26 September 2016 - The EMA and the U.S. 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 European Union and approximately the same number in the United States, 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 agencies will exchange information on various aspects of the development and scientific evaluation of medicines for rare diseases. These include topics such as:

• the design of clinical trials in small populations and the use of statistical analysis methods
• the selection and validation of trial endpoints, i.e. target outcomes of a trial
• preclinical evidence to support development programmes
• the design of post-marketing studies, in particular in the context of early access mechanisms such as EMA’s conditional marketing authorisation and FDA’s accelerated approval
• risk management strategies for long-term safety issues with medicines for rare diseases.

Read EMA press release