Reweighting estimators to extend the external validity of clinical trials: methodological considerations.
J Biopharm Stat
; 33(5): 515-543, 2023 09 03.
Article
en En
| MEDLINE
| ID: mdl-36688658
ABSTRACT
Methods to extend the strong internal validity of randomized controlled trials to reliably estimate treatment effects in target populations are gaining attention. This paper enumerates steps recommended for undertaking such extended inference, discusses currently viable choices for each one, and provides recommendations. We demonstrate a complete extended inference from a clinical trial studying a pharmaceutical treatment for Alzheimer's disease (AD) to a realistic target population of European residents diagnosed with AD. This case study highlights approaches to overcoming practical difficulties and demonstrates limitations of reliably extending inference from a trial to a real-world population.
Palabras clave
Texto completo:
1
Colección:
01-internacional
Base de datos:
MEDLINE
Asunto principal:
Ensayos Clínicos Controlados Aleatorios como Asunto
/
Enfermedad de Alzheimer
Tipo de estudio:
Clinical_trials
/
Diagnostic_studies
/
Guideline
Límite:
Humans
Idioma:
En
Revista:
J Biopharm Stat
Asunto de la revista:
FARMACOLOGIA
Año:
2023
Tipo del documento:
Article
País de afiliación:
Estados Unidos