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; Sydes, 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

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., Campbell, M. K., Quint, J. K., Robling, M., Sydes, 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), Article 94. 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: 2024
• Deposit Date: Apr 9, 2024
• Publicly Available Date: Apr 10, 2024
• Journal: Trials
• Electronic ISSN: 1745-6215
• Publisher: Springer Verlag
• Peer Reviewed: Peer Reviewed
• Volume: 25
• Issue: 1
• Article Number: 94
• 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://nottingham-repository.worktribe.com/output/30927415
• Publisher URL: https://trialsjournal.biomedcentral.com/articles/10.1186/s13063-024-07926-z
• Additional Information: Received: 13 September 2023; Accepted: 12 January 2024; First Online: 29 January 2024; : ; : Not applicable.; : Not applicable.; : 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.