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.

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.

A Litany of Problems With p-values

With the many problems that p-values have, and the temptation to “bless” research when the p-value falls below an arbitrary threshold such as 0.05 or 0.005, researchers using p-values should at least be fully aware of what they are getting. They need to know exactly what a p-value means and what are the assumptions required for it to have that meaning. ♦ A p-value is the probability of getting, in another study, a test statistic that is more extreme than the one obtained in your study if a series of assumptions hold.

Clinicians' Misunderstanding of Probabilities Makes Them Like Backwards Probabilities Such As Sensitivity, Specificity, and Type I Error

Optimum decision making in the presence of uncertainty comes from probabilistic thinking. The relevant probabilities are of a predictive nature: P(the unknown given the known). Thresholds are not helpful and are completely dependent on the utility/cost/loss function. Corollary: Since p-values are P(someone else’s data are more extreme than mine if H0 is true) and we don’t know whether H0 is true, it is a non-predictive probability that is not useful for decision making.

Classification vs. Prediction

It is important to distinguish prediction and classification. In many decisionmaking contexts, classification represents a premature decision, because classification combines prediction and decision making and usurps the decision maker in specifying costs of wrong decisions. The classification rule must be reformulated if costs/utilities or sampling criteria change. Predictions are separate from decisions and can be used by any decision maker. Classification is best used with non-stochastic/deterministic outcomes that occur frequently, and not when two individuals with identical inputs can easily have different outcomes.