Implementation of the PATH Statement

The recent PATH (Predictive Approaches to Treatment effect Heterogeneity) Statement outlines principles, criteria, and key considerations for applying predictive approaches to clinical trials to provide patient-centered evidence in support of decision making. Here challenges in implementing the PATH Statement are addressed with the GUSTO-I trial as a case study.

RCT Analyses With Covariate Adjustment

This article summarizes arguments for the claim that the primary analysis of treatment effect in a RCT should be with adjustment for baseline covariates. It reiterates some findings and statements from classic papers, with illustration on the GUSTO-I trial.

Implications of Interactions in Treatment Comparisons

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.

Assessing Heterogeneity of Treatment Effect, Estimating Patient-Specific Efficacy, and Studying Variation in Odds ratios, Risk Ratios, and Risk Differences

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.

Statistically Efficient Ways to Quantify Added Predictive Value of New Measurements

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.

In Machine Learning Predictions for Health Care the Confusion Matrix is a Matrix of Confusion

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.

Viewpoints on Heterogeneity of Treatment Effect and Precision Medicine

This article provides my reflections after the PCORI/PACE Evidence and the Individual Patient meeting on 2018-05-31. The discussion includes a high-level view of heterogeneity of treatment effect in optimizing treatment for individual patients.

Navigating Statistical Modeling and Machine Learning

This article elaborates on Frank Harrell's post providing guidance in choosing between machine learning and statistical modeling for a prediction project.

Road Map for Choosing Between Statistical Modeling and Machine Learning

This article provides general guidance to help researchers choose between machine learning and statistical modeling for a prediction project.

Is Medicine Mesmerized by Machine Learning?

Deep learning and other forms of machine learning are getting a lot of press in medicine. The reality doesn't match the hype, and interpretable statistical models still have a lot to offer.