The process for developing new drug and biologic products is extraordinarily expensive and time-consuming. Although large pharmaceutical companies may be able to afford the cost of development because they can expect a large return on investment, organizations developing drugs to treat rare and neglected diseases are unable to rely on such returns. On June 23, 2008, the Institute of Medicine's Forum on Drug Discovery, Development, and Translation held a public workshop, "Breakthrough Business Models: Drug Development for Rare and Neglected Diseases and Individualized Therapies," which sought to explore new and innovative strategies for developing drugs for rare and neglected diseases.
1 Front Matter; 2 1 Introduction and Overview; 3 2 Current Model for Financing Drug Development: From Concept Through Approval; 4 3 The Food and Drug Administration's Orphan Drug Program; 5 4 Diverse Funding Models; 6 5 Strategies for Facilitating Sharing of Research Materials and Data; 7 6 Strategies for Navigating Intellectual Property; 8 7 Strategies for Facilitating Clinical Trials; 9 8 Summary; 10 References; 11 Appendix A: Agenda; 12 Appendix B: Speaker Biographies; 13 Appendix C: Resources
National Research Council, Division of Behavioral and Social Sciences and Education, Institute of Medicine, and Families Board on Children, Youth, Steve Olson
Institute of Medicine, Board on the Health of Select Populations, and Transgender Health Issues and Research Gaps and Opportunities Committee on Lesbian, Gay, Bisexual
Institute of Medicine, Board on Population Health and Public Health Practice, Division of Health Promotion and Disease Prevention, Division of International Health
Institute of Medicine, Committee to Develop Methods Useful to the Department of Veteran Affairs in Estimating Its Physician Requirements, Joseph Lipscomb
Institute of Medicine, Board on Health Care Services, Committee on Cancer Clinical Trials and the NCI Cooperative Group Program, John Mendelsohn, Harold L. Moses, Sharyl J. Nass
Institute of Medicine, Board on Health Sciences Policy, and Translation Forum on Drug Discovery, Development, Anne B. Claiborne, Rebecca A. English, Neil Weisfeld
Institute of Medicine, Board on Health Care Services, Board on Health Sciences Policy, Roundtable on Translating Genomic-Based Research for Health, National Cancer Policy Forum, Forum on Neuroscience and Nervous System Disorders, and Translation Forum on Drug Discovery, Development, Steve Olson, Autumn Downey
and Medicine National Academies of Sciences, Engineering, Health and Medicine Division, Board on Health Sciences Policy, and Translation Forum on Drug Discovery, Development, Carolyn Shore, Amanda Wagner Gee, Theresa Wizemann
Board on Health Care Services, National Cancer Policy Forum, Institute of Medicine, An American Society of Clinical Oncology and Institute of Medicine Workshop, Margie Patlak, Sharyl J. Nass
Institute of Medicine, Board on Health Sciences Policy, and Translation Forum on Drug Discovery, Development, Roundtable on Value and Science-Driven Health Care, Rebecca A. English, Julia Sanders, Claudia Grossmann
and Medicine National Academies of Sciences, Engineering, Health and Medicine Division, Board on Health Sciences Policy, Roundtable on Genomics and Precision Health, Sarah Beachy, Theresa Wizemann, Meredith Hackmann, Siobhan Addie
and Medicine National Academies of Sciences, Engineering, Health and Medicine Division, Board on Health Sciences Policy, and Translation Forum on Drug Discovery, Development, Roundtable on Genomics and Precision Health, Sarah H. Beachy, Steve Olson, Amanda Wagner Gee, Siobhan Addie
and Medicine National Academies of Sciences, Engineering, Institute of Medicine, Board on Health Care Services, Committee on the State of the Science in Ovarian Cancer Research
and Medicine National Academies of Sciences, Engineering, Institute of Medicine, Board on Health Care Services, Committee on Policy Issues in the Clinical Development and Use of Biomarkers for Molecularly Targeted Therapies, Harold L. Moses, Jonathan K. Phillips, Laurene A. Graig
Institute of Medicine, Board on Health Sciences Policy, and Translation Forum on Drug Discovery, Development, Roundtable on Value and Science-Driven Health Care, Rebecca A. English, Julia Sanders, Claudia Grossmann