Cell and gene therapy outcomes-based payment programmes Market Outlook 2026–2035: Growth Drivers, Key Companies, Strategic Developments & Adoption Forecast — A Meticulous Next™ Foresight Brief

This Meticulous Next™ brief examines cell and gene therapy outcomes-based payment programmes, where payers and manufacturers tie the price of a one-time therapy to how well it works, through rebates, refunds, annuities or instalments paid over time. It tests how far outcomes-based payment becomes the standard route for gene therapies by 2035, and what that means for manufacturers, payers and treatment centres.

Federal negotiation now covers most Medicaid patients with sickle cell disease. The Centers for Medicare & Medicaid Services announced in July 2025 that 33 states, the District of Columbia and Puerto Rico had signed agreements to join its Cell and Gene Therapy Access Model, and these jurisdictions hold 84% of Medicaid beneficiaries with sickle cell disease. The conclusion is that one federal negotiation replaced dozens of state level talks, and manufacturers now face a single buyer for most Medicaid patients in this condition.

The model ties payment to results. CMS negotiated outcomes-based agreements with Vertex Pharmaceuticals Incorporated for Casgevy and with bluebird bio, Inc. for Lyfgenia, announced in December 2024 with the model starting in January 2025, and states receive guaranteed discounts and rebates if a therapy does not deliver the promised benefit. The conclusion is that the manufacturer carries part of the risk of therapy failure, which is the core of outcomes-based payment.

Prices explain why payers want protection. Casgevy lists at USD 2.2 million and Lyfgenia at USD 3.1 million, Zolgensma at USD 2.1 million, Hemgenix at USD 3.5 million and Lenmeldy at USD 4.25 million. The conclusion is that a single payment of this size is a large risk for any payer, and payers are more willing to pay these prices when part of that risk is shared.

Uptake has been uneven. Pfizer Inc. ended global sales of Beqvez in February 2025 at a price of USD 3.5 million, citing limited interest from patients and doctors, and BioMarin Pharmaceutical Inc. is withdrawing Roctavian. The conclusion is that a good payment model does not create demand alone, and programmes must be matched to conditions where patients and doctors want treatment.

The brief treats cell and gene therapy outcomes-based payment programmes as a high-impact signal with a nine year horizon to 2035. It identifies which manufacturers, payers and programmes move first and what each means for price, risk and patient access. It is a focused 105-page decision brief that presents an indicative trajectory rather than a segmented market model.

The federal model is the core structure. CMS negotiates outcomes-based agreements with manufacturers on behalf of state Medicaid agencies, and states receive guaranteed discounts and rebates if a therapy fails to deliver its benefit, while the federal government covers a defined scope of fertility preservation and costs such as travel, and up to USD 9.55 million per state for outreach and data tracking. The conclusion is that the federal model removes the setup burden from each state and pays for the data needed to judge outcomes.

Public payers already negotiate below list price. Public insurers paid USD 1.89 million per Zolgensma claim in 2019 and USD 1.36 million in 2022, nearly 40% below list price, and CMS spent about USD 87.25 million on about 63 Zolgensma treatments in 2022. The conclusion is that public programmes pay for these therapies in volume and use rebates to bring cost down, and an outcomes link adds protection against failure.

Lifetime cost is the basis of the commercial case. CSL Limited estimates that a patient with haemophilia B can cost an insurer USD 20 million over a lifetime, against a one-time price of USD 3.5 million for Hemgenix. The conclusion is that payers weigh a single high payment against years of treatment, and an annuity or rebate spreads that payment over the time the benefit is proven.

Spending remains small against total health spending. The Alliance for Regenerative Medicine cites an analysis from NEWDIGS and Tufts that projects US gene therapy spending of USD 7.5 billion by 2030, about 0.1% of projected health care spending that year. The conclusion is that the total budget impact is manageable, and the risk sits with individual payers that have a few very costly patients.

The limits are low uptake of some gene therapies, patient safety and the long-term follow-up burden, and the limits of evidence and follow-up periods. The brief tests each limit against the agreement evidence to 2035.

The cell and gene therapy outcomes-based payment programmes market, covering manufacturer net revenue from cell and gene therapies sold under outcomes-based rebate, refund, annuity or instalment agreements with payers, measured by annual manufacturer net revenue under such agreements, and excluding therapies sold at an upfront single payment, oncology cell therapies sold under standard coverage, and rebates unrelated to outcomes, is valued at USD 0.8–1.5 billion in 2026 and is projected to reach USD 4.79–8.98 billion by 2035, at a CAGR of 22%.

The 2026 base rests on manufacturer net revenue from Medicaid sickle cell agreements and from commercial rebate, refund and annuity agreements for rare disease and haemophilia therapies. Growth then follows the number of therapies and conditions added to federal models, the spread of annuity and instalment contracts at commercial payers and the number of patients treated.

The forecast is sensitive to the number of new gene therapy approvals, the uptake of therapies that payers cover and the share of revenue that agreements tie to outcomes.

Scenarios.

The base case assumes steady growth in therapies and patients under outcomes agreements, bringing the inflection to about 2030 and a 2035 value near the middle of the range. An accelerated case assumes fast addition of conditions to the federal model and wide use of annuities at commercial payers, bringing the inflection to 2028 and lifting the 2035 value to the top of the range. A delayed case assumes low uptake of approved therapies and slow growth in approvals, moving the inflection to 2033 and holding growth at the bottom of the range.

Growth drivers

• Payer demand for protection against therapy failure.
• Manufacturer need to win coverage and uptake.
• Federal programmes for Medicaid access.
• Growing number of approved gene therapies.

Enablers

• Registries and outcome data tools.
• Manufacturer and payer contracts.
• Contract and project evidence.
• Treatment centre and manufacturer partnerships.

Restraints and barriers

• Price pressure and return on investment.
• Low uptake of some gene therapies.
• Patient safety and long-term follow-up burden.
• Differences in national pricing and payment rules.

The direction of cell and gene therapy outcomes-based payment programmes is set by payer demand for protection against therapy failure, manufacturer need to win coverage and uptake, federal programmes for Medicaid access, registries and outcome data tools, and the readiness of payers and treatment centres to track outcomes over several years.

The key participants in cell and gene therapy outcomes-based payment programmes and outcomes-based gene therapy payment include Vertex Pharmaceuticals Incorporated, CRISPR Therapeutics AG, Novartis AG, CSL Limited, uniQure N.V., Pfizer Inc., BioMarin Pharmaceutical Inc., Sarepta Therapeutics, Inc., Bristol-Myers Squibb Company, Gilead Sciences, Inc., Johnson & Johnson, Ultragenyx Pharmaceutical Inc., PTC Therapeutics, Inc., Krystal Biotech, Inc., Rocket Pharmaceuticals, Inc., UnitedHealth Group Incorporated, CVS Health Corporation, The Cigna Group, Elevance Health, Inc., Centene Corporation, Regeneron Pharmaceuticals, Inc., Astellas Pharma Inc., Takeda Pharmaceutical Company Limited, Roche Holding Ltd, Bayer AG, Sanofi, GSK plc, AstraZeneca PLC, Intellia Therapeutics, Inc., Beam Therapeutics Inc., REGENXBIO Inc., Biogen Inc., Amgen Inc., Humana Inc., Molina Healthcare, Inc., the Centers for Medicare & Medicaid Services, the US Food and Drug Administration, the National Institute for Health and Care Excellence, NHS England, the European Medicines Agency, the Institute for Clinical and Economic Review and the Alliance for Regenerative Medicine.

The brief profiles representative participants in each archetype and assesses which are positioned to lead as multi-condition and commercial payer agreements reach scale.

Vertex Pharmaceuticals Incorporated, Novartis AG and CSL Limited lead among manufacturers with outcomes-based agreements, UnitedHealth Group Incorporated, CVS Health Corporation and The Cigna Group lead among commercial payers that can run annuity and rebate programmes, and the Centers for Medicare & Medicaid Services leads among public programmes. Competitive intensity is rising as new gene therapies reach approval and payers push for risk sharing.

Where value migrates.

In 2026 value sits with manufacturers of approved sickle cell and rare disease therapies that hold federal agreements and with the Medicaid programme that gains protection against failure. By 2030 it moves toward manufacturers and payers that run agreements across several conditions, because registries and outcome definitions can be reused. By 2035 it settles with outcomes-based payment as the standard route for new gene therapies, where long-term evidence, tracking capacity and trust carry the margin. Margins hold for manufacturers with strong outcome data, and narrow for those that sell a therapy with low demand and a rebate exposure that is hard to predict.

Federal negotiation, high prices and payer demand for protection against failure support growth, while low uptake of some therapies, the burden of long-term follow-up and limited evidence hold back the pace.

Weak uptake, slow approvals or complex tracking could slow progress. The brief therefore tracks four indicators: price per therapy and rebate terms, patients treated under outcomes agreements, therapies under outcomes agreements and the share of revenue paid under outcomes terms.

Medicaid sickle cell agreements first, then multi-condition and commercial payer agreements at scale, then standard status for outcomes-based payment is the expected order of change.