The feasibility of using real world data as external control arms in oncology trials

Lishi Lin; Merel J.J. Lucassen; Vincent van der Noort; Toine C.G. Egberts; Jos H. Beijnen; Alwin D.R. Huitema

doi:10.1016/j.drudis.2025.104324

The feasibility of using real world data as external control arms in oncology trials

Lishi Lin, Merel J.J. Lucassen, Vincent van der Noort, Toine C.G. Egberts, Jos H. Beijnen, Alwin D.R. Huitema^*

^*Corresponding author for this work

Research output: Contribution to journal › Short survey › peer-review

Abstract

Before real world data (RWD)-derived external control arms (ECAs) can be applied in clinical trials within the field of oncology, it must be determined whether these ECAs can function as appropriate and reliable control arms. This review provides an overview of studies in which RWD-derived ECAs were constructed and compared to a randomized clinical trial (RCT) arm. RWD-derived ECAs had similar survival outcomes as the RCT arm of comparison in six of the eight included studies. Therefore, when performing RCTs is not feasible, RWD-derived ECAs can act as control arms in single-arm clinical trials, provided that suitable RWD sources are used and suitable methodological decisions are made.

Original language	English
Article number	104324
Journal	Drug Discovery Today
Volume	30
Issue number	3
DOIs	https://doi.org/10.1016/j.drudis.2025.104324
Publication status	Published - Mar 2025

Keywords

external control arm
oncology
real world data

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1-s2.0-S1359644625000376-mainFinal published version, 212 KBLicence: CC BY

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title = "The feasibility of using real world data as external control arms in oncology trials",

abstract = "Before real world data (RWD)-derived external control arms (ECAs) can be applied in clinical trials within the field of oncology, it must be determined whether these ECAs can function as appropriate and reliable control arms. This review provides an overview of studies in which RWD-derived ECAs were constructed and compared to a randomized clinical trial (RCT) arm. RWD-derived ECAs had similar survival outcomes as the RCT arm of comparison in six of the eight included studies. Therefore, when performing RCTs is not feasible, RWD-derived ECAs can act as control arms in single-arm clinical trials, provided that suitable RWD sources are used and suitable methodological decisions are made.",

keywords = "external control arm, oncology, real world data",

author = "Lishi Lin and Lucassen, {Merel J.J.} and {van der Noort}, Vincent and Egberts, {Toine C.G.} and Beijnen, {Jos H.} and Huitema, {Alwin D.R.}",

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year = "2025",

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doi = "10.1016/j.drudis.2025.104324",

language = "English",

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journal = "Drug Discovery Today",

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T1 - The feasibility of using real world data as external control arms in oncology trials

AU - Lin, Lishi

AU - Lucassen, Merel J.J.

AU - van der Noort, Vincent

AU - Egberts, Toine C.G.

AU - Beijnen, Jos H.

AU - Huitema, Alwin D.R.

PY - 2025/3

Y1 - 2025/3

N2 - Before real world data (RWD)-derived external control arms (ECAs) can be applied in clinical trials within the field of oncology, it must be determined whether these ECAs can function as appropriate and reliable control arms. This review provides an overview of studies in which RWD-derived ECAs were constructed and compared to a randomized clinical trial (RCT) arm. RWD-derived ECAs had similar survival outcomes as the RCT arm of comparison in six of the eight included studies. Therefore, when performing RCTs is not feasible, RWD-derived ECAs can act as control arms in single-arm clinical trials, provided that suitable RWD sources are used and suitable methodological decisions are made.

AB - Before real world data (RWD)-derived external control arms (ECAs) can be applied in clinical trials within the field of oncology, it must be determined whether these ECAs can function as appropriate and reliable control arms. This review provides an overview of studies in which RWD-derived ECAs were constructed and compared to a randomized clinical trial (RCT) arm. RWD-derived ECAs had similar survival outcomes as the RCT arm of comparison in six of the eight included studies. Therefore, when performing RCTs is not feasible, RWD-derived ECAs can act as control arms in single-arm clinical trials, provided that suitable RWD sources are used and suitable methodological decisions are made.

KW - external control arm

KW - oncology

KW - real world data

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U2 - 10.1016/j.drudis.2025.104324

DO - 10.1016/j.drudis.2025.104324

M3 - Short survey

AN - SCOPUS:86000634326

SN - 1359-6446

VL - 30

JO - Drug Discovery Today

JF - Drug Discovery Today

IS - 3

M1 - 104324

ER -

The feasibility of using real world data as external control arms in oncology trials

Abstract

Keywords

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