Nested g-computation procedure

What is the difference in health care cost when two different treatments are used?  This question is challenging because cumulative health care cost is often censored either by death or lack of continuous enrollment.  Lin (2000) addressed this issue in his 2000 paper (see paper and my blog write-up). The problem with this approach, however,…

Berkson’s paradox

Berkson’s paradox happens when given two independent events, if you only consider outcomes where at least one occurs, then they become negatively dependent.  More technically, this paradox occurs when there is ascertainment bias in a study design. Let me provide an example. Consider the case where patients can have diabetes or HIV.  Assume that patients have a positive probability of…

Basket vs. Umbrella clinical trials

How do you determine if a drug is effective?  Typically, biostaticians rely on a randomized control trial where half the patients receive the treatment of interest and the remaining half receive either a placebo or the current standard of care depending on the trial design. Recent advances in cancer, however, call for more sophisticated designs.  Some cancer treatments are only…

Measuring cause-specific mortality?

This question is not so easy to answer, even when using data from a randomized trial.  Further, many studies do not have the statistical power to identify cause-specific mortality.  Consider the following example from Kim and Thompson: Consider a trial of an intervention only influencing a single cause of death, or a few specific causes…