Cost analysis for mirabegron use in the treatment of children with neurogenic bladder.

INTRODUCTION: Mirabegron is a beta-3 adrenergic receptor agonist that received FDA approval in 2021 to treat neurogenic detrusor overactivity (NDO) in children ages three years and older. Despite its safety and efficacy, access to mirabegron frequently remains restricted by payor coverage policies. OBJECTIVE: This cost minimization study sought to determine the cost implications from a payor perspective of mirabegron use at different points in the treatment pathway for pediatric NDO. STUDY DESIGN: A Markov decision analytic model was constructed to assess the costs for eight treatment strategies over a 10-year period, using six-month cycles (Table). Five strategies involve mirabegron use as first-, second-, third-, or fourth-line therapy. Two strategies, including the "base case," entail use of anticholinergic medications followed by onabotulinum toxin type A (Botox) injection and augmentation cystoplasty. A strategy involving first-line Botox was also modeled. The effectiveness, adverse event rates, attrition rates, and costs associated with each treatment option were obtained from the clinical literature and adjusted to a six-month cycle. Costs were adjusted to 2021-dollar value. A discount rate of 3% was used. To quantify uncertainty, costs and treatment transition probabilities were modeled as gamma and PERT distributions, respectively. One-way sensitivity analyses were performed. Probabilistic sensitivity analysis (PSA) was conducted using a Monte Carlo simulation with 100,000 iterations. Analyses were performed using Treeage Pro (Healthcare Version). RESULTS: The least costly strategy involved first-line mirabegron (expected cost $37,954). All strategies involving mirabegron were less costly than the base case ($56,417). On PSA, first-line mirabegron was the least costly strategy in 88.9% of cases (mean $37,604, 95% CI: $37,579-37,628); in 100% of cases, the least costly strategy involved mirabegron use. Cost savings associated with mirabegron use were attributable to decreased use of augmentation cystoplasty and Botox injections. DISCUSSION: This is the first study to compare costs across multiple strategies involving mirabegron to treat pediatric NDO. Mirabegron use likely yields cost savings for the payor: the least costly strategy involved first-line mirabegron, and all pathways incorporating mirabegron were less costly than those without mirabegron use. These findings provide an updated cost analysis for the treatment of NDO by investigating mirabegron use alongside more established treatment options. CONCLUSION: Use of mirabegron for the treatment of pediatric NDO is likely associated with cost savings as compared to treatment pathways without mirabegron. Expansion of payor coverage for mirabegron, as well as clinical studies to study first-line mirabegron use, should be considered.

Emergency department clinical leads' experiences of implementing primary care services where GPs work in or alongside emergency departments in the UK: a qualitative study.

BACKGROUND: To manage increasing demand for emergency and unscheduled care NHS England policy has promoted services in which patients presenting to Emergency Departments (EDs) with non-urgent problems are directed to general practitioners (GPs) and other primary care clinicians working within or alongside emergency departments. However, the ways that hospitals have implemented primary care services in EDs are varied. The aim of this study was to describe ED clinical leads' experiences of implementing and delivering 'primary care services' and 'emergency medicine services' where GPs were integrated into the ED team. METHODS: We conducted interviews with ED clinical leads in England (n = 19) and Wales (n = 2). We used framework analysis to analyse interview transcripts and explore differences across 'primary care services', 'emergency medicine services' and emergency departments without primary care services. RESULTS: In EDs with separate primary care services, success was reported when having a distinct workforce of primary care clinicians, who improved waiting times and flow by seeing primary care-type patients in a timely way, using fewer investigations, and enabling ED doctors to focus on more acutely unwell patients. Some challenges were: trying to align their service with the policy guidance, inconsistent demand for primary care, accessible community primary care services, difficulties in recruiting GPs, lack of funding, difficulties in agreeing governance protocols and establishing effective streaming pathways. Where GPs were integrated into an ED workforce success was reported as managing the demand for both emergency and primary care and reducing admissions. CONCLUSIONS: Introducing a policy advocating a preferred model of service to address primary care demand was not useful for all emergency departments. To support successful and sustainable primary care services in or alongside EDs, policy makers and commissioners should consider varied ways that GPs can be employed to manage variation in local demand and also local contextual factors such as the ability to recruit and retain GPs, sustainable funding, clear governance frameworks, training, support and guidance for all staff. Whether or not streaming to a separate primary care service is useful also depended on the level of primary care demand.

Urinary incontinence in spina bifida: Initial instrument validation.

The purpose of this study was to perform a psychometric assessment of the Incontinence Symptom Index-Pediatric (ISI-P) in a cohort of adolescents with spina bifida (SB) and neuropathic urinary incontinence (UI) to test its validity and reliability. The ISI-P, an 11-item instrument with domains for symptom severity and impairment, was self-administered by subjects 11-17 years old with SB and UI. Controls were 11-17 years old, with nephrolithiasis and no history of UI. Formal psychometric assessment included an evaluation of internal consistency, test re-test reliability and factor analysis. Of 78 study-eligible subjects we attempted to contact, 33 (66.7% female) with a median age of 13.1 years completed the ISI-P (42.3% response rate). 21 control patients also completed the ISI-P. Cronbach's alpha was 0.936 and 0.792 for the severity and bother factors respectively. The delta Chi-square test for the two-factor (vs. one-factor) model was significantly [χ(2)(89) = 107.823, p < 0.05] in favor of the former model with descriptive fit indices being excellent (e.g., comparative fit index = 0.969). Furthermore, category information analysis showed that all categories were associated with different threshold values, namely that each category contributed unique information for the measurement of the latent trait. In conclusion, the ISI-P has desirable psychometric properties for the measurement of UI symptom severity and impairment in adolescents with SB.