BACKGROUND: Eosinophilic esophagitis (EoE) is a chronic, immune-mediated, inflammatory disease characterized by esophageal dysfunction and eosinophilia. Dietary elimination therapy (‘diet’) and off-label treatments, including proton-pump inhibitors (PPIs), topical corticosteroids (CS), and in rare severe cases, systemic CS, are used to manage EoE. Currently, there is no US Food and Drug Administration (FDA)-approved treatment for EoE.
OBJECTIVE: We aimed to define the number of patients diagnosed with EoE and understand off-label treatment use patterns over time. These data were used to estimate the cost burden of EoE to the US health system.
METHODS: A 5-year analysis was conducted to define the patient population size and treatment patterns for adolescents (11–17 years old) and adults (≥ 18 years old) with EoE. The analysis used EoE prevalence and incidence data from a recent meta-analysis (2019), US population estimates from the US Census Bureau(2019), and unpublished market share assumptions (2020). Off-label drug costs were generated using wholesale acquisition costs from the Red Book 2020 drug pricing database and published literature for diet costs; costs were reported in US dollars.
RESULTS: It is estimated that 11 211 adolescents and 81 409 adults had EoE in the USA in 2019. Over the next 5 years, the US EoE patient population is estimated to grow by 20 565 patients per year on average. Off-label therapy use for EoE was estimated to be 40% for CS, 12% for diet and 12% for PPIs. Off-label CS use is projected to be substantially higher than diet use over the next 5 years (46% and 14% use in year 5, respectively) if no treatments are approved. Annual costs for off-label treatments are estimated to be over $128 million in 2020 and are expected to rise to over $247 million in the next 5 years if no new EoE therapies are approved by the FDA.
CONCLUSIONS: This analysis demonstrated that, given the rising number of patients with EoE, off-label treatment use and costs to the US healthcare system are expected to increase over the next 5 years, if current treatment patterns continue. Prevalence and incidence estimates were based on US claims data and were not adjusted for potential under-reporting. The approval of EoE treatments would alleviate the significant unmet medical need of this disease and reduce the use and costs of off-label therapy.
