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经济有效地使用生物制剂和靶向合成 DMARDs 治疗炎症性风湿病的考虑要点:伞式评价和国际 Delphi 研究的结果。

Points to consider for cost-effective use of biological and targeted synthetic DMARDs in inflammatory rheumatic diseases: results from an umbrella review and international Delphi study.

机构信息

Department of Rheumatology, Sint Maartenskliniek, Nijmegen, The Netherlands

Department of Rheumatology, Radboud Institute for Health Sciences, Radboudumc, Nijmegen, The Netherlands.

出版信息

RMD Open. 2023 Mar;9(1). doi: 10.1136/rmdopen-2022-002898.

Abstract

OBJECTIVES

To develop evidence-based points to consider for cost-effective use of biological and targeted synthetic disease-modifying antirheumatic drugs (b/tsDMARDs) in the treatment of inflammatory rheumatic diseases, specifically rheumatoid arthritis, psoriatic arthritis and axial spondyloarthritis.

METHODS

Following EULAR procedures, an international task force was formed, consisting of 13 experts in rheumatology, epidemiology and pharmacology from seven European countries. Twelve strategies for cost-effective use of b/tsDMARDs were identified through individual and group discussion. For each strategy, PubMed and Embase were systematically searched for relevant English-language systematic reviews and, for six strategies, additionally for randomised controlled trials (RCTs). Thirty systematic reviews and 21 RCTs were included. Based on the evidence, a set of overarching principles and points to consider was formulated by the task force using a Delphi procedure. Level of evidence (1a-5) and grade (A-D) were determined for each point to consider. Individual voting on the level of agreement (LoA; between 0 (completely disagree) and 10 (completely agree)) was performed anonymously.

RESULTS

The task force agreed on five overarching principles. For 10 of 12 strategies, the evidence was sufficient to formulate one or more points to consider, leading to 20 in total, regarding response prediction, drug formulary use, biosimilars, loading doses, low-dose initial therapy, concomitant conventional synthetic DMARD use, route of administration, medication adherence, disease activity-guided dose optimisation and non-medical drug switching. Ten points to consider (50%) were supported by level 1 or 2 evidence. The mean LoA (SD) varied between 7.9 (1.2) and 9.8 (0.4).

CONCLUSION

These points to consider can be used in rheumatology practices and complement inflammatory rheumatic disease treatment guidelines to incorporate cost-effectiveness in b/tsDMARD treatment.

摘要

目的

为生物制剂和靶向合成疾病修正抗风湿药物(b/tsDMARDs)在治疗炎症性风湿性疾病(特别是类风湿关节炎、银屑病关节炎和中轴型脊柱关节炎)中的经济有效使用制定基于证据的考虑要点。

方法

按照 EULAR 程序,成立了一个由来自 7 个欧洲国家的 13 名风湿病学、流行病学和药理学专家组成的国际工作组。通过个人和小组讨论确定了 12 项 b/tsDMARD 经济有效使用策略。对于每个策略,系统地在 PubMed 和 Embase 上搜索了相关的英语系统评价,对于其中 6 个策略,还搜索了随机对照试验(RCTs)。纳入了 30 项系统评价和 21 项 RCT。基于证据,工作组通过 Delphi 程序制定了一套总体原则和考虑要点。为每个考虑要点确定了证据级别(1a-5)和等级(A-D)。通过匿名投票对一致性水平(LoA;0 表示完全不同意,10 表示完全同意)进行了评估。

结果

工作组就五项总体原则达成一致。对于 12 项策略中的 10 项,有足够的证据来制定一个或多个考虑要点,总共制定了 20 个要点,涉及反应预测、药物处方使用、生物类似物、负荷剂量、低剂量初始治疗、同时使用传统合成 DMARD、给药途径、药物依从性、基于疾病活动的剂量优化和非药物药物转换。10 个考虑要点(50%)得到了 1 级或 2 级证据的支持。平均 LoA(SD)在 7.9(1.2)到 9.8(0.4)之间。

结论

这些考虑要点可用于风湿病学实践,并补充炎症性风湿性疾病治疗指南,以将成本效益纳入 b/tsDMARD 治疗。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/2448/9990692/91fd7d2e7073/rmdopen-2022-002898f01.jpg

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