Bayesian meta-experimental design: evaluating cardiovascular risk in new antidiabetic therapies to treat type 2 diabetes

Biometrics. 2012 Jun;68(2):578-86. doi: 10.1111/j.1541-0420.2011.01679.x. Epub 2011 Sep 28.

Abstract

Recent guidance from the Food and Drug Administration for the evaluation of new therapies in the treatment of type 2 diabetes (T2DM) calls for a program-wide meta-analysis of cardiovascular (CV) outcomes. In this context, we develop a new Bayesian meta-analysis approach using survival regression models to assess whether the size of a clinical development program is adequate to evaluate a particular safety endpoint. We propose a Bayesian sample size determination methodology for meta-analysis clinical trial design with a focus on controlling the type I error and power. We also propose the partial borrowing power prior to incorporate the historical survival meta data into the statistical design. Various properties of the proposed methodology are examined and an efficient Markov chain Monte Carlo sampling algorithm is developed to sample from the posterior distributions. In addition, we develop a simulation-based algorithm for computing various quantities, such as the power and the type I error in the Bayesian meta-analysis trial design. The proposed methodology is applied to the design of a phase 2/3 development program including a noninferiority clinical trial for CV risk assessment in T2DM studies.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Algorithms
  • Bayes Theorem
  • Biometry / methods*
  • Cardiovascular Diseases / etiology*
  • Cardiovascular Diseases / prevention & control
  • Clinical Trials, Phase II as Topic / statistics & numerical data
  • Clinical Trials, Phase III as Topic / statistics & numerical data
  • Computer Simulation
  • Diabetes Mellitus, Type 2 / drug therapy*
  • Humans
  • Hypoglycemic Agents / adverse effects
  • Hypoglycemic Agents / therapeutic use
  • Markov Chains
  • Meta-Analysis as Topic
  • Monte Carlo Method
  • Randomized Controlled Trials as Topic / statistics & numerical data
  • Risk Factors
  • Sample Size
  • Survival Analysis

Substances

  • Hypoglycemic Agents