CMS Announces Cell and Gene Therapy Access Model Will Focus on Sickle Cell Disease
The model was designed to help improve health outcomes, improve access to care, and lower costs for gene therapy.
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The model was designed to help improve health outcomes, improve access to care, and lower costs for gene therapy.
By Genoa Barrow | OBSERVER Senior Staff Writer - The FDA has approved the first two gene therapies to treat sickle cell disease, providing hope for patients, their families, and the entire sickle cell community, but the treatments are expensive and require specialized care to be effective.
He also provides insight for students training in classical hematology, oncology hematology, or oncology pharmacy.
The decision was supported by efficacy data from 36 patients from the ongoing phase 1/2 HGB-206 trial (NCT02140554) and 2 patients in the phase 3 HGB-210 trial (NCT04293185). It was the second approval from FDA for a gene therapy for sickle cell disease.
The president highlights a new curative treatment option for sickle cell disease while also expanding on the changing treatment landscape for hematologic conditions.