CGT (Cell and Gene Therapy Access) Model

Background

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.

Goals

The goals of the model and OBAs are:

• Improve patient health outcomes by increasing their ability to receive cell and gene therapies.
• Test innovative payment arrangements to reduce health care costs and administrative burden for Medicaid programs.

Innovation

The model is the first time the federal government has negotiated outcomes-based agreements with CGT manufacturers on behalf of state Medicaid agencies.  Other conditions might be added to the model over time.

Design

Under the CGT Access Model:

• CMS and pharmaceutical manufacturers negotiate a set of key terms, which would include pricing discounts and outcomes-based rebates that would form the basis for individual contracts between the manufacturer and participating states.
• State Medicaid agencies decide whether to sign the negotiated contract. Manufacturers are obligated to provide states that participate in the model with supplemental rebates that reflect model-negotiated terms. In turn, states must implement an agreed-upon standard access policy.
• CMS provides technical assistance and optional funding support to participating states. Technical assistance includes supporting the implementation, data collection, and reconciliation of the agreements and other model activities. Optional funding support is provided through a cooperative agreement and helps states cover the costs of participation in the model, including the state obligations related to provider requirements, data collection, and activities to ensure access to care.
   

Gene Therapy for Sickle Cell Disease

The model’s initial focus is 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 requires participating manufacturers to pay for a defined scope of fertility preservation services and supports for ancillary services related to receiving fertility preservations services, including travel expenses and lodging.

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
• Have Medicaid as their primary payer
• Receive a gene therapy from a participating manufacturer
• Meet standardized prior authorization criteria established through the OBAs

Participating Medicaid Programs: Arizona, Arkansas, California, Colorado, Connecticut, Delaware, Florida, Illinois, Kansas, Kentucky, Louisiana, Maryland, Michigan, Mississippi, Missouri, New Jersey, New York, North Carolina, Ohio, Oklahoma, Oregon, Pennsylvania, Rhode Island, South Carolina, Tennessee, Texas, Utah, Vermont, Virginia, Washington, West Virginia, and Wisconsin, as well as the District of Columbia and Puerto Rico.

Of these participating Medicaid programs, the following have applied for and been awarded Cooperative Agreement funding: Connecticut, District of Columbia, Illinois, Mississippi, North Carolina, Pennsylvania, Rhode Island, and Vermont.

Participating Drug Manufacturers: Genetix Biotherapeutics Inc. and Vertex Pharmaceuticals Incorporated.

Participation in the CGT Access Model is voluntary for both manufacturers and states. Participation was open to all states that met model requirements on a non-competitive basis.

• States: All states and U.S. territories that participate in the Medicaid Drug Rebate Program (MDRP) were 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 had a cell or gene therapy approved or licensed by the U.S. Food and Drug Administration for sickle cell disease that was launched by May 2024. CMS invited manufacturers to participate in OBA negotiations beginning in the spring of 2024.

Providers are not 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.

Participant Resources

• Letter to Treatment Centers that Administer Gene Therapy for Sickle Cell Disease (SCD)
• State RFA
• State NOFO
• Billing Information Guide

Background Resources

• Frequently Asked Questions
• Sickle Cell Disease Gene Therapy Care Journey (PDF)
• Press Release: CMS Expands Access to Lifesaving Gene Therapies Through Innovative State Agreements
• JAMA Viewpoint: The Cell and Gene Therapy Access Model

Sickle Cell Informational Resources

• CDC sickle cell disease webpage
• CDC fact sheets on sickle cell disease