Adaptive multi-interventional trial platform to improve patient care for fibrotic interstitial lung diseases.
Kawano-Dourado L., Kulkarni T., Ryerson CJ., Rivera-Ortega P., Baldi BG., Chaudhuri N., Funke-Chambour M., Hoffmann-Vold A-M., Johannson KA., Khor YH., Montesi SB., Piccari L., Prosch H., Molina-Molina M., Sellares Torres J., Bauer-Ventura I., Rajan S., Jacob J., Richards D., Spencer LG., Wendelberger B., Jensen T., Quintana M., Kreuter M., Gordon AC., Martinez FJ., Kaminski N., Cornelius V., Lewis R., Adams W., Jenkins G., REMAP-ILD consortium None.
BACKGROUND: 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). METHODS: 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. RESULTS: 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. CONCLUSION: 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.