Caregiver Global Impression Observations from EMBARK: A Phase 3 Study Evaluating Delandistrogene Moxeparvovec in Ambulatory Patients with Duchenne Muscular Dystrophy.

Introduction: Duchenne muscular dystrophy (DMD) is a rare, progressive, debilitating neuromuscular disease. The early childhood onset and debilitating nature of the disease necessitate decades of caretaking for most patients. Caregivers have a critical role in evaluating patients' physical functioning and/or response to treatment.

Dispensing pharmacy chains and direct anticoagulants: Potential associations with patient outcomes.

Purpose: Pharmacy chains can differ with respect to the characteristics of their patient populations as well as their nonprescription products, services, and practices, and thus may serve as a surrogate for potential unmeasured confounding in observational studies of prescription drugs. This study evaluates whether a single-source drug can have different patient outcomes based on the dispensing pharmacy chain.

Methods: Separate analyses for two anticoagulant drugs, rivaroxaban and apixaban, were conducted using Medicare Fee-for-Service claims evaluating the association between dispensing pharmacy chain and outcomes of acute myocardial infarction, ischemic stroke, intracranial hemorrhage, gastrointestinal (GI) bleeding, all-cause mortality, and major GI bleeding.

Incorporating Prior Data in Quantitative Benefit-Risk Assessments: Case Study of a Bayesian Method.

Background: Multiple criteria decision analysis (MCDA) and stochastic multi-criteria acceptability analysis (SMAA) in their current implementation cannot incorporate prior or external information on benefits and risks. We demonstrate how to incorporate prior data using a Bayesian mixture model approach while conducting quantitative benefit-risk assessments (qBRA) for medical products.

Methods: We implemented MCDA and SMAA in a Bayesian framework.

Scalable Incident Detection via Natural Language Processing and Probabilistic Language Models.

Post marketing safety surveillance depends in part on the ability to detect concerning clinical events at scale. Spontaneous reporting might be an effective component of safety surveillance, but it requires awareness and understanding among healthcare professionals to achieve its potential. Reliance on readily available structured data such as diagnostic codes risk under-coding and imprecision.

Evaluation of association of anti-PEG antibodies with anaphylaxis after mRNA COVID-19 vaccination.

Background: The mechanism for anaphylaxis following mRNA COVID-19 vaccination has been widely debated; understanding this serious adverse event is important for future vaccines of similar design. A mechanism proposed is type I hypersensitivity (i.e.

Early antibody responses associated with survival in COVID19 patients.

Neutralizing antibodies to the SARS CoV-2 spike proteins have been issued Emergency Use Authorizations and are a likely mechanism of vaccines to prevent COVID-19. However, benefit of treatment with monoclonal antibodies has only been observed in clinical trials in outpatients with mild to moderate COVID-19 but not in patients who are hospitalized and/or have advanced disease. To address this observation, we evaluated the timing of anti SARS-CoV-2 antibody production in hospitalized patients with the use of a highly sensitive multiplexed bead-based immunoassay allowing for early detection of antibodies to SARS-CoV-2.

Electronic phenotyping of health outcomes of interest using a linked claims-electronic health record database: Findings from a machine learning pilot project.

Objective: Claims-based algorithms are used in the Food and Drug Administration Sentinel Active Risk Identification and Analysis System to identify occurrences of health outcomes of interest (HOIs) for medical product safety assessment. This project aimed to apply machine learning classification techniques to demonstrate the feasibility of developing a claims-based algorithm to predict an HOI in structured electronic health record (EHR) data.

Materials And Methods: We used the 2015-2019 IBM MarketScan Explorys Claims-EMR Data Set, linking administrative claims and EHR data at the patient level.

Weighted estimators of the complier average causal effect on restricted mean survival time with observed instrument-outcome confounders.

A major concern in any observational study is unmeasured confounding of the relationship between a treatment and outcome of interest. Instrumental variable (IV) analysis methods are able to control for unmeasured confounding. However, IV analysis methods developed for censored time-to-event data tend to rely on assumptions that may not be reasonable in many practical applications, making them unsuitable for use in observational studies.

Sample size estimation for case-crossover studies.

Case-crossover study designs are observational studies used to assess postmarket safety of medical products (eg, vaccines or drugs). As a case-crossover study is self-controlled, its advantages include better control for confounding because the design controls for any time-invariant measured and unmeasured confounding and potentially greater feasibility as only data from those experiencing an event (or cases) are required. However, self-matching also introduces correlation between case and control periods within a subject or matched unit.

Evaluating center performance in the competing risks setting: Application to outcomes of wait-listed end-stage renal disease patients.

It is often of interest to compare centers or healthcare providers on quality of care delivered. We consider the setting where evaluation of center performance on multiple competing events is of interest. We propose estimating center effects through cause-specific proportional hazards frailty models that allow correlation among a center's cause-specific effects.

State-Level Awareness of Chronic Kidney Disease in the U.S.

Introduction: This study examined state-level variation in chronic kidney disease (CKD) awareness using national estimates of disease awareness among adults in the U.S. with CKD.

Food Insecurity, CKD, and Subsequent ESRD in US Adults.

Background: Poor access to food among low-income adults has been recognized as a risk factor for chronic kidney disease (CKD), but there are no data for the impact of food insecurity on progression to end-stage renal disease (ESRD). We hypothesized that food insecurity would be independently associated with risk for ESRD among persons with and without earlier stages of CKD.

Study Design: Longitudinal cohort study.

Measuring pharmacy performance in the area of medication adherence: addressing the issue of risk adjustment.

Background: Pharmacies and pharmacists play an important role in the health care system, improving health outcomes and enhancing quality through better pharmaceutical care. Yet, little information is available to accurately evaluate pharmacy store quality and thereby encourage quality improvement at the pharmacy store level.

Objectives: To (a) assess pharmacy performance in the area of medication adherence and (b) examine the impact of risk adjustment of performance scores on pharmacy rankings.