Semiparametric estimation exploiting covariate independence in two-phase randomized trials

Biometrics. 2009 Mar;65(1):178-87. doi: 10.1111/j.1541-0420.2008.01046.x. Epub 2008 May 13.

Abstract

Recent results for case-control sampling suggest when the covariate distribution is constrained by gene-environment independence, semiparametric estimation exploiting such independence yields a great deal of efficiency gain. We consider the efficient estimation of the treatment-biomarker interaction in two-phase sampling nested within randomized clinical trials, incorporating the independence between a randomized treatment and the baseline markers. We develop a Newton-Raphson algorithm based on the profile likelihood to compute the semiparametric maximum likelihood estimate (SPMLE). Our algorithm accommodates both continuous phase-one outcomes and continuous phase-two biomarkers. The profile information matrix is computed explicitly via numerical differentiation. In certain situations where computing the SPMLE is slow, we propose a maximum estimated likelihood estimator (MELE), which is also capable of incorporating the covariate independence. This estimated likelihood approach uses a one-step empirical covariate distribution, thus is straightforward to maximize. It offers a closed-form variance estimate with limited increase in variance relative to the fully efficient SPMLE. Our results suggest exploiting the covariate independence in two-phase sampling increases the efficiency substantially, particularly for estimating treatment-biomarker interactions.

Publication types

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

MeSH terms

  • Algorithms
  • Biomarkers
  • Biometry / methods*
  • Data Interpretation, Statistical
  • Humans
  • Models, Theoretical
  • Randomized Controlled Trials as Topic / statistics & numerical data*
  • Treatment Outcome

Substances

  • Biomarkers