Myocarditis in Patients Starting Combination Checkpoint Inhibitor Therapy: Analysis of a Commercial Claims Database.

Background: Immune checkpoint inhibitors have improved the clinical outcomes of several cancers but have also been associated with a greater risk of immune-related adverse effects, especially when combined. The objective of this study was to investigate the incidence of myocarditis in relation to the use of dual concurrent versus single immune checkpoint inhibitors therapies.

Methods And Results: A cohort study was conducted using medical and pharmacy claims data (2011-2022) from a large US commercial insurer.

Calibrating Observational Health Record Data Against a Randomized Trial.

Importance: The conditions required for health record data sources to accurately assess treatment effectiveness remain unclear. Emulation of randomized clinical trials (RCTs) with health record data and subsequent calibration of the results can help elucidate this.

Objective: To pilot an emulation of the KEYNOTE-189 RCT using a commercially available electronic health record (EHR) data source.

Sharing Is Caring? International Society for Pharmacoepidemiology Review and Recommendations for Sharing Programming Code.

Purpose: There is increasing recognition of the importance of transparency and reproducibility in scientific research. This study aimed to quantify the extent to which programming code is publicly shared in pharmacoepidemiology, and to develop a set of recommendations on this topic.

Methods: We conducted a literature review identifying all studies published in Pharmacoepidemiology and Drug Safety (PDS) between 2017 and 2022.

Serendipitous Pathways and Elusive Definitions: Leadership in Continuing Professional Development.

Introduction: Although the Continuing Professional Development (CPD) field has rapidly expanded in scope, breadth, and depth, there is a gap in how we understand CPD leadership and the role of the leader. Previous scholarship indicates that there is neither an agreed on set of competencies for CPD leadership roles nor a distinct pathway towards those roles. This study is aimed at answering the following question: How is leadership described or defined in CPD and what are the contextual issues that are and/or should be shaping its evolution?

Methods: Conducted between 2020 and 2022, CPD leadership program learners and CPD leaders with a range of leadership expertise were identified using convenience and purposive sampling and invited to participate in this study.

Efficacy Versus Effectiveness: The HORIZON-Pivotal Fracture Trial and Its Emulation in Claims Data.

Objective: The objective of this study is to evaluate the concordance of results between the Health Outcomes and Reduced Incidence with Zoledronic Acid Once Yearly (HORIZON)-Pivotal Fracture Trial (PFT) and a nonrandomized database study designed to emulate the trial.

Methods: HORIZON-PFT evaluated the efficacy of zoledronic acid versus placebo in reducing the risk of hip fractures and found a 41% risk reduction over a three-year treatment period (hazard ratio [HR] = 0.59; 95% confidence interval [95% CI] 0.

Invited commentary: A future of data-rich pharmacoepidemiology studies- transitioning to large-scale linked EHR+claims data.

Electronic health record (EHR) data are seen as an important source for Pharmacoepidemiology studies. In the US healthcare system, EHR systems often only identify fragments of patients' health information across the care continuum, including primary care, specialist care, hospitalizations, and pharmacy dispensing. This leads to unobservable information in longitudinal evaluations of medication effects causing unmeasured confounding, misclassification, and truncated follow-up times.

Real-world evidence from Germany and the United States: Treatment initiation on low-efficacy versus high-efficacy therapies in patients with multiple sclerosis.

Background: The hit-hard-and-early (HHAE) strategy where treatment is initiated with high-efficacy therapies opposed to low-efficacy therapies presents a potential paradigm shift in multiple sclerosis (MS) management. This study aimed to assess the adoption of the HHAE strategy in Germany and the United States (US) from 2020 to 2022 based on real-world data.

Methods: The analysis was based on longitudinal, patient-level data from Germany and the US.

Enhancing External Control Arm Analyses through Data Calibration and Hybrid Designs.

Almost all external control arm analyses to contextualize findings of a single arm trial struggle with two key issues: the lack of baseline randomization, and equally important, the difference in data collection between the experimental arm with its primary data collection, and the external control arm using secondary data. We illustrate the data calibration design to remedy issues arising from differential measurements in the two arms, and discuss the hybrid design that expands an underpowered randomized internal control arm with real-world data to mitigate the lack of randomization of the external control arm. We show how the two approaches fit into an evidence-development strategy that naturally builds on the incremental insights gained.

Health Care Utilization and Costs Associated With Empagliflozin in Older Adults With Type 2 Diabetes.

Objective: We compared health care resource utilization (HCRU) and costs for inpatient and outpatient services and dispensed medications in older adults with type 2 diabetes initiating empagliflozin versus dipeptidyl peptidase 4 inhibitors (DPP-4is).

Research Design And Methods: The study population included U.S.

A simulation-based bias analysis to assess the impact of unmeasured confounding when designing nonrandomized database studies.

Unmeasured confounding is often raised as a source of potential bias during the design of nonrandomized studies, but quantifying such concerns is challenging. We developed a simulation-based approach to assess the potential impact of unmeasured confounding during the study design stage. The approach involved generation of hypothetical individual-level cohorts using realistic parameters, including a binary treatment (prevalence 25%), a time-to-event outcome (incidence 5%), 13 measured covariates, a binary unmeasured confounder (u1; 10%), and a binary measured "proxy" variable (p1) correlated with u1.

Emulation of randomized trials of direct oral anticoagulants with claims data and implications for new Factor XI inhibitors.

Direct oral anticoagulants (DOACs) revolutionized the management of thromboembolic disorders. Clinical care may be further improved as Factor XIs undergo large-scale outcome trials. What role can non-randomized database studies play in expediting understanding of these drugs in clinical practice? The RCT-DUPLICATIVE Initiative emulated the design of eight DOAC randomized clinical trials (RCT) using non-randomized claims database studies.

Validation of alopecia coding in US claims data among women of childbearing age.

Background: Accurately identifying alopecia in claims data is important to study this rare medication side effect.

Objectives: To develop and validate a claims-based algorithm to identify alopecia in women of childbearing age.

Methods: We linked electronic health records from a large healthcare system in Massachusetts (Mass General Brigham) with Medicaid claims data from 2016 through 2018 to identify all women aged 18 to 50 years with an ICD-10 code for alopecia, including alopecia areata, androgenic alopecia, non-scarring alopecia, or cicatricial alopecia, from a visit to the MGB system.

Effectiveness and safety of empagliflozin: final results from the EMPRISE study.

Aims/hypothesis: Limited evidence exists on the comparative safety and effectiveness of empagliflozin against alternative glucose-lowering medications in individuals with type 2 diabetes with the broad spectrum of cardiovascular risk. The EMPagliflozin compaRative effectIveness and SafEty (EMPRISE) cohort study was designed to monitor the safety and effectiveness of empagliflozin periodically for a period of 5 years with data collection from electronic healthcare databases.

Methods: We identified individuals ≥18 years old with type 2 diabetes who initiated empagliflozin or dipeptidyl peptidase-4 inhibitors (DPP-4i) from 2014 to 2019 using US Medicare and commercial claims databases.

Patient Characteristics Associated With Using Transcatheter Left Atrial Appendage Occlusion Versus Oral Anticoagulants for Atrial Fibrillation.

Background: Transcatheter left atrial appendage occlusion (LAAO) is an alternative to oral anticoagulants (OACs) for stroke prevention in patients with atrial fibrillation, but the predictors of LAAO use in routine care are unclear. We aimed to assess the utilization trends of LAAO and compare the change in characteristics of LAAO users versus OACs since its marketing.

Methods: Using the US Medicare claims database (March 15, 2015, to December 31, 2020), we identified patients with atrial fibrillation, ≥65 years, and CHADS-VASc score ≥2 (men) or ≥3 (women), with either first implantation of an LAAO device or initiation of OACs, including apixaban, dabigatran, rivaroxaban, edoxaban, or warfarin.

Design differences and variation in results between randomised trials and non-randomised emulations: meta-analysis of RCT-DUPLICATE data.

Objective: To explore how design emulation and population differences relate to variation in results between randomised controlled trials (RCT) and non-randomised real world evidence (RWE) studies, based on the RCT-DUPLICATE initiative (Randomised, Controlled Trials Duplicated Using Prospective Longitudinal Insurance Claims: Applying Techniques of Epidemiology).

Design: Meta-analysis of RCT-DUPLICATE data.

Data Sources: Trials included in RCT-DUPLICATE, a demonstration project that emulated 32 randomised controlled trials using three real world data sources: Optum Clinformatics Data Mart, 2004-19; IBM MarketScan, 2003-17; and subsets of Medicare parts A, B, and D, 2009-17.

Cardiorenal effectiveness of empagliflozin vs. glucagon-like peptide-1 receptor agonists: final-year results from the EMPRISE study.

Background: No randomized clinical trials have directly compared the cardiorenal effectiveness of empagliflozin and GLP-1RA agents with demonstrated cardioprotective effects in patients with a broad spectrum of cardiovascular risk. We reported the final-year results of the EMPRISE study, a monitoring program designed to evaluate the cardiorenal effectiveness of empagliflozin across broad patient subgroups.

Methods: We identified patients ≥ 18 years old with type 2 diabetes who initiated empagliflozin or GLP-1RA from 2014 to 2019 using US Medicare and commercial claims databases.