UCL Great Ormond Street Institute of Child Health


Orphan Drug Designation (ODD)

An orphan designation allows a pharmaceutical company or another entity (including academic investigators) to benefit from incentives from the European Union to develop a medicine for a rare disease, such as reduced fees and protection from competition once the medicine is placed on the market.

At UCL ICH we are developing gene, cell and other novel therapies for a wide range of rare disease indications. Although most of our work is currently at the preclinical or early trial stage, there is a potential for these to go onto become licensed medicines. ODD status offers a number of benefits including protocol assistance in the next phase of development and also reduced fees. ODD will also attract the interest of commercial partners.

The gene therapy programme has recently gained experience in achieving ODD for a number of products and so wanted to share this experience so that other investigators can also achieve this status for their developments.

To qualify for orphan designation, a medicine must meet a number of criteria:

  • it must be intended for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating;
  • the prevalence of the condition in the EU must not be more than 5 in 10,000 or it must be unlikely that marketing of the medicine would generate sufficient returns to justify the investment needed for its development;
  • no satisfactory method of diagnosis, prevention or treatment of the condition concerned can be authorised, or, if such a method exists, the medicine must be of significant benefit to those affected by the condition.