General Model FAQs
What entities are eligible to apply for participation in the CGT Access Model?
There are two types of participants in the model, states, and pharmaceutical manufacturers. All states and U.S. territories that participate in the Medicaid Drug Rebate Program (MDRP) are eligible to apply to participate in the model. Pharmaceutical manufacturers that participate in the MDRP and market U.S. Food & Drug Administration (FDA)-approved or -licensed gene therapies for the treatment of severe sickle cell disease are also eligible to participate in the model. Participation in the CGT Access Model will be voluntary for both manufacturers and states.
Why does the CGT Access Model begin with a focus on sickle cell disease?
Sickle cell disease is a devastating and painful disease that affects more than 100,000 people in the United States, the majority of whom are Black Americans. Before December 8, 2023, there was only one cure: allogenic hematopoietic stem cell transplantation, which requires a close donor match (typically from a healthy sibling). There are other FDA-approved therapies used to treat the symptoms associated with sickle cell disease, but they do not hold the same transformative potential as gene therapies.
In December 2023, the FDA approved two gene therapies for the treatment of certain patients with sickle cell disease. “Casgevy” from Vertex Pharmaceuticals and CRISPR Therapeutics was listed at $2.2 million per patient and “Lyfgenia” from bluebird bio was listed at $3.1 million per patient. These potentially life-changing therapies have shown great promise in improving patients’ health, preventing further organ damage, and boosting quality of life. Between 50% and 60% of people living with sickle cell disease are on Medicaid and the high price of these treatments may strain state Medicaid agencies’ budgets.
This model will help make it easier for states to provide access to such therapies to people with Medicaid who are eligible for such treatments.
What is an outcomes-based agreement and how does it work in the model?
An outcomes-based agreement (OBA) ties payment to the manufacturer to patients’ health outcomes over a certain time period. Under the model, if the gene therapy is less successful at improving health outcomes than expected, the drug manufacturer will reimburse the health care payer some portion of what was paid for the therapy. Agreements will also include other price concessions, such as volume-based rebates or guaranteed rebates.
CMS will negotiate with manufacturers on behalf of states for cell and gene therapies that are administered to beneficiaries for whom Medicaid is the primary payer. Instead of each state creating its own OBAs with manufacturers, the model will allow CMS to bring states together and negotiate on a multi-state basis. CMS will also be responsible for establishing financial and clinical outcome measures, reconciling data, and evaluating results. The states will still be responsible for their share of the cost of the cell and gene therapy, but at a pricing rebate tied to specific outcomes, as negotiated by CMS.
How will the CGT Access Model contribute to CMS’ health equity strategy?
CMS is initially focusing the CGT Access Model on gene therapies for sickle cell disease to increase access to potentially life-changing therapies for all individuals with sickle cell disease for whom gene therapy may be an appropriate option, including groups who have experienced historic disparities associated with this disease. Sickle cell disease is a genetic blood disorder associated with significant health disparities. Black Americans are disproportionately affected by sickle cell disease and often face disparities in accessing health care in general, as well as critical specialized care and treatment for sickle cell disease.
The model also will provide optional funding to states to encourage access to critical supports and services for people with Medicaid with sickle cell disease undergoing gene therapy, including behavioral health services and case management.
Will this model qualify as an Advanced or MIPS Alternative Payment Model (APM)?
No, health care providers are not participants in the model and thus it will not qualify as an Advanced or MIPS APM under the Quality Payment Program (QPP).
When will the model start and when will results be published?
The CGT Access Model will go live with a “rolling start” in January 2025; states will have the option to begin participation at a time of their choosing between January 2025 and January 2026. If additional disease conditions are added to the model in future years, states will be permitted to apply for model participation pertaining to that condition.
The performance period of the model will include the OBA term; the measurement period; and a reconciliation period to allow for data to flow in, and for payments under the OBA to be made. The performance period of the model would depend on the length of the OBA term, the measurement period, and the reconciliation period as negotiated with the manufacturer(s). If, for example, the length of the OBA term is six years (that is, covering patients who receive the therapy from 2025-2030); the measurement period is three years; and the reconciliation period is two years, then the model performance period would be 11 years. In this example, the first evaluation results would be published in 2027 and the final evaluation report would be published in 2035.
This model starts in 2025, do Medicaid and Medicare cover this therapy now?
Improving access to these therapies — both before and after the launch of the model — is a key goal of CMS. Prior to the launch of the model, current Medicare and Medicaid access standards will apply, which will result in access as currently required under law.
CGT Access Model State Request for Applications (RFA) (PDF) FAQs
Which states are eligible to apply to participate in the model?
All states, the District of Columbia, and U.S. territories that participate in the Medicaid Drug Rebate Program (MDRP) are eligible to apply to participate in the model. Participation in the Cell and Gene Therapy (CGT) Access Model will be voluntary for both manufacturers and states.
What is the process and timeline for states to apply for and participate in the CGT Access Model?
States can submit applications to participate in the model after the negotiated Key Terms are communicated to all states in December 2024. The application portal will go live in December 2024, and will be open through February 28, 2025. States seeking to participate in the model must complete and submit the application (see Appendix A of the State RFA) at least 10 business days prior to their requested Performance Period Start Date.
CMS will acknowledge receipt of the application to the Primary Application Contact and will review the application within 10 business days. CMS will then work with states to execute State Agreements (SAs) on a rolling basis. Once the SA is signed, the state officially becomes a participant in the model.
If more than one manufacturer participates in the model, do participating states have to enter into supplemental rebate agreements (SRAs) with all of them?
No. Participating states must select at least one Model Drug from a participating manufacturer as their “State-Selected Model Drug(s)” and adopt the Key Terms associated with that therapy. States may change their State-Selected Model Drug(s) during the model performance period, so long as they continue to meet the participation requirements with respect to maintaining at least one Model Drug and associated Key Terms at all times.
What are the requirements of states that participate in the model?
Participating states must implement policy, operational, and system requirements to support the model. These include having or obtaining the necessary authority to implement the model, including CMS approval of a state plan amendment (SPA) to enter into a value-based purchasing (VBP) SRA; establishing a standardized State-Selected Model Drug access policy, consistent with the CMS-Manufacturer negotiated Key Terms; carving State-Selected Model Drugs out of an inpatient payment bundle, if necessary; and aligning with their managed care plans by 2026, to name a few.
For more detail, see Section 3 of the State RFA. All requirements will be detailed in the SA.
What are the model requirements related to managed care alignment?
States must join the model with both their fee-for-service (FFS) and managed care populations. FFS beneficiaries must be included in the model on the first day of the state’s performance in the model. States may include their managed care enrollees as late as January 1, 2026.
States must coordinate with their managed care plans to ensure alignment with all model requirements, but they are free to choose how to manage their relationships with managed care plans. For example, adjusting kick payments (i.e., one-time, fixed, supplemental payments made to plans) or capitation rates, or carving the gene therapies out of their managed care contracts.
For more detail, see Section 3.1.6 of the State RFA. All requirements will be detailed in the SA.
How will CMS help states implement model requirements?
CMS will offer direct technical assistance to all state participants to support them in implementing model requirements. States can also reach out to their pharmacy contact at the Center for Medicaid and CHIP Services (CMCS) for technical assistance. Additionally, states may choose to apply for funding for activities related to model implementation (among other things) through the optional Notice of Funding Opportunity (NOFO), which will be published later this summer.
Does the model change the share of state Medicaid costs funded by the state or federal government?
The Model will not alter the division of financial responsibility between states and the federal government. States will continue to be responsible for their share of the cost of the model gene therapy, but subject to supplemental rebates negotiated by CMS. States will be obligated to pay for the gene therapy and will receive Medicaid matching funds from the federal government at the current Federal Medical Assistance Percentage (FMAP), consistent with current Medicaid policy.
Can CMS provide more guidance about the model requirement to make a separate payment to health care providers for the gene therapy?
States participating in the CGT Access Model (and their managed care plans) must make payments to providers for a State-Selected Model Drug in the form of direct reimbursement. If a state typically pays for inpatient drugs as part of a bundled payment (e.g., Diagnosis-Related Groups (DRGs); Ambulatory Payment Classifications (APCs)), it must carve the State-Selected Model Drug out of the inpatient payment bundle. This allows the Model Drug to be considered a "covered outpatient drug" and included in the MDRP. For more information, states can reach out to their pharmacy contact at the Center for Medicaid and CHIP Services.
If a state chooses to participate in the model, can it attempt to negotiate additional discounts or terms other than those negotiated by CMS?
No. If a state opts to participate in the model, it must enter into an agreement with the manufacturer that reflects the terms negotiated by CMS. Limited state-to-state variation may be permitted by CMS, as may be necessary in certain situations for a state to comport with their legislative and regulatory requirements.
For participating states, does separate billing for an included gene therapy mean that a hospital could purchase the Model drug under the 340B program?
Under the Model, administration of gene therapy for sickle cell disease will be for individuals who are inpatient. The 340B program does not apply to drugs used for individuals who are inpatient. Please see the Health Resources and Services Administration (HRSA) website for additional information.
Are states required to pay for fertility preservation services under the model?
No. Under the CGT Access Model, participating manufacturers will be required to financially support a defined scope of fertility preservation services that includes collection, cryopreservation, and storage of reproductive material (i.e., eggs, sperm, ovarian tissue, and testicular tissue) for at least five years and up to 15 years. For more information, see the Manufacturer Request for Applications (PDF) published on March 7, 2024. These services would be provided at no cost to beneficiaries receiving treatment within the model and at no cost to other payers. State participants must not claim any costs for services paid for by a manufacturer as state expenses for purposes of federal financial participation (FFP). States may (but are not required to) provide additional fertility preservation services (not covered by manufacturer participants) through the Medicaid family planning benefit.
How does the model facilitate access to out-of-state treatment?
States participating in the model must ensure beneficiaries have access to at least one qualified SCD gene therapy provider, either in-state or out-of-state. If a state does not have an in-state SCD gene therapy provider, it must pay for out-of-state services as per 42 CFR § 431.52.
Several model requirements are designed to help states plan more proactively for their beneficiaries to be able to receive out-of-state treatment, thus reducing delays in care for individual beneficiaries (see section 3.3.1 of the State RFA). For example, states must maintain a contract with a treatment center qualified to administer each State-Selected Model Drug and must ensure that at least one qualified SCD gene therapy provider in each contracted treatment center is enrolled in the state Medicaid program. States must also submit a template single case agreement (SCA) for SCD gene therapy to CMS, indicating in-state and out-of-state services.
CMS will provide technical assistance to participating states. For more guidance, states are encouraged to review CMCS guidance on coordinating care from out-of-state providers (while the guidance is in the context of care for children with medically complex conditions, it includes information that applies more broadly). For assistance with out-of-state provider enrollment questions, States may contact the Center for Program Integrity at MedicaidProviderEnrollment@cms.hhs.gov.
What data will states be required to track and submit?
Participating states will be required to submit Medicaid claims data to CMS, primarily through the Transformed Medicaid Statistical Information System (T-MSIS). States will be expected to meet (or have a plan to meet) the data quality standards outlined in the T-MSIS Outcomes Based Assessment methodology. States will also have to submit quarterly reports related to Model implementation.
For more detail on data and reporting requirements, see Section 3.4 of the State RFA. All requirements will be detailed in the SA.
What CMS learning support is provided to states implementing the model?
CMS offers a national level learning system for states implementing the model, aimed at supporting state success in impacting the experience and quality of care for patients receiving cell and gene therapies. The purpose of the learning system is to:
- Facilitate connections between states for exchange of ideas, knowledge, tools, and resources.
- Disseminate successful strategies for how to effectively connect individuals with SCD to gene therapies.
- Offer data feedback to guide states in improving their activities.
Through learning support, state participants can engage in peer-to-peer learning, contribute to identifying and distributing promising practices, and participate in surveys or interviews to inform CMS or state needs.
Additionally, CMS will provide learning opportunities for Community Based Organizations (CBOs) partnering with participating states and gene therapy providers, focusing on increasing awareness and demand for cell and gene therapies and addressing social barriers to care. CMS will also organize virtual and/or in-person meetings for providers through existing forums to discuss changes to care delivery to meet increased demand and improve patient outcomes.
What can states do now to prepare to join the CGT Access Model?
Early and regular interaction with CMS will be useful as states prepare to join the model. States should review the requirements for participation in the State RFA and use this time to plan for any program changes necessary to participate in the model, including those related to SPAs, waivers, or data requirements (see Sections 3.1.1 and 3.4.1 of the State RFA).CMS will be holding a webinar about the State RFA. Information about the webinar will be posted on the Innovation Center CGT Access Model website. States can also stay informed about upcoming webinars and events hosted by CMS by joining the CGT Model listserv.
For any questions, states can reach the CGT Access Model team at CGTModel@cms.hhs.gov.
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