2019年甘肃省罕见病治疗费用核算分析

The treatment expenditure of rare diseases in Gansu province in 2019

目的 探究甘肃省2019年罕见病治疗费用的总量、人群受益分布、机构流向分布以及住院费用影响因素,为制定罕见病相关政策提供理论依据。方法 基于“卫生费用核算体系2011”(SHA2011)理论框架,利用甘肃省2019年官方统计资料提供的治疗费用总量数据和五级分层抽样调查获取的样本数据,采取“自上而下”的分摊方法,核算2019年甘肃省罕见病治疗费用,并采用K-Means聚类和支持向量机模型分析罕见病住院费用的影响因素。结果 2019年甘肃省罕见病治疗费用7 836.25万元;血液及造血器官疾病和其他某些涉及免疫系统的疾患为治疗费用最高的疾病类型,共2 801.21万元(35.75%);血友病、苯丙酮尿症和特发性肺动脉高压为治疗费用排名前三位的单病种,共占罕见病治疗费用的65.89%;<15岁人群发生的治疗费用最高;65.99%的治疗费用主要流向医院;97.67%的住院治疗费用发生在医院,以省级机构为主;罕见病次均治疗费用为2 284.94元,次均门诊费用为1 033.66元,次均住院费用为11 089.28元。罕见病住院费用的影响因素按重要性排序为:机构级别、药品费用占住院费用的比重、疾病类型、住院时间、年龄、是否并发。结论 甘肃省罕见病医疗释放不充分,疾病负担较重,应对重点罕见病人群给予更多的政策扶持,建立更完善的罕见病医疗保障和诊治体系。

Objective This study estimated the overall scale,population distribution,and institutional distribution of treatment expenditure of rare diseases as well as the factors affecting the hospitalization expenditure of rare diseases in Gansu province in 2019,so as to provide references for policies related to rare diseases in China.Methods Based on the theoretical framework of the System of Health Accounts 2011(SHA2011),the"top-down"decomposition accounting method was adopted to calculate the treatment cost of rare diseases in Gansu Province in 2019 using the total treatment cost data provided by the official statistics and data obtained from five-level stratified sampling survey.K-means clustering and support vector machine were used to investigate the influencing factors of hospitalization costs of rare diseases.Results In 2019,the total treatment cost of rare diseases in Gansu province was 78.3625 million yuan.The highest treatment cost was 28.0121 million yuan(35.75%)for the diseases of blood and hematopoietic organs and some diseases involving the immune system.The top three single rare diseases with the highest treatment cost were hemophilia,phenylketonuria and idiopathic pulmonary arterial hypertension,accounting for 65.89%of the total treatment cost of all rare diseases.Among the age groups,the highest treatment cost occurred in patients under 15 years.65.99%of the treatment expenses for rare diseases was mainly used in hospitals.97.67%of hospitalization costs occurred in hospitals,mostly in provincial ones.The average treatment cost,the average outpatient clinic visit cost,and the average hospitalization expense were 2284.94 yuan,1033.66 yuan,and 11089.28 yuan,respectively.The influencing factors of hospitalization cost of rare diseases,in the order of significance,was the level of health care facility,the proportion of drug cost in hospitalization cost,the type of disease,length of hospital stay,age,and comorbidities.Conclusions The medical resources for rare diseases may not be sufficiently utilized in Gansu province,and the burden of rare diseases is heavy.Therefore,more supporting policies should be implemented for the key patient population of rare diseases,and an improved medical insurance system as well as a diagnosis and treatment system be established for rare diseases.