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Background: Natalizumab (Tysabri) is a new disease-modifying therapy that has been shown to be clinically effective in patients with relapsing-remitting multiple sclerosis (RRMS) and has been licensed for use in patients with highly active RRMS (HARRMS). These patients are those who experience higher relapse rates and faster disability progression than the general RRMS population.Objectives: To estimate the cost effectiveness of natalizumab compared with interferon-beta, glatiramer acetate and best supportive care from various UK cost perspectives.Methods: A 30-year Markov model was developed, based on previously published models for multiple sclerosis, to estimate transition between disability states and the probability of relapse within disability states. The model was parameterized with data from the UK Multiple Sclerosis (MS) Survey 2005 and data from the AFFIRM study, a 2-year multicentre, randomized, double-blind, placebo-controlled trial of natalizumab in RRMS patients. Additional data were sourced from the literature. A UK societal cost perspective was used in the base case, with additional cost perspectives considered in the sensitivity analysis. The baseline characteristics for the patient group were taken from the patient population in the AFFIRM study (mean age 36 years, mean time since diagnosis 5 years and a mean Kurtzke Extended Disability Status Scale [EDSS] score of 2.5). The model and its parameterization were designed and developed to support a reimbursement application for natalizumab submitted to the UK National Institute for Health and Clinical Excellence (NICE). Efficacies for natalizumab and glatiramer acetate were taken from clinical trial data, and for interferon-beta from a meta-analysis of clinical trial data. Disutilities from adverse events for each comparator were also included in the model. Outcomes and costs were discounted at 3.5% per anum. Costs for interferon-beta and glatiramer acetate were based on published prices (year 2006 values) under the UK Risk Sharing Scheme, and for natalizumab the UK NHS list price was used. Diagnostic, administration and adverse event costs were also included. The incremental cost-effectiveness ratios (ICERs) were calculated for the base case, and a probabilistic sensitivity analysis was performed to assess the probability of cost effectiveness at different willingness-to-pay thresholds.Results: The ICER for natalizumab compared with interferon-beta was 2300 pound per QALY. Compared with glatiramer acetate, it was 2000 pound per QALY, and compared with best supportive care it was 8200 pound per QALY. From a health and social care cost perspective, the ICERs were 18,700 pound, 20,400 pound and 25,500 per QALY, respectively. At a willingness-to-pay threshold of 30,000 pound per QALY, the probability of natalizumab being cost effective against any comparator from a societal perspective was >89%.Conclusion: If UK society is willing to pay more than 8200 pound per QALY, or Health and Social Services are willing to pay more than 26,000 pound per QALY, this analysis suggests that natalizumab is likely to be a cost-effective treatment for all patients with HARRMS. [ABSTRACT FROM AUTHOR]

Introduction and aims Assessment of chest radiographs is a fundamental clinical skill, often taught opportunistically. Medical students are taught how to read adult chest radiographs, however, in our experience, there is often a lack of structured training for the interpretation of pediatric chest radiographs. Our aim was to develop and evaluate an online approach for medical students to learn this skill.  Materials and methods Ericsson's expertise acquisition theory was used to develop 10 sets of 10 practice radiographs which were graded using the X-ray difficulty score. Medical student volunteers (from Keele University School of Medicine) were recruited in the paediatric rotation of their first clinical year. Pre- and post-training tests of identical difficulty were offered. A semistructured focus group was conducted after the tests, the transcription of which was analyzed using grounded theory. Results Of 117 students in the year, 54 (46%) originally volunteered. The engagement was initially high but fell during the year, particularly during the pre-examination block. The high drop-out rate made the quantitative measurement of effectiveness difficult. The focus group suggested that pressure of other work, exam preparation, technical factors, and inflexibility of the study protocol reduced engagement. Conclusions Although the topic covered was seen as important and relevant to exams, the current system requires development to make it more effective and engaging., Competing Interests: The authors have declared that no competing interests exist., (Copyright © 2021, Rajendran et al.)

Background: Protracted bacterial bronchitis (PBB) is a chronic endobrochial infection and a leading cause of chronic wet cough in children. There is an urgent need for a randomised controlled trial to investigate the optimal treatment but there is no core outcome set (COS) to inform choice of outcomes. A COS is a standardised set of outcomes representing the minimum that should be measured and reported in clinical trials of a specific condition. We have developed a COS for PBB., Methods: Potential core outcomes were collated from a systematic review, interviews with parents and a clinician survey. A two-round Delphi survey of healthcare professionals identified which outcomes had consensus for inclusion. The final COS was agreed at a consensus meeting of parent representatives and clinicians., Results: 20 outcomes were identified for the Delphi survey. After two rounds, 10 reached consensus. These were combined and edited at the consensus meeting into the final six: 1) Resolution of cough assessed using a cough score/diary recorded daily by parent(s) during treatment; 2) relapse of chronic wet cough and/or cumulative antibiotic treatment during ≥12 months follow-up; 3) change in child's quality of life (parent-proxy reporting for young children); 4) emergence of antibiotic resistance; 5) development of bronchiectasis diagnosed on clinically indicated computed tomography scans; and 6) microbiological clearance of identified respiratory pathogen if samples readily available., Conclusions: We have developed a COS for PBB which will reduce the outcome heterogeneity and bias of future clinical trials, as well as promoting comparison between studies., Competing Interests: Conflict of interest: F.J. Gilchrist has nothing to disclose. Conflict of interest: I. Ali has nothing to disclose. Conflict of interest: M. Brodlie is supported by a Medical Research Council Clinician Scientist Fellowship (MR/M008797/1). Conflict of interest: W.D. Carroll has nothing to disclose. Conflict of interest: B. Kemball has nothing to disclose. Conflict of interest: J. Walker has nothing to disclose. Conflict of interest: I. Sinha has nothing to disclose., (Copyright ©ERS 2020.)