Hospital Virgen de las Nieves, Granada, Spain.
Hospital Gregorio Marañón, Madrid, Spain.
Orphanet J Rare Dis. 2018 Dec 10;13(1):220. doi: 10.1186/s13023-018-0966-4.
Pulmonary Arterial Hypertension (PAH) is a chronic rare disease that can lead to serious cardiovascular problems and death. Additional treatments that increase effectiveness, that are safe and with a convenient administration that improve outcomes and quality of life for patients are needed. The aim of this study was to assess the value contribution of the new, oral prostacyclin receptor agonist, selexipag, for PAH treatment in Spain through reflective Multicriteria Decision Analysis (MCDA) methodology.
A comprehensive literature review was performed to develop an evidence matrix, composed of twelve quantitative criteria and four contextual criteria, based on an EVIDEM MCDA framework adapted to orphan drugs evaluation by the Spanish region of Catalonia. Quantitative performance scores, qualitative impact of contextual criteria and individual reflections from stakeholders were collected for each MCDA framework criteria. The value contribution of selexipag to PAH treatment compared to inhaled iloprost was calculated.
Oral selexipag for PAH treatment was considered as a treatment which adds value, compared to iloprost, in the following MCDA quantitative criteria: comparative efficacy, patient reported outcomes, preventive benefit, therapeutic benefit, other medical costs and other non-medical costs, without significant differences in safety profile but with a higher acquisition cost than inhaled iloprost.
Selexipag was considered to provide value to PAH treatment. It was perceived as an intervention indicated for a severe rare disease with high unmet needs, supported by high quality clinical evidence. When compared to inhaled iloprost, oral selexipag has demonstrated improvements in efficacy and patient reported outcomes, with a similar safety profile and some additional costs. Reflective MCDA provided a standardised, transparent approach to evaluate multiple criteria relating to the overall value contribution of selexipag to PAH treatment facilitating decision-making.
肺动脉高压（PAH）是一种慢性罕见病，可导致严重的心血管问题和死亡。需要额外的治疗方法，这些方法不仅要提高疗效，而且要安全，给药方便，从而改善患者的预后和生活质量。本研究旨在通过反射性多准则决策分析（MCDA）方法评估新型口服前列环素受体激动剂 selexipag 对西班牙 PAH 治疗的价值贡献。
全面审查文献以制定证据矩阵，该矩阵由 12 个定量标准和 4 个情境标准组成，其基础是根据 EVIDEM MCDA 框架，该框架由西班牙加泰罗尼亚地区适应孤儿药评估的方法改编。为每个 MCDA 框架标准收集了定量绩效评分、情境标准的定性影响以及利益相关者的个人反思。计算了 selexipag 与吸入伊洛前列素相比治疗 PAH 的价值贡献。
与吸入伊洛前列素相比，口服 selexipag 治疗 PAH 在以下 MCDA 定量标准中被认为是一种具有附加值的治疗方法：比较疗效、患者报告的结果、预防效益、治疗效益、其他医疗费用和其他非医疗费用，且安全性方面无显著差异，但与吸入伊洛前列素相比，其购置成本更高。
selexipag 被认为对 PAH 治疗具有价值。它被认为是一种针对高未满足需求的严重罕见病的干预措施，具有高质量的临床证据支持。与吸入伊洛前列素相比，口服 selexipag 在疗效和患者报告的结果方面均有改善，安全性相似，且存在一些额外的成本。反射性 MCDA 为评估与 selexipag 对 PAH 治疗的总体价值贡献相关的多个标准提供了一种标准化、透明的方法，有助于决策。
