Spring Intro 2023

30/03/2023

Important points to consider

Not possible to combine an orphan & non-orphan indication on the same licence • Separate MAAs with separate trade names are needed Criteria for designation can be reassessed at year 5 after MAA approval if requested by a member state • 10-year market exclusivity can be reduced to 6 if determined that original criteria no longer met, e.g., prevalence >5/10 000 OD status can also be terminated • At the request of the sponsor at any time • If criteria for designation are no longer met at time of MA • At the end of the market exclusivity period Orphan market exclusivity: MA may be granted to a ‘similar’ medicinal product (derogation) only if • Holder of original orphan product gives consent • Holder of original orphan product cannot supply sufficient product • Second applicant demonstrates clinical superiority

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Ongoing Revision of EU Orphan (& Paediatric) Regulations

• Orphan Regulation now more than 20y old

• In Nov 2020, European Commission triggered the Revision of EU legislation on medicines for children and rare diseases (Ref. Ares(2020)7081640) • insufficient development in areas of greatest unmet medical needs (e.g. 95% of rare diseases still have no treatment option) • "one-size-fits all" incentives and rewards do not always provide an adequate tool to stimulate developments in areas of unmet needs • availability and accessibility varies considerably across EU Member States • review current legal definitions, including use of biomarkers • Fits within Commission’s wider EU Pharma Strategy Roadmap – revisions envisaged also to basic EU Pharmaceutical acts (Directive 2001/83/EC, Reg no 726/2001) which aims to ensure: • access to affordable medicines • supporting competitiveness, innovation of the EU’s pharmaceutical industry • enhancing crisis preparedness, diversified and secure supply chains, address medicines shortages • ensuring a strong EU voice in the world

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