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A status for drugs treating rare diseases (under 200,000 US patients, or under 5 in 10,000 in the EU) that gives the company tax credits, fee waivers and seven to ten years of market exclusivity, to make rare-disease drug development worthwhile.
Most cancers taken subtype by subtype qualify, so oncology accounts for a large share of orphan designations, and blockbuster drugs such as imatinib and rituximab began as orphans. The 1983 US Orphan Drug Act and the 2000 EU Orphan Regulation drove development for rare cancers, but critics note 'salami-slicing' of common cancers into orphan subsets, high prices, and exclusivity blocking competitors. Rare paediatric disease designation adds a transferable priority review voucher worth around $100 million, an incentive that has funded several paediatric oncology drugs (dinutuximab, eflornithine).
Shares Paediatric drug laws: BPCA and PREA, Regulation (EC) No 141/2000: EU orphan medicines, EU pharmaceutical package reform (2023 proposal), Rare cancers.
Shares Orphan Drug Act 1983, Regulation (EC) No 141/2000: EU orphan medicines, Breakthrough Therapy / Priority Review / Priority Voucher, Rare cancers.
Shares Paediatric drug laws: BPCA and PREA, Orphan Drug Act 1983, US regulatory exclusivity periods, Inflation Reduction Act 2022: Medicare drug price negotiation.
Shares Regulation (EC) No 141/2000: EU orphan medicines, EU pharmaceutical package reform (2023 proposal), US regulatory exclusivity periods.
Shares Fast Track and RMAT designations, Paediatric drug laws: BPCA and PREA, Breakthrough Therapy / Priority Review / Priority Voucher.
Shares AMNOG (Germany, 2011), EU pharmaceutical package reform (2023 proposal), Inflation Reduction Act 2022: Medicare drug price negotiation.
Shares Fast Track and RMAT designations, Breakthrough Therapy / Priority Review / Priority Voucher.
Shares Fast Track and RMAT designations, Regulation (EC) No 141/2000: EU orphan medicines, EU pharmaceutical package reform (2023 proposal).