title: Orphan drug development: an economically viable strategy for biopharma R&D.
abstract: Orphan 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.
authors.affiliation: Thomson Reuters Life Sciences Consulting, The Johnson Building, 77 Hatton Garden, London EC1N 8JS, UK. kiran.meekings@thomsonreuters.com
authors.forename: Kiran N
authors.initials: KN
authors.lastname: Meekings
authors.forename: Cory S M
authors.initials: CS
authors.lastname: Williams
authors.forename: John E
authors.initials: JE
authors.lastname: Arrowsmith
mesh_terms: D035843:Biomedical Research / Q000191:economics*; D002986:Clinical Trials as Topic / Q000191:economics; D003362:Cost-Benefit Analysis; D017277:Drug Approval / Q000191:economics; D055808:Drug Discovery / Q000191:economics* / Q000379:methods / Q000458:organization & administration / Q000639:trends; D005062:European Union; D006801:Humans; D040541:Marketing; D009965:Orphan Drug Production / Q000191:economics*; D035583:Rare Diseases / Q000188:drug therapy / Q000191:economics; D014481:United States; D014486:United States Food and Drug Administration