Total cost of care of Medicare Advantage beneficiaries participating in an appointment-based model in a national pharmacy chain.

Background: The appointment-based model (ABM) is a pharmacy service to improve medication-related health outcomes. ABM involves medication synchronization and medication review, plus other services such as medication reconciliation, medication therapy management, vaccine administration, and multimedication packaging. ABM can improve medication adherence, but the economic impact is unknown.

Assessment of algorithms for identifying patients with triple-class refractory multiple myeloma using real-world data.

Patients with triple-class refractory (TCR) multiple myeloma (MM) have limited treatment options and poor prognoses. This high unmet need has prompted the development of new therapies allowing for improved outcomes for these patients. Recently, new targeted therapies for the treatment of patients with relapsed or refractory MM have been approved based on single-arm clinical trial results.

Effectiveness of BNT162b2 BA.4/5 Bivalent COVID-19 Vaccine against Long COVID Symptoms: A US Nationwide Study.

Background: Long COVID has become a central public health concern. This study characterized the effectiveness of BNT162b2 BA.4/5 bivalent COVID-19 vaccine (bivalent) against long COVID symptoms.

Appropriateness of conducting and reporting random-effects meta-analysis in oncology.

A random-effects model is often applied in meta-analysis when considerable heterogeneity among studies is observed due to the differences in patient characteristics, timeframe, treatment regimens, and other study characteristics. Since 2014, the journals Research Synthesis Methods and the Annals of Internal Medicine have published a few noteworthy papers that explained why the most widely used method for pooling heterogeneous studies-the DerSimonian-Laird (DL) estimator-can produce biased estimates with falsely high precision and recommended to use other several alternative methods. Nevertheless, more than half of studies (55.

Regulatory agilities impacting review timelines for Pfizer/BioNTech's BNT162b2 mRNA COVID-19 vaccine: a retrospective study.

The appropriate use of regulatory agilities has the potential to accelerate regulatory review, utilize resources more efficiently and deliver medicines and vaccines more rapidly, all without compromising quality, safety and efficacy. This was clearly demonstrated during the COVID-19 pandemic where regulators and industry rapidly adapted to ensure continued supply of existing critical medicines and review and approve new innovative medicines. In this retrospective study, we analyze the impact of regulatory agilities on the review and approval of Pfizer/BioNTech's BNT162b2 mRNA COVID-19 Vaccine globally using regulatory approval data from 73 country/regional approvals.

Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints in Cancer Clinical Trials-Innovative Medicines Initiative (SISAQOL-IMI): stakeholder views, objectives, and procedures.

Patient-reported outcomes (PROs), such as symptoms, functioning, and other health-related quality-of-life concepts are gaining a more prominent role in the benefit-risk assessment of cancer therapies. However, varying ways of analysing, presenting, and interpreting PRO data could lead to erroneous and inconsistent decisions on the part of stakeholders, adversely affecting patient care and outcomes. The Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints in Cancer Clinical Trials-Innovative Medicines Initiative (SISAQOL-IMI) Consortium builds on the existing SISAQOL work to establish recommendations on design, analysis, presentation, and interpretation for PRO data in cancer clinical trials, with an expanded set of topics, including more in-depth recommendations for randomised controlled trials and single-arm studies, and for defining clinically meaningful change.

Comparing g-computation, propensity score-based weighting, and targeted maximum likelihood estimation for analyzing externally controlled trials with both measured and unmeasured confounders: a simulation study.

Objectives: To have confidence in one's interpretation of treatment effects assessed by comparing trial results to external controls, minimizing bias is a critical step. We sought to investigate different methods for causal inference in simulated data sets with measured and unmeasured confounders.

Methods: The simulated data included three types of outcomes (continuous, binary, and time-to-event), treatment assignment, two measured baseline confounders, and one unmeasured confounding factor.

Study protocol of Phase 2 open-label multicenter randomized controlled trial for granulocyte-colony stimulating factor (GCSF) in post-Kasai Type 3 biliary atresia.

Animal studies support RCT findings of improved liver function and short-term benefits using repurposed Granulocyte Colonic Stimulating Factor GCSF in adults with decompensated cirrhosis. We describe the protocol for phase 2 RCT of sequential Kasai-GCSF under an FDA-approved IND to test that GCSF improves early bile flow and post-Kasai biliary atresia BA clinical outcome. Immediate post-Kasai neonates, age 15-180 days, with biopsy-confirmed type 3 BA, without access to early liver transplantation, will be randomized 1:1 to standard of care SOC + GCSF at 10 ug/kg in 3 daily doses within 4 days of Kasai vs SOC + NO-GCSF (ClinicalTrials.

Evaluation of Intraoperative, Local Site Injections of Liposomal Bupivacaine as an Alternative to Standard Local Anesthetics in Patients Undergoing Total Hip Arthroplasty.

Achieving postsurgical pain control after total hip arthroplasty (THA) is a critical factor for successful recovery because inadequately treated pain may lead to a delay in ambulation and hospital discharge and have an adverse impact on a patient's quality of life. This study compares the effectiveness of immediate-release local anesthetics for pain control in THA vs liposomal bupivacaine (LB) related to patient outcomes and costs of care. This is a retrospective cohort study of consecutive patients undergoing THA at 3 hospitals from January 2013 to July 2016.

Hereditary, socio-behavioural, and immuno-hormonal predictors of incident rheumatoid arthritis and therapy response influences on survival versus matched control subjects using a generalised structural equation model.

Objectives: Incident onset and survival outcomes involve multiple risk factors and complex interactions preferably investigated in a single study. A generalized structural equation model (GSEM) was used to build an integrative framework to analyse multiple risk factors for incident rheumatoid arthritis (RA) and factors affecting long-term survival outcome.

Methods: Incident RA cases (n=54) had onsets between 1977 and 1994, after cohort entry in 1974.

Impact of liposomal bupivacaine on opioid use, hospital length of stay, discharge status, and hospitalization costs in patients undergoing total hip arthroplasty.

Effective postsurgical analgesia hastens recovery, reduces hospital length of stay (LOS), and decreases hospitalization costs for total hip arthroplasty (THA). Improving these outcomes is critical for value-based surgical bundled payment programs such as the Medicare Comprehensive Care for Joint Replacement and similar programs for commercial insurance providers. This study compared clinical outcomes and hospitalization costs for patients undergoing THA with and without liposomal bupivacaine (LB).

Association Between Exposure of Ipratropium and Salmeterol and Diagnosis of Multiple Sclerosis: A Matched Case-control Study.

Purpose: Ipratropium and salmeterol were found to stimulate oligodendrocyte differentiation in a high-throughput drug screening assay; thus, they may play a role in the risk reduction of multiple sclerosis (MS). So far, they have not been examined in any clinical data. This study aims at investigating the association between ipratropium and salmeterol and reduced diagnosis of MS with the use of real-world clinical data.

Economic burden and treatment patterns for patients with diffuse large B-cell lymphoma and follicular lymphoma in the USA.

Aim: Diffuse large B-cell lymphoma (DLBCL) and follicular lymphoma (FL) are common types of non-Hodgkin's lymphoma, and real-world evidence continues to be lacking for healthcare costs and utilization among DLBCL and FL patients. Our study aims to describe medical and pharmacy costs and health resource utilization and to characterize longitudinal treatment patterns among these patients.

Methods: A retrospective observational study was performed among adult patients with DLBCL or FL using the US MarketScan (Truven) administrative claims data from 1 January 2007 to 31 December 2015.

Uncomplicated Urinary Tract Infection in Ambulatory Primary Care Pediatrics: Are We Using Antibiotics Appropriately?

Objectives: Antimicrobial stewardship programs target antimicrobial use within the inpatient care setting. However, most antimicrobials are prescribed at ambulatory sites. We aim to determine the appropriateness of the diagnosis and treatment of uncomplicated urinary tract infection (UTI) in children within the outpatient setting at our institution, and to evaluate the cost of antibiotic treatment in our patient cohort.

Long-term effectiveness of a gambling intervention program among children in central Illinois.

Youth gambling is an increasing concern. As a response, the "Don't Gamble Away our Future (DGAOF)" program has been implemented among children in central Illinois. We aim to assess the long-term effectiveness of this school-based youth gambling prevention program in Illinois using the data from 2005 to 2009.

Universal Index for Cirrhosis (UIC index): The development and validation of a novel index to predict advanced liver disease.

Aim: The purpose of this study was to create and validate a novel serological diagnostic index to predict cirrhosis of all etiologies.

Methods: This was a retrospective observational study of 771 patients, age >18 years, who underwent a liver biopsy. The stage of fibrosis and routine laboratory values were recorded.

Impact of treatment with liposomal bupivacaine on hospital costs, length of stay, and discharge status in patients undergoing total knee arthroplasty at high-use institutions.

Post-surgical pain experienced by patients undergoing total knee arthroplasty (TKA) can be severe. Enhanced recovery after surgery programs incorporating multimodal analgesic regimens have evolved in an attempt to improve patient care while lowering overall costs. This study examined clinical and economic outcomes in hospitals using liposomal bupivacaine (LB) for pain control following TKA.

Liver function tests in identifying patients with liver disease.

Background And Aims: Many patients with liver disease come to medical attention once they have advanced cirrhosis or acute decompensation. Most often, patients are screened for liver disease via liver function tests (LFTs). There is very limited published data evaluating laboratory values with biopsy-proven stages of hepatic fibrosis.

Treatment patterns and health care resource utilization among patients with relapsed/refractory systemic light chain amyloidosis.

Background: Treatment for patients with systemic light chain (AL) amyloidosis remains challenging. Our study aims to describe treatment patterns for both newly diagnosed and relapsed/refractory AL (RRAL) amyloidosis, and to assess clinical outcomes, healthcare costs, and resource utilization during the first year following a diagnosis of RRAL amyloidsis.

Methods: This was a retrospective observational study of adult patients with AL amyloidosis using the US Optum administrative claims data during 1/1/2008 to 6/30/2015.