Estimands in hematologic oncology trials.
ICH E9 addendum
estimand
hematology clinical trials
intercurrent events
Journal
Pharmaceutical statistics
ISSN: 1539-1612
Titre abrégé: Pharm Stat
Pays: England
ID NLM: 101201192
Informations de publication
Date de publication:
07 2021
07 2021
Historique:
revised:
22
12
2020
received:
27
04
2020
accepted:
01
02
2021
pubmed:
10
3
2021
medline:
26
11
2021
entrez:
9
3
2021
Statut:
ppublish
Résumé
The estimand framework included in the addendum to the ICH E9 guideline facilitates discussions to ensure alignment between the key question of interest, the analysis, and interpretation. Therapeutic knowledge and drug mechanism play a crucial role in determining the strategy and defining the estimand for clinical trial designs. Clinical trials in patients with hematological malignancies often present unique challenges for trial design due to complexity of treatment options and existence of potential curative but highly risky procedures, for example, stem cell transplant or treatment sequence across different phases (induction, consolidation, maintenance). Here, we illustrate how to apply the estimand framework in hematological clinical trials and how the estimand framework can address potential difficulties in trial result interpretation. This paper is a result of a cross-industry collaboration to connect the International Conference on Harmonisation (ICH) E9 addendum concepts to applications. Three randomized phase 3 trials will be used to consider common challenges including intercurrent events in hematologic oncology trials to illustrate different scientific questions and the consequences of the estimand choice for trial design, data collection, analysis, and interpretation. Template language for describing estimand in both study protocols and statistical analysis plans is suggested for statisticians' reference.
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
793-805Informations de copyright
© 2021 John Wiley & Sons Ltd.
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