The IRDiRC’s Small Population Clinical Trials Task Force and the Scientific Secretariat have made a draft document available, which is currently open for comments and suggestion for the community at large.
Clinical research and trials in rare diseases face evident obstacles: very or exceptionally low disease prevalence, small and heterogeneous patient populations, difficulty to recruit such patients, disease severity, lack of or limited knowledge of disease natural history and high attrition rates during R&D processes. The traditional randomized controlled study designs are difficult to conduct in small populations because of the difficulty to create homogeneous groups and to assess changes adequately between variable groups. Controlled rigorous designs that allow within-patient comparisons and treat all subjects would assess therapies more accurately, if feasible. Therefore, the field needs to develop cost-effective, novel, rigorous controlled study designs and relevant analyses to assess treatment efficacy in heterogeneous small populations.
This area has been identified by IRDiRC’s Scientific Committees as a focal point; consequently a Task Force with several nominated experts in the field has been set up. In this Task Force, several international initiatives are represented, as well as industrial actors and agencies for the evaluation of medicinal products. Together, the goal of this Task Force is to advance discussions on ways to optimize and improve commonly adopted approaches and to reach agreement between the different stakeholders on appropriate small population studies.
More information about this Task Force can be found here.
The Small Population Clinical Trials Draft Document can be accessed here.