The European Medicines Agency (EMA) and the US FDA have set up a new ‘cluster’ on rare diseases to share experiences and best practices on each other’s regulatory approach to the development of medicines for these diseases.
While rare diseases are estimated to affect 30 million people in the EU and approximately the same number in the US, each disease individually concerns a limited number of patients.
Therefore, global collaboration in this area is particularly important to ensure that the limited number of studies conducted can benefit all patients regardless of where they live, the EMA said.