The cost-effectiveness of germline BRCA testing-guided olaparib treatment in metastatic castration resistant prostate cancer.

BACKGROUND: Olaparib targets the DNA repair pathways and has revolutionized the management of metastatic castration resistant prostate cancer (mCRPC). Treatment with the drug should be guided by genetic testing; however, published economic evaluations did not consider olaparib and genetic testing as codependent technologies. This study aims to assess the cost-effectiveness of BRCA germline testing to inform olaparib treatment in mCRPC. METHODS: We conducted a cost-utility analysis of germline BRCA testing-guided olaparib treatment compared to standard care without testing from an Australian health payer perspective. The analysis applied a decision tree to indicate the germline testing or no testing strategy. A Markov multi-state transition approach was used for patients within each strategy. The model had a time horizon of 5 years. Costs and outcomes were discounted at an annual rate of 5 percent. Decision uncertainty was characterized using probabilistic and scenario analyses. RESULTS: Compared to standard care, BRCA testing-guided olaparib treatment was associated with an incremental cost of AU$7,841 and a gain of 0.06 quality-adjusted life-years (QALYs). The incremental cost-effectiveness ratio (ICER) was AU$143,613 per QALY. The probability of BRCA testing-guided treatment being cost effective at a willingness-to-pay threshold of AU$100,000 per QALY was around 2 percent; however, the likelihood for cost-effectiveness increased to 66 percent if the price of olaparib was reduced by 30 percent. CONCLUSION: This is the first study to evaluate germline genetic testing and olaparib treatment as codependent technologies in mCRPC. Genetic testing-guided olaparib treatment may be cost-effective with significant discounts on olaparib pricing.

Embedding stakeholder preferences in setting priorities for health research: Using a discrete choice experiment to develop a multi-criteria tool for evaluating research proposals.

We determined weights for a multi-criteria tool for assessing the relative merits of clinical-trial research proposals, and investigated whether the weights vary across relevant stakeholder groups. A cross-sectional, adaptive discrete choice experiment using 1000minds online software was administered to consumers, researchers and funders affiliated with the Australian Clinical Trials Alliance (ACTA). We identified weights for four criteria-Appropriateness, Significance, Relevance, Feasibility-and their levels, representing their relative importance, so that research proposals can be scored between 0% (nil or very low merit) and 100% (very high merit). From 220 complete survey responses, the most important criterion was Appropriateness (adjusted for differences between stakeholder groups, mean weight 28.9%) and the least important was Feasibility (adjusted mean weight 19.5%). Consumers tended to weight Relevance more highly (2.7% points difference) and Feasibility less highly (3.1% points difference) than researchers. The research or grant writing experience of researchers or consumers was not associated with the weights. A multi-criteria tool for evaluating research proposals that reflects stakeholders' preferences was created. The tool can be used to assess the relative merits of clinical trial research proposals and rank them, to help identify the best proposals for funding.

Framing, moral foundations and health taxes: interpretive analysis of Ethiopia's tobacco excise tax policy passage.

BACKGROUND: In 2019-2020, the Ethiopian government ratified a suite of legislative measures that includes levying a tax on tobacco products. This study aims to examine stakeholders' involvement, position, power and perception regarding the Ethiopian Food and Drug Authority (EFDA) bill (Proclamation No.1112/2019). This includes their meaning-making and interaction with each other during the bill's formulation, adoption and implementation stages. METHODS: We employed a mixed-methods design drawing on three sources of data: (1) policy documents and media articles from government and/or civil society groups (n=27), (2) audio and video transcripts of parliamentary debates and (3) qualitative stakeholder interviews. RESULTS: Policy actors in both the public health camp and tobacco industry employed several framing moves, engaged in distinctive patterns of moral rhetoric, and strategically invoked moral languages to galvanise support for their policy objectives. Central to this framing debate are issues of public health and the danger of tobacco, and the protection of 'the economy and personal freedom'. The public health camp's arguments and persuasiveness-which led to the passage of the EFDA bill-centred around discrediting tobacco industry's cost-benefit assessments through frame disconnection, or by polarising their own position that the financial, psychological and lost productivity costs incurred by tobacco use outweighs any tax revenue. CONCLUSIONS: A successful cultivation of an epistemic community and engagement of policy entrepreneurs-both from government agencies and civil society organisations-was critical in creating a united front and a compelling affirmative policy narrative, thereby influence excise tax policy outcomes.

Valuing the Dental Caries Utility Index in Australia.

INTRODUCTION: The Dental Caries Utility Index (DCUI) is a new oral health-specific health state classification system for adolescents, consisting of 5 domains: pain/discomfort, difficulty eating food/drinking, worried, ability to participate in activities, and appearance. Each domain has 4 response levels. This study aims to generate an Australian-specific utility algorithm for the DCUI. METHODS: An online survey was conducted using a representative sample of the adult Australian general population. The discrete choice experiment (DCE) was used to elicit the preferences on 5 domains. Then, the latent utilities were anchored onto the full health-dead scale using the visual analogue scale (VAS). DCE data were modeled using conditional logit, and 2 anchoring procedures were considered: anchor based on the worst health state and a mapping approach. The optimal anchoring procedure was selected based on the model parsimony and the mean absolute error (MAE). RESULTS: A total of 995 adults from the Australian general population completed the survey. The conditional logit estimates on 5 dimensions and levels were monotonic and statistically significant, except for the second level of the "worried" and "appearance" domains. The mapping approach was selected based on a smaller MAE between the 2 anchoring procedures. The Australian-specific tariff of DCUI ranges from 0.1681 to 1. CONCLUSION: This study developed a utility algorithm for the DCUI. This value set will facilitate utility value calculations from the participants' responses for DCUI in economic evaluations of dental caries interventions targeted for adolescents. HIGHLIGHTS: Preference-based quality-of-life measures (PBMs), which consist of a health state classification system and a set of utility values (a scoring algorithm), are used to generate utility weights for economic evaluations.This study is the first to develop an Australian utility value set for the Dental Caries Utility Index (DCUI), a new oral health-specific classification system for adolescents.The availability of a utility value set will enable using DCUI in economic evaluations of oral health interventions targeted for adolescents and may ultimately lead to more effective and efficient planning of oral health care services.

Impact of information and deliberation on the consistency of preferences for prioritization in health care - evidence from discrete choice experiments undertaken alongside citizens' juries.

BACKGROUND: Public preferences are an important consideration for priority-setting. Critics suggest preferences of the public who are potentially naïve to the issue under consideration may lead to sub-optimal decisions. We assessed the impact of information and deliberation via a Citizens' Jury (CJ) or preference elicitation methods (Discrete Choice Experiment, DCE) on preferences for prioritizing access to bariatric surgery. METHODS: Preferences for seven prioritization criteria (e.g. obesity level, obesity-related comorbidities) were elicited from three groups who completed a DCE: (i) participants from two CJs (n = 28); (ii) controls who did not participate in the jury (n = 21); (iii) population sample (n = 1,994). Participants in the jury and control groups completed the DCE pre- and post-jury. DCE data were analyzed using multinomial logit models to derive "priority weights" for criteria for access to surgery. The rank order of criteria was compared across groups, time points and CJ recommendations. RESULTS: The extent to which the criteria were considered important were broadly consistent across groups and were similar to jury recommendations but with variation in the rank order. Preferences of jurors but not controls were more differentiated (that is, criteria were assigned a greater range of priority weights) after than before the jury. Juror preferences pre-jury were similar to that of the public but appeared to change during the course of the jury with greater priority given to a person with comorbidity. Conversely, controls appeared to give a lower priority to those with comorbidity and higher priority to treating very severe obesity after than before the jury. CONCLUSION: Being informed and undertaking deliberation had little impact on the criteria that were considered to be relevant for prioritizing access to bariatric surgery but may have a small impact on the relative importance of criteria. CJs may clarify underlying rationale but may not provide substantially different prioritization recommendations compared to a DCE.

Public preferences are an important consideration for priority-setting. However, some people worry that if the public doesn't know much about the issues, their opinions might not lead to the best decisions. To make these decisions, we used two different methods to get people's opinions: Deliberative methods and preference elicitation methods. Deliberative methods gather a small group of people and have them discuss an issue in detail, whereas preference elicitation methods seek opinions through surveying a large group of people.In this paper, we assessed the impact of information and deliberation via a deliberative method (Citizens' Jury, CJ) or a preference elicitation method (Discrete Choice Experiment, DCE) on preferences for prioritising access to bariatric surgery. We used data from two CJs and a DCE focussed on prioritising access to the surgery, to find out if the opinions of those in the CJs changed or stayed the same after they heard information from experts and discussed the topic.The results showed that the important criteria were rather similar across the groups, but the order of importance was a bit different. The people in CJs had more varied opinions after discussing it, while those who didn't discuss it had less varied opinions. The participants in CJs also prioritized those with other health problems more than they did at the beginning.This study helps us understand how different methods can be used to get the public's opinions on healthcare decisions.

Health outcomes of patients with type 2 diabetes following bariatric surgery: Results from a publicly funded initiative.

OBJECTIVE: Bariatric surgery is an effective treatment for type 2 diabetes and morbid obesity. This paper analyses the clinical and patient-reported outcomes of patients treated through the Bariatric Surgery Initiative, a health system collaboration providing bariatric surgery as a state-wide public service in Queensland, Australia. RESEARCH DESIGN AND METHODS: A longitudinal prospective cohort study was undertaken. Eligible patients had type 2 diabetes and morbid obesity (BMI ≥ 35 kg/m2). Following referral by specialist outpatient clinics, 212 patients underwent Roux-en-Y gastric bypass or sleeve gastrectomy. Outcomes were tracked for a follow-up of 12-months and included body weight, BMI, HbA1c, comorbidities, health-related quality of life, eating behaviour, and patient satisfaction. RESULTS: Following surgery, patients' average body weight decreased by 23.6%. Average HbA1c improved by 24.4% and 48.8% of patients were able to discontinue diabetes-related treatment. The incidence of hypertension, non-alcoholic steatohepatitis, and renal impairment decreased by 37.1%, 66.4%, and 62.3%, respectively. Patients' emotional eating scores, uncontrolled eating and cognitive restraint improved by 32.5%, 20.7%, and 6.9%, respectively. Quality of life increased by 18.8% and patients' overall satisfaction with the treatment remained above 97.5% throughout the recovery period. CONCLUSIONS: This study confirmed previous work demonstrating the efficacy of publicly funded bariatric surgery in treating obesity, type 2 diabetes and related comorbidities, and improving patients' quality of life and eating behaviour. Despite the short follow-up period, the results bode well for future weight maintenance in this cohort.

Approaches to prioritising research for clinical trial networks: a scoping review.

BACKGROUND: Prioritisation of clinical trials ensures that the research conducted meets the needs of stakeholders, makes the best use of resources and avoids duplication. The aim of this review was to identify and critically appraise approaches to research prioritisation applicable to clinical trials, to inform best practice guidelines for clinical trial networks and funders. METHODS: A scoping review of English-language published literature and research organisation websites (January 2000 to January 2020) was undertaken to identify primary studies, approaches and criteria for research prioritisation. Data were extracted and tabulated, and a narrative synthesis was employed. RESULTS: Seventy-eight primary studies and 18 websites were included. The majority of research prioritisation occurred in oncology and neurology disciplines. The main reasons for prioritisation were to address a knowledge gap (51 of 78 studies [65%]) and to define patient-important topics (28 studies, [35%]). In addition, research organisations prioritised in order to support their institution's mission, invest strategically, and identify best return on investment. Fifty-seven of 78 (73%) studies used interpretative prioritisation approaches (including Delphi surveys, James Lind Alliance and consensus workshops); six studies used quantitative approaches (8%) such as prospective payback or value of information (VOI) analyses; and 14 studies used blended approaches (18%) such as nominal group technique and Child Health Nutritional Research Initiative. Main criteria for prioritisation included relevance, appropriateness, significance, feasibility and cost-effectiveness. CONCLUSION: Current research prioritisation approaches for groups conducting and funding clinical trials are largely interpretative. There is an opportunity to improve the transparency of prioritisation through the inclusion of quantitative approaches.

The Australian Reproductive Genetic Carrier Screening Project (Mackenzie's Mission): Design and Implementation.

Reproductive genetic carrier screening (RGCS) provides people with information about their chance of having children with autosomal recessive or X-linked genetic conditions, enabling informed reproductive decision-making. RGCS is recommended to be offered to all couples during preconception or in early pregnancy. However, cost and a lack of awareness may prevent access. To address this, the Australian Government funded Mackenzie's Mission­the Australian Reproductive Genetic Carrier Screening Project. Mackenzie's Mission aims to assess the acceptability and feasibility of an easily accessible RGCS program, provided free of charge to the participant. In study Phase 1, implementation needs were mapped, and key study elements were developed. In Phase 2, RGCS is being offered by healthcare providers educated by the study team. Reproductive couples who provide consent are screened for over 1200 genes associated with >750 serious, childhood-onset genetic conditions. Those with an increased chance result are provided comprehensive genetic counseling support. Reproductive couples, recruiting healthcare providers, and study team members are also invited to complete surveys and/or interviews. In Phase 3, a mixed-methods analysis will be undertaken to assess the program outcomes, psychosocial implications and implementation considerations alongside an ongoing bioethical analysis and a health economic evaluation. Findings will inform the implementation of an ethically robust RGCS program.

The economic evaluation of an Internet-based parental wellbeing intervention.

BACKGROUND: Internet-based parental programmes may improve parental wellbeing and mitigate the burden of mental health issues during the perinatal period. However, few studies have explored the cost and clinical impacts of such interventions. In the present study, we sought to evaluate the cost-effectiveness associated with an online cognitive behaviour therapy intervention (Baby Steps Wellbeing) to an information-only programme (Baby Care). METHODS: An alongside-trial cost-effectiveness analysis was undertaken using data from a randomised clinical trial comparing the Baby Steps Wellbeing intervention to Baby Care. Direct healthcare costs, as well as indirect costs attributed to income loss, were considered. The Assessment of Quality of Life-8 Dimensions multi-attribute utility instrument was used to estimate participant utilities, and subsequently calculate quality-adjusted life years (QALYs). Incremental cost-effectiveness ratios were calculated to assess the cost-effectiveness of the intervention. The economic evaluation adopted a societal perspective. RESULTS: In total, 496 parents were randomised to either the Baby Steps Wellbeing intervention or the Baby Care control arm. No significant differences in costs (-$27, 95% confidence interval (CI): -$1189-$1134) or QALYs (0.051, 95% CI: -0.097-0.200) were identified. Bootstrapped results showed that the Baby Steps Wellbeing programme was cost-saving and health improving in 38% of simulations and cost-effective in another 37% of simulations. CONCLUSIONS: The Baby Steps Wellbeing programme was slightly cost-saving with slightly improved health outcomes compared with Baby Care. Bootstrapped results indicate the Baby Steps Wellbeing was cost-effective in 75% of simulations. Overall, the Baby Steps Wellbeing programme is an online programme that is cost-effective. TRIAL REGISTRATION: Australian & New Zealand Clinical Trials Registry: ANZCTR12614001256662.

Application of a risk-guided strategy to secondary prevention of coronary heart disease: analysis from a state-wide data linkage in Queensland, Australia.

OBJECTIVE: This study sought whether higher risk patients with coronary heart disease (CHD) benefit more from intensive disease management. DESIGN: Longitudinal cohort study. SETTING: State-wide public hospitals (Queensland, Australia). PARTICIPANTS: This longitudinal study included 20 426 patients hospitalised in 2010 with CHD as the principal diagnosis. Patients were followed-up for 5 years. PRIMARY AND SECONDARY OUTCOMES AND MEASURES: The primary outcome was days alive and out of hospital (DAOH) within 5 years of hospital discharge. Secondary outcomes included all-cause readmission and all-cause mortality. A previously developed and validated risk score (PEGASUS-TIMI54) was used to estimate the risk of secondary events. Data on sociodemography, comorbidity, interventions and medications were also collected. RESULTS: High-risk patients (n=6573, risk score ≥6) had fewer DAOH (∆=-142 days (95% CI: -152 to -131)), and were more likely to readmit or die (all p<0.001) than their low-risk counterparts (n=13 367, risk score <6). Compared with patients who were never prescribed a medication, those who consumed maximal dose of betablockers (∆=39 days (95% CI: 11 to 67)), angiotensin-converting enzyme inhibitors/angiotensin receptor blockers (∆=74 days (95% CI: 49 to 99)) or statins (∆=109 days (95% CI: 90 to 128)) had significantly greater DAOH. Patients who received percutaneous coronary intervention (∆=99 days (95% CI: 81 to 116)) or coronary artery bypass grafting (∆=120 days (95% CI: 92 to 148)) also had significantly greater DAOH than those who did not. The effect sizes of these therapies were significantly greater in high-risk patients, compared with low-risk patients (interaction p<0.001). Analysis of secondary outcomes also found significant interaction between both medical and interventional therapies with readmission and death, implicating greater benefits for high-risk patients. CONCLUSIONS: CHD patients can be effectively risk-stratified, and use of this information for a risk-guided strategy to prioritise high-risk patients may maximise benefits from additional resources spent on intensive disease management.

Development of a classification (descriptive) system for a preference-based quality of life measure for dental caries (dental caries utility index) among adolescents.

OBJECTIVES: Preference-based quality-of-life measures (PBMs) have been developed in many clinical areas to aid estimation of more accurate utility values for economic evaluations. Existing oral health-related quality-of-life (OHRQoL) instruments are non-PBM and hence, cannot be used to generate utility values. The objective of this study was to develop a classification system for a new PBM (dental caries utility index-DCUI) for the most prevalent childhood oral health condition: dental caries. METHODS: Possible domains and items to be included in the classification system were identified based on the reviewing available pediatric non-PBM OHRQoL instruments, studies eliciting utility values for oral health outcomes and clinical dentistry textbooks and based on the findings, a draft classification system was developed. To refine the draft classification system, semi-structured interviews were conducted among a convenience sample of 15 12-17-year-old adolescents who had experience with dental caries. The classification system was further refined and validated by a group of dental experts, using a modified Delphi technique. RESULTS: The classification system comprised five items (pain/discomfort, difficulty in eating food/drinking, worried, ability to participate in activities, and appearance) and each item had a four-level response scale. CONCLUSION: The classification system developed herein is considered an amenable tool for the subsequent development of a new PBM for dental caries. Once the scoring algorithm is completed, the classification system can be used to incorporate economic evaluations of dental caries health interventions.

Comparing multi-attribute utility instruments: CP-6D, a Cerebral palsy specific instrument, vs AQoL-4D.

BACKGROUND: Economic-evaluations of Cerebral palsy (CP) were based on utility estimates of health-related quality of life (HRQoL) from generic multi-attribute utility instruments (MAUIs). However, generic instruments had limited use as they could not capture some of the important aspects of living with CP. The Cerebral palsy 6 Dimension (CP-6D) is a disease specific MAUI. In this study, we compared the results of CP-6D with the Assessment of Quality of Life (AQoL-4D), a generic MAUI, and tested the criterion validity of the CP-6D in the general population. METHODS: An online survey of the Australian general population (n = 2002), who completed both the AQoL-4D and CP-6D MAUIs, was conducted. Validity was assessed from the correlations between the domains, items and instruments. ANOVA and t-tests were used to assess the instrument's discrimination in different social demographic categories. RESULT: There was a moderate correlation between the instruments (0.64). Differences in socio-demographic characteristics showed a medium effect size (p < 0.001) in both instruments and had a similar effect on utility weights in both instruments. Although the CP-6D was more sensitive to changes in income and education. CONCLUSIONS: Our results suggest that CP-6D and AQoL-4D were measuring a similar underlying construct. Both instruments responded similarly to socio-demographic differences.

The Baby Steps Web Program for the Well-Being of New Parents: Randomized Controlled Trial.

BACKGROUND: New parents face increased risks of emotional distress and relationship dissatisfaction. Digital interventions increase support access, but few preventive programs are optimized for both parents. OBJECTIVE: This study aims to conduct the first randomized controlled trial on universal self-guided digital programs to support positive perinatal adjustment of both mothers and fathers. Effects of childcare information (Baby Care) and information plus an interactive program (Baby Steps Wellbeing) were compared from the third trimester baseline to 3 and 6 months subsequently. METHODS: The study recruited 388 co-parenting male-female adult couples expecting their first single child (26-38 weeks' gestation), using web-based registration. Most (337/388, 86.8%) were obtained from prenatal hospital classes. Couples' randomization was automated and stratified by Edinburgh Postnatal Depression Scale (EPDS) scores (50% couples scored high if either mother >7, father >5). All assessments were web-based self-reports: the EPDS and psychosocial quality of life were primary outcomes; relationship satisfaction, social support, and self-efficacy for parenting and support provision were secondary. Linear mixed models provided intention-to-treat analyses, with linear and quadratic effects for time and random intercepts for participants and couples. RESULTS: Selection criteria were met by 63.9% (248/388) of couples, who were all randomized. Most participants were married (400/496, 80.6%), tertiary educated (324/496, 65.3%), employed full time (407/496, 82%), and born in Australia (337/496, 67.9%). Their mean age was 32.2 years, and average gestation was 30.8 weeks. Using an EPDS cutoff score of 13, 6.9% (18/248) of men, and 16.1% (40/248) of women screened positive for depression at some time during the 6 months. Retention of both partners was 80.6% (201/248) at the 6-month assessments, and satisfaction with both programs was strong (92% ≥50). Only 37.3% (185/496) of participants accessed their program more than once, with higher rates for mothers (133/248, 53.6%) than fathers (52/248, 20.9%; P<.001). The EPDS, quality of life, and social support did not show differential improvements between programs, but Baby Steps Wellbeing gave a greater linear increase in self-efficacy for support provision (P=.01; Cohen d=0.26) and lower reduction in relationship satisfaction (P=.03; Cohen d=0.20) than Baby Care alone. Mothers had greater linear benefits in parenting self-efficacy over time than fathers after receiving Baby Steps Wellbeing rather than Baby Care (P=.01; Cohen d=0.51). However, the inclusion of program type in analyses on parenting self-efficacy and relationship satisfaction did not improve model fit above analyses with only parent gender and time. CONCLUSIONS: Three secondary outcomes showed differential benefits from Baby Steps Wellbeing, but for one (parenting self-efficacy), the effect only occurred for mothers, perhaps reflecting their greater program use. Increased engagement will be needed for more definitive testing of the potential benefits of Baby StepsWellbeing for perinatal adjustment. TRIAL REGISTRATION: Australian New Zealand Clinical Trials Registry ACTRN12614001256662; https://www.anzctr.org.au/Trial/Registration/TrialReview.aspx?id=367277.

Cost-effectiveness of combined catheter ablation and left atrial appendage closure for symptomatic atrial fibrillation in patients with high stroke and bleeding risk.

BACKGROUND: Combined catheter ablation (CA) and left atrial appendage closure (LAAC) have been proposed for management of symptomatic atrial fibrillation (AF) in patients with high stroke and bleeding risk. We assessed the cost-effectiveness of combined CA and LAAC compared with CA and standard oral anticoagulation (OAC) in symptomatic AF. METHODS: A Markov model was developed to assess total costs, quality-adjusted life-years (QALYs), and the incremental cost-effectiveness ratio among 2 post-CA strategies: (1) standard OAC and (2) LAAC (combined CA and LAAC procedure). The base-case used a 10-year time horizon and consisted of a hypothetical cohort of patients aged 65 years with symptomatic AF, with high thrombotic (CHA2DS2-VASc = 3) and bleeding risk (HAS-BLED = 3), and planned for AF ablation. Values for transition probabilities, utilities, and costs were derived from the literature. Costs were converted to 2020 US dollars. Half-cycle correction was applied, and costs and QALYs were discounted at 3% annually. Sensitivity analyses were performed for significant variables and scenario analyses for higher embolic risk. RESULTS: In the base-case cohort of 10,000 patients followed for 10 years, total costs for the LAAC strategy were $29,027 and for OAC strategy were $27,896. The LAAC strategy was associated with 122 fewer disabling strokes and 203 fewer intracranial hemorrhages per 10,000 patients compared with the OAC strategy. The LAAC strategy had an incremental cost-effectiveness ratio of $11,072/QALY. In sensitivity analyses, although cost-effectiveness was highly dependent on the risk of intracranial hemorrhage in the LAAC strategy and the cost of the combined procedure, LAAC was superior to OAC under the most circumstances. Scenario analyses demonstrated that the combined procedure was more cost-effective in patients with higher stroke risk. CONCLUSIONS: In symptomatic AF patients with high stroke and bleeding risk who are planned for CA, the combined CA and LAAC procedure may be a cost-effective therapeutic option and be more beneficial to patients with CHA2DS2-VASc risk score ≥3.

Using Rasch and factor analysis to develop a Proxy-Reported health state classification (descriptive) system for Cerebral Palsy.

PURPOSE: The Cerebral Palsy quality of life instrument is a well-known health-related quality of life measure for children with Cerebral Palsy. Due to its length it is not suitable as the basis of a preference-based instrument. The aim of this study is to develop a short version of the Cerebral Palsy quality of life instrument that can subsequently be scored as a multi-attribute utility instrument through assigning preference-based values. METHODS: A sample of 473 participants who have a child with Cerebral Palsy completed the Cerebral Palsy quality of life instrument(proxy-version) instrument. After deleting questions related only to the proxy, the dimensional structure was obtained using exploratory factor analysis. Extended Rasch analysis was then undertaken to test the psychometric performance of items and select the best item to represent each dimension. Expert opinion was sought to confirm the dimensions and items. RESULTS: A six-dimension classification system was identified, in which four domains were extracted from the factor analysis. Following expert opinion, two other domains were also added, as these were considered to have significant impact on health-related quality of life in children with Cerebral Palsy. CONCLUSIONS: The combination of Factor and Rasch analysis along with consultation with patients, clinicians and experts in health-related quality-of-life instrument development, has resulted in a short version of the Cerebral Palsy quality of life instrument.IMPLICATION FOR REHABILITATIONThis study provides the first classification system for children with Cerebral Palsy.The Cerebral Palsy-six dimension (CP-6D) survey, which is a short version of Cerebral Palsy Quality Of Life instrument, can be timesaving when measuring quality of life in children with Cerebral Palsy.The short version (CP-6D) can be used in preference based measurement and generate quality adjusted life years for children with Cerebral Palsy.

Utility Values for the CP-6D, a Cerebral Palsy-Specific Multi-Attribute Utility Instrument, Using a Discrete Choice Experiment.

BACKGROUND AND OBJECTIVE: The CP-6D is a new preference-based measure derived from the CPQOL, a cerebral palsy-specific quality-of-life questionnaire. The CP-6D contains six dimensions, each with five levels. A preference-based value set is required to score the CP-6D on a utility scale and render it suitable for cost-utility analysis. This study aims to estimate the utility value set for the CP-6D for interventions for people with cerebral palsy (CP). METHODS: A discrete choice experiment was designed and administrated to an adult Australian online panel. Each respondent answered 12 choice sets. Each choice was presented as a combination of the health state from the CP-6D and duration spent in that health state before death. Conditional logit and mixed logit regression were used to analyse the data. The utility values were estimated as a ratio of the coefficient of each dimension to the coefficient of the duration. RESULTS: A total of 2002 participants completed the survey and responded to each choice. Generally, the dimension levels were monotonic, meaning the coefficients reflected the ordered nature of the levels in each dimension. The dimensions relating to manual ability, social well-being and acceptance had the greatest effect on choice. The value of the worst 'pits' health state is - 0.582. CONCLUSION: This study provides the first CP-specific utility value set that can potentially be used in cost-utility analyses of interventions for people with CP where the CPQOL has been applied, both prospectively and retrospectively.

Valuation study for a preference-based quality of life measure for dental caries (Dental Caries Utility Index - DCUI) among Australian adolescents - study protocol.

INTRODUCTION: A new health state classification system has been developed for dental caries - Dental Caries Utility Index (DCUI) to facilitate the assessment of oral health interventions in the cost-utility analysis (CUA). This paper reports the protocol for a valuation study, which aims to generate a preference-based algorithm for the classification system for the DCUI. METHODS AND ANALYSIS: Discrete choice experiments (DCEs) will be conducted to value health states generated by the DCUI classification system and preferences for these health states will be modelled to develop a utility algorithm. DCEs produce utility values on a latent scale and these values will be anchored into the full health-dead scale to calculate the quality-adjusted life years in CUA. There is no previous evidence for the most suitable anchoring method for dental caries health state valuation. Hence, we will first conduct pilot studies with two anchoring approaches; DCE including duration attribute and DCE anchoring to worst heath state in Visual Analogue Scale. Based on the pilot studies, the most suitable anchoring method among two approaches will be used in the main valuation survey, which will be conducted as an online survey among a representative sample of 2000 adults from the Australian general population. Participants will be asked to complete a set of DCE choice tasks along with anchoring tasks, basic social-demographic questions, DCUI, a generic preference-based measure and oral health quality of life instrument. ETHICS AND DISSEMINATION: Ethical approval for this study was obtained from the Human Research Ethics Committee, Griffith University (reference number HREC/2019/550). The generated algorithm will facilitate the use of the new dental caries preference-based measure in economic evaluations of oral health interventions. The results will be disseminated through journal articles and professional conferences.

Correction: Cost-effectiveness analysis of a mobile ear screening and surveillance service versus an outreach screening, surveillance and surgical service for indigenous children in Australia.

[This corrects the article DOI: 10.1371/journal.pone.0138369.].

Discrete choice experiments to generate utility values for multi-attribute utility instruments: a systematic review of methods.

OBJECTIVES: In recent years, discrete choice experiments (DCEs) have become frequently used to generate utility values, but there are a diverse range of approaches to do this. The primary focus of this systematic review is to summarise the methods used for the design and analysis of DCEs when estimating utility values in both generic and condition-specific preference-based measures. METHODS: Published literature using DCEs to estimate utility values from preference-based instruments were identified from MEDLINE, Embase, Cochrane Library and CINAHL using PRISMA guidelines. To assess the different DCE methods, standardised information was extracted from the articles including the DCE design method, the number of choice sets, the number of DCE pairs per person, randomisation of questions, analysis method, logical consistency tests and techniques for anchoring utilities. The CREATE checklist was used to assess the quality of the studies. RESULTS: A total of 38 studies with samples from the general population, students and patients were included. Values for health states described using generic multi attribute instruments (MAUIs) (especially the EQ-5D) were the most commonly explored using DCEs. The studies showed considerable methodology and design diversity (number of alternatives, attributes, sample size, choice task presentation and analysis). Despite these differences, the quality of articles reporting the methods used for the DCE was generally high. CONCLUSION: DCEs are an important approach to measure utility values for both generic and condition-specific instruments. However, a gold standard method cannot yet be recommended.