2021
DOI: 10.1177/09622802211017574
|View full text |Cite
|
Sign up to set email alerts
|

Frequentist rules for regulatory approval of subgroups in phase III trials: A fresh look at an old problem

Abstract: Background The number of Phase III trials that include a biomarker in design and analysis has increased due to interest in personalised medicine. For genetic mutations and other predictive biomarkers, the trial sample comprises two subgroups, one of which, say [Formula: see text] is known or suspected to achieve a larger treatment effect than the other [Formula: see text]. Despite treatment effect heterogeneity, trials often draw patients from both subgroups, since the lower responding [Formula: see text] subg… Show more

Help me understand this report

Search citation statements

Order By: Relevance

Paper Sections

Select...
2

Citation Types

0
2
0

Year Published

2022
2022
2022
2022

Publication Types

Select...
1

Relationship

0
1

Authors

Journals

citations
Cited by 1 publication
(2 citation statements)
references
References 16 publications
0
2
0
Order By: Relevance
“…To inform this decision, the efficacy in BM− is crucial. 37 Whether or not defined in the study protocol, the estimand for BM− may therefore be important. Its definition will typically follow directly from those of the other estimands, i.e., will mainly differ by the ”population” attribute.…”
Section: Estimands By Type Of Adaptation and Innovative Characteristicsmentioning
confidence: 99%
See 1 more Smart Citation
“…To inform this decision, the efficacy in BM− is crucial. 37 Whether or not defined in the study protocol, the estimand for BM− may therefore be important. Its definition will typically follow directly from those of the other estimands, i.e., will mainly differ by the ”population” attribute.…”
Section: Estimands By Type Of Adaptation and Innovative Characteristicsmentioning
confidence: 99%
“…While the trial sponsor may be interested in showing efficacy in AC (or BM+), regulators and payers may ask whether a proven efficacy in AC should lead to approval and reimbursement in the full population or, say, only in BM+. To inform this decision, the efficacy in BM− is crucial 37 . Whether or not defined in the study protocol, the estimand for BM− may therefore be important.…”
Section: Estimands By Type Of Adaptation and Innovative Characteristicsmentioning
confidence: 99%