Publications of the group


July 24, 2023

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

Accepted or published

  • 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 |
  • 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 |.
  • 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 |
  • 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.
  • 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.
  • 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.
  • 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 (2022). Statistics in Biopharmaceutical Research, accepted. doi.
  • 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.
  • 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 |.
  • 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, accepted. doi.
  • 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 |


  • 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.
  • 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). Submitted. | arXiv | github | markdown |
  • 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 |.