500 Fifth Street, N.W.
Washington, DC 20001

NOTICE: The project that is the subject of this report was approved by the Governing Board of the National Research Council, whose members are drawn from the councils of the National Academy of Sciences, the National Academy of Engineering, and the Institute of Medicine. The members of the committee responsible for the report were chosen for their special competences and with regard for appropriate balance.

This study was supported by Contract No. N01-OD-4-2139, TO # 215 between the National Academy of Sciences and the National Institutes of Health. Additional support was provided by the Food and Drug Administration. Any opinions, findings, conclusions, or recommendations expressed in this publication are those of the authors and do not necessarily reflect the view of the organizations or agencies that provided support for the project.

International Standard Book Number-13: 978-0-309-15806-0

International Standard Book Number-10: 0-309-15806-0

Additional copies of this report are available from the

National Academies Press,

500 Fifth Street, N.W., Lockbox 285, Washington, DC 20055; (800) 624-6242 or (202) 334-3313 (in the Washington metropolitan area); Internet,

For more information about the Institute of Medicine, visit the IOM home page at:

Copyright 2010 by the National Academy of Sciences. All rights reserved.

Printed in the United States of America

The serpent has been a symbol of long life, healing, and knowledge among almost all cultures and religions since the beginning of recorded history. The serpent adopted as a logotype by the Institute of Medicine is a relief carving from ancient Greece, now held by the Staatliche Museen in Berlin.

Front cover photographs (top to bottom):

Using electropheresis apparatus to separate proteins by molecular weight. Photo courtesy of National Institute of Arthritis and Musculoskeletal and Skin Diseases.

96-well, 384-well, and 1,536-well plates used in pharmaceutical and life science research. Photo courtesy of National Human Genome Research Institute.

Children with ectodermal dysplasia. Used with permission. Photo courtesy of the National Foundation for Ectodermal Dysplasias.

Image of chromosomal abnormalities in mouse cells from a study of leukemia-promoting effects of tumor necrosis factor-alpha in Fanconi anemia group C stem cells. Photo courtesy of the laboratory of Dr. Qishen Pang at Cincinnati Children’s Hospital Medical Center. U.S.A. Copyright 2007, American Society for Clinical Investigation. Used with permission.

Friedreich’s ataxia patient and Friedreich’s Ataxia Research Alliance (FARA) spokeperson, Kyle Bryant, on his recumbent trike during the cycling competition Race Across America. Copyright 2010, Used with permission.

Children with sickle cell disease. Used with permission. Photo courtesy of Cincinnati Children’s Hospital Medical Center.

Suggested citation: IOM (Institute of Medicine). 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press.

The National Academies | 500 Fifth St. N.W. | Washington, D.C. 20001
Copyright © National Academy of Sciences. All rights reserved.
Terms of Use and Privacy Statement