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Orphan Drug Designation

What is an Orphan Drug?

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 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

Legislation

Orphan Drugs are legislated for in the EU under Regulation (EC) No 141/2000 (1).

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

One of the key regulatory hurdles for companies developing Orphan Drugs is obtaining an Orphan Drug Designation (ODD).  Once obtained, it 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 Orphan Drug Designation (ODD) and the incentives available differ in the EU and US).

CambReg’s Orphan Drug Service

At CambReg we have found 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 stage
  • Successful ODD applications in the EU for several start-up companies
  • Evolving  to obtain ODD status in the US
  • ODD maintenance activities – EU & US
  • Developing an ODD product through to MAA stage

Our areas of expertise cover:

  • Advising on the various national/regional  incentives that are available to you
  • Advising on all stages of Orphan Drug development
  • Preparation and submission of ODD Applications in the EU, US and beyond
  • Optimising regulatory strategy and data adaptation for different regions
  • Preparation and submission of Marketing Authorisation Applications
  • Obtaining rapid approval by use of the ‘exceptional circumstances’ route

Here is what one of our clients has to say about our Orphan Drug service:

Morvus Technology Limited (Regulatory Advice and Strategy, Orphan Drug Designation Applications in EU and USA – 3 years)

“Morvus Technology has used Cambridge Regulatory Services for 3 years as the Company’s regulatory advisor.  During this time they have assisted us with an Orphan Drug Designation Application and given advice on a number of regulatory issues relating to drug development.  We have always found them most approachable and are happy with the work they have undertaken for us.  We would not hesitate to recommend them to other organisations requiring regulatory assistance.”

Dr. Phil Burke

Chief Executive

References

  1. Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products http://ec.europa.eu/health/files/eudralex/vol1/reg_2000_141/reg_2000_141_en.pdf