de Andrés-Nogales Fernando, Casado Miguel Ángel, Trillo José Luis, Ruiz-Moreno José María, Martínez-Sesmero José Manuel, Peralta Gemma, Poveda José Luis, Ortiz Pere, Ignacio Emilio, Zarranz-Ventura Javier, Udaondo Patricia, Mur Carlos, Álvarez Eloísa, Cervera Enrique, Martínez Mercedes, Llorente Iñaki, Zulueta Jacinto, Rodríguez-Maqueda Mariano, García-Layana Alfredo, Martínez-Olmos José
Pharmacoeconomics & Outcomes Research Iberia (PORIB), Madrid, Spain.
Departamento de Salud Clínico Malvarrosa, Valencia, Spain.
Pharmacoecon Open. 2020 Dec;4(4):615-624. doi: 10.1007/s41669-020-00201-2.
The clinical and economic management of retinal diseases has become more complex following the introduction of new intravitreal treatments. Multicriteria decision analysis (MCDA) offers the potential to overcome the challenges associated with traditional decision-making tools.
A MCDA to determine the most relevant criteria to decision-making in the management of diabetic macular edema (DME) based on the perspectives of multiple stakeholders in Spain was developed. This MCDA was termed the MULTIDEX-EMD study.
Nineteen stakeholders (7 physicians, 4 pharmacists, 5 health authorities and health management experts, 1 psychologist, and 2 patient representatives) participated in this three-phase project. In phase A, an advisory board defined all of the criteria that could influence DME treatment decision-making. These criteria were then screened using a discrete choice experiment (DCE) (phase B). Next, a multinomial logit model was fitted by applying the backward elimination algorithm (relevant criteria: p value < 0.05). Finally, the results were discussed in a deliberative process (phase C).
Thirty-one criteria were initially defined (phase A) and grouped into 5 categories: efficacy/effectiveness, safety, organizational and economic impact, patient-reported outcomes, and other therapeutic features. The DCE results (phase B) showed that 10 criteria were relevant to the decision-making process for a 50- to 65-year-old DME patient: mean change in best corrected visual acuity (p value < 0.001), percentage of patients with an improvement of ≥ 15 letters (p value < 0.001), effect duration per administration (p value = 0.008), retinal detachment (p value < 0.001), endophthalmitis (p value = 0.012), myocardial infarction (p value < 0.001), intravitreal hemorrhage (p value = 0.021), annual treatment cost per patient (p value = 0.001), health-related quality of life (HRQoL) (p value = 0.004), and disability level (p value = 0.021).
From a multi-stakeholder perspective, the selection of an appropriate treatment for DME patients should guarantee patient safety and maximize the visual acuity improvement and treatment effect duration. It should also contribute to system sustainability by being affordable, it should have a positive impact on HRQoL, and it should prevent disability.
随着新型玻璃体内注射治疗方法的引入，视网膜疾病的临床和经济管理变得更加复杂。多标准决策分析（MCDA）为克服传统决策工具带来的挑战提供了可能。
基于西班牙多个利益相关者的视角，开展一项多标准决策分析，以确定糖尿病性黄斑水肿（DME）管理中与决策最相关的标准。这项多标准决策分析被称为MULTIDEX-EMD研究。
19名利益相关者（7名医生、4名药剂师、5名卫生当局和卫生管理专家、1名心理学家以及2名患者代表）参与了这个分三个阶段的项目。在A阶段，一个咨询委员会确定了所有可能影响DME治疗决策的标准。然后使用离散选择实验（DCE）对这些标准进行筛选（B阶段）。接下来，通过应用向后消除算法拟合多项logit模型（相关标准：p值<0.05）。最后，在一个审议过程中讨论结果（C阶段）。
最初确定了31条标准（A阶段），并分为5类：疗效/有效性、安全性、组织和经济影响、患者报告的结果以及其他治疗特征。离散选择实验结果（B阶段）表明，10条标准与50至65岁DME患者的决策过程相关：最佳矫正视力的平均变化（p值<0.001）、视力提高≥15个字母的患者百分比（p值<0.001）、每次给药的效果持续时间（p值=0.008）、视网膜脱离（p值<0.001）、眼内炎（p值=0.012）、心肌梗死（p值<0.001）、玻璃体内出血（p值=0.021）、每位患者的年度治疗费用（p值=0.001）、健康相关生活质量（HRQoL）（p值=0.004）以及残疾程度（p值=0.021）。
从多利益相关者的角度来看，为DME患者选择合适的治疗方法应确保患者安全，并最大限度地提高视力改善和治疗效果持续时间。它还应通过价格可承受来促进系统可持续性，对健康相关生活质量产生积极影响，并预防残疾。
