国家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 Injection and Risperdal Oral Solution with Dispersions

关键词:  Mini HTA  国谈药品  诺西那生  利司扑兰

Key words:  Mini HTA  Nationally Negotiated Drugs  Nusinersen  Risperdal

摘要:

目的:积极推进用于治疗罕见病脊髓性肌萎缩症(SMA)的国谈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、其他属性五大项内容量化评价。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:经HTA后可知尽管利司扑兰口服溶液用散为口服给药,仍需每日给药且需严格按照体重计算给药剂量,长远用药来看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,证据级别及强度均低于诺西那生。评价结果为医疗机构准入及临床合理使用二者药物提供有价值的参考。

Abstract:

ABSTRACT: OBJECTIVE: To actively promote the clinical access of Nusinersen injection and Risperdal oral solution with powder 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 injection and risperdal for oral solution were quantitatively evaluated in five major components. RESULTS: After the evaluation of the optimized evaluation system, the scores of pharmacological properties of Nusinersen Injection and Risperdal Oral Solution for Dispersal were 24 and 22.8 respectively, the scores of effectiveness were 25 and 23 respectively, the scores of safety were 13 and 14.9 respectively, the scores of economy were 13 and 12.67 respectively, and the scores of other attributes were 5.3 and 5 respectively, and the total scores were 80.3, 78.37 points. CONCLUSION:After HTA, it can be seen that even though risperdal oral solution is administered orally, it still needs to be administered daily and the dosage needs to be calculated strictly according to the body weight, and the economy is not as good as that of Nusinersen injection in terms of long-term medication, risperdal is marketed at a later stage, and the consensus of the recommended guideline is less than that of Nusinersen, and the level and strength of the evidence are lower than that of Nusinersen. The results of the evaluation provide valuable references for the access to healthcare organizations and the rational use of both drugs in clinical practice.