World Orphan Drug Congress USA 2018
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World Orphan Drug Congress 2018 Charles River Associates Whitepaper

Building new access strategies in a disrupted industry


The arrival of gene and cell therapies for rare diseases could bring disruption in the biopharmaceutical industry. The ability to treat previously incurable conditions, but at prices that concentrate higher treatment costs for these patients over a short period of time is in the hands of only one industry stakeholder, the pharmaceutical manufacturer.


In this paper, we summarise key challenges for manufacturers relating to research and development, market access, commercialisation, and quantifying the value of these therapies to the multiple stakeholders. 


For more information on Charles River Associates visit our website. 


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