Points to consider in the design and analysis of clinical trials in small populations
Diseases that occur in populations with a low prevalence or in vulnerable populations lead to limitations in the number of available patients for drug development. In such a setting, when sample sizes for clinical development are limited, one usually speaks of small populations. Drug development in small populations poses some special challenges. This applies above all to the statistical planning of the clinical studies, their statistical evaluation and interpretation as well as decision-making. The challenges can often only be addressed using innovative statistical methods and data analysis approaches. This points-to-consider document is aimed at statisticians who are going to work in this exciting field intending to give them an initial introduction and to sensitize them to the peculiarities of small populations. Basic points to consider in clinical development for small populations are addressed and potential useful statistical approaches are proposed.
Historical controls, meta-analytic priors, and extrapolation - some useful publications for small populations
The SIG Small Populations has been working to identify publications that would be useful for statisticians working in studies with small populations. The topics that were chosen as of interest were extrapolation, historical controls, and meta-analytic priors.
Subgroups were set up to work on each of these topics. A literature search was performed to identify publications of potential interest. These were then reviewed within the subgroups and those deemed most relevant and useful were identified. The attached slides list the publications recommended for each topic with a brief description of the content and the reason for the recommendation.