Abstract
BACKGROUND AND OBJECTIVE: Half of patients with gastroesophageal reflux disease (GERD) experience persistent symptoms while on proton pump inhibitors (PPIs), thus driving efforts to develop novel adjunctive therapies for PPI-refractory GERD. An economic analysis was performed to establish at what cost and efficacy such potential medications are likely to become cost effective in clinical practice. METHODS: A Markov decision model was used to examine a hypothetical cohort of patients being evaluated for PPI-refractory GERD in the USA. The model compared 3 strategies: (1) usual care (i.e., upfront diagnostic testing with upper endoscopy ± ambulatory pH testing); (2) use of a PPI-adjunctive therapy after positive ambulatory pH testing; and (3) empiric use of a PPI-adjunctive therapy (i.e., diagnostic testing only after failing empiric treatment). The primary outcome was incremental cost per quality-adjusted life year (QALY) gained (third-party payer perspective) over a 10-year time horizon using a willingness to pay threshold of $100,000/QALY. RESULTS: In two-way sensitivity analyses varying the cost and effectiveness of the PPI-adjunctive therapy, most combinations revealed that use of the medication after positive pH testing was the most cost-effective approach. Empiric treatment was the preferred strategy only when the therapy was highly efficacious (≥ 87.5% response rate) and low cost (≤ $109/month). Use of PPI-adjunctive treatments were not cost effective when the cost exceeded $1150/month. CONCLUSION: Use of PPI-adjunctive therapies in those with persistent GERD symptoms may become cost effective when guided by ambulatory pH tests. These data can guide investigators, industry, and payers as they develop, validate, and price new treatments for PPI-refractory GERD.