Display Settings:

Format

Send to:

Choose Destination
See comment in PubMed Commons below
Ann Neurol. 2012 Oct;72(4):481-90. doi: 10.1002/ana.23672.

Unintended effects of orphan product designation for rare neurological diseases.

Author information

  • 1Medical 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.

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.

Copyright © 2012 American Neurological Association.

PMID:
23109143
[PubMed - indexed for MEDLINE]
PMCID:
PMC3490440
Free PMC Article

Images from this publication.See all images (1)Free text

Fig 1
PubMed Commons home

PubMed Commons

0 comments
How to join PubMed Commons

    Supplemental Content

    Full text links

    Icon for John Wiley & Sons, Inc. Icon for PubMed Central
    Loading ...
    Write to the Help Desk