Rare Disease Drug Development

Introduction to Rare Diseases and Market Overview.- The Patient Perspective.- Select Patient Narratives.- The Caregiver Perspective.- The Critical, Multidimensional Role of Patient Advocacy Groups in Rare Disease.- A Mental Health Perspective.- Investment Decisions Related to Rare Disease Drug Development.- Optimizing Rare Disease Registries and Natural History Studies.- Novel Approaches to Clinical Trials in Rare Diseases.- Patient Benefits from Innovative Designs in Rare Diseases.- Central Nervous System Rare Disease Drug Development.- Oncologic Rare Disease Drug Development .- Hematologic Rare Disease Drug Development.- Lessons From Rare Disease and Gene Therapy Clinical Studies in Ophthalmology.- Rare Diseases in the Pediatric Population.- Cell and Gene Therapy in Rare Diseases.- The Feasibility Assessment.- The Evolving Regulatory Space and the Advent of Patient-Focused Drug Development.- Operational Aspects of Rare Disease Drug Development.- Accelerating Rare Disease Drug Development.- Select Rare Disease Drug Approvals: Lessons Learned.- A Rapid Market Access Strategy for Orphan Medicinal Products (OMPs) with Highlights Regarding the Pricing and Reimbursement Process and Barriers to Patient Use.- Integrated Life Cycle Management for Rare and Orphan Products.- The Case for Real-World Data and Real-World Evidence Generation in Rare and Orphan Medicinal Drug Development.- Closing Remarks.-