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Unintended effects of orphan product designation for rare neurological diseases

Authors

  • Sinéad M. Murphy MB, BCh, MRCPI,

    1. Medical Research Council Centre for Neuromuscular Diseases, National Hospital for Neurology and Neurosurgery and Department of Molecular Neuroscience, University College London Institute of Neurology, London, United Kingdom
    2. Department of Neurology, Adelaide and Meath Hospitals incorporating the National Children's Hospital, Tallaght, Dublin, Ireland
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  • Araya Puwanant MD,

    Corresponding author
    1. Department of Neurology, School of Medicine and Dentistry, University of Rochester Medical Center, Rochester, NY
    • Department of Neurology, University of Rochester, 601 Elmwood Avenue, Box 673, Rochester, NY 14642
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  • Robert C. Griggs MD3 and the Consortium for Clinical Investigations of Neurological Channelopathies (CINCH) and Inherited Neuropathies Consortium (INC) Consortia of the Rare Disease Clinical Research Network,

    1. Department of Neurology, School of Medicine and Dentistry, University of Rochester Medical Center, Rochester, NY
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  • the Consortium for Clinical Investigations of Neurological Channelopathies (CINCH) and Inherited Neuropathies Consortium (INC) Consortia of the Rare Disease Clinical Research Network


Abstract

Since the introduction of the Orphan Drug Act in 1983, designed to promote development of treatments for rare diseases, at least 378 orphan drugs have been approved. Incentives include financial support, tax credits, and perhaps most importantly, extended market exclusivity. These incentives have encouraged industry interest and accelerated research on rare diseases, allowing patients with orphan diseases access to treatments. However, extended market exclusivity has been associated with unacceptably high drug costs, both for newly developed drugs and for drugs that were previously widely available. We suggest that a paradoxical effect of orphan product exclusivity can be reduced patient access to existing drugs. In addition, the costs of each new drug are arguably unsustainable for patients and for the American health care system. Of all the specialties, neurology has the third highest number of orphan product designations, and neurological diseases account for at least one-fifth of rare diseases. Citing the use of tetrabenazine for chorea in Huntington disease, adrenocorticotropic hormone for infantile spasms, and enzyme replacement therapy with alglucosidase alpha for Pompe disease, we highlight these paradoxical effects. ANN NEUROL 2012;72:481–490

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