Title: Orphan drugs: ten years of experience with the EU framework on stimulating innovation for treating rare diseases

Authors: Andreas Zaby

Addresses: Department of Business and Economics, Berlin School of Economics and Law, Badensche Str. 50-51, Berlin 10825, Germany

Abstract: This paper reviews the European Union|s (EU|s) legislative framework on orphan medicinal products in terms of the deliberations prior to its enactment as well as the key criteria, institutions and procedures established by it. This paper further examines the economic incentives, particularly the market exclusivity incentive, created for stimulating innovation by pharmaceutical companies for treating rare diseases. A quantitative analysis of the impact this innovation policy has had to date is provided by analysing the development of the number of active orphan drug designations and corresponding marketing authorisations during the ten-year period since the enactment of this legislation. The EU framework is compared to the US Orphan Drug Act and critical perspectives, such as pricing, patient access and off-label use, are discussed.

Keywords: orphan drugs; medicinal products; innovation policies; rare diseases; economic incentives; Europe; medicines; pharmaceutical companies; EU; European Union; legislative frameworks; market exclusivity; drug designations; active designations; marketing authorisations; USA; United States; pricing; prices; acts; statutes; patient access; off-label use; European Community; laws; legislation; new product development; NPD; regulations; regulatory frameworks; management.

DOI: 10.1504/IJTPM.2011.042088

International Journal of Technology, Policy and Management, 2011 Vol.11 No.3/4, pp.291 - 306

Published online: 31 Mar 2015 *

Full-text access for editors Full-text access for subscribers Purchase this article Comment on this article