Adaptive Optimal Designs for Dose-Finding Studies with Time-to-Event Outcomes

AAPS J. 2017 Dec 28;20(1):24. doi: 10.1208/s12248-017-0166-5.

Abstract

We consider optimal design problems for dose-finding studies with censored Weibull time-to-event outcomes. Locally D-optimal designs are investigated for a quadratic dose-response model for log-transformed data subject to right censoring. Two-stage adaptive D-optimal designs using maximum likelihood estimation (MLE) model updating are explored through simulation for a range of different dose-response scenarios and different amounts of censoring in the model. The adaptive optimal designs are found to be nearly as efficient as the locally D-optimal designs. A popular equal allocation design can be highly inefficient when the amount of censored data is high and when the Weibull model hazard is increasing. The issues of sample size planning/early stopping for an adaptive trial are investigated as well. The adaptive D-optimal design with early stopping can potentially reduce study size while achieving similar estimation precision as the fixed allocation design.

Keywords: D-optimal design; Weibull distribution; adaptive design; censoring; dose finding.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Clinical Trials as Topic / methods*
  • Dose-Response Relationship, Drug
  • Drug Development / methods
  • Humans
  • Likelihood Functions
  • Pharmaceutical Research / methods*
  • Research Design*
  • Sample Size