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Equity, Access and Economic Evaluation in Rare Diseases

The Impact of Orphan Drug Legislation on Health Policy and Patient Care

  • Current Opinion
  • Published:
Pharmaceutical Development and Regulation

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.

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Acknowledgements

Preparation of this review was made possible by financial support from Serono International SA, Switzerland. The authors have provided no information on conflicts of interest directly relevant to the content of this study.

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Authors and Affiliations

  1. Faculty of Medicine, Imperial College of Science, Technology and Medicine, London, England

    Nick Bosanquet

  2. Department of Econometrics and Political Economy (DEEP), Institute of Economics and Health Management (IEMS), University of Lausanne, Lausanne, Switzerland

    Gianfranco Domenighetti

  3. Corporate Pharmacoeconomics, Serono International SA, 15 bis Chemin des mines, Geneva, CH, 1211, Switzerland

    Ariel Beresniak

  4. National Center of Scientific Research (CNRS), University of Lyon 1, Lyon, France

    Jean-Paul Auray

  5. Department of Economics and Management, University of Applied Sciences of Southern Switzerland, University of Lugano, Lugano, Switzerland

    Luca Crivelli

  6. Heron Evidence Development Ltd, Stevenage, England

    Lance Richard & Paul Howard

Authors
  1. Nick Bosanquet
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  2. Gianfranco Domenighetti
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  3. Ariel Beresniak
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  4. Jean-Paul Auray
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  5. Luca Crivelli
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  6. Lance Richard
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  7. Paul Howard
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Correspondence to Ariel Beresniak.

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Bosanquet, N., Domenighetti, G., Beresniak, A. et al. Equity, Access and Economic Evaluation in Rare Diseases. Pharm Dev Regul 1, 151–157 (2003). https://doi.org/10.1007/BF03257374

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  • DOI: https://doi.org/10.1007/BF03257374

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