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

Abstract

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 long-term 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.</p> 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.

Medication adherence, hospitalization, and healthcare resource utilization and costs in patients with pulmonary arterial hypertension treated with endothelin receptor antagonists or phosphodiesterase type‐5 inhibitors

“…Patients were considered to be adherent to therapy if their PDC was !80%, the commonly accepted threshold for good adherence to PAH therapies 16,17 and other pulmonary and cardiovascular medications. 13,22 Hospitalizations and rehospitalizations within 30 days of discharge were quantified as rates (i.e.…”

“…Overlapping days from an early refill were counted only once from the end of the previous days’ supply. Patients were considered to be adherent to therapy if their PDC was ≥80%, the commonly accepted threshold for good adherence to PAH therapies 16 , 17 and other pulmonary and cardiovascular medications 13 , 22 …”

“…Previous retrospective studies of PAH-specific therapies have indicated that adherence to PAH-specific drugs may vary by drug class, as high adherence to treatment has been reported with endothelin receptor antagonists (ERAs), 15 whereas relatively low adherence to treatment with phosphodiesterase type-5 inhibitors (PDE-5is) has been observed in other studies. 16,17 ERAs and PDE-5is are readily comparable because all are oral agents that are often used in first-line therapy. 5 In contrast, PAH medications that target the prostacyclin pathway include intravenous, subcutaneous, inhaled, and oral agents, most of which are typically reserved for later in the treatment pathway and for patients at high risk for clinical worsening.…”

“…Previous retrospective studies of PAH‐specific therapies have indicated that adherence to PAH‐specific drugs may vary by drug class, as high adherence to treatment has been reported with endothelin receptor antagonists (ERAs), 15 whereas relatively low adherence to treatment with phosphodiesterase type‐5 inhibitors (PDE‐5is) has been observed in other studies 16 , 17 . ERAs and PDE‐5is are readily comparable because all are oral agents that are often used in first‐line therapy 5 .…”

Excess healthcare resource utilization and costs for commercially insured patients with pulmonary arterial hypertension: A real‐world data analysis

“… 5 Delayed diagnosis and initiation of therapy leads to a poor prognosis, 6 which could lead to a significant economic burden for PAH patients. Published studies have estimated healthcare resource utilization (HCRU) and associated costs among patients with PAH 7 , 8 , 9 ; however, there are few studies estimating the excess burden of PAH relative to patients without PH. For example, a recent study reported the direct healthcare costs (2018 USD) were $9915 PPPM for 1293 commercially insured patients with prevalent PAH as compared to $359 for demographically (age, sex, region) matched non‐PAH patients.…”