Providing treatment, no matter the cost, may even out long-standing disparities

While an investigational gene therapy for severe sickle cell disease could bring substantial benefits for patients—and potentially even a cure—it may have significant budgetary impacts on many Medicaid plans. The mean 10-year budget impact was estimated at nearly $30 million per state Medicaid program studied, according to results from a budget impact analysis recently published in JAMA Pediatrics.

“Gene therapy for sickle cell disease is likely to provide an early demonstration of the unique financial challenges associated with this emerging drug class,” wrote researchers led by Patrick DeMartino, MD, of Doembecher Children’s Hospital, Portland, OR. “However, this therapy is likely to cost more than $1 million, presenting a variety of fiscal challenges to U.S. health care payers covering large numbers of patients with SCD. At least 55,000 patients with SCD are enrolled in Medicaid nationally.”

Philip O. Ozuah, MD, PhD, of Albert Einstein College of Medicine and Montefiore Medicine, Bronx, NY, commented on DeMartino et al’s study in an accompanying editorial, and reflected on the burden of SCD and that the majority of those affected are of African ancestry.

“The most commonly inherited blood disorder in the world, SCD is routinely diagnosed at birth and can affect virtually every organ and system in the body. Only approximately 10% of patients can get a matched bone marrow transplant from a sibling, a harrowing procedure that carries a 90% cure rate. Beyond that, treatments primarily target pain-management and the complications that patients experience. For many, SCD means years of painful crises, blood transfusions, and hospitalizations. I know all too well the agony of this condition, having spent more than 3 decades as a physician providing care for patients with SCD in this country and in Africa,” he wrote.

Ozuah noted that the study from DeMartino and coauthors “amounts to an early warning about what may well be the next manifestation of the systemic disparities that continue to plague health care in the U.S.” He pointed out that the good news from the study is that it has uncovered “the rarest of all therapeutic interventions — a potential cure.” But the bad news is that cost of the treatment, as the authors noted, could make it unattainable for many, if not most.

The gene therapy DeMartino et al studied was a lentiviral-based therapy—betibeglogene autotemcel—which was approved by the European Medicines Agency in 2019 for the treatment of β-thalassemia and for which early efficacy has been shown on an ongoing phase I/II trial (HGB-206).

They analyzed the budgetary impact of lentiviral-based therapy on 10 state Medicaid plans over a 5-year horizon time point, using state-level disease prevalence data from 2012, with prevalence, Medicaid enrollment, and expenditures calculated from existing literature.

The cohort was comprised of patients with severe sickle cell disease identified based on modified clinical trial inclusion. In all, an estimated 5,464 Medicaid enrollees (age 13-45 years) were deemed eligible for the therapy, and 2,315 patients were enrolled in the 10 Medicaid programs included in the analysis.

DeMartino and colleagues based this analysis on the assumption that lentiviral-based therapy was to be administered to 7% of the eligible population annually and was curative, thereby incurring no more subsequent disease-related spending. The price of the gene therapy was $1.85 million, and baseline disease-related spending was $42,200 annually.

In modeling based on these parameters, DeMartino et al found that the mean projected 10-year budget impact was $29.96 million per state Medicaid program, at $1.91 per member per month (PMPM).

The 5-year annuity payment reduced the impact of this short-term budget, with savings accruing and fewer patients remaining eligible for the treatment. In the 10 states studied, the mean budget impact decreased from $1.91 PMPM during year 1, to $1.27 in year 5. DeMartino and colleagues used costs in Mississippi to illustrate, with costs decreasing from year 1 ($23.8M), to $21.6M in year 2; $19.5M in year 3, $17.6M in year 4, and $15.8M in year 5.

In his commentary, Ozuah also reflected on the disparities in care that people of African descent have endured in this country, more recently including the Tuskegee syphilis experiments, and the doubling of the death rate from Covid-19 for Black individuals compared with White.

In the U.S., the cost to cure the 5,464 Medicaid enrollees who have severe SCD and are eligible for lentiviral based gene therapy is approximately $5.5 billion, noted Ozuah, adding “I am too well versed in the harsh realities of health care funding, and Medicaid specifically, to pretend that is not an intimidating number. However, viewed in the light of the decades of neglect and disparities that patients with SCD have experienced in the U.S., it amounts to little more than a down payment for the pain they have endured.”

“I have spent most of 2020 fighting a novel coronavirus whose lethality is amplified by the poverty, unemployment, hunger, substandard public housing, and other social determinants of health that our neighbors in the Bronx have to deal with day in and day out. As a physician and health care leader, I find it maddening to think that people who live within mere blocks of my hospital are made more vulnerable by their physical and social environment,” he wrote.

“Sickle cell disease, caused by a point mutation in DNA, a single amino acid substitution in a single gene, has long been recognized as a good target for gene therapy, including CRISPR (clustered regularly interspaced short palindromic repeats)– Cas9 (CRISPR-associated protein 9) technology, the revolutionary genome editing method that recently won its creators the 2020 Nobel Prize in chemistry. The irony here is inescapable—some of the most underserved patients in the world are ideal candidates for the most advanced medical treatment yet conceived. We are presented with the opportunity to cure a terrible disease and, perhaps someday, eliminate it altogether. There is a debt to be paid here, and we as a society should welcome the chance to pay it. There are, after all, so many others that will never be paid,” Ozuah concluded.

  1. Gene therapy for sickle cell disease will likely present affordability challenges to several Medicaid plans.

  2. For a gene therapy for sickle cell disease, researchers estimated a mean 1-year budget impact of $29.96 million per state Medicaid program, or $1.91 per member per month increase in spending.

Liz Meszaros, Deputy Managing Editor, BreakingMED™

DeMartino and Ozuah reported no conflicts of interest.

Cat ID: 497

Topic ID: 495,497,585,497,730,118,192,151,462,925