CBC’s Ideas has been running a series of shows on heart disease called “Heart of the Matter”. Episode 2 is particularly interesting from a statistical perspective, as the episode discusses several difficulties with the analysis of drug efficacy. Some highlights include:
Effect sizes Some of the best cited studies for the use of drugs to treat heart disease show a statistically significant effect of only a few percentage points improvement. Contrast this with a dramatic, vastly superior improvement from diet alone.
Response variables The focus of many drug studies has been on the reduction of cholesterol, rather than reductions in heart disease. Diet studies, for example, have shown dramatic improvements in reducing heart attacks while having no effect on cholesterol levels. Conversely, drug studies that show a reduction in cholesterol show no change in mortality rates.
Blocking of data Separate analyses of drug efficacy on female or elderly patients tend to show that drug therapy increases overall mortality. Lumping these data in with the traditional middle-aged male patients removes this effect and, instead, shows a significant decrease in heart disease with drug use.
The point here isn’t to make a comment on the influence of drug companies on medical research. Rather, such statistical concerns are common to all research disciplines. The primary concern of such analyses should be: what is the magnitude of the effect of a specific treatment on my variable of interest? The studies discussed in the Ideas program suggest that much effort has been devoted to detecting significant effects of drugs on surrogate response variables regardless of the size of the effect.