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评估精准医学的成本效益:系统评价和荟萃分析方案。

Assessing the cost-effectiveness of precision medicine: protocol for a systematic review and meta-analysis.

机构信息

Saw Swee Hock School of Public Health, National University of Singapore, Singapore

Department of Family Medicine, Faculty of Medicine, Ramathibodi Hospital, Mahidol University, Salaya, Nakhon Pathom, Thailand.

出版信息

BMJ Open. 2022 Apr 5;12(4):e057537. doi: 10.1136/bmjopen-2021-057537.

DOI:10.1136/bmjopen-2021-057537
PMID:35383079
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC8984003/
Abstract

INTRODUCTION

Precision medicine (PM) involves gene testing to identify disease risk, enable early diagnosis or guide therapeutic choice, and targeted gene therapy. We aim to perform a systematic review and meta-analysis to quantify the cost-effectiveness profile of PM stratified by intervention type, identify sources of heterogeneity in the value-for-money of PM.

METHODS AND ANALYSIS

We will perform a systematic search in Embase, MEDLINE, EconLit and CRD databases for studies published in English language or with translation in English between 1 January 2011 and 8 July 2021 on the topic of cost-effectiveness analysis of PM interventions. The focus will be on studies that reported health and economic outcomes. Study quality will be assessed using the Biases in Economic Studies checklist. The incremental net benefit of PM screening, diagnostic, treatment-targeting and therapeutic interventions over conventional strategies will be respectively pooled across studies using a random-effect model if heterogeneity is present, otherwise a fixed-effect model. Subgroup analyses will be performed based on disease area, WHO region and World Bank country-income level. Additionally, we will identify the potential sources of heterogeneity with random-effect meta-regressions. Finally, biases will be detected using jackknife sensitivity analysis, funnel plot assessment and Egger's tests.

ETHICS AND DISSEMINATION

For this type of study ethics approval or formal consent is not required. The results will be disseminated at various presentations and feedback sessions, in conference abstracts and manuscripts that will be submitted to peer-reviewed journals.

PROSPERO REGISTRATION NUMBER

CRD42021272956.

摘要

简介

精准医学(PM)涉及基因检测以识别疾病风险,实现早期诊断或指导治疗选择,并进行靶向基因治疗。我们旨在进行系统评价和荟萃分析,以量化按干预类型分层的 PM 的成本效益概况,并确定 PM 的性价比存在差异的原因。

方法和分析

我们将在 Embase、MEDLINE、EconLit 和 CRD 数据库中进行系统检索,检索 2011 年 1 月 1 日至 2021 年 7 月 8 日期间以 PM 干预措施的成本效益分析为主题的英文发表或有英文翻译的研究。重点将放在报告健康和经济结果的研究上。使用经济研究偏倚清单评估研究质量。如果存在异质性,则使用随机效应模型对 PM 筛查、诊断、治疗靶向和治疗干预相对于传统策略的增量净效益进行跨研究汇总;否则,使用固定效应模型。将根据疾病领域、世界卫生组织区域和世界银行国家收入水平进行亚组分析。此外,我们将使用随机效应荟萃回归来确定潜在的异质来源。最后,将使用刀切敏感性分析、漏斗图评估和 Egger 检验来检测偏差。

伦理和传播

对于这种类型的研究,不需要伦理批准或正式同意。研究结果将在各种演讲和反馈会议、会议摘要和提交给同行评议期刊的手稿中进行传播。

PROSPERO 注册号:CRD42021272956。

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