US and EU regulators collaborate on rare diseases

The European Medicines Agency is working increasingly closely with its US counterpart on a range of issues – and the latest collaboration is in the field of rare diseases.

The EMA said that it has set up a new “cluster” with the US Food and Drug Administration dedicated to confidentially sharing experiences and best practices about regulation of orphan medicines.

Rare diseases affect 30 million people in the EU and around the same number in the US, even though each disease concerns a limited number of patients.

Global collaboration is important to ensure that the limited number of studies conducted due to the small populations, can benefit all patients regardless of where they live.

The agencies will exchange information on:

  • Clinical trial design and use of statistical analysis and methods;
  • Selection and validation of trial endpoints;
  • Preclinical evidence to support development programmes;
  • Design of post-marketing studies, such as the EMA’s conditional marketing authorisation and the FDA’s accelerated approval;
  • Risk management strategies for long-term safety issues.

An existing EMA/FDA ‘cluster on orphan medicinal products’ will continue to focus on information sharing and collaboration on orphan designation and exclusivity, the agencies’ mechanisms to encourage the development of medicines for rare diseases.

The currently existing EMA/FDA clusters discuss issues related to patient engagement, biosimilars, orphan medicines, medicines to treat cancer, medicines for children, and pharmacovigilance, among other topics.

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