idem: An R Package for Inferences in Clinical Trials with Death and Missingness

idem: An R Package for Inferences in Clinical Trials with Death and Missingness

clinical trial, truncation due to death, composite endpoint, imputation, missing
data, R, SACE, sensitivity analysis, shiny, Stan

In randomized controlled trials of seriously ill patients, death is common and often
defined as the primary endpoint. Increasingly, non-mortality outcomes such as functional
outcomes are co-primary or secondary endpoints. Functional outcomes are not defined
for patients who die, referred to as “truncation due to death”, and among survivors,
functional outcomes are often unobserved due to missed clinic visits or loss to follow-up.
It is well known that if the functional outcomes “truncated due to death” or missing are
handled inappropriately, treatment effect estimation can be biased. In this paper, we
describe the package idem that implements a procedure for comparing treatments that is
based on a composite endpoint of mortality and the functional outcome among survivors.
Among survivors, the procedure incorporates a missing data imputation procedure with
a sensitivity analysis strategy. A web-based graphical user interface is provided in the
idem package to facilitate users conducting the proposed analysis in an interactive and
user-friendly manner. We demonstrate idem using data from a recent trial of sedation
interruption among mechanically ventilated patients

Chenguang Wang

https://www.jstatsoft.org/article/view/v093i12

Johns Hopkins University

April 2020

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