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

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2024年1月29日

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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

摘要

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.