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and incidence data from a recent meta-analysis (2019), US population estimates from the US Census Bureau (2019), and unpublished market share assumptions (2020).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 RedBook 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 underreporting. 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.
