. 2024 Jan 29;25(1):94.

doi: 10.1186/s13063-024-07926-z.

Using healthcare systems data for outcomes in clinical trials: issues to consider at the design stage

Alice-Maria Toader^#¹, Marion K Campbell², Jennifer K Quint³, Michael Robling⁴, Matthew R Sydes^{5

6}, Joanna Thorn⁷, Alexandra Wright-Hughes⁸, Ly-Mee Yu⁹, Tom E F Abbott¹⁰, Simon Bond¹¹, Fergus J Caskey⁶, Madeleine Clout¹², Michelle Collinson⁸, Bethan Copsey⁸, Gwyneth Davies¹³, Timothy Driscoll¹⁴, Carrol Gamble¹⁵, Xavier L Griffin¹⁶, Thomas Hamborg¹⁷, Jessica Harris¹², David A Harrison¹⁸, Deena Harji^{8

19}, Emily J Henderson^{20

21}, Pip Logan²², Sharon B Love⁵, Laura A Magee²³, Alastair O'Brien²⁴, Maria Pufulete²⁵, Padmanabhan Ramnarayan²⁶, Athanasios Saratzis²⁷, Jo Smith²⁸, Ivonne Solis-Trapala²⁸, Clive Stubbs²⁹, Amanda Farrin^#⁸, Paula Williamson^#³⁰

Affiliations

¹ MRC-NIHR Trials Methodology Research Partnership, Department of Health Data Science, University of Liverpool, Liverpool, UK. Sgatoade@liverpool.ac.uk.
² Health Services Research Unit, University of Aberdeen, Aberdeen, AB25 2ZD, UK.
³ School of Public Health &, National Heart and Lung Institute, Imperial College London, London, UK.
⁴ Centre for Trials Research, Cardiff University, Cardiff, CF14 4YS, UK.
⁵ MRC Clinical Trials Unit at UCL, Institute of Clinical Trials and Methodology, UCL, London, UK.
⁶ BHF Data Science Centre, Health Data Research UK, London, UK.
⁷ Health Economics Bristol, Population Health Sciences, University of Bristol, Bristol, UK.
⁸ Clinical Trials Research Unit, Leeds Institute of Clinical Trials Research,, University of Leeds, Leeds, LS2 9JT, UK.
⁹ Oxford Primary Care CTU, Nuffield Department of Primary Care Health Sciences, University of Oxford, Oxford, UK.
¹⁰ William Harvey Research Institute, Queen Mary University of London, London, EC1M 6BQ, UK.
¹¹ Cambridge Clinical Trials Unit, Cambridge, UK.
¹² Bristol Trials Centre, Population Health Sciences, Bristol Medical School, University of Bristol, Bristol, BS8 2PS, UK.
¹³ UCL Great Ormond Street Institute of Child Health, London, WC1N 1EH, UK.
¹⁴ Swansea Trials Unit, Swansea University, Swansea, SA2 8PP, UK.
¹⁵ Liverpool Clinical Trials Centre, University of Liverpool, Liverpool, UK.
¹⁶ Barts Bone and Joint Health, Blizard Institute, Barts and The London School of Medicine and Dentistry, Queen Mary University of London, London, UK.
¹⁷ Pragmatic Clinical Trials Unit, Wolfson Institute of Population Health, Queen Mary University of London, London, E1 2AB, UK.
¹⁸ Intensive Care National Audit & Research Centre, London, UK.
¹⁹ Manchester University NHS Foundation Trust, Manchester, UK.
²⁰ Ageing and Movement Research Group, Bristol Medical School, University of Bristol, Bristol, UK.
²¹ Older People's Unit, Royal United Hospitals NHS Foundation Trust, Bath, UK.
²² School of Medicine, University of Nottingham and Nottingham City Care Partnership, Nottingham, UK.
²³ Department of Women and Children's Health, King's College London, London, UK.
²⁴ Division of Medicine, UCL Institute for Liver and Digestive Health, Royal Free Campus, Upper 3Rd FloorRowland Hill Street, London, NW3 2PF, UK.
²⁵ Bristol Medical School, University of Bristol, Bristol, BS8 2PS, UK.
²⁶ Department of Surgery and Cancer, Imperial College London, London, W21PB, UK.
²⁷ Department of Cardiovascular Sciences, University of Leicester, Leicester, UK.
²⁸ Keele Clinical Trials Unit, Faculty of Medicine and Health Sciences, Keele University, Staffordshire, UK.
²⁹ Birmingham Clinical Trials Unit (BCTU), Institute of Applied Health Research College of Medical and Dental Sciences, The University of Birmingham, Edgbaston, Birmingham, B15 2TT, UK.
³⁰ MRC-NIHR Trials Methodology Research Partnership, Department of Health Data Science, University of Liverpool, Liverpool, UK.

^# Contributed equally.

PMID: 38287428
PMCID: PMC10823676
DOI: 10.1186/s13063-024-07926-z

Using healthcare systems data for outcomes in clinical trials: issues to consider at the design stage

Alice-Maria Toader et al. Trials. 2024.

. 2024 Jan 29;25(1):94.

doi: 10.1186/s13063-024-07926-z.

Authors

Affiliations

¹ MRC-NIHR Trials Methodology Research Partnership, Department of Health Data Science, University of Liverpool, Liverpool, UK. Sgatoade@liverpool.ac.uk.
² Health Services Research Unit, University of Aberdeen, Aberdeen, AB25 2ZD, UK.
³ School of Public Health &, National Heart and Lung Institute, Imperial College London, London, UK.
⁴ Centre for Trials Research, Cardiff University, Cardiff, CF14 4YS, UK.
⁵ MRC Clinical Trials Unit at UCL, Institute of Clinical Trials and Methodology, UCL, London, UK.
⁶ BHF Data Science Centre, Health Data Research UK, London, UK.
⁷ Health Economics Bristol, Population Health Sciences, University of Bristol, Bristol, UK.
⁸ Clinical Trials Research Unit, Leeds Institute of Clinical Trials Research,, University of Leeds, Leeds, LS2 9JT, UK.
⁹ Oxford Primary Care CTU, Nuffield Department of Primary Care Health Sciences, University of Oxford, Oxford, UK.
¹⁰ William Harvey Research Institute, Queen Mary University of London, London, EC1M 6BQ, UK.
¹¹ Cambridge Clinical Trials Unit, Cambridge, UK.
¹² Bristol Trials Centre, Population Health Sciences, Bristol Medical School, University of Bristol, Bristol, BS8 2PS, UK.
¹³ UCL Great Ormond Street Institute of Child Health, London, WC1N 1EH, UK.
¹⁴ Swansea Trials Unit, Swansea University, Swansea, SA2 8PP, UK.
¹⁵ Liverpool Clinical Trials Centre, University of Liverpool, Liverpool, UK.
¹⁶ Barts Bone and Joint Health, Blizard Institute, Barts and The London School of Medicine and Dentistry, Queen Mary University of London, London, UK.
¹⁷ Pragmatic Clinical Trials Unit, Wolfson Institute of Population Health, Queen Mary University of London, London, E1 2AB, UK.
¹⁸ Intensive Care National Audit & Research Centre, London, UK.
¹⁹ Manchester University NHS Foundation Trust, Manchester, UK.
²⁰ Ageing and Movement Research Group, Bristol Medical School, University of Bristol, Bristol, UK.
²¹ Older People's Unit, Royal United Hospitals NHS Foundation Trust, Bath, UK.
²² School of Medicine, University of Nottingham and Nottingham City Care Partnership, Nottingham, UK.
²³ Department of Women and Children's Health, King's College London, London, UK.
²⁴ Division of Medicine, UCL Institute for Liver and Digestive Health, Royal Free Campus, Upper 3Rd FloorRowland Hill Street, London, NW3 2PF, UK.
²⁵ Bristol Medical School, University of Bristol, Bristol, BS8 2PS, UK.
²⁶ Department of Surgery and Cancer, Imperial College London, London, W21PB, UK.
²⁷ Department of Cardiovascular Sciences, University of Leicester, Leicester, UK.
²⁸ Keele Clinical Trials Unit, Faculty of Medicine and Health Sciences, Keele University, Staffordshire, UK.
²⁹ Birmingham Clinical Trials Unit (BCTU), Institute of Applied Health Research College of Medical and Dental Sciences, The University of Birmingham, Edgbaston, Birmingham, B15 2TT, UK.
³⁰ MRC-NIHR Trials Methodology Research Partnership, Department of Health Data Science, University of Liverpool, Liverpool, UK.

^# Contributed equally.

PMID: 38287428
PMCID: PMC10823676
DOI: 10.1186/s13063-024-07926-z

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: Clinical trials; Data validity; Healthcare systems data; Outcomes; Registries; Routinely collected data.

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Conflict of interest statement

GD reports speaker honoraria from Chiesi Ltd and Vertex Pharmaceuticals outside the submitted work.

PR reports consultancy fees from Vyaire Medical and Sanofi outside the submitted work.

EJH reports honoraria and travel support from Kyowa Kirin; Abbvie; Ever; Bial, The Neurology Academy and CME Institute outside the submitted work.

JKQ has received grants from MRC, HDR UK, GSK, BI, asthma + lung UK, and AZ and personal fees for advisory board participation, consultancy or speaking fees from GlaxoSmithKline, Evidera, Chiesi, AstraZeneca, Insmed.

MKC was a member of the CONSORT-ROUTINE group. The Health Services Research Unit, where MKC works, receives core funding from the Scottish Government Health Directorates.

SBL reports no conflicts of interest.

MRSy reports speaker fees at clinical trial statistics training meeting for clinicians (no discussion of particular drugs) from Lilly Oncology; Speaker fees at clinical trial statistics training meeting for clinicians (no discussion of particular drugs) from Janssen; and Educational video on clinical trial statistics (no discussion of particular drugs) from Eisai.

MClout reports no conflicts of interest.

MC reports no conflicts of interest.

JThorn reports no conflicts of interest.

MR reports no conflicts of interest.

JH reports no conflicts of interest.

TJD reports no conflicts of interest.

AJF reports no conflicts of interest.

None of the other authors reported any conflicts of interest.

Figures

See this image and copyright information in PMC

References

1. Sydes MR, Barbachano Y, Bowman L, Denwood T, Farmer A, Garfield-Birkbeck S, et al. Realising the full potential of data-enabled trials in the UK: a call for action. BMJ Open. 2021;11(6):e043906. doi: 10.1136/bmjopen-2020-043906. - DOI - PMC - PubMed
1. Lensen S, Macnair A, Love SB, Yorke-Edwards V, Noor NM, Martyn M, et al. Access to routinely collected health data for clinical trials–review of successful data requests to UK registries. Trials. 2020;21(1):1–11. doi: 10.1186/s13063-020-04329-8. - DOI - PMC - PubMed
1. McKay AJ, Jones AP, Gamble CL, Farmer AJ, Williamson PR. Use of routinely collected data in a UK cohort of publicly funded randomised clinical trials. F1000Research. 2021;9:323. doi: 10.12688/f1000research.23316.3. - DOI - PMC - PubMed
1. Alice-Maria Toader, Carrol Gamble, Susanna Dodd, et al. The use of healthcare systems data for RCTs, 17 October 2023, PREPRINT (Version 1) available at Research Square [10.21203/rs.3.rs-3373403/v1]. - PMC - PubMed
1. Williams AD, Davies G, Farrin AJ, Mafham M, Robling M, Sydes MR, et al. A DELPHI study priority setting the remaining challenges for the use of routinely collected data in trials: COMORANT-UK. Trials. 2023;24(1):1–8. doi: 10.1186/s13063-023-07251-x. - DOI - PMC - PubMed

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Using healthcare systems data for outcomes in clinical trials: issues to consider at the design stage

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Using healthcare systems data for outcomes in clinical trials: issues to consider at the design stage

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Conflict of interest statement

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