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Background: Pulmonary arterial hypertension (PAH) is described by proliferation of small pulmonary arteries leading to increased pulmonary vascular resistance, right ventricular failure, and death. Research confirms longterm improvement in composite morbidity and mortality endpoints on some endothelin receptor antagonists alone and in combination with phosphodiesterase type 5 inhibitors (PDE-5is) but not with PDE-5i monotherapy. While current treatment guidelines incorporate these findings, a substantial number of patients are started or maintained on PDE-5i monotherapy. Objectives: This study describes real-world clinical practice and treatment patterns with PDE-5i monotherapy including events indicative of clinical worsening, treatment modifications, adherence, allcause healthcare resource utilization, and costs. Methods: This retrospective study analyzed PharMetrics Plus claims data including 150 million lives; study period was January 1, 2009 through December 31, 2013. Eligible patients were ≥18 years with ≥1 inpatient or ≥2 outpatient claims ≥30 days apart, a diagnosis of pulmonary hypertension or other chronic pulmonary heart disease, and an initial PDE-5i prescription. To include only World Health Organization group 1 PAH patients, ≥1 encounter for right-heart catheterization or Doppler echocardiogram was required during the pre-index period. Results: PDE-5i monotherapy for PAH treatment was associated with high treatment modification rates, low adherence, increased healthcare resource utilization, and high costs. At 12 months post index, 41.5% of patients experienced treatment modification. For the index therapy, 47% of patients had ≥80% adherence to therapy. Almost 50% of patients had ≥1 hospitalization, with costs increased three fold to $197 111 compared to $59 164 for non-hospitalized patients. Conclusions: Initial treatment with PDE-5i monotherapy was associated with substantial direct medical costs, including hospitalizations and emergency department visits, low therapy adherence and a high rate of treatment modifications.

journal of health economics and outcomes research

Using Real World Evidence to Describe Pulmonary Arterial Hypertension Treatment Patterns, Healthcare Resource Utilization, and Costs Associated with PDE-5 Inhibitor Monotherapy