Abad María Reyes, Alerany Carmen, González Luis Ignacio, Neth Olaf, Payares-Herrera Concepción, Rodríguez-Gallego Carlos, Trillo Jose Luis, Herrmann Kirsten H, Figueiredo Raquel, Gil Alicia
Pharmacy Department, H.U. Miguel Servet, Zaragoza - Spain.
Pharmacy Department, H.U. Vall d' Hebron, Barcelona - Spain.
Glob Reg Health Technol Assess. 2025 Jan 27;12:9-15. doi: 10.33393/grhta.2025.3199. eCollection 2025 Jan-Dec.
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.
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.
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.
Based on MCDA methodology and stakeholder experience in APDS management, leniolisib is seen as a value-added treatment option compared to SoC in Spain.
活化磷脂酰肌醇3激酶（PI3K）δ综合征（APDS）是一种极为罕见、可能危及生命的疾病，在西班牙尚无获批的治疗方法。本研究旨在应用多标准决策分析（MCDA）来评估西班牙针对APDS的首个药物治疗的价值。
一个由8名专家组成的多学科小组将选择性PI3Kδ抑制剂来那度胺与标准治疗（SoC）进行了评估。使用了一个为孤儿药（ODs）量身定制的MCDA框架，该框架由5个比较性标准和2个定量非比较性标准组成。重新评分是在小组讨论之后进行的。
来那度胺在所有标准（包括疗效和安全性）上的得分均高于SoC。它被认为作为首个疾病改善治疗具有很高的价值，具有积极的治疗效果和改善患者生活质量的潜力。此外，来那度胺可能会节省成本。支持数据被认为质量很高。
基于MCDA方法以及利益相关者在APDS管理方面的经验，与西班牙的SoC相比，来那度胺被视为一种增值治疗选择。
