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Orphan Drugs


These are medicines which are developed specifically for rare diseases.




Companies that are looking to gain "orphan medicinal product designation" for their products in Europe must submit their application to the Committee for Orphan Medicinal Products (COMP). They need to demonstrate that they intend to develop their product(s) "for the diagnosis, prevention or treatment of life-threatening or very serious conditions that affect not more than 5 in 10,000 persons in the European Union" (this equates to about 185,000 cases) (Ref: EMA/COMP).




The FDA Office of Orphan Products Development (OOPD) is responsible for the evaluation of scientific and clinical data submissions from sponsors seeking Orphan Drug Designation for their product.


In the US an 'Orphan Drug' is a drug or biologic:


“intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug.”


As of 13 December 2016, Section 3052 of the 21st Century Cures Act (Pub. L. No. 114-255) changed the population estimate to qualify for Humanitarian Use Device (HUD) designation.  The HUD designation is now defined as “a medical device intended to benefit patients in the treatment or diagnosis of a disease or condition that affects or is manifested in not more than 8,000 individuals in the United States per year”.  Previously the requirement stated that designation was for “fewer than 4,000”.




Under normal circumstances the Pharmaceutical Industry doesn't develop medicines or devices for such small numbers of patients.  There are a range of worldwide incentives, however, to encourage the life science industry to develop Orphan Drugs.  Many companies are now taking advantage of these incentives.


Please refer to our regulatory services for further information.


Please contact us with your specific regulatory compliance requirements.  We look forward to working with you.