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