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1. Formulating causal questions and principled statistical answers
1. Formulating causal questions and principled statistical answers
Goetghebeur E, le Cessie S, De Stavola B, Moodie EE, Waernbaum I. Formulating causal questions and principled statistical answers. Statistics in Medicine. 2020;39:4922–4948. https://doi.org/10.1002/sim.8741
2. Single-arm studies involving patient-reported outcome data in oncology: a literature review on current practice
2. Single-arm studies involving patient-reported outcome data in oncology: a literature review on current practice
Liu L, Choi J, Musoro JZ, et al. Single-arm studies involving patient-reported outcome data in oncology: a literature review on current practice. Lancet Oncol. 2023;24(5):e197-e206. doi:10.1016/S1470-2045(23)00110-9
3. The Role of the Estimand Framework in the Analysis of Patient-Reported Outcomes in Single-Arm Trials
3. The Role of the Estimand Framework in the Analysis of Patient-Reported Outcomes in Single-Arm Trials
Thomassen, D., Roychoudhury S., Delphin Amdal C., Reynders D., Musoro J. , Sauerbrei W., Goetghebeur E., and le Cessie S. and on behalf of SISAQOLIMI Work Package 3 “The Role of the Estimand Framework in the Analysis of Patient-Reported Outcomes in Single-Arm Trials.” BMC Medical Research Methodology (2024) 24:290 https://doi.org/10.1186/s12874-024-02408-x
4. Handling missing values in patient-reported outcome data in the presence of intercurrent events
4. Handling missing values in patient-reported outcome data in the presence of intercurrent events
Thomassen, D., Roychoudhury S., Delphin Amdal C., Reynders D., Musoro J. , Sauerbrei W., Goetghebeur E., and le Cessie S. and on behalf of SISAQOLIMI Work Package 3. ``Handling missing values in patient-reported outcome data in the presence of intercurrent events". BMC Medical Research Methodology (2025) 25:56 https://doi.org/10.1186/s12874-025-02510-8
5. Evaluating treatment effects on longitudinal outcomes with attrition due to death: Methods for While-Alive estimands with a case study in Quality of Life
5. Evaluating treatment effects on longitudinal outcomes with attrition due to death: Methods for While-Alive estimands with a case study in Quality of Life
Reynders D., Thomassen D., Roychoudhury S., Delphin Amdal C., Musoro J., Sauerbrei W., le Cessie S. and Goetghebeur E. and on behalf of SISAQOLIMI Work Package 3. ``Evaluating treatment effects on longitudinal outcomes with attrition due to death: Methods for While-Alive estimands with a case study in Quality of Life." arXiv:2509.25548
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