Willingness to pay for colorectal cancer screening: a comparison of elicitation formats

Willingness to pay is increasingly being used in health technology assessment, although a number of methodological issues remain unresolved. Using data collected from four studies, this thesis presents the findings from a direct comparison between alternative format designs to elicit willingness to pay for two alternative colorectal cancer screening tests; faecal occult blood (FOB) testing and flexible sigmoidoscopy (FS) testing. Along with the willingness to pay values estimated using the open-ended, payment scale, closed-ended and iterative bidding formats, information is collected on household income, attitudes toward health promotion and personal risk perceptions to determine the nature and value of responses. In comparison with the alternative formats, the closed-ended question design produced significantly higher WTP valuations and different justifications for those valuations. It is hypothesised that the yea-saying effect may explain this difference. The payment scale format achieved a higher completion rate compared to the open-ended design and both formats produced broadly similar valuations. Although a subsequent study suggested evidence of range bias within the payment scale design. The iterative bidding format produced higher valuations than the open-ended and payment scale but lower than the closed-ended, it is hypothesised that valuations obtained using different initial bids demonstrate the existence of starting point bias. Across all studies, respondents who have a high health motivation, are well educated, have a high household income and who are particularly worried about the disease have a positive effect on the willingness to pay for colorectal cancer screening

Willingness to pay for colorectal cancer screening: a comparison of elicitation formats

Willingness to pay is increasingly being used in health technology assessment, although a number of methodological issues remain unresolved. Using data collected from four studies, this thesis presents the findings from a direct comparison between alternative format designs to elicit willingness to pay for two alternative colorectal cancer screening tests; faecal occult blood (FOB) testing and flexible sigmoidoscopy (FS) testing. Along with the willingness to pay values estimated using the open-ended, payment scale, closed-ended and iterative bidding formats, information is collected on household income, attitudes toward health promotion and personal risk perceptions to determine the nature and value of responses. In comparison with the alternative formats, the closed-ended question design produced significantly higher WTP valuations and different justifications for those valuations. It is hypothesised that the yea-saying effect may explain this difference. The payment scale format achieved a higher completion rate compared to the open-ended design and both formats produced broadly similar valuations. Although a subsequent study suggested evidence of range bias within the payment scale design. The iterative bidding format produced higher valuations than the open-ended and payment scale but lower than the closed-ended, it is hypothesised that valuations obtained using different initial bids demonstrate the existence of starting point bias. Across all studies, respondents who have a high health motivation, are well educated, have a high household income and who are particularly worried about the disease have a positive effect on the willingness to pay for colorectal cancer screening

Willingness to pay for colorectal cancer screening: a comparison of elicitation formats

Willingness to pay is increasingly being used in health technology assessment, although a number of methodological issues remain unresolved. Using data collected from four studies, this thesis presents the findings from a direct comparison between alternative format designs to elicit willingness to pay for two alternative colorectal cancer screening tests; faecal occult blood (FOB) testing and flexible sigmoidoscopy (FS) testing. Along with the willingness to pay values estimated using the open-ended, payment scale, closed-ended and iterative bidding formats, information is collected on household income, attitudes toward health promotion and personal risk perceptions to determine the nature and value of responses. In comparison with the alternative formats, the closed-ended question design produced significantly higher WTP valuations and different justifications for those valuations. It is hypothesised that the yea-saying effect may explain this difference. The payment scale format achieved a higher completion rate compared to the open-ended design and both formats produced broadly similar valuations. Although a subsequent study suggested evidence of range bias within the payment scale design. The iterative bidding format produced higher valuations than the open-ended and payment scale but lower than the closed-ended, it is hypothesised that valuations obtained using different initial bids demonstrate the existence of starting point bias. Across all studies, respondents who have a high health motivation, are well educated, have a high household income and who are particularly worried about the disease have a positive effect on the willingness to pay for colorectal cancer screening

Is utility-based quality of life associated with overweight in children? Evidence from the UK WAVES randomised controlled study

BACKGROUND: Quality-Adjusted Life Years (QALYs) are often used to make judgements about the relative cost-effectiveness of competing interventions and require an understanding of the relationship between health and health-related quality of life (HRQOL) when measured in utility terms. There is a dearth of information in the literature concerning how childhood overweight is associated with quality of life when this is measured using utilities. This study explores how weight is associated with utility-based HRQOL in 5–6 year olds and examines the psychometric properties of a newly developed pediatric utility measure – the CHU9D instrument. METHODS: Weight and HRQOL were examined using data collected from 1334 children recruited within a UK randomised controlled trial (WAVES) (ISRCTN97000586). Utility-based HRQOL was measured using the CHU9D, and general HRQOL measured using the PedsQL instrument. The association between weight and HRQOL was examined through a series of descriptive and multivariate analysis. The construct validity of the CHU9D was further assessed in relation to weight status, in direct comparison to the PedsQL instrument. RESULTS: The HRQOL of children who were either overweight or obese was not statistically different from children who were healthy or underweight. This result was the same for when HRQOL was measured in utility terms using the CHU9D instrument, and in general terms using the PedsQL instrument. Furthermore, the results support the construct validity of the newly developed CHU9D as the PedsQL total HRQOL scores corresponded well with the individual CHU9D dimensions. CONCLUSION: At age 5–6 years, the inverse association between overweight and HRQOL is not being captured by either the utility-based CHU9D instrument nor the PedsQL instrument. This result has implications for how the cost-effectiveness of childhood obesity interventions is measured in children aged 5–6 years. TRIAL REGISTRATION: ISRCTN Registry: ISRCTN97000586 19(th) May 2010

Levonorgestrel-releasing intrauterine system vs. usual medical treatment for menorrhagia: An economic evaluation alongside a randomised controlled trial

Objective: To undertake an economic evaluation alongside the largest randomised controlled trial comparing Levonorgestrel-releasing intrauterine device ('LNG-IUS') and usual medical treatment for women with menorrhagia in primary care; and compare the cost-effectiveness findings using two alternative measures of quality of life. Methods: 571 women with menorrhagia from 63 UK centres were randomised between February 2005 and July 2009. Women were randomised to having a LNG-IUS fitted, or usual medical treatment, after discussing with their general practitioner their contraceptive needs or desire to avoid hormonal treatment. The treatment was specified prior to randomisation. For the economic evaluation we developed a state transition (Markov) model with a 24 month follow-up. The model structure was informed by the trial women's pathway and clinical experts. The economic evaluation adopted a UK National Health Service perspective and was based on an outcome of incremental cost per Quality Adjusted Life Year (QALY) estimated using both EQ-5D and SF-6D. Results: Using EQ-5D, LNG-IUS was the most cost-effective treatment for menorrhagia. LNG-IUS costs £100 more than usual medical treatment but generated 0.07 more QALYs. The incremental cost-effectiveness ratio for LNG-IUS compared to usual medical treatment was £1600 per additional QALY. Using SF-6D, usual medical treatment was the most cost-effective treatment. Usual medical treatment was both less costly (£100) and generated 0.002 more QALYs. Conclusion: Impact on quality of life is the primary indicator of treatment success in menorrhagia. However, the most costeffective treatment differs depending on the quality of life measure used to estimate the QALY. Under UK guidelines LNG-IUS would be the recommended treatment for menorrhagia. This study demonstrates that the appropriate valuation of outcomes in menorrhagia is crucial. Copyright: © 2014 Sanghera et al

A cluster-randomised controlled trial to assess the effectiveness and cost-effectiveness of a childhood obesity prevention programme delivered through schools, targeting 6-7 year old children: the WAVES study protocol.

BACKGROUND: There is some evidence that school-based interventions are effective in preventing childhood obesity. However, longer term outcomes, equity of effects and cost-effectiveness of interventions have not been assessed. The aim of this trial is to assess the clinical and cost-effectiveness of a multi-component intervention programme targeting the school and family environment through primary schools, in preventing obesity in 6-7 year old children, compared to usual practice. METHODS: This cluster randomised controlled trial is set in 54 primary schools within the West Midlands, UK, including a multi-ethnic, socioeconomically diverse population of children aged 6-7 years. The 12-month intervention consists of healthy diet and physical activity promotion. These include: activities to increase time spent doing physical activity within the school day, participation in the 'Villa Vitality' programme (a programme that is delivered by an iconic sporting institution (Aston Villa Football Club), which provides interactive learning opportunities for physical activity and healthy eating), healthy cooking skills workshops in school time for parents and children, and provision of information to families signposting local leisure opportunities. The primary (clinical) outcome is the difference in body mass index (BMI) z-scores between arms at 3 and 18 months post-intervention completion. Cost per Quality Adjusted Life Year (QALY) will also be assessed. The sample size estimate (1000 children split across 50 schools at follow-up) is based on 90% power to detect differences in BMI z-score of 0.25 (estimated ICC ≤ 0.04), assuming a correlation between baseline and follow-up BMI z-score of 0.9. Treatment effects will be examined using mixed model ANCOVA. Primary analysis will adjust for baseline BMI z-score, and secondary analysis will adjust for pre-specified baseline school and child level covariates. DISCUSSION: The West Midlands ActiVe lifestyle and healthy Eating in School children (WAVES) study is the first trial that will examine the cost-effectiveness and long term outcomes of a childhood obesity prevention programme in a multi-ethnic population, with a sufficient sample size to detect clinically important differences in adiposity. The intervention was developed using the Medical Research Council framework for complex interventions, and outcomes are measured objectively, together with a comprehensive process evaluation. TRIAL REGISTRATION: Current Controlled Trials ISRCTN97000586 (registered May 2010)

Practice nurse-supported weight self-management delivered within the national child immunisation programme for postnatal women:a feasibility cluster RCT

Background: Pregnancy is a high-risk time for excessive weight gain. The rising prevalence of obesity in women, combined with excess weight gain during pregnancy, means that there are more women with obesity in the postnatal period. This can have adverse health consequences for women in later life and increases the health risks during subsequent pregnancies. Objective: The primary aim was to produce evidence of whether or not a Phase III trial of a brief weight management intervention, in which postnatal women are encouraged by practice nurses as part of the national child immunisation programme to self-monitor their weight and use an online weight management programme, is feasible and acceptable. Design: The research involved a cluster randomised controlled feasibility trial and two semistructured interview studies with intervention participants and practice nurses who delivered the intervention. Trial data were collected at baseline and 3 months later. The interview studies took place after trial follow-up. Setting: The trial took place in Birmingham, UK. Participants: Twenty-eight postnatal women who were overweight/obese were recruited via Birmingham Women’s Hospital or general practices. Nine intervention participants and seven nurses were interviewed. Interventions: The intervention was delivered in the context of the national child immunisation programme. The intervention group were offered brief support that encouraged self-management of weight when they attended their practice to have their child immunised at 2, 3 and 4 months of age. The intervention involved the provision of motivation and support by nurses to encourage participants to make healthier lifestyle choices through self-monitoring of weight and signposting to an online weight management programme. The role of the nurse was to provide regular external accountability for weight loss. Women were asked to weigh themselves weekly and record this on a record card in their child’s health record (‘red book’) or using the online programme. The behavioural goal was for women to lose 0.5-1 kg per week. The usual-care group received a healthy lifestyle leaflet. Main outcome measures: The primary outcome was the feasibility of a Phase III trial to test the effectiveness of the intervention, as assessed against three traffic-light stop-go criteria (recruitment, adherence to regular self-weighing and registration with an online weight management programme). Results: The traffic-light criteria results were red for recruitment (28/80, 35% of target), amber for registration with the online weight loss programme (9/16, 56%) and green for adherence to weekly self-weighing (10/16, 63%). Nurses delivered the intervention with high fidelity. In the qualitative studies, participants indicated that the intervention was acceptable to them and they welcomed receiving support to lose weight at their child immunisation appointments. Although nurses raised some caveats to implementation, they felt that the intervention was easy to deliver and that it would motivate postnatal women to lose weight. Limitations: Fewer participants were recruited than planned. Conclusions: Although women and practice nurses responded well to the intervention and adherence to self-weighing was high, recruitment was challenging and there is scope to improve engagement with the intervention.Future work: Future research should focus on investigating other methods of recruitment and, thereafter, testing the effectiveness of the intervention.Trial registration: Current Controlled Trials ISRCTN12209332.Funding: This project was funded by the National Institute for Health Research (NIHR) Health Technology Assessment programme and will be published in full in Health Technology Assessment; Vol. 25, No. 49. See the NIHR Journals Library website for further project information.</p

Sixteen-week versus standard eight-week prednisolone therapy for childhood nephrotic syndrome: the PREDNOS RCT.

BackgroundThe optimal corticosteroid regimen for treating the presenting episode of steroid-sensitive nephrotic syndrome (SSNS) remains uncertain. Most UK centres use an 8-week regimen, despite previous systematic reviews indicating that longer regimens reduce the risk of relapse and frequently relapsing nephrotic syndrome (FRNS).ObjectivesThe primary objective was to determine whether or not an extended 16-week course of prednisolone increases the time to first relapse. The secondary objectives were to compare the relapse rate, FRNS and steroid-dependent nephrotic syndrome (SDNS) rates, requirement for alternative immunosuppressive agents and corticosteroid-related adverse events (AEs), including adverse behaviour and costs.DesignRandomised double-blind parallel-group placebo-controlled trial, including a cost-effectiveness analysis.SettingOne hundred and twenty-five UK paediatric departments.ParticipantsTwo hundred and thirty-seven children presenting with a first episode of SSNS. Participants aged between 1 and 15 years were randomised (1 : 1) according to a minimisation algorithm to ensure balance of ethnicity (South Asian, white or other) and age (≤ 5 or ≥ 6 years).InterventionsThe control group (n = 118) received standard course (SC) prednisolone therapy: 60 mg/m2/day of prednisolone in weeks 1-4, 40 mg/m2 of prednisolone on alternate days in weeks 5-8 and matching placebo on alternate days in weeks 9-18 (total 2240 mg/m2). The intervention group (n = 119) received extended course (EC) prednisolone therapy: 60 mg/m2/day of prednisolone in weeks 1-4; started at 60 mg/m2 of prednisolone on alternate days in weeks 5-16, tapering by 10 mg/m2 every 2 weeks (total 3150 mg/m2).Main outcome measuresThe primary outcome measure was time to first relapse [Albustix® (Siemens Healthcare Limited, Frimley, UK)-positive proteinuria +++ or greater for 3 consecutive days or the presence of generalised oedema plus +++ proteinuria]. The secondary outcome measures were relapse rate, incidence of FRNS and SDNS, other immunosuppressive therapy use, rates of serious adverse events (SAEs) and AEs and the incidence of behavioural change [using Achenbach Child Behaviour Checklist (ACBC)]. A comprehensive cost-effectiveness analysis was performed. The analysis was by intention to treat. Participants were followed for a minimum of 24 months.ResultsThere was no significant difference in time to first relapse between the SC and EC groups (hazard ratio 0.87, 95% confidence interval 0.65 to 1.17; log-rank p = 0.3). There were also no differences in the incidence of FRNS (SC 50% vs. EC 53%; p = 0.7), SDNS (44% vs. 42%; p = 0.8) or requirement for other immunosuppressive therapy (56% vs. 54%; p = 0.8). The total prednisolone dose received following completion of study medication was 5475 mg vs. 6674 mg (p = 0.07). SAE rates were not significantly different (25% vs. 17%; p = 0.1) and neither were AEs, except poor behaviour (yes/no), which was less frequent with EC treatment. There were no differences in ACBC scores. EC therapy was associated with a mean increase in generic health benefit [0.0162 additional quality-adjusted life-years (QALYs)] and cost savings (£4369 vs. £2696).LimitationsStudy drug formulation may have prevented some younger children who were unable to swallow whole or crushed tablets from participating.ConclusionsThis trial has not shown any clinical benefit for EC prednisolone therapy in UK children. The cost-effectiveness analysis suggested that EC therapy may be cheaper, with the possibility of a small QALY benefit.Future workStudies investigating EC versus SC therapy in younger children and further cost-effectiveness analyses are warranted.Trial registrationCurrent Controlled Trials ISRCTN16645249 and EudraCT 2010-022489-29.FundingThis project was funded by the National Institute for Health Research (NIHR) Health Technology Assessment programme and will be published in full in Health Technology Assessment; Vol. 23, No. 26. See the NIHR Journals Library website for further project information

Effectiveness of a childhood obesity prevention programme delivered through schools, targeting 6 and 7 year olds: cluster randomised controlled trial (WAVES study).

OBJECTIVE: To assess the effectiveness of a school and family based healthy lifestyle programme (WAVES intervention) compared with usual practice, in preventing childhood obesity. DESIGN: Cluster randomised controlled trial. SETTING: UK primary schools from the West Midlands. PARTICIPANTS: 200 schools were randomly selected from all state run primary schools within 35 miles of the study centre (n=980), oversampling those with high minority ethnic populations. These schools were randomly ordered and sequentially invited to participate. 144 eligible schools were approached to achieve the target recruitment of 54 schools. After baseline measurements 1467 year 1 pupils aged 5 and 6 years (control: 28 schools, 778 pupils) were randomised, using a blocked balancing algorithm. 53 schools remained in the trial and data on 1287 (87.7%) and 1169 (79.7%) pupils were available at first follow-up (15 month) and second follow-up (30 month), respectively. INTERVENTIONS: The 12 month intervention encouraged healthy eating and physical activity, including a daily additional 30 minute school time physical activity opportunity, a six week interactive skill based programme in conjunction with Aston Villa football club, signposting of local family physical activity opportunities through mail-outs every six months, and termly school led family workshops on healthy cooking skills. MAIN OUTCOME MEASURES: The protocol defined primary outcomes, assessed blind to allocation, were between arm difference in body mass index (BMI) z score at 15 and 30 months. Secondary outcomes were further anthropometric, dietary, physical activity, and psychological measurements, and difference in BMI z score at 39 months in a subset. RESULTS: Data for primary outcome analyses were: baseline, 54 schools: 1392 pupils (732 controls); first follow-up (15 months post-baseline), 53 schools: 1249 pupils (675 controls); second follow-up (30 months post-baseline), 53 schools: 1145 pupils (621 controls). The mean BMI z score was non-significantly lower in the intervention arm compared with the control arm at 15 months (mean difference -0.075 (95% confidence interval -0.183 to 0.033, P=0.18) in the baseline adjusted models. At 30 months the mean difference was -0.027 (-0.137 to 0.083, P=0.63). There was no statistically significant difference between groups for other anthropometric, dietary, physical activity, or psychological measurements (including assessment of harm). CONCLUSIONS: The primary analyses suggest that this experiential focused intervention had no statistically significant effect on BMI z score or on preventing childhood obesity. Schools are unlikely to impact on the childhood obesity epidemic by incorporating such interventions without wider support across multiple sectors and environments. TRIAL REGISTRATION: Current Controlled Trials ISRCTN97000586.This study was funded by the National Institute for Health Research (NIHR) Health Technology Assessment Programme (project reference No 06/85/11)