Innovative methodology for small populations research

Professor Nigel Stallard will lead the InSPiRE (Innovation in Small Populations Research) project, with an EU grant of €2.3m to bring together international experts in innovative clinical trial design methodology from across the globe. The focus will be 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.

Speaking about the InSPiRe project, Professor Stallard said, “The conduct of clinical trials in small populations is exceedingly challenging and this acts as a brake on the development of new treatments. This project will develop methods that will enable more reliable results to be obtained from clinical trials more quickly, ultimately leading to improved healthcare for these small population groups.”

“It’s important that these new, improved methods enable rapid evaluation of treatments whilst maintaining scientific and statistical rigour. New methods will include the combination of trial data with information from other studies, adaptive trial designs that allow most efficient use of the data and optimal decision-making processes that allow a conclusion to be made as quickly as possible.”

 

 

Click here for the The InSPiRe Team