An exciting two day workshop on Clinical Trials in Small Populations took place on the 30th November and 1st December 2015 at the Royal Statistical Society in London. The event brought together researchers and practitioners to discuss state-of-the-art methods for trials in small populations. Day 1 involved a groundwork interactive training workshop involving discussion sessions motivated by case studies of real trials and tutorials on methods for small trials. Day 2 comprised of invited talks from medical statisticians from industry and academia, patient representatives, and regulators, including representatives from the three projects recently funded by the EU FP7 programme to develop innovative methods for clinical research in small populations.
Clinical trial design is an increasingly important area of research that the MRC Biostatistics Unit (BSU) are playing a vital role in. Clinical trials give the best evidence about the benefits and harms of drugs and other health interventions. Clinical trials to study new drugs and treatments for rare diseases pose several methodological, practical and ethical challenges. This workshop had a two-fold goal: to train practitioners in the novel methods specifically developed for trials in small populations and to discuss what further research is needed based on the current experience of regulators, practitioners and patients.
Sofia Villar who works in the ‘Design and Analysis of Randomised Trials’ theme at BSU, led by Adrian Mander, played an active part in organising this highly successful event funded by the MRC Network of Hubs for Trials Methodology Research. Sofia was a co-organiser of the event, she gave a tutorial on `Response-adaptive randomisation’ during Day 1 and also contributed to one of the talks during Day 2 entitled ‘Bringing patient population size into clinical trial design using response-adaptive randomisation’. The workshop project was led by Lisa Hampson (Lancaster University) and further co-organised by Tim Morris and Matt Sydes (MRC CTU at UCL), Prof Nigel Stallard (University of Warwick), and Catrin Tudur-Smith (University of Liverpool).
Sofia found the event an exciting and valuable experience, saying: “Traditional trial designs require recruiting large numbers of patients. This is simply not possible for ‘rare diseases’, in which the prevalence of a disease is low. Even if the number of patients of each such disease is small, the overall number of patients suffering from rare diseases is on the rise. As a young researcher it was a very enriching experience to organise and be part of an event that brought together everyone involved in the process of developing the alternative approaches that are needed to give these patients access to new effective medicines.”
For further information on this event, please click here.