Economic Burden of Pompe Disease in China: Empirical Evidences and Simulation of Policy Effects
Author(s)
Dong D1, Chen S2, WU Y3, Zhu J4, Jin J5, Wang Y6, Guo P7
1The Chinese University of Hong Kong, Shatin, China, 2University of Cambridge, Cambridge, CAM, Great Britain, 3The Chinese University of Hong Kong, Shatin, Hong Kong, 4Fudan University, Shanghai, China, 5Tsinghua University, Beijing, China, 6The Illness Challenge Foundation, Beijing, China, 7China Pompe Care Center, Beijing, China
OBJECTIVES To explore the economic burden for people affected by Pompe Disease (PD) in China, and to simulate impacts of local reimbursement policies on attenuating such burden. METHODS A nation-based, cross-sectional, self-response survey was conducted in early 2018. Ninety-two PD patients responded, constituting all registered members of the only national PD patient organization in China. Twenty-four were child patients (age ≤18 years) and Sixty-eight were adult patients (age >18 years). The catastrophic health expenditure (CHE) and impoverishment rate due to illness were adopted to quantify the degree of economic burden. Two pilot reimbursement policies – the medicine-based pattern and the cost-based pattern – are analyzed to simulate policy impacts. RESULTS 94.12% of child patients and 67.21% of adults have suffered from CHE. Government-sponsored health insurance helps mildly with CHE. For child patients, the percentage of having CHE decreases to 88.24% after such reimbursement; and for adults, the percentage declines less significantly, on to 62.30%. 15.79 % of the child and 18.03% of the adult patient families live under the poverty line even before using medications. The percentages of poverty soar to 84.21% and 44.26%, respectively, after using medications. Health insurance reimbursement can only help decrease the burden very slightly to 78.95% and 42.62%. Assuming patients completely follow clinical dose of Alglucosidase alfa, under both pilot reimbursement policies, the ratio of CHE decreases 50% among child patients’ and 55% among adult families. However, for impoverishment, neither policies are effective in alleviating the situation. For both groups, the poverty rate rises to almost 100% under both policies. CONCLUSIONS The economic burden of PD, mainly caused by direct medical cost, is enormous, especially for families with child patients. Existing general health insurance can hardly alleviate such burden. The two pilot policies will be significantly effective in reducing the occurrence of CHE but not on impoverishment.
Conference/Value in Health Info
2020-09, ISPOR Asia Pacific 2020, Seoul, South Korea
Value in Health Regional, Volume 22S (September 2020)
Code
PRO7
Topic
Health Policy & Regulatory
Topic Subcategory
Reimbursement & Access Policy
Disease
Diabetes/Endocrine/Metabolic Disorders, Rare and Orphan Diseases