The Burden of Spinal Muscular Atrophy Type 2 (SMA2) on Caregivers in Japan, United Kingdom, and United States: Results of a Global Survey

Author(s)

Toro W1, Tanaka S2, Patel A1, Dabbous O1, Motrunich A3, Aballéa S4, Toumi M5, El-Maghrabi T4, Saito K6
1Novartis Gene Therapies, Inc., Bannockburn, IL, USA, 2Novartis Pharma K.K., Tokyo, 13, Japan, 3Creativ-Ceutical, ROTTERDAM, ZH, Netherlands, 4Creativ-Ceutical, Paris, France, 5Aix Marseille University, Marseille, France, 6Institute of Medical Genetics, Tokyo Women’s Medical University, Tokyo, Japan

OBJECTIVES: SMA2, a rare, genetic neuromuscular disease that causes loss of both voluntary motor and bulbar functions, accounts for 4050% of all SMA cases worldwide. Because data on caregiver burden associated with SMA2 are sparse, we sought to describe out-of-pocket expenditures and time costs for the families/caregivers of these patients, as well as health care resource utilization (HCRU). Here, we present initial data reflecting caregiver burden in Japan, UK, and US.

METHODS: Online surveys were disseminated through patient advocacy groups (Cure SMA, Japan Network for SMA, Muscular Dystrophy, ACT for SMA, TreatSMA, and SMA UK) and external recruitment companies from 07/2020–12/2021 across Japan, UK, and US. Eligible respondents were voluntary non-health care professional adult caregivers.

RESULTS: One hundred twenty-four caregivers (mean age, 43.6 years) managing 127 patients with SMA2 responded. In 93/127 cases (73.2%), the respondent was the patient’s mother. Mean patient care time was 69.6 hours/week. Of 127 patients, 48 (37.8%) had a caregiver who stopped working to provide care; 31 (24.4%) had a caregiver who reduced working hours (mean reduction, 14.3 hours/week); 15 (11.8%) had a caregiver who changed jobs. Within the last 6 months, 58/79 (73.4%) employed caregivers took days off from work for caregiving (mean, 21.6 days). Impact on net income was reported by 84 caregivers representing 127 patients (median monthly reduction, $1,286 USD equivalent). Within the last 6 months, 33 patients (26.0%) had ≥1 overnight hospitalizations, excluding SMA-related surgeries.

CONCLUSIONS: Caregivers of patients with SMA2 in Japan, UK, and US reported a substantial burden on time, employment status, income, and out-of-pocket costs. Lost productivity and HCRU associated with SMA2 impose greater direct and indirect societal burdens. Early SMA identification via newborn screening and early access to disease-modifying treatments may reduce these burdens and associated costs. Work is ongoing to describe caregiver burden globally.

Conference/Value in Health Info

2022-11, ISPOR Europe 2022, Vienna, Austria

Value in Health, Volume 25, Issue 12S (December 2022)

Code

PCR61

Topic

Economic Evaluation, Patient-Centered Research

Topic Subcategory

Patient-reported Outcomes & Quality of Life Outcomes

Disease

SDC: Pediatrics, SDC: Rare & Orphan Diseases

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