Orphan drugs

Does my product qualify as an orphan drug?

For a product to be classed as an orphan medicine, it must be intended for the diagnosis, prevention or treatment of either:

  • a life-threatening or chronically debilitating condition affecting no more than 5 in 10,000 people in the EU at the time of submission of the application for orphan designation; or
  • a life-threatening, seriously debilitating or serious and chronic condition that, without incentives, it would be unlikely that the revenue after marketing of the medicinal product would cover the investment in its development.

There must also be either no satisfactory method of diagnosis, prevention or treatment of the condition concerned, or, if there is such a method, the new medicine must be of significant benefit to those affected by the condition.

Are there any advantages to a medicine having orphan status?

European legislationThere are special provisions in European legislation to encourage companies to develop new drugs and to carry out research on existing drugs to treat these rare diseases, as sales of such drugs are expected to be low.

The following incentives are provided in relation to orphan drugs:

  • Market exclusivity.  For a 10 year period following the grant of a marketing authorisation for the orphan product, no directly competing or similar product can be placed on the market.
  • Protocol assistance.  The European Medicines Agency will provide scientific advice and guidance on preparing the marketing authorisation dossier.
  • Reduced fees.  The European Medicines Agency may grant fee reductions, which it funds through a centralised fund from the European Commission.  Reduced fees may be applicable to applications for marketing authorisation, inspections, variations and protocol assistance.
  • Grant funding.  The EU and most member states have programmes and initiatives in place for granting funding to pharmaceutical companies developing orphan drugs, such as the European Commission framework programme for research and technological development.

How do I get an orphan designation?

Any company may apply to the European Medicines Agency for their drug to be given an orphan designation. This is a separate application to an application for a marketing authorisation. Orphan designation may be obtained at any stage before an application for marketing authorisation is made, provided there is proper scientific justification for it.

If a drug is successfully designated as an orphan drug, it will be registered on the Community Register of Orphan Medicinal Products.

If you have any questions on this article or would like to propose a subject to be addressed by Synapse please contact us.

Orphan drugs

Tim Worden

Tim is a partner in the Patents and Life Sciences group based in our Cambridge office.

"For a 10 year period following the grant of marketing authorisation for the orphan product, no directly competing or similar product can be placed on the market."