The model was designed to help improve health outcomes, improve access to care, and lower costs for gene therapy.
In a statement, the Biden-Harris Administration announced that sickle cell disease (SCD) will be the first focus of the Cell and Gene Therapy (CGT) Access Model, originally announced in February 2023. The access model was designed to help improve health outcomes, improve access to care, and lower costs for gene therapy.1
This announcement follows the FDA approval of Casgevy (Vertex Pharmaceuticals Inc) and Lyfgenia (Bluebird Bio Inc), 2 cell-based gene therapy treatments which were approved in December 2023 for those aged 12 years and older with SCD.2 Casgevy was the first treatment with CRISPR/Cas9 to be approved by the FDA, according to an article in Pharmacy Times.2
In a statement released by the American Society of Hematology (ASH) President Mohandas Narla, DSc, from the New York Blood Center Enterprises, said, “We recently celebrated the approval of the first gene therapies for SCD, a significant step forward for the SCD community. However, we also voiced concerns regarding the high cost of these treatments and are glad to see CMS trying to address this issue with the development of this model . . . The Society commends [the Centers for Medicare & Medicaid Services (CMS)] for incorporating coverage of fertility preservation services, travel expenses, case management, and behavioral health services into the model’s framework, and continues to advocate for increased access to all available treatments and interventions for those living with this blood condition.”3
With the CGT Access Model, CMS will work with participating states and manufactures to cultivate new framework that expands access to gene therapies for SCD. CMS will also negotiate outcome-based agreements with various manufacturers to line up pricing with health outcomes of the therapies. As part of the negotiations, CMS will also include pricing rebates and a standardized access policy.1
Additionally, states that are participating can also decide to enter into an agreement with the manufacturers based on negotiate terms as well as offer the agreed-upon standard access policy for rebates that were negotiated by CMS.1
Furthermore, CMS will negotiate the financial and clinical outcome measures with the manufacturers, including reconciling data, monitoring results, and evaluating outcomes. The CGT Access Model will begin January 2025, and states can choose to begin participation between January 2025 and January 2026, according to the statement.
As part of the model, CMS also intends to address additional care gaps and barriers to access for those with cell and gene therapy during the negotiation process, including a defined scope of fertility preservation services, according to the release. Funding will also be offered to states that participate in activities increasing access to cell and gene therapy as well as promoting comprehensive care for those with Medicaid undergoing gene therapy for SCD.1
The CGT Access Model could be expanded to other types of CGTs in the future.1
According to an article published by Pharmacy Times, individuals who have severe SCD have a high interest in potentially curative gene therapy treatment and are even willing to accept the risk for a potential cure. The current standard of care for SCD is hydroxyurea, which is for symptom management. Bone marrow transplantation can cure the disease if successful, though transplantation includes finding a matching donor and has a high risk of rejection.4
However, such advancements in bone marrow transplantation have been made. In an interview with Pharmacy Times, Robert A Brodsky, MD, the 2023 president of the American Society of Hematology and professor of medicine at Johns Hopkins University School of Medicine, highlighted a new treatment option for SCD, haploidentical bone marrow transplant.5
“You no longer need a perfectly matched sibling, and the cure rate is 90%. It's also 10 times cheaper than gene therapy, and this is available now,” he said in the interview. “There is a something called a Wiz transcription factor that is very important for hemoglobin switching, and so this is kind of the future.”5
He added that although this is an improvement in care for SCD, not everyone has needs or can get bone marrow transplants, highlighting the need for more drug development for the disease.5