Investigator methodological publications

Selected methodological publications from Chief and Associate Investigators.

For full publication listings please see listings given on researcher profiles.


Bowden R, Forbes AB, Kasza J. On the centrosymmetry of treatment effect estimators for stepped wedge and related cluster randomized trial designs. Statistics & Probability Letters. 2021. doi: 10.1016/j.spl.2020.109022.

Brilleman SL, Wolfe R, Moreno-Betancur M, Crowther MJ. Simulating survival data using the simsurv R package. Journal of Statistical Software. 97(3):1-27;2021. doi: 10.18637/jss.v097.i03.

Kasza J, Bowden R, Forbes AB. Information content of stepped wedge designs with unequal cluster‐period sizes in linear mixed models: Informing incomplete designs. Statistics in Medicine. 2021; 1-16. doi: 10.1002/sim.8867.

Marschner IC. A general framework for the analysis of adaptive experiments. Statistical Science. 2021; 36: 465-492. doi: 10.1214/20-STS803.


Brilleman SL, Elci EM, Buros Novik J, Wolfe R. Bayesian survival analysis using the rstanarm R package. arXiv:2002.09633. [stat.CO]. Published online 22 February 2020.

Dansie K, Viecelli AK, Pascoe EM, Johnson DW, McDonald S, Clayton P, Hawley C. Novel trial strategies to enhance the relevance, efficiency, effectiveness, and impact of nephrology research. Kidney International. 2020;98 (3) 572-578. doi: 10.1016/j.kint.2020.04.050.

Denholm JT, Davis J, Paterson D, Roberts J, Morpeth S, Snelling T, Zentner D, Rees M, O’Sullivan M, Price D, Bowen A, Tong SYC. The Australasian COVID-19 Trial (ASCOT) to assess clinical outcomes in hospitalised patients with SARS-CoV-2 infection (COVID-19) treated with lopinavir/ritonavir and/or hydroxychloroquine compared to standard of care: A structured summary of a study protocol for a randomised controlled trial. Trials. 2020: 21 (646). doi: 10.1186/s13063-020-04576-9.

De Silva AP, De Livera AM, Lee KJ, Moreno‐Betancur M, Simpson JA. Multiple imputation methods for handling missing values in longitudinal studies with sampling weights: Comparison of methods implemented in Stata. Biometrical Journal. 2020; 1–18. doi: 10.1002/bimj.201900360.

Gebski V, Byth K, Asher R, Marschner I. Recurrent time-to-event models with ordinal outcomes. Pharmaceutical Statistics. 2021;20(1):77-92. doi: 10.1002/pst.2057. Epub 2020 Oct 2.

Hemming K, Hughes JP, McKenzie JE, Forbes AB. Extending the I-squared statistic to describe treatment effect heterogeneity in cluster, multi-centre randomized trials and individual patient data meta-analysis. Statistical Methods in Medical Research. September 2020. doi: 10.1177/0962280220948550.

Hooper R, Kasza J, Forbes AB. The hunt for efficient, incomplete designs for stepped wedge trials with continuous recruitment and continuous outcome measures. BMC Medical Research Methodology. 2020; 20:279. doi: 10.1186/s12874-020-01155-z.

Hemming K, Kasza J, Hooper R, Forbes AB, Taljaard M. A tutorial on sample size calculation for multiple-period cluster randomised parallel, cross-over and stepped-wedge trials using the Shiny CRT Calculator. International Journal of Epidemiology, 2020. doi: 10.1093/ije/dyz237.

Huque M, Moreno-Betancur M, Quartagno M, Simpson J, Carlin J, Lee K. Multiple imputation methods for handling incomplete longitudinal and clustered data where the target analysis is a linear mixed effects model. Biometrical Journal. 2020;62(2). doi: 10.1002/bimj.201900051.

Hemming K, Taljaard M, Weijer C, Forbes AB. Use of the multiple period, cluster randomised, crossover trial designs for comparative effectiveness research. British Medical Journal. 2020, 371:m3800. doi: 10.1136/bmj.m3800.

Jones MA, Graves G, Middleton B, Totterdell J, Snelling T, Marsh J. The ORVAC Trial: a phase IV, double-blind, randomised, placebo-controlled clinical trial of a third scheduled dose of Rotarix rotavirus vaccine in Australian Indigenous infants to improve protection against gastroenteritis: a Statistical Analysis Plan. Trials. 2020. 21 (741). doi: 10.1186/s13063-020-04602-w.

Kasza J, Hooper R, Copas A, Forbes AB. Sample size and power calculations for open cohort longitudinal cluster randomized trials. Statistics in Medicine. 2020; 39: 1871–1883. doi: 10.1002/sim.8519.

Marschner IC, Askie LM, Schou IM. Sensitivity analyses assessing the impact of early stopping on systematic reviews: Recommendations for interpreting guidelines. Res Syn Meth. 2020; 11: 287-300. doi: 10.1002/jrsm.1394.

McGuinness M, Kasza J, Karahalios A, Guymer R, Finger R, Simpson J. A comparison of methods to estimate the survivor average causal effect in the presence of missing data: a simulation study (vol 19, 223, 2019). BMC Medical Research Methodology. 2020;20(1). doi: 10.1186/s12874-020-00935-x.

McLeod C, Marsh J, Tong A, Blyth C, Norman R, Webb S, Snelling T. A clinical researcher’s guide for estimands: how they relate to outcomes, endpoints and measures of treatment effect in clinical trials. Archives of Clinical Research and Trials. 2020; 1(1): 106. doi: 10.36879/ACRT.20.000106.

Perez Chacon G, Estcourt M, Totterdell J, Campbell DE, Perrett KP, Marsh JA, Richmond P, Wood N, Gold MS, Holt PG, Waddington C, Snelling TL. The OPTIMUM Study Protocol: an adaptive randomised controlled trial of a mixed whole-cell/acellular pertussis vaccine schedule. BMJ Open. 2020 Dec 17;10(12):e042838. doi: 10.1136/bmjopen-2020-042838.

Sullivan TR, Latimer NR, Gray J, Sorich MJ, Salter AB, Karnon J. Adjusting for Treatment Switching in Oncology Trials: A Systematic Review and Recommendations for Reporting. Value Health. 2020 Mar;23(3):388-396. doi: 10.1016/j.jval.2019.10.015. Epub 2020 Jan 23. PMID: 32197735.

Thompson J, Hemming K, Forbes A, Fielding K, Hayes R. Comparison of small-sample standard-error corrections for generalised estimating equations in stepped wedge cluster randomised trials with a binary outcome: A simulation study. Statistical Methods in Medical Research. September 2020. doi: 10.1177/0962280220958735.

Turner S, Karahalios E, Forbes AB, Taljaard M, Grimshaw J, Cheng A, Bero L, McKenzie J. Design characteristics and statistical methods used in interrupted time series studies evaluating public health interventions: A review. Journal of Clinical Epidemiology. 2020 Jun;122:1-11. doi: 10.1016/j.jclinepi.2020.02.006.

Wijesuriya R, Moreno-Betancur M, Carlin JB, Lee KJ. Evaluation of approaches for multiple imputation of three-level data. BMC Research Methodology. 20(1); 2020. doi: 10.1186/s12874-020-01079-8.

Young PJ, Bagshaw SM, Forbes AB, Nichol AD, Wright SE, Bellomo R, et al. Opportunities and challenges of clustering, crossing over, and using registry data in the PEPTIC trial. Critical Care & Resuscitation. 2020 Jun;22(2):105-109.


De Silva AP, Moreno-Betancur M, De Livera AM, Lee KJ, Simpson JA. Multiple imputation methods for handling missing values in a longitudinal categorical variable with restrictions on transitions over time: a simulation study. BMC Medical Research Methodology. 2019 Jan 10;19(1):14. doi: 10.1186/s12874-018-0653-0.

Grantham K, Forbes AB, Heritier S, Kasza J. Time Parameterizations in Cluster Randomized Trial Planning. The American Statistician, 2020, 74:2, 184-189. doi: 10.1080/00031305.2019.1623072. Epub 2019 May 28.

Grantham K, Kasza J, Heritier S, Hemming K, Forbes AB. Accounting for a decaying correlation structure in cluster randomised trials with continuous recruitment. Statistics in Medicine. 2019, 38, 1918-1934. doi: 10.1002/sim.8089.

Grantham K, Kasza J, Heritier S, Hemming K, Litton E, Forbes AB. How many times should a cluster randomised crossover trial cross over? Statistics in Medicine. 2019 Nov;38(25):5021-5033. doi: 10.1002/sim.8349.

Hemming, K, Carroll K, Thompson J, Forbes AB, Taljaard M, Jacobsen P. Quality of stepped-wedge trial reporting can be reliably assessed using an updated CONSORT: crowd-sourcing systematic review. Journal of Clinical Epidemiology. 2019, 107, 77-88. doi: 10.1016/j.jclinepi.2018.11.017.

Jachno K, Heritier S, Wolfe R. Are non-constant rates and non-proportional treatment effects accounted for in the design and analysis of randomised controlled trials? A review of current practice. BMC Medical Research Methodology. 2019 May 16;19(1):103. doi: 10.1186/s12874-019-0749-1.

Kasza J, Forbes AB. Inference for the treatment effect in multiple-period cluster randomised trials when random effect correlation structure is misspecified. Statistical Methods in Medical Research. 2019;28(10-11):3112-3122. doi: 10.1177/0962280218797151.

Kasza J, Forbes AB. Information content of cluster-period cells in stepped wedge trials. Biometrics. 2019 Mar;75(1):144-152. doi: 10.1111/biom.12959.

Kasza J, Hemming K, Hooper R, Matthews J, Forbes AB. Impact of non-uniform correlation structure on sample size and power in multiple-period cluster randomised trials. Statistical Methods in Medical Research. 2019 Mar;28(3):703-716. doi: 10.1177/0962280217734981.

Lee KJ, Moreno-Betancur M, Kasza J, Marschner IC, Barnett AG, Carlin JB. Biostatistics: a fundamental discipline at the core of modern health data science. Medical Journal of Australia. 2019 Nov;211(10):444-446.e1.  doi: 10.5694/mja2.50372.

Li F, Forbes AB, Turner EL, Preisser JS. Power and sample size requirements for GEE analyses of cluster randomized crossover trials. Statistics in Medicine. 2019 Feb 20;38(4):636-649. doi: 10.1002/sim.7995.

Marschner IC, Schou IM. Underestimation of treatment effects in sequentially monitored clinical trials that did not stop early for benefit. Statistical Methods in Medical Research. 2019; 28: 3027-3041. doi: 10.1177/0962280218795320.

Palamuthusingam D, Johnson DW, Hawley C, Pascoe E, Fahim M. Health data linkage research in Australia remains challenging. Internal Medicine Journal. 2019 Apr;49(4):539-544. doi: 10.1111/imj.14244.

Thao LTP, Geskus R. A comparison of model selection methods for prediction in the presence of multiply imputed data. Biometrical Journal. 2019;61:343–356. doi: 10.1002/bimj.201700232.