Budget Impact Analysis of High-priced Orphan Medicinal Products intended for the treatment of Rare Diseases in China: Evidence from a densely populated metropolis of Chengdu

Abstract Background In China, in addition to cost-related reasons, most high-priced orphan medicinal products (OMPs) remain excluded from the coverage of the social health insurance program due to the lack of reimbursement budget via economic evaluation approaches. Purpose To estimate the budget of high-priced OMPs for life-threatening rare diseases (RDs) from the societal and health care payers’ perspectives, while analyzing the budget sustainability. Methods Based on the patient data in a densely populated metropolis of Chengdu, China in 2019, the budget impact posed by high-priced OMPs was analyzed from a societal perspective. Next, a scenario analysis was performed based on 3 parameters, namely the assumed policy scenarios, the number of patients, and the OMPs price to assess the variation of the budget from the payers’ perspective. Finally, the budget for OMPs was estimated for the next three years. Results According to the survey, 98 rare disease patients were in the need of high-priced OMPs in Chengdu, China in 2019, which suggested the necessity of projecting a budget of CNY 179 million for this group of patients without the coverage of reimbursement policies from the societal perspective. Such budget would range from CNY 32 million to CNY 156 million under the context of six assumed policy scenarios. For the next three years, the budget for each single year was estimated to range from CNY 200 million to CNY 1.303 billion. Conclusions The study found that the financial burden posed by OMPs on each patient was exceptionally heavy, while the budget impact of OMPs for RDs on the whole society and payers was relatively limited due to the limited number of patients. The estimated budgets based on scenario analysis were expected to inform the formulation regarding OMPs.