Draft guidelines issued on marketing exclusivity review for orphan drugs

The European Commission has produced draft guidelines for reviewing the 10-year marketing exclusivity period for certain orphan drugs after five years.

A European regulation that came into effect in April 2000 created a procedure for the designation of orphan drugs in the EU and set up incentives for the development of orphan products. The flagship incentive was the granting of 10 years' marketing exclusivity for products approved in an orphan indication. But the regulation also said that the period could be reduced to six years under certain conditions, and that the Commission could draw up detailed guidelines on these conditions.

The Commission's guidelines give details of procedures for the review of the marketing exclusivity period and the conditions under which it might be reduced to six years.

To be designated an orphan product, a drug must be developed either for a disease that affects fewer than five people in 10,000 in the EU or for another serious disease for which the treatment is unlikely to generate an adequate return on investment if no incentives are provided. Additionally, there must either be no existing satisfactory method of diagnosis, prevention or treatment for this disease, or, if one does exist, the product in question must offer 'significant benefit' to the patient.

The regulation says that 10 years' marketing exclusivity may be reduced to six if at the end of the fifth year after marketing authorisation, the product no longer meets these designation criteria.

The guidelines say that the review procedure is triggered only if a member state considers that the designation criteria are no longer met. In this case, the member state should inform the European Medicines Agency (EMEA). The EMEA's Committee of Orphan Medicinal Products (COMP) will then issue an opinion explaining why orphan medicine status should or should not be extended beyond the sixth year.

The COMP first looks at whether the initial designation criteria are still met. If this is the case, it will recommend that the marketing exclusivity period is not reduced. However, if the initial criteria are no longer met, the COMP then has to consider whether the alternative criteria in the regulation are met. If the alternative criteria are met, the COMP will recommend that the period is not reduced.

The Commission's guidelines are open to comments from concerned parties until 7 May.