• Drug discovery today · Jul 2012

    Orphan drug development: an economically viable strategy for biopharma R&D.

    • Kiran N Meekings, Cory S M Williams, and John E Arrowsmith.
    • Thomson Reuters Life Sciences Consulting, The Johnson Building, 77 Hatton Garden, London EC1N 8JS, UK. kiran.meekings@thomsonreuters.com
    • Drug Discov. Today. 2012 Jul 1; 17 (13-14): 660-4.

    AbstractOrphan drug incentives have stimulated research into diseases with significant unmet medical need. Although the targeting of orphan diseases is seen by industry as an attractive strategy, there are limited economic data available to support its use. In this paper we show that the revenue-generating potential of orphan drugs is as great as for non-orphan drugs, even though patient populations for rare diseases are significantly smaller. Moreover, we suggest that orphan drugs have greater profitability when considered in the full context of developmental drivers including government financial incentives, smaller clinical trial sizes, shorter clinical trial times and higher rates of regulatory success. The data support the targeting of rare diseases as an important component of a successful biopharma R&D strategy.Copyright © 2012 Elsevier Ltd. All rights reserved.

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