Response to Provincial Governments’ Decisions Regarding Monitoring for Adults with Spinal Muscular Atrophy

Guidelines for care of SMA have indicated that a multidisciplinary approach in a clinic with expertise in motor neuron disease is standard of care.1 Recent positive trials of therapies in children with SMA have revolutionized care, with significant impacts on survival and quality of life.2–4 The efficacy of treatment is optimal when patients are treated soon after or before symptoms become apparent. There are divided opinions in the medical community as to the potential benefit of SMA therapies in adults; while some say that real-world studies provide the necessary data offering support for use, others worry that the inherent bias of an open label study and the training effect of regularly applied outcome measures is insufficient to support decision-making. Additional SMA-modifying therapies currently under review by Health Canada include the gene replacement onasemnogene abeparvovec-xioi (Zolgensma) for children under 2 years of age (all studies done without a concurrent control group and already approved in the USA and in Europe), and the splice modifier RG 7916 (Risdiplam – studies done in infants without concurrent control group and in patients 2–25 years with a placebo group). Provincial adult coverage and monitoring for nusinersen Organization/province Expanded coverage date Adult coverage Standard of care assessment schedule (months) Required monitoring schedule (months) Health Canada June 28, 2017 All SMA – – CADTH February 27, 2019 None (type 1 and 2, 12 years and under) – – INESSS (Québec) December 18, 2018 Types 1, 2, and 3 of all ages 12 12 Saskatchewan April 22, 2019 Physicians encouraged to submit application for those 18 years of age and older and/or who walked independently 12 4 Ontario June 12, 2019 Adults can be approved exceptionally on a case-by-case basis 12 4 Alberta July 5, 2019 May be considered on an exceptional basis 12–18 6 New Brunswick August 26, 2019 Requests considered on a case-by-case basis 12 n/a Yukon August 8, 2019 Case-by-case basis, based on clinical status and SMA disease course, age at symptom onset, ventilation status, motor outcome score, and patient-specific goals # Nova Scotia October 1, 2019 Individuals over the age of 18 may be considered on a case-by-case basis 12 n/a Manitoba October 22, 2019 12–18 British Columbia December 16, 2019 None (case-by-case for types 1 and 2, 12 years and under) 12–24 n/a Northwest Territories January 1, 2020 # Prince Edward Island – Nunavut – # CADTH = Canadian Association of Drug