Experts Pitch National Risk Pool to Fund Gene Therapy Access

Cell and gene therapies (CGTs) are spreading beyond oncology and rare diseases, but the way health plans pay for them has not kept pace. At the October 28 session, “From Pipeline to Patient: Navigating the Next Wave of Cell & Gene Therapies,” at the Academy of Managed Care Pharmacy (AMCP) Nexus 2026 meeting in San Diego, experts argued for a national, all-payer risk pool and presented early numbers suggesting it could be affordable.¹ Adam Colborn, JD, vice president of government affairs at the AMCP, moderated.

A Growing Pipeline

Carmen G. Witsken, PharmD, senior manager, pharmacy policy, Alliance of Community Health Plans, opened with the pipeline. She reported 4487 cell, gene, and RNA therapies in development globally—49% are gene therapies, including modified cells; 21% are non–genetically modified therapies; and 30% are RNA therapies²—that the FDA has approved 54 CGTs, and 438 gene therapies are in the US pipeline as of January. Compared with last year, gene therapy programs in phases 1 through 3 each grew, while preclinical programs fell by 129.

“I want to flag here that most gene therapies, by far, are in the oncology or rare disease space,” Witsken said, with oncology and rare disease leading. Immunology, neurology, sensory, and cardiovascular indications are expanding, and “the need for health plans to have financing and reimbursement pathways grows as the number of patients who are potentially treated by these gene therapies also grows.”

Witsken highlighted InspiroGene’s 2026 survey: oncologists reported treating an average of 32 patients on CGTs each year, up from 17 in 2024, and 34% named insurance approval as the main reason for patient drop-off.³ In a separate finding, 76% of surveyed payers expect affordability to be a moderate or a major challenge over the next 2 to 3 years.⁴ With list prices from $2 million to more than $4 million, paid as a single claim, the cost “can be incredibly difficult to manage and threatens the viability of the health plan,” Witsken said, especially for small nonprofit plans. Her proposed fix has 4 elements: all-payer participation, per-member monthly fees paired with manufacturer outcomes-based contracts, federal administration through an independent designee, and eligibility limited to the rarest or highest-cost therapies. Contracts would also allow patients to be tracked when they change plans.

Why Not Stop-Loss?

Sujith Ramachandran, PhD, associate director of Trinity Life Sciences, grouped access barriers into 3 buckets: upfront financial burden, risk portability, and administration facilities. On costs, he cited a Mercer survey in which 57% of employers named gene therapy cost their top concern.⁵

On portability, he said, “all of these costs for gene therapies are up front, but the savings that might come from these potentially curative medical miracles actually don’t accrue for years afterwards,” and 1 in 5 Americans leave their health plan each year. Regarding administration, he said over half of patients with sickle cell disease do not live in the same metro area as an authorized treatment center, and manufacturing capacity may be as low as 1 to 2 patients per year.

Stop-loss insurance, he said, does not address outcomes tracking, may exclude known patients, leaves portability unsolved, and invites adverse selection. “We need a more innovative policy solution,” he emphasized.

Ramachandran presented an AMCP-published analysis modelling sickle cell gene therapy: 90,000 US patients, 5% eligible, 5% uptake, even market share between exagamglogene autotemcel (Casgevy; Vertex/Crispr Therapeutics) at $2.2 million and lovotibeglogene autotemcel (Lyfgenia; Genetix) at $3.1 million, and 25% rebates. Funding the pool for all insured people, he said, “would cost 11 cents per member per month.” Sensitivity analyses ranged from 2 cents (75% rebate) to 22 cents (10% uptake).⁶

The range, he said, “importantly demonstrates that such a national risk pool might actually be financially feasible,” although the analysis covered only 1 disease state and 2 products. Open design questions include which therapies qualify, how eligibility and contributions are set, and how centers are reimbursed.

Outcomes-Based Agreements and Medicaid

Ravi Singh, vice president, market access, advocacy and policy, Genetix Biotherapeutics, said he was not speaking for his company, which manufactures lovotibeglogene autotemcel. He said, “really successful outcomes agreements combine meaningful risk-sharing with a process that can be administered consistently,” and he listed 4 necessary traits of said agreements: they are clinically grounded, easily measurable, operationally feasible, and scalable.

He then reviewed the CMS Innovation Center’s CGT Access Model, in which 34 states and territories participate for sickle cell gene therapy. This is the first time the federal government has negotiated outcomes-based agreements with CGT manufacturers for state Medicaid agencies. States must publish coverage criteria, pay separately for the therapy, reimburse at least the acquisition cost, and contract with a qualified treatment center, including out of state, if needed. Singh said CMS announced on October 27 a request for applications to add other therapies.⁷ The model is “a great step forward in terms of helping address some of the challenges with financing CGTs. But I wouldn’t say that it addresses everything.”

What Is Lifetime Value?

During the audience question-and-answer period, Singh said sickle cell disease’s lifetime direct medical costs may run $2 million to $6 million,⁸ so “lifetime costs of care are relevant when putting the price of gene therapy in context.” Asked whether manufacturing efficiencies could cut costs, he said, “Whether that yields sort of list price reductions, I’m not so sure. But in the context of net price costs, you could see savings over time.”

Ramachandran said CGTs force a rethink of value, but not its formula. “The way we think about the value that these products bring to society is still the same,” he said. The challenge, he added, is that plan turnover can keep payers from realizing that value.

References

1. Colborn A, Witsken C, Ramachandran S, Singh R. From pipeline to patient: navigating the next wave of cell & gene therapies. Presented at: AMCP Nexus; October 26-29, 2026; San Diego, CA.
2. Gene, cell, & RNA therapy landscape report: Q2 2026 quarterly data report. American Society of Gene & Cell Therapy and Citeline. July 2026. Accessed October 29, 2026. https://www.asgct.org/uploads/files/general/Final-ASGCT-Citeline-Q2-2026.pdf
3. 2026 cell and gene therapy report. InspiroGene. Accessed October 29, 2026. https://inspirogene.com/cgt-report/
4. Lonergan M, Gregg R, Lee M, Rayburg R. 2026 trends in specialty drug benefits report. Pharmaceutical Strategies Group. Accessed October 29, 2026. https://www.psgconsults.com/industry-report/2026-trends-in-specialty-drug-benefits-report/
5. Belousova A, Brown R, Peel A. Employers top four questions about cell and gene therapies. Mercer. August 13, 2025. Accessed October 29, 2026. https://www.mercer.com/en-us/insights/us-health-news/employers-top-four-questions-about-cell-and-gene-therapies/
6. White paper: ESTIMATING the PMPM of gene therapies for sickle cell disease in a national all-payer risk pool AMCP. Accessed October 29, 2026. https://www.amcp.org/sites/default/files/2026-10/AMCP_CGT_RiskPool_WhitePaper_102226.pdf
7. Innovation insight: CGT access model builds on initial success with new therapies to be added following manufacturer negotiations. CMS. October 27, 2026. Accessed October 29, 2026. https://www.cms.gov/priorities/innovation/innovation-insight-cgt-access-model-builds-initial-success-new-therapies-be-added-following
8. Johnson KM, Jiao B, Ramsey SD, Bender MA, Devine B, Basu A. Lifetime medical costs attributable to sickle cell disease among nonelderly individuals with commercial insurance. Blood Adv. 2022;7(3):365-374. doi:10.1182/bloodadvances.2021006281