Study on the Economic Burden and Multi-Level Medical Security System of SMA Patients in Shaanxi Province, China

OBJECTIVES: The aim is to analyze the economic burden of spinal muscular atrophy (SMA) patients in Shaanxi Province and the alleviating effects of medical security, evaluating the impact of rare disease medical security policies on patients' economic burdens.

METHODS: A questionnaire collected data on the economic burden of SMA patients in Shaanxi Province from 2021 to 2023, measuring direct and indirect medical expenses. Annual insurance data for Nusinersen from the Shaanxi Medical Security Bureau were collected and cleaned. Descriptive statistics analyzed patient costs under different levels of the medical security system after Nusinersen was included in the national basic medical insurance catalog.

RESULTS: A total of 37 valid questionnaires (88.10%) were collected. The per capita total economic burden showed an increasing trend from 2021 to 2023, at 134,200, 216,500, and 217,400 yuan, respectively. The direct medical burden per capita increased most significantly, with an average annual growth rate of 109.87%. Direct non-medical expenses showed a slow decline, with an average annual reduction rate of 34.62%, while the indirect burden remained stable at 45,000-46,000 yuan per person. The increase in direct medical expenses was mainly due to Nusinersen's inclusion in the medical insurance catalog. The first-level medical security costs rose from 15,500 yuan per person (21.06%) in 2021 to 113,200 yuan per person (69.77%) in 2023. Expenses for the second and third levels were lower, and patient out-of-pocket expenses decreased from 57,700 yuan per person (78.20%) in 2021 to 43,200 yuan per person (26.66%) in 2023.

CONCLUSIONS: While the multi-tiered medical security system has somewhat reduced the financial burden on SMA patients, families still face high medical expenses due to low incomes and insufficient labor force. It is recommended to further enhance policy interventions, improve the accessibility and affordability of rare disease drugs, and optimize the medical security system.