.
What keeps you going as a patient advocate?
When Spinraza first came out, my thoughts went to families with newly diagnosed children. I was encouraged to think the drug will allow them to have a very different life experience from my own. I envision a future where all people have fair and equal access to such life-changing treatments, from the day they are born. My advocacy work is about making this vision a reality.
Newborn screening gives children with SMA a chance to realize their developmental potential. Late last year, Ontario became the first province to add SMA screening to its newborn screening panel. Not content to rest on these laurels, Muscular Dystrophy Canada, an umbrella organization representing people impacted by SMA and other neuromuscular disorders, has embarked on a collaboration with Novartis Canada to deploy SMA screening across Canada. Novartis has committed up to $2 million to the project. “It’s an excellent example of what industry can do to help improve access to therapies,” says Boivin.
References
38 CADTH final recommendation for Spinraza. Feb. 27, 2019. https://www.cadth.ca/sites/default/files/cdr/complete/SR0576-Spinraza-Resubmission-Mar-1-19.pdf
39 INESSS recommendation for Spinraza. December 2018. https://bit.ly/3rhDwwx
40 Muscular Dystrophy Canada statement. Dec. 4, 2020. https://muscle.ca/2020/12/04/mdc-statement/