Clinical Trials in Small Populations (CTSP) was a two-day event, that brought together researchers and practitioners to discuss the state of the art of methods and perspectives for this very particular and challenging type of study. The meeting was held at the Royal Statistical Society, in London, on 30 November and 1st December 2015.
Each day was independent in terms of work dynamics and attendance; but complementary in terms of its contents. The first day was a workshop, organised in the following way: sessions where some participants delivered short presentations on real life trials (case studies) were followed by questions and answers from the audience. The case studies sessions were interspersed with tutorial sessions delivered by the organisers, focusing mainly on (1) Bayesian Methods (the most popular statistical approach to deal with issues like sample size calculation and randomisation of subjects in CTSP); and (2) Adaptive Clinical Trial Design (a relatively new methodological approach for CTSP, used to optimise time and resources).
The second day had a different dynamic and was organised more in a seminar/conference style, with guest speakers delivering talks on selected issues. Once again, the main focus was on Bayesian methods, as this is still being the "Statistical School" that provides the most commonly used methods for the development and conduction of CTSP.
The regulatory process that drugs for rare diseases need to go through was highlighted in a few talks, including a very passionate one given by Nick Catlin from Action Duchenne, as a barrier that is still avoiding more treatment options to become available in a faster manner.
At the end of the day, the feeling was that a lot of progress has happened recently. The interest in CTSP has grown amongst all involved parts (stakeholders, researchers, policy makers), as well as the number of novel methods to plan robust studies for such needed populations. A more flexible view, focusing on smaller (but statistically significant) clinical effects, seems to be gradually replacing a more conservative position. This is a very important achievement, since even a small improvement in quality of life might mean a huge difference for those patients suffering from conditions with very few - or sometime even no - treatment options.
"Take home" messages:
* More interest and importance is rapidly being given to CTSP by stakeholders, researchers and policy makers;
* Many different methods are available for planning robust and adequately powered CTSP;
* The regulatory process of new drugs for rare diseases is still an important barrier that needs to be tackled, but;
* The perspectives are good and, as this is a growing field in the area of medical research, many patients with rare diseases may take advantage of the results from CTSP in the near future.