Burden of Spinal Muscular Atrophy (SMA) on Patients and Caregivers in Canada.

BACKGROUND: Spinal muscular atrophy (SMA) is a rare neurodegenerative disease characterized by progressive muscular weakness, which occurs in one in 6,000 to 10,000 live births. The burden of SMA on Canadian patients and caregivers is not known. OBJECTIVE: To characterize the burden of SMA in Canada as reported by patients and caregivers, including disease and treatment impacts, indirect costs, and caregiver burden. METHODS: Surveys were distributed by Cure SMA Canada and Muscular Dystrophy Canada to individuals with SMA and their caregivers. The online surveys were anonymous and completed between January 28 and February 21, 2020. RESULTS: 965 patient and 962 caregiver responses met the eligibility criteria. Patients reported SMA subtypes as: type I (25.0%), type II (41.3%), type III (29.3%). Using the EQ-5D, patients were shown to have impaired quality of life with an average health utility index of 0.49 (SD: 0.26). The median expenditure was $4,500 CAD (IQR: $1,587 - $11,000) for assistive devices; $6,800 CAD (IQR: $3,900-$13,000) on health professional services; and $1,200 CAD (IQR: $600 -$3,100) on SMA-related travel and accommodation in the past 12 months. Caregivers reported needing respite care (45.7%), physiotherapy for an injury from a lift/transfer (45.7%), or other health impacts (63.3%). Caregivers reported changes to personal plans, sleep disturbances, and work adjustments, with a mean Caregiver Strain Index score of 7.5 [SD: 3.3]. CONCLUSION: SMA in Canada is associated with a significant burden for patients and their caregivers.

Evaluation of subcutaneous rituximab administration on Canadian systemic therapy suites.

Background: Non-Hodgkin lymphoma (nhl) is the most common hematologic malignancy. Diffuse large B-cell lymphoma (dlbcl) and follicular lymphoma (fl) constitute 55% of new nhl cases and are initially treated with rituximab-based chemoimmunotherapy. Relative to intravenous (IV) rituximab, a subcutaneous (sc) formulation approved in 2016 has comparable pharmacokinetics, efficacy, and safety, and a greatly reduced administration time; it is also preferred by patients. The objective of the present study was to estimate the effect (on systemic therapy suite time and on the costs of drug acquisition and administration) of implementing sc rituximab in the initial chemoimmunotherapy for fl and dlbcl over 3 years in the Canadian market. Methods: An Excel (Microsoft Corporation, Redmond, WA, U.S.A.)-based model was created with a population size based on epidemiologic data and current rituximab use, duration of use considering initial therapy, time savings for sc rituximab administration from published studies, costs from standard Canadian sources, and assumed uptake in implementing provinces of 65%, 75%, and 80% over 3 years. Key parameters and sensitivity analysis values were validated by clinical experts located in various Canadian jurisdictions. Costs are reported in 2017 Canadian dollars from the perspective of the health care system. Results: More than 3 years after implementation of sc rituximab, we estimated that 5762 Canadians would be receiving sc rituximab, resulting in savings of 128,715 hours in systemic therapy suite time and approximately $40 million in drug and administration costs. Sensitivity analyses suggest that the model is most sensitive to sc market uptake, number of induction therapy cycles, and eligible patients. Conclusions: Subcutaneous administration of rituximab can significantly reduce systemic therapy suite time and achieve substantial savings in drug and administration costs.