Transatlantic collaboration to boost research and development of medicines for rare diseases

A new working group (“cluster”) has been set up by the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA), with the aim of sharing experiences and best practices in relation to the regulatory approach taken for the development of medicines for rare diseases. Historically, the main hurdle faced by companies wishing to invest in development in these areas has been the limited number of patients available – despite there being an estimated 60 million patients across both continents that are affected by such diseases.

The proposed collaboration will allow the agencies to share information on the various aspects in relation to the scientific evaluation and development of medicines in these rare disease areas. This will allow a wider patient population to benefit, regardless of location, since to date there have been limited numbers of studies undertaken in these areas and thus limited patient access.

The agencies will particularly focus on the following common information platforms:

• Clinical trial endpoints and how they are selected and validated
• Clinical trial design – specifically in small populations – and the use of statistical analysis methods
• Post-marketing study designs and, in particular, early access mechanisms such as EMA’s conditional marketing authorisation and FDA’s accelerated approval
• Development programs supported by preclinical evidence
• Risk management strategies for long-term safety issues with medicines for rare diseases

The emergence of this cluster will allow companies focusing in the area of rare disease therapies to have an open dialogue and a more robust pathway for scientific advice and procedural clarity. The agencies themselves will be better placed to exchange thoughts on policies under development, draft documents and well as scientific rationale in relation to assessments of medicine development plans. This data exchange is covered by confidentiality agreements.

The creation of this cluster is the latest step in the EMA and FDA’s wider objective to expand and reinforce international collaboration. The cluster will initially meet once a month via teleconference and will be chaired jointly by the FDA and EMA. The clusters established by the EMA and FDA focus on areas where the parties involved could benefit from an intensified exchange of information and strengthened collaboration. 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. The 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.

This continually developing area is a key space in the portfolio focus for bespoke pharma companies and will be a space to watch as the collaborations grow and develop.