OBJECTIVES:As improving value of health care is a goal supported by all stakeholders in health sector, it is crucial to develop a tool to measure value, especially for rare diseases. This study aims to establish a patient-centered value framework for hemophilia in China to help identify values.

METHODS: Literature review was employed to explore key value elements. By adopting multiple criteria decision analysis (MCDA) to build value framework, two-round Delphi survey was conducted to collect opinions concerning the importance, availability and sensitivity of each value element from 44 experts including physicians, pharmacists, health insurance officials, and patients. The weight of indicators was analyzed through Fuzzy evaluation method.

RESULTS: A patient-centered value framework for hemophilia has been developed with 5 tiers and 36 indicators based on Porter and IHASC’s model. Tier 1, health status achieved or retained, includes survival and hemophilia-specific health outcomes such as bleeding frequency, pain frequency/level, joint function, quality of life, etc. Tier 2, process of recovery, includes time to recovery such as bed days and disutility of care such as inhibitor development, pathogen transmission. Tier 3, sustainability of health, includes sustainability of recovery and long-term consequences of therapy measured by labor capacity, times of visits, long-term disutility of insufficient or inappropriate therapy. Tier 4, patient expense, refers to annual direct cost including total medical expenses, out-of-pocket expenses and long-term medical expenses. Tier 5, patient experience, is measured by patient’s satisfaction with the treatment process and compliance to medication. The results of indicator weights reveal that bleeding control, quality of life and drug cost are value indicators greatest concerned by stakeholders.

CONCLUSIONS: This value framework for hemophilia developed via MCDA provides a useful tool for multiple stakeholders to understand values of health care comprehensively and good basis for further value assessment for decision making.