Accelerating Payor, Legal and Procurement Pathways Paves the Way for Breakthrough Therapy for Pediatric Patient with Rare Disease at Children's National Hospital
BOSTON, Jan. 16, 2024 -- Clearway Health, a specialty pharmacy accelerator partnering with hospitals and health systems, has pioneered a model to support hospitals in enabling access to novel gene therapy treatments. The model was used to execute and accelerate payor, legal and procurement processes for Children's National Hospital in Washington, D.C., to treat their first pediatric patient with ZYNTEGLO®, a one-time, potentially curative breakthrough gene therapy treatment for transfusion-dependent beta-thalassemia, a rare blood disorder requiring regular red blood cell transfusions.
"Novel gene therapies represent the advancement of emerging therapies addressing the possibility of untethering patients from a lifetime of burdensome, costly, chronic care with a single treatment. While very promising, the full clinical impact of these treatments is not yet clear, driving questions on coverage, cost efficiency and delivery," said Shawn Francis, PharmD, director of specialty pharmacy at Clearway Health...
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