One approach to CMS is Cell and Gene Therapy (CGT) Access Model. Under the CGT access model, CMS (federal level) will negotiate an Outcomes-Based Agreement (OBA) with the manufacturer, which will include pricing terms and outcomes to be evaluated. States can then choose to participate in the terms of the contract negotiated by CMS, or they can opt out and negotiate on their own. From CMS's perspective, they hope that increased purchasing power will lead to lower prices (or equivalently, more negotiated rebates). For manufacturers, the hope is that the CGT access model will speed up patient access and reduce transaction costs because manufacturers must negotiate with fewer than 50 state Medicaid agencies.Outcomes-based contracts are cumbersome to implement for both parties; having a CMS will support states in implementing, monitoring, coordinating, and evaluating the financial and clinical outcomes outlined in the OBA
What CGT are currently planned to be included in the CGT Access model? How much do they cost?
The program incorporates two CGTs used to treat sickle cell disease into the model. Both were approved in December 2023. Vertex Pharmaceuticals and CRISPR Therapeutics’ “Casgevy” is priced at $2.2 million per patient; Bluebird Bio’s “Lyfgenia” is priced at $3.1 million per patient.
What types of rebates are available to state Medicaid agencies under the capital gains tax entry model?
These include not only standard Medicaid Drug Rebate Program (MDRP) rebates (also known as Medicaid Best Prices), but also additional guaranteed and volume rebates as well as future rebates paid based on patient and population outcomes.
When is the rebate paid?
The timing of the rebate depends on the type of rebate. Statutory Rebates (MDRP) and negotiated guaranteed rebates will be paid immediately following treatment. Volume-based discounts may take effect by the end of the year. Finally, patient outcome and population outcome rebates will be paid out years after treatment. Note that patient outcome rebates can be paid annually over time to account for patient response to treatment, but may not be indefinite.
What does this process look like from a patient’s perspective?
this CGT access model: Overview of country presentations Have a helpful schedule.
If a state chooses to participate in this model, can it attempt to negotiate additional discounts or terms beyond those negotiated by CMS?
If a state chooses 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 variations are allowed, which may be necessary to comply with state legislation and regulatory requirements.
What do states need to do to participate in the CGT access model?
Requirements include:
- Execute Value-Based Purchasing Supplemental Rebate Agreements (VBP SRA) with manufacturers to reflect key terms negotiated by CMS
- State Plan Amendments (SPA) where appropriate
- Develop standardized access policies for included gene therapies
- Incorporate gene therapy into any hospitalization payment bundle
- Providers are required to follow data reporting and claims submission requirements
- Ensure beneficiaries have access to care from qualified in-state or out-of-state gene therapy providers
- Ensure beneficiaries’ necessary transportation and related travel expenses (NEMT)
- Meet minimum T-MSIS profile requirements
What legislation created the CGT admission model?
CMS Statement:
The CGT access model was developed in response to President Biden’s Executive Order 14087,”Lowering prescription drug costs for Americans” and was first proposed in Reports in response to executive orders Under the direction of the Secretary of Health and Human Services.
https://www.cms.gov/priorities/innovation/innovation-models/cgt
How can I learn more?
Some useful reference materials about CMS CGT visits model web page include:
