The role of the estimand framework in the analysis of patient-reported outcomes in single-arm trials: a case study in oncology
The results show that decisions made in the estimand framework are not trivial. Trial results and their interpretation strongly depend on the chosen estimand. The estimand framework provides a structure to match a research question with a clear target of estimation, supporting specific clinical decisions. Adherence to this framework can help improve the quality of data collection, analysis and reporting of PROs in SATs, impacting decision making in clinical practice.
Authors
- Ragnhild Sørum Falk (ORCID: https://orcid.org/0000-0001-8398-3492)
- Tove Ragna Reksten (ORCID: https://orcid.org/0000-0001-8704-4943)
- Willi Sauerbrei (ORCID: https://orcid.org/0000-0002-6792-4123)
- Ahu Alanya (ORCID: https://orcid.org/0009-0007-0627-4300)
- Jennifer A. Black (ORCID: https://orcid.org/0000-0001-7049-3814)
- Doranne Thomassen (ORCID: https://orcid.org/0000-0002-9450-6015)
- Karen Keating (ORCID: https://orcid.org/0000-0001-6993-3114)
- Jaap C. Reijneveld (ORCID: https://orcid.org/0000-0002-2257-3147)
- Satrajit Roychoudhury (ORCID: https://orcid.org/0000-0003-4001-3036)
- Gerhard Rumpold (ORCID: https://orcid.org/0000-0002-6037-793X)
- Jammbe Musoro (ORCID: https://orcid.org/0000-0002-6228-3531)
- Rajesh Kamalakar
- Samantha Cruz Rivera (ORCID: https://orcid.org/0000-0002-1566-6804)
- Geert Molenberghs (ORCID: https://orcid.org/0000-0002-6453-5448)
- Kavita Sail
- Antoine Regnault (ORCID: https://orcid.org/0000-0001-8234-2896)
- Anja Schiel (ORCID: https://orcid.org/0000-0002-1258-4731)
- Paul G. Kluetz (ORCID: https://orcid.org/0000-0002-9796-4791)
- Khadija Rantell (ORCID: https://orcid.org/0000-0002-6747-7650)
- Cecilie Delphin Amdal (ORCID: https://orcid.org/0000-0001-8422-8085)
- Kelly Van Lancker (ORCID: https://orcid.org/0000-0003-3458-3788)
- Melanie Calvert (ORCID: https://orcid.org/0000-0002-1856-837X)
- Claudia Rutherford (ORCID: https://orcid.org/0000-0002-4637-4572)
- Vishal Bhatnagar (ORCID: https://orcid.org/0000-0003-4650-0513)
- Saskia le Cessie (ORCID: https://orcid.org/0000-0003-2154-4923)
- Dries Reynders (ORCID: https://orcid.org/0000-0001-6875-048X)
- Mallorie H. Fiero (ORCID: https://orcid.org/0000-0001-5536-116X)
- Silene ten Seldam
- Olalekan Lee Aiyegbusi (ORCID: https://orcid.org/0000-0001-9122-8251)
- Ethan Basch (ORCID: https://orcid.org/0000-0003-3813-9318)
- Els Goetghebeur (ORCID: https://orcid.org/0000-0002-8896-0721)
- Joseph C. Cappelleri (ORCID: https://orcid.org/0000-0001-9586-0748)
- Limin Liu
- Ting‐Yu Chen (ORCID: https://orcid.org/0000-0002-3445-9907)
- Yun Su (ORCID: https://orcid.org/0000-0002-1408-8459)
- Alicyn Campbell
- David Ness
- Michael Schlichting
- Alexander Russell-Smith
- Maxime Sasseville
Institutions
- Oslo University Hospital (NO)
- University of Freiburg (DE)
- Pfizer (United States) (US)
- Leiden University Medical Center (NL)
- Ghent University (BE)
- European Organisation for Research and Treatment of Cancer (BE)
Publication Details
- Journal
- BMC Medical Research Methodology
- Published
- 2024-11-23
- DOI
- https://doi.org/10.1186/s12874-024-02408-x
- Citations
- 7
- Primary Topic
- Health Systems, Economic Evaluations, Quality of Life
- Type
- article
- Field-Weighted Citation Impact
- 7.90
Funders
- American Society of Clinical Oncology
- European Federation of Pharmaceutical Industries and Associations
- European Commission
- European Society for Medical Oncology
- Health Canada
- National Cancer Institute