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 ContentsFront MatterSummary1 Introduction2 Profile of Rare Diseases3 Regulatory Framework for Drugs for Rare Diseases4 Discovery Research for Rare Diseases and Orphan ProductDevelopment5 Development of New Therapeutic Drugs and Biologics for RareDiseases6 Coverage and Reimbursement: Incentives and Disincentives forProduct Development7 Medical Devices: Research and Development for Rare Diseases8 Toward an Integrated Approach to Accelerating Research andProduct Development for Rare DiseasesReferencesAppendix A: Study ActivitiesAppendix B: Innovation and the Orphan Drug Act, 1983-2009:Regulatory and Clinical Characteristics of Approved Orphan DrugsAppendix C: Medicare Part D Coverage and Reimbursement of OrphanDrugsAppendix D: Glossary, Abbreviations, and Public LawsAppendix E: Rare Diseases Clinical Research NetworkAppendix F: Advocacy Group Approaches to Accelerating Research andProduct Development: Illustrative ExamplesAppendix G: Committee and Staff BiographiesIndex