The European Medicines Agency (EMA) and the United States Food and Drug Administration (FDA) have established a new cluster to boost the development of medicines for rare diseases.
The new cluster will share information and best practices on each other’s regulatory approach to support medicine development for these diseases.
It will provide a forum for confidential exchange of authorities draft documents, policies under development, and furnish detailed information to support the scientific basis for decision making for the medicine development.
The recent activity is a part of the regulatory authority’s objective to expand international collaboration.
A global collaboration on this area has been deemed important to ensure the number of studies conducted benefit patients regardless of their location.
The information being exchanged in the cluster will range from design of clinical trials in small populations, use of statistical analysis methods to selection of trial endpoints and pre-clinical evidence to support development programmes.
The authorities will also discuss design of post-marketing studies such as EMA’s conditional marketing authorisation and FDA’s accelerated approval, as well as risk management strategies addressing long-term safety issues with medicines for rare diseases.
The cluster will be jointly chaired by EMA and FDA and initially meet each month through a teleconference.