Abstract
BACKGROUND: According to the selection criteria outlined in China's current basic medical insurance system, the majority of drugs for rare diseases fail to meet the requirements for inclusion. Consequently, patients with rare diseases lack sufficient protection for their fundamental rights to life and health. The purpose of this paper is to explore the construction of a multi-criteria decision analysis (MCDA) system for the inclusion of rare disease drugs in medical insurance. METHODS: A preliminary evaluation system was constructed through a systematic literature review, followed by the establishment of a formal evaluation system through expert investigation. Based on the formal system, expert surveys were conducted to score the system attributes, and the pairwise comparison method was used to calculate the weight contribution value of each attribute in the system. The robustness of the system was tested using the bootstrap method. RESULTS: A multi-criteria decision value evaluation system for the inclusion of rare disease drugs in medical insurance, comprising four primary attributes and 16 secondary attributes, was developed. The four primary attributes and their respective weight contributions are as follows: disease-related aspects (26.6%), treatment-related aspects (25.8%), economic-related aspects (24.0%), and social-related aspects (23.6%). Bootstrap validation confirmed the stability of the system results (p < 0.05). CONCLUSION: This study initially constructed a value evaluation system for the inclusion of rare disease drugs in medical insurance, based on MCDA and multi-stakeholder perspectives. However, the system relied heavily on expert opinions and lacks empirical analysis. Given the complexity of real-world applications, further validation is necessary to assess the system's applicability and feasibility.