A Budget Impact Analysis of Gene Therapy for Sickle Cell Disease

Abstract

Hundreds of gene therapies are undergoing clinical testing and are likely to be priced more than $1 million per course of treatment. The association that high prices will have with insurance coverage of gene therapy remains unclear. Gene therapy for sickle cell disease has shown early success and would be one of the first gene therapies available for a relatively large population.OBJECTIVES To estimate the budget impact and affordability of a gene therapy for severe sickle cell disease from the perspective of US Medicaid programs with the highest prevalence of sickle cell disease while exploring the impact of an annuity payment model. DESIGN, SETTING, AND PARTICIPANTSA budget impact analysis was performed from January 1 to May 31, 2020, for a sickle cell disease gene therapy from the perspective of 10 state Medicaid plans with a 5-year time horizon, using state-level disease prevalence data from 2012. Disease prevalence, Medicaid enrollment, and expenditures were derived from the available literature. The eligible population was based on modified clinical trial inclusion criteria including individuals aged 13 to 45 years with severe disease.EXPOSURES The gene therapy was assumed to be administered to 7% of the eligible population annually and was curative (no subsequent disease-related expenditures). The gene therapy price was $1.85 million in the base case, and baseline disease-related expenditures were $42 200 per year. MAIN OUTCOMES AND MEASURESThe main outcomes were total budget impact and budget impact per member per month in years 1 through 5. One-way sensitivity analysis was used to evaluate uncertainty of market diffusion, size of eligible population, price of therapy, and cost of routine care. RESULTSAn estimated 5464 Medicaid enrollees would be eligible for the gene therapy nationally, with 2315 individuals in the 10 Medicaid programs of interest (16 per 100 000 enrollees). The model projected a mean 1-year budget impact of $29.96 million per state Medicaid program in the sample ($1.91 per member per month). A 5-year annuity payment reduced the short-term budget impact. CONCLUSIONS AND RELEVANCEThis study suggests that a gene therapy for severe sickle cell disease is likely to produce a considerable budget impact for many Medicaid plans while potentially offering substantial benefit to patients. Payers may need to take steps to ensure affordability and access. Gene therapy for sickle cell disease is likely to provide an early demonstration of the unique financial challenges associated with this emerging drug class.

Development of a conceptual model for evaluating new non-curative and curative therapies for sickle cell disease

“…Following the eligibility criteria of several clinical trials, we will initially restrict treatment to patients with severe SCD, defined based on their history of severe adverse events [ 58 ]. The cost of treatment will be based on the price of genetic therapy for beta-thalassemia [ 14 , 59 ]. We will use scenario analyses to account for considerable uncertainty in the price of genetic therapies, its effectiveness over a patient’s lifetime and among patients with less severe SCD, and the potential for induced mutagenesis and other treatment-related complications.…”

Gene Therapy for Neuromuscular Diseases: Health Economic Challenges and Future Perspectives

“…Therefore, the reimbursement of a new gene therapy is likely to put significant pressure on the payer’s budget [ 68 ]. For example, DeMartino et al [ 69 ] concluded that the reimbursement of a gene therapy for sickle cell disease would likely present affordability challenges to several Medicaid health plans, and Alhakamy et al [ 17 ] estimated that the total cost of gene therapies for only 10% of US patients with genetic diseases (accounting to about 1% of the total population, according to the authors) could be as large as $3 trillion, roughly 75% of the total national healthcare expenditure in 2020 [ 70 ].…”

Lifetime medical costs attributable to sickle cell disease among nonelderly individuals with commercial insurance

“…2 SCD-related lifetime medical costs were estimated at $1.2 million (2020 US dollars). 8 , 9 Despite being nearly 20 years old and derived from a single state’s Medicaid program, this estimate is widely used to establish the baseline costs of medical care for SCD. 9 , 10 , 11 , 12 Other studies of the burden of medical costs are limited to descriptive summaries, 5 , 6 , 7 , 8 , 13 and none provide clear estimates of the lifetime incremental medical costs paid by health insurers or, importantly, the out-of-pocket (OOP) burden that SCD individuals face compared with those without SCD or related blood disorders.…”