Value contribution of leniolisib in the Treatment of Activated PI3Kδ syndrome (APDS) in Spain using Multi-Criteria Decision Analysis (MCDA).

IF 0.4 Q4 HEALTH CARE SCIENCES & SERVICES

Global & Regional Health Technology Assessment Pub Date : 2025-01-27 eCollection Date: 2025-01-01 DOI:10.33393/grhta.2025.3199

María Reyes Abad, Carmen Alerany, Luis Ignacio González, Olaf Neth, Concepción Payares-Herrera, Carlos Rodríguez-Gallego, Jose Luis Trillo, Kirsten H Herrmann, Raquel Figueiredo, Alicia Gil

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引用次数: 0

Abstract

Background: Activated phosphoinositide 3-kinase (PI3K) δ Syndrome (APDS) is an ultra-rare, potentially life-threatening disease that lacks approved treatments in Spain. This study aimed to apply Multi-Criteria Decision Analysis (MCDA) to assess the value of the first pharmacological treatment for APDS in Spain.

Methods: A multidisciplinary group of 8 experts evaluated the selective PI3Kδ inhibitor leniolisib against Standard of Care (SoC). An MCDA framework tailored for Orphan Drugs (ODs), consisting of 5 comparative and 2 quantitative non-comparative criteria, was used. Re-scoring followed a group discussion.

Results: Leniolisib scored higher than SoC in all criteria, including efficacy and safety. It was deemed highly valuable as the first disease-modifying treatment, with a positive therapeutic impact and potential to improve patients' quality of life. Additionally, leniolisib may lead to cost savings. The supporting data was considered of high quality.

Conclusion: Based on MCDA methodology and stakeholder experience in APDS management, leniolisib is seen as a value-added treatment option compared to SoC in Spain.

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应用多标准决策分析（MCDA）研究lenolisib在西班牙治疗活化PI3Kδ综合征（APDS）中的价值贡献。

背景：活化磷酸肌肽3-激酶(PI3K) δ综合征（APDS）是一种超罕见的潜在威胁生命的疾病，在西班牙缺乏批准的治疗方法。本研究旨在应用多标准决策分析（MCDA）来评估西班牙APDS首次药物治疗的价值。方法：一个由8名专家组成的多学科小组评估了选择性PI3Kδ抑制剂lenolisib与标准护理（SoC）的关系。采用了为孤儿药量身定制的MCDA框架，包括5个比较标准和2个定量非比较标准。小组讨论后重新打分。结果：lenolisib的疗效和安全性评分均高于SoC。作为第一种改善疾病的治疗方法，它被认为具有很高的价值，具有积极的治疗效果和改善患者生活质量的潜力。此外，宽松化可能会节省成本。支持数据被认为质量高。结论：基于MCDA方法和利益相关者在APDS管理中的经验，与西班牙的SoC相比，leniolisib被视为一种增值治疗选择。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Global & Regional Health Technology Assessment HEALTH CARE SCIENCES & SERVICES-

CiteScore

0.80

自引率

20.00%

发文量

审稿时长

8 weeks

期刊介绍： Global & Regional Health Technology Assessment (GRHTA) is a peer-reviewed, open access journal which aims to promote health technology assessment and economic evaluation, enabling choices among alternative therapeutical paths or procedures with different clinical and economic outcomes. GRHTA is a unique journal having three different editorial boards who focus on their respective geographical expertise.