InSPiRe: Innovative methodology for small populations research.

An European project which the focus is on the development of novel methods for the design and analysis of clinical trials in rare diseases or small populations defined, for example, by a rare genetic marker. A rare disease is defined to be one that effects less than one in 2,000 of the general population, though many are much less common than this, perhaps affecting only a handful of people in any country. Around 30 million people in Europe suffer from a rare disease.

In such settings where numbers are scarce, the large clinical trials that are generally used to evaluate new drugs and other healthcare interventions are infeasible or often impossible. As a result, new approaches to the design of such studies, or improved methods of data analysis and subsequent decision-making, are needed.