Outcomes-based payment for cell and gene therapies
A plain explanation for patients, families and doctors, checked against the regulator's own pages on 17 October 2026.
Reviewed by the Reserve Meds clinical operations team on 17 October 2026. Sources accessed 17 October 2026.
The short answer
Outcomes-based (pay-for-performance) agreements link what a payer pays for a medicine to how well it works. The clearest public example is the US Cell and Gene Therapy Access Model, in which CMS negotiates outcomes-based terms with manufacturers for gene therapies for sickle cell disease on behalf of state Medicaid programs. It applies to US Medicaid patients, not to international buyers.
- Example program: CMS Cell and Gene Therapy (CGT) Access Model.
- First focus: Gene therapy for sickle cell disease.
- Who it covers: Medicaid or CHIP enrollees in participating US states.
- International patients: Price and terms are quoted case by case.
What an outcomes-based agreement is
Cell and gene therapies can transform lives, and payers fund them through specific programmes. An outcomes-based agreement ties payment to outcomes: if the therapy does not deliver agreed results, the payer receives rebates or pays less.[1]
The CMS Cell and Gene Therapy Access Model
The CGT Access Model is a multi-year, voluntary model for states and manufacturers. CMS and manufacturers negotiate key terms, including pricing discounts and outcomes-based rebates, which form the basis of contracts between each manufacturer and participating states. CMS describes it as the first time the federal government has negotiated outcomes-based agreements with cell and gene therapy manufacturers on behalf of state Medicaid agencies.[1]
Its initial focus is gene therapy for sickle cell disease. States began participating between January 2025 and January 2026. To be eligible, a person must be enrolled in Medicaid or CHIP in a participating state at the time of therapy and meet standardized prior authorization criteria. CMS also notes that sickle cell gene therapy involves an extended inpatient stay and possibly several visits to a gene therapy center.[1]
The two FDA-approved gene therapies for sickle cell disease are Casgevy and Lyfgenia, both on FDA's list of approved cellular and gene therapy products.[2]
What this means for families outside the US
Outcomes-based agreements like the CGT Access Model are negotiated by payers for their own populations. A family or hospital outside the US is not covered by them, and we do not claim access to their terms.
For international patients, cell and gene therapies are usually given at a qualified treatment center chosen by the manufacturer, and the center confirms eligibility, funding and scheduling.
Ask the treating specialist whether a local center offers the therapy, and ask your insurer or government program about funding before planning travel. Price: quote on request.
What outcomes-based terms usually measure
In the CGT Access Model, CMS and manufacturers negotiate key terms, including pricing discounts and outcomes-based rebates, and manufacturers give participating states supplemental rebates that reflect those terms. States, in turn, must implement an agreed standard access policy. CMS also offers participating states technical assistance and optional funding support for data collection and reconciliation.[1]
Questions to ask the treating specialist
- Is the therapy offered by a center in our country or region, and who funds it?
- What tests confirm eligibility before referral?
- How long is the hospital stay and follow-up after treatment?
- Will our insurer or government program cover any part of the cost? Ask in writing.
How Reserve Meds helps
Reserve Meds is a digital health company. Medicines are supplied through a US-licensed pharmaceutical wholesale partner. We help families and treating doctors understand the route, prepare the documents the regulator or hospital asks for, arrange supply from the US once approval is in place, and keep everyone updated. We do not diagnose, prescribe or decide what a regulator approves.
We coordinate access to FDA-approved medicines only. Investigational medicines are only available through a manufacturer's expanded-access program requested by the treating physician; we can explain that route, but we do not supply them. Your coordination fee is quoted in writing for your case before you commit. No hidden charges. We give you a written timeline estimate for your case before you commit.
Ask us whether a medicine can reach your patient
Tell us the medicine, your country and a WhatsApp or phone number. We reply within one business day. On WhatsApp we usually reply the same day.
Frequently asked questions
What is a pay-for-performance or outcomes-based agreement?
A payment arrangement where the price a payer pays for a medicine is linked to whether it achieves agreed outcomes, often through rebates if it does not.
What is the CMS CGT Access Model?
A voluntary US model in which CMS negotiates outcomes-based terms with cell and gene therapy manufacturers on behalf of participating state Medicaid programs, starting with gene therapy for sickle cell disease.
Can a family in the Gulf benefit from the CGT Access Model?
No. It covers Medicaid or CHIP enrollees in participating US states. International treatment is priced and arranged case by case.
Which gene therapies are FDA-approved for sickle cell disease?
Casgevy and Lyfgenia, both listed on FDA's page of approved cellular and gene therapy products.
Sources for these answers: [1, 2]
Sources
All sources were opened and checked on 17 October 2026. Rules and labels change, so the treating hospital or regulator confirms the current position before anything is filed.
- US Centers for Medicare and Medicaid Services: Cell and Gene Therapy (CGT) Access Model (accessed 17 October 2026)
- US FDA: Approved Cellular and Gene Therapy Products (accessed 17 October 2026)
Related
- Hematology medicines and access
- Managed access programs
- All access pathways
- US expanded access (compassionate use)
- Named-patient access, explained
- Answers to common access questions
This page is general information, not medical or legal advice. It does not replace advice from your own doctor or the regulator.