Paragraph 1 Prevalence of a condition in the United KingdomF1F5
The following provisions apply for the purposes of establishing, pursuant to regulation 50G(2)(a) and (b)(i), that a medicinal product is intended for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition affecting not more than five in 10,000 persons in F2the United Kingdom.
The material provided pursuant to regulation 50G(3) must include—
material which demonstrates that the disease or condition for which the medicinal product would be authorised affects not more than five in 10,000 persons in F3the United Kingdom at the time at which the application for an orphan marketing authorisation is submitted, where this is available;
details of the condition intended to be treated and a justification of the life-threatening or chronically debilitating nature of the condition, supported by scientific or medical references; and
copies of, or references to, relevant scientific literature, as well as copies of information from relevant databases in F4the United Kingdom, where available.
If there are no databases as referred to in paragraph (2)(c), information from relevant databases in other countries may be supplied, provided appropriate extrapolations are made.
Annotations
Textual amendments
- F1
Sch. 9A inserted (31.12.2020) by The Human Medicines (Amendment etc.) (EU Exit) Regulations 2019 (S.I. 2019/775), reg. 1, Sch. 4 (as amended by S.I. 2020/1488, reg. 1, Sch. 2 para. 191); 2020 c. 1, Sch. 5 para. 1(1)
- F2
Words in Sch. 9A para. 1(1) substituted (1.1.2025) by The Human Medicines (Amendments relating to the Windsor Framework) Regulations 2024 (S.I. 2024/832), regs. 1(1), 139(b)
- F3
Words in Sch. 9A para. 1(2)(a) substituted (1.1.2025) by The Human Medicines (Amendments relating to the Windsor Framework) Regulations 2024 (S.I. 2024/832), regs. 1(1), 139(b)
- F4
Words in Sch. 9A para. 1(2)(c) substituted (1.1.2025) by The Human Medicines (Amendments relating to the Windsor Framework) Regulations 2024 (S.I. 2024/832), regs. 1(1), 139(b)
- F5
Words in Sch. 9A para. 1 heading substituted (1.1.2025) by The Human Medicines (Amendments relating to the Windsor Framework) Regulations 2024 (S.I. 2024/832), regs. 1(1), 139(a)