Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2006 Jan 17;174(2):189-90.
doi: 10.1503/cmaj.050706.

Is the current approach to reviewing new drugs condemning the victims of rare diseases to death? A call for a national orphan drug review policy

Joe T R Clarke  1
Affiliations

Is the current approach to reviewing new drugs condemning the victims of rare diseases to death? A call for a national orphan drug review policy

Joe T R Clarke. CMAJ. .
No abstract available

PubMed Disclaimer

References

    1. United States Food and Drug Administration. Orphan Drug Act (P.L. 97-414), Jan. 4, 1983. Available: www.fda.gov/orphan/oda.htm (accessed 2005 Dec 07).
    1. Canadian Coordinating Office for Health Technology Assessment. Common Drug Review (CDR). Available: www.ccohta.ca/CDR/cdr_intro_e.cfm (accessed 2005 Dec 07).
    1. Clarke JTR, Iwanochko RM. Enzyme replacement therapy of Fabry disease. Mol Neurobiol 2005;32:43-50. - PubMed
    1. Summaries of all reviews and CEDAC recommendations are available on the CCOHTA website at www.ccohta.ca/CDR/cdr_recommendations.cfm (accessed 2005 Dec 07).
    1. Clarke JT, Amato D, Deber RB. Managing public payment for high-cost, high-benefit treatment: enzyme replacement therapy for Gaucher's disease in Ontario. CMAJ 2001;165:595-6. - PMC - PubMed