Objectives: Patients with rare diseases in China face extremely high medical expenses. The current coverage framework remains inadequate in terms of coverage depth and proactive risk control, underscoring an urgent need for institutional reform. Methods: This study employs a policy content analysis approach to review the current landscape of rare disease protection in China. Drawing on risk management theory and the health capital model, it constructs an analytical framework to examine potential institutional reforms through the lens of risk response pathways and the efficiency of health investment. Results: The findings reveal that basic medical insurance (BMI) provides limited financial protection for patients with rare diseases. Among China’s 31 provincial-level administrative centers, 24 have set general outpatient reimbursement ceilings under the urban and rural resident basic medical insurance (URRBMI) at 1000 RMB or less. In comparison, 24 cities have set outpatient reimbursement limits under the urban employee basic medical insurance (UEBMI) at 6000 RMB or less. The security system relies predominantly on the BMI, while supplementary mechanisms have failed to provide effective support or continuity in coverage. Current policies are generally reactive, with coverage typically triggered only after a confirmed diagnosis and often lacking early intervention or preventive strategies. Conclusions: China’s rare disease security system urgently requires structural improvements in coverage depth and proactive risk management. The proposed Dedicated Insurance Scheme for Rare Diseases (DISRD) presents a feasible and sustainable model for China’s multi-tiered system of securing rare diseases. It provides valuable institutional insights for other countries and regions seeking to build public health systems with proactive risk control capabilities.
Zhang et al. (Sun,) studied this question.