Economic Considerations in Designs and Modifications of Multiarm, Multistage Adaptive and Adaptive Platform Randomized Controlled Trials: A Systematic Literature Review.

Objectives: There is uncertainty around whether, and under what circumstances, there is value in embedding economic considerations into multiarm, multistage (MAMS) adaptive, and adaptive platform trial designs. This systematic review was conducted to assess the analytical methods and factors that are considered when incorporating health economic analyses when designing and modifying MAMS adaptive and adaptive platform trials.

Methods: The review searched for health economic analyses, including planned analyses, of interventions assessed through MAMS adaptive, and adaptive platform trials.

Cardiac prehabilitation, rehabilitation and education in first-time atrial fibrillation (AF) ablation (CREED AF): Study protocol for a randomised controlled trial.

Background: Atrial fibrillation (AF) is associated with significant morbidity/mortality. AF-ablation is an increasingly used treatment. Currently, first-time AF-ablation success is 40-80% at 1-year, depending on individual factors.

Health-related quality of life at 5 years of age for children born very preterm with congenital anomalies: a multi-national cohort study.

Background: This study aimed to investigate the health-related quality of life (HRQoL) at 5 years of age of European children born very preterm across multi-dimensional outcomes by presence and severity of congenital anomalies.

Methods: The study used data from a European cohort of children born very preterm (<32 weeks of gestation) and followed up to 5 years of age (N = 3493). Multilevel Ordinary Least Squares (OLS) regression were used to explore the associations between the presence and severity of congenital anomalies.

Considerations Around the Inclusion of Children and Young People's Time in Economic Evaluation: Findings from an International Delphi Study.

Background: People's time is a finite resource and a valuable input that ought to be considered in economic evaluations taking a broad, societal perspective. Yet, evaluations of interventions focusing on children and young people (CYP) rarely account for the opportunity cost of time in this population. As a key reason for this, health economists have pointed to uncertainty around when it is appropriate to include CYP time-related costs in an economic evaluation and highlighted the lack of clear guidance on the topic.

Impact of colonoscopy on health-related quality of life: findings from the RECEDE study.

Background: Colonoscopy is a valuable diagnostic tool but the procedure and the preparation for it cause anxiety and discomfort that impacts on patients' health-related quality of life (HRQoL). The 'disutility' of undergoing an invasive colonoscopy needs to be considered and accounted for in comprehensive cost-utility analyses that compare different diagnostic strategies, yet there is little empirical evidence that can be used in such studies. To fill this gap, we collected and analysed data on the effect of a colonoscopy examination on patients' HRQoL that can be used in economic evaluations.

Variation in follow-up for children born very preterm in Europe.

Background: Children born very preterm (<32 weeks of gestation) face high risks of neurodevelopmental and health difficulties compared with children born at term. Follow-up after discharge from the neonatal intensive care unit is essential to ensure early detection and intervention, but data on policy approaches are sparse.

Methods: We investigated the characteristics of follow-up policy and programmes in 11 European countries from 2011 to 2022 using healthcare informant questionnaires and the published/grey literature.

Views, obstacles, and uncertainties around the inclusion of children and young people's time in economic evaluations: Findings from an international survey of health economists.

People's time is a limited resource and, in economic evaluations that adopt a societal perspective, it is important that it is valued and accounted for. Yet, in economic evaluations of interventions for children and young people (CYP), attempts to take into account the opportunity cost of their time are rare. To understand why this is the case, we need to first understand what views health economists hold in relation to CYP time, and what challenges they face in incorporating this in their evaluations.

Impact of updated trial data on the cost-effectiveness of percutaneous mitral repair.

When updated clinical trial data becomes available reassessing the cost-effectiveness of technologies may modify estimates and influence decision-making. We investigated the impact of updated trial outcomes on the cost-effectiveness of percutaneous mitral repair (PR) for secondary mitral regurgitation. We updated our previous three-state time-varying Markov model to assess the cost-effectiveness of PR + guideline directed medical treatment (GDMT) versus GDMT alone.

Health-related quality of life of children born very preterm: a multinational European cohort study.

Purpose: This study aims to (1) describe the health-related quality of life (HRQoL) outcomes experienced by children born very preterm (28-31 weeks' gestation) and extremely preterm (< 28 weeks' gestation) at five years of age and (2) explore the mediation effects of bronchopulmonary dysplasia (BPD) and severe non-respiratory neonatal morbidity on those outcomes.

Methods: This investigation was based on data for 3687 children born at < 32 weeks' gestation that contributed to the EPICE and SHIPS studies conducted in 19 regions across 11 European countries. Descriptive statistics and multi-level ordinary linear squares (OLS) regression were used to explore the association between perinatal and sociodemographic characteristics and PedsQL GCS scores.

REducing Colonoscopies in patients without significant bowEl DiseasE: the RECEDE Study - protocol for a prospective diagnostic accuracy study.

Introduction: Demand for colonoscopies and CT colonography (CTC) is exceeding capacity in National Health Service Trusts. In many patients colonoscopies and CTCs show no significant bowel disease (SBD). Faecal Immunochemical Testing (FIT) is being introduced to prioritise patients for colonoscopies but is insufficient to identify non-SBD patients meaning colonoscopy and CTC demand remains high.

Economic costs at age five associated with very preterm birth: multinational European cohort study.

Background: This study aims to estimate the economic costs of care provided to children born very preterm and extremely preterm across 11 European countries, and to understand what perinatal and socioeconomic factors contribute to higher costs.

Methods: Generalised linear modelling was used to explore the association between perinatal and sociodemographic characteristics and total economic costs (€, 2016 prices) during the fifth year of life.

Results: Lower gestational age was associated with increased mean societal costs of €2755 (p < 0.

Procedural and 12-month in-hospital costs of primary infra-popliteal bypass surgery, infrapopliteal best endovascular treatment, and major lower limb amputation for chronic limb threatening ischemia.

Objective: Chronic limb-threatening ischemia (CLTI) is a growing global problem due to the widespread use of tobacco and increasing prevalence of diabetes. Although the financial consequences are considerable, few studies have compared the relative cost-effectiveness of different CLTI management strategies. The Bypass vs Angioplasty in Severe Ischaemia of the Leg (BASIL)-2 trial is randomizing patients with CLTI to primary infrapopliteal (IP) vein bypass surgery (BS) or best endovascular treatment (BET) and includes a comprehensive within-trial cost-utility analysis.

Study protocol randomised controlled trial comparison of cost-utility and cost-effectiveness of a face-to-face rehabilitation programme versus a telemedicine programme in the treatment of patients with chronic low back pain.

Introduction: Chronic lower back pain is a highly prevalent medical condition in Western countries, which that incurs a considerable social and economic burden. Although prescription exercise at home for chronic pain has become a widely used alternative to reduce healthcare costs, the evidence regarding patient adherence and decreased in costs in European countries is scarce and inconclusive. The objective of this study is to examine the cost-utility and cost-effectiveness in patients with chronic lower back pain treated with the McKenzie Method and electroanalgesia via a telemedicine programme versus a face-to-face programme.

The PLANES study: a protocol for a randomised controlled feasibility study of the placental growth factor (PlGF) blood test-informed care versus standard care alone for women with a small for gestational age fetus at or after 32 + 0 weeks' gestation.

Background: Stillbirth remains a major concern across the globe and in some high-resource countries, such as the UK; efforts to reduce the rate have achieved only modest reductions. One third of stillborn babies are small for gestational age (SGA), and these pregnancies are also at risk of neonatal adverse outcomes and lifelong health problems, especially when delivered preterm. Current UK clinical guidance advocates regular monitoring and early term delivery of the SGA fetus; however, the most appropriate regimen for surveillance of these babies remains unclear and often leads to increased intervention for a large number of these women.

Will the US$5 million onasemnogene abeparvosec treatment for spinal muscular atrophy represent 'value for money' for the NHS? A rapid inquiry into suggestions that it may be cost-effective.

Objectives: Nusinersen (Spinraza®, Biogen) and onasemnogene abeparvosec (Zolgensma®, Novartis) are novel gene-based therapies for the orphan disease Spinal Muscular Atrophy. Onasemnogene abeparvosec has been allocated an acquisition cost of up to US$5 million per patient. We undertook a rapid inquiry to evaluate if onasemnogene abeparvosec is likely to be cost-effective for the UK NHS.

The economic case for prevention of population vitamin D deficiency: a modelling study using data from England and Wales.

Background: Vitamin D deficiency (VDD) affects the health and wellbeing of millions worldwide. In high latitude countries such as the United Kingdom (UK), severe complications disproportionally affect ethnic minority groups.

Objective: To develop a decision-analytic model to estimate the cost effectiveness of population strategies to prevent VDD.

Measuring, valuing and including forgone childhood education and leisure time costs in economic evaluation: Methods, challenges and the way forward.

Economic evaluations carried out to inform the allocation of finite public funds ought to take into account all relevant costs and benefits. When such evaluations adopt a societal perspective, it is important that they include 'time-related' costs arising from productivity and leisure time losses due to receipt of care, ill health or both. For programmes that relate to children, similar costs arise from forgone time, though there is a distinct lack of insights into how such costs should be identified, measured and valued.

Three wound-dressing strategies to reduce surgical site infection after abdominal surgery: the Bluebelle feasibility study and pilot RCT.

Background: Surgical site infection (SSI) affects up to 20% of people with a primary closed wound after surgery. Wound dressings may reduce SSI.

Objective: To assess the feasibility of a multicentre randomised controlled trial (RCT) to evaluate the effectiveness and cost-effectiveness of dressing types or no dressing to reduce SSI in primary surgical wounds.

Comparative survival benefit of currently licensed second or third line treatments for epidermal growth factor receptor (EGFR) and anaplastic lymphoma kinase (ALK) negative advanced or metastatic non-small cell lung cancer: a systematic review and secondary analysis of trials.

Background: A review of therapies for advanced cancers licenced by the EMA between 2009 and 2013 concluded that for more than half of these drugs there was little evidence of overall survival or quality of life benefit. Recent years have witnessed a growing number of licensed second-line pharmacotherapies for advanced/metastatic non-small cell lung cancer (NSCLC). With the aim of gauging patient survival benefit, we conducted a systematic review of randomised controlled trials (RCT) and compared survival outcomes from available licensed treatments for patients with advanced/metastatic NSCLC.

Micronutrient deficiencies and health-related quality of life: the case of children with vitamin D deficiency.

Objective: To explore the extent to which micronutrient deficiencies (MND) affect children's health-related quality of life (HRQoL), using vitamin D deficiency (VDD) as a case study.

Design: Proxy valuation study to estimate the impact of VDD on the HRQoL of younger (0-4 years) and older (>4 years) children. We used the Child Health Utility 9 Dimension (CHU9D) questionnaire to estimate HRQoL for children within six VDD-related health states: 'hypocalcaemic cardiomyopathy', 'hypocalcaemic seizures', 'active rickets', 'bone deformities', 'pain and muscle weakness' and 'subclinical VDD'.

Are acceptance and mindfulness-based interventions 'value for money'? Evidence from a systematic literature review.

Objectives: Acceptance and mindfulness-based interventions (A/MBIs) are recommended for people with mental health conditions. Although there is a growing evidence base supporting the effectiveness of different A/MBIs for mental health conditions, the economic case for these interventions has not been fully explored. The aim of this systematic review was to identify and appraise all available economic evidence of A/MBIs for the management of mental health conditions.