Adaptive multi-interventional trial platform to improve patient care for fibrotic interstitial lung diseases
Leticia Kawano-Dourado1,2,3, Tejaswini Kulkarni4, Christopher J Ryerson5
1Hcor Research Institute, Hcor Hospital, Sao Paulo, Brazil ldourado@hcor.com.br.
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.
Insights
This study introduces REMAP-ILD, a novel adaptive platform to accelerate drug development for fibrotic interstitial lung diseases (fILD). This approach aims to improve patient care by overcoming limitations of traditional clinical trials.
Area of Science:
- Pulmonology
- Clinical Trial Design
- Drug Development
Background:
- Fibrotic interstitial lung diseases (fILDs) present significant morbidity and mortality.
- Current treatments for fILDs are limited, with only two therapies approved to slow fibrosis progression.
- There is an urgent need to accelerate drug development for fILDs due to a lengthy pipeline.
Purpose of the Study:
- To introduce the concept and design of an innovative research approach for drug development in fILD.
- To describe the REMAP-ILD (Randomised Embedded Multifactorial Adaptive Platform in fILD) platform.
- To outline the potential of REMAP-ILD to expedite the delivery of effective therapies.
Main Methods:
- The REMAP-ILD platform employs a multifactorial, adaptive design.
- It incorporates responsive adaptive randomization and prespecified thresholds for success and futility.
- The platform targets adult patients with fILD and focuses on a disease progression model using forced vital capacity and mortality.
Main Results:
- The primary outcome measure is a disease progression model over 12 months.
- Effectiveness and safety of interventions will be assessed using adaptive randomization.
- REMAP-ILD prioritizes diversity, equity, inclusion, and an open-science approach to data sharing.
Conclusions:
- REMAP-ILD offers an innovative and efficient adaptive platform to accelerate care for fILD patients.
- Worldwide collaboration, novel analytical methods, and pragmatic trial delivery are key components.
- This approach aims to overcome limitations of conventional randomized controlled trials for fILD.
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