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

Kimmett Edgar | Zendy; Dan Jackson | Zendy; Kirsty Rhodes | Zendy; Tomás Duffy | Zendy; C-F Burman | Zendy; Linda Sharples | Zendy

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

Author(s) -

Kimmett Edgar,

Dan Jackson,

Kirsty Rhodes,

Tomás Duffy,

C-F Burman,

Linda Sharples

Publication year - 2021

Publication title -

statistical methods in medical research

Language(s) - English

Resource type - Journals

SCImago Journal Rank - 1.952

H-Index - 85

eISSN - 1477-0334

pISSN - 0962-2802

DOI - 10.1177/09622802211017574

Subject(s) - frequentist inference , subgroup analysis , sample size determination , type i and type ii errors , multiple comparisons problem , econometrics , statistics , covariate , medicine , population , statistical power , meta analysis , mathematics , actuarial science , bayesian probability , bayesian inference , economics , environmental health

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 B + is known or suspected to achieve a larger treatment effect than the other B - . Despite treatment effect heterogeneity, trials often draw patients from both subgroups, since the lower responding B - subgroup may also gain benefit from the intervention. In this case, regulators/commissioners must decide what constitutes sufficient evidence to approve the drug in the B - population.

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