Objective: From the perspective of the right to life and health, new evaluation indicators for rare disease drugs are proposed based on rescue rules and experience at home and abroad. Methods: The paper uses literature research method and comparative analysis method to systematically review domestic and foreign literature on the right to life and health, health technology assessment, and experience of medical insurance for orphan drugs, to provide theoretical basis and extract appropriate indicators. Results: Based on the rule of rescue, 20 indicators are proposed to evaluate orphan drugs considering the right to life and health, and some new dimensions for the fairness of orphan drugs are provided. Conclusion: To improve the accessibility of medicines for rare diseases, it is necessary to establish some special evaluation criteria. In this paper, a set of evaluation indicators for orphan drugs considering the right to life and health is suggested based on the rule of rescue, providing a new perspective to improve the coverage of rare diseases in China.
Key words
orphan drugs /
right to life and heath /
rule of rescue /
evaluation indicators
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