Comparative effectiveness research and the rise of orphan indications
International Journal of Pharmaceutical and Healthcare Marketing
ISSN: 1750-6123
Article publication date: 27 May 2014
Abstract
Purpose
The purpose of this paper is to show the link between the comparative effectiveness research (CER) and the interest in developing drugs for rare disease by the pharmaceutical industry. Total healthcare spending is on the rise without having a corresponding better health outcome. As such, with the growing role of government in healthcare, measuring and demonstrating value is beginning to expand beyond the private sector to metrics applied in the public sector. A modern approach to comparative effective research began its rapid rise in the USA when the American Recovery and Reinvestment Act of 2009 allocated $1.1 billion for CER. This paper analyzes the implication and impact on the pharmaceutical industry.
Design/methodology/approach
The paper reviews the basic definitions of CER and its areas of strength and weakness. It used real examples of drugs to show the impact of the Reinvestment Act of 2009 on the rise of orphan drugs.
Findings
The study shows that the act encouraged the development of orphan drugs, mainly because of the low budget impact due to a smaller patient base. Provisions of the Affordable Care Act provide incentives for such rare disease indications, as no one can be denied coverage with pre-existing conditions.
Research limitations/implications
The study is limited by the number of available rare drugs and the ongoing process of implementation of the Affordable Care Act.
Practical implications
The study shows the cost-effective method of treating medical conditions.
Social implications
Development of orphan drugs opens up access to care for many patients at a cost-effective price.
Originality/value
This paper shows the link between the CER and the interest in developing drugs for rare disease by the pharmaceutical industry. It also brings out the possible implication of the Affordable Care Act on the pharmaceutical industry with respect to its strategies for drug development and drug portfolio.
Keywords
Acknowledgements
The authors acknowledge the support received from the Rutgers Business School Alumni Association and the Blanche and Irwin Lerner Center for the Study of Pharmaceutical Management Issues.
Citation
Jeffers, S., Slomiany, M., Bitar, R., Kruse, S. and Hassan, M. (2014), "Comparative effectiveness research and the rise of orphan indications", International Journal of Pharmaceutical and Healthcare Marketing, Vol. 8 No. 2, pp. 151-163. https://doi.org/10.1108/IJPHM-01-2014-0007
Publisher
:Emerald Group Publishing Limited
Copyright © 2014, Emerald Group Publishing Limited