The role of the estimand framework in the analysis of patient-reported outcomes in single-arm trials: a case study in oncology: a case study in oncology
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| Názov: | The role of the estimand framework in the analysis of patient-reported outcomes in single-arm trials: a case study in oncology: a case study in oncology |
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| Autori: | Thomassen, Doranne, Roychoudhury, Satrajit, Amdal, Cecilie Delphin, Reynders, Dries, Musoro, Jammbe Z., Sauerbrei, Willi, Goetghebeur, Els, le Cessie, Saskia, SISAQOL-IMI Work Package 3, Kamalakar, Rajesh, Sail, Kavita, Basch, Ethan, Reijneveld, Jaap, Keating, Karen, Su, Yun, Alanya, Ahu, Rumpold, Gerhard, Sasseville, Maxime, Black, Jennifer, Molenberghs, Geert, Rantell, Khadija, Schlichting, Michael, Regnault, Antoine, Ness, David, ten Seldam, Silene, Reksten, Tove Ragna, Schiel, Anja, Falk, Ragnhild Sorum, Campbell, Alicyn, Cappelleri, Joseph C., Russell-Smith, Alexander, Calvert, Melanie, Cruz Rivera, Samantha, Aiyegbusi, Olalekan Lee, Liu, Limin, Van Lancker, Kelly, Rutherford, Claudia, Bhatnagar, Vishal, Chen, Ting-Yu, Fiero, Mallorie, Kluetz, Paul |
| Zdroj: | BMC Med Res Methodol BMC Medical Research Methodology, Vol 24, Iss 1, Pp 1-13 (2024) BMC MEDICAL RESEARCH METHODOLOGY BMC Medical Research Methodology |
| Informácie o vydavateľovi: | Springer Science and Business Media LLC, 2024. |
| Rok vydania: | 2024 |
| Predmety: | Quality of life, Medicine (General), Lung Neoplasms, patient reported outcomes, Medical Oncology, 01 natural sciences, Single-arm trial, 03 medical and health sciences, R5-920, 0302 clinical medicine, estimand, Humans, Patient Reported Outcome Measures, 0101 mathematics, Repeated measurements, Clinical Trials as Topic, Patient-reported outcomes, Research, Estimand, Treatment Outcome, quality of life, Humans [MeSH], Medical Oncology/methods [MeSH], Treatment Outcome [MeSH], Oncology, Research Design [MeSH], Patient-centric approaches, Patient Reported Outcome Measures [MeSH], Clinical Trials as Topic/methods [MeSH], Quality of Life [MeSH], Lung Neoplasms/drug therapy [MeSH], Medical Oncology/standards [MeSH], Research Design, oncology, single arm trials, Quality of Life |
| Popis: | Background Patient-reported outcomes (PROs) play an increasing role in the evaluation of oncology treatments. At the same time, single-arm trials are commonly included in regulatory approval submissions. Because of the high risk of biases, results from single-arm trials require careful interpretation. This benefits from a clearly defined estimand, or target of estimation. In this case study, we demonstrated how the ICH E9 (R1) estimand framework can be implemented in SATs with PRO endpoints. Methods For the global quality of life outcome in a real single-arm lung cancer trial, a range of possible estimands was defined. We focused on the choice of the variable of interest and strategies to deal with intercurrent events (death, treatment discontinuation and disease progression). Statistical methods were described for each estimand and the corresponding results on the trial data were shown. Results Each intercurrent event handling strategy resulted in its own estimated mean global quality of life over time, with a specific interpretation, suitable for a corresponding clinical research aim. In the setting of this case study, a ‘while alive’ strategy for death and a ‘treatment policy’ strategy for non-terminal intercurrent events were deemed aligned with a descriptive research aim to inform clinicians and patients about expected quality of life after the start of treatment. Conclusions 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. |
| Druh dokumentu: | Article Other literature type |
| Popis súboru: | application/pdf; pdf |
| Jazyk: | English |
| ISSN: | 1471-2288 |
| DOI: | 10.1186/s12874-024-02408-x |
| Prístupová URL adresa: | https://pubmed.ncbi.nlm.nih.gov/39580440 https://doaj.org/article/0d129c75262d4b7fb9e5a10fb3adf508 https://hdl.handle.net/1887/4239196 https://pure.amsterdamumc.nl/en/publications/cafc698d-c972-486d-92ce-50bba0b73b9a https://doi.org/10.1186/s12874-024-02408-x https://repository.publisso.de/resource/frl:6494732 https://biblio.ugent.be/publication/01JMFJ6BBMAYW704GQ811YQTE4/file/01JMFJHBADN5ET4KE139X5B4WQ http://hdl.handle.net/1854/LU-01JMFJ6BBMAYW704GQ811YQTE4 https://biblio.ugent.be/publication/01JMFJ6BBMAYW704GQ811YQTE4 http://doi.org/10.1186/s12874-024-02408-x |
| Rights: | CC BY |
| Prístupové číslo: | edsair.doi.dedup.....323518ac672b481294b1c6ee718a3dcd |
| Databáza: | OpenAIRE |
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Nájsť tento článok vo Web of Science | Abstrakt: | Background Patient-reported outcomes (PROs) play an increasing role in the evaluation of oncology treatments. At the same time, single-arm trials are commonly included in regulatory approval submissions. Because of the high risk of biases, results from single-arm trials require careful interpretation. This benefits from a clearly defined estimand, or target of estimation. In this case study, we demonstrated how the ICH E9 (R1) estimand framework can be implemented in SATs with PRO endpoints. Methods For the global quality of life outcome in a real single-arm lung cancer trial, a range of possible estimands was defined. We focused on the choice of the variable of interest and strategies to deal with intercurrent events (death, treatment discontinuation and disease progression). Statistical methods were described for each estimand and the corresponding results on the trial data were shown. Results Each intercurrent event handling strategy resulted in its own estimated mean global quality of life over time, with a specific interpretation, suitable for a corresponding clinical research aim. In the setting of this case study, a ‘while alive’ strategy for death and a ‘treatment policy’ strategy for non-terminal intercurrent events were deemed aligned with a descriptive research aim to inform clinicians and patients about expected quality of life after the start of treatment. Conclusions 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. |
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| ISSN: | 14712288 |
| DOI: | 10.1186/s12874-024-02408-x |