Fool Me Twice? The Reemergence of Rofecoxib and the Orphan Drug Act

2019 ◽  
Vol 171 (8) ◽  
pp. 578
Author(s):  
Theodore T. Lee ◽  
Daniel H. Solomon ◽  
Aaron S. Kesselheim
Keyword(s):  
Orphan Drug ◽  
Orphan Drug Act

scholarly journals The Orphan Drug Act: An Appropriate Approval Pathway for Treatments of Rare Diseases?

2019 ◽  
Vol 54 (5) ◽  
pp. 283-284 ◽  
Author(s):  
Michael Gabay
Keyword(s):  
Orphan Drug ◽  
Rare Diseases ◽  
Financial Incentives ◽  
Annual Number ◽  
Recent Past ◽  
Regulatory Requirements ◽  
Drug Products ◽  
Orphan Drug Act ◽  
Pharmaceutical Manufacturers ◽  
Financial Benefits

The Orphan Drug Act provides financial incentives to pharmaceutical manufacturers to develop treatments for rare diseases affecting limited patient populations. Since passage of the Act in 1983, the U.S. Food and Drug Administration (FDA) has approved more than 600 orphan drug indications from greater than 450 distinct drug products. The annual number of orphan drug designation approvals has increased significantly in the recent past with much of this increase driven by approval of secondary indications for previously approved treatments. This recent increase has led to concerns regarding the Act as some pharmaceutical manufacturers have reaped outsized financial benefits while avoiding the regulatory requirements and costs associated with nonorphan drug development.

Editorial by concerned physicians: Unintended effect of the orphan drug act on the potential cost of 3,4-diaminopyridine

2015 ◽  
Vol 53 (2) ◽  
pp. 165-168 ◽  
Author(s):  
Ted M. Burns ◽  
Gordon A. Smith ◽  
Jeffrey A. Allen ◽  
Anthony A. Amato ◽  
W. David Arnold ◽  
...  
Keyword(s):  
Orphan Drug ◽  
Potential Cost ◽  
Orphan Drug Act ◽  
Unintended Effect

The Orphan Drug Act

1993 ◽  
Vol 153 (23) ◽  
pp. 2623
Author(s):  
Charles A. Sanders
Keyword(s):  
Orphan Drug ◽  
Orphan Drug Act

scholarly journals Biomarker-Defined Subsets of Common Diseases: Policy and Economic Implications of Orphan Drug Act Coverage

PLoS Medicine ◽  
2017 ◽  
Vol 14 (1) ◽  
pp. e1002190 ◽  
Author(s):  
Aaron S. Kesselheim ◽  
Carolyn L. Treasure ◽  
Steven Joffe
Keyword(s):  
Orphan Drug ◽  
Economic Implications ◽  
Common Diseases ◽  
Orphan Drug Act
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