Trials (Jan 2024)
Using healthcare systems data for outcomes in clinical trials: issues to consider at the design stage
- Alice-Maria Toader,
- Marion K. Campbell,
- Jennifer K. Quint,
- Michael Robling,
- Matthew R Sydes,
- Joanna Thorn,
- Alexandra Wright-Hughes,
- Ly-Mee Yu,
- Tom. E. F. Abbott,
- Simon Bond,
- Fergus J. Caskey,
- Madeleine Clout,
- Michelle Collinson,
- Bethan Copsey,
- Gwyneth Davies,
- Timothy Driscoll,
- Carrol Gamble,
- Xavier L. Griffin,
- Thomas Hamborg,
- Jessica Harris,
- David A. Harrison,
- Deena Harji,
- Emily J. Henderson,
- Pip Logan,
- Sharon B. Love,
- Laura A. Magee,
- Alastair O’Brien,
- Maria Pufulete,
- Padmanabhan Ramnarayan,
- Athanasios Saratzis,
- Jo Smith,
- Ivonne Solis-Trapala,
- Clive Stubbs,
- Amanda Farrin,
- Paula Williamson
Affiliations
- Alice-Maria Toader
- MRC-NIHR Trials Methodology Research Partnership, Department of Health Data Science, University of Liverpool
- Marion K. Campbell
- Health Services Research Unit, University of Aberdeen
- Jennifer K. Quint
- School of Public Health &, National Heart and Lung Institute, Imperial College London
- Michael Robling
- Centre for Trials Research, Cardiff University
- Matthew R Sydes
- MRC Clinical Trials Unit at UCL, Institute of Clinical Trials and Methodology, UCL
- Joanna Thorn
- Health Economics Bristol, Population Health Sciences, University of Bristol
- Alexandra Wright-Hughes
- Clinical Trials Research Unit, Leeds Institute of Clinical Trials Research,, University of Leeds
- Ly-Mee Yu
- Oxford Primary Care CTU, Nuffield Department of Primary Care Health Sciences, University of Oxford
- Tom. E. F. Abbott
- William Harvey Research Institute, Queen Mary University of London
- Simon Bond
- Cambridge Clinical Trials Unit
- Fergus J. Caskey
- BHF Data Science Centre, Health Data Research UK
- Madeleine Clout
- Bristol Trials Centre, Population Health Sciences, Bristol Medical School, University of Bristol
- Michelle Collinson
- Clinical Trials Research Unit, Leeds Institute of Clinical Trials Research,, University of Leeds
- Bethan Copsey
- Clinical Trials Research Unit, Leeds Institute of Clinical Trials Research,, University of Leeds
- Gwyneth Davies
- UCL Great Ormond Street Institute of Child Health
- Timothy Driscoll
- Swansea Trials Unit, Swansea University
- Carrol Gamble
- Liverpool Clinical Trials Centre, University of Liverpool
- Xavier L. Griffin
- Barts Bone and Joint Health, Blizard Institute, Barts and The London School of Medicine and Dentistry, Queen Mary University of London
- Thomas Hamborg
- Pragmatic Clinical Trials Unit, Wolfson Institute of Population Health, Queen Mary University of London
- Jessica Harris
- Bristol Trials Centre, Population Health Sciences, Bristol Medical School, University of Bristol
- David A. Harrison
- Intensive Care National Audit & Research Centre
- Deena Harji
- Clinical Trials Research Unit, Leeds Institute of Clinical Trials Research,, University of Leeds
- Emily J. Henderson
- Ageing and Movement Research Group, Bristol Medical School, University of Bristol
- Pip Logan
- School of Medicine, University of Nottingham and Nottingham City Care Partnership
- Sharon B. Love
- MRC Clinical Trials Unit at UCL, Institute of Clinical Trials and Methodology, UCL
- Laura A. Magee
- Department of Women and Children’s Health, King’s College London
- Alastair O’Brien
- Division of Medicine, UCL Institute for Liver and Digestive Health, Royal Free Campus
- Maria Pufulete
- Bristol Medical School, University of Bristol
- Padmanabhan Ramnarayan
- Department of Surgery and Cancer, Imperial College London
- Athanasios Saratzis
- Department of Cardiovascular Sciences, University of Leicester
- Jo Smith
- Keele Clinical Trials Unit, Faculty of Medicine and Health Sciences, Keele University
- Ivonne Solis-Trapala
- Keele Clinical Trials Unit, Faculty of Medicine and Health Sciences, Keele University
- Clive Stubbs
- Birmingham Clinical Trials Unit (BCTU), Institute of Applied Health Research College of Medical and Dental Sciences, The University of Birmingham
- Amanda Farrin
- Clinical Trials Research Unit, Leeds Institute of Clinical Trials Research,, University of Leeds
- Paula Williamson
- MRC-NIHR Trials Methodology Research Partnership, Department of Health Data Science, University of Liverpool
- DOI
- https://doi.org/10.1186/s13063-024-07926-z
- Journal volume & issue
-
Vol. 25,
no. 1
pp. 1 – 9
Abstract
Abstract Background Healthcare system data (HSD) are increasingly used in clinical trials, augmenting or replacing traditional methods of collecting outcome data. This study, PRIMORANT, set out to identify, in the UK context, issues to be considered before the decision to use HSD for outcome data in a clinical trial is finalised, a methodological question prioritised by the clinical trials community. Methods The PRIMORANT study had three phases. First, an initial workshop was held to scope the issues faced by trialists when considering whether to use HSDs for trial outcomes. Second, a consultation exercise was undertaken with clinical trials unit (CTU) staff, trialists, methodologists, clinicians, funding panels and data providers. Third, a final discussion workshop was held, at which the results of the consultation were fed back, case studies presented, and issues considered in small breakout groups. Results Key topics included in the consultation process were the validity of outcome data, timeliness of data capture, internal pilots, data-sharing, practical issues, and decision-making. A majority of consultation respondents (n = 78, 95%) considered the development of guidance for trialists to be feasible. Guidance was developed following the discussion workshop, for the five broad areas of terminology, feasibility, internal pilots, onward data sharing, and data archiving. Conclusions We provide guidance to inform decisions about whether or not to use HSDs for outcomes, and if so, to assist trialists in working with registries and other HSD providers to improve the design and delivery of trials.
Keywords
