On March 3, 2016, a workshop on Small Population Clinical Trials was organized. This workshop, jointly organized by the European Medicine Agency (EMA) and the International Rare Disease Research Consortium (IRDiRC), was held at the EMA premises in London, UK. The purpose of this workshop was to contribute technical solutions to make the best use of scarce clinical data in the context of small population trials and to identify points of agreement between the different stakeholders regarding non-classical designs. The workshop was also aimed at identifying further areas where research is needed. Furthermore, the purpose was to issue recommendations that are useful to clinicians and researchers when planning trials.
The workshop, chaired by Dr Simon Day and Dr Kristina Larsson, started with a general discussion on the topic, followed by two breakout sessions on different topics, being: a matrix of the different study methods, obtaining adequate safety data, multi-arm designs, decision analytic approaches, extrapolation and patient’ engagement. The day was finalized in a joint session, in which participants gave feedback from their breakouts, and discussed and agreed on proposed conclusions and recommendations.
More information about the IRDiRC Small Population Clinical Trials Task Force can be found here.