Abstract
Background
As China works to establish a mature medical security system by 2030, the heavy medical expenses faced by patients with rare diseases and the inadequacies in the existing protection mechanisms have become increasingly prominent and problematic, creating an urgent need for further research and policy solutions.
Objectives and methods
This study used an explanatory sequential design. First, a questionnaire survey was conducted to investigate the status of the protection provided to patients by the three major security channels (government, market, and social organizations) and to assess the medical burden on patients. Then, interviews were conducted to identify the shortcomings of the three current major security channels, with the goal of developing an innovative mechanism for addressing the medical expenses of patients with rare diseases.
Results
The results from the 720 questionnaires showed that, among rare disease patients who could be contacted through patient organizations and related networks, the three major protection channels currently available are still insufficient to cover their high medical expenses, resulting in a heavy financial burden. The 14 interviews revealed two types of deficiencies in the three channels. First, each channel demonstrated limited protection effectiveness. Second, coordination among the three protection channels was lacking.
Conclusion
Policy and mechanism innovations are needed at two levels. First, to improve the effectiveness of the three channels, the government should expand the breadth and depth of coverage and gradually equalize protection levels across regions; the market should lower access thresholds for patients, develop a multi-tiered product supply system, and improve operational risk prevention and control mechanisms, while social organizations should strengthen their capacity to raise charitable resources, establish a sustainable project operation model, and build collaborative networks. Second, at the level of cross-channel coordination, the expense-sharing responsibilities of each entity must be clarified, the types of expenses and target patient populations covered by multiple entities should be better defined, and a multi-entity collaborative reimbursement mechanism should be established. Ultimately, a collaborative, multi-entity mechanism for covering the medical expenses of patients with rare diseases should be established.