Quality-adjusted Life Year Estimates Research Articles

18F FDG PET/MRI with hepatocyte-specific contrast agent for M staging of rectal cancer: a primary economic evaluation

PurposeRectal cancer is one of the most frequent causes of cancer-related morbidity and mortality in the world. Correct identification of the TNM state in primary staging of rectal cancer has critical implications on patient management. Initial evaluations revealed a high sensitivity and specificity for whole-body PET/MRI in the detection of metastases allowing for metastasis-directed therapy regimens. Nevertheless, its cost-effectiveness compared with that of standard-of-care imaging (SCI) using pelvic MRI + chest and abdominopelvic CT is yet to be investigated. Therefore, the aim of this study was to analyze the cost-effectiveness of whole-body 18F FDG PET/MRI as an alternative imaging method to standard diagnostic workup for initial staging of rectal cancer.MethodsFor estimation of quality-adjusted life years (QALYs) and lifetime costs of diagnostic modalities, a decision model including whole-body 18F FDG PET/MRI with a hepatocyte-specific contrast agent and pelvic MRI + chest and abdominopelvic CT was created based on Markov simulations. For obtaining model input parameters, review of recent literature was performed. Willingness to pay (WTP) was set to $100,000/QALY. Deterministic sensitivity analysis of diagnostic parameters and costs was applied, and probabilistic sensitivity was determined using Monte Carlo modeling.ResultsIn the base-case scenario, the strategy whole-body 18F FDG PET/MRI resulted in total costs of $52,186 whereas total costs of SCI were at $51,672. Whole-body 18F FDG PET/MRI resulted in an expected effectiveness of 3.542 QALYs versus 3.535 QALYs for SCI. This resulted in an incremental cost-effectiveness ratio of $70,291 per QALY for PET/MRI. Thus, from an economic point of view, whole-body 18F FDG PET/MRI was identified as an adequate diagnostic alternative to SCI with high robustness of results to variation of input parameters.ConclusionBased on the results of the analysis, use of whole-body 18F FDG PET/MRI was identified as a feasible diagnostic strategy for initial staging of rectal cancer from a cost-effectiveness perspective.

Economic evaluation of point of care universal newborn screening for glucose-6-Phosphate dehydrogenase deficiency in United States

Background and objectives Glucose-6-Phosphate Dehydrogenase (G6PD) deficiency is frequent inherited enzymopathy that poses potentially preventable risk for extreme hyperbilirubinemia (EHB) which can, rarely, lead to acute bilirubin encephalopathy, childhood kernicterus and death. We aimed to estimate quality adjusted life years (QALY) lost due to G6PD deficiency associated with EHB and economic costs to best estimate value of universal pre-discharge screening. Methods We did a cost utility analysis for US birth cohort utilizing pre-discharge screening decision tree model to estimate population burden and EHB outcomes, based on literature search and expert opinions. Employing human capital approach, we measured health benefits in terms of QALYs and economic losses. QALYs and costs were discounted at 3%; one-way sensitivity analysis was used for decision variables. Results We determined for USA live births of 3.86 million in 2017, 1464 cases of EHB were estimated to be due to G6PD deficiency (CI 95%; range: 1270–1656) and contributed 2 deaths (CI 95%; range 1.3–3.2) and 14 (CI 95%; range: 9.1–21.5) cases of kernicterus. Over lifetime horizon, the model predicted undiscounted and discounted gains of 165 (102–252) life years; 241 (183–433) QALYs and 16 (9.9–24.5) life years; 89 (67.9–160.5) QALYs, respectively. Assuming 50% effectiveness, benefit cost ratios ranged from 0.19 to 3.42 for diverse operational settings. The cost to prevent a single case of kernicterus was $2.7 to 6.8 million per annum with cost per QALY gained at $35,946 to $89,159. Conclusion At incremental cost-effective threshold of $100,000/life year, pre-discharge screening would be expected to prove cost effective in preventing EHB related morbidities and mortality attributed to G6PD deficiency.

Impact of Pharmacogenomic Information on Values of Care and Quality of Life Associated with Codeine and Tramadol-Related Adverse Drug Events

ObjectiveTo assess the potential impact of Pharmacogenomic (PGx) variation in cytochrome P450 2D6 (CYP2D6) enzyme function, using loss in quality-adjusted life years (QALYs) associated with treatment problems, and the willingness to pay to avoid treatment problems from patients’ and payers’ perspectives. Patients and MethodsThe study included patients prescribed tramadol or codeine, or both, between January 1, 2005, and December 31, 2017. Demographic information and adverse drug events, including adverse drug events and poor pain control, were collected from the electronic health records using natural language processing techniques and review by trained abstractors. Patients’ willingness to pay and QALY estimates were based on comprehensive literature review. The CYP2D6 phenotypes were divided into 4 groups: ultra-rapid metabolizers, normal metabolizers, intermediate metabolizers, and poor metabolizers. ResultsAmong the 2860 identified patients, 63 (2%) were ultrarapid metabolizers, 1449 (50%) were normal metabolizers, 1155 (40%) were intermediate metabolizers, and 193 (7%) were poor metabolizers. The patients’ average estimated willingness-to-pay value to avoid treatment problems was $23 per month; poor metabolizers developed problems with the highest estimated willingness-to-pay value ($32 per month). The mean QALY loss among all patients was 0.024 QALYs (8.8 healthy days); poor metabolizers had the highest loss (0.027 QALYs, 9.9 healthy days). ConclusionPatients with various phenotypes developed different treatment problem profiles. Poor CYP2D6 metabolizers developed problems with highest willingness to pay, and they might potentially benefit most from PGx-guided treatment and problem prevention.

CL2 Measuring Health-Related Quality of Life in the Context of Episodic Conditions

Cost-effectiveness analyses require the measurement of health-related quality of life (HRQoL) to estimate quality–adjusted life years (QALYs). Challenges with measuring HRQoL arise in the context of episodic conditions such as epilepsy, because symptoms may not have occurred within the recall period of the HRQoL instrument. The aim of this paper is to explore whether HRQoL measured at regular time intervals correctly reflects the HRQoL of patients with episodic conditions, using the example of refractory epilepsy. Follow-up data from the first year of the EPISODE-study on the (cost-)effectiveness of seizure dogs were used. In the stepped-wedge randomised controlled trial HRQoL is collected in 23 respondents at month 3, 6 and 9 with the EQ-5D and seizure frequency is recorded continuously using a seizure diary. Descriptive statistics, Student’s t-tests and graphical displays were used to compare the average seizure count on the day of completion of the EQ-5D with the average seizure counts over the preceding 7, 14 and 28 days. The average seizure count on the day of EQ-5D completion was lower than the average seizure count for the preceding period (2.88±5.10 vs. respectively 3.76±5.62, 3.81±5.69 and 3.75±5.39, p < 0.01). The same pattern was seen at the individual measurements. Graphical displays confirm the hypothesis that patients appear to complete the questionnaire on a day that seizure count is relatively low. When HRQoL is measured at regular time intervals, people with an episodic condition such as epilepsy appear to complete these measurements on relatively good days. Consequently, the HRQoL is potentially overestimated in such populations, especially for bad days. When treatment is targeted at reducing the frequency or the intensity of such bad days, measuring HRQoL at regular time intervals might lead to underestimation of the effectiveness of treatment, and therefore to biased estimates of cost-effectiveness.

PIN114 Estimation of Quality-Adjusted Life YEARS (QALY) Losses Associated with COVID-19 Deaths in Ukraine

Objectives: This study aims to assess possible loss of quality-adjusted life years (QALY) associated with premature death from COVID-19 in Ukraine Methods: Based on the adaptation of the previously published model (Briggs et al, 2020), a health economics markov model was built to estimate the QALY burden of COVID-19 in Ukraine Local Life Tables, age specific COVID-19 mortality rates and the potential impact of comorbidities (1 - no exposure, 2 - moderate exposure and 3 - severe exposure) were taken into account Age structure of mortality from COVID-19 in Ukraine is based on data provided by the Center of Public Health of the Ministry of Health of Ukraine (08 05 2020) Results: Model estimated that COVID-19 public health burden is characterized with a substantial loss of QALY in all three scenarios, which differ by the level of impact of comorbidities on mortality rates According to the three scenarios QALY losses per person in Ukraine were within 6-13 years per person, in the United Kingdom these figures were in the range of 4-8 years, which represents a 55 2% higher QALY loss per person in Ukraine compared to the United Kingdom) The annual COVID-19 associated QALY loss due to mortality in Ukraine ranges from 2295 to 4525 QALY, depending on the impact of comorbidities Conclusions: The study showed that high QALY losses in Ukraine are associated with the high mortality from COVID-19 in the relatively young age QALY is shown to be an important metric to consider in the selection of healthcare programmes and pharmaceutical provision during the pandemic

Assessing the Impact of EQ-5D Country-specific Value Sets on Cost-utility Outcomes.

To assess the impact of EQ-5D country-specific value sets on cost-utility outcomes. Data from 2 randomized controlled trials on low back pain (LBP) and depression were used. 3L value sets were identified from the EuroQol Web site. A nonparametric crosswalk was employed for each tariff to obtain the likely 5L values. Differences in quality-adjusted life years (QALYs) between countries were tested using paired t tests, with United Kingdom as reference. Cost-utility outcomes were estimated for both studies and both EQ-5D versions, including differences in QALYs and cost-effectiveness acceptability curves. For the 3L, QALYs ranged between 0.650 (Taiwan) and 0.892 (United States) in the LBP study and between 0.619 (Taiwan) and 0.879 (United States) in the depression study. In both studies, most country-specific QALY estimates differed statistically significantly from that of the United Kingdom. Incremental cost-effectiveness ratios ranged between &OV0556;2044/QALY (Taiwan) and &OV0556;5897/QALY (Zimbabwe) in the LBP study and between &OV0556;38,287/QALY (Singapore) and &OV0556;96,550/QALY (Japan) in the depression study. At the NICE threshold of &OV0556;23,300/QALY (≈£20,000/QALY), the intervention's probability of being cost-effective versus control ranged between 0.751 (Zimbabwe) and 0.952 (Taiwan) and between 0.230 (Canada) and 0.396 (Singapore) in the LBP study and depression study, respectively. Similar results were found for the 5L, with extensive differences in ICERs and moderate differences in the probability of cost-effectiveness. This study indicates that the use of different EQ-5D country-specific value sets impacts on cost-utility outcomes. Therefore, to account for the fact that health state preferences are affected by sociocultural differences, relevant country-specific value sets should be used.

Alternative Approaches to Quality-Adjusted Life-Year Estimation Within Standard Cost-Effectiveness Models: Literature Review, Feasibility Assessment, and Impact Evaluation.

The quality-adjusted life-year (QALY) has been long debated, but alternative estimation approaches have not been comprehensively evaluated. Our objective was to identify alternatives, characterize them by implementation feasibility, and evaluate the impact of implementing feasible options in cost-effectiveness models developed for the Institute for Clinical and Economic Review reports. We conducted a literature review combining keywords relating to QALYs, methodology alternatives, and cost-effectiveness in PubMed, EconLit, Web of Science, and MEDLINE. Articles that discussed alternatives to the conventional QALY were included. Alternatives were characterized by type, data availability, calculation burden, and overall implementation feasibility. The subset of feasible alternatives, that is, sufficient data and methodology compatible with incorporation into common modeling approaches, were evaluated according to impact on incremental QALYs, incremental net monetary benefit (iNMB), intervention rankings, and proportion of interventions with a positive iNMB. We identified 28 articles discussing 9 alternatives. Feasible alternatives were using patient preference (PP) data; equity weighting according to baseline utility, fair innings, or proportional QALY shortfall; and the equal value of life-years-gained approach. All alternatives affected the incremental QALY and iNMB outcomes, rankings, and proportion of interventions with a positive iNMB. The PP alternative had the largest and most consistent impact. The PP impact on the proportion of interventions with a positive iNMB, was in the negative direction. Our work is the first comprehensive evaluation of proposed alternatives to the conventional QALY. We found robust literature but few options that were feasible to be implemented in current healthcare decision-making processes.

An exploration of methods for obtaining 0\u2009=\u2009dead anchors for latent scale EQ-5D-Y values

ObjectivesDiscrete choice experiments (DCEs) can be used to obtain latent scale values for the EQ-5D-Y, but these require anchoring at 0 = dead to meet the conventions of quality-adjusted life year (QALY) estimation. The primary aim of this study is to compare four preference elicitation methods for obtaining anchors for latent scale EQ-5D-Y values.MethodsFour methods were tested: visual analogue scale (VAS), DCE (with a duration attribute), lag-time time trade-off (TTO) and the location-of-dead (LOD) approach. In computer-assisted personal interviews, UK general public respondents valued EQ-5D-3L health states from an adult perspective and EQ-5D-Y health states from a 10-year-old child perspective. Respondents completed valuation tasks using all four methods, under both perspectives.Results349 interviews were conducted. Overall, respondents gave lower values under the adult perspective compared to the child perspective, with some variation across methods. The mean TTO value for the worst health state (33333) was about equal to dead in the child perspective and worse than dead in the adult perspective. The mean VAS rescaled value for 33333 was also higher in the child perspective. The DCE produced positive child perspective values and negative adult perspective values, though the models were not consistent. The LOD median rescaled value for 33333 was negative under both perspectives and higher in the child perspective.DiscussionThere was broad agreement across methods. Potential criteria for selecting a preferred anchoring method are presented. We conclude by discussing the decision-making circumstances under which utilities and QALY estimates for children and adults need to be commensurate to achieve allocative efficiency.

Estimation of quality-adjusted life years (QALY) losses associated with COVID-19 deaths in Ukraine

Резюме. Цель — оценка вероятных потерь лет жизни с поправкой на качество (QALY), связанных с преждевременной смертью от COVID-19 в Украине, на основе адаптации международной методологии (Briggs A., 2020a; b) и стандартных методов с использованием таблиц смертности и учетом возрастной структуры, а также потенциального влияния сопутствующих заболеваний в локальных условиях. Объект и методы исследования. Адаптированы входные параметры модели, базово представленные данными Великобритании, к украинским локальным условиям на основе опубликованных таблиц смертности и возрастной структуры смертности от COVID-19 в Украине с использованием программы «Microsoft Excel». Возрастная структура смертности от COVID-19 представлена по данным Центра общественного здоровья Министерства здравоохранения Украины (по состоянию на 08.05.2020 г.). Результаты. Смерть от COVID-19 в Украине связана со значительной потерей QALY по трем сценариям — от 6 до 13 лет потерянных лет качественной жизни. Потери вследствие смертности от COVID-19 в Украине составляют от 2295 до 4525 QALY на популяцию в зависимости от влияния сопутствующих заболеваний. Выводы. В Украине на одного человека теряется на 55,2% больше QALY по сравнению с Великобританией, что обусловлено разной возрастной структурой смертности от COVID-19. Следовательно, в странах с невысокой заболеваемостью показатель потери QALY является важным параметром, который требует учета для совершенствования оказания медицинской помощи и фармацевтического обеспечения в период пандемии.

Affording Immune Checkpoint Inhibitors (ICI) in the treatment of advanced non-small cell lung cancer (NSCLC): Argentinean National Cancer Institute (ANCI) perspective.

e19404 Background: ICI agents proved clinical effectiveness in metastatic NSCLC; irrespectively PDL1 expression, but they increase costs considerably. Thus its cost-effectiveness (CE) needs to be established in low and middle-income countries. We aimed to assess CE of Pembrolizumab plus carboplatin and pemetrexed (PCP) vs. carboplatin and pemetrexed (CP) in the first-line setting for non-squamous NSCLC, according to PDL1expresion, in Argentinian public healthcare system (APHS). Methods: Clinical outcomes, utilities, and transition probabilities were collected from the available literature (Keynote 189/024) after a systematic review. The certainty of evidence according to GRADE methodology was qualified as high. A Markov model was developed. Model outcomes were measured in quality adjusted-life-years (QALY) and CE was estimated as the incremental cost-effectiveness ratio (ICER) over a 20 years time horizon. Only direct costs were considered from a public price database of the drugs accounted in the model. The WHO ICER threshold, defined as 1-3 GDP per capita, was selected to established CE. Sensitivity analyses were performed to assess the robustness of results. Results: In PDL1≥ 50 % subgroup, PCP increased life expectancy of patients by 1.06 QALY for an incremental cost of USD 71.551 vs. CP with an ICER of 67.352 per QALY. An indirect comparison of PCP vs. Pembolizumab monotherapy found no differences in QALY estimates, however, it was associated with an incremental cost of USD 5.516. QALY estimate for PDL1 1-49% was 0.73 with an ICER of USD 85.984/ QALY gained. PDL1 &lt; 1% obtained a QALY of 0.42 related to the largest ICER (USD 97.095) for the same comparison. The sensitivity analyses using a threshold between 1- 3 Argentinian GDP per capita in 2018 (USD 11.8602) found that PCP has no chance of being considered CE in untreated NSCLC patients. If a 6 GDP threshold is adopted in PDL1≥ 50 %, PCP would have a 50% possibility of being CE. Conclusions: This model was part of the decision-making process in the development of treatment guidelines for metastatic NSCLC in the ANCI. PCP cannot be considered acceptable for the APHS. Strategies to reduce costs are mandatory to improve affordability in our region.

Cost-Utility Analysis of rhBMP-2 Use in Adult Spinal Deformity Surgery.

Economic modeling of data from a multicenter, prospective registry. The aim of this study was to analyze the cost utility of recombinant human bone morphogenetic protein-2 (BMP) in adult spinal deformity (ASD) surgery. ASD surgery is expensive and presents risk of major complications. BMP is frequently used off-label to reduce the risk of pseudarthrosis. Of 522 ASD patients with fusion of five or more spinal levels, 367 (70%) had at least 2-year follow-up. Total direct cost was calculated by adding direct costs of the index surgery and any subsequent reoperations or readmissions. Cumulative quality-adjusted life years (QALYs) gained were calculated from the change in preoperative to final follow-up SF-6D health utility score. A decision-analysis model comparing BMP versus no-BMP was developed with pseudarthrosis as the primary outcome. Costs and benefits were discounted at 3%. Probabilistic sensitivity analysis was performed using mixed first-order and second-order Monte Carlo simulations. One-way sensitivity analyses were performed by varying cost, probability, and QALY estimates (Alpha = 0.05). BMP was used in the index surgery for 267 patients (73%). The mean (±standard deviation) direct cost of BMP for the index surgery was $14,000 ± $6400. Forty patients (11%) underwent revision surgery for symptomatic pseudarthrosis (BMP group, 8.6%; no-BMP group, 17%; P = 0.022). The mean 2-year direct cost was significantly higher for patients with pseudarthrosis ($138,000 ± $17,000) than for patients without pseudarthrosis ($61,000 ± $25,000) (P < 0.001). Simulation analysis revealed that BMP was associated with positive incremental utility in 67% of patients and considered favorable at a willingness-to-pay threshold of $150,000/QALY in >52% of patients. BMP use was associated with reduction in revisions for symptomatic pseudarthrosis in ASD surgery. Cost-utility analysis suggests that BMP use may be favored in ASD surgery; however, this determination requires further research. 2.

Case manager–assisted rehabilitation for lumbar spinal fusion patients: an economic evaluation alongside a randomized controlled trial with two-year follow-up

Objective: To examine the cost-effectiveness of case manager–assisted rehabilitation as an add-on to usual physical rehabilitation after lumbar spinal fusion, given the lack of any clinical benefits found on analysing the clinical data. Design: Economic evaluation alongside a randomized controlled trial with two-year follow-up. Setting: Patients from the outpatient clinics of a university hospital and a general hospital. Subjects: A total of 82 lumbar spinal fusion patients. Interventions: Patients were randomized one-to-one to case manager–assisted rehabilitation programme as an add-on to usual physical rehabilitation or to usual physical rehabilitation. Main measures: Oswestry Disability Index and EuroQol 5-dimension. Danish preference weights were used to estimate quality-adjusted life years. Costs were estimated from micro costing and national registries. Multiple imputation was used to handle missing data. Costs and effects were presented with means (95% confidence interval (CI)). The incremental net benefit was estimated for a range of hypothetical values of willingness to pay per gain in effects. Results: No impact of case manager–assisted rehabilitation on the Oswestry Disability Index or estimate quality-adjusted life years was observed. Intervention cost was Euros 3984 (3468; 4499), which was outweighed by average reductions in inpatient resource use and sickness leave. A cost reduction of Euros 1716 (–16,651; 20,084) was found in the case manager group. Overall, the probability for the case manager–assisted rehabilitation programme being cost-effective did not exceed a probability of 56%, regardless of willingness to pay. Sensitivity analysis did not change the conclusion. Conclusion: This case manager–assisted rehabilitation programme was unlikely to be cost-effective.

Identification of the Most Cost-effective Position of Vedolizumab Among the Available Biologic Drugs for the Treatment of Ulcerative Colitis.

There are limited data on the most cost-effective sequencing of biologics for ulcerative colitis [UC]. We used Markov modelling to identify the most cost-effective position for vedolizumab among biologics for steroid-dependent UC, with a base-case of a 35-year-old male. We assessed three treatment algorithms, with vedolizumab use: prior to an initial anti-tumour necrosis factor alpha [anti-TNFα] and azathioprine [Algorithm 1]; prior to a second anti-TNF and azathioprine [Algorithm 2]; and prior to colectomy [Algorithm 3]. The initial anti-TNF could be either infliximab or adalimumab. Transition probabilities, costs, and quality-adjusted life-year estimates were derived from published estimates, Medicare, and the Nationwide Inpatient Sample. Primary analyses included 100 trials of 100 000 individuals over 1 year, with a willingness-to-pay threshold of US$100,000. Multiple sensitivity analyses were conducted to assess our findings. From a population perspective, when both infliximab and adalimumab are available, vedolizumab was preferred as the first biologic if ≥14% of initial anti-TNF use was adalimumab. If infliximab is the primary biologic, vedolizumab use after infliximab [Algorithm 2] and prior to adalimumab was the most cost-effective strategy. All models were sensitive to biologic pricing. This simulation demonstrated that the most cost-effective strategy in UC depends on the proportion of patients using adalimumab as the initial anti-TNF. If adalimumab was ≥14%, vedolizumab was preferred as the first biologic. When only infliximab was available for first-line therapy, the most cost-effective position of vedolizumab was prior to cycling to adalimumab.

The construct validity and responsiveness of the EQ-5D-5L, AQL-5D and a bespoke TTO in acute asthmatics

PurposeMeasuring quality of life in acute asthmatics is challenging, especially when asthma attacks can occur sporadically. Several questionnaires can be used to measure quality of life in this patient group; however, psychometric testing is limited on questionnaires that can be used to estimate Quality Adjusted Life years. The objective of this study is to assess the construct validity (convergent and discriminative validity) and responsiveness of the EuroQol-5-Dimensions 5-Level (EQ-5D-5L), Asthma Quality of Life Utility Index-5 Dimensions (AQL-5D) and Time Trade-Off (TTO) in acute asthma patients.MethodsData from a prospective cohort study were used to test the validity and responsiveness of the EQ-5D-5L, AQL-5D and TTO in asthma patients who were recruited from UK accident & emergency departments or hospital wards. The spearman’s rank correlation coefficient, the Kruskal–Wallis test statistic and the standardized response mean were used to test for convergent validity, discriminative validity and responsiveness, respectively.ResultsOne hundred and twenty-one participants were included in the available case analysis. The EQ-5D-5L and AQL-5D showed moderate to strong correlations for convergent validity at baseline, week 4 and week 8. The AQL-5D and TTO showed moderate correlations at week 4 and week 8. No statistical significance was observed for discriminative validity at baseline. Both the EQ-5D-5L and the AQL-5D also showed that they were sensitive to change for the recovery responses.ConclusionsThe EQ-5D-5L and AQL-5D showed stronger construct validity and responsiveness compared to the TTO. Therefore, both the EQ-5D-5L and AQL-5D should be considered for use in future economic evaluations.

PNS394 DEVELOPMENT OF A MULTIPLICATIVE MULTI-ATTRIBUTE AND SINGLE-ATTRIBUTE UTILITY FUNCTION FOR THE HUI3 IN JAPAN

The Health Utilities Index Mark 3 (HUI3) is a generic multiattribute preference-based measure of health status and health-related quality of life that is widely used as an outcome measure in clinical studies, in population health surveys, in the estimation of quality-adjusted life years. We aimed to present a multi-attribute utility function and eight single-attribute utility functions for the HUI3 system based on community preferences in Japan. Two preference surveys were conducted. One, the modeling survey, collected preference scores for the estimation of the utility functions. The other, the direct survey, provided independent scores to assess the predictive validity of the utility functions. Preference measures included value scores obtained on the Feeling Thermometer and standard gamble utility scores obtained using the Chance Board. We recruited 1,043 respondents (aged 20–79) from the general population, stratified by gender and age group, from five Japanese cities. Estimates were obtained for eight single-attribute utility functions and an overall multi-attribute utility function (MAUF). The Japanese HUI3 MAUF is u=1.016*(b1*b2*b3*b4*b5*b6*b7*b8)-0.016. The minimum predicted multi-attribute utility score was -0.002. The intraclass correlation coefficient between directly measured utility scores and scores generated by the multi-attribute function for 53 health states was 0.742. The HUI3 scoring function was developed in Japan has strong theoretical and empirical foundations. It seems to perform well in predicting directly measured scores for health technology assessment in Japan.

Modeling the Economic Impact of Interventions for Older Populations with Multimorbidity: A Method of Linking Multiple Single-Disease Models.

Introduction. Individuals from older populations tend to have more than 1 health condition (multimorbidity). Current approaches to produce economic evidence for clinical guidelines using decision-analytic models typically use a single-disease approach, which may not appropriately reflect the competing risks within a population with multimorbidity. This study aims to demonstrate a proof-of-concept method of modeling multiple conditions in a single decision-analytic model to estimate the impact of multimorbidity on the cost-effectiveness of interventions. Methods. Multiple conditions were modeled within a single decision-analytic model by linking multiple single-disease models. Individual discrete event simulation models were developed to evaluate the cost-effectiveness of preventative interventions for a case study assuming a UK National Health Service perspective. The case study used 3 diseases (heart disease, Alzheimer's disease, and osteoporosis) that were combined within a single linked model. The linked model, with and without correlations between diseases incorporated, simulated the general population aged 45 years and older to compare results in terms of lifetime costs and quality-adjusted life-years (QALYs). Results. The estimated incremental costs and QALYs for health care interventions differed when 3 diseases were modeled simultaneously (£840; 0.234 QALYs) compared with aggregated results from 3 single-disease models (£408; 0.280QALYs). With correlations between diseases additionally incorporated, both absolute and incremental costs and QALY estimates changed in different directions, suggesting that the inclusion of correlations can alter model results. Discussion. Linking multiple single-disease models provides a methodological option for decision analysts who undertake research on populations with multimorbidity. It also has potential for wider applications in informing decisions on commissioning of health care services and long-term priority setting across diseases and health care programs through providing potentially more accurate estimations of the relative cost-effectiveness of interventions.

Erectile dysfunction management after failed phosphodiesterase-5-inhibitor trial: a cost-effectiveness analysis.

To evaluate the cost-effectiveness of alternate erectile dysfunction (ED) management options after failed first line phosphodiesterase-5-inhibitors (PDE5-I). An empiric, repetitive decision tree analysis model was constructed using literature review and expert clinical judgement. This assessed the expected costs and quality adjusted life years (QALYs) of decision alternatives over a 10-year period. The model incorporated interventions including alternate PDE5-Is, intracorporal injections (ICI) with alprostadil or trimix (alprostadil, phentolamine, and papaverine), and inflatable penile prosthesis placement (IPP) and included respective risks of failure, subsequent interventions, and other complications (including priapism risk). Average model QALY estimates obtained from the literature were as follows: ED =0.56, successful alternate PDE5-I =0.70, successful ICI =0.70, and successful IPP =0.78. Cost data were calculated from a high-volume academic center and published manufacturer data. Over the 10-year period, IPP placement was the most cost-effective management option per preserved QALY (QALY =7.82, cost =$22,009/10 years) as compared to ICI alprostadil (QALY =8.51, cost =$62,890/10 years), ICI trimix (QALY =8.47, cost =$48,617/10 years) and alternate PDE5-I (QALY =7.73, $52,883/10 years). Using expert opinion and published utility, cost, and complication data in a decision analysis, we demonstrated that IPP placement is the most cost-effective ED intervention following failed initial PDE5-I over a 10-year period as compared to alternate treatment options. Such cost-effectiveness outcomes may be used in ED management counseling.

Integration of Pharmacometrics and Pharmacoeconomics to Quantify the Value of Improved Forgiveness to Nonadherence: A Case Study of Novel Xanthine Oxidase Inhibitors for Gout.

Linked pharmacometric and pharmacoeconomic models provide a structured approach for assessing the value of candidate drugs in development. The aim of this study was to assess the utility of such an approach for identifying the properties of xanthine oxidase inhibitors (XOi) providing improved forgiveness to nonadherence and estimate the maximum reimbursement price. The pharmacometric and pharmacoeconomic models were used to simulate the time course of serum uric acid concentrations and estimate quality-adjusted life years and costs for the XOi febuxostat and a range of hypothetical analogues. Compounds with reduced clearance or increased potency were more forgiving to missed doses, however, even following relatively large changes in these properties the predicted maximum reimbursement prices represented an increase of only 19% above febuxostat 80mg. Linked pharmacometric and pharmacoeconomic modeling methods have the potential to inform early drug development by providing an indication of pricing options that may permit reimbursement.

Investigating the relative value of health and social care related quality of life using a discrete choice experiment

A key outcome in the evaluation of health technologies is the quality adjusted life year (QALY) which is often estimated using health measures such as the EuroQol instruments (EQ-5D-3L and EQ-5D-5L). The impacts of many interventions extend beyond a narrow definition of health to include non-health impacts such as social care related dimensions of quality of life (QoL). This means that there are circumstances where the QALY does not capture the full value of an intervention. In response to this, instruments with a wider measurement framework, such as the Adult Social Care Outcomes Toolkit (ASCOT), which measures social care related QoL, have been developed. Given the range of instruments available, it is important that decision-makers have tools to assess value for money comprehensively and consistently. To date, preference elicitation of different aspects of QoL combined within the same valuation procedure has not been tested. We investigate the relationship between health and social care aspects of QoL when assessed jointly by combining EQ-5D-5L and ASCOT in an online discrete choice experiment (DCE). In July 2016, 975 respondents recruited from internet panels completed 15 choice sets from an underlying design of 300. Conditional logit regression was used to estimate coefficient decrements for each attribute and examine their relative magnitude. Latent class and mixed logit modelling were used to understand preference heterogeneity. The results suggest trading across health and social care aspects indicated by coefficient estimates of differing magnitude. Dimensions with the largest disutility include four from EQ-5D-5L and one from ASCOT. There is evidence of preference heterogeneity at more severe dimension levels. We have used an established method to test the joint valuation of concepts measuring different aspects of QoL. The results have implications for the aspects of QoL that are included in QALY estimation and used in resource allocation decision-making.

PMU1 MEASUREMENT HEALTH OUTCOMES ASSOCIATED WITH MEDICINES AT A NATIONAL LEVEL

Discussion on medicines provision at a national level focuses on overall expenditure and cost-containment measures. Expenditure is a critical metric and every healthcare system has a finite budget. However, measurement of alternative indicators can provide a method to benchmark or improve medicines provision. This research examined the practicality of providing national level estimates of the following health outcomes metrics for Ireland; number of patients treated, Quality Adjusted Life Year (QALY) gain and Life Year Gain (LYG). A pilot study was undertaken using the top 10 medicine by total drug expenditure in the Pfizer product portfolio in Ireland for 2017. This information was obtained from a supply database maintained by IQVIA (formerly IMS Health). The quantities of Pfizer medicines supplied were translated into a patient number estimate based on the primary licensed indication and approved posology. The number of patients treated was then matched with LYG and QALY gains associated with the treatment the primary indication for each medicine. Previously published economic evaluation and HTA reports provided required QALY and LYG input data. An estimate of the number of patients treated was available for all 10 medicines examined. QALY estimates were possible for 9 agents. LYG outcomes were non-quantifiable for 7 of the medicines examined. Health outcomes measurements were most difficult to provide for medicines categorised as anti-infectives. Patients treated and QALY were viable outcomes to measure. Application of this type of assessment could provide a useful method to examine the implications of payer interventions and national level drugs policy. The methods described are not without limitation and development of patient electronic healthcare records together with utilisation data would reduce the assumptions around adherence and duration of treatment required for calculations.