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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. METHODS: With this in mind, we carried out a Delphi study to establish a list of relevant considerations for researchers to utilise whilst making decisions about whether and when to include CYP time in their economic evaluations. Delphi panellists were asked to propose and rate a set of possible considerations and provide additional thoughts on their ratings. Ratings were summarised using descriptive statistics, and text comments were interrogated through thematic analysis. FINDINGS: A total of 73 panellists across 16 countries completed both rounds of a two-round Delphi study. Panellists' ratings showed that, when thinking about whether to include displaced CYP time in an economic evaluation, it is very important to consider whether: (1) inclusion would be in line with specified perspective(s) (median score: 9), (2) CYP's time may already be accounted for in other parts of the evaluation (median score: 8), (3) the amount of forgone time is substantial, either in absolute or relative terms (median score: 7) and (4) inclusion of CYP's time costs would be of interest to decision-makers (median score: 7). Respondents thought that considerations such as (1) whether inclusion would be of interest to the research community (median score: 6), (2) whether CYP's time displaced by receiving treatment is 'school' or 'play' time (median score: 5), and (3) whether CYP's are old enough for their time to be considered valuable (median score: 5) are moderately important. A range of views was offered to support beliefs and ratings, many of which were underpinned by compelling normative questions.
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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. We further explored how one aspect of follow-up, its recommended duration, may be reflected in the percent of parents reporting that their children are receiving follow-up services at 5 years of age in these countries using data from an area-based cohort of very preterm births in 2011/12 (N = 3635). RESULTS: Between 2011/12 and 22, the number of countries with follow-up policies or programmes increased from 6 to 11. The policies and programmes were heterogeneous in eligibility criteria, duration and content. In countries that recommended longer follow-up, parent-reported follow-up rates at 5 years of age were higher, especially among the highest risk children, born <28 weeks' gestation or with birthweight <1000 g: between 42.1% and 70.1%, vs. <20% in most countries without recommendations. CONCLUSIONS: Large variations exist in follow-up policies and programmes for children born very preterm in Europe; differences in recommended duration translate into cross-country disparities in reported follow-up at 5 years of age.
Subject(s)
Infant, Extremely Premature , Premature Birth , Infant, Newborn , Child , Female , Humans , Infant , Follow-Up Studies , Premature Birth/epidemiology , Gestational Age , Europe/epidemiologyABSTRACT
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. We planned and carried out an international survey of health economists. We used a combination of approaches to identify potential survey respondents (the survey's sampling frame), we developed a questionnaire that sought to capture respondents' views and practice through close- and open-ended questions, we piloted the questionnaire through a series of cognitive interviews, and we e-mailed unique links to the final version of the questionnaire to 1956 individuals in the sampling frame. We analysed data using quantitative (descriptive and inferential statistics) and qualitative (thematic analysis) methods. We received 274 complete responses. Most respondents (87%) believe CYP time should be considered for inclusion in economic evaluations conducted from a societal perspective. However, they identify a number of obstacles to doing so, most prominently uncertainties around appropriate practice (e.g., when CYP's time should or should not be included in calculations), methodological gaps (e.g., what value to attach to CYP's time), and practical difficulties in measuring displaced time in CYP. Reporting on their own practice, most respondents found it challenging to consider CYP time in their studies, and stressed the need for clear guidance on when, and further research on how, to appropriately account for CYP's time in economic evaluations. We offer our views on how to move the topic forwards and make suggestions further research.
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Cost-Benefit Analysis , Child , Humans , Adolescent , UncertaintyABSTRACT
Avoiding excessive dialysis-associated volume depletion may help preserve residual kidney function (RKF). To establish whether knowledge of the estimated normally hydrated weight from bioimpedance measurements (BI-NHW) when setting the post-hemodialysis target weight (TW) might mitigate rate of loss of RKF, we undertook an open label, randomized controlled trial in incident patients receiving HD, with clinicians and patients blinded to bioimpedance readings in controls. A total of 439 patients with over 500 ml urine/day or residual GFR exceeding 3 ml/min/1.73m2 were recruited from 34 United Kingdom centers and randomized 1:1, stratified by center. Fluid assessments were made for up to 24 months using a standardized proforma in both groups, supplemented by availability of BI-NHW in the intervention group. Primary outcome was time to anuria, analyzed using competing-risk survival models adjusted for baseline characteristics, by intention to treat. Secondary outcomes included rate of RKF decline (mean urea and creatinine clearance), blood pressure and patient-reported outcomes. There were no group differences in cause-specific hazard rates of anuria (0.751; 95% confidence interval (0.459, 1.229)) or sub-distribution hazard rates (0.742 (0.453, 1.215)). RKF decline was markedly slower than anticipated, pooled linear rates in year 1: -0.178 (-0.196, -0.159)), year 2: -0.061 (-0.086, -0.036)) ml/min/1.73m2/month. Blood pressure and patient-reported outcomes did not differ by group. The mean difference agreement between TW and BI-NHW was similar for both groups, Bioimpedance: -0.04 kg; Control: -0.25 kg. Thus, use of a standardized clinical protocol for fluid assessment when setting TW is associated with excellent preservation of RKF. Hence, bioimpedance measurements are not necessary to achieve this.
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Anuria , Kidney Failure, Chronic , Humans , Dielectric Spectroscopy/methods , Renal Dialysis/adverse effects , Renal Dialysis/methods , Urea , Kidney , Kidney Failure, Chronic/diagnosis , Kidney Failure, Chronic/therapy , Randomized Controlled Trials as TopicABSTRACT
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. Key clinical inputs (overall survival (OS) and heart failure hospitalisations (HFH)) were obtained using the 3-year trial findings from the COAPT (Cardiovascular Outcomes Assessment of the MitraClip Percutaneous Therapy) RCT. We calculated incremental cost-effectiveness ratios (ICER) and report how these differ between analyses based on early (2-year) and updated (3-year) evidence. Updated trial data showed an increase in mortality in the intervention arm between two and three years follow-up that was not seen in the control arm. Deterministic and multivariate cost-effectiveness modelling yielded incremental cost effectiveness ratios ICERs of 38,123 and 31,227 /QALY. Compared to our 2-year based estimate (21,918 / QALY) these results imply an approximate 1.5-fold increase in ICER. The availability of updated survival analyses from the COAPT pivotal trial suggests previous estimates based on 2-year trial findings were over optimistic for the intervention.
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Heart Failure , Mitral Valve Insufficiency , Humans , Cost-Benefit Analysis , Mitral Valve Insufficiency/complications , Heart Failure/therapy , Outcome Assessment, Health Care , Treatment OutcomeABSTRACT
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. A mediation analysis that applied generalised structural equation modelling explored the association between potential mediators and PedsQL™ GCS scores. RESULTS: The multi-level OLS regression (fully adjusted model) revealed that birth at < 26 weeks' gestation, BPD status and experience of severe non-respiratory morbidity were associated with mean decrements in the total PedsQL™ GCS score of 0.35, 3.71 and 5.87, respectively. The mediation analysis revealed that the indirect effects of BPD and severe non-respiratory morbidity on the total PedsQL™ GCS score translated into decrements of 1.73 and 17.56, respectively, at < 26 weeks' gestation; 0.99 and 10.95, respectively, at 26-27 weeks' gestation; and 0.34 and 4.80, respectively, at 28-29 weeks' gestation (referent: birth at 30-31 weeks' gestation). CONCLUSION: The findings suggest that HRQoL is particularly impaired by extremely preterm birth and the concomitant complications of preterm birth such as BPD and severe non-respiratory morbidity.
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Bronchopulmonary Dysplasia , Premature Birth , Pregnancy , Female , Infant, Newborn , Humans , Child , Cohort Studies , Infant, Extremely Premature , Quality of Life/psychology , Bronchopulmonary Dysplasia/epidemiologyABSTRACT
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. The REducing Colonoscopies in patients without significant bowEl DiseasE (RECEDE) study aims to test urine volatile organic compound (VOC) analysis alongside FIT to improve detection of SBD and to reduce the number of colonoscopies and CTCs. METHODS AND ANALYSIS: This is a multicentre, prospective diagnostic accuracy study evaluating whether stool FIT plus urine VOC compared with stool FIT alone improves detection of SBD in patients referred for colonoscopy or CTC due to persistent lower gastrointestinal symptoms. To ensure SBD is not missed, the dual test requires a high sensitivity, set at 97% with 95% CI width of 5%. Our assumption is that to achieve this sensitivity requires 200 participants with SBD. Further assuming 19% of all participants will have SBD and 55% of all participants will return both stool and urine samples we will recruit 1915 participants. The thresholds for FIT and VOC results diagnosing SBD have been pre-set. If either FIT or VOC exceeds the respective threshold, the participant will be classed as having suspected SBD. As an exploratory analysis we will be testing different thresholds. The reference comparator will be a complete colonoscopy or CTC. Secondary outcomes will look at optimising the FIT and VOC thresholds for SBD detection. An economic evaluation, using a denovo decision analytic model, will be carried out determine the costs, benefits and overall cost-effectiveness of FIT +VOC vs FIT followed by colonoscopy. ETHICS AND DISSEMINATION: Ethical approval was obtained by Liverpool Central Research Ethics Committee (20/NW/0346). TRIAL REGISTRATION NUMBER: RECEDE is registered on Clinicaltrials.gov NCT04516785 & ISRCTN14982373. This protocol was written and published before results of the trial were available.
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Colonoscopy , State Medicine , Colonoscopy/methods , Humans , Occult Blood , Prospective Studies , Sensitivity and SpecificitySubject(s)
Child Development , Child Welfare/statistics & numerical data , Infant, Extremely Premature , Patient Discharge/statistics & numerical data , Aftercare/statistics & numerical data , Child, Preschool , Europe , Follow-Up Studies , Humans , Infant , Infant, Newborn , Premature Birth , Prevalence , Risk FactorsABSTRACT
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. The aim of this study is to compare over a 12-month time horizon, the costs of primary IP BS, IP best endovascular treatment (BET), and major limb major amputation (MLLA) to inform the BASIL-2 cost-utility analysis. METHODS: We compared procedural human resource (HR) costs and total in-hospital costs for the index admission, and over the following 12-months, in 60 consecutive patients undergoing primary IP BS (n = 20), IP BET (n = 20), or MLLA (10 transfemoral and 10 transtibial) for CLTI within the BASIL prospective cohort study. RESULTS: Procedural HR costs were greatest for BS (BS £2551; 95% confidence interval [CI], £1934-£2807 vs MLLA £1130; 95% CI, £1046-£1297 vs BET £329; 95% CI, £242-£390; P < .001, Kruskal-Wallis) due to longer procedure duration and greater staff requirement. With regard to the index admission, MLLA was the most expensive due to longer hospital stay (MLLA £13,320; 95% CI, £8986-£18,616 vs BS £8714; 95% CI, £6097-£11,973 vs BET £4813; 95% CI, £3529-£6097; P < .001, Kruskal-Wallis). The total cost of the index admission and in-hospital care over the following 12 months remained least for BET (MLLA £26,327; 95% CI, £17,653-£30,458 vs BS £20,401; 95% CI, £12,071-£23,926 vs BET £12,298; 95% CI, £6961-£15,439; P < .001, Kruskal-Wallis). CONCLUSIONS: Over a 12-month time horizon, MLLA and IP BS are more expensive than IP BET in terms of procedural HR costs and total in-hospital costs. These economic data, together with quality of life data from BASIL-2, will inform the calculation of incremental cost-effectiveness ratios for different CLTI management strategies within the BASIL-2 cost-utility analysis.
Subject(s)
Amputation, Surgical/economics , Angioplasty/economics , Chronic Limb-Threatening Ischemia/surgery , Hospital Costs/statistics & numerical data , Limb Salvage/economics , Aged , Aged, 80 and over , Amputation, Surgical/statistics & numerical data , Angioplasty/methods , Angioplasty/statistics & numerical data , Chronic Limb-Threatening Ischemia/economics , Cost-Benefit Analysis/statistics & numerical data , Female , Follow-Up Studies , Humans , Limb Salvage/methods , Limb Salvage/statistics & numerical data , Lower Extremity/blood supply , Lower Extremity/surgery , Male , Middle Aged , Operative Time , Patient Readmission/economics , Patient Readmission/statistics & numerical data , Popliteal Artery/surgery , Prospective Studies , Treatment OutcomeABSTRACT
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 < 0.001), 752 (p < 0.01) and 657 (p < 0.01) for children born at < 26, 26-27 and 28-29 weeks, respectively, in comparison to the reference group born at 30-31 weeks. A sensitivity analyses that excluded variables (BPD, any neonatal morbidity and presence of congenital anomaly) plausibly lying on the causal pathway between gestational age at birth and economic outcomes elevated incremental societal costs by 1482, 763 and 144 at < 26, 26-27 and 28-29 weeks, respectively, in comparison to the baseline model. CONCLUSION: This study provides new evidence about the main cost drivers associated with preterm birth in European countries. Evidence identified by this study can act as inputs within cost-effectiveness models for preventive or treatment interventions for preterm birth. IMPACT: What is the key message of your article? This study provides new evidence about the magnitude and drivers of economic costs associated with preterm birth in European countries. What does it add to the existing literature? Lower gestational age is associated with increased mean societal costs during mid-childhood with indirect costs representing a key driver of increased costs. What is the impact? For policy makers, this study adds to sparse evidence about the main cost drivers associated with preterm birth in European countries beyond the first 2 years of life.
Subject(s)
Premature Birth , Child , Cohort Studies , Cost-Benefit Analysis , Female , Gestational Age , Humans , Infant, Newborn , Pregnancy , Socioeconomic FactorsABSTRACT
OBJECTIVES: To describe parent-reported healthcare service use at age 5 years in children born very preterm and investigate whether perinatal and social factors and the use of very preterm follow-up services are associated with high service use. STUDY DESIGN: We used data from an area-based cohort of births at <32 weeks of gestation from 11 European countries, collected from birth records and parental questionnaires at 5 years of age. Using the published literature, we defined high use of outpatient/inpatient care (≥4 sick visits to general practitioners, pediatricians, or nurses, ≥3 emergency room visits, or ≥1 overnight hospitalization) and specialist care (≥2 different specialists or ≥3 visits). We also categorized countries as having either a high or a low rate of children using very preterm follow-up services at age 5 years. RESULTS: Overall, 43% of children had high outpatient/inpatient care use and 48% had high specialist care use during the previous year. Perinatal factors were associated with high outpatient/inpatient and specialist care use, with a more significant association with specialist services. Associations with intermediate parental educational level and unemployment were stronger for outpatient/inpatient services. Living in a country with higher rates of very preterm follow-up service use was associated with lower use of outpatient/inpatient services. CONCLUSIONS: Children born very preterm had high healthcare service use at age 5 years, with different patterns for outpatient/inpatient and specialist care by perinatal and social factors. Longer follow-up of children born very preterm may improve care coordination and help avoid undesirable health service use.
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Infant, Extremely Premature , Parents , Child , Child, Preschool , Cohort Studies , Delivery of Health Care , Female , Humans , Infant, Newborn , Pregnancy , Surveys and QuestionnairesABSTRACT
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. This pilot trial will determine the feasibility of a large-scale trial refining the risk of adverse pregnancy outcome in SGA pregnancies using biomarkers of placental function sFlt-1/PlGF, identifying and intervening in only those deemed at highest risk of stillbirth. METHODS: PLANES is a randomised controlled feasibility study of women with an SGA fetus that will be conducted at two tertiary care hospitals in the UK. Once identified on ultrasound, women will be randomised into two groups in a 3:1 ratio in favour of sFlt-1/PlGF ratio led management vs standard care. Women with an SGA fetus and a normal sFlt-1/PlGF ratio will have a repeat ultrasound and sFlt-1/PlGF ratio every 2 weeks with planned birth delayed until 40 weeks. In those women with an SGA fetus and an abnormal sFlt-1/PlGF ratio, we will offer birth from 37 weeks or sooner if there are other concerning features on ultrasound. Women assigned to standard care will have an sFlt-1/PlGF ratio taken, but the results will be concealed from the clinical team, and the woman's pregnancy will be managed as per the local NHS hospital policy. This integrated mixed method study will also involve a health economic analysis and a perspective work package exploring trial feasibility through interviews and questionnaires with participants, their partners, and clinicians. DISCUSSION: Our aim is to determine feasibility through the assessment of our ability to recruit and retain participants to the study. Results from this pilot study will inform the design of a future large randomised controlled trial that will be adequately powered for adverse pregnancy outcome. Such a study would provide the evidence needed to guide future management of the SGA fetus. TRIAL REGISTRATION: ISRCTN58254381 . Registered on 4 July 2019.
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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. METHODS: We used publicly available cost-effectiveness data and recommended methodology to perform cost-utility evaluation of onasemnogene abeparvosec versus best supportive care and nusinersen. RESULTS: Our evaluations highlight wide variations in cost and benefit estimates of nusinersen and indicate that onasemnogene abeparvosec is unlikely to represent value for money according to current standards of reimbursement. Results are discussed in the context of reimbursement decisions for orphan diseases. CONCLUSION: Commonly implemented commercial confidentiality practices combined with uncertain data obscure scrutiny and justification of past and present reimbursement decisions for orphan drugs. Future cutting edge expensive therapies will be numerous, they will entail very substantial economic strains. We conclude that there is an urgent and increasing need for the development of improved procedures that can lead to equitable, consistent, and transparent decision-making.
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Genetic Therapy/economics , Muscular Atrophy, Spinal/therapy , Biological Products/therapeutic use , Cost-Benefit Analysis , Humans , Oligonucleotides/therapeutic use , Palliative Care , Quality of Life , Recombinant Fusion Proteins/therapeutic use , Regression AnalysisABSTRACT
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'. SETTING: Sampling was not restricted to any particular setting and worldwide experts were recruited. PARTICIPANTS: Respondents were paediatric bone experts recruited through network sampling. RESULTS: Thirty-eight experts completed the survey. The health state with the largest detrimental impact (mean score (se)) on children's HRQoL was hypocalcaemic cardiomyopathy (0·47 (0·02)), followed by hypocalcaemic seizures (0·50 (0·02)) and active rickets (0·62 (0·02) in young children; 0·57 (0·02) in older children). Asymptomatic VDD had a modest but noticeable negative impact on HRQoL, attributed mostly to tiredness in both age groups and pain in the older paediatric population. CONCLUSIONS: Elicitation of HRQoL from clinical experts suggests a negative impact of VDD on HRQoL, even if there is no recognizable clinical manifestation. HRQoL data from populations of patients with MND will inform public health policy decisions. In some settings, routine collection of HRQoL data alongside national nutrition surveys may help capture the full burden of MND and prioritize resources towards effective prevention.
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Micronutrients/deficiency , Quality of Life , Vitamin D Deficiency/epidemiology , Adolescent , Cardiomyopathies/epidemiology , Child , Child, Preschool , Female , Humans , Infant , Infant, Newborn , Male , Rickets/epidemiology , Surveys and Questionnaires , Vitamin D/blood , Vitamin D Deficiency/bloodABSTRACT
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. We set out to explore how forgone time-including absence from formal education and childhood leisure time-can be estimated and incorporated into economic evaluations. To do so, we look at theories and approaches to time valuation proposed in different disciplines and we discuss their suitability for use in health economics research. We find that, while there is a sizeable literature on time valuation methods in education, labour and transportation economics, much of this is not directly applicable to economic evaluation of health care interventions for children. We identify gaps in existing methods and practice, we outline challenges in moving forwards and we provide a list of considerations aiming to assist researchers in deciding whether, and how, to include foregone time-related costs in economic evaluation.
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Education/economics , Leisure Activities/economics , Value of Life , Age Factors , Child , Costs and Cost Analysis , Humans , Time Factors , Value of Life/economicsABSTRACT
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. METHODS: RCTs of second/third line treatments in participants with advanced/metastatic NSCLC and negative/low expression of Anaplastic Lymphoma Kinase (ALK) and of Epidermal Growth Factor Receptor (EGFR) were included. We searched electronic databases (MEDLINE; EMBASE; Web of Science) from January, 2000 up to July, 2017. Two or more independent reviewers screened bibliographic records, extracted data, and assessed risk of bias of studies. Published Kaplan Meier plots for OS and PFS along with restricted-mean-survival methods and parametric modelling were used to estimate the survival outcomes as mean number of months of survival. Network meta-analysis was undertaken to rank interventions and to make indirect comparisons. RESULTS: We included 11 RCTs with data for 7581 participants that compared nine different drugs. In studies of patients regardless of histology groups, targeted drugs (ramucirumab and nintedanib) yielded small overall survival gains of < 2.5 months over docetaxel, erlotinib provided no benefit, while immunotherapies (atezolizumab and pembrolizumab) delivered 5 to 6 months gain. Studies with patients stratified by histology confirmed the apparent superiority of immunotherapy (nivolumab and atezolizumab) over targeted treatments (ramucirumab, nintedanib, afatinib) providing between about 4 to 8 months OS gain over docetaxel. In network analysis immunotherapies consistently ranked higher than alternatives irrespective of population histology and outcome measure. CONCLUSION: Our review indicates that nivolumab, pembrolizumab and atezolizumab provide superior survival benefits compared to other licensed drugs for late stage NSCLC. Patient gains from these immunotherapies are substantial compared to the expected average survival with chemotherapy (docetaxel) of < 1 year for people with squamous histology and about 1.25 year for those with non-squamous histology.
Subject(s)
Anaplastic Lymphoma Kinase/genetics , Antineoplastic Combined Chemotherapy Protocols/therapeutic use , Carcinoma, Non-Small-Cell Lung/drug therapy , Antibodies, Monoclonal/therapeutic use , Antibodies, Monoclonal, Humanized , Carcinoma, Non-Small-Cell Lung/genetics , Carcinoma, Non-Small-Cell Lung/pathology , ErbB Receptors/genetics , Erlotinib Hydrochloride/therapeutic use , Humans , Immunotherapy , Neoplasm Metastasis , Neoplasms, Second Primary/drug therapy , Neoplasms, Second Primary/genetics , Neoplasms, Second Primary/pathology , Progression-Free Survival , Protein Kinase Inhibitors/therapeutic use , Quality of Life , Randomized Controlled Trials as Topic , RamucirumabABSTRACT
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. METHODS: Eight electronic bibliographic databases (MEDLINE, MEDLINE In-Process & Other Non-Indexed Citations, EMBASE, Web of Science, NHS Economic Evaluation Database (EED), Database of Abstracts of Reviews of Effects (DARE), Health Technology Assessment (HTA) database, and EconLit) were searched for relevant economic evaluations published from each database's inception date until November 2017. Study selection, quality assessment, and data extraction were carried out according to published guidelines. RESULTS: Ten relevant economic evaluations presented in 11 papers were identified. Seven of the included studies were full economic evaluations (i.e., costs and effects assessed), and three studies were partial economic evaluations (i.e., only costs were considered in the analysis). The A/MBIs that had been subjected to economic evaluation were acceptance and commitment therapy (ACT), dialectical behaviour therapy (DBT), mindfulness-based cognitive therapy (MBCT), and mindfulness-based stress reduction (MBSR). In terms of clinical presentations, the evaluation of cost-effectiveness of A/MBIs has been more focused on depression and emotional unstable personality disorder with three and four economic evaluations, respectively. Three out of seven full economic evaluations observed that A/MBIs were cost-effective for the management of mental health conditions. Nevertheless, the heterogeneity of included populations, interventions, and economic evaluation study types limits the extent to which firm conclusions can currently be made. CONCLUSION: This first substantive review of economic evaluations of A/MBIs indicates that more research is needed before firm conclusions can be reached on the cost-effectiveness of A/MBIs for mental health conditions. PRACTITIONER POINTS: The findings of the review provide information that may be relevant to mental health service commissioners and decision-makers as all economic evidence available on acceptance and mindfulness-based interventions for mental health conditions is summarized. Evidence relating to the cost-effectiveness and cost-saving potential of acceptance and mindfulness-based interventions is focused mainly on depression and emotional unstable personality disorder to date. Heterogeneity in the specific forms of acceptance and mindfulness-based interventions may limit generalizability of the findings. The number of health economic evaluations relating to acceptance and mindfulness-based interventions remains relatively small. Further research in this area is required.
Subject(s)
Cost-Benefit Analysis/methods , Mindfulness/methods , Female , Humans , MaleABSTRACT
BACKGROUND: Ventilator-associated pneumonia is associated with significant morbidity, mortality and healthcare costs. Most of the cost data that are available relate to general intensive care patients in privately remunerated institutions. This study assessed the cost of managing ventilator-associated pneumonia in a cardiac intensive care unit in the National Health Service in the United Kingdom. METHODS: Propensity-matched study of prospectively collected data from the cardiac surgical database between April 2011 and December 2014 in all patients undergoing cardiac surgery (n = 3416). Patients who were diagnosed as developing ventilator-associated pneumonia, as per the surveillance definition for ventilator-associated pneumonia (n = 338), were propensity score matched with those who did not (n = 338). Costs of treating post-op cardiac surgery patients in intensive care and cost difference if ventilator-associated pneumonia occurred based on Healthcare Resource Group categories were assessed. Secondary outcomes included differences in morbidity, mortality and cardiac intensive care unit and in-hospital length of stay. RESULTS: There were no significant differences in the pre-operative characteristics or procedures between the groups. Ventilator-associated pneumonia developed in 10% of post-cardiac surgery patients. Post-operatively, the ventilator-associated pneumonia group required longer ventilation (p < 0.01), more respiratory support, longer cardiac intensive care unit (8 vs 3, p < 0.001) and in-hospital stay (16 vs 9) days. The overall cost for post-operative recovery after cardiac surgery for ventilator-associated pneumonia patients was £15,124 compared to £6295 for non-ventilator-associated pneumonia (p < 0.01). The additional cost of treating patients with ventilator-associated pneumonia was £8829. CONCLUSION: Ventilator-associated pneumonia was associated with significant morbidity to the patients, generating significant costs. This cost was nearer to the lower end for the cost for general intensive care unit patients in privately reimbursed systems.
ABSTRACT
BACKGROUND: Ventilator-associated pneumonia (VAP) is common and costly. In a recent randomized controlled trial, the Venner-PneuX (VPX) endotracheal tube system (Qualitech Healthcare Limited, Maidenhead, United Kingdom) was found to be superior to standard endotracheal tubes (SET) in preventing VAP. However, VPX is considerably more expensive. We evaluated the costs and benefits of VPX to determine whether replacing SET with VPX is a cost-effective option for intensive care units. METHODS: We developed a decision analytic model to compare intubation with VPX or SET for patients requiring mechanical ventilation after cardiac operations. The model was populated with existing evidence on costs, effectiveness, and quality of life. Cost-effectiveness and cost-utility analyses were conducted from a National Health Service hospital perspective. Uncertainty was assessed through deterministic and probabilistic sensitivity analyses. RESULTS: Compared with SET, VPX is associated with an expected cost saving of £738 per patient. VPX led to a small increase in quality-adjusted life years, indicating that the device is overall less costly and more effective than SET. The probability of VPX being cost-effective at £30,000 per quality-adjusted life year is 97%. VPX would cease to be cost-effective if (1) it led to a risk reduction smaller than 0.02 compared with SET, (2) the acquisition cost of VPX was as high as £890, or (3) the cost of treating a case of VAP was lower than £1,450. CONCLUSIONS: VPX resulted in improved outcomes and savings that far offset the cost of the device, suggesting that replacing SET with VPX is overall beneficial. Findings were robust to extreme values of key variables.
Subject(s)
Cardiac Surgical Procedures/methods , Cost-Benefit Analysis , Intubation, Intratracheal/instrumentation , Pneumonia, Ventilator-Associated/prevention & control , Postoperative Care/economics , Cardiac Surgical Procedures/adverse effects , Decision Support Techniques , Equipment Design , Female , Humans , Intubation, Intratracheal/economics , Intubation, Intratracheal/methods , Male , Postoperative Care/methods , Postoperative Complications/diagnosis , Postoperative Complications/therapy , Respiration, Artificial/adverse effects , Respiration, Artificial/methods , United KingdomABSTRACT
BACKGROUND: Intrathecal drug delivery (ITDD) systems are one of a limited number of management options for chronic noncancer pain, cancer pain, and spasticity. Concerns over their effectiveness and high initial costs led National Health Service (NHS) England to decommission ITDD for patients with chronic noncancer pain. However, the extent to which this decision is in line with existing economic evidence is unclear. The aim of this systematic review was to identify and review the existing evidence on the cost effectiveness of ITDD for chronic noncancer pain. METHODS: Full and partial economic evaluations on ITDD were identified through systematic searches in MEDLINE, Embase, Web of Science, and the NHS for Reviews and Dissemination databases. Database searches were complemented by hand searching of reference lists of relevant studies and searches of grey literature. Study selection was carried out by 2 assessors, independently. Study quality assessment was performed to inform critical appraisal of health economics studies. Data were extracted using a data extraction form developed for the purposes of this study. RESULTS: Four thousand four hundred and sixty-four unique studies were identified, of which 7 met the inclusion criteria. With the exception of 1 study, the studies found ITDD to be either cost saving or cost effective compared to conventional medical management. ITDD became cost ineffective in 1 further study following price year adjustment to 2016. CONCLUSIONS: Study findings showed ITDD to be not cost effective only in extremely conservative scenarios. There is limited evidence on the effectiveness of ITDD in noncancer pain; however, the available economic evidence controverts arguments to refute the treatment on economic grounds.