Analysis estimates 11 cents per member per month for sickle cell gene therapy risk pool

What the 11 cents assumes

The AMCP analysis modeled 90,000 US sickle cell patients, 5% eligible and 5% uptake, with even market share between Casgevy at $2.2 million and Lyfgenia at $3.1 million and 25% rebates, said Sujith Ramachandran, PhD, associate director of Trinity Life Sciences. Funding the pool for all insured people would cost 11 cents per member per month, he said. Sensitivity analyses ranged from 2 cents to 22 cents under different rebate and uptake assumptions.

Ramachandran said the analysis was early modeling and that the range shows a national risk pool might be financially feasible, though it covered one disease and two products. The session identified open design questions: which therapies qualify, how eligibility and contributions are set, and how centers are reimbursed.

Pipeline pressure

Carmen G. Witsken, senior manager of pharmacy policy at the Alliance of Community Health Plans, said most gene therapies are in oncology or rare disease, while immunology, neurology, sensory and cardiovascular indications are expanding. She counted 4,487 cell, gene and RNA therapies in development worldwide, 49% gene therapies including modified cells, 21% non-genetically modified and 30% RNA therapies. The FDA has approved 54 cell and gene therapies, and 438 gene therapies are in the US pipeline as of January, she said.

List prices run from $2 million to more than $4 million, paid as a single claim, Witsken said. That can threaten the viability of a health plan, especially small nonprofit plans, she said. Her proposed fix has four parts: 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.

In a Mercer survey cited by Ramachandran, 57% of employers named gene therapy cost as their top concern. Witsken also cited InspiroGene's 2026 survey, in which 76% of surveyed payers expected affordability to be a moderate or major challenge over the next two to three years.

Barriers beyond price

Ramachandran grouped access barriers into upfront financial burden, risk portability and administration. Costs land up front while savings accrue years later, he said, and 1 in 5 Americans leaves a health plan each year. Stop-loss insurance does not track outcomes, may exclude known patients, leaves portability unsolved and invites adverse selection, he said. "We need a more innovative policy solution," he said.

Manufacturing is another bottleneck. Ramachandran put capacity as low as 1 to 2 patients per year and said more than half of people with sickle cell disease do not live in the same metro area as an authorized treatment center.

Cost context

Separate research published in Blood in June found stem cell transplantation was the most cost-effective option for long-term care of adults with sickle cell disease, compared with gene therapy and standard of care. It found gene therapy's cost would need to be 66% to 71% lower than previously determined to have a competitive value for price.

The Blood study cites nearly 8 million people with sickle cell disease worldwide and approximately 100,000 in the United States, compared with the 90,000 patients used in the AMCP model.