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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.
Background:
Phase 3 clinical trials for disease-modifying therapies in relapsing-remitting multiple sclerosis (RRMS) have utilized a limited number of conventional designs with a high degree of success. However, these designs limit the types of questions that can be addressed, and the time and cost required. Moreover, trials involving people with progressive multiple sclerosis (MS) have been less successful.
Objective:
The objective of this paper is to discuss complex innovative trial designs, intermediate and composite outcomes and to improve the efficiency of trial design in MS and broaden questions that can be addressed, particularly as applied to progressive MS.
Methods:
We held an international workshop with experts in clinical trial design.
Results:
Recommendations include increasing the use of complex innovative designs, developing biomarkers to enrich progressive MS trial populations, prioritize intermediate outcomes for further development that target therapeutic mechanisms of action other than peripherally mediated inflammation, investigate acceptability to people with MS of data linkage for studying long-term outcomes of clinical trials, use Bayesian designs to potentially reduce sample sizes required for pediatric trials, and provide sustained funding for platform trials and registries that can support pragmatic trials.
Conclusion:
Novel trial designs and further development of intermediate outcomes may improve clinical trial efficiency in MS and address novel therapeutic questions.
Insights
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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