Rare Diseases and Orphan Products

Rare diseases collectively affect millions of Americans of all ages, but developing drugs and medical devices to prevent, diagnose, and treat these conditions is challenging. The Institute of Medicine (IOM) recommends implementing an integrated national strategy to promote rare diseases research and product development. Table of Contents Front Matter Summary 1 Introduction 2 Profile of Rare Diseases 3 Regulatory Framework for Drugs for Rare Diseases 4 Discovery Research for Rare Diseases and Orphan Product Development 5 Development of New Therapeutic Drugs and Biologics for Rare Diseases 6 Coverage and Reimbursement: Incentives and Disincentives for Product Development 7 Medical Devices: Research and Development for Rare Diseases 8 Toward an Integrated Approach to Accelerating Research and Product Development for Rare Diseases References Appendix A: Study Activities Appendix B: Innovation and the Orphan Drug Act, 1983-2009: Regulatory and Clinical Characteristics of Approved Orphan Drugs Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs Appendix D: Glossary, Abbreviations, and Public Laws Appendix E: Rare Diseases Clinical Research Network Appendix F: Advocacy Group Approaches to Accelerating Research and Product Development: Illustrative Examples Appendix G: Committee and Staff Biographies Index