Delaware Youth Rare Disease Advocacy
Delaware Rare Disease Landscape
This report outlines the current state of rare diseases in Delaware, including prevalence, access to care, and key gaps in services and research. It brings together public data and local insights to highlight issues and policy recommendations relevant to patients and families throughout the state. All information was accurate to the author's knowledge as of December 2025. The report in its current form has been presented to several legislators.
Download it below.
Data Sources
This report draws on reputable publicly available sources.
Takeaways
Rare diseases affect 7 to 8 percent of the nation's population, and Delaware is no exception. However, the state lacks the infrastructure to quantify the incidence and prevalence of these conditions within its borders.
Estimates suggest that up to 30 million Americans live with rare disease. The exact number of patients or diagnoses in Delaware remains unknown.
The FDA has approved more than 40 gene therapies, highlighting the need for transparent prior authorization criteria for treatment.
The state is currently exploring several mechanisms to pay for high-investment gene and cellular therapies that can cure rare diseases.
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Op Eds: ​
Lee: Those Living With Rare Disease Need Aid
Lee: Why We Should Embrace biomarker Testing
Lee: The Case for a Rare Disease Registry in Delaware
Lee: The Case for Newborn Genetic Testing