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
  • Brexit and orphan designation
  • Orphan designation—essential criteria
  • Defining the disease
  • Establishing prevalence
  • Orphan designation—procedure
  • Orphan Marketing Authorisation
  • Incentives—orphan market exclusivity
  • Orphan market exclusivity—derogations
  • more

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 affect an estimated 30 million people (68% of the population) in Europe. About 80% of rare diseases are thought to be genetic in origin which means they are often incurable, life-long conditions. The low incidence of individual rare diseases means that it is often not commercially viable for pharmaceutical companies to develop and market OMPs. Of the 7,000 plus rare diseases known to exist today, treatment is only available for just 200–300 of them.

Based on the principle that patients suffering from rare diseases deserve access to the same quality of medicinal products as other patients, the EU introduced a regulatory regime designed to stimulate research and development of medicinal products for rare diseases by providing incentives and rewards to the pharmaceutical industry. The primary reward is a ten-year period of market exclusivity for orphan drugs following marketing authorisation (MA) approval. This means that no directly competing products will be allowed onto the market during this ten-year period.

The regulatory regime has proved