Posted by Michael Wonder on 05 Jan 2020
Beyond “Intent‐to‐treat” and “Per protocol”: improving assessment of treatment effects in clinical trials through the specification of an estimand
27 December 2019 - There is a key problem in randomised clinical trials as outcomes can be distorted due to informative post‐randomisation events.
This is inadequately addressed by the use of traditional intention‐to‐treat or per protocol analysis sets and often either ignored or wrongly labelled as missing data.
As a consequence, the treatment effects of interest in a clinical trial are not well defined and their estimates might be misinterpreted.
The estimand framework should help all those planning, conducting and analysing clinical trials as well as those interpreting the results to better define, estimate and understand the treatment effects of interest.
