Budgetary Impact of Adding Riociguat to a US Health Plan for the Treatment of Patients with Pulmonary Arterial Hypertension or Chronic Thromboembolic Pulmonary Hypertension.

Background: Pulmonary arterial hypertension (PAH) and chronic thromboembolic pulmonary hypertension (CTEPH) are chronic, debilitating, and life-threatening conditions. Riociguat is the first and only pharmacotherapy approved by the US Food and Drug Administration (FDA) for the treatment of PAH and for CTEPH in patients who are either inoperable or have persistent pulmonary hypertension after surgery.

Objective: To estimate the budgetary impact of adding riociguat to a US health plan's formulary for the treatment of patients with PAH or CTEPH using a budget impact analytic model.

Methods: A customizable, Microsoft Excel-based decision analytic tool was developed to estimate the impact of riociguat on per-member per-month (PMPM) and per-member per-year (PMPY) bases in Medicare and non-Medicare health plans. The economic impact was calculated based on 1 million insured lives, published prevalence estimates of PAH and CTEPH, pharmacotherapy-eligible patients with PAH or CTEPH, administration costs, and monitoring costs related to pharmacotherapy. The drug costs were based on wholesale acquisition costs, and the medical costs were derived from Truven Health MarketScan claims data and the Medicare 2013 Clinical Diagnostic Laboratory Fee Schedule and Physician Fee Schedule. The market share for approved treatments was based on a tracking study of physicians treating patients with PAH or CTEPH. A sensitivity analysis was used to test the model's robustness.

Results: In a hypothetical plan population of 1 million members, the model estimated that 7 patients with PAH and 2 patients with CTEPH would be suitable for pharmacotherapy. Overall, 3 patients (1 with PAH and 2 with CTEPH) were receiving riociguat in a health plan consisting of patients with commercial and with Medicare insurance coverage. The incremental PMPY and PMPM costs for providing insurance coverage for riociguat were $0.27 and $0.02, respectively, for non-Medicare and Medicare health plans. Sensitivity analyses indicated that the budget impact increased by $0.01 PMPM, with a 25% increase in base-case parameter values.

Conclusion: Riociguat is a first-in-class and the only FDA-approved treatment for patients with PAH or CTEPH-2 debilitating, chronic, and life-threatening conditions with poor prognosis. This drug offers health plans an effective and safe treatment option, with a minimal economic impact. The financial impact to a health plan of providing coverage for riociguat in the first year of treatment was as low as $0.02 PMPM. The real-world budget impact of riociguat needs to be measured using real-world evidence to validate our results.