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Cover of Rare Diseases and Orphan Products

Rare Diseases and Orphan Products

Accelerating Research and Development

; Editors: Marilyn J Field and Thomas F Boat.

Washington (DC): National Academies Press (US); .
ISBN-13: 978-0-309-15806-0ISBN-10: 0-309-15806-0


This Institute of Medicine (IOM) study grew out of discussions with the NIH Office of Rare Diseases Research and the FDA Office of Orphan Products Development about opportunities to accelerate rare diseases research and orphan product development. As discussions progressed, the focus expanded from drugs and biologics to include medical devices. In 2009, the IOM appointed a 14-person committee to oversee the study. Consistent with its charge (which is presented in full in Appendix A), the committee examined the epidemiology, impact, and treatment of rare diseases as context for an assessment of research and development; investigated the strengths and limitations of the current development pathways for new drugs, medical devices, and biologics for rare diseases; assessed public policies that may influence research and development decisions involving rare diseases and orphan products; and developed recommendations for an integrated national policy on rare diseases research and orphan product development.

This report presents the committee's conclusions and recommendations. It is written for a broad and diverse audience, including public officials in research and regulatory agencies; advocacy and philanthropic groups that support rare diseases research and orphan product development; companies that develop pharmaceutical, medical device, and biologic products; academic medical centers, research institutes, and researchers engaged in basic and clinical research; and the interested general public.


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.

Suggested citation:

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

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.

Copyright © 2010, National Academy of Sciences.
Bookshelf ID: NBK56189PMID: 21796826DOI: 10.17226/12953


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