Sickle Cell Gene Therapy May Be Unaffordable For Some State Medicaid Programs, Jeopardizing Access, Study Finds

It can seem like there’s almost as much research on how to pay for gene therapy treatments as there is on finding new ones, and for good reason: There’s not much use in developing a treatment that no one can afford.

Gene therapy for sickle cell disease (SCD) presents perhaps the biggest affordability challenge so far. The two gene therapies approved by the FDA last December carry list prices of roughly $2 million and $3 million. The treatments apply to a population far larger than any existing commercial gene therapy, and one-half of the estimated 100,000 people with the disease in the United States are enrolled in Medicaid.

In a research letter published in the Sept. 10 issue of Blood Advances, scientists updated earlier findings of a model published in 2021 in JAMA Pediatrics that projected the impact on Medicaid program budgets of yet-to-be approved gene therapy treatments would be difficult for some states to afford.

Using updated inputs based on the actual approvals and other new data, first

Patrick DeMartino, M.D., M.P.H.

author Patrick C. DeMartino, M.D., Ph.D., an assistant professor of pediatrics at Oregon Health & Sciences University and a pediatric hematologist/oncologist at its Doernbecher Children’s Hospital in Portland, and colleagues, found that they have since become substantially more unaffordable.

DeMartino and his colleagues researchers estimated first year and fifth year costs to state Medicaid program budgets nationally and in each of 10 states with the highest prevalence of SCD. They made multiple assumptions: Only the cheaper of the two new gene therapies, both potentially curative with a single treatment, would be used: Vertex Pharmaceuticals' Casgevy (exagamglogene autotemcel), which has a wholesale acquisition cost price of $2.2 million plus, they estimated, $900,000 for necessary one-time hospitalization. The eligible population would be people aged 13 to 45 with a severe type of SCD (two or more episodes of severe pain annually), estimated at 35% of all those with sickle cell disease.

The researchers estimated a 7% annual market diffusion rate which they conceded would be far lower in the first year. They also assumed that the single treatment would work forever (far beyond what the clinical trials could conclude), have no adverse effects and no medical expenses beyond the initial treatment.

The results: An estimated 542 Medicaid enrollees with severe SCD would receive gene therapy the first year nationwide, according to the model, costing the state programs a total $1.2 billion after subtracting the costs for a current treatment that must be given for a lifetime and is not widely used. The budgetary impact for Medicaid programs nationwide comes out to an average $1.13 per Medicaid member per month, declining to $0.75 in year 5.

The numbers may seem relatively small, but they vary widely by state and can mount in some. The model estimated that Alabama and Virginia, for example, each would provide gene therapy to 24 Medicaid enrollees with severe SCD in the first year with a total budget impact of roughly $54.3 million in each state. the cost per member per month works out to $3.90 in Alabama the first year, declining to $2.60 by year 5, compared with $2.30 in Virginia the first year, going down to $1.54.

Although “there are no thresholds for budgetary significance,” wrote DeMartino and his coauthors wrote, an impact of more than $2 per member per month “is notable.” Many state Medicaid programs faced significant budget challenges in 2014 and 2015 with the newly approved and pricey Sovaldi (sofosbuvir) – also a single, potentially curative treatment, although not gene therapy – for hepatitis C.

The average per member per month cost for hepatitis C treatment in the same 10 states was $1.89 (in 2024 dollars), “which proved unaffordable for many Medicaid plans,” the authors wrote. The 10-state average for gene therapy would be $3.11 per member per month the first year.

“The two new gene therapies for severe SCD will likely produce a considerable budget impact for some Medicaid plans, and the ensuing affordability challenge may jeopardize patient access,” concluded DeMartino and his colleagues.