Policy Process Lessons from the Orphan Drug Act: Applications for Health Policy Advocates

2016 ◽  
Author(s):  
Barbara Teresa Andraka-Christou
Keyword(s):  
Health Policy ◽  
Orphan Drug ◽  
Policy Process ◽  
Orphan Drug Act

Policy process lessons from the Orphan Drug Act: applications for health policy advocates

2015 ◽  
Vol 4 (3) ◽  
pp. 278-297 ◽  
Author(s):  
Barbara Andraka-Christou
Keyword(s):  
Public Policy ◽  
Orphan Drug ◽  
Policy Process ◽  
Public Attention ◽  
Content Type ◽  
The Public ◽  
Orphan Drug Act ◽  
Public Policy Process ◽  
History Of ◽  
The Impact

Purpose – The Orphan Drug Act has provided the pharmaceutical industry with incentives to research and develop drugs for orphan diseases: rare diseases with little profit potential. It is considered very successful legislation by legal scholars, the Food and Drug Administration (FDA), and orphan drug activists. The policy process of the Act provides an important model of the policy process for future incentive-based pharmaceutical legislation. The purpose of this paper is to summarize the important incentives of the Act and the historical events leading up to the Act. The paper applies three different theoretical models of the public policy process to understand the emergence of the Orphan Drug Act: Kingdon’s Multiple Streams Model, the Advocacy Coalition Framework, and Social Constructionism Theory. The paper then synthesizes the public policy process lessons from each perspective and provides four recommendations for other social activists seeking to propel incentive-based pharmaceutical legislation for under-researched diseases. Design/methodology/approach – The author analyzes the history of the Orphan Drug Act based on publicly available scholarly research, government documents, and interest group publications. The author then applies three public policy theories to the history of the Orphan Drug Act to explain the emergence of the Act and to extract policy process lessons for future disease activists. Findings – Regardless of which theoretical perspective the Orphan Drug Act is analyzed from, some common themes of the policy process emerge. First, focussing events are instrumental in capturing the public’s sympathy and Congress’s attention. Second, in its activities and proposed legislation, a coalition should provide a role for all relevant and important actors. Third, the target groups of the legislation were construed positively, increasing the pressure for Congressmen to pass some kind of bill. Finally, the proper construction of “the problem” is instrumental to passing effective legislation as a “solution.” Originality/value – The Orphan Drug Act is widely considered successful incentive-based pharmaceutical legislation. However, because it was originally passed in 1983 and has not had public attention since the early 1990s (when it was amended), it has rarely been written about in recent years. However, its lessons are still highly relevant to policy activists, especially disease activists. Furthermore, existing articles focus on the impact of the legislation and ways to amend it, rather than on the passage of the Act.

scholarly journals Framing and the health policy process: a scoping review

2016 ◽  
Vol 31 (6) ◽  
pp. 801-816 ◽  
Author(s):  
Adam D Koon ◽  
Benjamin Hawkins ◽  
Susannah H Mayhew
Keyword(s):  
Health Policy ◽  
Scoping Review ◽  
Policy Process

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.

The impact of political context upon the health policy process in Pakistan

Public Health ◽  
2007 ◽  
Vol 121 (4) ◽  
pp. 278-286 ◽  
Author(s):  
M.M. Khan ◽  
W. Van den Heuvel
Keyword(s):  
Health Policy ◽  
Policy Process ◽  
Political Context ◽  
The Impact

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
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