CGT (Cell and Gene Therapy) Access Model: Executive Summary and Strategic Architecture Analysis

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1. Executive Summary and Strategic Context

What Is This Model and What Problem Does It Solve?

The Cell and Gene Therapy (CGT) Access Model is a voluntary, multi-year CMMI innovation model designed to expand Medicaid beneficiary access to high-cost, potentially curative gene therapies through a novel federal negotiating framework. Operating under Section 1115A of the Social Security Act, the model tests whether CMS-led negotiation of outcomes-based agreements (OBAs) on behalf of state Medicaid agencies can increase access to transformative treatments, improve patient health outcomes, and reduce financial burden on state Medicaid programs.

The core problem the model addresses is structural: gene therapies for rare and severe diseases carry list prices measured in millions of dollars per patient. The two FDA-approved gene therapies for sickle cell disease (SCD) — Casgevy (exagamglogene autotemcel) from Vertex Pharmaceuticals and Lyfgenia (lovotibeglogene autotemcel) from bluebird bio (subsequently transferred to Genetix Biotherapeutics Inc.) — were priced at $2.2 million and $3.1 million per dose, respectively, following their December 2023 FDA approvals. These prices create an acute affordability crisis for Medicaid, which is the primary payer for approximately 50–60% of the more than 100,000 Americans living with SCD. The total annual economic burden of SCD on the U.S. health system is estimated at $2.98 billion per year.

State Medicaid agencies are legally required to cover FDA-approved drugs under the Medicaid Drug Rebate Program (MDRP) but have historically been unable to negotiate meaningful value-based or outcomes-based rebates for gene therapies independently. States have reported three specific barriers: (1) inadequate leverage against manufacturers due to small individual SCD populations, (2) insufficient in-house expertise and capacity to negotiate complex OBA endpoints, and (3) prohibitive administrative burden of multi-year data collection and reconciliation required to operationalize outcomes-based payments.

The CGT Access Model directly addresses all three barriers. CMS negotiates centralized key terms — including outcomes-based rebates, volume-based rebates, guaranteed rebates, and fertility preservation service obligations — on behalf of all participating states simultaneously, pooling beneficiary populations and institutional expertise at the federal level. States then decide whether to adopt those pre-negotiated terms through individual Supplemental Rebate Agreements (SRAs) with manufacturers, without having to conduct independent negotiations.

Strategic Significance for the VBC Market

This model represents the first instance of the federal government negotiating outcomes-based agreements with cell and gene therapy manufacturers on behalf of state Medicaid agencies. That distinction carries significant implications for the broader value-based care and pharmaceutical contracting landscape.

For Medicaid programs, the model creates a replicable procurement template for any high-cost gene or cell therapy that may receive FDA approval in the future. CMS has explicitly stated that other conditions and therapies may be added to the model over time, suggesting this framework is designed to scale beyond SCD. The model's early evaluation — covering March 2024 through December 2025 — confirmed that CMS-led negotiation was "a reasonably efficient alternative to state-by-state discussions" and that both participating manufacturers signed participation agreements on schedule.

The participation numbers underscore rapid market adoption: 34 state Medicaid programs and territories joined the model, representing states with and without prior value-based contracting experience. The evaluation found that 84% of Medicaid beneficiaries with SCD age 12 and older are covered by participating states, and that participation rates were more than twice as high among states with the highest SCD prevalence compared to those with the lowest. Non-participating states cited small SCD populations, state-specific legal or statutory barriers, or a preference to negotiate independently — not opposition to the OBA concept itself.

The model's first evaluation report found three concrete advantages of the CMS-led approach: (1) pooling beneficiaries across states gave CMS negotiating leverage no single state could achieve; (2) states lacking resources or negotiating experience benefited disproportionately; and (3) centralized rebate determinations reduced administrative burden for both states and manufacturers. The evaluation also noted that CMS's role as a neutral third-party intermediary was valued specifically for dispute resolution in cases of failed-outcome rebate triggers — a function no individual state could replicate in bilateral manufacturer negotiations.

For manufacturer participants, the model offered an important incentive: CMS provided reimbursement assurances to qualified treatment centers, which facilitated market access. Additionally, the CMS-sponsored model safe harbor under 42 CFR § 1001.952(ii) allows manufacturers to provide manufacturer-funded fertility preservation services to beneficiaries without running afoul of Anti-Kickback Statute exposure — a protection that could only be structured through an official CMS-sponsored model and would be unavailable in independent state negotiations.

Political Context and Administrative Alignment

The model was initiated under the Biden administration and launched January 1, 2025, the final days of that administration. Its model page was last modified October 27, 2026, indicating active continuation and operational status under the Trump administration led by CMS Administrator Dr. Mehmet Oz and CMMI Director Abe Sutton. The CMMI Innovation Insight referenced in the model update notes CMS is "broadening the framework of the CGT Access Model following positive initial results from the first model evaluation report," signaling Trump-era CMMI leadership has affirmatively chosen to expand rather than contract this initiative.

This is strategically significant. The CGT Access Model's design aligns with several durable policy priorities that transcend administrations: reducing drug costs for government programs, tying payment to outcomes, expanding access to innovative therapies, and reducing federal and state administrative burden. The model's reliance on voluntary participation, manufacturer-funded patient supports, and market-negotiated rebates rather than regulatory price controls also fits comfortably within the current administration's market-oriented policy preferences.

Market Transformation Implications

Beyond SCD, the CGT Access Model is best understood as infrastructure for Medicaid's engagement with the next generation of curative therapies. Gene therapies are in active development for hemophilia, Duchenne muscular dystrophy, certain forms of blindness, Parkinson's disease, and other rare conditions — many of which carry projected list prices in the millions of dollars per patient. The model's architecture — federal OBA negotiation, standardized access policy, CIBMTR-linked patient registry, manufacturer-funded ancillary services, and cooperative agreement funding for states — provides a replicable template for each of these future approvals.

The model also accelerates the creation of real-world evidence infrastructure. By requiring providers to participate in the Center for International Blood and Marrow Transplant Research (CIBMTR) registry and conditioning Medicaid payment on registry participation and standardized claims submission, CMS is building a longitudinal outcomes dataset for gene therapy that will directly inform future rebate structures, FDA labeling updates, and coverage policy decisions. This data infrastructure has value extending well beyond the current 11-year model performance period ending December 31, 2035.

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2. Model Architecture and Payment Structure

Participation Structure

The CGT Access Model operates as a three-party voluntary structure involving CMS (as negotiator and administrator), participating manufacturers, and participating state Medicaid agencies. Providers are not formal model participants but bear critical obligations as conditions of Medicaid payment.

Manufacturer Eligibility and Participation: Manufacturers must have held FDA approval or licensure for a gene therapy for SCD that was launched by May 2024. CMS invited manufacturers to participate beginning spring 2024, with the pre-implementation period running from May 1, 2024 through November 29, 2024. Manufacturers that reached agreement with CMS executed a Participation Agreement (PA) by November 29, 2024. Both eligible manufacturers — Vertex Pharmaceuticals Incorporated (Casgevy) and Genetix Biotherapeutics Inc. (Lyfgenia) — signed on schedule. Participating manufacturers must offer negotiated key terms to all participating states without exclusion; variation in key terms is permitted only as necessary to comply with state law, and only with CMS approval.

State Eligibility and Participation: All states, the District of Columbia, and U.S. territories participating in the MDRP were eligible to apply. States submitted applications under the State RFA, originally posted June 2024 and updated February 18, 2025 to extend the application deadline to March 14, 2025. States must execute a State Agreement (SA) with CMS by no later than June 1, 2025 to become participants. Upon SA execution, states then execute individual Value-Based Purchasing Supplemental Rebate Agreements (VBP SRAs) directly with participating manufacturers.

The 34 current participating Medicaid programs span 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, plus the District of Columbia and Puerto Rico.

Cooperative Agreement (Optional Funding): States may separately apply for optional funding through a Notice of Funding Opportunity (NOFO). Eight states/territories have been awarded Cooperative Agreement funding: Connecticut, District of Columbia, Illinois, Mississippi, North Carolina, Pennsylvania, Rhode Island, and Vermont. Cooperative Agreements begin as early as June or July 2025 and extend through December 31, 2035. Two funding types are available:

Funding Type	Purpose
Implementation Funding	Staff time, infrastructure costs, community-based organization partnerships
Milestone Funding	Achievement of access milestones for SCD gene therapy and multidisciplinary care

Model Beneficiary Eligibility: To receive gene therapy under the model, a beneficiary must:

• Have a documented medical diagnosis of sickle cell disease
• Be enrolled in Medicaid or CHIP in a participating state at time of therapy
• Have Medicaid as the primary payer
• Receive a gene therapy from a participating manufacturer
• Meet standardized prior authorization criteria established through the OBAs
• Be age 12 or older (per FDA approval requirements for both therapies)

Separate CHIP beneficiary inclusion is optional for both states and manufacturers but is subject to distinct "separate CHIP Key Terms" negotiated by CMS and subject to a model-specific waiver excluding separate CHIP units from Average Sales Price (ASP) calculations under 42 U.S.C. § 1847A(c).

Payment Methodology

The financial architecture of the CGT Access Model rests on a multi-layered rebate structure negotiated centrally by CMS and operationalized through state-level VBP SRAs.

Rebate Type	Description
Outcomes-Based Rebates	Triggered when pre-specified clinical Outcome Measure Benchmarks are not achieved; amount linked to pricing calculation
Guaranteed Rebates	Applied to all units regardless of outcome or volume; model-specific and additive to existing statutory MDRP rebates
Volume-Based Rebates	Paid by manufacturer based on number of eligible units administered during the Volume Accrual Period
Statutory MDRP Rebates	Continue in parallel; model OBA supplements but does not replace existing statutory rebate obligations

The Key Terms — the central parameters negotiated between CMS and each manufacturer — govern all financial arrangements and include:

• Outcome Measures: Agreed-upon clinical or utilization-based factors (e.g., vaso-occlusive crisis rates, hemoglobin F levels, patient-reported outcomes) linked to rebate triggers
• Outcome Measure Benchmarks: Measurable performance thresholds assessed at defined intervals during the Measurement Period
• Measurement Period: The time period following drug administration during which outcomes are monitored for each patient cohort
• Reconciliation Period: The period following the Measurement Period's conclusion in which final rebate calculations are settled; may include interim and final payment stages
• OBA Term: Expected to be no more than 6 years (e.g., PYs 1–6 in an 11-year model)
• Volume Accrual Period: Time window during which administered units accumulate toward volume-based rebate thresholds before resetting

Provider Reimbursement Requirements: States must reimburse providers (hospitals, treatment centers, specialty pharmacies) for Model Drugs via direct reimbursement, not as part of a bundled DRG or APC inpatient payment. Payment must not be less than the provider's Actual Acquisition Cost (AAC) as defined in 42 CFR § 447.502. States must carve Model Drugs out of inpatient bundles where necessary to preserve their status as "covered outpatient drugs" eligible for MDRP rebate treatment.

Billing specifics differ by drug:

• Casgevy: Must be reimbursed separately from ancillary services including gene therapy administration, pre- and post-treatment regimens, and hospitalization
• Lyfgenia: May be billed as separate claims or as part of inclusive payments if drug costs are itemized on the provider's bill with unit count, NDC identifier, and corresponding HCPCS codes

340B Program: Does not apply to inpatient-administered Model Drugs; states may direct covered entities not to acquire Model Drugs through the 340B program for model beneficiaries.

Manufacturer-Funded Fertility Preservation: A uniquely structured financial obligation requires participating manufacturers to fund a defined scope of fertility preservation services at no cost to Medicaid beneficiaries or state programs. This covers harvesting, freezing, and storage of reproductive material — necessary because myeloablative chemotherapy administered prior to gene therapy infusion typically renders patients permanently infertile. Coverage also extends to ancillary services including travel expenses and lodging associated with fertility preservation services. This obligation is protected from Anti-Kickback liability through the CMS-sponsored model safe harbor at 42 CFR § 1001.952(ii).

Performance Measures and Quality Requirements

Registry Participation: Providers must be active members of the CIBMTR (Center for International Blood and Marrow Transplant Research) registry and must seek patient consent for the CIBMTR Research Database study protocol as a condition of Medicaid payment. CIBMTR collects both clinical data and patient-reported outcomes (PRO) data. Providers that do not comply with registry requirements cannot bill for Model Drugs or their administration under participating state Medicaid programs.

Data and Reporting Obligations (Duration by Requirement Type):

Requirement Category	Duration
Operational requirements (Sections 3.1–3.2)	Duration of the OBA Term (up to PYs 1–6)
Beneficiary access requirements (Section 3.3)	OBA Term + Measurement Period (approximately PYs 1–9)
Data and reporting requirements (Section 3.4)	Full model duration (PYs 1–11, through December 31, 2035)

Access Policy Standardization: States must establish and publish a standardized access policy aligned with CMS-negotiated Key Terms, covering prior authorization criteria, utilization management, and provider qualification standards. State policies may be more permissive but may not be more restrictive than the Key Terms access policy. The same policy must apply uniformly to FFS and managed care beneficiaries within the state.

Managed Care Alignment: States must ensure Medicaid managed care organizations (MCOs), prepaid inpatient health plans (PIHPs), and prepaid ambulatory health plans (PAHPs) apply the same access, reimbursement, and claims standards as FFS. States must amend managed care contracts, submit state-directed payment preprints as needed, and ensure continuity of care for beneficiaries switching between FFS and managed care — including honoring prior authorizations and maintaining access to the same gene therapy providers for at least one year after gene therapy infusion.

Model Timeline

Milestone	Date
Manufacturer RFA released	March 7, 2024
Model pre-implementation period begins	May 1, 2024
CMS invites manufacturers to negotiate	Spring 2024
Model pre-implementation period ends	November 29, 2024
Manufacturer PA execution deadline	November 29, 2024
Key Terms disclosed to states	December 2024
Letter to Treatment Centers issued	December 4, 2024
Model Performance Period Year 1 begins	January 1, 2025
State RFA updated (deadline extended)	February 18, 2025
State RFA application deadline	March 14, 2025
State Agreement execution deadline	June 1, 2025
Cooperative Agreements begin	June/July 2025
Managed care beneficiaries required by	January 1, 2026 (PY2 start)
Separate CHIP beneficiaries required by (if applicable)	January 1, 2026
OBA Term maximum end date	December 31, 2030
Model Performance Period ends	December 31, 2035
Total model duration	11 Performance Years

Cohort Structure: Under the model's example framework, gene therapy administration occurs during PYs 1–6. Beneficiaries treated in each year constitute a separate cohort with its own measurement timeline. Outcome measurement begins the year after administration. Final rebate reconciliation follows completion of each cohort's Measurement Period. This staggered structure means states maintain data collection and reporting obligations through the full 11-year model duration (PY11 ending December 31, 2035), even though drug administration under the OBA Term ends after PY6 (December 31, 2030).

States may select their individual Performance Period Start Date from January 1, 2025 to January 1, 2026, giving early adopters up to 12 additional months of cohort accrual compared to late joiners — a meaningful difference given the small eligible population and per-patient financial stakes at these therapy price points.