Module 6

03/10/2024

Orphan Regulation

Orphan designation where the drug:

• is to treat a condition that affects not more than five in 10,000 persons in the UK/EU; OR • is intended for a life-threatening, seriously debilitating or serious and chronic condition, and the marketing of the product is unlikely to generate a sufficient return on investment

AND

• there exists no satisfactory method of diagnosis, prevention or treatment of the condition in question authorised in the UK/EU or, if such method exists, the product will be of significant benefit to those affected by that condition

The Organisation for Professionals in Regulatory Affairs

Data Exclusivity

47

Orphan Regulation

• Art. 8(1) of Regulation 141/2000 awards orphan drugs a ten (10) year period of market exclusivity (retained in UK law post-Brexit)

• The 10 year exclusivity is per orphan condition approved

• Subject to Art 8(3), during this period no other marketing authorisation will be issued for the same therapeutic indication in respect of a "similar medicinal product" • Orphan drug market exclusivity may be reduced to 6 years - Art 8(2) • No mix of orphan and non-orphan indications in the same MA allowed

The Organisation for Professionals in Regulatory Affairs

Data Exclusivity

48

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