Publications of the group


December 7, 2023

Here we list the publications that the group has written or contributed to.

Accepted or published

  1. Lawrence, R., Degtyarev, E., Griffiths, P., Trask, P., Lau, H., D’Alessio, D., Griebsch, I., Wallenstein, G., Cocks, K., Rufibach, K. What is an estimand & how does it relate to quantifying the effect of treatment on patient-reported quality of life outcomes in clinical trials (2020). Journal of Patient-Reported Outcomes, 4(1), 68. | doi |
  2. Degtyarev, E., Rufibach, K., Shentu, Y., Yung, G., Casey, M., Englert, S., Liu, F., Liu, Y., Sailer, O., Siegel, J., Sun, S., Tang, R., Zhou, J. Assessing the impact of COVID-19 on the objective and analysis of oncology clinical trials – application of the estimand framework (2020). Statistics in Biopharmaceutical Research, 12(4), 427-437. | doi | arxiv |.
  3. Casey M., Degtyarev E., Lechuga M.J., Aimone P., Ravaud A., Motzer R., Liu F., Stalbovskaya V., Tang R., Butler E., Sailer O., Halabi S., George D. Estimand framework: Are we asking the right question? A case study in the solid tumor setting (2020). Pharmaceutical Statistics, 20, 324–334. | doi |
  4. Sun, S., Weber, J., Butler, E., Rufibach, K., Roychoudhury, S. Estimands in Hematology Trials (2021). Pharmaceutical Statistics, 20, 793-805. | doi | arxiv |. This paper was the 3rd most downloaded paper that appeared in 2021 in Pharmaceutical Statistics.
  5. Bornkamp, B., Rufibach, K., Lin, J., Liu, Y., Mehrotra, D., Roychoudhury, S., Schmidli, H., Shentu, Y., Wolbers, M. Principal Stratum Strategy: Potential Role in Drug Development (2021). Pharmaceutical Statistics, 20, 737-751. | doi | arxiv | github | markdown | This paper was the 4th most downloaded paper that appeared in 2021 in Pharmaceutical Statistics.
  6. Manitz, J., Kan-Dobrosky, N., Buchner, H., Casadebaig, M.L., Degtyarev, E., Dey, J., Haddad, V., Fei, J., Martin, E., Mo, M., Rufibach, K., Shentu, Y., Stalbovskaya, V., Tang, R., Yung, G., Zhu, J. Estimands for Overall Survival in Clinical Trials with Treatment Switching in Oncology (2022). Pharmaceutical Statistics, 21, 150-162. doi. This paper was among the most downloaded papers in the first twelve months following publication among those papers that appeared in 2021 in Pharmaceutical Statistics.
  7. Hampson, L.V., Degtyarev, E., Tang, R., Lin, J., Rufibach, K., Zheng, C. Comment on Biostatistical considerations when using RWD and RWE in clinical studies for regulatory purposes: A landscape assessment (2023). Statistics in Biopharmaceutical Research, 15(1), 23–26. doi.
  8. Rajeshwari, S., Barksdale, E., Marchenko, O., Jiang, Q., Ando, Y., Bloomquist, E., Coory, M., Crouse, M., Degtyarev, E., Framke, T., Freidlin, B., Gerber, D.E., Gwise, T., Josephson, F., Hess, L., Kluetz, P., Li, D., Mandrekar, S., Posch, M., Rantell, K., Ratitch, B., Raven, A., Roes, K., Rufibach, K., Sarac, S.B., Simon, R., Singh, H., Theoret, M., Thomson, A., Zuber, E., Shen, Y.L., Pazdur, R. Cancer Clinical Trials Beyond Pandemic: Report of an American Statistical Association Biopharmaceutical Section Open Forum Discussion (2023). Statistics in Biopharmaceutical Research, 15(2), 444–449. doi.
  9. Rufibach, K., Grinsted, L., Li, J., Weber, H.J., Zheng, C., and Zhou, J. Quantification of Follow-up Time in Oncology Clinical Trials with a Time-to-Event Endpoint: Asking the Right Questions. (2023). Pharmaceutical Statistics, to appear. | doi | arXiv | github | markdown |.
  10. Liu, Y., Yang, M., Kil, S., Li, J., Mondal, S., Shentu, Y., Tian, H., Wang, L., Yung, G. From logic-respecting efficacy estimands to logic-ensuring analysis principle for time-to-event endpoint in randomized clinical trials with subgroups. (2023). Statistics in Biopharmaceutical Research, 15(3), 560–573. doi.
  11. Englert, S., Mercier, F., Pilling, E. A., Homer, V., Habermehl, C., Kan-Dobrosky, N. Defining estimands for efficacy assessment in single arm Phase 1b or Phase 2 clinical trials in oncology early development. (2023+). Pharmaceutical Statistics, to appear. | doi |
  12. Weber, H. J., Corson, S., Li, J., Mercier, F., Roychoudhury, S., Sailer, M. O., Sun, S., Todd, A., Yung, G. Duration of and time to response in oncology clinical trials from the perspective of the estimand framework. (2023+). Pharmaceutical Statistics, to appear. | arXiv | doi | github | markdown |
  13. Lawrance, R., Skaltsa, K., Regnault, A., Floden, L. Reflections on estimands for patient-reported outcomes in cancer clinical trials. (2023+). Journal of Biopharmaceutical Statistics, to appear. | doi |


  • Siegel, J.M. Weber, J., Englert, S. The Role of Occlusion: Potential Extension of the ICH E9 (R1) Addendum on Estimands and Sensitivity Analysis for Time-to-Event Oncology Studies (2022). Submitted. | arxiv |.
  • Mercier, F., Homer, V., Geng, J., Zhang, H., Englert, S., Kan-Dobrosky, N. Estimands in oncology early clinical development: Assessing the impact of intercurrent events on the dose-toxicity relationship. (2022). Submitted.
  • Siegel, J.M., Weber, H.J., Englert, S., Liu, F. Time-to-event estimands and loss to follow-up in oncology in light of the estimands framework (2023). Submitted. | arxiv |.