Orphan Drugs: In Development

PMA Commission See s Corporate Parents
For Orphan Dus in Development
By Thomas L. Copmann, Ph.D.
Executive Director, PMA Commission on Drugs for Rare Diseases

With 10 million to 20 million Americans suffering from
nearly 5,000 rare diseases, the PMA Commission on Drugs
for Rare Diseases plays an important role in orphan drug
research. In the 11 years since its creation by the PMA
Board of Directors, the Commission has reviewed and
evaluated at least 44 proposals and has been directly
responsible for placing 18 drugs with pharmaceutical
industry sponsors.
The Commission is made up of senior industry scientists
with expertise in the problems associated with the development of drugs for rare diseases. The Commission reviews
proposals from individual researchers for corporate sponsorship of research and development of drugs for rare
diseases. After a favorable review, the Commission searches
for corporate sponsors for those promising therapies.
The Commission currently is searching for corporate
sponsors for five potential therapies for rare diseases.

Information about them is contained in a table accompanying this article. The PMA Commission has thoroughly
reviewed these orphan drugs and has found that they
merit development.
In addition to finding corporate sponsors for research
projects, the Commission also has the ability to link
investigators with suppliers of rare chemicals or with
experts on various rare diseases. The Commission, which
predates the Orphan Drug Act by more than two years, is
a program created by the research-based pharmaceutical
industry to help stimulate orphan drug research. The
Commission is dedicated to contributing its special expertise to the betterment of life for people with rare diseases.
Information concerning the submission of proposals to
the Commission can be obtained by writing to: PMA Commission on Drugs for Rare Diseases, 1100 15th Street NW,
Washington, DC 20005.

PMA Commission on Drugs for Rare Diseases
Is Seeking Sponsors for These Orphan Drugs
DRUGS TARGET DISEASE INVESTIGATOR/AFFILIATION
1-cycloserine Gaucher's disease Meir Lev, M.D.
The City College of New York
(New York, NY)
dextran sulfate cystic fibrosis Thomas Kennedy, M.D.
McGuire Clinic
(Richmond, VA)
3,4-diaminopyridine Lambert-Eaton syndrome Mayo Clinic
(Rochester, MN)
dichloroacetate sodium congenital lactic acidosis; Peter Stacpoole, M.D.
homozygous familial hypercholesterolemia University of Florida
(Gainesville, FL)
gossypol metastatic adrenocarcinoma Marcus Reidenberg, M.D.
Cornell Medical Center
(New York, NY)
PMA Commission on Drugs for Rare Diseases
Chairman Jack W. Reich, Ph.D. Sandra K. Mahkorn, M.D., M.P.H.
Bert A. Spilker, M.D., Ph.D. Vice President Deputy Assistant Secretary for
Director, Project Coordination Regulatory Affairs Public Health Policy
Burroughs Wellcome Co. Gensia Pharmaceuticals Health Planning & Evaluation
Department of Health and
Vice Chairman Robert L. Zerbe, M.D. Human Services
Marvin E. Jaffe, M.D. Executive Director
President Clinical Investigation & Marlene E. Haffner, M.D.
R.W. Johnson Pharmaceutical Regulatory Affairs Director
Research Institute Eli Lilly and Company Office of Orphan Products
Development
Joann L. Data, M.D., Ph.D. Food and Drug Administration
Vice President Liaison Representatives:
Project Management and Jay Moskowitz, Ph.D.
Special Assignments Claire V. Broome, M.D. Associate Director
The Upjohn Company Assistant Director for Science Science Policy and Legislation
Centers for Disease Control Office of the Director
L. Patrick Gage, Ph.D. National Institutes of Health
Executive Vice President
Scientific Affairs
Genetics Institute, Inc.
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