Digital endpoints in clinical trials: emerging themes from a multi-stakeholder Knowledge Exchange event.


Journal

Trials
ISSN: 1745-6215
Titre abrégé: Trials
Pays: England
ID NLM: 101263253

Informations de publication

Date de publication:
03 Aug 2024
Historique:
received: 29 04 2024
accepted: 24 07 2024
medline: 3 8 2024
pubmed: 3 8 2024
entrez: 2 8 2024
Statut: epublish

Résumé

Digital technologies, such as wearable devices and smartphone applications (apps), can enable the decentralisation of clinical trials by measuring endpoints in people's chosen locations rather than in traditional clinical settings. Digital endpoints can allow high-frequency and sensitive measurements of health outcomes compared to visit-based endpoints which provide an episodic snapshot of a person's health. However, there are underexplored challenges in this emerging space that require interdisciplinary and cross-sector collaboration. A multi-stakeholder Knowledge Exchange event was organised to facilitate conversations across silos within this research ecosystem. A survey was sent to an initial list of stakeholders to identify potential discussion topics. Additional stakeholders were identified through iterative discussions on perspectives that needed representation. Co-design meetings with attendees were held to discuss the scope, format and ethos of the event. The event itself featured a cross-disciplinary selection of talks, a panel discussion, small-group discussions facilitated via a rolling seating plan and audience participation via Slido. A transcript was generated from the day, which, together with the output from Slido, provided a record of the day's discussions. Finally, meetings were held following the event to identify the key challenges for digital endpoints which emerged and reflections and recommendations for dissemination. Several challenges for digital endpoints were identified in the following areas: patient adherence and acceptability; algorithms and software for devices; design, analysis and conduct of clinical trials with digital endpoints; the environmental impact of digital endpoints; and the need for ongoing ethical support. Learnings taken for next generation events include the need to include additional stakeholder perspectives, such as those of funders and regulators, and the need for additional resources and facilitation to allow patient and public contributors to engage meaningfully during the event. The event emphasised the importance of consortium building and highlighted the critical role that collaborative, multi-disciplinary, and cross-sector efforts play in driving innovation in research design and strategic partnership building moving forward. This necessitates enhanced recognition by funders to support multi-stakeholder projects with patient involvement, standardised terminology, and the utilisation of open-source software.

Sections du résumé

BACKGROUND BACKGROUND
Digital technologies, such as wearable devices and smartphone applications (apps), can enable the decentralisation of clinical trials by measuring endpoints in people's chosen locations rather than in traditional clinical settings. Digital endpoints can allow high-frequency and sensitive measurements of health outcomes compared to visit-based endpoints which provide an episodic snapshot of a person's health. However, there are underexplored challenges in this emerging space that require interdisciplinary and cross-sector collaboration. A multi-stakeholder Knowledge Exchange event was organised to facilitate conversations across silos within this research ecosystem.
METHODS METHODS
A survey was sent to an initial list of stakeholders to identify potential discussion topics. Additional stakeholders were identified through iterative discussions on perspectives that needed representation. Co-design meetings with attendees were held to discuss the scope, format and ethos of the event. The event itself featured a cross-disciplinary selection of talks, a panel discussion, small-group discussions facilitated via a rolling seating plan and audience participation via Slido. A transcript was generated from the day, which, together with the output from Slido, provided a record of the day's discussions. Finally, meetings were held following the event to identify the key challenges for digital endpoints which emerged and reflections and recommendations for dissemination.
RESULTS RESULTS
Several challenges for digital endpoints were identified in the following areas: patient adherence and acceptability; algorithms and software for devices; design, analysis and conduct of clinical trials with digital endpoints; the environmental impact of digital endpoints; and the need for ongoing ethical support. Learnings taken for next generation events include the need to include additional stakeholder perspectives, such as those of funders and regulators, and the need for additional resources and facilitation to allow patient and public contributors to engage meaningfully during the event.
CONCLUSIONS CONCLUSIONS
The event emphasised the importance of consortium building and highlighted the critical role that collaborative, multi-disciplinary, and cross-sector efforts play in driving innovation in research design and strategic partnership building moving forward. This necessitates enhanced recognition by funders to support multi-stakeholder projects with patient involvement, standardised terminology, and the utilisation of open-source software.

Identifiants

pubmed: 39095915
doi: 10.1186/s13063-024-08356-7
pii: 10.1186/s13063-024-08356-7
doi:

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

521

Subventions

Organisme : NIHR Cambridge Biomedical Research Centre
ID : NIHR203312
Organisme : Medical Research Council
ID : MC UU 00002/15
Pays : United Kingdom
Organisme : Medical Research Council
ID : MC UU 00002/15
Pays : United Kingdom
Organisme : Medical Research Council
ID : MC UU 00040/03
Pays : United Kingdom
Organisme : Medical Research Council
ID : MC UU 00004/07
Pays : United Kingdom
Organisme : Medical Research Council
ID : MC UU 00040/03
Pays : United Kingdom
Organisme : Horizon 2020 Framework Programme
ID : 101016775
Organisme : HORIZON EUROPE Framework Programme
ID : 821511
Organisme : Innovative Medicines Initiative 2 Joint Undertaking
ID : 806999
Organisme : MRC Experimental Medicine Award
ID : MR/W026279/1

Informations de copyright

© 2024. The Author(s).

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Auteurs

Mia S Tackney (MS)

MRC-Biostatistics Unit, University of Cambridge, Cambridge, UK. mst35@cam.ac.uk.

Amber Steele (A)

Strategic Funding Partnerships Hub (SFPH), Cambridge University Hospitals, Cambridge, UK.

Joseph Newman (J)

Department of Medicine, University of Cambridge and Royal Papworth Hospital, Cambridge, UK.

Marie-Christine Fritzsche (MC)

Institute of History and Ethics in Medicine, TUM School of Medicine and Health, Technical University of Munich, Munich, Germany.
School of Social Sciences and Technology, Technical University of Munich, Munich, Germany.

Federica Lucivero (F)

Ethox Centre, Nuffield Department of Population Health, University of Oxford, Oxford, UK.

Zarnie Khadjesari (Z)

School of Health Sciences, University of East Anglia, Norwich, England.

Jennifer Lynch (J)

University of Hertfordshire, Hatfield, UK.

Rosemary A Abbott (RA)

ICON PLC, Reading, UK.

Vicki S Barber (VS)

Oxford Clinical Trials Research Unit (OCTRU), University of Oxford, Oxford, UK.

James R Carpenter (JR)

MRC Clinical Trials Unit at University College London, London, UK.
Department of Medical Statistics, London School of Hygiene and Tropical Medicine, London, UK.

Bethan Copsey (B)

Leeds Clinical Trials Research Unit, University of Leeds, Leeds, UK.

Elin H Davies (EH)

Aparito, a wholly owned subsidiary company of Eli Lilly and Company, Wrexham, Wales, UK.

William G Dixon (WG)

Centre for Epidemiology Versus Arthritis, Manchester Academic Health Sciences Centre, University of Manchester, Manchester, UK.

Lisa Fox (L)

Clinical Trials and Statistics Unit (ICR-CTSU), The Institute of Cancer Research, London, UK.

Javier González (J)

Microsoft Research Cambridge, Cambridge, UK.

Jessica Griffiths (J)

Clinical Trials and Statistics Unit (ICR-CTSU), The Institute of Cancer Research, London, UK.

Chloe H L Hinchliffe (CHL)

Translational and Clinical Research Institute, Newcastle University, Newcastle, UK.

Magdalena A Kolanko (MA)

UK Dementia Research Institute Care Research and Technology Centre, London, UK.
Imperial College London, London, UK.

Dylan McGagh (D)

Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Sciences, University of Oxford, Oxford, UK.
Big Data Institute, Li Ka Shing Centre for Health Information and Discovery, University of Oxford, Oxford, UK.

Aryelly Rodriguez (A)

The University of Edinburgh, Edinburgh, UK.

George Roussos (G)

School of Computing and Mathematical Sciences, Birkbeck College, University of London, London, UK.

Karen B E So (KBE)

Alexion Rare Oncology, AstraZeneca, Cambridge, UK.

Louise Stanton (L)

Southampton Clinical Trials Unit, University of Southampton, Southampton, UK.

Mark Toshner (M)

Royal Papworth Hospital and Department of Medicine, Victor Phillip Dahdaleh Heart and Lung Research Institute, University of Cambridge, Cambridge, UK.

Frances Varian (F)

University of Sheffield, Sheffield, UK.

Paula R Williamson (PR)

Institute of Population Health, University of Liverpool, Liverpool, UK.

Belay B Yimer (BB)

Centre for Epidemiology, University of Manchester, Manchester, UK.

Sofía S Villar (SS)

MRC-Biostatistics Unit, University of Cambridge, Cambridge, UK.

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