Innovative methods for clinical trials

• Sabine Braat

  Associate Professor

  s.braat@unimelb.edu.au
  

Project Details

Members of the Biostatistics Unit have led the design, conduct, analysis, and reporting of innovative clinical trial designs, including highly adaptive platform trial designs (e.g., SNAP (https://www.snaptrial.com.au/)) and sequential multiple assignment randomized trial (SMART) designs. Furthermore, our biostatisticians collaborate closely with clinical investigators to provide tailored methodological rigour to national and international clinical trials for example via state-of-the-art analysis approaches to handle missing data.

Our related biostatistical research is linked with the main research streams of the Australian Trials Methodology (AusTriM) Research Network (https://www.monash.edu/medicine/sphpm/austrim/home), which is an NHMRC Centre of Research Excellence of which several of our biostatisticians are associate investigators and early to mid-career researchers. Our main research themes focus on adaptive platform trials, dealing with missing data during follow-up of participants in trials, and the estimand framework.

Researchers

Associate Professor Sabine Braat

Dr Anurika De Silva

Associate Professor Karen Lamb

Ms Peixuan Li

Dr Rob Mahar

Dr Alistair McLean

Ms Fiona McManus

Professor Julie Simpson

Graduate Researchers

Gethmini Siriwardena

Jessica Xu

Collaborators

We collaborate on numerous national and international clinical trials, including trials led by researchers at The University of Melbourne, Murdoch Children’s Research Institute (https://www.mcri.edu.au/), and partners of the Melbourne Centre for Clinical Trials (https://mdhs.unimelb.edu.au/mcct).

Research Opportunities

This research project is available to PhD students, Masters by Research, Honours students, Master of Biomedical Science, Post Doctor Researchers to join as part of their thesis.
Please contact the Research Group Leader to discuss your options.

Research Publications

Mahar RK, McGlothlin A, Dymock M, Barina L, Bonten M, Bowen A, Cheng MP, Daneman N, Goodman AL, Lee TC, Lewis RJ. Statistical documentation for multi-disease, multi-domain platform trials: our experience with the Staphylococcus aureus Network Adaptive Platform trial. Trials. 2025 Feb 11;26(1):49.

Mahar RK, McGlothlin A, Dymock M, Lee TC, Lewis RJ, Lumley T, Mora J, Price DJ, Saville BR, Snelling T, Turner R. A blueprint for a multi-disease, multi-domain Bayesian adaptive platform trial incorporating adult and paediatric subgroups: the Staphylococcus aureus Network Adaptive Platform trial. Trials. 2023 Dec 6;24(1):795.

Mahar RK, McGlothlin A, Dymock M, Barina L, Bonten M, Bowen A, Cheng MP, Daneman N, Goodman AL, Lee TC, Lewis RJ, Lumley T, McLean ARD, McQuilten Z, Mora J, Paterson DL, Price DJ, Roberts J, Snelling T, Tverring J, Webb SA, Yahav D, Davis JS, Tong SYC, Marsh JA; SNAP Global Trial Steering Committee. Statistical documentation for multi-disease, multi-domain platform trials: our experience with the Staphylococcus aureus Network Adaptive Platform trial. Trials. 2025 Feb 11;26(1):49.

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. BMC Medical Research Methodology. 2025 Jul 1;25(1):162.

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. 2021 Feb;63(2):354-71.

De Silva AP, Leslie K, Braat S, Grobler AC. Application of the Estimand Framework to Anesthesia Trials. Anesthesiology. 2024 Jun 6;141(1):13-23.

Selman CJ, Lee KJ, Ferguson KN, Whitehead CL, Manley BJ, Mahar RK. Statistical analyses of ordinal outcomes in randomised controlled trials: a scoping review. Trials. 2024 Apr 6;25(1):241.

Hong W, McLachlan SA, Moore M, Mahar RK. Improving clinical trials using Bayesian adaptive designs: a breast cancer example. BMC Med Res Methodol. 2022 May 4;22(1):133.