Beyond the budget impact model: A case study for glucarpidase and the real cost of managing curable toxicities.

The greatest challenge in healthcare today lies in managing limited resources to deliver high-quality care to the patients who need it the most. Payers heavily rely on budget impact models to assess the net costs or potential savings associated with adding a new intervention and to inform their formulary decision. Drugs developed for rare conditions are often prohibitively expensive due to the complex manufacturing processes and investments required to undertake clinical trials.

Economic Model of Uridine Triacetate Versus Supportive Care for the Treatment of Patients with Life-Threatening Early-Onset Severe Toxicity.

Background: Early-onset severe toxicity following the administration of 5-fluorouracil (5-FU) or capecitabine occurs in approximately 10-30% of patients receiving fluoropyrimidine therapy in the USA and is fatal to at least 0.5% of patients treated. Supportive care measures used to manage symptoms of toxicity are associated with extended hospital length of stay, high cost of care, and poor survival.

Bone Health Management in Elective Orthopaedic Surgery: A Claims-Based Observational Study.

Introduction: There are limited data on the management of bone health, including bone mineral density (BMD) evaluation and osteoporosis (OP) treatment, in patients undergoing elective orthopaedic surgeries.

Methods: This was a retrospective cohort study using administrative claims data from Symphony Health, PatientSource for patients aged ≥50 years with documented kyphoplasty/vertebroplasty (KP/VP), total knee arthroplasty (TKA), and total hip arthroplasty (THA). Risk stratification to identify patients at very high risk for fracture (VHRFx) was based on clinical practice guideline recommendations to the extent information on variables of interest were available from the claims database.

Treatment patterns in women with postmenopausal osteoporosis using abaloparatide: a real-world observational study.

Unlabelled: Review of medical records from 173 women with osteoporosis who received abaloparatide treatment revealed that 96.0% had at least one visit for osteoporosis management and 55.5% had medication support group access.

Real-World Evidence to Support the Registration of a New Osteoporosis Medicinal Product in Europe.

Real-World Evidence (RWE), which has historically been used to support post-approval safety studies, has recently gained acceptance for new drug applications as supportive evidence or as new clinical evidence for medicinal products with orphan designation and/or in disease areas with high unmet need. Here, we present a case study for the use of RWE in the approval of abaloparatide in the European Union (EU) under the tradename Eladynos. In addition to data from the pivotal Phase 3 study, the marketing authorization application (MAA) included clinical data from additional interventional and observational studies, as well as post-marketing data obtained from the United States (US) market since approval of abaloparatide by the Food and Drug Administration (FDA) in 2017.

Real-World Management of Patients With Osteoporosis at Very High Risk of Fracture.

Introduction: Lack of consideration for risk-based assessments that inform osteoporosis treatment decisions may contribute to disease burden. In this study, we examined the prevalence of patients at very high risk of fracture and evaluated real-world treatment practices for these patients.

Methods: This retrospective observational cohort study used real-world data linked to commercial and Medicare medical claims from Symphony Health PatientSource.

An Evaluation of Treatment Patterns for Osteoporosis and Outcomes After a Fragility Fracture in a Real-World Setting.

Objectives: Treatment initiation and persistence after a fragility fracture are critical to reduce the risk of subsequent fractures. The authors evaluated osteoporosis management and outcomes after index fracture.

Methods: This retrospective cohort study used real-world data for patients (≥50 years), including pharmacy claims linked to commercial and Medicare medical claims from Symphony Health Patient Source.

A Qualitative Study to Assess US Patient Preferences between new Transdermal System and Injectable Anabolic Therapies for Osteoporosis Treatment.

Unlabelled: US patients with osteoporosis included in three focus groups identified efficacy, safety, cost, and convenience as important attributes of treatment when choosing between anabolic therapies with high stated preference for the solid Microstructured Transdermal System.

Objective: The current study evaluated patient perspective and relative importance of treatment attributes of in-home daily self-administration of abaloparatide-solid Microstructured Transdermal System (sMTS) compared with other anabolic agents (i.e.

Outcomes post fragility fracture among members of an integrated healthcare organization.

Unlabelled: This study highlights an unmet need in osteoporosis management, suggesting that beyond bone mineral density and fracture history, gender, fracture type, and age should be considered for fracture risk assessment. Following fragility fracture, men, patients with a spine or hip fracture, and those aged ≥ 65 have a higher disease burden.

Introduction: The objective of this study was to characterize osteoporosis-related fracture incidence and identify predictors of subsequent fractures and mortality.

Development and validation of a one year predictive model for secondary fractures in osteoporosis.

The number of osteoporosis-related fractures in the United States is no longer declining. Existing risk-based assessment tools focus on long-term risk. Payers and prescribers need additional tools to identify patients at risk for imminent fracture.

Gender Disparities in Osteoporosis Screening and Management Among Older Adults.

Introduction: One in two women and one in four men experience an osteoporosis-related fracture in their lifetime. Related morbidity and mortality rates are higher in men versus women. Current guidelines are inconsistent in the screening recommendations for osteoporosis in men.

Risk Factors for Incident Fracture in Older Adults With Type 2 Diabetes: The Framingham Heart Study.

Objective: To identify risk factors for fracture in type 2 diabetes.

Research Design And Methods: This prospective study included members of the Framingham Original and Offspring Cohorts. Type 2 diabetes was defined as fasting plasma glucose >125 mg/dL or use of type 2 diabetes therapy.

Abaloparatide Real-World Patient Experience Study.

Despite the availability of various osteoporosis treatments, adherence remains suboptimal. One contributing factor may be patient experience with therapy. This US, multicenter, combined retrospective chart review and patient questionnaire study included postmenopausal women at high risk for fracture and is the first study to describe real-world patient experience with abaloparatide (ABL) injection.

Phase 1b Evaluation of Abaloparatide Solid Microstructured Transdermal System (Abaloparatide-sMTS) in Postmenopausal Women with Low Bone Mineral Density.

Background And Objective: Abaloparatide, an anabolic osteoporosis treatment administered by subcutaneous (SC) injection, increases bone mineral density (BMD) and reduces fracture risk in postmenopausal women with osteoporosis. The abaloparatide-solid Microstructured Transdermal System [abaloparatide-sMTS (Kindeva, St Paul, MN, USA)], which delivers abaloparatide intradermally, is in development to provide an alternative method for abaloparatide delivery. The objective of this study was to evaluate the ability of subjects to self-administer abaloparatide-sMTS, based on pharmacokinetic and pharmacodynamic markers.

Economic Burden of Osteoporosis-Related Fractures in the US Medicare Population.

Background: Osteoporosis-related fractures are an important public health burden.

Objective: To examine health care costs in Medicare patients with an osteoporosis-related fracture.

Methods: Medicare fee-for-service members with an osteoporosis-related fracture between January 1, 2010, to September 30, 2014 were included.

Effect of Abaloparatide on Bone Mineral Density and Fracture Incidence in a Subset of Younger Postmenopausal Women with Osteoporosis at High Risk for Fracture.

Purpose: Current treatment guidelines recommend treatment for postmenopausal women with a T score <2.5 regardless of age. This subgroup analysis evaluated the efficacy and safety of abaloparatide in younger postmenopausal women considered to be at high risk for fracture.

Economic burden of osteoporotic fractures in US managed care enrollees.

Objectives: To examine healthcare resource utilization (HRU) and costs in a population of managed care enrollees who experienced an osteoporotic fracture.

Study Design: Retrospective cohort study using the Optum Research Database (January 2007 to May 2017).

Methods: All-cause and osteoporosis-related HRU and costs were analyzed in patients 50 years and older with a qualifying index fracture and continuous enrollment with medical and pharmacy benefits for 12 months preindex (baseline period).

Cost-effectiveness of sequential treatment with abaloparatide followed by alendronate vs. alendronate monotherapy in women at increased risk of fracture: A US payer perspective.

Objectives: Emerging evidence supports sequential therapy with anabolic followed by antiresorptive in patients at high-risk of fragility fractures. This study assessed the cost-effectiveness of sequential treatment with abaloparatide (ABL) followed by alendronate (ALN) [(ABL/ALN)] compared to ALN monotherapy and to sequential treatment starting with antiresorptive therapy (ALN/ABL/ALN).

Methods: A previously validated Markov microsimulation model was used to estimate the cost-effectiveness of sequential ABL/ALN compared to ALN monotherapy and to sequential ALN/ABL/ALN from a lifetime US payer perspective.

Impact of fractures on quality of life in patients with osteoporosis: a US cross-sectional survey.

To evaluate the impact of osteoporosis-related fractures on health-related quality of life (HRQoL). Data were obtained from the 2016 Adelphi US Osteoporosis Disease Specific Programme™, a cross-sectional survey of physicians and their male and female patients with osteoporosis. Patient-reported outcomes (PRO) measures included the European Quality of Life 5 Domains (EQ-5D), European Quality of Life Visual Analog Scale (EQ-VAS), and Osteoporosis Assessment Questionnaire short-version (OPAQ-SV; physical, emotional, and symptom domains).

Challenges in osteoporosis awareness and management: results from a survey of US postmenopausal women.

Osteoporotic fractures are a serious public health burden, leading to excess morbidity, mortality, and high costs for post-fracture care. To evaluate the perception of osteoporotic fracture risk and to identify potential barriers associated with osteoporosis diagnosis and treatment in US postmenopausal women. An online survey was conducted by The Harris Poll in 2017 that included 1,012 postmenopausal women 50 years and older.

Cost-effectiveness of sequential treatment with abaloparatide vs. teriparatide for United States women at increased risk of fracture.

Objectives: There is emerging evidence supporting sequential therapy with an osteoanabolic followed by an antiresorptive in patients at high-risk of fragility fractures. This study assessed the cost-effectiveness of sequential treatment with abaloparatide (ABL) followed by alendronate (ALN) [(ABL/ALN)] compared with teriparatide (TPTD) followed by ALN (TPTD/ALN).

Methods: A previously validated Markov microsimulation model was adapted to estimate the cost-effectiveness of sequential ABL/ALN compared with sequential TPTD/ALN and no treatment with a lifetime horizon from the US payer perspective.

Discontinuation of Biologic Therapy in Rheumatoid Arthritis: Analysis from the Corrona RA Registry.

Introduction: Despite the availability of multiple effective therapies, discontinuation/switching of treatment is common for many patients with rheumatoid arthritis (RA). This study was designed to examine initiation of biologic disease-modifying anti-rheumatic drugs (bDMARDs) within the Consortium of Rheumatology Researchers of North America (Corrona) RA Registry, and characterize reasons for discontinuation.

Methods: Inclusion criteria were: Corrona-registered adults (≥18 years) with RA (2002-2011); age of RA onset: ≥16 years; ≥6 months' follow-up after initiation of first/subsequent bDMARD.

Health Care Utilization and Costs After Initiating Budesonide/Formoterol Combination or Fluticasone/Salmeterol Combination Among COPD Patients New to ICS/LABA Treatment.

Background: Chronic obstructive pulmonary disease (COPD) affects approximately 15 million people in the United States and accounts for approximately $36 billion in economic burden, primarily due to medical costs. To address the increasing clinical and economic burden, the Global Initiative for Chronic Obstructive Lung Disease emphasizes the use of therapies that help prevent COPD exacerbations, including inhaled corticosteroid/long-acting beta2-agonist (ICS/LABA).

Objective: To evaluate health care costs and utilization among COPD patients newly initiating ICS/LABA combination therapy with budesonide/formoterol (BFC) or fluticasone/salmeterol (FSC) in a managed care system.

Comparative effectiveness of budesonide/formoterol combination and tiotropium bromide among COPD patients new to these controller treatments.

Background: Inhaled corticosteroid/long-acting β2-agonist combinations and/or long-acting muscarinic antagonists are recommended first-line therapies for preventing chronic obstructive pulmonary disease (COPD) exacerbation. Comparative effectiveness of budesonide/formoterol combination (BFC, an inhaled corticosteroid/long-acting β2-agonist combination) vs tiotropium (long-acting muscarinic antagonist) in the US has not yet been studied.

Methods: Using US claims data from the HealthCore Integrated Research Environment, COPD patients (with or without comorbid asthma) ≥40 years old initiating BFC or tiotropium between March 1, 2009 and February 28, 2012 and at risk for exacerbation were identified and followed for 12 months.