Multistate models as a framework for estimand specification in clinical trials of complex processes.
Stat Med
; 42(9): 1368-1397, 2023 04 30.
Article
en En
| MEDLINE
| ID: mdl-36721334
ABSTRACT
Intensity-based multistate models provide a useful framework for characterizing disease processes, the introduction of interventions, loss to followup, and other complications arising in the conduct of randomized trials studying complex life history processes. Within this framework we discuss the issues involved in the specification of estimands and show the limiting values of common estimators of marginal process features based on cumulative incidence function regression models. When intercurrent events arise we stress the need to carefully define the target estimand and the importance of avoiding targets of inference that are not interpretable in the real world. This has implications for analyses, but also the design of clinical trials where protocols may help in the interpretation of estimands based on marginal features.
Palabras clave
Texto completo:
1
Bases de datos:
MEDLINE
Asunto principal:
Proyectos de Investigación
/
Modelos Estadísticos
Tipo de estudio:
Clinical_trials
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Guideline
/
Prognostic_studies
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Risk_factors_studies
Límite:
Humans
Idioma:
En
Revista:
Stat Med
Año:
2023
Tipo del documento:
Article
País de afiliación:
Canadá