Affiliation:
1. Sichuan University West China School of Public Health
2. Sichuan University
Abstract
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.
Publisher
Research Square Platform LLC
Reference41 articles.
1. Can the Consideration of Societal Costs Change the Recommendation of Economic Evaluations in the Field of Rare Diseases? An Empirical Analysis;Aranda-Reneo I;Value in Health,2021
2. Rare diseases research and policy in Australia: On the journey to equitable care;Bhattacharya K;J Paediatr Child Health,2021
3. Market Exclusivity for Drugs with Multiple Orphan Approvals (1983–2017) and Associated Budget Impact in the US;Padula WV;PharmacoEconomics
4. Li Q, Chen F, Yang M, et al. The Effect of China’s National Essential Medicine Policy on Health Expenses: Evidence From a National Study. INQUIRY: The Journal of Health Care Organization, Provision, and Financing. 2018/01/01 2018;55:0046958018787057. doi:10.1177/0046958018787057.
5. Franco P. Orphan drugs: the regulatory environment. Drug Discovery Today. 2013/02/01/ 2013;18(3):163–172. doi:https://doi.org/10.1016/j.drudis.2012.08.009.