Hcor Research Institute, Hcor Hospital, Sao Paulo, Brazil
Pulmonary Division, Heart Institute (InCor), University of Sao Paulo, Sao Paulo, Brazil.
Thorax. 2024 Jul 16;79(8):788-795. doi: 10.1136/thorax-2023-221148.
Fibrotic interstitial lung diseases (fILDs) are a heterogeneous group of lung diseases associated with significant morbidity and mortality. Despite a large increase in the number of clinical trials in the last 10 years, current regulatory-approved management approaches are limited to two therapies that prevent the progression of fibrosis. The drug development pipeline is long and there is an urgent need to accelerate this process. This manuscript introduces the concept and design of an innovative research approach to drug development in fILD: a global Randomised Embedded Multifactorial Adaptive Platform in fILD (REMAP-ILD).
Description of the REMAP-ILD concept and design: the specific terminology, design characteristics (multifactorial, adaptive features, statistical approach), target population, interventions, outcomes, mission and values, and organisational structure.
The target population will be adult patients with fILD, and the primary outcome will be a disease progression model incorporating forced vital capacity and mortality over 12 months. Responsive adaptive randomisation, prespecified thresholds for success and futility will be used to assess the effectiveness and safety of interventions. REMAP-ILD embraces the core values of diversity, equity, and inclusion for patients and researchers, and prioritises an open-science approach to data sharing and dissemination of results.
By using an innovative and efficient adaptive multi-interventional trial platform design, we aim to accelerate and improve care for patients with fILD. Through worldwide collaboration, novel analytical methodology and pragmatic trial delivery, REMAP-ILD aims to overcome major limitations associated with conventional randomised controlled trial approaches to rapidly improve the care of people living with fILD.
纤维化间质性肺疾病（fILD）是一组异质性肺病，与较高的发病率和死亡率相关。尽管在过去 10 年中，临床试验的数量大幅增加，但目前监管部门批准的治疗方法仅限于两种可预防纤维化进展的疗法。药物研发的过程漫长，迫切需要加快这一进程。本文介绍了一种纤维化间质性肺疾病药物研发的创新研究方法的概念和设计：纤维化间质性肺疾病全球随机嵌入式多因素适应性平台（REMAP-ILD）。
描述 REMAP-ILD 的概念和设计：具体术语、设计特点（多因素、适应性特征、统计方法）、目标人群、干预措施、结局、使命和价值观以及组织结构。
目标人群将是纤维化间质性肺疾病的成年患者，主要结局将是包含用力肺活量和 12 个月死亡率的疾病进展模型。将使用响应适应性随机化、预先设定的成功和无效阈值来评估干预措施的有效性和安全性。REMAP-ILD 秉持患者和研究人员多样性、公平性和包容性的核心价值观，并优先采用开放科学方法来共享数据和传播结果。
通过使用创新和高效的适应性多干预试验平台设计，我们旨在加速和改善纤维化间质性肺疾病患者的治疗。通过全球合作、新颖的分析方法和实用的试验实施，REMAP-ILD 旨在克服传统随机对照试验方法的主要局限性，快速改善纤维化间质性肺疾病患者的治疗。
