An orphan drug is a pharmaceutical product aimed at rare diseases or disorders. The development oorphan drugs has been nancially incentivised through US law via the ‘Orphan Drug Act o 1983’. TheNational Organization or Rare Disorders (NORD), which was instrumental in establishing the 1983 Act,currently estimates 30 million Americans suer rom 7,000 rare diseases. Prior to the 1983 Act, 38 orphandrugs were approved. To date, 425 indication designations covering 347 drugs have been approved.The success o the original Orphan Drug Act in the US led to it being adopted in other key markets, mostnotably in Japan in 1993 and the European Union in 2000.
r Diss pi polios Dfd i Lw s:
USA: <200,000 patients (<6.37 in 10,000, based on US population o 314m)
EU: <5 in 10,000 (<250,000 patients, based on EU population o 506m)
Japan: <50,000 patients (<4 in 10,000 based on Japan population o 128m)
Ficil Icivs by Lw Icld:
USA: 7 Years o marketing exclusivity rom approval; Note: Majority o orphan drugs have acompound patent beyond 7 years. The market exclusivity blocks ‘same drug’ recombinant products.E.g. Fabrazyme (Genzyme, now Sano) vs. Replagal (Transkaryotic, now Shire). ‘Same drug’exclusion can be overturned i clinically superior (mix o ecacy/ side eects). E.g. Rebi overturnedAvonex’s orphan drug exclusivity (7 MAR 2002)
EU: 10 Years o marketing exclusivity rom approval
rdcd r&D Coss
USA: 50% Tax Credit on R&D Cost
USA: R&D Grants or Phase I to Phase III Clinical Trials ($30m or each o scal years 2008-12)
USA: User ees waived [FFDCA Section 526: Company WW Revenues <$50m]
Modoloy o Clssiyi O D
has identied all products that have orphan drug designations led in the US, EU orJapan. These will be made available as part o the core EvaluatePharma
service. To urther enhanceanalysis EvaluatePharma
has dened a clean ‘Orphan’ sub-set o products which excludes productswhere less than 25% o lietime sales are expected to come rom orphan indications. This has led to theexclusion o products, such as Avastin, Enbrel, Herceptin, Humira and Remicade, all o which have orphandesignations or indications contributing less than 25% to lietime sales. All sales analysis in the reportis based on this clean ‘Orphan’ sub-set o products.
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The success o the original Orphan Drug Act in the US ledto it being adopted in other key markets, most notably inJapan in 1993 and the European Union in 2000.