The role of the estimand framework in the analysis of patient-reported outcomes in single-arm trials: a case study in oncology
Doranne Thomassen, Satrajit Roychoudhury, Cecilie Delphin Amdal, Dries Reynders, Jammbe Z Musoro, Willi Sauerbrei, Els Goetghebeur, Saskia le Cessie, SISAQOL-IMI Work Package 3 · 2024
Read The role of the estimand framework in the analysis of patient-reported outcomes in single-arm trials: a case study in oncology by Doranne Thomassen, Satrajit Roychoudhury, Cecilie Delphin Amdal, Dries Reynders, Jammbe Z Musoro, Willi Sauerbrei, Els Goetghebeur, Saskia le Cessie, SISAQOL-IMI Work Package 3 (2024) in the Tower of Babel, a free online library of public-domain works preserved in clean, searchable text. No sign-up. The full text is served as a plain-text file at a predictable URL, where text is present: https://antarctic-labs.com/tower-of-babel/pmc-11585159.txt.
Description
Thomassen and co-authors present a 2024 case study on sharpening patient-reported outcomes in single-arm cancer trials. Such trials feed drug approvals, yet their lack of a control arm invites bias. A clear estimand, the exact quantity a trial aims to measure, helps readers judge the numbers. The team applied a standard framework to quality of life data from a real lung cancer trial.
Each choice for handling death, dropout, and disease spread gave its own quality-of-life path over time. Each path also carried its own meaning for a matching research aim. A while-alive strategy for death proved one workable option. The study shows trial teams how to state their target before analysis.
Catalog record
| CREATOR | Doranne Thomassen, Satrajit Roychoudhury, Cecilie Delphin Amdal, Dries Reynders, Jammbe Z Musoro, Willi Sauerbrei, Els Goetghebeur, Saskia le Cessie, SISAQOL-IMI Work Package 3 |
|---|---|
| YEAR | 2024 |
| COLLECTION | PAPERS |
| FORMAT | TXT |
| FILE SIZE | 49 KB |
| VERSION | 1.0 |
| LICENSE | CC BY 4.0 |
| RIGHTS | CC BY |
| SOURCE | Europe PMC |