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Breakthrough Business Models: Drug Development for Rare and Neglected Diseases and Individualized Therapies: Workshop Summary [Minkštas viršelis]

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  • Formatas: Paperback / softback, 150 pages, aukštis x plotis: 229x152 mm
  • Išleidimo metai: 17-Mar-2009
  • Leidėjas: National Academies Press
  • ISBN-10: 0309120888
  • ISBN-13: 9780309120883
Kitos knygos pagal šią temą:
Breakthrough Business Models: Drug Development for Rare and Neglected Diseases and Individualized Therapies: Workshop SummaryDidesnis paveikslėlis
  • Formatas: Paperback / softback, 150 pages, aukštis x plotis: 229x152 mm
  • Išleidimo metai: 17-Mar-2009
  • Leidėjas: National Academies Press
  • ISBN-10: 0309120888
  • ISBN-13: 9780309120883
Kitos knygos pagal šią temą:
Pastovi nuoroda: https://www.kriso.lt/db/9780309120883.html
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.

Table of Contents



Front Matter 1 Introduction and Overview 2 Current Model for Financing Drug Development: From Concept Through Approval 3 The Food and Drug Administration's Orphan Drug Program 4 Diverse Funding Models 5 Strategies for Facilitating Sharing of Research Materials and Data 6 Strategies for Navigating Intellectual Property 7 Strategies for Facilitating Clinical Trials 8 Summary References Appendix A: Agenda Appendix B: Speaker Biographies Appendix C: Resources
1 INTRODUCTION AND OVERVIEW 1
Scope' of the Workshop,
3
Key Themes,
4
Organization of the Report,
5
2 CURRENT MODEL FOR FINANCING DRUG DEVELOPMENT: FROM CONCEPT THROUGH APPROVAL 7
Investors in Drug Development,
7
Current Status of Investments,
9
Ways to Facilitate Drug Development,
11
3 THE FOOD AND DRUG ADMINISTRATION'S ORPHAN DRUG PROGRAM 12
Strategies,
15
4 DIVERSE FUNDING MODELS 19
Institute for OneWorld Health: A Not-for-Profit Pharmaceutical Company,
19
Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT): A Virtual Company for Managing Drug Discovery and Development Alliances,
24
Genzyme: Surviving and Thriving as a For-Profit Company in the Rare Disease Arena,
29
Celtic Therapeutics, LLLP: A Private-Equity Model for Addressing Global Health,
32
Panel Discussion,
37
Open Discussion,
39
5 STRATEGIES FOR FACILITATING SHARING OF RESEARCH MATERIALS AND DATA 42
Finding and Bargaining for Research Materials and Data,
42
The Alzheimer's Disease Neuroimaging Initiative (ADNI): A Public–Private Partnership,
45
Genetic Alliance BioBank,
51
6 STRATEGIES FOR NAVIGATING INTELLECTUAL PROPERTY 55
Overview: Creating an Enabling Intellectual Property Environment for Rare and Neglected Diseases,
55
Innovation in Alliances and Licensing: Vertex Pharmaceuticals Transforming Now for the Future,
64
The Myelin Repair Foundation: Accelerating Intellectual Property Sharing to Facilitate Translation,
67
The University of California at Berkeley's Approach to Management of Intellectual Property,
71
Open Discussion,
78
7 STRATEGIES FOR FACILITATING CLINICAL TRIALS 82
FDA Review and Regulation of Small Clinical Trials: Successes, Barriers, and Directions for the Future,
83
Approaches to Accelerating Clinical Trials,
91
Muscular Dystrophy Association's Approach to Maximizing Assets in Clinical Trials,
94
Open Discussion,
97
8 SUMMARY 98
Scientific and Regulatory Elements of the Translational Research Process,
98
Diverse Funding Organizations,
99
Sharing of Materials and Data,
100
Intellectual Property Strategies,
101
Approaches to Facilitating Clinical Trials,
101
Areas for Further Discussion,
102
REFERENCES 104
APPENDIXES
A Agenda
106
B Speaker Biographies
113
C Resources
128