Current Business, Legal, and Ethical Issues Impacting the Orphan Drug Segment of the American Pharmaceutical Industry

Author

Karen Lulek Scharnhorst, Fontbonne College

Year of Award

1986

Degree

Master of Business Administration (MBA)

College

College of Business & Professional Studies

Degree Program

Business

Department

Business Administration

Keywords

Orphan Disease, Pharmaceutical, DHHS, Drug Research, Toxic, Ethics, Government Regulation

Abstract

In the United States, orphan diseases are those illnesses or conditions defined as uncommon or occurring with an incidence of 1% or less. The markets for orphan products are very small so that many pharmaceutical firms are reluctant to commit substantial funds to the research and development of drugs with low return on investment. Orphan disease victims and their families have articulated their predicament and raised the public consciousness in recent years such that the Department of Health and Human Services (DHHS) has set about providing incentives for orphan drug research and development. The Orphan Products Board (established through the DHHS) is working in concert with the FDA, the Pharmaceutical Manufacturers Association, the Generic Pharmaceutical Industry Association, and other groups to facilitate orphan drug development.

Screening and testing of orphan drugs prior to marketing is much more difficult than performing the same steps for nonorphan products. The drug toxicities may be greater and securing a sufficient number of patients for adequate pre-market testing of an orphan drug is difficult. Another major barrier to orphan drug production is the tension between pharmaceutical manufacturers' obligation to shareholders, and therein to maximize profits, and society's obligation to meet the needs of orphan disease victims. Drug companies cite rising research costs, increasing drug approval constraints, and litigation risks as reasons for not developing orphan drugs.

Recently, the federal government has assumed more of a facilitation rather than a regulation role, and collaborative programs among pharmaceutical firms, academia, government, and private groups are enhancing orphan drug sponsorship and development. Ethical issues surrounding orphan drugs have not been resolved, but some believe that new technologies will resolve many concerns.

Document Type

Restricted Thesis

Recommended Citation

Scharnhorst, Karen Lulek, "Current Business, Legal, and Ethical Issues Impacting the Orphan Drug Segment of the American Pharmaceutical Industry" (1986). All Theses, Dissertations, and Capstone Projects. 646.
https://griffinshare.fontbonne.edu/all-etds/646