Evidence map›Paper›PMID 37555492›Full record

ReviewMultiple sclerosis (Houndmills, Basingstoke, England)2023

Improving the efficiency of clinical trials in multiple sclerosis.

Ruth Ann Marrie, Maria Pia Sormani, Sean Apap Mangion, Francesca Bovis, Winson Y Cheung, Gary R Cutter, Peter Feys, Michael D Hill, Marcus Werner Koch, Morgan McCreary and 5 more

Abstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in Multiple sclerosis (Houndmills, Basingstoke, England), 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
9citing papers in PubMed, 1 pooled it
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

9 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Article
  3. Article
  4. Article
  5. Article
  6. Article
  7. Article
  8. Enhancing diversity of clinical trial populations in multiple sclerosis.Multiple sclerosis (Houndmills, Basingstoke, England) · 2023
    Review
  9. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

15 authors.

Ruth Ann MarrieDepartments of Internal Medicine and Community Health Sciences, Max Rady College of Medicine, Rady Faculty of Health Sciences, University of Manitoba, Winnipeg, MB, Canada.ORCID 0000-0002-1855-5595
Maria Pia SormaniDepartment of Health Sciences, University of Genoa, Genoa, Italy/IRCCS Ospedale Policlinico San Martino, Genoa, Italy.ORCID 0000-0001-6892-104X
Sean Apap MangionQueen Square Multiple Sclerosis Centre, Department of Neuroinflammation, UCL Queen Square Institute of Neurology, Faculty of Brain Sciences, University College London, London, UK.
Francesca BovisDepartment of Health Sciences, University of Genoa, Genoa, Italy.
Winson Y CheungDepartment of Oncology, Cumming School of Medicine, University of Calgary, Calgary, AB, Canada.
Gary R CutterDepartment of Biostatistics, University of Alabama at Birmingham, Birmingham, AL, USA.ORCID 0000-0002-8455-980X
Peter FeysREVAL Rehabilitation Research Center, REVAL, Faculty of Rehabilitation Sciences, Hasselt University, Hasselt, Belgium/Universitair MS Centrum, UMSC, Hasselt, Belgium.
Michael D HillDepartments of Clinical Neurosciences, Community Health Sciences, Medicine, and Radiology, Hotchkiss Brain Institute, Cumming School of Medicine, University of Calgary, Calgary, AB, Canada.
Marcus Werner KochDepartments of Clinical Neurosciences, Community Health Sciences, Hotchkiss Brain Institute, Cumming School of Medicine, University of Calgary, Calgary, AB, Canada.ORCID 0000-0001-9972-5092
Morgan McCrearyDepartment of Neurology, Section on Statistical Planning and Analysis, UT Southwestern Medical Center, Dallas, TX, USA.ORCID 0000-0002-8378-460X
Ellen M MowryDepartment of Neurology, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
Jay Jh ParkDepartment of Health Research Methods, Evidence and Impact, McMaster University, Hamilton, ON, Canada.
Fredrik PiehlDepartment of Clinical Neuroscience, Karolinska Institutet, Stockholm, Sweden.ORCID 0000-0001-8329-5219
Amber SalterDepartment of Neurology, Section on Statistical Planning and Analysis, UT Southwestern Medical Center, Dallas, TX, USA.ORCID 0000-0002-1088-110X
Jeremy ChatawayQueen 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.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundPhase 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.

objectiveThe 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.

methodsWe held an international workshop with experts in clinical trial design.

resultsRecommendations 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.

conclusionNovel trial designs and further development of intermediate outcomes may improve clinical trial efficiency in MS and address novel therapeutic questions.

Indexed as

Multiple SclerosisMultiple Sclerosis, Chronic ProgressiveMultiple Sclerosis, Relapsing-RemittingAdaptive Clinical Trials as TopicBayes TheoremChildClinical Trials as TopicHumansSample Sizeadaptive trial designsBayesian statisticsclinical trialsfutility trialsMultiple sclerosisplatform trials

Identifiers

PMID37555492
PMCPMC10413792

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.