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Orphan Drug Designation

Orphan Drug Designation

What is an Orphan Drug (EU)?

An Orphan Drug is a medicinal product for a rare disease. As the market for such medicines is small, companies are encouraged to develop such treatments by being offered incentives.

  • A medicinal product can be designated as an orphan medicinal product if it is intended for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition:
  • Affecting no more than five in 10,000 persons in the UK/EU at the time of submission
  • Without incentives it is unlikely that expected sales of the medicinal product would cover the investment in its development, and
  • No satisfactory method of diagnosis, prevention or treatment of the condition concerned is authorised, or, if such method exists, the medicinal product will be of significant benefit to those affected by the condition

Do you have an Orphan Drug? – Let us advise

It is worthwhile finding out whether your product can be designated as an orphan drug as there are many advantages to developing a product in this category.

Advantages of Orphan Drug Designation

Orphan Drug Designation (ODD) gives automatic access to the numerous incentives some of which are listed below.

  • EU Funded Research
  • National incentives
  • Access to the Centralised Procedure
  • Protocol Assistance (Scientific Advice)
  • 10-year marketing exclusivity post marketing authorisation approval
  • Possibility of an accelerated marketing authorisation assessment
  • Fee reductions
  • Two years additional market exclusivity for orphan paediatric medicines

These incentives also apply to medicines already on the market, so the addition of an ‘orphan’ indication for marketed drugs is naturally a very attractive proposition and can be used to exploit products in an entirely new market sector.


It should be noted that the criteria for obtaining an EU Orphan Drug Designation (ODD) and the incentives available differ in the US.

CambReg’s Orphan Drug Service

We find that helping companies to develop new drugs / new drug uses for patients where there currently are no credible alternatives to be tremendously successful, challenging and rewarding.

Our experience can help you

CambReg have been working in this arena since 1999 and have therefore have plenty of experience in obtaining orphan drug designation (ODD) and can advise you on all regulatory aspects related to Orphan Drug approval

CambReg has been involved in numerous Orphan Drug projects including:

  • Developmental advice for an ODD product through to partnering/MAA stage
  • Successful ODD applications in the EU for several start-up companies
  • Obtaining ODD status in the US or making parallel applications
  • ODD maintenance activities for both EU & US
  • Developing an ODD product through to MAA stage
  • Preparation and submission of Marketing Authorisation Applications
  • Obtaining rapid approval by use of the ‘exceptional circumstances’ route


For more information or an estimate, contact us »

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