Emulations of Oncology Trials Using Real-World Data: A Systematic Literature Review.

By evaluating published emulations of oncology RCTs studies in which both the active and comparator groups are sourced from RWD and target trial results are available for benchmarking, this systematic review aims to gain insight into factors related to emulation performance. Thirteen oncology emulation studies using various types of RWD were identified through an online database search of PubMed through 2022. Based on the ROBINS-I tool, most studies (N=8) had a serious risk of overall bias driven by risk of bias from confounding.

Providing community intravenous therapy during the COVID-19 pandemic.

Community intravenous therapy is well-established in most regions of the UK. Although there are national good practice recommendations detailing the service provision of outpatient parenteral antibiotic therapy (OPAT), examples of extended service delivery of other therapies are limited. This article describes the development of a community IV therapy service through integration with acute and primary care teams.

Remdesivir Treatment in Hospitalized Patients With Coronavirus Disease 2019 (COVID-19): A Comparative Analysis of In-hospital All-cause Mortality in a Large Multicenter Observational Cohort.

Background: Remdesivir (RDV) improved clinical outcomes among hospitalized patients with coronavirus disease 2019 (COVID-19) in randomized trials, but data from clinical practice are limited.

Methods: We examined survival outcomes for US patients hospitalized with COVID-19 between August and November 2020 and treated with RDV within 2 days of hospitalization vs those not receiving RDV during their hospitalization using the Premier Healthcare Database. Preferential within-hospital propensity score matching with replacement was used.

Achieving clinical response in postpartum depression leads to improvement in health-related quality of life.

Objective: To evaluate the health-related quality of life (HRQoL) burden associated with postpartum depression (PPD), determine the extent to which clinical response impacts HRQoL, and estimate the impact of PPD and clinical response on healthcare resource utilization (HRU) and productivity.

Methods: Patient data ( = 127) from two multicenter, randomized, double-blind, placebo-controlled phase 3 clinical trials evaluating the safety and efficacy of brexanolone injection in adults with PPD were employed for these posthoc analyses. HRQoL and health utility was assessed with the SF-36-v2 Health Survey (SF-36v2) acute version.

Brexanolone in Postpartum Depression: Analyses to Help Inform Clinical Decision-Making.

Brexanolone (BRX) injection was approved by the United States Food and Drug Administration in 2019 for the treatment of adults with postpartum depression (PPD) based on two Phase 3 clinical trials. Data from the three trials were combined. PPD-specific 17-item Hamilton Rating Scale for Depression (HAMD-17) group-level minimal important difference (MID) and patient-level meaningful change (meaningful change threshold [MCT]) were estimated and applied to differences in BRX versus placebo (PBO) at hour 60 (primary endpoint) and day 30 (end of trial follow-up).

Associations between commonly used patient-reported outcome tools in postpartum depression clinical practice and the Hamilton Rating Scale for Depression.

The objective of this study is to explore the associations between the patient-reported Edinburgh Postnatal Depression Scale (EPDS) and Patient Health Questionnaire (PHQ)-9 and clinician-reported 17-item Hamilton Depression Rating Scale (HAMD-17) in order to facilitate clinical decision-making. An integrated efficacy dataset of three randomized placebo-controlled trials (NCT02614547, NCT02942004, and NCT02942017) evaluating brexanolone injection, a neuroactive steroid chemically identical to allopregnanolone, in women with postpartum depression was used for this post hoc analysis. Data were pooled across treatment arms.

What Is the Health and Well-Being Burden for Parents Living With a Child With ADHD in the United Kingdom?

To explore the burden associated with childhood ADHD in a large observational study. We recruited familes with at least one child (6-18 years) with ADHD via 15 NHS trusts in the UK, and collected data from all family members. We made careful adjustments to ensure a like-for-like comparison with two different control groups, and explored the impact of controlling for a positive parental/carer ADHD screen, employment, and relationship status.

The Cystic Fibrosis Impact Questionnaire: qualitative development and cognitive evaluation of a new patient-reported outcome instrument to assess the life impacts of cystic fibrosis.

Background: Patients with cystic fibrosis (CF) experience significant disease burden, including progressive pulmonary decline and reduced survival. This multicenter qualitative study was conducted to develop a new patient-reported outcome (PRO) measure to assess the impact of CF on patients' quality of life: the Cystic Fibrosis Impact Questionnaire (CF-IQ). Semi-structured qualitative concept elicitation (CE) interviews with patients and caregivers documented CF-related symptoms, impacts, and treatment experiences.

Cost-Effectiveness of Brexanolone Versus Selective Serotonin Reuptake Inhibitors for the Treatment of Postpartum Depression in the United States.

Background: Brexanolone injection (BRX) was approved by the FDA in 2019 for the treatment of adult patients with postpartum depression (PPD), but its cost-effectiveness has not yet been evaluated.

Objective: To estimate the cost-effectiveness of BRX compared with treatment with selective serotonin reuptake inhibitors (SSRIs) for PPD.

Methods: We projected costs (2018 U.

Using Matching-Adjusted Indirect Comparisons and Network Meta-analyses to Compare Efficacy of Brexanolone Injection with Selective Serotonin Reuptake Inhibitors for Treating Postpartum Depression.

Background: Brexanolone injection, the first therapy approved by the US FDA for the treatment of postpartum depression (PPD) in adults, has been shown to produce a significantly greater decrease in the Hamilton Rating Scale for Depression (HAM-D) total score than placebo in randomised controlled trials (RCTs) of women with PPD.

Objectives: Given the rapid effect of brexanolone injection (within 60 h) sustained throughout the length of the trials (30 days), we sought to compare its efficacy data against selective serotonin reuptake inhibitors (SSRIs), the class of antidepressants most commonly prescribed for PPD, using HAM-D and Edinburgh Postnatal Depression Scale (EPDS) outcomes from currently available RCTs.

Methods: We extracted data from 26 studies identified in a systematic literature review of pharmacological and pharmacological/nonpharmacological combination therapies in PPD.

The burden of cystic fibrosis in the Medicaid population.

Purpose: To conduct an analysis describing clinical characteristics, pulmonary exacerbation (PEx) events, and health care resource utilization among Medicaid-insured patients with cystic fibrosis (CF).

Patients And Methods: A retrospective analysis of the Truven Health MarketScan Medicaid Multi-State administrative claims database (2010-2014) was undertaken. Patients aged ≥6 years with a CF diagnosis, continuously enrolled for 12 months, were identified.

The economic consequences of attention-deficit hyperactivity disorder in the Scottish prison system.

Background: Attention-deficit hyperactivity disorder (ADHD) is highly prevalent amongst prison inmates and the criminal justice system (CJS) likely bears considerable costs for offenders with ADHD. We aimed to examine the relationship between ADHD and health-related quality of life (HRQoL) and quality-adjusted life years (QALY) amongst imprisoned adults; and to estimate the annual expenditure associated with ADHD status in prison.

Methods: An observational study was performed in 2011-2013, at Porterfield Prison, Inverness, United Kingdom (UK).

Health-related quality of life in prisoners with attention-deficit hyperactivity disorder and head injury.

Background: Attention-deficit hyperactivity disorder (ADHD) and head injury (including traumatic brain injury (TBI)) manifest in high levels across prison samples and guidance from the National Institute for Health and Care Excellence notes that people with acquired brain injury may have increased prevalence of ADHD. We aimed to examine the association of ADHD with TBI and the impact of the association upon health-related quality of life (HRQoL) and service use among imprisoned adults.

Methods: An observational study was performed in 2011-2013, at Porterfield Prison, Inverness, United Kingdom (UK).

Community intravenous therapy provision.

Many healthcare services that were once only available in acute settings are now common in the community. Intravenous (IV) therapy is increasingly available as a community service. Given the option, most patients would choose to receive their treatment in a community setting, rather than in hospital.

Frequency and costs of pulmonary exacerbations in patients with cystic fibrosis in the United States.

Background: Information is limited regarding the cost of pulmonary exacerbations (PEx) among patients with cystic fibrosis in the United States.

Methods: To examine PEx costs, medical chart data were linked to insurance claims for patients aged ≥6 years who had commercial coverage from a large US health insurer affiliated with Optum during 2008-2013. A PEx was categorized as an episode requiring newly started (1) oral antibiotics (PEx-O) or (2) intravenous (IV) antibiotics and/or inpatient stay (PEx-IV).

Preferences for Attention-Deficit/Hyperactivity Disorder (ADHD) Non-Stimulant Treatment Characteristics Among Children and Adolescents With ADHD and Their Caregivers.

Understanding patient and caregiver preferences for treatment is important for optimizing treatment decisions. Non-stimulant therapies are an alternative treatment option to stimulant therapy for attention-deficit/hyperactivity disorder (ADHD). Guanfacine extended release (GXR) and atomoxetine (ATX) are two non-stimulant medications approved in the United States for the treatment of ADHD.

The impact of ADHD on the health and well-being of ADHD children and their siblings.

Childhood attention-deficit/hyperactivity disorder (ADHD) has been associated with reduced health and well-being of patients and their families. The authors undertook a large UK survey-based observational study of the burden associated with childhood ADHD. The impact of ADHD on both the patient (N = 476) and their siblings (N = 337) on health-related quality of life (HRQoL) and happiness was quantified using multiple standard measures [e.

Estimating minimal important differences for several scales assessing function and quality of life in patients with attention-deficit/hyperactivity disorder.

Objective: Defining minimal important difference (MID) is critical to interpreting patient-reported outcomes data and treatment efficacy in clinical trials. This study estimates the MID for the Weiss Functional Impairment Rating Scale-Parent Report (WFIRS-P) and the Child Health and Illness Profile-Parent Report (CHIP-CE-PRF76) among parents of young people with attention-deficit/hyperactivity disorder (ADHD) in the UK.

Methods: Parents of children (6-12 years; n=100) and adolescents (13-17 years; n=117) with ADHD completed a socio-demographic form, the CHIP-CE-PRF76, the WFIRS-P, and the Pediatric Quality of Life scale at baseline and 4 weeks later.

A Cost-Utility Analysis of Lisdexamfetamine Versus Atomoxetine in the Treatment of Children and Adolescents with Attention-Deficit/Hyperactivity Disorder and Inadequate Response to Methylphenidate.

Background: An economic analysis from the perspective of the UK National Health Service (NHS) evaluated the cost effectiveness of lisdexamfetamine dimesylate (LDX) compared with atomoxetine in children and adolescents with attention-deficit/hyperactivity disorder who have had an inadequate response to methylphenidate.

Methods: A 1-year decision-analytic model was constructed, with the health outcomes "response", "nonresponse", and "unable to tolerate". Clinical data were taken from a head-to-head, randomized controlled trial in inadequate responders to methylphenidate.