Orphan Drug Designations


Shining lights for rare diseases

Almost 27 to 36 million Europeans are suffering from rare diseases, which are serious, often chronic and progressive diseases. The majority affect children and almost 30% of these children die before the age of 5. In view of these facts, the development of medicinal "orphan" drug products for treatment or even for a progression-free life is significant.
To stimulate the research on orphan drugs the European Commission offers orphan drug developers incentives such as an up to 10 years market exclusivity or fee reductions.

Orphan drug designation procedures are complex and a lot of Regulatory knowledge is needed to successfully apply and maintain the orphan drug designation.

My services include

Orphan drug designation applications
  • Dossier preparation and submission
  • Communication on behalf of the sponsor

Annual reports
  • Report preparation and submission

Orphan drug designation maintenance
  • Dossier preparation and submission
  • Coordination of the procedure
  • Oral hearings: planning and execution


Nicolodi C
Orphan drug designation in Europe - Procedural guidance and challenges. 
IJDRA. 2019; 7(3): 1-7