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

Toader, Alice-Maria; Campbell, Marion K.; Quint, Jennifer K.; Robling, Michael; ydes, Matthew R.; Thorn, Joanna; Wright-Hughes, Alexandra; Yu, Ly-Mee; Abbott, Tom E. F.; Bond, Simon; Caskey, Fergus J.; Clout, Madeleine; Collinson, Michelle; Copsey, Bethan; Davies, Gwyneth; Driscoll, Timothy; Gamble, Carrol; Griffin, Xavier L.; Hamborg, Thomas; Harris, Jessica; Harrison, David A.; Harji, Deena; Henderson, Emily J.; Logan, Pip; Love, Sharon B.; Magee, Laura A.; O’Brien, Alastair; Pufulete, Maria; Ramnarayan, Padmanabhan; Saratzis, Athanasios; Smith, Jo; Solis-Trapala, Ivonne; Stubbs, Clive; Farrin, Amanda; Williamson, Paula

doi:10.1186/s13063-024-07926-z

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

Toader, Alice-Maria; Campbell, Marion K.; Quint, Jennifer K.; Robling, Michael; ydes, Matthew R.; Thorn, Joanna; Wright-Hughes, Alexandra; Yu, Ly-Mee; Abbott, Tom E. F.; Bond, Simon; Caskey, Fergus J.; Clout, Madeleine; Collinson, Michelle; Copsey, Bethan; Davies, Gwyneth; Driscoll, Timothy; Gamble, Carrol; Griffin, Xavier L.; Hamborg, Thomas; Harris, Jessica; Harrison, David A.; Harji, Deena; Henderson, Emily J.; Logan, Pip; Love, Sharon B.; Magee, Laura A.; O’Brien, Alastair; Pufulete, Maria; Ramnarayan, Padmanabhan; Saratzis, Athanasios; Smith, Jo; Solis-Trapala, Ivonne; Stubbs, Clive; Farrin, Amanda; Williamson, Paula

Authors

Alice-Maria Toader

Marion K. Campbell

Jennifer K. Quint

Michael Robling

Matthew R. ydes

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 i.solis-trapala@keele.ac.uk

Clive Stubbs

Amanda Farrin

Paula Williamson

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.

Citation

Toader, A.-M., Campbell, M. K., Quint, J. K., Robling, M., ydes, M. R., Thorn, J., …Williamson, P. (2024). Using healthcare systems data for outcomes in clinical trials: issues to consider at the design stage. Trials, 25(1), https://doi.org/10.1186/s13063-024-07926-z

Journal Article Type	Article
Acceptance Date	Jan 12, 2024
Online Publication Date	Jan 29, 2024
Publication Date	Jan 29, 2024
Deposit Date	Feb 5, 2024
Publicly Available Date	Feb 5, 2024
Journal	Trials
Publisher	Springer Verlag
Peer Reviewed	Peer Reviewed
Volume	25
Issue	1
DOI	https://doi.org/10.1186/s13063-024-07926-z
Keywords	Healthcare systems data, Outcomes, Data validity, Registries, Routinely collected data, Clinical trials
Public URL	https://keele-repository.worktribe.com/output/721704
Publisher URL	https://trialsjournal.biomedcentral.com/articles/10.1186/s13063-024-07926-z

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