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Pricing for Orphan Drugs

Pricing for Orphan Drugs Opinion VIEWPOINT Will the Market Bear What Society Cannot? Cystic fibrosis (CF) is an inherited condition that af- ditionally, the foundation made its Therapeutics Devel- Brian P. O’Sullivan, MD University of fects approximately 30 000 individuals in the United opment Network available for conducting the clinical Massachusetts Medical States. Rare genetic diseases such as CF are increas- studies that enabled the drug to gain FDA approval and School, Worcester. ingly seen as unique niche markets for pharmaceutical come to market. Ironically, the patients who assumed companies. An unsustainable price structure for novel the risks of participating in the clinical trials necessary David M. Orenstein, drugs developed by pharmaceutical companies for this to bring this drug to market and who devoted count- MD, MA University of Pittsburgh rare condition and others is developing. In January 2012, less hours to raising money for the CF Foundation to un- School of Medicine, the US Food and Drug Administration approved Vertex derwrite early work are now being asked to pay, most Pittsburgh, Pharmaceuticals’ drug ivacaftor (Kalydeco) for use in a often through their insurers, an exorbitant price for the Pennsylvania. subpopulation of patients with CF who carry a specific product that resulted from http://www.deepdyve.com/assets/images/DeepDyve-Logo-lg.png JAMA American Medical Association

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Publisher
American Medical Association
Copyright
Copyright 2013 American Medical Association. All Rights Reserved. Applicable FARS/DFARS Restrictions Apply to Government Use.
ISSN
0098-7484
eISSN
1538-3598
DOI
10.1001/jama.2013.278129
pmid
24084916
Publisher site
See Article on Publisher Site

Abstract

Opinion VIEWPOINT Will the Market Bear What Society Cannot? Cystic fibrosis (CF) is an inherited condition that af- ditionally, the foundation made its Therapeutics Devel- Brian P. O’Sullivan, MD University of fects approximately 30 000 individuals in the United opment Network available for conducting the clinical Massachusetts Medical States. Rare genetic diseases such as CF are increas- studies that enabled the drug to gain FDA approval and School, Worcester. ingly seen as unique niche markets for pharmaceutical come to market. Ironically, the patients who assumed companies. An unsustainable price structure for novel the risks of participating in the clinical trials necessary David M. Orenstein, drugs developed by pharmaceutical companies for this to bring this drug to market and who devoted count- MD, MA University of Pittsburgh rare condition and others is developing. In January 2012, less hours to raising money for the CF Foundation to un- School of Medicine, the US Food and Drug Administration approved Vertex derwrite early work are now being asked to pay, most Pittsburgh, Pharmaceuticals’ drug ivacaftor (Kalydeco) for use in a often through their insurers, an exorbitant price for the Pennsylvania. subpopulation of patients with CF who carry a specific product that resulted from

Journal

JAMAAmerican Medical Association

Published: Oct 2, 2013

References