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EP OrphanDrugReport2013

EP OrphanDrugReport2013

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Published by John Cronin
stae of orphan drugs in US
stae of orphan drugs in US

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Categories:Types, Research
Published by: John Cronin on May 10, 2013
Copyright:Attribution Non-commercial


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Orphan DrugrepOrt 2013
Wlcom o  evlpm
 O D ro 2013
The rst edition o EvaluatePharma
’s Orphan Drug Report brings together many o our analyses toprovide top-level insight, rom the world’s nancial markets, into the expected perormance o the orphandrug market between now and 2018. Based on EvaluatePharma
’s coverage o over 4,000 o the world’sleading pharmaceutical and biotech companies, the Orphan Drug Report 2013 highlights trends in prescriptionsales or orphan vs. non-orphan drugs, orphan designation analysis in US and Europe, product andcompany perormance, phase III R&D spend and return on investment. Additional copies are availableat: www.evaluategroup.com/orphandrug2013.Since 1996 EvaluatePharma
has been providing senior decision makers within the pharmaceutical industrywith an essential service that models the sector rom the viewpoint o the world’s nancial markets.EvaluatePharma
’s orward-looking view o the market is hugely infuential as it displays the consensuso expectations, which infuence company stock market valuations. The orecasts o equity analysts revealtheir perspectives on individual company perormance, industry trends and the macro economic environment.EvaluatePharma
has captured the consensus orecasts o equity analysts and seamlessly integratedthem with the historic results, as reported by companies. From this comprehensive view o the industry,its past and expected uture perormance emerges and can be analysed using EvaluatePharma
. Analysesrange rom total market trends and therapeutic overviews to individual company perormance andproduct progress.Whatever your view on the uture o the industry, EvaluatePharma
can help you nd value in the pharmaand biotech sector.To nd out more about this report and Evaluate’s range o services contact us:
 no amic:
 Debbie PaulTel: +1 617 573 9453Email: debbie.paul@evaluategroup.com
 rs o  Wold
:Will HauTel: +44 (0)20 7377 0800Email: will.hau@evaluategroup.com
 Hiroshi YamazakiTel: + 81 (0) 80 1164 4754Email: hiroshi.yamazaki@evaluategroup.com
 Fo l qsios:
 Christine LindgrenTel: +1 617 866 3906Email: christine.lindgren@evaluategroup.com
This report may not be relent, resold, hired out or otherwise disposed o by way o trade in any orm o binding or cover other than thatin which it is published, without the prior consent o Evaluate Ltd. While all reasonable steps have been taken to ensure that the datapresented are accurate, Evaluate Ltd. cannot accept responsibility or errors or omissions. Neither does Evaluate Ltd. warrant the accuracy,merchantability or ftness o use or a particular purpose o the data.
ail 2013
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An orphan drug is a pharmaceutical product aimed at rare diseases or disorders. The development oorphan 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 suer 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 Diss pi polios  Dfd i Lw 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)
Ficil Icivs by Lw Icld:
Mk exclsiviy
 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 ecacy/ side eects). E.g. Rebi overturnedAvonex’s orphan drug exclusivity (7 MAR 2002) 
EU: 10 Years o marketing exclusivity rom approval
rdcd r&D Coss
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]
Modoloy o Clssiyi  O D
has identied 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 dened a clean ‘Orphan’ sub-set o products which excludes productswhere less than 25% o lietime 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 lietime sales. All sales analysis in the reportis based on this clean ‘Orphan’ sub-set o products.
 O D Ovviw
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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.

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