Can SMA Innovative Treatments Be Reimbursed? A Rapid Review

IF 0.4 4区医学 Q4 PEDIATRICS

Iranian Journal of Pediatrics Pub Date : 2024-07-24 DOI:10.5812/ijp-145056

Fateme Yaftian, M. Mobinizadeh, A. Olyaeemanesh, E. Mohamadi, Farhad Habibi

{"title":"Can SMA Innovative Treatments Be Reimbursed? A Rapid Review","authors":"Fateme Yaftian, M. Mobinizadeh, A. Olyaeemanesh, E. Mohamadi, Farhad Habibi","doi":"10.5812/ijp-145056","DOIUrl":null,"url":null,"abstract":"Context: Spinal muscular atrophy (SMA) is a rare genetic disorder that significantly impacts the quality of life. This paper aims to gather current understanding of the safety, efficacy, and economic aspects of common SMA treatments to assist Iranian policymakers in adopting these novel treatments for this vulnerable population. Methods: This rapid health technology assessment (HTA) research was conducted in four steps: Database review, screening, data extraction, and thematic analysis. Inclusion criteria consisted of studies focused on assessing the safety, efficacy, and economic aspects of medical interventions in SMA patients compared to those who did not receive such interventions. Results: Based on current data, nusinersen was found to be the most effective treatment (increasing mobility achievements in SMA types 1 and 2) with the least side effects for SMA types 1 and 2. In terms of economic evaluations, none of the treatments were found to be cost-effective. Conclusions: The decision regarding reimbursement for such medical drugs should not focus solely on their cost-effectiveness but rather on creating access to essential care, meeting patient needs, and considering national budget limitations. Managed entry agreements (MEAs) are flexible tools that service providers or payers can use to negotiate and reduce the financial burden for both patients and payers.","PeriodicalId":14593,"journal":{"name":"Iranian Journal of Pediatrics","volume":null,"pages":null},"PeriodicalIF":0.4000,"publicationDate":"2024-07-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Iranian Journal of Pediatrics","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.5812/ijp-145056","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q4","JCRName":"PEDIATRICS","Score":null,"Total":0}

引用次数: 0

Abstract

Context: Spinal muscular atrophy (SMA) is a rare genetic disorder that significantly impacts the quality of life. This paper aims to gather current understanding of the safety, efficacy, and economic aspects of common SMA treatments to assist Iranian policymakers in adopting these novel treatments for this vulnerable population. Methods: This rapid health technology assessment (HTA) research was conducted in four steps: Database review, screening, data extraction, and thematic analysis. Inclusion criteria consisted of studies focused on assessing the safety, efficacy, and economic aspects of medical interventions in SMA patients compared to those who did not receive such interventions. Results: Based on current data, nusinersen was found to be the most effective treatment (increasing mobility achievements in SMA types 1 and 2) with the least side effects for SMA types 1 and 2. In terms of economic evaluations, none of the treatments were found to be cost-effective. Conclusions: The decision regarding reimbursement for such medical drugs should not focus solely on their cost-effectiveness but rather on creating access to essential care, meeting patient needs, and considering national budget limitations. Managed entry agreements (MEAs) are flexible tools that service providers or payers can use to negotiate and reduce the financial burden for both patients and payers.

查看原文本刊更多论文

SMA 创新疗法可以报销吗？快速回顾

背景：脊髓性肌萎缩症（SMA）是一种罕见的遗传性疾病，严重影响患者的生活质量。本文旨在收集目前对常见 SMA 治疗方法的安全性、有效性和经济性方面的了解，以帮助伊朗决策者为这一弱势群体采用这些新型治疗方法。方法：这项快速卫生技术评估 (HTA) 研究分四个步骤进行：数据库审查、筛选、数据提取和专题分析。纳入标准包括重点评估与未接受医疗干预的 SMA 患者相比，医疗干预的安全性、有效性和经济性的研究。结果：根据目前的数据发现，对于 1 型和 2 型 SMA 患者，纽西奈森是最有效的治疗方法（可提高 SMA 1 型和 2 型患者的活动能力），且副作用最小。在经济评估方面，没有发现任何一种治疗方法具有成本效益。结论在决定此类医疗药物的报销时，不应只关注其成本效益，而应关注是否能获得基本医疗服务、是否能满足患者需求以及是否考虑到国家预算限制。管理性进入协议（MEA）是一种灵活的工具，服务提供者或支付者可以利用它来协商并减轻患者和支付者的经济负担。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

求助全文

约1分钟内获得全文求助全文

来源期刊

Iranian Journal of Pediatrics 医学-小儿科

CiteScore

0.90

自引率

20.00%

发文量

审稿时长

6-12 weeks

期刊介绍： Iranian Journal of Pediatrics (Iran J Pediatr) is a peer-reviewed medical publication. The purpose of Iran J Pediatr is to increase knowledge, stimulate research in all fields of Pediatrics, and promote better management of pediatric patients. To achieve the goals, the journal publishes basic, biomedical, and clinical investigations on prevalent diseases relevant to pediatrics. The acceptance criteria for all papers are the quality and originality of the research and their significance to our readership. Except where otherwise stated, manuscripts are peer-reviewed by minimum three anonymous reviewers. The Editorial Board reserves the right to refuse any material for publication and advises that authors should retain copies of submitted manuscripts and correspondence as the material cannot be returned. Final acceptance or rejection rests with the Editors.