A comparative study of in vitro dose–response estimation under extreme observations

Xinying Fang, Shouhao Zhou

Research output: Contribution to journalArticlepeer-review

1 Scopus citations

Abstract

Quantifying drug potency, which requires an accurate estimation of dose–response relationship, is essential for drug development in biomedical research and life sciences. However, the standard estimation procedure of the median–effect equation to describe the dose–response curve is vulnerable to extreme observations in common experimental data. To facilitate appropriate statistical inference, many powerful estimation tools have been developed in R, including various dose–response packages based on the nonlinear least squares method with different optimization strategies. Recently, beta regression-based methods have also been introduced in estimation of the median–effect equation. In theory, they can overcome nonnormality, heteroscedasticity, and asymmetry and accommodate flexible robust frameworks and coefficients penalization. To identify a reliable estimation method(s) to estimate dose–response curves even with extreme observations, we conducted a comparative study to review 14 different tools in R and examine their robustness and efficiency via Monte Carlo simulation under a list of comprehensive scenarios. The simulation results demonstrate that penalized beta regression using the mgcv package outperforms other methods in terms of stable, accurate estimation, and reliable uncertainty quantification.

Original languageEnglish (US)
Article number2200092
JournalBiometrical Journal
Volume66
Issue number1
DOIs
StatePublished - Jan 2024

All Science Journal Classification (ASJC) codes

  • Statistics and Probability
  • Statistics, Probability and Uncertainty

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