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Focus on Regulation

EMA and FDA set up new working group on rare diseases

On 26 September 2016, a new EU-US collaboration between the European Medicines Agency (EMA) and the United States Food and Drug Administration (FDA) was announced. This new collaboration is intended to contribute to sharing experiences and best practices on the approach of the two regulators to the development and scientific evaluation of medicines for rare diseases.


Rare diseases are estimated to affect 30 million people in the European Union and the United States, though each individual disease only concerns a limited number of patients. Both the EMA and the FDA call for more global collaboration 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.

This sort of collaboration where the EMA and the FDA hold regular meetings by teleconference, with the participation of other agencies globally are known as ‘clusters’ in the EU.

The cluster on rare diseases

The agencies’ collaboration will focus on exchanging information on 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;
  • Preclinical evidence to support development programmes;
  • The design of post-marketing studies, in particular in the context of early access mechanisms;
  • Risk management strategies for long-term safety issues with rare disease treatments.

On 23 September 2016, the first meeting of the rare diseases cluster took place by teleconference. The cluster will initially meet once a month via teleconference and will be chaired jointly by FDA and EMA. The information exchange between the two regulators is covered by confidentiality arrangements.

More generally, clusters established by EMA and FDA focus on areas where the parties involved could benefit from an intensified exchange of information and strengthened international collaboration. The currently existing clusters discuss issues related to patient engagement, orphan medicinal products, biosimilars, medicines to treat cancer, medicines for children and pharmacovigilance amongst others.

The cluster will allow for confidential exchange of draft documents, policies under development, and provide more detailed information to support the scientific basis for decision-making on medicine development.

For further information visit: http://www.ema.europa.eu/docs/en_GB/document_library/Other/2016/09/WC500213119.pdf