The impact of operational trial approaches on representativeness: Comparison of decentralized clinical trial participants, conventional trial participants, and patients in daily practice

Trials@Home Consortium; Amos J de Jong; Mira G P Zuidgeest; Yared Santa-Ana-Tellez; Christine E Hallgreen; Thomas T van Sloten; Anthonius de Boer; Helga Gardarsdottir

doi:10.1016/j.drudis.2025.104304

The impact of operational trial approaches on representativeness: Comparison of decentralized clinical trial participants, conventional trial participants, and patients in daily practice

Trials@Home Consortium, Amos J de Jong, Mira G P Zuidgeest, Yared Santa-Ana-Tellez, Christine E Hallgreen, Thomas T van Sloten, Anthonius de Boer, Helga Gardarsdottir

Research output: Contribution to journal › Review article › peer-review

Abstract

Decentralized clinical trial (DCT) approaches - in which trial activities are conducted at participants' homes - have the potential to improve representativeness. We present a study that compared the demographics and cardiovascular risk factors of participants from a DCT (ASCEND) and a conventional trial with a similar trial objective (POPADAD) to those of patients in daily practice. We adjudicate that there are relevant differences when comparing the participants of the conventional trial and the DCT, with the latter providing better representativeness in terms of age, insulin use, smoking status, and body mass index, whereas conventional trial participants were more representative in terms of biological sex. Differences in these characteristics were not explained by the eligibility criteria, but are considered attributable to the operational trial approach.

Original language	English
Article number	104304
Number of pages	11
Journal	Drug Discovery Today
Volume	30
Issue number	2
Early online date	28 Jan 2025
DOIs	https://doi.org/10.1016/j.drudis.2025.104304
Publication status	Published - Feb 2025

Bibliographical note

Publisher Copyright:
© 2025 The Author(s)

Funding

The Trials@Home project has received funding from the Innovative Medicines Initiative 2 Joint Undertaking under grant agreement No 831458. This Joint Undertaking received support from the European Union's Horizon 2020 research and innovation programme and EFPIA. A grant from the Van Leersum Grant/KNAW Medical Sciences Fund, Royal Netherlands Academy of Arts & Sciences, facilitated a research visit to the Copenhagen Centre for Regulatory Science. The Trials@Home project has received funding from the Innovative Medicines Initiative 2 Joint Undertaking under grant agreement No 831458. This Joint Undertaking received support from the European Union\u2019s Horizon 2020 research and innovation programme and EFPIA. A grant from the Van Leersum Grant/KNAW Medical Sciences Fund, Royal Netherlands Academy of Arts & Sciences, facilitated a research visit to the Copenhagen Centre for Regulatory Science.

Funders	Funder number
European Federation of Pharmaceutical Industries and Associations
Horizon 2020 Framework Programme
European Commission
Koninklijke Nederlandse Akademie van Wetenschappen
Innovative Medicines Initiative	831458

Keywords

clinical trial operations
decentralized clinical trials
generalizability
participant representativeness

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1-s2.0-S1359644625000170-mainFinal published version, 1 MBLicence: CC BY

Cite this

Trials@Home Consortium, de Jong, A. J., Zuidgeest, M. G. P., Santa-Ana-Tellez, Y., Hallgreen, C. E., van Sloten, T. T., de Boer, A., & Gardarsdottir, H. (2025). The impact of operational trial approaches on representativeness: Comparison of decentralized clinical trial participants, conventional trial participants, and patients in daily practice. Drug Discovery Today, 30(2), Article 104304. https://doi.org/10.1016/j.drudis.2025.104304

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abstract = "Decentralized clinical trial (DCT) approaches - in which trial activities are conducted at participants' homes - have the potential to improve representativeness. We present a study that compared the demographics and cardiovascular risk factors of participants from a DCT (ASCEND) and a conventional trial with a similar trial objective (POPADAD) to those of patients in daily practice. We adjudicate that there are relevant differences when comparing the participants of the conventional trial and the DCT, with the latter providing better representativeness in terms of age, insulin use, smoking status, and body mass index, whereas conventional trial participants were more representative in terms of biological sex. Differences in these characteristics were not explained by the eligibility criteria, but are considered attributable to the operational trial approach.",

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Trials@Home Consortium, de Jong, AJ, Zuidgeest, MGP, Santa-Ana-Tellez, Y, Hallgreen, CE, van Sloten, TT, de Boer, A & Gardarsdottir, H 2025, 'The impact of operational trial approaches on representativeness: Comparison of decentralized clinical trial participants, conventional trial participants, and patients in daily practice', Drug Discovery Today, vol. 30, no. 2, 104304. https://doi.org/10.1016/j.drudis.2025.104304

The impact of operational trial approaches on representativeness: Comparison of decentralized clinical trial participants, conventional trial participants, and patients in daily practice. / Trials@Home Consortium; de Jong, Amos J; Zuidgeest, Mira G P et al.
In: Drug Discovery Today, Vol. 30, No. 2, 104304, 02.2025.

Research output: Contribution to journal › Review article › peer-review

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AU - Trials@Home Consortium

AU - de Jong, Amos J

AU - Zuidgeest, Mira G P

AU - Santa-Ana-Tellez, Yared

AU - Hallgreen, Christine E

AU - van Sloten, Thomas T

AU - de Boer, Anthonius

AU - Gardarsdottir, Helga

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Trials@Home Consortium, de Jong AJ, Zuidgeest MGP, Santa-Ana-Tellez Y, Hallgreen CE, van Sloten TT et al. The impact of operational trial approaches on representativeness: Comparison of decentralized clinical trial participants, conventional trial participants, and patients in daily practice. Drug Discovery Today. 2025 Feb;30(2):104304. Epub 2025 Jan 28. doi: 10.1016/j.drudis.2025.104304

The impact of operational trial approaches on representativeness: Comparison of decentralized clinical trial participants, conventional trial participants, and patients in daily practice

Abstract

Bibliographical note

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Keywords

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