Orphan drug designation
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 many blockbuster drugs began as orphans (imatinib, rituximab). 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).
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not linked directly; found by shared links- TermBLA / NDA / MAA (marketing applications) and PDUFA dates
Shares Fast Track and RMAT designations, Label, indication and label expansion, Breakthrough Therapy / Priority Review / Priority Voucher.
- TermOrphan drug
Shares Breakthrough Therapy / Priority Review / Priority Voucher, Rare cancers.
- TermAccelerated approval
Shares Fast Track and RMAT designations, Label, indication and label expansion.