Skip to main content

A practical guide to the estimands framework

Published on March 31, 2025   47 min

A selection of talks on Methods

Please wait while the transcript is being prepared...
0:00
Hello. My name is Brennan Kahan. I'm based at the MRC Clinical Trials Unit at UCL and I'll be talking about a practical guide to the estimands framework.
0:14
I'll be giving some of the background and motivation to why estimands are important. I'll be covering what an estimand is. Going through the estimand framework, I'm giving an example of applying the framework to a real trial and offering some conclusions.
0:32
So, we know that randomized trials are used to answer questions about the safety and effectiveness of different interventions, but trials can actually be used to answer different types of questions about how safe and effective an intervention is. So, if we had a trial where some patients stopped their assigned treatment early, you might be interested in understanding the effective treatment if all patients have actually taken treatment for its full course or else we might be interested in the effect of the intervention regardless of whether the fact some patients had to stop early. These different questions might actually lead to quite different answers about how safe and effective an intervention is. So, if we're someone who's using trial results to inform decision making, it's important to understand which of these questions a trial has been designed to answer.
1:26
However, historically, most randomized trials have not reported this information. So, they don't say exactly which question or which treatment effect they're trying to estimate. So, if we're someone who's using results from this trial to inform decision making, we need to instead try to infer what the question is from the study methods. So, how they've designed the trial and done the analysis. Now, the issue is that this is actually really challenging doom, and I'll give you an example of that.

Quiz available with full talk access. Request Free Trial or Login.

Hide

A practical guide to the estimands framework

Embed in course/own notes