{"entity":{"id":"orphan-designation","kind":"term","name":"Orphan drug designation","aka":["orphan","orphan drug","orphan designation","orphan drug status","orphan status","orphan indication","Orphan Drug Act","orphan exclusivity","rare disease designation","rare paediatric disease designation","priority review voucher","priority review vouchers"],"tldr":"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.","summary":"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).","asOf":"2026-09-09","wikipedia":"https://en.wikipedia.org/wiki/Orphan_drug","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Orphan_drug"}],"tags":[],"related":["breakthrough-designation","fast-track-rmat","label-indication","orphan-drug-act","eu-orphan-regulation","inflation-reduction-act","us-regulatory-exclusivity","amnog","eu-pharma-package","bpca-prea"],"cancers":[],"sections":["drug-discovery"],"technologies":["rare-cancers"],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"category":"Regulation & policy"},"route":"/terms/orphan-designation/","neighbours":{"term":[{"id":"amnog","kind":"term","name":"AMNOG (Germany, 2011)","route":"/terms/amnog/"},{"id":"breakthrough-designation","kind":"term","name":"Breakthrough Therapy / Priority Review / Priority Voucher","route":"/terms/breakthrough-designation/"},{"id":"eu-pharma-package","kind":"term","name":"EU pharmaceutical package reform (2023 proposal)","route":"/terms/eu-pharma-package/"},{"id":"fast-track-rmat","kind":"term","name":"Fast Track and RMAT designations","route":"/terms/fast-track-rmat/"},{"id":"inflation-reduction-act","kind":"term","name":"Inflation Reduction Act 2022: Medicare drug price negotiation","route":"/terms/inflation-reduction-act/"},{"id":"label-indication","kind":"term","name":"Label, indication and label expansion","route":"/terms/label-indication/"},{"id":"orphan-drug-act","kind":"term","name":"Orphan Drug Act 1983","route":"/terms/orphan-drug-act/"},{"id":"bpca-prea","kind":"term","name":"Paediatric drug laws: BPCA and PREA","route":"/terms/bpca-prea/"},{"id":"rare-cancers","kind":"term","name":"Rare cancers","route":"/terms/rare-cancers/"},{"id":"eu-orphan-regulation","kind":"term","name":"Regulation (EC) No 141/2000: EU orphan medicines","route":"/terms/eu-orphan-regulation/"},{"id":"us-regulatory-exclusivity","kind":"term","name":"US regulatory exclusivity periods","route":"/terms/us-regulatory-exclusivity/"}],"section":[{"id":"drug-discovery","kind":"section","name":"Drug Discovery Platforms","route":"/fronts/drug-discovery/"}]}}