Cell and Gene Therapy (CGT) Access Model

The Cell and Gene Therapy (CGT) Access Model aims to improve the lives of people with Medicaid living with rare and severe diseases by increasing access to potentially transformative treatments. Cell and gene therapies have high upfront costs but have the potential to reduce health care spending over time by addressing the underlying causes of disease, reducing the severity of illness, and reducing health care utilization. Initially, the model will focus on access to gene therapy treatments for people living with sickle cell disease, a genetic blood disorder that disproportionately affects Black Americans.

The CGT Access Model is a multi-year voluntary model for states and manufacturers. CMS anticipates states to begin with a rolling start in January 2025. CMS released a Request for Application (RFA) to manufacturers (PDF) in March 2024 and released an RFA to states (PDF) in June 2024. A Notice of Funding Opportunity (NOFO) to states is anticipated for later in summer 2024. States may choose to participate in the model under a participation agreement with CMS by responding to the state RFA. States may apply for model funding by applying to the NOFO, but they are not required to respond to the NOFO to participate in the model. States may begin participation anytime between January 2025 and January 2026.

Highlights

  • Cell and gene therapies can transform the lives of people living with rare and severe diseases that are hard to treat, but gaining access to these potentially life-changing treatments can be difficult because they can cost millions of dollars.
  • The Cell and Gene Therapy (CGT) Access Model aims to improve health outcomes for people with Medicaid who could benefit from cell and gene therapies by supporting outcomes-based agreements between states and manufacturers that will provide for treatments within a framework that lowers prices for states and ties payment to outcomes. This framework is intended to make it easier for states to pay for cell and gene therapies.
  • By increasing access to transformative therapies, this model can potentially help address the historic disparities, poor health outcomes, and low life expectancy associated with sickle cell disease. Other conditions might be added to the model over time.

Background

The CGT Access Model was developed in response to President Biden’s Executive Order 14087, “Lowering Prescription Drug Costs for Americans” and was first proposed in a report responding to the executive order directed by the Secretary of the Department of Health and Human Services.

Cell and gene therapies are a rapidly growing class of one-time treatments, many of which are developed to treat rare and severe diseases, such as sickle cell disease. They can correct underlying causes of a disease, address symptoms, and halt disease progression. However, the high cost of these treatments makes it difficult for state Medicaid agencies to pay for them.

Model Overview

The CGT Access Model is a voluntary model for states and manufacturers that tests whether a CMS-led approach to developing and administering outcomes-based agreements (OBAs) for cell and gene therapies improves Medicaid beneficiaries’ access to innovative treatment, improves their health outcomes, and reduces health care costs and burdens to state Medicaid programs. Under the model:

  • CMS and pharmaceutical manufacturers negotiate a set of key terms, which would include pricing (that is reflective of rebates paid by the manufacturer) and outcome measures that would form the basis for individual contracts between the manufacturer and participating states.
  • State Medicaid agencies decide whether to sign the negotiated contract. If agreed, manufacturers will be obligated to provide states with supplemental rebates that reflect model-negotiated terms. In turn, states will be obligated to implement an agreed-upon standard access policy.
  • CMS will provide support through technical assistance and funding to participating states. Technical assistance would include supporting the implementation, data collection, and reconciliation of the agreements and other model activities. Funding support would be conducted through a cooperative agreement and will help states cover the costs of participation in the model, including the state obligations related to provider requirements, data collection, and activities to ensure equitable access to care.
OBA Process - Step 1: CMS directly negotiates OBA Key Terms with a manufacturer. Step 2:States decide whether to enter into agreements with the manufacturer reflecting OBA Key Terms. Step 3: CMS supports implementation, reconciliation, and evaluation.

The OBAs will include additional sources of savings, such as pricing concessions or rebates tied to volume.

The goals of the model and the OBAs are to:

  • Test innovative payment and service delivery models to reduce program expenditures under the applicable titles while preserving or enhancing the quality of care.
  • Improve patient health outcomes by increasing their ability to receive cell and gene therapies.
CGT Access Model Infographic, showing start dates and providing information on Sickle Cell Disease

The model will focus initially on expanding access to gene therapy for sickle cell disease. The model will help address access to critical supports and services for people with Medicaid who receive gene therapy for sickle cell disease in a participating state. Patients receiving gene therapy for sickle cell disease will need to go through an extended inpatient hospital stay, and potentially multiple additional visits to a gene therapy center. Additionally, the process of preparing for this gene therapy involves myeloablative chemotherapy, which typically renders patients (male or female) infertile. Therefore, the model will include coverage of a defined scope of fertility preservation services and supports for ancillary services, including travel expenses, case management, and behavioral health services.

Health Equity Strategy

Sickle cell disease, the initial focus of the CGT Access Model, is a devastating and painful genetic blood disorder, which afflicts about 1 out of every 365 Black or African American births and about 1 out of every 16,300 Hispanic American births, according to the Centers for Disease Control and Prevention sickle cell disease fact sheet (PDF). People with sickle cell disease have a life expectancy more than 20 years shorter than the average life expectancy in the United States. Many people who have sickle cell disease are enrolled in Medicaid and are from underserved communities with limited access to specialized care and experience a host of health disparities.

By increasing access to transformative therapies, this model can potentially help address the historic disparities, poor health outcomes, and low life expectancy associated with sickle cell disease.

CMS might include additional cell and gene therapies for other diseases in the model at a future time.

Model Participation

Participation in the CGT Access Model will be voluntary for both manufacturers and states, with participation open to all states that meet model requirements on a non-competitive basis.

  • States: All states and U.S. territories that participate in the Medicaid Drug Rebate Program (MDRP) are eligible to apply to participate in the model. Participating states must implement management, operational and system requirements to support the model. These include changing reimbursement structures to appropriately reimburse for gene therapy; aligning coverage policies with Medicaid managed care organizations, as applicable; and contracting with out-of-state providers as may be necessary.
  • Manufacturers: Participating manufacturers must have a cell or gene therapy approved or licensed by the U.S. Food and Drug Administration for sickle cell disease launched by May 2024. CMS invited manufacturers to participate in OBA negotiations beginning in the spring of 2024.

Providers will not be formal participants in the model. However, providers will play an important role in delivering care and submitting data. CMS plans to work with existing patient registry infrastructure to facilitate data flows.

Qualifying for Gene Therapy as Part of the Model

To be eligible for gene therapy to treat sickle cell disease as part of this model, a person must:

  • Have a documented medical diagnosis for sickle cell disease.
  • Be enrolled in Medicaid or CHIP (if applicable) in a state participating in the model at time of therapy.
  • Beneficiaries for whom Medicaid is the primary payer.
  • Receive a gene therapy from a participating manufacturer.
  • Meet standardized prior authorization criteria established through the OBAs.

Additional Information

Model Background Resources

Executive Order on Lowering Prescription Drug Costs for Americans

Sickle Cell Informational Resources

Technical Documents

Past Events

Outreach

If you are interested in receiving additional information, updates or have questions about the Cell and Gene Therapy Access Model, please:

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