Orphan medicinal products
Orphan medicinal products

The following Life Sciences guidance note provides comprehensive and up to date legal information covering:

  • Orphan medicinal products
  • EU regulatory framework
  • Orphan designation—essential criteria
  • Defining the disease
  • Establishing prevalence
  • Orphan designation—procedure
  • Orphan Marketing Authorisation
  • Incentives—orphan market exclusivity
  • Orphan market exclusivity—derogations
  • Similar medicinal product
  • more

On 31 January 2020, the UK ceased to be an EU Member State and entered an implementation period during which it continues to be treated by the EU as a Member State for many purposes. As a third country, the UK can no longer participate in the EU’s political institutions, agencies, offices, bodies (except to the limited extent agreed), but it continues to be subject to EU law and must submit to the continuing jurisdiction of the Court of Justice of the EU in accordance with the transitional arrangements in Part 4 of the Withdrawal Agreement. For further reading, see: Brexit—Life Sciences and News Analysis: Brexit Bulletin—key updates, research tips and resources.

Orphan medicinal products (OMPs) are medicines used to prevent, diagnose or treat rare medical conditions and diseases. Given that the number of patients affected is very low, in the absence of incentives, pharmaceutical companies may be unable to recover through sales, the costs of research and development of medical products to diagnose, prevent and treat these rare diseases.

'Rare' is defined in the relevant European legislation as affecting less than five in 10,000 people in Europe. However, most rare diseases affect less than one in 100,000 people. Despite the low incidence of individual rare diseases, they are of significant public health importance. There are over 7,000 known rare diseases which together