EU-US collaboration to boost medicine development for rare diseases

The European Medicines Agency and the US Food and Drug Administration 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 European Union and around the same number in the USA, 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 that can be conducted, due to the small populations, can benefit all patients regardless of where they live, the regulators explain.

The agencies will exchange information on various aspects of the development and scientific evaluation of medicines for rare diseases. These include topics such as: