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Innovative new project looks to improve opportunities for rare disease research

Warwick Medical School is to lead one of three prestigious EU projects on developing methodology for clinical trials within small populations, aiming to create further opportunities for new treatments of rare diseases.

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.

Source: Rare Diseases UK
75% of rare diseases affect children
30% of rare disease patients die before their fifth birthday
Includes rare cancers such as childhood cancers, and some other well known conditions such as cystic fibrosis and Huntington’s disease.

There are between 6,000 and 8,000 known rare diseases, many of which have a genetic component, and the majority of which affect children.

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.”

stallard“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.”

The announcement today coincides with the international Rare Disease Day, an annual, awareness-raising event co-ordinated by EURORDIS.

Professor Stallard added, “The hope for this, as with any methodological research, is that we’re able to pave the way for improved treatments. When we consider the vast array of rare diseases that we know of, the potential impact is huge.”


For more information on Rare Diseases in Europe, view the 2014 Report from Orphanet:

Further information

For further information, or to arrange interviews with Professor Stallard, contact Luke Harrison, Press Officer, on +44 (0) 2476 574255/150483 or +44 (0) 7920531221, or by email on

Useful links

Warwick Medical School on Twitter

Rare Disease Day