The Impact of Spinal Muscular Atrophy (SMA) Type 1 on Caregivers in Taiwan: Results of a Global Survey
Speaker(s)
Patel A1, Yang K2, Toro W1, Dabbous O1, Jong YJ3
1Novartis Gene Therapies, Inc., Bannockburn, IL, USA, 2Novartis (Taiwan) Co., Ltd., Taipei, Taipei, Taiwan, 3Kaohsiung Medical University (KMU), Kaohsiung, Sanmin, Taiwan
Presentation Documents
OBJECTIVES: Data on the impact of costs associated with care for SMA type 1 patients are limited. We sought to describe sociodemographics, health care resource use (HCRU), and time costs for patients with SMA type 1 in Taiwan.
METHODS: An online survey was disseminated to voluntary non-health care provider (HCP) caregivers in Taiwan from May 2022 to December 2022. The survey collected data on HCRU from the caregivers’ perspective, as well as time spent as a caregiver and out-of-pocket costs for caring for patients with SMA type 1.
RESULTS: Sixteen caregivers (mean age, 40.6 years) managing 16 patients with SMA type 1 responded. In 11 of 16 cases (68.8%), the respondent was the patient’s primary or co-primary caregiver. Mean patient care time was 79.4 hours/week. Caregivers reported entertainment and feeding as their most time-consuming activities. Of the 16 patients, five (31.2%) had a caregiver who stopped working to provide care; four (25%) had a caregiver who reduced their working hours (median reduction, 30 hours/week); and one (6.2%) had a caregiver who changed jobs. Ten of 16 (62.5%) caregivers reported impact on income, and, within the last 6 months, eight of 11 (72.7%) caregivers took days off from work for caregiving (mean, 5.4 days). Most caregivers consulted at least one HCP during the last 6 months for their patient with SMA type 1, and five patients (45.5%) had ≥1 overnight hospitalizations, excluding SMA-related surgeries.
CONCLUSIONS: Caregivers of SMA type 1 patients in Taiwan reported a substantial impact on their time for providing care, as well as financial and employment status. Direct/indirect societal burdens established from HCRU and lost productivity associated with SMA type 1 creates the need to understand the impact of early diagnosis and access to disease-modifying therapies on the severity of these burdens and associated costs.
Code
RWD164
Topic
Health Policy & Regulatory, Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes, Pricing Policy & Schemes, Reimbursement & Access Policy, Value Frameworks & Dossier Format
Disease
Pediatrics, Rare & Orphan Diseases