Improving the efficiency of clinical trials in multiple sclerosis.

Autor:	Marrie RA; Departments of Internal Medicine and Community Health Sciences, Max Rady College of Medicine, Rady Faculty of Health Sciences, University of Manitoba, Winnipeg, MB, Canada., Sormani MP; Department of Health Sciences, University of Genoa, Genoa, Italy/IRCCS Ospedale Policlinico San Martino, Genoa, Italy., Apap Mangion S; Queen Square Multiple Sclerosis Centre, Department of Neuroinflammation, UCL Queen Square Institute of Neurology, Faculty of Brain Sciences, University College London, London, UK., Bovis F; Department of Health Sciences, University of Genoa, Genoa, Italy., Cheung WY; Department of Oncology, Cumming School of Medicine, University of Calgary, Calgary, AB, Canada., Cutter GR; Department of Biostatistics, University of Alabama at Birmingham, Birmingham, AL, USA., Feys P; REVAL Rehabilitation Research Center, REVAL, Faculty of Rehabilitation Sciences, Hasselt University, Hasselt, Belgium/Universitair MS Centrum, UMSC, Hasselt, Belgium., Hill MD; Departments of Clinical Neurosciences, Community Health Sciences, Medicine, and Radiology, Hotchkiss Brain Institute, Cumming School of Medicine, University of Calgary, Calgary, AB, Canada., Koch MW; Departments of Clinical Neurosciences, Community Health Sciences, Hotchkiss Brain Institute, Cumming School of Medicine, University of Calgary, Calgary, AB, Canada., McCreary M; Department of Neurology, Section on Statistical Planning and Analysis, UT Southwestern Medical Center, Dallas, TX, USA., Mowry EM; Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, MD, USA., Park JJ; Department of Health Research Methods, Evidence and Impact, McMaster University, Hamilton, ON, Canada., Piehl F; Department of Clinical Neuroscience, Karolinska Institutet, Stockholm, Sweden., Salter A; Department of Neurology, Section on Statistical Planning and Analysis, UT Southwestern Medical Center, Dallas, TX, USA., Chataway J; Queen Square Multiple Sclerosis Centre, Department of Neuroinflammation, UCL Queen Square Institute of Neurology, Faculty of Brain Sciences, University College London, London, UK/National Institute for Health Research, University College London Hospitals, Biomedical Research Centre, London, UK/Medical Research Council Clinical Trials Unit at UCL, Institute of Clinical Trials and Methodology, University College London, London, UK.
Jazyk:	angličtina
Zdroj:	Multiple sclerosis (Houndmills, Basingstoke, England) [Mult Scler] 2023 Aug; Vol. 29 (9), pp. 1136-1148.
DOI:	10.1177/13524585231189671
Abstrakt:	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.
Databáze:	MEDLINE
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