How to evaluate rare disease policy effectiveness based on policy modeling consistency (PMC) index model: A quantitative assessment of policy implementation in China

Based on the Policy Modeling Consistency (PMC) index model, this study systematically evaluates 19 national-level and 13 regional-level rare disease policy documents from 2016 to 2025, aiming to reveal the structural characteristics and consistency level of China's rare disease policy system. Using ROSTCM 6.0 software, high-frequency keywords and semantic networks from the policy texts were extracted to identify policy focus and thematic evolution. A PMC evaluation system was constructed to assign values and conduct a visual analysis of the sample policies. Results show that the average PMC index for China's rare disease policies is 5.31 (national level) and 4.94 (local level), falling within the "excellent-acceptable" range overall, indicating that the structure of China's rare disease policy system is well-developed. Among the indicators, policy nature (X2), policy function (X8), and outcome orientation (X4) scored higher, reflecting strategic planning and institutional characteristics at the national level. In contrast, incentive measures (X5), patient support (X6), and social support (X9) scored lower, highlighting deficiencies in research and development incentives, social integration, and long-term support mechanisms. Further comparisons reveal that national policies emphasize top-level design and institutional development. In contrast, local policies focus more on exploratory efforts in medical assistance and policy innovation. To optimize China's rare disease policy system, it is essential to improve incentive and social support systems, promote coordinated legislation between national and local levels, establish a national rare disease information platform and a dedicated fund, and build a comprehensive policy chain.