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Duong T, Krosschell KJ, James MK, et al. Consensus Guidelines for Improving Quality of Assessment and Training for Neuromuscular Diseases. Front Genet. 2021;12:735936doi: 10.3389/fgene.2021.735936.
Duong, T., Krosschell, K. J., James, M. K., Nelson, L., Alfano, L. N., Eichinger, K., Mazzone, E., Rose, K., Lowes, L. P., Mayhew, A., Florence, J., King, W., Senesac, C. R., & Eagle, M. (2021). Consensus Guidelines for Improving Quality of Assessment and Training for Neuromuscular Diseases. Frontiers in genetics, 12735936. https://doi.org/10.3389/fgene.2021.735936
Duong, Tina, et al. "Consensus Guidelines for Improving Quality of Assessment and Training for Neuromuscular Diseases." Frontiers in genetics vol. 12 (2021): 735936. doi: https://doi.org/10.3389/fgene.2021.735936
Duong T, Krosschell KJ, James MK, Nelson L, Alfano LN, Eichinger K, Mazzone E, Rose K, Lowes LP, Mayhew A, Florence J, King W, Senesac CR, Eagle M. Consensus Guidelines for Improving Quality of Assessment and Training for Neuromuscular Diseases. Front Genet. 2021 Nov 10;12:735936. doi: 10.3389/fgene.2021.735936. eCollection 2021. PMID: 34858470; PMCID: PMC8631528.
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Front Genet. 2021 Nov 10;12:735936. doi: 10.3389/fgene.2021.735936. eCollection 2021.

Consensus Guidelines for Improving Quality of Assessment and Training for Neuromuscular Diseases.

Frontiers in genetics

Tina Duong, Kristin J Krosschell, Meredith K James, Leslie Nelson, Lindsay N Alfano, Katy Eichinger, Elena Mazzone, Kristy Rose, Linda P Lowes, Anna Mayhew, Julaine Florence, Wendy King, Claudia R Senesac, Michelle Eagle

Affiliations

  1. Department of Neurology, Stanford University, Palo Alto, CA, United States.
  2. Children's National Hospital, Washington, DC, United States.
  3. Department of Physical Therapy and Human Movement Sciences, Northwestern University Feinberg School of Medicine, Chicago, IL, United States.
  4. The John Walton Muscular Dystrophy Research Centre, Newcastle Hospitals NHS Foundation Trust, Newcastle University, Newcastle upon Tyne, United Kingdom.
  5. Department of Physical Therapy, University of Texas Southwestern Medical Center, Dallas, TX, United States.
  6. Department of Pediatrics, The Ohio State University, Columbus, OH, United States.
  7. Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, United States.
  8. Department of Neurology, University of Rochester Medical Center, Rochester, NY, United States.
  9. Department of Child Neurology, Catholic University Policlinico Gemelli, Rome, Italy.
  10. Discipline of Physiotherapy, Faculty of Medicine and Health, The Sydney Children's Hospital Network, University of Sydney, Sydney, NSW, Australia.
  11. Department of Neurology, Washington University School of Medicine, St. Louis, MO, United States.
  12. Department of Neurology, The Ohio State University, Columbus, OH, United States.
  13. Department of Physical Therapy, University of Florida, Gainesville, FL, United States.
  14. ATOM International Ltd., Newcastle upon Tyne, United Kingdom.

PMID: 34858470 PMCID: PMC8631528 DOI: 10.3389/fgene.2021.735936

Abstract

Critical components of successful evaluation of clinical outcome assessments (COAs) in multisite clinical trials and clinical practice are standardized training, administration, and documented reliability of scoring. Experiences of evaluators, alongside patient differences from regional standards of care, may contribute to heterogeneity in clinical center's expertise. Achieving low variability and high reliability of COA is fundamental to clinical research and to give confidence in our ability to draw rational, interpretable conclusions from the data collected. The objective of this manuscript is to provide a framework to guide the learning process for COAs for use in clinics and clinical trials to maximize reliability and validity of COAs in neuromuscular disease (NMD). This is a consensus-based guideline with contributions from fourteen leading experts in clinical outcomes and the field of clinical outcome training in NMD. This framework should guide reliable and valid assessments in NMD specialty clinics and clinical trials. This consensus aims to expedite study start up with a progressive training pathway ranging from research naïve to highly experienced clinical evaluators. This document includes recommendations for education guidelines and roles and responsibilities of key stakeholders in COA assessment and implementation to ensure quality and consistency of outcome administration across different settings.

Copyright © 2021 Duong, Krosschell, James, Nelson, Alfano, Eichinger, Mazzone, Rose, Lowes, Mayhew, Florence, King, Senesac and Eagle.

Keywords: clinical evaluation education; clinical outcomes assessment; clinical trial readiness; evaluator training; neuromuscular disease (NMD)

Conflict of interest statement

TD has served on medical advisory boards and/or consultant for Scholar Rock, Genentech, F. Hoffman La Roche, Biogen, Sarepta, Novartis, Solid Biosciences, Dynacure, Dyne, and Audentes Consultancy also

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