Based on advances in our understanding of genetics and pathophysiology, we are in the midst of an exciting therapeutic development period in rare diseases. The Orphan Drug Act defines as rare conditions those affecting <200 000 individuals in the United States.1 Examples of rare diseases include cystic fibrosis, hemophilia, spinal muscular atrophy, and most lysosomal storage diseases. Collectively, rare diseases are common, affecting almost 30 million individuals in the United States.2 Unfortunately, patients with rare diseases experience barriers to comprehensive care, and 95% of rare diseases lack US Food And Drug Administration (FDA)-approved disease-specific treatments. Key barriers to the delivery of high-quality care and therapeutic innovation include lack of access to disease experts, limited knowledge of disease course, and few partnerships between disease stakeholders. We propose a new health care model for patients with rare diseases, called the Care Continuum Model, which focuses on 3 concepts: (1) telehealth;...

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