NCATS Researchers Propose Innovative Approach to Test Drugs in Rare Diseases

Author: NIH
Date Published: June-2014
Source: NCATS

Developing effective treatments is a slow and costly process, and more than 80 percent of investigational drugs tested in clinical trials fail. Discovering treatments for rare diseases can be even more challenging because the small numbers of patients with these diseases make it difficult to find enough people who can participate in clinical trials. The small numbers of rare disease patients also make gathering information about the diseases difficult. As a result, scientists often know little about the symptoms and biology of these conditions, which adds to the complexity of designing drug studies. Additionally, pharmaceutical companies may find it difficult to justify the cost of developing drugs for such small markets of rare disease patients.