Rare diseases affect about 60 millions of people in EU and the US. FDA considers disease as an orphan one if it affects less than 200000 persons in the US. EMA defines orphan disease as a disease with prevalence under 5 per 10000 people in EU. Despite the difference in definition, both regulators agree that all these rare diseases combined seriously affect health and wealth of EU and US citizens. Agencies began to collaborate in orphan products back in 2008 and since then already achieved some results, for instance,
Agencies began to collaborate in orphan products back in 2008 and since then already achieved some results, for instance, the common application form for orphan drug approval for medical use in the US and EU has been developed. Regulators have set up a new cluster to provide flexibility in orphan medicines development and assessment. Agencies plan to share clinical trial design and statistical analysis experience, endpoint selection and validation approach, drug preclinical evaluation data and risk management strategies in this regulatory area. The first meeting of rare diseases cluster has been already conducted on September 23. The cluster will meet once a month via teleconference to discuss current regulatory issues for an hour and a half. Meeting highlights will be then published online.