Overcoming Obstacles to Access, Affordability, and Availability of Rare Disease Treatment
Pharma Live • Mon Aug 04 2025
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. 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.
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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About AscellaHealth
Healthcare today is more complex than ever—especially when it comes to therapies for people living with rare and chronic conditions. These high-cost, high-impact treatments account for only a small fraction of prescriptions, but they make up the largest and fastest-growing share of total drug spend. As a result, balancing affordability and quality of care remains a significant challenge.
We’re here to change that.
Because at AscellaHealth, we believe that all patients in need should have the ability to access life-changing therapies.
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