Views expressed here are neither those of Vanderbilt University nor the US FDA
PhD in Biostatistics, 1979
UNC Chapel Hill
BS in Mathematics, 1973
University of Alabama Birmingham
This article explains how the generalizability of randomized trial findings depends primarily on whether and how patient characteristics modify (interact with) the treatment effect. For an observational study this will be related to overlap in the propensity to receive treatment.
Reasons are given for why heterogeneity of treatment effect must be demonstrated, not assumed. An example is presented that shows that HTE must exceed a certain level before personalizing treatment results in better decisions than using the average treatment effect for everyone.
This article shows an example formally testing for heterogeneity of treatment effect in the GUSTO-I trial, shows how to use penalized estimation to obtain patient-specific efficacy, and studies variation across patients in three measures of treatment effect.
Researchers have used contorted, inefficient, and arbitrary analyses to demonstrated added value in biomarkers, genes, and new lab measurements. Traditional statistical measures have always been up to the task, and are more powerful and more flexible. It’s time to revisit them, and to add a few slight twists to make them more helpful.
The performance metrics chosen for prediction tools, and for Machine Learning in particular, have significant implications for health care and a penetrating understanding of the AUROC will lead to better methods, greater ML value, and ultimately, benefit patients.
Enhancing capabilities of CDER and its Office of Biostatistics in Bayesian clinical trial design and analysis
This is a free almost-weekly web course in introductory and intermediate biostatistics. Details are on the course web page.
I teach the BIOS7330 Regression Modeling Strategies course in the Biostatistics Graduate Program at Vanderbilt University in the spring semester. The course web page is here. I teach a 4-day version of this course each May at Vanderbilt. For 2020 the course will be held by webconference. Registration information for the short course may be found here.
I co-teach this course at Vanderbilt each February for postdoctoral medical and surgical fellows and junior faculty in the MSCI program.
stackexchangedatamethods.org is a discussion site where data methodologists meet each other and subject matter experts including clinical trialists and clinical researchers. Its development is documented here. Datamethods is provided by the Department of Biostatistics, Vanderbilt University School of Medicine.
I have written some short articles on the site, listed below.