摘要
目的:运用多维度决策分析的方法,对孤儿药的遴选条件进行研究。方法:采用问卷调查法对包括政府官员、医保部门管理人员、药企工作人员和医务科研在内的61位特定人群进行认知调查,采用SSPS进行统计分析,计算平均数和标准差,对上述4组调查对象的调查结果还进行了kendall等级相关的一致性检验。结果:前三位因素影响罕见病孤儿药价格高的主要原因依次为患病人数少、药品研发费用高和药品生产成本高。从遴选罕见病孤儿药的8条标准评价结果来看,重要性依次为孤儿药的治疗效果、药物的安全性、疾病的严重程度、医保基金的可负担性或治疗的公平性。在与波黑、波兰、捷克、希腊及匈牙利调查的结果进行比较发现,被调查的我国医保管理者对罕见病的严重程度、临床治疗效果、药物安全性、治疗公平性和医保基金的可负担性比较关心,而对未满足的需要、患者的偏好方面还不够重视,与国外调查结果略有不同。结论:我国已开始启动罕见病的防治政策,多渠道筹资机制正在形成,但对罕见病认知和对患者偏好的重视程度依然不够。
Objective： The selective criteria of orphan drugs in medical insurance reimbursement list were studied by using muhiple criteria decision analysis（MCDA） in China. Methods： A broad spectrum of stakeholders, including 61 government officials, medi- cal insurance managers, pharmaceutical Co. staff and academia were surveyed by questionnaires. SPSS was applied to conduct the sta- tistical analysis on the mean and standard deviation. Four groups of stakeholders were tested hy Kendall rank consistency. Results： The top 3 factors influenced on high price of orphan drugs were low number of rare diseases（RD） cases, high cost of R＆D（research and development） and high cost of production. From the 8 selective criteria for selecting RD orphan drugs, the order of importance was as follows： efficacy, safety, and severity of disease, the affordability of insurance budget and the equity of treatment. The results of the study in China were compared with that in Bosnia and Herzegovina, Poland, Czech, Greece and Hungary. It found that the govern- ment officials and medical insurance managers in China were paid more attention on the severity of RD, clinical efficacy, drug safety and the affordability of insurance budget. The unmet need and the preference of patients seemed not as importance as the other attrib- uted. The results were slightly different from that in foreign countries. Conclusion： In China, the prevention and control policy of RD had been started, and the muhi,channel financing mechanism were emerging. However, the recognition of RD and the preference for patients were still not enough.
出处
《中国卫生经济》
北大核心
2018年第3期67-70,共4页
Chinese Health Economics
关键词
罕见病
孤儿药
药物遴选
多维度决策
rare disease
orphan drug
drug selection
multiple decision
