UNIVERSITY OF MALTA
LIFE SCIENCE RESEARCH SEMINARS
Web: http://events.um.edu.mt/scisem/
Email: scisem@um.edu.mt
Abstract form
Title: Development of orphan drugs in the United States: an academic
perspective
Presenter: James Cloyd, PharmD
Contact address: Rm 4-204, MTRF, 2001 6th St, University of Minnesota,
Minneapolis, MN, USA 55455
Tel: +1 612 624 4609
Fax: +1 612 626 9985
Email: cloyd001@umn.edu
Presentation date: 7 April 2008
Abstract
Rare disorders are medical conditions that afflict a relatively small percentage of the
population. As a result of advances in biomedical sciences, especially genomics, the number
of rare disorders is increasing and now exceeds 6000. Although only a few individuals have
a particular rare disorder, when all conditions are combined as many as 25 million
individuals in the US and 35 million in Europe are affected. The medical, social, and
economic costs of rare disorders are enormous. Therapies are limited because of an
inadequate understanding of pathophysiology and the lack of financial incentives for
companies to develop drugs. The medications used to treat rare disorders are collectively
known as orphan drugs, because they often have no commercial sponsors. In the late 1970s,
a group in the US began working with Congress to enact legislation intended to increase the
number of orphan drugs. The Orphan Drug Act, which was passed in 1983 give companies a
tax credit of 50% of clinical investigation expenses, exemption form FDA filing fees, and 7
years of market exclusivity for orphan drug that have been approved. Similar incentives are
now available in the EU. The FDA has designated more than 1700 products as orphan
drugs, but has only approved 300 for marketing. Although these medications are important
additions, a greater emphasis on drug discovery and development is needed to meet the
needs of patients and families. Academic centers are increasingly focusing on rare disorder
research and are well positioned to accelerate the discovery and development of new
therapies for rare disorders. However, substantial increases in funding, expansion of
research facilities, and the establishment of clinical trials network are needed. With adequate
support and collaboration from government agencies and the pharmaceutical industry,
academic centers throughout the world can significantly contribute to improving the
treatment of rare disorders.