国家医保谈判药品诺西那生钠注射液、利司扑兰口服溶液用散的数字化Mini卫生技术评估  

Digital Mini Health Technology Assessment of the Nationally Negotiated Drugs Nusinersen Sodium Injection and Risdiplam Powder for Oral Solution

作　　者：李宵[1] 邱志宏[1] 于泽芳 董占军[1] LI Xiao;QIU Zhihong;YU Zefang;DONG Zhanjun(Hebei Key Laboratory of Clinical Pharmacy,Hebei General Hospital,Shijiazhuang 050051,China)

机构地区：[1]河北省人民医院,河北省临床药学重点实验室,石家庄050051

出　　处：《中国现代应用药学》2025年第4期653-664,共12页Chinese Journal of Modern Applied Pharmacy

基　　金：河北省自然科学基金项目(H2022307063);河北省卫生健康委医学科学研究课题(20230312)。

摘　　要：目的积极推进用于治疗罕见病脊髓性肌萎缩症(spinal muscular atrophy,SMA)的国家医保谈判药品诺西那生钠注射液、利司扑兰口服溶液用散的临床准入工作,促进二者临床合理用药。方法通过运用《中国医疗机构药品评价与快速遴选指南(第二版)》,对诺西那生钠注射液、利司扑兰口服溶液用散进行有效性、药学特性、安全性、经济性、其他属性5大项内容量化评价。结果经优化后的评价体系评价后,诺西那生钠注射液、利司扑兰口服溶液用散药学特性得分分别为24、22.8分,有效性得分分别为25、23分,安全性得分分别为13、14.9分,经济性得分分别为13、12.67分,其他属性得分分别为5.3、5分,总分分别为80.3、78.37分。结论利司扑兰口服溶液用散为口服给药,需严格按照体质量计算给药剂量进行每日给药,长期用药经济性不及诺西那生钠注射液。利司扑兰上市较晚,推荐的指南共识少于诺西那生钠,证据级别及强度均低于诺西那生钠。评价结果为医疗机构准入及临床合理使用2种药物提供有价值的参考。OBJECTIVE To actively promote the clinical access of nusinersen sodium injection and risdiplam powder for oral solution for the treatment of spinal muscular atrophy(SMA),a rare disease,and to promote the rational use of these two drugs in clinical practice.METHODS By applying the"Guidelines for Drug Evaluation and Rapid Selection in Chinese Medical Institutions(Second Edition)",the effectiveness,pharmacological properties,safety,economy,and other attributes of nusinersen sodium injection and risdiplam powder for oral solution were quantitatively evaluated in 5 major components.RESULTS After the evaluation of the optimized evaluation system,the scores of pharmacological properties of nusinersen sodium injection and risdiplam powder for oral solution were 24 and 22.8 points respectively;the scores of effectiveness were 25 and 23 points respectively;the scores of safety were 13 and 14.9 points respectively,the scores of economy were 13 and 12.67 points respectively;the scores of other attributes were 5.3 and 5 points respectively;and the total scores were 80.3,78.37 points respectively.CONCLUSION Risdiplam powder for oral solution is administered orally in powder form,and the dosage needs to be calculated strictly according to the body weight for daily administration.The economy is not as good as that of nusinersen sodium injection in terms of long-term medication.Risdiplam is marketed at a later stage,and the consensus of the recommended guideline is less than that of nusinersen sodium,and the level and strength of the evidence are lower than that of nusinersen sodium.The results of the evaluation provide valuable references for the access to healthcare organizations and the rational use of both drugs in clinical practice.

关 键 词：Mini卫生技术评估 国家医保谈判药品 诺西那生钠 利司扑兰 

分 类 号：R969.3[医药卫生—药理学]