Optimal designs for phase II/III drug development programs including methods for discounting of phase II results.

ABSTRACT:

Background

Go/no-go decisions after phase II and sample size chosen for phase III are usually based on phase II results (e.g., the treatment effect estimate of phase II). Due to the decision rule (only promising phase II results lead to phase III), treatment effect estimates from phase II that initiate a phase III trial commonly overestimate the true treatment effect. Underpowered phase III trials are the consequence. Optimistic findings may then not be reproduced, leading to the failure of potentially expensive drug development programs. For some disease areas these failure rates are described to be quite high: 62.5%.

Methods

We integrate the ideas of multiplicative and additive adjustment of treatment effect estimates after go decisions in a utility-based framework for opt

SUBMITTER: Erdmann S 

PROVIDER: S-EPMC7547445 | biostudies-literature | 2020 Oct

REPOSITORIES: biostudies-literature