Regulatory Processes for Rare Disease Drugs in the United States and European Union

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• Front MatterSummary1 Introduction2 FDA Flexibilities, Authorities, and Mechanisms3 EMA Flexibilities, Authorities, and Mechanisms4 Alternative and Confirmatory Data5 FDA and EMA CollaborationAppendix A: Biographical Sketches of Committee Members and StaffAppendix B: Disclosures of Unavoidable Conflicts of InterestAppendix C: Public Meeting AgendasAppendix D: Centre for Innovation in Regulatory Science Data Analysis MethodologyAppendix E: Qualitative Interview Summary and MethodologyAppendix F: Non-Exhaustive List of Patient Focused Drug Development Meetings and Patient Listening Sessions for Rare Diseases Between 2013 and 2023Appendix G: List of Orphan Approvals by FDA or EMA Between 2018 and 2022Appendix H: Select Examples of Rare Disease Drug ProductsAppendix I: FDA and EMA Resources, Policies, and Programs Relevant for Drug Development for Rare Diseases and Conditions