Bluebird and Vertex’s sickle cell gene therapies to kick off new Medicaid access model

The Biden Administration said Wednesday that it has secured agreements with Vertex Pharmaceuticals and bluebird bio for their sickle cell disease gene therapies to be the first in a new access model from the Centers for Medicare and Medicaid Services.

The voluntary model looks to solve one of the major issues with cell and gene therapies so far: the lack of commercial uptake. It will test outcomes-based agreements for cell and gene therapies, tying payments to whether the therapy improves health outcomes for people in Medicaid.

CMS said in a statement that it negotiated outcomes-based agreements with both Vertex and bluebird on behalf of states, which can sign up to participate.

“The Cell and Gene Therapy Access Model launches in January 2025, and states may choose to begin participation anytime between January 2025 and January 2026. The state application portal goes live this month — December 2024 — and will remain open through February 28, 2025,” CMS said.

The FDA approved the gene therapies, known as Lyfgenia (lovotibeglogene autotemcel) from bluebird bio, and Casgevy (exagamglogene autotemcel) from Vertex, in December 2023. Casgevy has a list price of $2.2 million in the US, and Lyfgenia’s list price was set at $3.1 million per treatment — but may potentially offer a curative therapy for a lifelong and costly disease.

Liz Fowler, deputy administrator and director of the CMS Innovation Center, said in a statement that the therapies’ high costs “can pose challenges to state budgets. This model will afford state Medicaid agencies more budget predictability while helping improve access to these innovative therapies for people with Medicaid with sickle cell disease.”

CMS said states can apply for optional model funding (totaling $95 million) by responding to the notice of funding opportunity.

PackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.

Related News

Related Services