Nearly one year ago, the Food and Drug Administration (FDA) approved two new gene therapies for the treatment of sickle cell disease.\u00a0 The Sickle Cell Disease Association of America Inc. (SCDAA) is pleased that the manufacturers of these two FDA-approved gene therapy treatments have entered into agreements with the Centers for Medicare & Medicaid Services (CMS) to participate in the Cell and Gene Therapy (CGT) Access Model. These cutting-edge treatments are poised to make a difference in the lives of many sickle cell warriors, but their high price tags are a barrier to access. The CGT Access Model is a promising effort to reduce cost for these potentially curative therapies for eligible individuals, allowing more patients to benefit from these significant advancements in treating diseases. According to an announcement from the Department of Health and Human Services, the model \u201cwill test outcomes-based agreements for cell and gene therapies, with the aim to improve health outcomes, increase access to cell and gene therapies, and lower health care costs.\u201d We are also heartened to see that the model will provide fertility preservation for patients, marking an important acknowledgment of quality-of-life standards for our community.<\/p>\n
The Model will launch in January 2025, and all 50 states may choose to begin participation anytime between January 2025 and January 2026. SCDAA looks forward to working with our community-based organizations and other stakeholders to advocate for state enrollment. The CGT Access Model will provide crucial support to patients where available, and we encourage full participation across the country.<\/p>\n