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Breakthrough Business Models

Drug Development for Rare and Neglected Diseases and Individualized Therapies: Workshop Summary


Washington (DC): National Academies Press (US); .
ISBN-13: 978-0-309-12088-3ISBN-10: 0-309-12088-8


The Institute of Medicine’s Forum on Drug Discovery, Development, and Translation held a public workshop on June 23, 2008, titled “Breakthrough Business Models: Drug Development for Rare and Neglected Diseases and Individualized Therapies.” The purpose of the workshop was to explore innovative strategies for orphan drug development. As outlined by workshop chair Nancy Sung, the workshop was designed to: provide an overview of how drug development is financed; review the state of orphan product development at FDA; explore new models for funding translational research and technologies; and examine and discuss the adequacy of the regulatory, legislative, and policy tools currently in place to help advance the development of drugs for rare and neglected diseases.

The workshop presentations and discussions considered a range of strategies for reducing the risk to industry and venture capitalists of investing in the development of such therapies by filling critical funding gaps along the drug development pathway and pursuing highly targeted approaches to early-phase development. Such strategies include sharing data and materials, managing intellectual property, launching clinical trials, and incorporating progress milestones. Speakers and participants examined the factors that contribute to successful approaches. They further discussed the use of these approaches by other organizations and in other disease areas in order to accelerate research on rare and neglected diseases. In addition, the workshop provided an opportunity for organizations with an interest in such diseases to share ideas and identify potential collaborative activities.


Rapporteurs: Theresa Wizemann, Sally Robinson, and Robert Giffin

Support for this project was provided by the American Diabetes Association; American Society for Microbiology; Amgen, Inc.; Association of American Medical Colleges; AstraZeneca Pharmaceuticals; Blue Cross Blue Shield Association; Burroughs Wellcome Fund; Department of Health and Human Services (Contract Nos. N01-OD-4-2139 and 223-01-2460); Doris Duke Charitable Foundation; Eli Lilly and Company; Entelos, Inc.; Genentech; GlaxoSmithKline; March of Dimes Foundation; Merck & Co.; Pfizer Inc.; and UnitedHealth Group.

Suggested citation:

IOM (Institute of Medicine). 2009. Breakthrough business models: Drug development for rare and neglected diseases and individualized therapies: Workshop summary. Washington, DC: The National Academies Press.

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 this project.

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.

Copyright © 2009, National Academy of Sciences.
Bookshelf ID: NBK50977PMID: 21210560DOI: 10.17226/12219


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