Describing the Patient Journey of Women with Claims for Uterine Fibroids and Heavy Menstrual Bleeding Using a Commercial Database (2011-2020).

Introduction: This retrospective database claims analysis describes the clinical characteristics and treatment patterns of commercially insured United States women with uterine fibroids (UF) and heavy menstrual bleeding (HMB). Methods: Women age 18-55 years with an incident UF diagnosis (index date) between 1/1/2012 and 12/31/2019 and ≥1 claim for HMB (UF-HMB), were identified from the Optum® Clinformatics® database. Outcomes included clinical characteristics, pharmacologic therapy use, and surgeries/procedures. Regression models were used to identify factors associated with time to post-diagnosis hormonal therapy and hysterectomy. Results: A total of 85,428 women had UF-HMB (mean [SD] age, 43.7 [6.4] years). The median follow-up was 3.2 years. After HMB, the most common symptoms were pelvic pressure/pain (27.6%) and backache (17.5%). Within 6 months of UF diagnosis, 40.2% of patients had received only pharmacologic therapy; 25.5% had received no treatment; 24.3% had a hysterectomy, and 10.0% had other procedures. By the end of follow-up, 50.0% had received a hysterectomy. Multiple factors were predictive of a higher likelihood of receiving hormonal therapy (geographic region, infertility, pre-index pregnancy) or hysterectomy (older age, prior hormonal treatment, specific bulk symptoms, White race). Conclusion: Within 6 months of UF diagnosis, fewer than one-half of women with UF-HMB had received hormonal therapy, one-quarter received no treatment, and one-quarter had received a hysterectomy or another gynecologic procedure. Patients who received a hysterectomy were more likely to be older, White, and to have bulk symptoms.

Treatment Patterns in Patients with Uterine Fibroids With and Without a Diagnosis of Heavy Menstrual Bleeding: Results from a Large U.S. Claims Database.

Background: This retrospective database analysis describes clinical characteristics and treatment patterns of U.S. women with a diagnosis for uterine fibroids (UF), both with and without heavy menstrual bleeding (HMB). Materials and Methods: Two cohorts aged 18-50 years with an incident UF diagnosis, comprising women with and without claims for HMB (UF-HMB and UF-only), were identified from the IQVIA PharMetrics® Plus database (January 1, 2010-December 31, 2019). The index date was the first UF claim following diagnosis; treatment patterns were documented for postindex years 1 and 2 and the full duration of postindex follow-up. Also identified were claims for symptoms or signs potentially associated with UF. Outcomes were the proportion of patients treated with pharmacologic therapies of interest and gynecologic procedures. Logistic regression was used to identify factors associated with postdiagnosis hysterectomy and hormonal therapy. Results: A total of 66,313 (71.8%) women were included in the UF-HMB cohort (mean age [standard deviation]) 42.6 [5.4] years), and 26,068 (28.2%) in the UF-only cohort (41.8 [6.3]). Median follow-up was ∼4 years. Pain was the most common symptom (42.7% in patients with UF-HMB and 36.6% with UF-only); also common were abnormal bleeding (15.6%, 11.5%) and fatigue (22.2%, 15.5%). Within 1 year of UF diagnosis, 28.8% and 49.2% of women with UF-HMB and UF-only, respectively, had no claims for relevant pharmacologic or surgical treatment. In logistic regression, multiple factors were associated with a higher likelihood of receiving hysterectomy or hormonal therapy. Conclusions: Patients with UF-HMB were more likely to receive UF treatment, either surgical or pharmacologic, than women with UF-only. Apart from HMB, pain was the most commonly documented symptom of UF.

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.

Adherence to therapy for pulmonary arterial hypertension is essential to optimize patient outcomes, but data on real-world adherence to different pulmonary arterial hypertension drug classes are limited. This retrospective database analysis evaluated relationships between adherence, hospitalization, and healthcare costs in pulmonary arterial hypertension patients treated with endothelin receptor antagonists or phosphodiesterase type-5 inhibitors. From the IQVIA Adjudicated Health Plan Database, patients with pulmonary arterial hypertension were identified based on diagnostic codes and prescriptions for endothelin receptor antagonists (ambrisentan, bosentan, macitentan) or phosphodiesterase type-5 inhibitors (sildenafil, tadalafil) approved for pulmonary arterial hypertension. Patients were assigned to the class of their most recently initiated (index) pulmonary arterial hypertension therapy between 1 January 2009 and 30 June 2015. Medication adherence was measured by proportion of days covered; patients with proportion of days covered ≥80% were considered adherent. The proportion of adherent patients was higher for endothelin receptor antagonists (571/755; 75.6%) than for phosphodiesterase type-5 inhibitors (970/1578; 61.5%; P < 0.0001). In both groups, hospitalizations declined as proportion of days covered increased. Among adherent patients, those on endothelin receptor antagonists had a significantly lower hospitalization rate than those on phosphodiesterase type-5 inhibitors (23.1% versus 28.5%, P = 0. 0218), fewer hospitalizations (mean (standard deviation) 0.4 (0.8) versus 0.5 (0.9); P = 0.02), and mean hospitalization costs during the six-month post-index ($9510 versus $15,726, P = 0.0318). Increasing adherence reduced hospitalization risk more for endothelin receptor antagonists than for phosphodiesterase type-5 inhibitors (hazard ratio 0.176 versus 0.549, P = 0.001). Rates and numbers of rehospitalizations within 30 days post-discharge were similar between groups. Mean total costs were higher with endothelin receptor antagonists than phosphodiesterase type-5 inhibitors in all patients ($91,328 versus $72,401, P = 0.0003) and in adherent patients ($88,867 versus $56,300, P < 0.0001), driven by higher drug costs.

The myth of the stable pulmonary arterial hypertension patient.

Pulmonary arterial hypertension (PAH) is a rare, progressive disease that often leads to right heart failure and premature death. Despite increased awareness and an expanding treatment landscape in recent decades, long-term prognosis is poor for patients with PAH. Recently, emphasis has evolved from goal-oriented therapy to risk-assessment and achieving low-risk status. Findings from recent clinical trials suggest that functional class II patients, long assumed to be stable, are not stable. Therefore, frequent assessment of all patients with PAH is essential toward escalating treatment as indicated to optimize clinical outcomes. Lowering mortality risk, preventing disease progression, and optimizing quality of life of patients with PAH is paramount.

Prostacyclin Use Among Patients with Pulmonary Arterial Hypertension in the United States: A Retrospective Analysis of a Large Health Care Claims Database.

BACKGROUND: Prostacyclins play an important role in the management of pulmonary arterial hypertension (PAH). Intravenous prostacyclin was the first disease-specific treatment for patients with PAH. Subcutaneous and nonparenteral (oral or inhaled) formulations have subsequently become available. However, data are lacking on how these different prostacyclin formulations are being used in clinical practice. OBJECTIVES: To (a) conduct retrospective analyses of a large U.S. health care claims database to describe the characteristics of patients with PAH initiating prostacyclin therapy, and (b) evaluate their treatment patterns, health care resource use, and associated costs. METHODS: Truven Commercial and Medicare databases were used to define annual cohorts of adults with PAH between January 1, 2010, and October 31, 2015. These patients were identified based on claims with ICD-9-CM diagnoses indicative of PAH (codes 416.0 or 416.8) and claims for PAH-specific medications and PAH-related procedures. Patients with evidence of receiving a prostacyclin were identified, and prostacyclin use was categorized as parenteral versus nonparenteral. Health care costs were assessed alternatively employing an all-cause and PAH-related perspective. RESULTS: Of 13,633 adults with identified PAH, 3,006 (22.0%) received a prostacyclin during at least 1 year of the study period, and annual prevalence of prostacyclin use ranged from 19.9% to 22.6%. Across calendar years, the median age of prostacyclin users ranged from 56 to 58 years, and 71.9%-75.8% were female. Among prostacyclin users, parenteral prostacyclin use declined from 63.2% in 2010 to 46.5% in 2015, while use of nonparenteral prostacyclins increased from 39.7% to 56.2% over the same period (both P < 0.001). Few patients (2.7%-4.1%) received both parenteral and nonparenteral formulations in a given calendar year. Among patients using prostacyclins, receipt of other PAH-specific medications increased from 62.1% in 2010 to 79.2% in 2015. Comparing the 6 months preceding the first prostacyclin prescription (any formulation) to the 6 months subsequent, mean overall health care costs rose from $61,243 to $119,283, and PAH-related health care costs increased from $58,815 to $116,661, driven mainly by PAH-specific medications, spending on which increased from $15,053 to $73,705 (all P < 0.001). CONCLUSIONS: While overall use of prostacyclins was relatively constant from 2010 to 2015, our findings revealed a shift from parenteral to nonparenteral formulations, coupled with increased prescribing of PAH-related medications from other drug classes. Further research is needed to better understand how these changes in patterns of prostacyclin use affect levels of health care resource utilization and costs and patients' overall quality of life. DISCLOSURES: This research was funded by Actelion Pharmaceuticals US, a Janssen pharmaceutical company of Johnson & Johnson. Burger has received grant funding from Actelion, Gilead Sciences, and United Therapeutics; personal fees from Actelion and Gilead Sciences; and nonfinancial support from Actelion. Pruett, Lickert, and Drake are employees of Actelion. Pruett and Lickert own shares in Actelion. Berger and Murphy are employees of Evidera, a consultancy that received payment from Actelion to conduct this research. Pruett, Lickert, Berger, and Drake contributed to study conception and participated with Burger in study design. Lickert and Murphy performed the data analyses. Burger, Pruett, Lickert, Murphy, and Drake interpreted the data. All authors participated in manuscript drafting and/or critical revision, approved the final manuscript, and agree to be accountable for all aspects of the work.

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

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. 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.

Healthcare resource utilization and costs for patients with pulmonary arterial hypertension: real-world documentation of functional class.

BACKGROUND AND AIMS: Pulmonary arterial hypertension (PAH) is a rare medical disease in which patients experience increased pulmonary vascular resistance (PVR) and pulmonary arterial pressure that can result in remodeling of the pulmonary vasculature and heart, and eventually lead to right heart failure and death. As PAH progresses, patients become unable to perform even routine daily tasks without severe shortness of breath (dyspnea), fatigue, dizziness, and fainting (syncope). Treatment strategies largely depend on assessment of an individual patient's WHO Functional Class. The aim of the present study was to determine whether PAH functional decline, as described by the WHO Functional class (FC), is associated with increased healthcare costs for patients. METHODS: Patients with a prescription for a FDA-approved treatment for PAH and a medical claim indicating chronic pulmonary heart disease or right heart catheterization were identified from an administrative claims database. Provider-reported data from prior authorization forms required for advanced PAH therapies and medical charts were examined for reported FC. Healthcare resource utilization and costs were the primary outcomes of interest. Costs were accounted in 2014 US dollars ($) from a healthcare payer perspective. RESULTS: Patients with a reported FC-IV were observed to have the worst outcomes; averaging significantly more inpatient admissions, longer average lengths of stay, and more emergency department visits than the other FC sub-groups, resulting in higher medical costs. CONCLUSIONS: Using administrative data to document disease severity, this study replicates and expands on findings obtained from the registry study; disease severity was associated with higher healthcare resource utilization and costs. Stakeholders' implications for patient management are discussed.

Combination therapy in pulmonary arterial hypertension: is this the new standard of care?

Pulmonary arterial hypertension (PAH) is a rare, progressive, and potentially fatal cardiopulmonary syndrome that imposes a significant burden on patients in terms of morbidity and mortality, and on managed care organizations in terms of resource utilization. The majority of PAH-approved therapies are high-touch, high-management, high-cost treatments dispensed through specialty pharmacies. Current treatment guidelines recommend combination therapy for patients who show inadequate clinical response or who deteriorate on monotherapy. Combination therapies target 2, or sometimes 3, distinct PAH-associated signaling pathways: the endothelin, prostacyclin, and nitric oxide pathways. Registry data suggest that combination therapy is utilized in more than half of patients with PAH in the United States. Evidence supporting the use of combination therapy is provided through clinical trials, retrospective research, registry data, and expert guidelines. Managed care decision makers are charged with making population-based decisions on resource allocation. These decision makers must always consider cost, but must also be aware that clinical evidence suggests that early treatment with combination therapy can significantly reduce disease burden, may reduce hospitalizations, and should be considered when making coverage decisions.