Orphan Drug Commercial Models: Sustaining the rare disease business
May 2016
A FirstWord ExpertViews Dossier Report
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Orphan Drug Commercial Models:Sustaining the rare disease business
Published May 2016 Copyright 2016 Doctors Guide Publishing Limited
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Orphan Drug Commercial Models: Sustaining the rare disease business
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ContentsExecutive summary .............................................................................................................................. 1
Research objectives and methodology .................................................................................2
Contributors ...............................................................................................................................................3
Current structures .................................................................................................................................4Key findings .................................................................................................................................................4
Orphan drugs market overview .............................................................................................................5
What are orphan drugs? .....................................................................................................................5
The orphan drugs market is increasing in value ..........................................................................5
There are a large number of orphan drugs being approved each year ........................... 7
Some orphan drugs can generate significant revenue .......................................................10
New products will continue to drive growth ............................................................................ 11
Companies involved in orphan drugs ...............................................................................................15
Orphan drugs commercial models and leading companies ................................. 17Key findings ............................................................................................................................................... 17
There are different structures for orphan drugs companies ....................................................... 17
A fully integrated model is favoured by larger pharmaceutical companies ........................18
Partnership or partially integrated models are being increasingly used .............................20
Independent and focused on orphan drugs ................................................................................ 21
Shire: increasing its focus on rare diseases ..........................................................................24
BioMarin: a blueprint for orphan drug success .....................................................................26
Strategies for success ......................................................................................................................29Key findings ...............................................................................................................................................29
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Orphan Drug Commercial Models: Sustaining the rare disease business
Past orphan drugs success has been largely down to the unmet need .................................. 31
Product Acquisition removes the risk but can deliver returns ................................................33
A focused R&D strategy is essential for success ......................................................................35
Education and awareness is an important part of the commercialisation process .............. 37
Stakeholder engagement is fundamental for orphan drugs ...................................................38
Patients are seen as the most important stakeholder by many .......................................38
Companies have a role to play in educating and supporting physicians ......................40
Determining the value proposition is an important element for commercial success ......42
A successful model is not necessarily the one that delivers the highest revenues .............. 47
Traditional commercial measures of success are used for orphan drugs .......................... 47
Patient outcomes are increasingly important for orphan drugs ............................................49
Key challenges for current orphan drugs models ....................................................... 51Key findings ............................................................................................................................................... 51
Pricing of orphan drugs is expected to become more challenging .....................................52
The orphan drugs market place is becoming more competitive ...........................................56
There are emerging challenges in the approval processes ....................................................60
The definition of an orphan drug is being scrutinised ...............................................................62
Evolution of commercial models ..............................................................................................66Key findings ...............................................................................................................................................66
A more focused model is perceived as being most effective ................................................. 67
Patient engagement will continue to be critical for the orphan drugs model .....................69
Innovation will be key for future orphan drug success ............................................................ 71
Out-sourcing commercial activities can maintain focus and competitiveness ..................72
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Orphan Drug Commercial Models: Sustaining the rare disease business
ContributorsnTony Hall, Co-founder of FindaCure
nLuigi Longinotti, Portfolio Manager Orphan Drugs at Recordati
n Pedro Lendinez Ortega, Products Affairs Manager, Orphan Drugs Especialist en Orphan Europe
nWendy White, SVP Rare Disease Dohmen Life Science Services
nChairman and CEO, Rare Disease Company
nChris Garabedian, Former CEO of Sarepta
nVP, Rare Diseases, Biopharmaceutical Company.
nCEO and Rare Disease Expert, Bioscience and Biopharmaceutical Company.
nRare Disease Expert, Healthcare regulatory policy and government relations consulting firm
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Orphan Drug Commercial Models: Sustaining the rare disease business
Partnership or partially integrated models are being increasingly usedThe unique challenges that the orphan drugs market entails means that some companies prefer to use a hybrid partnership approach rather than fully integrate their rare disease interests. One such example is Sanofi and Genzyme.
Sanofi, for example, bought Genzyme, which is very much a rare diseases company. This worked because large companies are not really set up for the rare disease market. It requires a different kind of approach to drug development and a different kind of approach to commercialisation really. Thats why a company like Sanofi buys a company that knows how to do it, like Genzyme.
Tony Hall
Sanofi acquired rare disease leader Genzyme in 2011 and it is now a wholly owned subsidiary. However, Sanofi has retained the name and kept Genzyme as a la
