To stimulate the development of therapies for patients affected by orphan diseases with unmet medical needs, regulatory authorities worldwide introduced the designation of orphan drug.
These incentives consist of:
- seven years of market exclusivity from the grant date of marketing approval
- assistance in clinical research study designs
- tax credits for the costs of clinical research
- FDA fee waiver
- eligibility for FDA grants
In Europe, an orphan disease is defined as a disease or condition affecting no more than five in 10,000 European citizens with no satisfactory method of diagnosis, prevention or treatment.
The adopted incentives consist of:
- ten years of market exclusivity from the grant date of marketing approval in the EU
- protocol assistance and scientific advice
- fee reductions on EMA procedural activities
- eligibility for EU grants