Clinical trials are a crucial part of developing new treatments. However, their cost has raised considerably and so has the time it takes to bring a new treatment to market. This is partly caused by the fact that modern medicine is focusing on more challenging diseases, usually rare and life-threatening. Researchers in many fields are looking for possible ways of addressing these issues. A promising statistical approach is the use of “adaptive designs” where scheduled looks at the data are pre-planned while the trial is ongoing, and pre-specified changes to the trial can be made based on analyses of accumulating data.
Sofia Villar’s research focuses on developing statistically robust procedures to alter the randomisation probabilities of patients to treatments during a clinical trial in order to meet ethical and efficiency objectives. The methods in Sofia’s work aim at placing more patients on more promising treatments whilst maintaining the validity and integrity of the trial. A novel element of her work is the formal inclusion of patient outcomes during the trial as a design feature to be optimised. The tradeoffs between efficiency, estimation and ethical goals are formally studied in Sofia’s work.
Her research was awarded the first Biometrika fellowship in 2014. Currently, an ongoing collaboration of hers resulted in the first NIHR Career Development Fellowship awarded to a surgeon whose programme is trying to understand what part of the brain tumour surgeons should remove to improve local control, and what cannot be removed without impacting quality of life or cognitive function for patients with glioblastomas. A final outcome of this collaboration is to develop a novel clinical trial using Sofia’s work to test if the resulting clinical research can be used to deliver better treatment options to patients with these aggressive and incurable brain tumours.
Sofia Villar has been awarded 3 new grants to continue her research on designing adaptive trials, working with researchers from Cancer Research UK, Papworth Hospital, and University of Cambridge.