Facilitating access to treatments for sickle cell disease will be the first order of business of the Biden-Harris Administration’s Cell and Gene Therapy (CGT) Access Model, a blueprint for alleviating health care burdens for sickle cell disease patients enrolled in Medicaid.
Starting next year, the Centers for Medicare and Medicaid Services (CMS) will act as a proxy between states and manufacturers to negotiate access policies, outcome-based agreements, and pricing rebates for cell and gene therapies. Once an agreement has been reached, states partaking in the model will be given the option of partnering with the manufacturers based on the terms that were agreed upon.
“By negotiating with manufacturers on behalf of states, CMS can ease the administrative burden on state Medicaid programs so they can focus on improving access and health outcomes for people with sickle cell disease,” said Liz Fowler, CMS Deputy Administrator and Director of the CMS Innovation Center, which will test these agreements
The CGT model provide participating states the opportunity to select any start date between January 2025 and January 2026. In addition, the Centers for Medicare & Medicaid Services (CMS) plans to offer optional funding to states that take steps to increase equitable access to cell and gene therapies and advocate for quality care in sickle cell disease patients that are undergoing gene therapy and enrolled in Medicaid.
The CGT Access Model is the first of three models—proposed in February 2023 to address health care burdens—to be implemented. The other two models called “The Medicare $2 Drug List” and “The Accelerating Clinical Evidence Model” are aimed at lowering drug costs for chronic conditions and reducing Medicare spending on drugs that have yet to demonstrate clinical efficacy respectively.
The focus on sickle cell disease treatment access comes after recent high-profile approvals that have introduced new therapies. In December 2023, the US Food and Drug Administration (FDA) approved Vertex Pharmaceuticals and CRISPR Therapeutics’ Casgevy (exa-cel) and bluebird bio’s Lyfgenia (lovo-cel) as its first two gene therapies for the treatment of SCD in patients 12 years and older.
Casgevy is the first FDA-approved therapy to utilise CRISPR/Cas9 genome editing technology to modify a patient’s haematopoetic stem cells. GlobalData predicts Casgevy to bring in $593m in 2029.
Lyfgenia, on the other hand, uses a lentiviral vector delivery vehicle to modify a patient’s blood stem cells to produce new haemoglobin that functions in place of the dysfunctional inherent haemoglobin. GlobalData expects Lyfgenia usher in $306m in 2029.
GlobalData is the parent company of Pharmaceutical Technology.
Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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