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.
Orphan Drugs are legislated for in the EU under Regulation (EC) No 141/2000 (1).
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.
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.
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).
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.
CambReg have been working in this arena since 1999 and have therefore have plenty of experience in obtaining ODD and can advise you on all regulatory aspects related to Orphan Drug approval
CambReg has been involved in numerous Orphan Drug projects including:
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