Defining Efficacy Estimands in Clinical Trials: Examples Illustrating ICH E9(R1) Guidelines.
Ther Innov Regul Sci
; 54(2): 370-384, 2020 03.
Article
em En
| MEDLINE
| ID: mdl-32072586
ABSTRACT
This paper provides examples of defining estimands in real-world scenarios following ICH E9(R1) guidelines. Detailed discussions on choosing the estimands and estimators can be found in our companion papers. Three scenarios of increasing complexity are illustrated. The first example is a proof-of-concept trial in major depressive disorder where the estimand is chosen to support the sponsor decision on whether to continue development. The second and third examples are confirmatory trials in severe asthma and rheumatoid arthritis respectively. We discuss the intercurrent events expected during each trial and how they can be handled so as to be consistent with the study objectives. The estimands discussed in these examples are not the only acceptable choices for their respective scenarios. The intent is to illustrate the key concepts rather than focus on specific choices. Emphasis is placed on following a study development process where estimands link the study objectives with data collection and analysis in a coherent manner, thereby avoiding disconnect between objectives, estimands, and analyses.
Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Asma
/
Transtorno Depressivo Maior
Tipo de estudo:
Guideline
/
Prognostic_studies
/
Qualitative_research
Limite:
Humans
Idioma:
En
Ano de publicação:
2020
Tipo de documento:
Article