On the choice of doses for phase III clinical trials
Artikel i vetenskaplig tidskrift, 2013

Many potential new medicines fail in phase III clinical trials, because of either insufficient efficacy or intolerability. Such failures may be caused by the absence of an effect and also if a suboptimal dose is being tested. It is thus important to consider how to optimise the choice of dose or doses that continue into the confirmatory phase. For many indications, it is common to test one single active dose in phase III. However, phase IIB dose-finding trials are relatively small and often lack the ability of precisely estimating the dose–response curves for efficacy and tolerability. Because of this uncertainty in dose response, it is reasonable to consider bringing more than one dose into phase III. Using simple but illustrative models, we find the optimal doses and compare the probability of success, for fixed total sample sizes, when one or two active doses are included in phase III.

benefit/risk

optimal doses

Bayesian decision analysis

design of clinical trials

probability of success

multiple testing

Författare

Vera Lisovskaja

Göteborgs universitet

Chalmers, Matematiska vetenskaper, matematisk statistik

Carl-Fredrik Burman

Göteborgs universitet

Chalmers, Matematiska vetenskaper, matematisk statistik

Statistics in Medicine

0277-6715 (ISSN) 1097-0258 (eISSN)

Vol. 32 1661-1676

Ämneskategorier

Sannolikhetsteori och statistik

DOI

10.1002/sim.5632