#estimand — Public Fediverse posts

Reading a paper on the estimands framework and in one of the examples, analysis strategies to deal with data from patients dying before the end of the clinical trial are considered.

A hypothetical strategy what if all patients would have survived, is considered but dismissed because
“… no mechanism to avoid death exists …”

And I really do like how methodological papers suddenly turn to deadpan humor, just for a part of a sentence.

#estimand #statistics #darkhumor

The estimand framework, formalized in ICH E9(R1) regulatory guidance, provides a structured approach to define scientific objectives with precision. We apply the estimand framework to dose–exposure–response analyses. ... strategy to improve exposure–response analyses for dose selection, particularly when the relevant evidence includes data from multiple studies.

#estimand #exposure-response #dose-response #causal #pharmacometrics #pmx

https://doi.org/10.1002/psp4.70202

The estimand framework, formalized in ICH E9(R1) regulatory guidance, provides a structured approach to define scientific objectives with precision. We apply the estimand framework to dose–exposure–response analyses. ... strategy to improve exposure–response analyses for dose selection, particularly when the relevant evidence includes data from multiple studies.

#estimand #exposure-response #dose-response #causal #pharmacometrics #pmx

https://doi.org/10.1002/psp4.70202

A review of UK MHRA protocols (k=122) of the use of #Estimand -s offers insights for improvement of protocols due to incomplete or incorrect specifications:
https://trialsjournal.biomedcentral.com/articles/10.1186/s13063-025-08991-8

#RCT #StudyDesign #Registration #Causality

Further resources:

How estimands support stating the exact research question of a study and the interpretation of results:
https://www.bmj.com/content/384/bmj-2023-076316

Application of the estimand framework to for studies with Patient-Reported Outcomes:
https://jpro.springeropen.com/articles/10.1186/s41687-020-00218-5
#HRQOL

Trigg et al discussed conceptualisations of meaningful between-group differences:
https://rdcu.be/ehHdD

A comment by Kevin Weinfurt advances the discussion w 4 points
https://rdcu.be/ehHfS

#RCT #Estimand #HRQL

From the abstract:

(1) rather than “between-group difference,” specify the level at which you wish to infer a treatment effect: population or individual;

(2) points of reference may be different for interpreting individual- and population-level treatment effect estimates;
...

But in all these years I found it difficult to get a grasp on how #EducationalResearch understands and conceptualises #ControlGroups.

In my health-oriented research this discussion is very prevalent.
#OutcomeDefinition #Estimand

And while I admired the early uptake of...

But in all these years I found it difficult to get a grasp on how #EducationalResearch understands and conceptualises #ControlGroups.

In my health-oriented research this discussion is very prevalent.
#OutcomeDefinition #Estimand

And while I admired the early uptake of...