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Abstract:
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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. To facilitate appropriate statistical inference, many powerful estimation tools have been developed in R, including various dose-response packages based on nonlinear least squares method. Recently, beta regression-based methods have been introduced in estimation of the median-effect equation. In theory, they can overcome non-normality, heteroscedasticity and asymmetry and accommodate flexible robust frameworks and coefficients penalization. To identify a reliable estimation method(s) to estimate dose-response curves, we conducted a comparative study to review 14 different tools in R and examine their robustness and efficiency via Monte Carlo simulation. 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.
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