Orphan medicine and market exclusivity

An orphan medicinal product is a medicinal product intended for the diagnosis, prevention or treatment of a rare condition, the development of which would not be profitable without incentives. The European Regulation on orphan medicinal products organises a designation procedure by the European Medicines Agency and grants the authorised orphan medicinal product ten years of market exclusivity. This exclusivity is a regulatory protection distinct from the patent, often decisive in the strategy of pharmaceutical companies.

Designation of the orphan medicinal product

First, the disease must be rare: it must not affect more than five in ten thousand persons in the Union, or the medicinal product would not be developed without incentive for lack of return on investment. Next, there must be no satisfactory method of diagnosis, prevention or treatment, or the medicinal product must be of significant benefit compared with existing methods. Finally, designation is granted before authorisation, on the opinion of the Committee for Orphan Medicinal Products, and must be confirmed at the time of authorisation. The texts and the list of designations can be consulted on the website of theEuropean Medicines Agency.

Market exclusivity

For ten years from authorisation, the authorities may not accept an application for authorisation, nor grant an authorisation, for a similar medicinal product in the same indication. The exclusivity is extended to twelve years where the results of a paediatric investigation plan are included. It may be reduced to six years if, after five years, the designation criteria are no longer met, in particular if the medicinal product has become sufficiently profitable. Derogations allow the authorisation of a similar medicinal product with the holder’s consent, in case of insufficient supply, or if the second product is safer, more effective or clinically superior. The notion of similar medicinal product has been the subject of decisions of the General Court of the European Union.

Key points

In practice, the market exclusivity of the orphan medicinal product protects the indication and not the molecule: a competitor may obtain an authorisation for another indication, and the originator may be protected by several exclusivities for several indications. It is cumulative with the patent, the supplementary protection certificate and the’data exclusivity, in a protection timetable that must be drawn up precisely. The firm handles these issues, as presented on the page pharmaceutical patents and regulation.