Overcoming Obstacles to Access, Affordability, and Availability of Rare Disease Treatment

By Clayton Edwards, AscellaHealth

Any parent can relate to the anxiety of caring for a sick child – but when a newborn or young child is diagnosed with a rare disease, the uncertainty and lack of clear answers can be devastating. A rare disease is defined by the FDA as a disease or condition that affects less than 20,000 individuals in the U.S. alone. There are currently 7,000 known rare diseases which are impacting over 30 million Americans. For families affected by rare conditions, the emotional and physical toll is immense. Caregivers often find themselves stepping into multiple roles: researcher, advocate, care coordinator and educator – navigating a fragmented healthcare system while managing the complexities of the disease itself.

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