The IRDiRC Small Population Clinical Trials (SPCT) Task Force has published its report, which includes recommendations and guidelines for the design of small population clinical trials in the field of rare diseases. The report and recommendations have resulted from the IRDiRC SPCT workshop that was held its workshop at the EMA premises on March 3, 2016 in London, UK.
The Task Force members discussed technical solutions to make the best use of scarce data in the context of small studies, typically in rare diseases, and to identify further areas where research is needed. These recommendations have been set up in order to contribute to ensure that ultimately, effective therapies are available for patients. The recommendations are intended to be of guidance to clinicians, researchers and regulators.
Six different topics are covered in the recommendations:
- Different study methods/designs versus types of conditions
- Adequate safety data
- Multi-arm designs and platform trial designs
- Decision analytic approaches and rational approaches to adjusting levels of evidence
- Extrapolation problems and opportunities
- Patients’ engagement in study design
Overall, it was concluded that randomized, clinical trial is considered the traditional golden standard, but it is strongly advised to look systematically at alternative design options when setting up a clinical trial for a rare disease. Not every rare disease trial is as challenging as others, but if a randomized control design is not feasible, other trial options should be considered. Also, a strong emphasis was put on the promotion of the use of scientific advice from regulators regarding small population clinical trials.
To read this workshop report, including the recommendations, click here.
More information about this Task Force can be found here.