EMA and FDA Set Up New Working Group on Rare Diseases

The new collaboration between the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) announced Monday will improve the sharing of information on various aspects of the development and scientific evaluation of medicines for rare diseases.

According to EMA, rare diseases affect 30 million people in the EU and approximately the same number in the US, though each disease individually concerns a limited number of patients. And since only a limited number of studies can be conducted for such small populations, both regulators are calling for more global collaboration.

The collaboration, known in the EU as a cluster, will focus on:

• 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 programs
• opportunities for regulatory flexibility (approval supported by other than two adequate and well-controlled studies and/or use of a novel endpoint)
• 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 or breakthrough designation
• risk management strategies for long-term safety issues with rare disease treatments

Both regulators will allow for the confidential exchange of draft documents, policies under development and the discussion of more detailed information supporting the scientific basis for decision making.

The first meeting of the rare diseases cluster took place by teleconference on 23 September and future teleconferences will occur monthly, subject to need and predefined in advance, for approximately one hour and 30 minutes.

EMA also clarified that the existing EMA/FDA “cluster on orphan medicinal products” will continue to focus on information sharing and collaboration on orphan designation and exclusivity, as well as the agencies’ mechanisms to encourage the development of medicines for rare diseases.

Other currently existing EMA/FDA clusters discuss issues related to patient engagement, biosimilars, orphan medicines, cancer treatments, pediatric medicines and pharmacovigilance.

Terms of reference for the EMA/FDA cluster on rare diseases