New Methods for Evaluation of Therapy

Date: September 4, 2013

By: Rare Disorders

An International Research Team is Developing New Methods for Therapy Evaluation in Rare Diseases


Aachen, 9/2/2013-In the EU diseases that affect 5 in 10,000 people or less are defined as rare.  Worldwide more than 7,000 such rare diseases are registered. The resulting groups of patients who are affected by a specific rare disease can be very small.  The ability of conventional statistical methods to evaluate new therapeutic approaches for any given rare disease is limited due to the small number of patients.  Thus, there is an urgent need not only to develop new therapeutic approaches to treat diseases, but also to develop new statistical methods to establish which approaches work.