CHA Applauds Actions Taken to Increase Access to Sickle Cell Disease Therapies

Children’s Hospital Association (CHA) issued the following statement today applauding the Centers for Medicare and Medicaid Services (CMS) and the gene therapy manufacturers Bluebird Bio™ and Vertex Pharmaceuticals™ for entering into a new agreement with a goal of increasing access to FDA-approved gene therapies for sickle cell disease:

"This agreement could transform the lives of many children and adolescents living with sickle cell disease. One year ago this month, the FDA approved two groundbreaking gene therapies for sickle cell disease, a condition that disproportionately affects Black and Latino communities. According to the CDC, approximately 1 in 365 Black children are born with sickle cell disease and about 1 in every 16,300 Hispanic American births, highlighting the urgent need for improved treatment options.

"Children’s Hospital Association applauds CMS for entering into an agreement to participate in the Cell and Gene Therapy Access Model (CGT Access Model). By partnering with states that participate in the program, the model aims to increase access to treatment while reducing costs.

"Children’s hospitals are leaders in the development and provision of these potentially curative therapies for sickle cell disease. At children’s hospitals across the country, world class pediatric specialists and care teams, along with hospital coordinators, provide care to children and adolescents and support their families as they undergo treatment to become healthier and live longer. The care is extraordinarily complex and specialized, requiring long, resource-intensive hospital stays, made more challenging by inconsistent insurance coverage.

"As states participate in the model, Medicaid beneficiaries will have greater access to these treatments. Most importantly, the model offers additional support for patients including fertility preservation and other health-related social needs. Children’s hospitals welcome the expansion of treatment access for individuals with sickle cell disease. CHA remains committed to collaborating with CMS to advocate for and support the model while encouraging all state Medicaid programs to participate, advancing health equity in addressing this life-altering disease."