Rare diseases, such as sickle cell disease and thalassemia, affect up to 30 million people in the United States and at least 300 million across the globe. Congress called on the U.S. Food and Drug Administration (FDA) to sponsor a National Academies study on processes for evaluating the safety and efficacy of drugs for rare diseases or conditions in the United States and the European Union. The resulting report provides recommendations for enhancing and promoting rare disease drug development by improving engagement with people affected by a rare disease, advancing regulatory science, and fostering collaboration between FDA and the European Medicines Agency.
Table of Contents
Front Matter Summary 1 Introduction 2 FDA Flexibilities, Authorities, and Mechanisms 3 EMA Flexibilities, Authorities, and Mechanisms 4 Alternative and Confirmatory Data 5 FDA and EMA Collaboration Appendix A: Biographical Sketches of Committee Members and Staff Appendix B: Disclosures of Unavoidable Conflicts of Interest Appendix C: Public Meeting Agendas Appendix D: Centre for Innovation in Regulatory Science Data Analysis Methodology Appendix E: Qualitative Interview Summary and Methodology Appendix F: Non-Exhaustive List of Patient Focused Drug Development Meetings and Patient Listening Sessions for Rare Diseases Between 2013 and 2023 Appendix G: List of Orphan Approvals by FDA or EMA Between 2018 and 2022 Appendix H: Select Examples of Rare Disease Drug Products Appendix I: FDA and EMA Resources, Policies, and Programs Relevant for Drug Development for Rare Diseases and Conditions
