TY - JOUR
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.
N1 - Copyright © 2025 The Authors. Published by Elsevier Ltd.. All rights reserved.
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 - Clinical Trials as Topic/methods
KW - Feasibility Studies
KW - Humans
KW - Medical Oncology/methods
KW - Neoplasms/drug therapy
KW - Randomized Controlled Trials as Topic/methods
KW - Research Design
UR - https://www.scopus.com/pages/publications/86000634326
UR - https://www.mendeley.com/catalogue/5d464a2e-e69e-39dd-bb98-bc547ed6615c/
U2 - 10.1016/j.drudis.2025.104324
DO - 10.1016/j.drudis.2025.104324
M3 - Review article
C2 - 40054765
AN - SCOPUS:86000634326
SN - 1359-6446
VL - 30
SP - 104324
JO - Drug Discovery Today
JF - Drug Discovery Today
IS - 3
M1 - 104324
ER -