Orphan Drug Designation

Orphan Drug Designation (ODD) is a key regulatory procedure available for Drugs intended to treat, prevent or diagnose rare diseases. It provides a valuable set of incentives for companies.

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

  • Intended to treat, prevent or diagnose a disease that is life-threatening or chronically debilitating.
  • The prevalence in the EU must not be more than 5 in 10,000.
  • No satisfactory method of diagnosis, prevention or treatment of the condition is already authorized, or, if such a method already exists, the medicine must be of significant additional benefit to those affected by the condition.

Applications for ODD are assessed by the EMA’s Committee for Orphan Medicinal Products (COMP). The Agency sends the COMP opinion to the European Commission, which is responsible for granting the orphan designation.

There is little commercial incentive to develop medicines intended for small numbers of patients, therefore, the EU offers some incentives to encourage the development of Orphan Drugs (OD).Amongst others, these include receiving protocol assistance, certain fee reductions and market exclusivity.

In terms of Pediatrics medicines, some 60% of ODs are intended for pediatric use. Medicines authorized across the EU through the results of studies from a Paediatric Investigation Plan (PIP) are eligible for an extension of their supplementary protection certificate. For ODs, the incentive is an additional two years of market exclusivity.

How can DDR help?

DDR has successfully conducted challenging ODD processes for companies worldwide and has extensive experience in ODD singularities in Europe.

  • DDR provides regulatory support throughout the whole designation procedure, including the submission of orphan annual reports.
  • DDR’s expertise ensures an in-depth understanding of the key ODD criteria, such as medical plausibility, prevalence and significant benefit.
  • DDR will elaborate robust arguments to successfully substantiate these elements.
  • DDR’s network provides personalized support at EMA oral explanations, resulting in successful outcomes.
  • DDR will assist you in all management activities via the EMA IRIS portal.

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