Quality-adjusted Life Year Estimates Research Articles

Mapping functions for the PHQ-9 and GAD-7 to generate EQ-5D-3L for economic evaluation.

Generic preferenced-based measures, such as EQ-5D-3L, that are used to estimate quality adjusted life years (QALYs) for economic evaluation are not always available in clinical trials. Predicting EQ-5D-3L values from the commonly used Patient Health Questionnaire 9 (PHQ-9) and Generalised Anxiety Disorder-7 (GAD-7) would allow estimation of QALYs from such trials. The aim was to provide mapping functions to estimate EQ-5D-3L from PHQ-9 and GAD-7 to facilitate economic evaluation. Data was drawn from four trials of patients with symptoms of depression testing collaborative care or computerised cognitive behavioural therapy. Patients completed PHQ-9, GAD-7, and EQ-5D-3L at different timepoints. Mapping was undertaken using adjusted limited dependent variable mixture models (ALDVMM), ordinary least squares (OLS), and Tobit models based on PHQ-9, GAD-7 scores or questions, and age to predict EQ-5D-3L utilities. Models were selected based on mean error (ME), mean absolute error (MAE), root mean squared error (RMSE), model goodness of fit, and visual inspection of the predictions. There were 5583 and 3942 observations for EQ-5D-3L combined with PHQ-9 and GAD-7 respectively. ALDVMM models had low ME ( ≤|0.0018|) and MAE ranging from 0.189 to 0.192, while RMSE was from 0.251 to 0.254 and had better predictions than OLS and Tobit models. ALDVMM models with four components based on PHQ-9 and GAD-7 scores are recommended for estimating EQ-5D-3L utilities. Recommended mapping functions provide users with an approach to estimate EQ-5D-3L utilities for economic evaluation using PHQ-9, GAD-7, or both scores where they have been used together.

Artificial intelligence support in MR imaging of incidental renal masses: an early health technology assessment.

This study analyzes the potential cost-effectiveness of integrating an artificial intelligence (AI)-assisted system into the differentiation of incidental renal lesions as benign or malignant on MR images during follow-up. For estimation of quality-adjusted life years (QALYs) and lifetime costs, a decision model was created, including the MRI strategy and MRI + AI strategy. Model input parameters were derived from recent literature. Willingness to pay (WTP) was set to $100,000/QALY. Costs of $0 for the AI were assumed in the base-case scenario. Model uncertainty and costs of the AI system were assessed using deterministic and probabilistic sensitivity analysis. Average total costs were at $8054 for the MRI strategy and $7939 for additional use of an AI-based algorithm. The model yielded a cumulative effectiveness of 8.76 QALYs for the MRI strategy and of 8.77 for the MRI + AI strategy. The economically dominant strategy was MRI + AI. Deterministic and probabilistic sensitivity analysis showed high robustness of the model with the incremental cost-effectiveness ratio (ICER), which represents the incremental cost associated with one additional QALY gained, remaining below the WTP for variation of the input parameters. If increasing costs for the algorithm, the ICER of $0/QALY was exceeded at $115, and the defined WTP was exceeded at $667 for the use of the AI. This analysis, rooted in assumptions, suggests that the additional use of an AI-based algorithm may be a potentially cost-effective alternative in the differentiation of incidental renal lesions using MRI and needs to be confirmed in the future. These results hint at AI's the potential impact on diagnosing renal masses. While the current study urges careful interpretation, ongoing research is essential to confirm and seamlessly integrate AI into clinical practice, ensuring its efficacy in routine diagnostics. • This is a model-based study using data from literature where AI has been applied in the diagnostic workup of incidental renal lesions. • MRI + AI has the potential to be a cost-effective alternative in the differentiation of incidental renal lesions. • The additional use of AI can reduce costs in the diagnostic workup of incidental renal lesions.

Comparison of partitioned survival modeling with state transition modeling approaches with or without consideration of brain metastasis: a case study of Osimertinib versus pemetrexed-platinum

BackgroundThe partitioned survival model (PSM) and the state transition model (STM) are widely used in cost-effectiveness analyses of anticancer drugs. Using different modeling approaches with or without consideration of brain metastasis, we compared the quality-adjusted life-year (QALY) estimates of Osimertinib and pemetrexed-platinum in advanced non-small cell lung cancer with epidermal growth factor receptor mutations.MethodsWe constructed three economic models using parametric curves fitted to patient-level data from the National Health Insurance Review and Assessment claims database from 2009 to 2020. PSM and 3-health state transition model (3-STM) consist of three health states: progression-free, post-progression, and death. The 5-health state transition model (5-STM) has two additional health states (brain metastasis with continuing initial therapy, and with subsequent therapy). Time-dependent transition probabilities were calculated in the state transition models. The incremental life-year (LY) and QALY between the Osimertinib and pemetrexed-platinum cohorts for each modeling approach were estimated over seven years.ResultsThe PSM and 3-STM produced similar incremental LY (0.889 and 0.899, respectively) and QALY (0.827 and 0.840, respectively). However, 5-STM, which considered brain metastasis as separate health states, yielded a slightly higher incremental LY (0.910) but lower incremental QALY (0.695) than PSM and 3-STM.ConclusionsOur findings indicate that incorporating additional health states such as brain metastases into economic models can have a considerable impact on incremental QALY estimates. To ensure appropriate health technology assessment decisions, comparison and justification of different modeling approaches are recommended in the economic evaluation of anticancer drugs.

Subthreshold micropulse laser versus standard laser for the treatment of central-involving diabetic macular oedema with central retinal thickness of

ObjectivesTo estimate the economic costs, health-related quality-of-life outcomes and cost-effectiveness of subthreshold micropulse laser (SML) versus standard laser (SL) for the treatment of diabetic macular oedema (DMO) with central retinal...

Cost-effectiveness of closed incision negative pressure wound therapy in preventing surgical site infection among obese women giving birth by caesarean section: An economic evaluation (DRESSING trial).

There is growing evidence regarding the potential of closed incision negative pressure wound therapy (ci-NPWT) to prevent surgical site infections (SSIs) in healing wounds by primary closure following a caesarean section (CS). To assess the cost-effectiveness of ci-NPWT compared to standard dressings for prevention of SSI in obese women giving birth by CS. Cost-effectiveness and cost-utility analyses from a health service perspective were undertaken alongside a multicentre pragmatic randomised controlled trial, which recruited women with a pre-pregnancy body mass index ≥30 kg/m2 giving birth by elective/semi-urgent CS who received ci-NPWT (n=1017) or standard dressings (n=1018). Resource use and health-related quality of life (SF-12v2) collected during admission and for four weeks post-discharge were used to derive costs and quality-adjusted life years (QALYs). ci-NPWT was associated with AUD$162 (95%CI -$170 to $494) higher cost per person and an additional $12 849 (95%CI -$62 138 to $133 378) per SSI avoided. There was no detectable difference in QALYs between groups; however, there are high levels of uncertainty around both cost and QALY estimates. There is a 20% likelihood that ci-NPWT would be considered cost-effective at a willingness-to-pay threshold of $50 000 per QALY. Per protocol and complete case analyses gave similar results, suggesting that findings are robust to protocol deviators and adjustments for missing data. ci-NPWT for the prevention of SSI in obese women undergoing CS is unlikely to be cost-effective in terms of health service resources and is currently unjustified for routine use for this purpose.

Incorporating Prior Beliefs Into Meta-Analyses of Health-State Utility Values Using the Bayesian Power Prior

ObjectivesHealth-state utility values (HSUVs) directly affect estimates of Quality-Adjusted Life-Years and thus the cost-utility estimates. In practice a single preferred value (SPV) is often selected for HSUVs, despite meta-analysis being an option when multiple (credible) HSUVs are available. Nevertheless, the SPV approach is often reasonable because meta-analysis implicitly considers all HSUVs as equally relevant. This article presents a method for the incorporation of weights to HSUV synthesis, allowing more relevant studies to have greater influence. MethodsUsing 4 case studies in lung cancer, hemodialysis, compensated liver cirrhosis, and diabetic retinopathy blindness, a Bayesian Power Prior (BPP) approach is used to incorporate beliefs on study applicability, reflecting the authors’ perceived suitability for UK decision making. Older studies, non-UK value sets, and vignette studies are thus downweighted (but not disregarded). BPP HSUV estimates were compared with a SPV, random effects meta-analysis, and fixed effects meta-analysis. Sensitivity analyses were conducted iteratively updating the case studies, using alternative weighting methods, and simulated data. ResultsAcross all case studies, SPVs did not accord with meta-analyzed values, and fixed effects meta-analysis produced unrealistically narrow CIs. Point estimates from random effects meta-analysis and BPP models were similar in the final models, although BPP reflected additional uncertainty as wider credible intervals, particularly when fewer studies were available. Differences in point estimates were seen in iterative updating, weighting approaches, and simulated data. ConclusionsThe concept of the BPP can be adapted for synthesizing HSUVs, incorporating expert opinion on relevance. Because of the downweighting of studies, the BPP reflected structural uncertainty as wider credible intervals, with all forms of synthesis showing meaningful differences compared with SPVs. These differences would have implications for both cost-utility point estimates and probabilistic analyses.

Estimation of Health State Utility Values in Fabry Disease Using Vignette Development and Valuation

Background: Health state utilities are measures of health-related quality of life that reflect the value placed on improvements in patients’ health status and are necessary for estimation of quality-adjusted life-years. Health state utility data on Fabry disease (FD) are limited. In this study we used vignette (scenario) construction and valuation to develop health state utilities. Objectives: The aim of this study was to use vignette construction and valuation to estimate health state utility values suitable for inclusion in economic models of FD treatments. Methods: Health state vignettes were developed from semistructured qualitative telephone interviews with patients with FD and informed by published literature and input from an expert. Each vignette was valued in an online survey by members of the United Kingdom (UK) general population using the composite time trade-off (TTO) method, which aims to determine the time the respondent would trade to live in full health compared with each impaired health state. Results: Eight adults (50% women) with FD from the UK were interviewed. They were recruited via various approaches, including patient organizations and social media. The interviewees’ responses, evidence from published literature, and input from a clinical expert informed the development of 6 health state vignettes (pain, moderate clinically evident FD [CEFD], severe CEFD, end-stage renal disease [ESRD], stroke, and cardiovascular disease [CVD]) and 3 combined health states (severe CEFD + ESRD, severe CEFD + CVD, and severe CEFD + stroke). A vignette valuation survey was administered to 1222 participants from the UK general population who were members of an external surveying organization and agreed to participate in this study; 1175 surveys were successfully completed and included in the analysis. Responses to TTO questions were converted into utility values for each health state. Pain was the highest valued health state (0.465), and severe CEFD + ESRD was the lowest (0.033). Discussion: Overall, mean utility values declined as the severity of the vignettes increased, indicating that respondents were more willing to trade life-years to avoid a severe health state. Conclusions: Health state vignettes reflect the effects of FD on all major health-related quality-of-life domains and may help to support economic modeling for treatment of FD.

Estimation of Health State Utility Values in Fabry Disease Using Vignette Development and Valuation.

Background: Health state utilities are measures of health-related quality of life that reflect the value placed on improvements in patients' health status and are necessary for estimation of quality-adjusted life-years. Health state utility data on Fabry disease (FD) are limited. In this study we used vignette (scenario) construction and valuation to develop health state utilities. Objectives: The aim of this study was to use vignette construction and valuation to estimate health state utility values suitable for inclusion in economic models of FD treatments. Methods: Health state vignettes were developed from semistructured qualitative telephone interviews with patients with FD and informed by published literature and input from an expert. Each vignette was valued in an online survey by members of the United Kingdom (UK) general population using the composite time trade-off (TTO) method, which aims to determine the time the respondent would trade to live in full health compared with each impaired health state. Results: Eight adults (50% women) with FD from the UK were interviewed. They were recruited via various approaches, including patient organizations and social media. The interviewees' responses, evidence from published literature, and input from a clinical expert informed the development of 6 health state vignettes (pain, moderate clinically evident FD [CEFD], severe CEFD, end-stage renal disease [ESRD], stroke, and cardiovascular disease [CVD]) and 3 combined health states (severe CEFD + ESRD, severe CEFD + CVD, and severe CEFD + stroke). A vignette valuation survey was administered to 1222 participants from the UK general population who were members of an external surveying organization and agreed to participate in this study; 1175 surveys were successfully completed and included in the analysis. Responses to TTO questions were converted into utility values for each health state. Pain was the highest valued health state (0.465), and severe CEFD + ESRD was the lowest (0.033). Discussion: Overall, mean utility values declined as the severity of the vignettes increased, indicating that respondents were more willing to trade life-years to avoid a severe health state. Conclusions: Health state vignettes reflect the effects of FD on all major health-related quality-of-life domains and may help to support economic modeling for treatment of FD.

The impact of different types of NHS expenditure on health: Marginal cost per QALY estimates for England for 2016/17

English data from 2003 to 2012 suggests that it costs the NHS £10,000 to generate an additional quality-adjusted life year (QALY). This estimate relates to all NHS expenditure and no attempt was made to explore possible heterogeneity within this total. Different types of expenditure – such as secondary care, primary care and specialized commissioning – may have different productivities and estimates of these may help policymakers decide where additional investment is most beneficial. We use the two-stage least squares estimator and data for 2016 to explore the mortality response to three types of healthcare expenditure. Three specifications are estimated for each type of expenditure: backward selection and regularized regression are used to identify parsimonious specifications, and a full specification with all covariates is also estimated. The regression results are combined with information about survival and morbidity disease burden to calculate the marginal cost per QALY for each type of expenditure: the most conservative results suggest that this is about £8,000 for locally (CCG) commissioned services, while estimates for specialized commissioning and primary care are more uncertain. When this heterogeneity is taken into account, the estimated marginal cost per QALY for all NHS expenditure increases slightly, from about £6,000 to £7,000. Our results suggest that additional investment is likely to be most productive in primary care and in locally commissioned services.

Abstract P1-10-13: Economic comparison of standard external beam whole breast (WBI) versus accelerated partial breast irradiation (ABPI) in postmenopausal women with early-stage breast cancer. Results from the French SHARE randomized trial

Abstract Purpose: The economic evaluation reports the incremental cost utility ratio and budget impact of APBI vs standard external beam WBI for the treatment of post-menopausal women with early stage breast cancer. Methods and materials: We compared 488 women in the standard arm (1 fraction per day delivered 5 days per week over 3 or 6/6.5 weeks) to 490 women in the ABPI arm (ten fractions delivered twice per day over one week). We took the perspective of the healthcare system, a 3-year time horizon; the outcomes were quality adjusted life years (QALYs). QALYs were calculated from the EQ5D5L questionnaires at baseline, 3 months, 6 months, 12 months and yearly after irradiation; scores were converted into utilities using the French value set and QALYs computed with the area under the curve approach. Measures of within-trial use of hospital resources were based on routine hospital data via patient-level information. We used the itemized and DRG cost data from each individual patient. Transportation costs were added in a sensitivity analysis. A 2.5% discount rate was applied to costs and QALYs. An incremental analysis with differences in costs and QALYs was performed to calculate the cost utility ratio. Bootstrapping was used to quantify uncertainty on the joint distribution of costs and outcomes, and 1,000 paired estimates of mean differential costs and QALYs were reported on a cost-effectiveness plane. A budget impact analysis based on incidence of breast cancer estimates was added. All analyses followed the intent to treat principle. Results: Cost and utilities were available for the entire population. Costs and QALY results are presented in table 1. The 2 925 € (95% IC, -3 364 €; - 2 452 €) significant difference in total costs favoring ABPI was driven by the difference in radiotherapy costs and partly by lower transportation costs. No significant difference was found in QALYs. Figure 1 shows the uncertainty of the joint distribution of costs and QALYs. All replication are in the lower half of the plane indicating that ABPI is cost saving with QALYs distributed on each side of the vertical axis indicating equal distribution of QALYs. would be eligible for treatment with ABPI. The uptake of ABPI for 16% of these women would result in a 16 million€ cost saving. Conclusions: At three years, ABPI for the treatment of postmenopausal women with early-stage breast cancer was found to be cost saving, with no difference in outcome measured by QALYs. Table 1. Figure 1. Citation Format: Alicia Le Bras, Yazid Belkacemi, céline Bourgier, Isabelle Gabelle-Flandin, Adeline Petit, Philippe Guilbert, Julien Geffrelot, Christian Carrie, Eleonor RIVIN DEL CAMPO, Chantal Hanzen, claire charra-brunaud, Guillaume Auzac, Thomas Lacornerie, Jérôme Lemonnier, Eric Lartigau, Isabelle Durand-Zaleski. Economic comparison of standard external beam whole breast (WBI) versus accelerated partial breast irradiation (ABPI) in postmenopausal women with early-stage breast cancer. Results from the French SHARE randomized trial [abstract]. In: Proceedings of the 2022 San Antonio Breast Cancer Symposium; 2022 Dec 6-10; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2023;83(5 Suppl):Abstract nr P1-10-13.

The Estimated Lifetime Quality-Adjusted Life-Years Lost Due to Chlamydia, Gonorrhea, and Trichomoniasis in the United States in 2018.

Comprehensive evaluation of the quality-adjusted life-years (QALYs) lost attributable to chlamydia, gonorrhea, andtrichomoniasis in the United States is lacking. We adapted a previous probability-tree model to estimate the average number of lifetime QALYs lost due to genital chlamydia, gonorrhea, and trichomoniasis, per incident infection and at the population level, by sex and age group. We conducted multivariate sensitivity analyses to address uncertainty around key parameter values. The estimated total discounted lifetime QALYs lost for men and women, respectively, due to infections acquired in 2018, were 1541 (95% uncertainty interval [UI], 186-6358) and 111 872 (95% UI, 29 777-267 404) for chlamydia, 989 (95% UI, 127-3720) and 12 112 (95% UI, 2 410-33 895) for gonorrhea, and 386 (95% UI, 30-1851) and 4576 (95% UI, 13-30 355) for trichomoniasis. Total QALYs lost were highest among women aged 15-24 years with chlamydia. QALYs lost estimates were highly sensitive to disutilities (health losses) of infections and sequelae, and to duration of infections and chronic sequelae for chlamydia and gonorrhea in women. The 3 sexually transmitted infections cause substantial health losses in the United States, particularly gonorrhea and chlamydia among women. The estimates of lifetime QALYs lost per infection help to prioritize prevention policies and inform cost-effectiveness analyses of sexually transmitted infection interventions.

Economic costs, health-related quality of life outcomes and cost-utility of a physical and psychological group intervention targeted at older adults with neurogenic claudication

BackgroundEmerging evidence suggests that structured and progressive exercise underpinned by a cognitive behavioural approach can improve functional outcomes in patients with neurogenic claudication (NC). However, evidence surrounding its economic benefits is lacking.ObjectivesTo estimate the economic costs, health-related quality of life outcomes and cost-effectiveness of a physical and psychological group intervention (BOOST programme) versus best practice advice (BPA) in older adults with NC.MethodsAn economic evaluation was conducted based on data from a pragmatic, multicentre, superiority, randomised controlled trial. The base-case economic evaluation took the form of an intention-to-treat analysis conducted from a UK National Health Service (NHS) and personal social services (PSS) perspective and separately from a societal perspective. Costs (£ 2018–2019 prices) were collected prospectively over a 12 month follow-up period. A bivariate regression of costs and quality-adjusted life-years (QALYs), with multiple imputation of missing data, was conducted to estimate the incremental cost per QALY gained and the incremental net monetary benefit (INMB) of the BOOST programme in comparison to BPA. Sensitivity and pre-specified subgroup analyses explored uncertainty and heterogeneity in cost-effectiveness estimates.ResultsParticipants (N = 435) were randomised to the BOOST programme (n = 292) or BPA (n = 143). Mean (standard error [SE]) NHS and PSS costs over 12 months were £1,974 (£118) in the BOOST arm versus £1,827 (£169) in the BPA arm (p = 0.474). Mean (SE) QALY estimates were 0.620 (0.009) versus 0.599 (0.006), respectively (p = 0.093). The probability that the BOOST programme is cost-effective ranged between 67 and 83% (NHS and PSS perspective) and 79–89% (societal perspective) at cost-effectiveness thresholds between £15,000 and £30,000 per QALY gained. INMBs ranged between £145 and £464 at similar cost-effectiveness thresholds. The cost-effectiveness results remained robust to sensitivity analyses.ConclusionsThe BOOST programme resulted in modest QALY gains over the 12 month follow-up period. Future studies with longer intervention and follow-up periods are needed to address uncertainty around the health-related quality of life impacts and cost-effectiveness of such programmes.Trial registration This study has been registered in the International Standard Randomised Controlled Trial Number registry, reference number ISRCTN12698674. Registered on 10 November 2015.

Translation and cultural adaptation of the Health Utilities Preschool to Brazilian Portuguese

IntroductionHealth research is particularly important in low- and middle-income countries (LMICs), where improvements must be achieved with limited resources, and where the great majority of the world's population, especially children, live. Improvements in public health detection in Brazil have resulted in cancer becoming the most prevalent cause of death by disease in the group aged 1 to 19 years, hence, delivering cost-effective care to the group is a priority. Preference-based measures of health status and health-related quality of life (HRQL) integrate morbidity and mortality and provide utility scores for the estimation of quality-adjusted life years to be used in cost-effectiveness analyses and economic evaluation. The generic preference-based instrument Health Utilities - Preschool (HuPS) measures the health status of young children and is applicable to the age group 2 to 5 years, who carry the highest incidence of cancer in childhood. MethodsThe translation of the HuPS classification system followed recommended protocols from published guidelines. Forward and backward translations were performed by a team of six qualified professionals and linguistic validation was undertaken with a sample of parents of preschool children. Main resultsInitial disagreements on individual words occurring in 0.5-1.5% were resolved by consensus. A final version of the instrument was validated by the sample of parents. ConclusionsThe translation and cultural adaptation of the HuPS into Brazilian Portuguese were accomplished as the first step in the validation of the HuPS instrument in Brazil.

Cost-effectiveness of benralizumab versus mepolizumab and dupilumab in patients with severe uncontrolled eosinophilic asthma in Spain

Objective To assess the cost-effectiveness of benralizumab (benra) vs. mepolizumab (mepo) and dupilumab (dupi) for the treatment of patients with severe uncontrolled asthma from the Spanish Health System perspective. Methods Exacerbations avoided, quality-adjusted life years (QALYs) gained and costs in a 5-year period were estimated with a Markov model for a cohort of 1,000 patients in which, based on published evidence, 31% of the patients received biologics + oral corticosteroids (OCS) and 69% received only biologics. Efficacy data (exacerbation reduction and OCS elimination) were derived from a matching-adjusted indirect comparison. Published EQ-5D utilities per health state (biologic alone, biologic + OCS, standard of care + OCS, exacerbations, and post-exacerbations) were used for QALY estimation. Utility decrements associated with exacerbation management [-0.1 (OCS or emergency visits), −0.2 (hospitalization)] derived from the literature were applied. Costs (€, 2022) included drug acquisition (ex-factory price), administration and disease management. An expert panel (2 pneumologists and 1 pharmacist) validated all inputs. Results Benra was more effective (52.21 QALYs) than mepo (51.39 QALYs) and dupi (51.30 QALYs). Benra avoided more exacerbations (2.87 exacerbations) compared to mepo (4.70 exacerbations) and dupi (5.11 exacerbations) for the 5-year horizon. Total costs/patient were €56,093.77 (benra), €59,280.45 (mepo) and €62,991.76 (dupi), resulting in benra dominating (more QALYs with lower costs) vs. mepo and dupi. Conclusions Benralizumab can be considered as a dominant treatment alternative vs. other biologic drugs for the treatment of uncontrolled severe eosinophilic asthma patients in Spain.

Quality-adjusted life years for digital cognitive behavioural therapy for insomnia (Sleepio): a secondary analysis.

Insomnia is common, and difficulty with daytime functioning is a core symptom. Studies show cognitive behavioural therapy (CBT) improves functioning, but evidence is needed on its value for money. Quality-adjusted life years (QALYs), capturing length and quality of life, provide a standard metric by which to judge whether a treatment is worth its cost. Studies have found QALY gains with therapist-delivered and therapist-guided CBT, but most have not reached statistical significance. Estimates of QALY gains with fully automated digital CBT (dCBT) for insomnia are lacking. To assess whether dCBT (Sleepio) for insomnia is associated with gains in QALYs compared with a sleep hygiene education control. A secondary analysis of a large effectiveness trial of 1711 participants from the UK, US, and Australia. EQ-5D scores, the National Institute for Health and Care Excellence's (NICE's) preferred measure of health-related quality of life (HRQoL), were predicted (mapped) from the 10-item Patient-Reported Outcomes Measurement Information System (PROMIS-10) Global Health scores and used to determine QALYs from baseline to 24 weeks (controlled), and to 48 weeks (uncontrolled). At week 24, QALYs were significantly higher for the dCBT group, with mean QALYs 0.375 and 0.362 in the dCBT and control groups, respectively. The mean difference was 0.014 (95% confidence interval [CI] = 0.008 to 0.019), and this difference was maintained over the 48-week study period (0.026, 95% CI = 0.016 to 0.036). The difference of 0.026 QALYs is equivalent to 9.5 days in perfect health. Sleepio is associated with statistically significant gains in QALYs over time compared with control. Findings may be used to power future studies and inform cost-effectiveness analyses of automated dCBT for insomnia scaled to a population level.

Abstract 170: Cost-effectiveness Of Icosapent Ethyl In Reduce-it USA: Results From Patients Randomized In The United States

Background: The Reduction of Cardiovascular Events with Icosapent Ethyl (IE)-Intervention Trial (REDUCE-IT) demonstrated the efficacy of IE among patients with elevated triglyceride levels despite the use of statins. This study aimed to examine the cost-effectiveness (CE) of IE among US adults using both in-trial and lifetime time horizons. Methods: US patients in REDUCE-IT were included in the in-trial analysis. We included all cardiovascular and serious adverse events from the REDUCE-IT database where rates differed between the study arms; we used patient-level data from REDUCE-IT USA based on 2019 US costs and $4.16/day for IE. The lifetime analysis used a microsimulation Markov model. Both analyses considered value from a US health sector perspective. The primary result is the incremental CE ratio (ICER), measured as incremental costs divided by incremental quality-adjusted life-years (QALY) of IE compared with placebo. We performed univariate and probabilistic sensitivity analyses (PSA) to capture the uncertainties involved in the estimation of costs and QALYs. Results: Based on 3146 REDUCE-IT USA participants, there was an incremental gain in QALYs with IE compared with placebo using in-trial (3.28 vs. 3.13) and lifetime (10.36 vs. 9.83) time horizons. Total healthcare costs were lower with IE compared with placebo for both in-trial ($20,221 vs. $20,357) and lifetime ($201,842 vs. $204,701). IE was a dominant strategy compared to placebo using a lifetime time horizon with a 74.8% probability of being more effective and costing less and had a 99.6% probability of costing below $50,000 per QALY. The lifetime PSA showed that IE was a dominant strategy in 65.6% of simulations and cost-effective in 98.8%, 99.6%, and 99.9% of simulations at the $50,000, $100,000, and $150,000 per QALY gained thresholds, respectively. Conclusions: The REDUCE-IT USA cost-effectiveness analysis has shown that IE provides better outcomes with lower costs, dominant both in-trial and lifetime as well in the majority of sensitivity analyses and subgroups, both in primary and secondary prevention. These results, with the clinical evidence of efficacy, suggest that at $4.16 per day, IE therapy should be strongly considered in patients similar to those enrolled in REDUCE-IT USA.

Monetised estimated quality-adjusted life year (QALY) losses for non-fatal injuries

BackgroundQuality-adjusted life years (QALYs) provide a means to compare injuries using a common measurement which allows quality of life and duration of life from an injury to be considered. A...

Methodological challenges surrounding QALY estimation for paediatric economic evaluation

Cost-utility analysis remains the preferred form of economic evaluation for health technology assessment, pricing and reimbursement authorities in several countries. The results of cost-utility analyses are commonly expressed in terms of incremental cost per quality-adjusted life year (QALY) gained where the QALY combines length of life and health-related quality of life in a single metric. This commentary provides an overview of key methodological challenges surrounding QALY estimation for paediatric economic evaluation. These challenges include issues surrounding the relevant attributes to incorporate into measurement instruments, appropriate respondents for the measurement and valuation tasks, perspectives adopted when completing valuation tasks, potential sources of bias in the description and valuation processes, and the paucity of psychometric evidence for existing measures. In addition, the commentary considers methodological challenges raised by research aimed at assessing whether a QALY gain by a child should be valued equally to a QALY gain by an adult.

Estimating Cost-Effectiveness Using Alternative Preference-Based Scores and Within-Trial Methods: Exploring the Dynamics of the Quality-Adjusted Life-Year Using the EQ-5D 5-Level Version and Recovering Quality of Life Utility Index

ObjectivesThis study aimed to explore quality-adjusted life-year (QALY) and subsequent cost-effectiveness estimates based on the more physical health–focused EQ-5D 5-level version (EQ-5D-5L) value set for England or cross-walked EQ-5D 3-level version UK value set scores or more mental health recovery-focused Recovering Quality of Life Utility Index (ReQoL-UI), when using alternative within-trial statistical methods. We describe possible reasons for the different QALY estimates based on the interaction between item scores, health state profiles, preference-based scores, and mathematical and statistical methods chosen. MethodsQALYs are calculated over 8 weeks from a case study 2:1 (intervention:control) randomized controlled trial in patients with anxiety or depression. Complete case and with missing cases imputed using multiple-imputation analyses are conducted, using unadjusted and regression baseline-adjusted QALYs. Cost-effectiveness is judged using incremental cost-effectiveness ratios and acceptability curves. We use previously established psychometric results to reflect on estimated QALYs. ResultsA total of 361 people (241:120) were randomized. EQ-5D-5L crosswalk produced higher incremental QALYs than the value set for England or ReQoL-UI, which produced similar unadjusted QALYs, but contrasting baseline-adjusted QALYs. Probability of cost-effectiveness <£30 000 per QALY ranged from 6% (complete case ReQoL-UI baseline-adjusted QALYs) to 64.3% (multiple-imputation EQ-5D-5L crosswalk unadjusted QALYs). The control arm improved more on average than the intervention arm on the ReQoL-UI, a result not mirrored on the EQ-5D-5L nor condition-specific (Patient-Health Questionnaire-9, depression; Generalized Anxiety Disorder-7, anxiety) measures. ConclusionsReQoL-UI produced contradictory cost-effectiveness results relative to the EQ-5D-5L. The EQ-5D-5L’s better responsiveness and “anxiety/depression” and “usual activities” items drove the incremental QALY results. The ReQoL-UI’s single physical health item and “personal recovery” construct may have influenced its lower 8-week incremental QALY estimates in this patient sample.

Comparison of the PROMIS Preference Score (PROPr) and EQ-5D-5L Index Value in General Population Samples in the United Kingdom, France, and Germany

ObjectivesThe Patient-Reported Outcome Measurement Information System (PROMIS) Preference score (PROPr) can be used to assess health state utility (HSU) and estimate quality-adjusted life-years in cost-effectiveness analyses. It is based on item response theory and promises to overcome limitations of existing HSU scores such as ceiling effects. The PROPr contains 7 PROMIS domains: cognitive abilities, depression, fatigue, pain, physical function, sleep disturbance, and ability to participate in social roles and activities. We aimed to compare the PROPr with the 5-level EQ-5D (EQ-5D-5L) in terms of psychometric properties using data from 3 countries. MethodsWe collected PROMIS-29 profile and EQ-5D-5L data from 3 general population samples (United Kingdom = 1509, France = 1501, Germany = 1502). Given that cognition is not assessed by the PROMIS-29, it was predicted by the recommended linear regression model. We compared the convergent validity, known-groups construct validity, and ceiling and floor effects of the PROPr and EQ-5D-5L. ResultsThe mean PROPr (0.48, 0.53, 0.48; P<.01) and EQ-5D-5L scores (0.82, 0.85, 0.83; P<.01) showed significant differences of similar magnitudes (d = 0.34; d = 0.32; d = 0.35; P<.01) across all samples. The differences were invariant to sex, income, occupation, education, and most conditions but not for age. The Pearson correlation coefficients between both scores were r = 0.74, r = 0.69, and r = 0.72. PROPr’s ceiling and floor effects both were minor to moderate. The EQ-5D-5L’s ceiling (floor) effects were major (negligible). ConclusionsBoth the EQ-5D-5L and the PROPr assessed by the PROMIS-29 show high validity. The PROPr yields considerably lower HSU values than the EQ-5D-5L. Consequences for quality-adjusted life-year measurements should be investigated in future research.