Designing precision medicine trials to yield a greater population impact.
Biometrics
; 76(2): 643-653, 2020 06.
Article
em En
| MEDLINE
| ID: mdl-31598964
ABSTRACT
Traditionally, a clinical trial is conducted comparing treatment to standard care for all patients. However, it could be inefficient given patients' heterogeneous responses to treatments, and rapid advances in the molecular understanding of diseases have made biomarker-based clinical trials increasingly popular. We propose a new targeted clinical trial design, termed as Max-Impact design, which selects the appropriate subpopulation for a clinical trial and aims to optimize population impact once the trial is completed. The proposed design not only gains insights on the patients who would be included in the trial but also considers the benefit to the excluded patients. We develop novel algorithms to construct enrollment rules for optimizing population impact, which are fairly general and can be applied to various types of outcomes. Simulation studies and a data example from the SWOG Cancer Research Network demonstrate the competitive performance of our proposed method compared to traditional untargeted and targeted designs.
Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Ensaios Clínicos como Assunto
/
Medicina de Precisão
Tipo de estudo:
Prognostic_studies
/
Risk_factors_studies
Limite:
Humans
/
Male
Idioma:
En
Revista:
Biometrics
Ano de publicação:
2020
Tipo de documento:
Article