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Improving the efficiency of clinical trials in multiple sclerosis
Ruth Ann Marrie1, Maria Pia Sormani2, Sean Apap Mangion3
1Departments of Internal Medicine and Community Health Sciences, Max Rady College of Medicine, Rady Faculty of Health Sciences, University of Manitoba, Winnipeg, MB, Canada.
Multiple Sclerosis (Houndmills, Basingstoke, England)
|August 9, 2023
Summary
Innovative clinical trial designs and intermediate outcomes can enhance efficiency for multiple sclerosis (MS) research, especially for progressive MS. These approaches aim to address broader questions and accelerate therapeutic development.
Area of Science:
- Clinical trial methodology
- Neuroimmunology
- Drug development
Background:
- Conventional Phase 3 trial designs for relapsing-remitting multiple sclerosis (RRMS) are successful but limited in scope, time, and cost.
- Trials for progressive multiple sclerosis (MS) have historically faced greater challenges and lower success rates.
- Existing designs restrict the types of research questions that can be effectively investigated.
Purpose of the Study:
- To explore complex innovative trial designs and intermediate outcomes to improve clinical trial efficiency in MS.
- To broaden the scope of questions addressable by clinical trials, with a specific focus on progressive MS.
- To enhance the development of effective therapies for all forms of MS.
Main Methods:
- An international workshop convened experts in clinical trial design.
- Discussions focused on innovative methodologies and outcome measures relevant to MS research.
- Expert consensus was sought on recommendations for future trial development.
Main Results:
- Recommendations include adopting complex innovative designs and utilizing biomarkers to enrich progressive MS trial populations.
- Prioritizing intermediate outcomes targeting non-inflammatory mechanisms and assessing data linkage acceptability are advised.
- Bayesian designs for pediatric trials and sustained funding for platform trials and registries are suggested.
Conclusions:
- Novel trial designs and advanced intermediate outcomes hold the potential to significantly improve the efficiency of MS clinical trials.
- These advancements can enable the investigation of new therapeutic questions, particularly for progressive MS.
- The findings offer a roadmap for more effective and comprehensive clinical research in multiple sclerosis.
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