Taking the long view: how to design a series of Phase III trials to maximize cumulative therapeutic benefit.

ABSTRACT:

Background

Traditional clinical trial designs strive to definitively establish the superiority of an experimental treatment, which results in risk-adverse criteria and large sample sizes. Increasingly, common cancers are recognized as consisting of small subsets with specific aberrations for targeted therapy, making large trials infeasible.

Purpose

To compare the performance of different trial design strategies over a long-term research horizon.

Methods

We simulated a series of two-treatment superiority trials over 15 years using different design parameters. Trial parameters examined included the number of positive trials to establish superiority (one-trial vs. two-trial rule), α level (2.5%-50%), and the number of trials in the 15-year period, K (thus, trial sample s

SUBMITTER: Deley MC 

PROVIDER: S-EPMC3904223 | biostudies-literature | 2012 Jun

REPOSITORIES: biostudies-literature