- General Model FAQs
- State Request for Applications (RFA) FAQs
- Notice of Funding Opportunity (NOFO) FAQs
General Model FAQs
What is the status of the Cell and Gene Therapy Access Model?
Following a successful negotiation period, the two drug manufacturers of gene therapies for treating sickle cell disease — bluebird bio, Inc. and Vertex Pharmaceuticals, Incorporated — have each entered into separate agreements with CMS to participate in the Cell and Gene Therapy Access Model. Under the model, these manufacturers will offer new, outcomes-based arrangements to state Medicaid agencies. States have until February 28, 2025, to review the outcomes-based arrangements negotiated by CMS and offered by the manufacturers and decide whether to apply to participate in the model. In addition, states may choose to apply for optional model funding by responding to the Notice of Funding Opportunity (NOFO), but they are not required to respond to the NOFO to participate in the model. The deadline for responding to the NOFO is also February 28, 2025. States may begin participation in the model anytime between January 2025 and January 2026. CMS is providing technical assistance to all interested states to help them understand the terms of each outcomes-based agreement and assist in their decision-making regarding model participation.
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 is voluntary for both manufacturers and states.
Why does the CGT Access Model begin with a focus on sickle cell disease?
Sickle cell disease can be 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, Incorporated was listed at $2.2 million per patient and LYFGENIA™ from bluebird bio, Inc 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 have Medicaid coverage 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 guaranteed rebates.
CMS has negotiated 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 an administration period; a 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 administration period, the measurement period, and the reconciliation period as negotiated with the manufacturer(s). If, for example, the length of the administration period 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.
State Request for Applications (RFA) FAQs
More information can be found in the State Request for Applications (RFA) (PDF) published on June 28, 2024.
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 is voluntary for both manufacturers and states.
What is the process and timeline for states to apply for and participate in the CGT Access Model?
The application portal went 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. All SAs must be signed by June 1, 2025.
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.
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).
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 held a webinar on Monday, July 29, 2024 for states to learn about the State RFA. Interested states can view the webinar slides (PDF) and can contact the CGT Access Model team to request the webinar transcript and recording.
For any questions, states can reach the CGT Access Model team at CGTModel@cms.hhs.gov.
Notice of Funding Opportunity (NOFO) FAQs
More information can be found in the Notice of Funding Opportunity (NOFO) published on August 15, 2024.
What entities are eligible to apply for the CGT Access Model NOFO?
Eligible applicants are states, the District of Columbia, and any U.S. Territory participating in the Medicaid Drug Rebate Program (MDRP). To be considered for Cooperative Agreement funding the applicant must:
- Submit an application in response to the State Request for Applications (RFA) (PDF) no later than February 28, 2025
- Submit an application in response to the NOFO no later than February 28, 2025
- Sign a State Agreement with CMS no later than June 1, 2025.
For more information about the eligibility criteria and conditions, please refer to Sections A6.1.1 and C1 of the NOFO.
Are all states that are interested in participating in the CGT Access Model required to respond to the NOFO?
No. States that are interested in participating in the CGT Access Model are only required to respond to the State RFA. Only states that are interested in optional Cooperative Agreement funding are required to respond to the NOFO.
Cooperative Agreement funding, available to states that successfully apply to the NOFO, is intended to support state model implementation activities and to support states that take steps to improve equitable access to gene therapy and multi-disciplinary, comprehensive care in conjunction with the model test.
Why should states consider applying for Cooperative Agreement funding under the NOFO?
Cooperative Agreement funding can play a critical role in supporting the patient care journey for sickle cell disease (SCD) gene therapy. The journey for patients receiving SCD gene therapy can be long and challenging, and includes initial patient identification as a possible gene therapy candidate, evaluation for gene therapy, preparation phases including disease management and apheresis, optional fertility preservation services, gene therapy infusion, and extensive follow-up care.
Optional Cooperative Agreement funding can provide states with financial support to implement benefit enhancements, partner with community-based organizations (CBOs) to deliver services that may not be covered by Medicaid, and increase beneficiary access to comprehensive, multi-disciplinary care.
For a detailed description of the care journey, refer to Section A5.2 in the NOFO, which outlines the steps involved in this care journey. Additionally, the patient journey map (PDF) provides a visual guide of the care journey for SCD gene therapy.
What is the funding amount and duration of award for the CGT Access Model?
The CGT Access Model’s anticipated total available funding amount is a maximum of $95.5 million, subject to the availability of funds. Each applicant may receive a maximum of $9.55 million, depending on the number of states that apply to the NOFO.
The duration of the award is anticipated to be up to 10.5 years. The Model Performance Period and the Cooperative Agreement Period of Performance are anticipated to conclude on December 31, 2035.
Will all states that apply be awarded funding or is there a limit on the number of states that will be awarded funding?
Up to 52 awards may be issued, which includes the 50 states, the District of Columbia, and Puerto Rico (currently the only U.S. territory participating in the MDRP). However, the final number of awards will depend on the quality of applications and availability of funds. A merit review committee will evaluate each application using the criteria described in Section E1 of the NOFO.
Can multiple applications for the NOFO be submitted from the same state or territory?
As stated in Section C5 of the NOFO, each state may submit only one application for the CGT Access Model NOFO.
What are key dates related to the NOFO?
The negotiated Key Terms will be disclosed to all states in December 2024. After the Key Terms have been disclosed, states may apply to participate in the model in response to the State RFA and may apply for optional Cooperative Agreement funding in response to the NOFO. Important dates are listed below.
- State RFA Electronic Application Due Date: February 28, 2025, 11:59 pm EST
- NOFO Electronic Application Due Date: February 28, 2025, 11:59 pm EST
- State Agreement Signing Deadline: June 1, 2025
- NOFO Anticipated Issuance Notice(s) of Award: July 1, 2025
- Periods of Performance: There are two periods of performance to be aware of in this model:
- Model Performance Period (anticipated): January 1, 2025-December 31, 2035
Cooperative Agreement Period of Performance (anticipated): July 1, 2025-December 31, 2035
What is the difference between Implementation and Milestone Funding?
Implementation Funding and Milestone Funding are two distinct types of financial support available under the Cooperative Agreement in the CGT Access Model. Implementation Funding is designed to support required and optional model activities that involve staff/contractor time and infrastructure costs. Milestone Funding is designed to support states that successfully complete research projects related to increasing equitable access to SCD gene therapy and promoting multi-disciplinary, comprehensive care for beneficiaries with SCD who are considering or receiving SCD gene therapy.
How can Implementation Funding be used?
Implementation Funding, as outlined in Section A6.8.1 of the NOFO, is designed to support required and optional model activities that involve staff/contractor time and infrastructure costs. States can use these funds to engage in a variety of activities such as data collection, coordination with managed care plans and out-of-state providers, and partnerships with CBOs.
Specifically, states may collaborate with CBOs to provide enhanced services focusing on increasing awareness and education about gene therapy among patients and healthcare providers, improving access to supportive services necessary for receiving gene therapy (like transportation and lodging), providing services related to addressing health-related social needs, and providing community health worker/patient navigator and peer supports. As detailed in Section F5.1.1, states are required to report at least quarterly on how Implementation Funding is utilized, ensuring accountability and alignment with the overall objectives of the model.
How can Milestone Funding be used?
Milestone Funding, as outlined in Section A6.8.2 of the NOFO, is designed to support research projects related to increasing equitable access to SCD gene therapy and promoting multi-disciplinary, comprehensive care for beneficiaries with SCD who are considering or receiving SCD gene therapy. Milestone Funding is restricted (unavailable) unless the state demonstrates satisfactory completion of the research project in a submitted performance report. Unrestricted Milestone Funding can only be used for cost-based reimbursement of completed Milestone Funding projects. This means that the funds are specifically allocated to cover the expenses related to the research activities once the project is satisfactorily completed and reported.
Are there restrictions on the use of funds?
CMS prohibits the use of Cooperative Agreement funding to reimburse pre-award costs or to supplant existing state, local, tribal, or private funding of state costs.
For a full list of all the applicable limitations on the use of funds, please review Sections D6 and F2 of the NOFO. These sections provide comprehensive guidelines to ensure the funds are used appropriately and in alignment with the overall objectives of the CGT Access Model.
What steps should a state take to apply for the CGT Access Model NOFO?
Applicants need to register with the System for Award Management (SAM) database and obtain a Unique Entity Identifier (UEI) number. Applications must be submitted electronically through Grants.gov using the Grants.gov Workspace. For detailed instructions about how to apply, see Appendix II of the NOFO.
Applicants also need to apply to the CGT Access Model itself through the State RFA.
What happens if an application is submitted after the deadline?
Applications are due by February 28, 2025, 11:59 pm EST. Applications submitted after the deadline will not be accepted or reviewed. The official date and time that Grants.gov receives the application will serve as proof of timely submission. Applications received after the established due date will be considered late and will not be considered for funding by CMS.
It is also important to note that notification of an incomplete application may not be received until close to or after the deadline, preventing the applicant from correcting errors and resubmitting the application on time. Therefore, applicants are encouraged not to wait until the application deadline to apply.
What are the reporting requirements for the award?
Recipients will be required to comply with both federal reporting requirements and model-specific reporting requirements. For example, states will be required to submit quarterly progress reports on activities for which Implementation Funding is used and performance reports to demonstrate completion of Milestone Funding research projects. For a detailed list of all reporting requirements, including specific conditions and procedures, refer to Section F5 of the NOFO.
How can states partner with organizations under this NOFO?
States can use Implementation Funding to partner with CBOs with a focus on providing services to individuals with SCD who are considering or undergoing gene therapy for SCD. Implementation Funding can be used to pay CBOs for any of the following purposes: increasing awareness and education of gene therapy among patients and health care providers, increasing awareness and access to supportive ancillary services necessary for beneficiaries to receive gene therapy, providing services related to addressing health-related social needs (HRSNs), and providing community health worker/patient navigator and peer supports.
States can also partner with organizations to conduct Milestone Funding research projects. Partner organizations may include CBOs, treatment centers qualified to administer gene therapy for SCD, or academic institutions.
What are the terms and conditions for partnerships with CBOs?
Partnerships with CBOs have specific terms and conditions under the NOFO. Recipients seeking funding for these partnerships are required to include specific items in their reporting submissions. Furthermore, recipients must submit a full performance report of all funded CBOs on an annual basis. In each quarterly progress report, recipients must submit an updated Partnership List that includes details of all funded CBOs such as their name, mailing address, primary contact details, and amount of award. This ensures transparency, accountability, and effective monitoring of the funded CBOs.
What can states do now to prepare to apply for CGT Access Model Cooperative Agreement funding?
States should review both the requirements for model participation in the State RFA and the requirements for Cooperative Agreement funding in the NOFO. States can also use this time to coordinate with potential partner organizations, such as CBOs, treatment centers qualified to administer gene therapy for SCD, or academic institutions.
CMS held a webinar to provide an overview of the NOFO on Tuesday, September 24, 2024 at 2 p.m. ET. Interested states can view the webinar slides (PDF), transcript (PDF), and recording (MP4). States can also stay informed about upcoming webinars and events hosted by CMS by joining the CGT Access Model listserv.
For any questions, states can reach the CGT Access Model team at CGTModel@cms.hhs.gov.