CADTH Ruling on SPINRAZA™ (nusinersen) Extinguishes Hope for Adults Needing Treatment for Spinal Muscular Atrophy (SMA) Français
- CADTH recommends against reimbursement and access to potentially life-altering treatment for adult SMA patients, discounting the large body of real-world evidence of SPINRAZA's safety and effectiveness across age groups
- Provinces to ultimately decide on CADTH's reimbursement recommendation and the fate of adult patients living with this progressive, neurodegenerative rare disease
- Quebec leads by example as the only province that commits to funding SMA treatment for all patients, regardless of their age, consistent with the practice of more than 25 countries across the globe
TORONTO, ON, Aug. 30, 2022 /CNW/ - Biogen Canada is deeply saddened by the Canadian Agency for Drugs and Technologies in Health's (CADTH) final recommendation against public reimbursement of SPINRAZATM (nusinersen) for adults with spinal muscular atrophy (SMA), and by what the decision means for this rare disease community. Most adults living with SMA remain without access to treatment and face continuous functional decline, worsening disability, and an increasing loss of independence.
In its recommendation, CADTH acknowledges the unmet medical need for adult patients as well as the real-world evidence (RWE) generated from observational studies suggesting that treatment with SPINRAZA may improve or maintain physical abilities. However, CADTH discounted the RWE as it deemed it inconclusive without randomized clinical trial data for adult patients.
"We are absolutely devastated for the SMA community and the adult patients who have been relentlessly advocating for years for their right to access potentially life-changing treatment," says Eric Tse, General Manager of Biogen Canada. "While CADTH's negative position reinforces access inequities across the country and keeps an urgently needed medication out of reach for the vast majority of adult patients, individual provinces are not bound by this recommendation and will ultimately decide whether to expand funding for SMA treatment."
SPINRAZA was the first treatment approved in Canada for SMA, a severe neurodegenerative disease that causes progressive loss of muscle, mobility, and independence. Since its approval in 2017, SPINRAZA has been widely used and reimbursed by most provinces for patients under 18, and only on a case-by-case basis for adults, with many waiting years for a decision on their applications. Quebec is currently the only province that commits to funding treatment for Canadian SMA patients regardless of their age or SMA type, consistent with the practice and policies of more than 25 countries across the globe.
CADTH's final recommendation comes pursuant to a draft position published in May 2022, which advised against SPINRAZA reimbursement for adult patients, despite the large body of RWE1 from global and local disease registries that consistently demonstrates the treatment's safety and effectiveness across age groups when used in practice. In response to the draft recommendation, CADTH received feedback from multiple medical experts and patient groups across Canada, who opposed the agency's recommendation against expanding SPINRAZA access.
"The SMA community is in stunned disbelief and feels devastated that government agencies are not able to see the value in saving Canadian patients' lives," says Susi Vander Wyk, Executive Director of Cure SMA Canada. "We as a country cannot possibly accept healthcare based on age and postal code, where in one jurisdiction you are able to access treatment, while in another you can't – it is the same disease and same life-saving treatment, no matter where an SMA patient lives, and their lives all have equal value."
"We are disappointed with CADTH's decision not to revise their recommendations; it is extremely challenging for patients not to have access to treatments in Canada when real world evidence shows a treatment can be beneficial," says Stacey Lintern, Chief Executive Officer of Muscular Dystrophy Canada. "MDC will continue to advocate and work with government and other like-minded organizations to influence policy and processes so that the neuromuscular community has access to treatments and can make informed decisions concerning their treatment options."
"As a clinician with over three years of experience with this treatment and others like it, this decision is incomprehensible," says Dr. Xavier Rodrigue, doctor of physical medicine and rehabilitation at Hôpital de l'Enfant-Jésus, Quebec City. "I can only express my disappointment and sadness."
"Disappointingly, SMA doctors like myself will now need to provide support, guidance and explanation to our adult patients with SMA, on the rationale of denying persons based on age, for the drug treatment nusinersen," says Dr. Colleen O'Connell, doctor of physical medicine and rehabilitation and Medical Director & Research Chief, Stan Cassidy Centre for Rehabilitation, Fredericton, NB. "There has been so much hope among our patients and families, given this treatment benefiting those already receiving the medication; these will no doubt be distressing and potentially devastating conversations."
Although CADTH's recommendation is final, decision-making authority ultimately rests with individual provinces which will determine whether to extend or deny access to life-altering treatment to adult SMA patients. Biogen Canada remains unwavering in its commitment to the SMA community and will continue to support sustainable solutions that ensure equitable access for all SMA patients across Canada.
SMA is a rare, debilitating neurodegenerative condition that is characterized by loss of motor neurons in the spinal cord and lower brain stem, resulting in severe and progressive muscular atrophy and weakness.2
Due to a deletion of, or mutation in, the SMN1 gene, people with SMA do not produce enough survival motor neuron (SMN) protein, which is critical for the maintenance of motor neurons. The severity of SMA correlates with the amount of SMN protein an individual has. Ultimately, individuals with SMA can become paralyzed and have difficulty performing the basic functions of life, like breathing and swallowing.
People with Type I (infantile-onset) SMA, the form that requires the most intensive and supportive care, produce very little SMN protein and do not achieve the ability to sit without support or typically live beyond two years without respiratory support. People with Type II and Type III SMA produce greater amounts of SMN protein and have less severe, but still life-altering forms of SMA.
SPINRAZATM (nusinersen) is approved to treat infants, children, and adults with all types of spinal muscular atrophy (SMA)3. As a foundation of care in SMA, more than 13,000 individuals have been treated with SPINRAZATM worldwide.4 It is supported by the largest clinical data set in SMA with a clinical development program that encompasses 10 clinical studies, which have included more than 300 individuals across a broad spectrum of patient populations.3 SPINRAZATM has demonstrated sustained efficacy across ages and SMA types with a well-established safety profile based on data in patients treated up to 7 years, combined with the largest dataset of real-world experience.
As pioneers in neuroscience, Biogen discovers, develops, and delivers worldwide innovative therapies for people living with serious neurological diseases as well as related therapeutic adjacencies. One of the world's first global biotechnology companies, Biogen was founded in 1978 by Charles Weissmann, Heinz Schaller, Sir Kenneth Murray, and Nobel Prize winners Walter Gilbert and Phillip Sharp. Today, Biogen has the leading portfolio of medicines to treat multiple sclerosis, has introduced the first approved treatment for spinal muscular atrophy, and developed the first and only approved treatment to address a defining pathology of Alzheimer's disease. Biogen is also commercializing biosimilars and focusing on advancing one of the industry's most diversified pipelines in neuroscience that will transform the standard of care for patients in several areas of high unmet need.
Since 1998, Biogen Canada has been delivering life-changing medicines and services that support unmet treatment needs and the lives of Canadians affected by neurological conditions. As a company that founds its culture on principles of equity, diversity and inclusion, Biogen Canada is proud to have been named a Best Workplace™ in Healthcare, for Inclusion, Giving Back and Hybrid Work. To learn more, please visit www.biogen.ca.
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1 |
Coratti et al. Motor function in type 2 and 3 Nusinersen: a critical SMA patients treated with review. Orphanet Journal of Rare Diseases. 2021. 16:430. |
2 |
Kolb et al., 2015 Nov;33(4):831-46. doi: 10.1016/j.ncl.2015.07.004. J Kolb et al. |
3 |
SPINRAZA product monogrtaph. Accessible at www.biogen.ca/content/dam/corporate/en_CA/pdfs/products/SPINRAZA/SPINRAZAProduct_Monograph_EN.pdf |
4 |
Biogen data on file. |
SOURCE Biogen Canada
Media Contact: Biogen, Marija Mandic, + 1 416 712 8399, [email protected]
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