Drug development for orphan diseases in the context of personalized medicine

Translational Research : the Journal of Laboratory and Clinical Medicine
George J Brewer

Abstract

Orphan diseases are diseases that are found in less than 200,000 patients in the United States, which is the cutoff point for the number of patients for a drug to be profitable. Because many thousands of orphan diseases exist in the aggregate (about 20 to 30 million Americans have orphan diseases), these patients are disenfranchised from drug development by the pharmaceutical industry. Orphan drugs are a large part of personalized medicine. The orphan diseases are often so rare that a physician may observe only 1 case a year or less. So proper treatment is a personalized encounter between doctor and patient. Academic physician-scientists have tried to fill this therapy vacuum by working on developing orphan drugs. But many disincentives are involved, which include career disincentives, lack of funding, and the multiple areas of expertise that are required. Positive developments include formation of the National Organization for Rare Diseases, the Orphan Drug Act, the development of a grant program to fund orphan drug development, the formation of the National Institutes of Health Office of Rare Diseases, and the passage of orphan drug legislation by other countries. Progress has increased, but the 300 orphan drugs and devices a...Continue Reading

References

Nov 10, 1978·JAMA : the Journal of the American Medical Association·A S PrasadP Rabbani
May 1, 1987·Hepatology : Official Journal of the American Association for the Study of Liver Diseases·G M HillD E Hartmann
Apr 1, 1982·Journal of Inorganic Biochemistry·I BremnerB W Young
Apr 1, 1981·Journal of Inorganic Biochemistry·C F MIllsG Weham
Jun 1, 1981·Journal of Inorganic Biochemistry·C F MillsI Bremner
Feb 1, 1996·Seminars in Liver Disease·M L Schilsky
Feb 3, 2000·Hepatology : Official Journal of the American Association for the Study of Liver Diseases·G J BrewerK J Kluin
Jun 17, 2008·Lancet·Marlene E HaffnerPaul D Maher
Jul 22, 2008·The Annals of Thoracic Surgery·Harvey I PassSofia Merajver

❮ Previous
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Citations

Apr 30, 2010·The Journal of Clinical Endocrinology and Metabolism·Leona Cuttler, J B Silvers
Nov 24, 2011·Indian Journal of Pediatrics·Paul Saenger
Jan 21, 2012·Genetics in Medicine : Official Journal of the American College of Medical Genetics·David C LandyRichard R Sharp
Dec 25, 2010·Journal of Pharmacy & Bioallied Sciences·Aarti SharmaDushyant Kumar
Jan 29, 2016·Wiener medizinische Wochenschrift·Claude-Henry VolmarShaun P Brothers
Oct 27, 2012·Drug Discovery Today·Sean EkinsAntony J Williams
Jun 23, 2012·Nitric Oxide : Biology and Chemistry·Antonia PatrunoRoberta Fruttero
Mar 8, 2011·Drug Discovery Today·Sean EkinsJoel S Freundlich
Mar 13, 2014·Annals of the New York Academy of Sciences·Christelle Gateau, Pascale Delangle
May 16, 2015·Seminars in Pediatric Surgery·Corey W IqbalMichael R Harrison
Apr 14, 2011·Policy, Politics & Nursing Practice·Pamela Holtzclaw Williams
Jul 28, 2015·Journal of Materials Chemistry. B, Materials for Biology and Medicine·Murthi S KandanapitiyeSongping D Huang
Jan 21, 2018·Cell Death and Differentiation·Zinaida DedeićXin Lu
Mar 30, 2016·The European Journal of Health Economics : HEPAC : Health Economics in Prevention and Care·Julio López-BastidaPedro Serrano-Aguilar
Dec 1, 2012·Molecular Informatics·Sean EkinsAntony J Williams
Jan 21, 2014·Journal of Materials Chemistry. B, Materials for Biology and Medicine·Vindya S PereraSongping D Huang
Nov 11, 2014·Expert Opinion on Orphan Drugs·Jouni UittoSharon F Terry
Feb 14, 2012·Dalton Transactions : an International Journal of Inorganic Chemistry·Pascale Delangle, Elisabeth Mintz
Feb 11, 2021·Trends in Pharmacological Sciences·Helen I RoesslerGijs van Haaften
May 26, 2021·Seminars in Cancer Biology·Albrecht StenzingerUNKNOWN GMS working groups and ZPM working groups
Jan 1, 2013·Chemistry of Materials : a Publication of the American Chemical Society·Vindya S PereraSongping D Huang
Sep 14, 2021·Protein Science : a Publication of the Protein Society·Tucker L Apgar, Charles R Sanders

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