New Strategy for Gene Therapy Treatment of Sickle Cell Disease May Serve as a Blueprint
The Centers for Medicare and Medicaid Services (CMS) has announced a new voluntary program aimed at improving patient access and lowering costs for gene therapies targeting sickle cell disease (SCD). This initiative, known as the Cell and Gene Therapy (CGT) Access Model, was initiated by the Biden administration and continued by the Trump administration [1].
As of mid-2025, 33 states, along with the District of Columbia and Puerto Rico, have joined the program. This covers approximately 84% of Medicaid beneficiaries with SCD [3][4]. The program focuses on two novel gene therapies: Lyfgenia, manufactured by bluebird bio, and Casgevy, produced by Vertex Pharmaceuticals [2].
The CGT Access Model establishes outcomes-based agreements (OBAs) between CMS and these manufacturers. Medicaid payments are tied to the clinical outcomes of the therapies. If the therapies fail to deliver promised benefits, manufacturers must provide discounts or rebates to the states [3][4]. This approach aims to protect Medicaid budgets and potentially lower costs.
California's Medicaid program (Medi-Cal) has already received CMS approval to cover Casgevy effective September 1, 2025, and Lyfgenia as of July 1, 2025 [2]. The program offers optional federal support of up to $9.55 million per state for implementation, outreach, and data tracking [1].
SCD predominantly affects people of sub-Saharan African descent, with approximately 100,000 individuals living with the disease in the United States [1]. The most common SCD type is sickle cell anemia. Gene therapies hold the promise of groundbreaking improvements in health outcomes across multiple disease areas, making overcoming access obstacles crucial [1].
In the cell and gene therapy space, access to costly treatments is a challenge. Payers must find novel ways to pay while generating evidence on effectiveness and safety [5]. All cell and gene therapy manufacturers face a challenging environment with regulatory hurdles, manufacturing challenges, patient preparation issues, and coverage restrictions imposed by payers [6].
The launch prices for Casgevy and Lyfgenia are $2.2 million and $3.1 million, respectively [1]. These therapies have the potential to decrease or potentially eliminate pain crises in SCD patients [1]. Manufacturers of these therapies must provide states with supplemental rebates reflecting model-negotiated terms [2].
Insurers must address questions about real-world health outcomes, treatment promises, safety concerns, and manageable side effects. The disease alters the structure of hemoglobin, causing severe pain, anemia, organ damage, and infections, and results in a shorter life expectancy [1].
The CGT Access Model seeks to make transformative gene therapies for sickle cell disease more accessible to Medicaid patients, improve health outcomes by incentivizing effective treatment, and reduce long-term healthcare expenses through value-based purchasing agreements [1][3][4]. The program is voluntary for states and manufacturers but currently focuses on sickle cell gene therapies, with plans to potentially add other rare disease CGTs in the future [2][3].
The SCD model being experimented with could serve as a blueprint for other cell and gene therapies that have faced considerable barriers with respect to patient access. By coordinating evidence gathering and contracts across state Medicaid agencies, a more efficient process and improved access for a majority of SCD sufferers can be achieved.
- With the new Cell and Gene Therapy (CGT) Access Model, the Centers for Medicare and Medicaid Services (CMS) aims to improve access and lower costs for gene therapies targeting sickle cell disease (SCD), focusing on science and medical-conditions within the health-and-wellness sector.
- As a result of the program, 84% of Medicaid beneficiaries with SCD in 33 states, along with the District of Columbia and Puerto Rico, will have access to two novel gene therapies: Lyfgenia and Casgevy, suggesting an intersection between finance, lifestyle, technology, and education-and-self-development.
- The program establishes outcomes-based agreements (OBAs) between CMS and manufacturers like bluebird bio and Vertex Pharmaceuticals, linking Medicaid payments to the clinical outcomes of the therapies, demonstrating an overlap between science, general-news, and nutrition.
- In order to protect Medicaid budgets and potentially lower costs, the agreements require manufacturers to provide discounts or rebates if the therapies fail to deliver promised benefits, highlighting the importance of financial considerations in the health-and-wellness sector.
- The program could serve as a blueprint for other cell and gene therapies, addressing access obstacles and paving the way for groundbreaking improvements in health outcomes across multiple disease areas, bridging the gap between science, technology, and education-and-self-development.
