Healthcare Resource Utilization, Economic Burden, and Multi-Level Medical Security System for Individuals with Spinal Muscular Atrophy in Shaanxi Province, China.

Abstract

Objectives: The objective of this study is to quantify healthcare resource utilization, economic burden, and the multi-level medical security system for Spinal Muscular Atrophy (SMA) patients in Shaanxi Province, China, from a societal perspective using a survey. Methods: This observational study employed an online survey with a retrospective cross-sectional design in Shaanxi Province, China. The survey examined various aspects of SMA, including resource utilization, direct and indirect economic burdens, and co-payment mechanisms within a multi-level medical security system. Results: Following the inclusion of nusinersen in the National Reimbursement Drug List (NRDL) in 2022, the treatment rate for SMA patients increased significantly. After risdiplam was added to the NRDL in 2023, its use also saw a marked increase. Treatment costs varied by SMA type: Type 1 incurred the highest costs (RMB 300,000 or USD 41,000), followed by Type 2 (RMB 270,000 or USD 37,000), Type 3 (RMB 200,000 or USD 27,000), and Type 4 (RMB 80,000 or USD 11,000). The primary sources of costs were productivity losses due to primary caregivers (32.94%), nusinersen usage (29.29%), and risdiplam usage (17.33%). Out-of-pocket costs for SMA patients accounted for 29.29% of the total costs. In 2023, basic medical insurance covered 49% of direct costs and 32% of total costs. Patients still had to pay 25.73% of the total cost for the direct costs. Conclusions: Basic medical insurance is a critical foundation for patient security and plays a pivotal role in reimbursement. In contrast, commercial insurance has a relatively limited impact on covering the costs for SMA patients. These findings highlight the substantial healthcare burden faced by SMA patients under the current healthcare system in China.