Projects
Sustainable orphan drug development
The number of distinct rare (orphan) diseases is estimated to be between 5,000 and 8,000, with around 29 million rare disease sufferers in the European Union. Rare diseases are life threatening or chronically debilitating diseases with a prevalence of less than 5:10,000 inhabitants in the European Union. For most rare diseases, including many inborn errors of metabolism, therapy has not been developed due to lack of general awareness and gaps in knowledge. This consortium is set up in order to build a disease registry framework to collect information on rare diseases, encourage drug development and optimize patient care.
Partners: Academic Medical Center (AMC), Erasmus MC (University Medical Center Rotterdam), Genzyme, Health Care Insurance Board (CVZ - College voor zorgverzekeringen), University Medical Center Utrecht, Shire, and ZonMW/Steering Committee on Orphan Drugs
Full project title: Sustainable orphan drug development through registries and monitoring
Relevant information:
26 02 2010 Persbericht: 'Eén miljoen Nederlanders met zeldzame ziekte’
06 04 2009 Press release: Registry framework for rare disease