Investigator methodological publications

Selected methodological publications from Chief and Associate Investigators.

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

Preprints and works Accepted/In press

Xu J, De Silva AP, Lee KJ, Mahar RK, Simpson JA. Optimising dynamic treatment regimens using sequential multiple assignment randomised trials data with missing data. 2024, PREPRINT (Version 1) available at Research Square.

2025

Lee KJ, Middleton M, Mahar RK. Review article: Primer for clinical researchers on innovative trial designs for emergency medicine. Emergency Medicine Australasia. 37(14532):2025.

McKenzie JE, Taljaard M, Hemming K, Arnup SJ, Giraudeau B, Eldridge S, Kahan BC, Li T, Moher D, Turner EL, Grimshaw JM, Forbes AB. Reporting of cluster randomised crossover trials: extension of the CONSORT 2010 statement with explanation and elaboration. BMJ; 388 :e080472. 2025.

2024

Caille A, Billot L, Kasza J. Practical and methodological challenges when conducting a cluster randomized trial: examples and recommendations. Journal of Epidemiology and Population Health. 72:202199.: 2024.

Descallar J, Ma J, Zhu H, Heritier S, Wolfe R. Cause-specific hazard Cox models with partly interval censoring – Penalized likelihood estimation using Gaussian quadrature. Statistical Methods in Medical Research. 0:0 2024.

Grantham KL, Forbes AB, Hooper R, Kasza J. The staircase cluster randomised trial design: a pragmatic alternative to the stepped wedge. Statistical Methods in Medical Research. 33:24-41.: 2024.

Hemming K, Copas A, Forbes AB, Kasza J. What type of cluster randomized trial for which setting? Journal of Epidemiology and Population Health. 72:202195: 2024.

Hooper R, Quintin O, Kasza J. Efficient designs for three-sequence stepped wedge trials with continuous recruitment. Clinical Trials. 0:0 2024.

Kanukula R, McKenzie JE, Bero L, Dai Z, McDonald S, Kroeger CM, Korevaar E, Forbes AB, Page MJ. Investigation of bias due to selective inclusion of study effect estimates in meta-analyses of nutrition research. Research Synthesis Methods; 15(4): 524-542. 2024.

Korevaar E, Turner SL, Forbes AB, Karahalios A, Taljaard M, McKenzie JE. Comparison of statistical methods used to meta-analyse results from interrupted time series studies: an empirical study. BMC Medical Research Methodology. 24, 31: 2024.

Long E, Lee KJ, Davidson A, Babl F. Adaptive platform trials rather than randomised controlled trials for paediatric sepsis. Emergency Medicine Australasia. 36(3); 488-490: 2024.

Marschner IC, Schou IM. Analysis of Nonconcurrent Controls in Adaptive Platform Trials: Separating Randomized and Nonrandomized Information. Biometrical Journal. 66: e202300334:2024.

Ryan EG, Gao CX, Grantham KL, Thao LTP, Charles-Nelson A, Bowden R, Herschtal A, Lee KJ, Forbes AB, Heritier S, Phillipou A, Wolfe R. Advancing randomized controlled trial methodologies: The place of innovative trial design in eating disorders research. International Journal of Eating Disorders. 57(6):1337-1349: 2024.

Selman C, Lee KJ and Mahar R. Statistical analyses of ordinal outcomes in randomised controlled trials: a scoping review. Trials. 25:241 2024.

Soon YY, Marschner IC, Schou M, Sweeney CJ, Davis ID, Stockler MR, Martin AJ. Challenges of estimating treatment effects after a positive interim analysis. Eur J Cancer. 209:114230:2024.

Thao LTP, Wolfe R, Heritier S, Geskus R. Handling missing disease information in diseases that need two visits to diagnose. Statistics in Medicine. 43(9):1708–1725: 2024.

2023

Hinman RS, Kasza J. Research note: Non-inferiority trials. Journal of Physiotherapy. 69:129-132 2023.

Kasza J, Bowden R, Ouyang Y, Taljaard M, Forbes AB. Does it decay? Obtaining decaying correlation parameter values from previously analysed cluster randomised trials. Statistical Methods in Medical Research. 32(11):2123-2134. 2023.

Korevaar E, Turner SL, Forbes AB, Karahalios A, Taljaard M, McKenzie JE. Evaluation of statistical methods used to meta-analyse results from interrupted time series studies: A simulation study. Research Synthesis Methods. 14, 6:882-902 21 2023.

Lange KM, Kasza J, Sullivan TR, Yelland LN. Partially clustered designs for clinical trials: unifying existing designs using consistent terminology. Clinical Trials. 20:99-110. 2023.

Lee KJ, Carlin JB, Simpson JA, Moreno-Betancur M. Assumptions and analysis planning in studies with missing data in multiple variables: moving beyond the MCAR/MAR/MNAR classification. International Journal of Epidemiology. 8: 2023.

Li F, Kasza J, Turner EL, Rathouz PJ, Forbes AB, Preisser JS. Generalizing the information content for stepped wedge designs: A marginal modeling approach. Scandinavian Journal of Statistics. 3:1048-1067 2023.

Rezaei-Darzi E, Grantham K, Forbes AB, Kasza J. The impact of iterative removal of low-information cluster-period cells from a stepped wedge design. BMC Medical Research Methodology. 23:160 2023.

Selman CJ, Lee KJ, Whitehead CL, Manley BJ, Mahar RK. Statistical analyses of ordinal outcomes in randomised controlled trials: protocol for a scoping review. Trials. 24(1): 7: 2023.

Teerenstra S, Kasza J, Leontjevas R, Forbes AB. Sample size for partially nested designs and other nested or crossed designs with a continuous outcome when adjusted for baseline. Statistics in Medicine. 42(19):3568–3592. 2023.

Turner SL, Korevaar E, Cumpston MS, Kanukula R, Forbes AB, McKenzie JE. Effect estimates can be accurately calculated with data digitally extracted from interrupted time series graphs. Research Synthesis Methods. 14, 4:622-638 17 2023.

Yelland LN, Louise J, Kahan BC, Morris TP, Lee KJ, Sullivan TR. Handling misclassified stratification variables in the analysis of randomised trials with continuous outcomes. Statistics in Medicine. 42(19):3529-3546 2023.

2022

Braat S, Lee KJ. Left in the dark: the importance of publicly available clinical trial protocols. Medical Journal of Australia. 217(10): 519-519: 2022.

Falconer N, Paterson DL, Peel N, Welch A, Freeman C, Burkett E, Hubbard R, Comans T, Hanjani LS, Pascoe EM, Hawley CM and Gray L. A multimodal intervention to optimise antimicrobial use in residential aged care facilities (ENGAGEMENT): protocol for a stepped-wedge cluster randomised trial. Trials. 2022; 23(1): 427. doi: 10.1186/s13063-022-06323-8.

Felmingham C, MacNamara S, Cranwell W, Williams N, Wada M, Adler N, Ge Z, Sharfe A, Bowling A, Haskett M, Wolfe R, Mar V. Improving Skin cancer Management with ARTificial Intelligence (SMARTI): protocol for a pre-post intervention trial of an Artificial Intelligence system used as a diagnostic aid for skin cancer management in a specialist dermatology setting. BMJ Open. 2022;12:e050203. doi:10.1136/bmjopen-2021-050203.

Grantham KL, Kasza J, Heritier S, Carlin JB, Forbes AB. Evaluating the performance of Bayesian and restricted maximum likelihood estimation for stepped wedge cluster randomized trials with a small number of clusters. BMC Med Res Methodol. 2022;22:112. doi:10.1186/s12874-022-01550-8.

Grobler AC, Lee KJ, Wong A, Currow DC, Braat S. Handling missing data and drop out in hospice/palliative care trials through the estimand framework. Journal of Pain and Symptom Management. 2022; 63(4): e431-e439. doi: 10.1016/j.jpainsymman.2021.12.022.

Heller G, Robledo K, Marschner IM. Distributional regression in clinical trials: treatment effects on parameters other than the mean. BMC Medical Research Methodology. 2022; 22(1): 56-1-56-12. doi: 10.1186/s12874-022-01534-8.

Hong W, McLachlan SA, Moore M, Mahar RK. Improving clinical trials using Bayesian adaptive designs: a breast cancer example. BMC Medical Research Methodology. 22(1):133: 2022.

Jachno K, Heritier S, Woods RL, Mahady S, Chan AT, Tonkin A, Murray A, McNeil JJ, Wolfe R. Examining evidence of time-dependent treatment effects: an illustration using regression methods. Trials. 23:857 2022.

Kasza J, Bowden R, Hooper R, Forbes AB. The batched stepped wedge design: A design robust to delays in cluster recruitment. Statistics in Medicine. 41:3627–3641. 2022.

Korevaar E, Karahalios A, Turner SL, Forbes AB, Taljaard M, Cheng AC, Grimshaw JM, Bero L, McKenzie JE. Methodological systematic review recommends improvements to conduct and reporting when meta-analyzing interrupted time series studies. Journal of Clinical Epidemiology. 2022; 145: 55–69. doi: 10.1016/j.jclinepi.2022.01.010.

Li F, Kasza J, Turner EL, Rathouz PJ, Forbes AB, Preisser JS. Generalizing the information content for stepped wedge designs: a marginal modelling approach. Scandinavian Journal of Statistics. 1-20. 2022.

Lo S, Ma J, Manuguerra M, Moreno-Betancur M, Scolyer R, Thompson J. Competing risks analysis with missing cause-of-failure penalized likelihood estimation of cause-specific Cox models. Statistical Methods in Medical Research. 2022; 31(5): 978-994. doi: 10.1177/09622802211070254.

Lo S, Thompson J. Adaptive designs for clinical trials have potential advantages, but statistical challenges lurk! British Journal of Dermatology. 2022; 186(2): 205-206. doi: 10.1111/bjd.20828.

Mahar RK, Lee KJ, Chakraborty B, Salim A, Simpson JA. Making SMART decisions in prophylaxis and treatment studies. 2022 [Pre-print]

Mainzer RM, Nguyen CD, Carlin JB, Moreno-Betancur M, White IR, Lee KJ. A comparison of strategies for selecting auxiliary variables for multiple imputation. 2022 [Pre-print]

Marschner IC, Schou IM. Analysis of adaptive platform trials using a network approach. Clinical Trials. 2022; 19(5): 479-489. doi:10.1177/17407745221112001.

Neumann JT, Le TTP, Callender E, Carr PR, Qaderi V, Nelson MR, Reid CM, Woods RL, Orchard SG, Wolfe R, Polekhina G, Williamson JD, Trauer JM, Newman AB, Murray AM, Ernst ME, Tonkin AM, McNeil JJ. A multistate model of health transitions in older people: a secondary analysis of ASPREE clinical trial data. Lancet Healthy Longev. 2022;3(2):e89-e97. doi:10.1016/s2666-7568(21)00308-1.

Pascoe EM, Chadban SJ, Fahim MA, Hawley CM, Johnson DW, Collins MG. Statistical analysis plan for Better Evidence for Selecting Transplant Fluids (BEST-Fluids): a randomised controlled trial of the effect of intravenous fluid therapy with balanced crystalloid versus saline on the incidence of delayed graft function in deceased donor kidney transplantation. Trials. 2022; 23(1): 52. doi: 10.1186/s13063-021-05989-w.

Rezaei-Darzi E, Kasza J, Forbes AB, Bowden R. Use of information criteria for selecting a correlation structure for longitudinal cluster randomised trials. Clinical Trials. 2022; 19(3), 316-325. doi: 10.1177/17407745221082227.

Ryan EG, Couturier DL, Heritier S. Bayesian adaptive clinical trial designs for respiratory medicine. Respirology. 2022;27(10):834–843. doi:10.1111/resp.14337.

Wijesuriya R, Moreno‐Betancur M, Carlin JB, De Silva AP, Lee KJ. Multiple imputation approaches for handling incomplete three‐level data with time‐varying cluster‐memberships. Stat Med. 2022;1:18. doi:10.1002/sim.9515.

Woodford R, Zhou D, Kok P, Lord S, Friedlander M, Marschner IM, Simes R, Lee C. The validity of progression-free survival 2 as a surrogate trial end point for overall survival. Cancer. 2022; 128(7): 1449-1457. doi: 10.1002/cncr.34085.

Yan MK, Adler NR, Heriot N, Shang C, Zalcberg JR, Evans S, Wolfe R, Mar VJ. Opportunities and barriers for the use of Australian cancer registries as platforms for randomised clinical trials. Asia Pac J Clin Oncol. 2022;18(4):344-352. doi:10.1111/ajco.13670.

Yelland LN, Scurrah KJ, Ferreira P, Calais-Ferreira L, Rankin M, Denton J, Harvey M, Lee KJ, Kendal E and Craig JM. Conducting Clinical Trials in Twin Populations: A Review of Design, Analysis, Recruitment and Ethical Issues for Twin-Only Trials. Twin Research and Human Genetics. 24(6):359-364: 2022.

2021

Agius P, Simpson JA, Dini S, Gething P, Devine A, Fowkes F. Applying novel multi-disciplinary methodologies to accelerate malaria elimination. International Journal of Epidemiology. 2021; 50: 8. doi: 10.1093/ije/dyab168.014.

Bowden R, Forbes AB, Kasza J. Inference for the treatment effect in longitudinal cluster randomized trials when treatment effect heterogeneity is ignored. Statistical Methods in Medical Research. 2021; 30(11): 2503–2525. doi: 10.1177/09622802211041754.

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.

Chondros P, Ukoumunne OC, Gunn JM, Carlin JB. When should matching be used in the design of cluster randomized trials? Statistics in Medicine. 2021; 40(26): 5765-5778. doi: 10.1002/sim.9152.

Gebski V, Byth Wilson K, Asher R, Marschner I. Recurrent time-to-event models with ordinal outcomes. Pharmaceutical Statistics: the journal of applied statistics in the pharmaceutical industry. 2021; 20(1): 77-92. doi: 10.1002/pst.2057.

Gebski V, Marschner I, Asher, R, Byth Wilson K. Using recurrent time-to-event models with multinomial outcomes to generate toxicity profiles. Pharmaceutical Statistics: the journal of applied statistics in the pharmaceutical industry. 2021; 20(4): 840-849. doi: 10.1002/pst.2113.

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. 2021; 30(2): 376–395. doi: 10.1177/0962280220948550.

Hemming K, Taljaard M, Moerbeek M, Forbes AB. Contamination: How much can an individually randomized trial tolerate? Statistics in Medicine. 2021; 40: 10. doi: 10.1002/sim.8958.

Hockham C, Kotwal S, Wilcox A, Bassi A, McGree J, Pollock C, Burrell LM, Bathla N, Kunigari M, Rathore V, John M, Enmoore L, Jenkins C, Ritchie A, McLachlan A, Snelling T, Jones, M, Jha V, Jardine M. Protocol for the Controlled evaLuation of Angiotensin Receptor blockers for COVID-19 respIraTorY disease (CLARITY): a randomised controlled trial. Trials. 2021; 22(1): 1. doi: 10.1186/s13063-021-05521-0.

Jachno K, Heritier S, Wolfe R. Impact of a non-constant baseline hazard on detection of time-dependent treatment effects. A simulation study. BMC Medical Research Methodology. 2021; 21: 177. doi: 10.1186/s12874-021-01372-0.

Kasza J. Research Note: Estimating the complier average causal effect when participants in randomised trials depart from allocated treatment. Journal of physiotherapy. 2021; 67(2): 147–149. doi: 10.1016/j.jphys.2021.02.002.

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.

Kok P, Yoon W, Lord S, Marschner I, Friedlander M, Lee C. Tumor Response End Points as Surrogates for Overall Survival in Immune Checkpoint Inhibitor Trials: A Systematic Review and Meta-Analysis. JCO Precision Oncology. 2021; 5: 1151-1159. doi: 10.1200/PO.21.00108.

Korevaar E, Kasza J, Taljaard M, Hemming K, Haines T, Turner EL, Thompson JA, Hughes JP, Forbes AB. Intra-cluster correlations from the CLustered OUtcome Dataset bank to inform the design of longitudinal cluster trials. Clinical trials. 2021; 18(5): 529–540. doi: 10.1177/17407745211020852.

Litton E, Atkinson H, Anstey J, Anstey M, Campbell LT, Forbes AB, Hahn R, Hooper K, Kasza J, Knapp S, McGain F, Ngyuen N, Pilcher D, Reddi B, Reid C, Robinson S, Thompson K, Webb S, Young P. Optimising a targeted test reduction intervention for patients admitted to the intensive care unit: The Targeted Intensive Care Test Ordering Cluster Trial intervention. Aust Crit Care. 2021; 34(5): 419-426. doi: 10.1016/j.aucc.2020.11.003.

Ma J, Couturier D, Heritier S and Marschner IC. Penalized likelihood estimation of the proportional hazards model for survival data with interval censoring. The International Journal of Biostatistics. 2021, 000010151520200104. doi: 10.1515/ijb-2020-0104.

Mahar R, Mcguinness M, Chakraborty B, Carlin JB, Ijzerman M, Simpson JA. A scoping review of studies using observational data to optimise dynamic treatment regimens. BMC Medical Research Methodology. 2021; 21(1): 13. doi: 10.1186/s12874-021-01211-2.

Marschner IM. A General Framework for the Analysis of Adaptive Experiments. Statistical Science. 2021; 36(3): 465-492. doi: 10.1214/20-STS803.

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

Nguyen CD, Carlin JB, Lee KJ. Practical strategies for handling breakdown of multiple imputation procedures. Emerg Themes Epidemiol. 2021; 18: 5. doi: 10.1186/s12982-021-00095-3.

Pittet LF, Messina NL, Gardiner K, Orsini F, Abruzzo V, Bannister S, Bonten M, Campbell JL, Croda J, Dalcolmo M, et al. BCG vaccination to reduce the impact of COVID-19 in healthcare workers: Protocol for a randomised controlled trial (BRACE trial). BMJ Open. 2021; 11(10): e052101. doi: 10.1136/bmjopen-2021-052101.

Robledo K, Marschner I. A new algorithm for fitting semi-parametric variance regression models. Computational Statistics. 2021; 36(4): 2313-2335. doi: 10.1007/s00180-021-01067-6.

Sullivan TR, Yelland LN, Moreno‐Betancur M, Lee KJ. Multiple imputation for handling missing outcome data in randomized trials involving a mixture of independent and paired data. Statistics in Medicine. 2021; 40(27): 6008 -6020. doi: 10.1002/sim.9166.

Thompson JA, Hemming K, Forbes AB, 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. 2021; 30(2): 425–439. doi: 10.1177/0962280220958735.

Turner SL, Forbes AB, Karahalios A, Taljaard M, McKenzie JM. Evaluation of statistical methods used in the analysis of interrupted time series studies: a simulation study. BMC Medical Research Methodology. 2021; 21: 181. doi: 10.1186/s12874-021-01364-0.

Turner SL, Karahalios A, Forbes AB, Taljaard M, Grimshaw JM, Korevaar E, Cheng AC, Bero L, McKenzie JM. Creating effective interrupted time series graphs: Review and recommendations. Research Synthesis Methods. 2021; 12: 106-117. doi: 10.1002/jrsm.1435.

Turner SL, Karahalios A, Forbes AB, Taljaard M, Grimshaw JM, McKenzie JE. Comparison of six statistical methods for interrupted time series studies: empirical evaluation of 190 published series. BMC Medical Research Methodology. 2021;21: 134. doi: 10.1186/s12874-021-01306-w.

Turner, EL, Platt AC, Gallis JA, Tetreault L, Easter C, McKenzie JE, Nash S, Forbes AB, Hemming K, and Madurasinghe VW. Completeness of Reporting and Risks of Overstating Impact in Cluster Randomised Trials: a Systematic Review. Lancet Global Health. 2021; 9(8): e1163–e1168. doi: 10.1016/S2214-109X(21)00200-X.

Wijesuriya R, Moreno‐Betancur M, Carlin JB, De Silva AP, Lee KJ. Evaluation of approaches for accommodating interactions and non‐linear terms in multiple imputation of incomplete three‐level data. Biometrical Journal. 2021; 1-22. doi: 10.1002/bimj.202000343.

Yelland LN, Scurrah KJ, Ferreira P, Calais-Ferreira L, Rankin M, Denton J, Harvey M, Lee KJ, Kendal E, Craig JM. Conducting Clinical Trials in Twin Populations: A Review of Design, Analysis, Recruitment and Ethical Issues for Twin-Only Trials. Twin Research and Human Genetics. 2021; 24(6): 359-364. doi: 10.1017/thg.2021.52.

2020

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.

Grobler AC, Lee KJ. Intention-to-treat Analyses for Randomized Controlled Trials in Hospice/Palliative Care enhanced by principled methods to handle missing data. Journal of Pain and Symptom Management. 2020; 60(4): e28 -e29. doi: 10.1016/j.jpainsymman.2020.06.038.

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.

Mace A, Martin AC, Ramsey J, Totterdell J, Marsh JA, Snelling T. FeBRILe3 Project: protocol for a prospective pragmatic, multisite observational study and safety evaluation assessing Fever, Blood cultures and Readiness for discharge in Infants Less than 3 months old. BMJ Open. 2020; 10: e035992. doi: 10.1136/bmjopen-2019-035992.

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.

Ramsay J, Marsh J, Pedrana A, Andric N, Norman R, Cheng W, Webb S, Zeps N, Bellgard M, Graves T, Hellard M, Snelling T. A platform in the use of medicines to treat chronic hepatitis C (PLATINUM C): protocol for a prospective treatment registry of real-world outcomes for hepatitis C. BMC Infect Dis. 2020; 20: 802. doi: 10.1186/s12879-020-05531-4.

Snyders K, Cho D, Hong J, Lord S, Asher R, Marschner I, Lee C. Benchmarking single-arm studies against historical controls from non-small cell lung cancer trials - an empirical analysis of bias. Acta Oncologica. 2020; 59(1): 90-95. doi: 10.1080/0284186X.2019.1674452.

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.

2019

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.