Value contribution of etranacogene dezaparvovec gene therapy in moderately severe and severe haemophilia B through multi-criteria decision analysis.

IF 3 2区医学 Q2 HEMATOLOGY

Haemophilia Pub Date : 2024-09-28 DOI:10.1111/hae.15096

Daniel-Aníbal García-Diego, Xavier Badia, Olga Benítez-Hidalgo, Víctor Jiménez, Juan Carlos Juárez, Ramiro Núñez, José Luis Poveda, José Luis Trillo, Joan-Antoni Vallés

{"title":"Value contribution of etranacogene dezaparvovec gene therapy in moderately severe and severe haemophilia B through multi-criteria decision analysis.","authors":"Daniel-Aníbal García-Diego, Xavier Badia, Olga Benítez-Hidalgo, Víctor Jiménez, Juan Carlos Juárez, Ramiro Núñez, José Luis Poveda, José Luis Trillo, Joan-Antoni Vallés","doi":"10.1111/hae.15096","DOIUrl":null,"url":null,"abstract":"Introduction: The value of gene therapies for haemophilia needs to be assessed holistically.Aim: To determine the value of etranacogene dezaparvovec (ED) compared to current extended half-life (EHL) recombinant factors (rFIX), using multi-criteria decision analysis (MCDA).Method: MCDA EVIDEM methodology adapted to orphan drugs was used, with nine quantitative criteria and four contextual criteria. The MCDA framework was rated by 28 multidisciplinary experts. Descriptive statistics were performed for quantitative and qualitative criteria.Results: Haemophilia B (HB) was considered a severe disease (mean ± SD: 4.3 ± 0.7) with some unmet needs (mean ± SD 3.3 ± 0.9). Experts found ED more effective (mean ± SD 2.0 ± 2.3) and provide better quality of life (QoL) (mean ± SD: 1.8 ± 1.5) than the comparative HB treatments but with safety uncertainties (mean ± SD -1.2 ± 1.8). ED could lead to medical cost and non-medical cost savings over time (mean ± SD: 1.6 ± 2.0 and 2.0 ± 1.5, respectively). The quality of the evidence was high (mean ± SD: 3.9 ± 0.9). ED was considered aligned with the priorities of the National Health System (NHS) and the specific interests of patients. ED's value contribution was 0.45 (+1 = highest value).Conclusions: ED brings added value in the treatment of moderately severe and severe HB (sHB) compared to current EHL rFIX, addressing the severity of the disease and increasing efficacy and patients' QoL especially related to the single dose and low bleeding rate. Concerns about long-term safety need to be addressed.","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":null,"pages":null},"PeriodicalIF":3.0000,"publicationDate":"2024-09-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Haemophilia","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1111/hae.15096","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"HEMATOLOGY","Score":null,"Total":0}

引用次数: 0

Abstract

Introduction: The value of gene therapies for haemophilia needs to be assessed holistically.

Aim: To determine the value of etranacogene dezaparvovec (ED) compared to current extended half-life (EHL) recombinant factors (rFIX), using multi-criteria decision analysis (MCDA).

Method: MCDA EVIDEM methodology adapted to orphan drugs was used, with nine quantitative criteria and four contextual criteria. The MCDA framework was rated by 28 multidisciplinary experts. Descriptive statistics were performed for quantitative and qualitative criteria.

Results: Haemophilia B (HB) was considered a severe disease (mean ± SD: 4.3 ± 0.7) with some unmet needs (mean ± SD 3.3 ± 0.9). Experts found ED more effective (mean ± SD 2.0 ± 2.3) and provide better quality of life (QoL) (mean ± SD: 1.8 ± 1.5) than the comparative HB treatments but with safety uncertainties (mean ± SD -1.2 ± 1.8). ED could lead to medical cost and non-medical cost savings over time (mean ± SD: 1.6 ± 2.0 and 2.0 ± 1.5, respectively). The quality of the evidence was high (mean ± SD: 3.9 ± 0.9). ED was considered aligned with the priorities of the National Health System (NHS) and the specific interests of patients. ED's value contribution was 0.45 (+1 = highest value).

Conclusions: ED brings added value in the treatment of moderately severe and severe HB (sHB) compared to current EHL rFIX, addressing the severity of the disease and increasing efficacy and patients' QoL especially related to the single dose and low bleeding rate. Concerns about long-term safety need to be addressed.

查看原文本刊更多论文

通过多标准决策分析确定 etranacogene dezaparvovec 基因疗法对中度和重度血友病 B 的价值贡献。

简介：血友病基因疗法的价值需要全面评估：目的：采用多标准决策分析（MCDA）方法，确定 etranacogene dezaparvovec（ED）与当前的延长半衰期（EHL）重组因子（rFIX）相比的价值：方法：采用适用于孤儿药的 MCDA EVIDEM 方法，其中包括 9 个定量标准和 4 个背景标准。28 位多学科专家对 MCDA 框架进行了评分。对定量和定性标准进行了描述性统计：结果：血友病 B（HB）被认为是一种严重疾病（平均值±标准差：4.3±0.7），有一些需求尚未得到满足（平均值±标准差：3.3±0.9）。专家们发现，与 HB 治疗相比，ED 更有效（平均值±标准差：2.0 ± 2.3），并能提供更好的生活质量（QoL）（平均值±标准差：1.8 ± 1.5），但安全性不确定（平均值±标准差：-1.2 ± 1.8）。随着时间的推移，ED可节省医疗费用和非医疗费用（平均值±标准差：分别为1.6±2.0和2.0±1.5）。证据质量较高（平均值±标准差：3.9±0.9）。ED 被认为符合国家卫生系统（NHS）的优先事项和患者的具体利益。ED 的价值贡献为 0.45（+1 = 最高价值）：与目前的 EHL rFIX 相比，ED 为中重度和重度 HB（sHB）的治疗带来了附加值，解决了疾病的严重性问题，提高了疗效和患者的 QoL，特别是与单剂量和低出血率有关。对长期安全性的担忧需要解决。

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

Haemophilia 医学-血液学

CiteScore

6.50

自引率

28.20%

发文量

226

审稿时长

3-6 weeks

期刊介绍： Haemophilia is an international journal dedicated to the exchange of information regarding the comprehensive care of haemophilia. The Journal contains review articles, original scientific papers and case reports related to haemophilia care, with frequent supplements. Subjects covered include: clotting factor deficiencies, both inherited and acquired: haemophilia A, B, von Willebrand''s disease, deficiencies of factor V, VII, X and XI replacement therapy for clotting factor deficiencies component therapy in the developing world transfusion transmitted disease haemophilia care and paediatrics, orthopaedics, gynaecology and obstetrics nursing laboratory diagnosis carrier detection psycho-social concerns economic issues audit inherited platelet disorders.