Equity, Access and Economic Evaluation in Rare Diseases: The Impact of Orphan Drug Legislation on Health Policy and Patient Care
Authors: Bosanquet N.1; Domenighetti G.2; Beresniak A.3; Auray J-P.4; Crivelli L.5; Richard L.6; Howard P.6
Source: Pharmaceutical Development and Regulation, Volume 1, Number 3, 2003 , pp. 151-157(7)
Publisher: Adis International
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Abstract:
Currently, there are in excess of 5000 rare diseases, that is, diseases that affect only a small proportion of a given population. The majority of these conditions lack appropriate treatments for a variety of reasons, including limited markets for the development of new pharmaceutical products coupled with prohibitively high costs associated with research, manufacturing and marketing. This paper discusses the issues associated with the equity of and access to orphan drugs used in rare diseases. Options for healthcare policy across Europe are presented. The interest in using robust evaluation parameters is emphasized, despite this not being in agreement with the use of artificial indicators such as quality-adjusted life years (QALYs), which appear to lead to divergent and erroneous results. New decision-making methods must govern the policy, statistics, and economics relevant to the use of orphan drugs in rare diseases.Keywords: Orphan drugs; Economic implications; Health policy; Legislation
Document Type: Research article
Affiliations: 1: 1 Faculty of Medicine, Imperial College of Science, Technology and Medicine, London, England 2: 2 Department of Econometrics and Political Economy (DEEP), Institute of Economics and Health Management (IEMS), University of Lausanne, Lausanne, Switzerland 3: 3 Corporate Pharmacoeconomics, Serono International SA, Geneva, Switzerland 4: 4 National Center of Scientific Research (CNRS), University of Lyon 1, Lyon, France 5: 5 Department of Economics and Management, University of Applied Sciences of Southern Switzerland, University of Lugano, Lugano, Switzerland 6: 6 Heron Evidence Development Ltd, Stevenage, England
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