脊髓性肌萎缩药物经济学评价的系统综述

朱晓红; 刘世贤; 李顺平; 窦蕾; 王瑞雪; 宋泽华; 陈豪

doi:10.6039/j.issn.1001-0408.2024.15.12

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脊髓性肌萎缩药物经济学评价的系统综述

药物经济学 | 更新时间：2024-08-11

- 脊髓性肌萎缩药物经济学评价的系统综述
- Systematic review for pharmacoeconomics evaluation in spinal muscular atrophy
- 中国药房 2024年35卷第15期页码：1868-1875
- 作者机构：
  
  1.山东大学齐鲁医学院公共卫生学院卫生管理与政策研究中心，济南;250012
  2.国家卫生健康委员会卫生经济与政策研究重点实验室（山东大学），济南　250012
  3.山东大学健康偏好研究中心，济南　250012
- 作者简介：
  
  硕士研究生。研究方向：罕见病的药物经济学和卫生技术评估。E-mail：2870797942@qq.com
  教授，博士生导师，博士。研究方向：健康偏好与健康结果测量、罕见病诊疗与保障、卫生技术评估。E-mail：lishunping@ sdu.edu.cn
- 基金信息：
  
  国家自然科学基金面上项目(72174110)
- DOI：10.6039/j.issn.1001-0408.2024.15.12
  中图分类号： R956;R971
- 纸质出版日期：2024-08-15，
  
  收稿日期：2023-12-12，
  
  修回日期：2024-06-05，
- 稿件说明：
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朱晓红,刘世贤,李顺平等.脊髓性肌萎缩药物经济学评价的系统综述 ^Δ[J].中国药房,2024,35(15):1868-1875.

ZHU Xiaohong,LIU Shixian,LI Shunping,et al.Systematic review for pharmacoeconomics evaluation in spinal muscular atrophy[J].ZHONGGUO YAOFANG,2024,35(15):1868-1875.
朱晓红,刘世贤,李顺平等.脊髓性肌萎缩药物经济学评价的系统综述 ^Δ[J].中国药房,2024,35(15):1868-1875. DOI： 10.6039/j.issn.1001-0408.2024.15.12.

ZHU Xiaohong,LIU Shixian,LI Shunping,et al.Systematic review for pharmacoeconomics evaluation in spinal muscular atrophy[J].ZHONGGUO YAOFANG,2024,35(15):1868-1875. DOI： 10.6039/j.issn.1001-0408.2024.15.12.

摘要

目的

综述脊髓性肌萎缩（SMA）药物经济学评价的研究新进展，以期为相关临床治疗、筛查和医保支付决策提供参考。

方法

计算机检索PubMed、Web of Science、Embase、Scopus、Cochrane Library、EBSCOhost、中国知网、维普、中国生物医学文献数据库、万方数据等数据库和英国国家健康研究所、国际卫生保健技术评估协会、美国医疗保健研究与质量局等卫生技术评估（HTA）机构的网站，搜集与SMA相关的药物经济学评价研究。检索时限为建库至2023年12月31日。由2名研究人员严格按照纳入、排除标准进行文献/报告筛选，并使用Excel 2019软件提取所纳入文献/报告的基本信息；采用卫生经济学评价报告标准共识2022评价纳入文献/报告的研究质量。

结果

最终纳入9篇文献和15篇HTA报告，文献的质量整体较好，但HTA报告的质量较差。SMA药物经济学评价研究共计24项，治疗方案包括诺西那生钠、索伐瑞韦、利司扑兰和最佳支持治疗等。综述结果显示，诺西那生钠治疗SMA不具有经济性；利司扑兰和索伐瑞韦等治疗方案的经济性尚无一致结论；新生儿/产前筛查联合药物治疗方案具有经济性。

结论

新生儿/产前筛查联合药物治疗SMA具有经济学优势。建议未来基于我国本土化参数和医保价格继续探讨SMA新型治疗药物与SMA筛查相关的经济性，逐步将SMA筛查纳入新生儿遗传病检测范围，从而缓解患者家庭和卫生体系沉重的经济负担。

Abstract

OBJECTIVE

To review the current research progress on pharmacoeconomics evaluation related to spinal muscular atrophy （SMA）， in order to provide valuable insights for clinical treatment， screening and medical insurance payment decision-making.

METHODS

A computerized search was conducted across multiple databases including PubMed， Web of Science， Embase， Scopus， Cochrane Library， EBSCOhost， CNKI， VIP， CBM and Wanfang database as well as other important health technology assessment （HTA） websites， such as National Institute for Health and Care Research，International Society of Technology Assessment in Health Care， Agency for Healthcare Research and Quality， etc. The pharmacoeconomics evaluation studies related to SMA were collected from the inception to December 31st， 2023. The literature/reports were rigorously screened based on predefined inclusion and exclusion criteria by two researchers， and the essential information from the included literature/reports was extracted using Excel 2019. The quality of the included literature/reports was evaluated by Consolidated Health Economic Evaluation Reporting Standards 2022.

RESULTS

Finally， 9 articles and 15 HTA reports were included， with overall good quality of literature， but poor quality of HTA reports. There were a total of 24 studies on the pharmacoeconomics evaluation of SMA， including treatment options such as nusinersen sodium， sovaprevir， risperidone， and best supportive therapy. The review results showed that nusinersen sodium was not cost-effective in the treatment of SMA； there was no consensus on the economic viability of treatment options such as risperidone and sovaprevir； newborn/prenatal screening combined with medication therapy was cost-effective.

CONCLUSIONS

newborn/prenatal screening combined with SMA medication therapy demonstrates economic advantages. It is suggested to further investigate the cost-effectiveness of new SMA drugs and SMA screening in China， taking localization parameters and medical insurance prices into account， and gradually incorporate SMA screening into the scope of neonatal genetic disease detection， in order to alleviate the financial burden of patients’ families and healthcare systems.

关键词

脊髓性肌萎缩药物经济学系统综述诺西那生钠索伐瑞韦利司扑兰新生儿筛查

Keywords

pharmacoeconomicssystematic reviewnusinersen sodiumsovaprevirrisperidonenewborn screening

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