Augmenting randomized clinical trial data with historical control data: Precision medicine applications.
J Natl Cancer Inst
; 115(1): 14-20, 2023 01 10.
Article
en En
| MEDLINE
| ID: mdl-36161487
ABSTRACT
As precision medicine becomes more precise, the sizes of the molecularly targeted subpopulations become increasingly smaller. This can make it challenging to conduct randomized clinical trials of the targeted therapies in a timely manner. To help with this problem of a small patient subpopulation, a study design that is frequently proposed is to conduct a small randomized clinical trial (RCT) with the intent of augmenting the RCT control arm data with historical data from a set of patients who have received the control treatment outside the RCT (historical control data). In particular, strategies have been developed that compare the treatment outcomes across the cohorts of patients treated with the standard (control) treatment to guide the use of the historical data in the analysis; this can lessen the potential well-known biases of using historical controls without any randomization. Using some simple examples and completed studies, we demonstrate in this commentary that these strategies are unlikely to be useful in precision medicine applications.
Texto completo:
1
Colección:
01-internacional
Base de datos:
MEDLINE
Asunto principal:
Proyectos de Investigación
/
Medicina de Precisión
Tipo de estudio:
Clinical_trials
Límite:
Humans
Idioma:
En
Revista:
J Natl Cancer Inst
Año:
2023
Tipo del documento:
Article
País de afiliación:
Estados Unidos