1) 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.
2) 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.
3) 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.
4) If a state chooses to participate in the model, can it attempt to negotiate additional discounts or terms other than those negotiated by CMS?
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. Some limited state-to-state variation may be permitted, as may be necessary to comport with state legislative and regulatory requirements.
5) 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.
6) When will more details on state participation be released?
CMS expects to release further details on how states can participate in the model later in 2024. All states and territories that participate in the Medicaid Drug Rebate Program (MDRP) are encouraged to apply for the model, provided they meet requirements for participation.
7) 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).
8) 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.
9) 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.
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