New collaboration among EU and US to boost medicine development on Rare Diseases

The clusters established by EMA and FDA focus on areas where the parties involved could benefit from an intensified exchange of information and strengthened collaboration.

November 7, 2016

A new working group on rare diseases has been established through a collaboration among the European Medicines Agency (EMA) and the United States Food and Drug Administration (FDA) in order to boost medicine development for rare diseases.

The aim of this new cluster will be too share experiences and best practices on each other’s regulatory approach to the development of medicines for these diseases, always assuring a secure exchange of information covered by the confidentiality arrangements between the two regulators. 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.

The cluster will provide a forum for confidential exchange of draft documents, policies under development, and more detailed information supporting the scientific basis for decision making on medicine development.



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