Study On The Evaluation Indicator System And Influencing Factors Of Orphan Drug Accessibility In China

Objective:To identify the evaluation indicator system and obstacle factors for orphan drug accessibility in China, and explore the policy and management strategies to help improve access to orphan drugs and the quality of life for the patients suffered with rare diseases.Methods and contents:In literature-based study, some concepts such as rare disease, orphan drug and drug accessibility are defined. And the laws and policies about rare disease and orphan drug and health insurance in China and abroad are illustrated. The evaluation indicator system for drug accessibility and orphan drug accessibility in abroad research literatures are analyzed comparatively. According to the Delphi expert survey method, we construct the evaluation indicator system for orphan drug accessibility. And based on the subjective weighting method, we determine the weight of indicators.The importance and feasibility of the indicators are identified and classified with the Fuzzy pattern recognition. Then, we used the structure survey method of questionnaire to interview the patients with rare diseases nationwide, and analyzed the obstacle factors for the patients with rare diseases to access to orphan drugs and found some effective management strategies to improve orphan drug accessibility.Results:The evaluation indicator system for orphan drug accessibility was constructed from availability, utilizability, affordability and responsiveness of health system through two rounds of experts’s survey. The levels of evaluation indicators consist of four indicators at first level, nine indicators at second level and seventy-nine indicators at third level. The weight of evaluation indicators was determined by subjective weighting method. The importance and feasibility of the indicators was classified and ranked. The result of the structure survey for patients with rare diseases showed that some barrier factors of orphan drug accessibility include mainly the unaffordable medical expenses, lack of medical insurance payment, unavailability of orphan drug, the big difficulty in diagnosing and treating rare disease, safe question of medication, the difficulties of patients with rare disease being accepted into society and insufficient measures of rare disease prevention and treatment in China.Conclusion:Our government should establish orphan drug incentive policies, and develop guidelines for diagnosis and treatment, construct rare disease diagnosis and treatment system, build up the orphan drug information communication platform, improve the medical insurance coverage and reimbursement level of rare diseases, support the patient organizations for rare disease, enhance the responsiveness of health system, set up the orphan drug reserve system and rare disease preventions systems in China.