scholarly journals Dutch utility weights for the EORTC cancer-specific utility instrument: the Dutch EORTC QLU-C10D

Author(s):  
Femke Jansen ◽  
◽  
Irma M. Verdonck-de Leeuw ◽  
Eva Gamper ◽  
Richard Norman ◽  
...  

Abstract Purpose To measure utilities among cancer patients, a cancer-specific utility instrument called the European Organization for Research and Treatment of Cancer (EORTC) QLU-C10D has been developed based on EORTC quality of life core module (QLQ-C30). This study aimed to provide Dutch utility weights for the QLU-C10D. Methods A cross-sectional valuation study was performed in 1017 participants representative in age and gender of the Dutch general population. The valuation method was a discrete choice experiment containing 960 choice sets, i.e. pairs of QLU-C10D health states, each health state described in terms of the 10 QLU-C10D domains and the duration of that health state. Each participant considered 16 choice sets, choosing their preferred health state from each pair. Utility scores were derived using generalized estimation equation models. Non-monotonic levels were combined. Results Utility decrements were generated for all 10 QLU-C10D domains, with largest decrements for pain (− 0.242), physical functioning (− 0.228), and role functioning (− 0.149). Non-monotonic levels of emotional functioning, pain, fatigue, sleep problems, and appetite loss were combined. No decrement in utility was seen in case of a little or quite a bit impairment in emotional functioning or a little pain. The mean QLU-C10D utility score of the participants was 0.85 (median = 0.91, interquartile range = 0.82 to 0.96). Conclusion Dutch utility decrements were generated for the QLU-C10D. These are important for evaluating the cost-utility of new cancer treatments and supportive care interventions. Further insight is warranted into the added value of the QLU-C10D alongside other utility instruments.

2019 ◽  
Vol 4 (1) ◽  
pp. 238146831984253 ◽  
Author(s):  
Helen McTaggart-Cowan ◽  
Madeleine T. King ◽  
Richard Norman ◽  
Daniel S. J. Costa ◽  
A. Simon Pickard ◽  
...  

Objective. The EORTC QLQ-C30 is widely used for assessing quality of life in cancer. However, QLQ-C30 responses cannot be incorporated in cost-utility analysis because they are not based on general population’s preferences, or utilities. To overcome this limitation, the QLU-C10D, a cancer-specific utility algorithm, was derived from the QLQ-C30. The aim of this study was to obtain Canadian population utility weights for the QLU-C10D. Methods. Respondents from a Canadian research panel expressed their preferences for 16 choice sets in an online discrete choice experiment. Each choice set consisted of two health states described by the 10 QLU-C10D domains plus an attribute representing duration of survival. Using a conditional logit model, responses were converted into utility decrements by evaluating the marginal rate of substitution between each QLU-C10D domain level with respect to duration. Results. A total of 3,363 individuals were recruited. A total of 2,345 completed at least one choice set and 2,271 completed all choice sets. The largest utility decrements were associated with the worse levels of Physical Functioning (−0.24), Pain (−0.18), Role Functioning (−0.15), Emotional Functioning (−0.12), and Nausea (−0.12). The remaining domains and levels had decrements of −0.05 to −0.09. The utility of the worst possible health state was −0.15. Conclusion. Respondents from the general population were most concerned with generic health domains, but Nausea and Bowel Problems also had an impact on the individual’s utility. It is unclear as to whether cancer-specific domains will affect cost-utility analysis when evaluating cancer treatments; this will be tested in the next phase of the study.


Author(s):  
Vitaly Omelyanovskiy ◽  
Nuriya Musina ◽  
Svetlana Ratushnyak ◽  
Tatiana Bezdenezhnykh ◽  
Vlada Fediaeva ◽  
...  

Abstract Purpose The most widely used generic questionnaire to estimate the quality of life for yielding quality-adjusted life years in economic evaluations is EQ-5D. Country-specific population value sets are required to use EQ-5D in economic evaluations. The aim of this study was to establish an EQ-5D-3L value set for Russia. Methods A representative sample aged 18+ years was recruited from the Russia`s general population. Computer-assisted face–to–face interviews were conducted based on the standardized valuation protocol using EQ-Portable Valuation Technology. Population preferences were elicited utilizing both composite time trade-off (cTTO) and discrete choice experiment (DCE) techniques. To estimate the value set, a hybrid regression model combining cTTO and DCE data was used. Results A total of 300 respondents who successfully completed the interview were included in the primary analysis. 120 (40.0%) respondents reported no health problems of any dimension, and 56 (18.7%) reported moderate health problems in one dimension of the EQ‐5D‐3L. Median self-rated health using EQ‐VAS was 80 with IQR 70–90. Comparing cTTO and DCE-predicted values for 243 health states resulted in a similar pattern. This supports the use of hybrid models. The predicted value based on the preferred model for the worst health state “33333” was −0.503. Mobility dimension had the most significant impact on the utility decrement, and anxiety/depression had the lowest decrement. Conclusion Determining a Russian national value set may be considered the first step towards promoting cost-utility analysis use to increase comparability among studies and improve the transferability of healthcare decision-making in Russia.


2019 ◽  
Vol 21 (3) ◽  
pp. 397-407
Author(s):  
Louis S. Matza ◽  
L. Clark Paramore ◽  
Katie D. Stewart ◽  
Hayley Karn ◽  
Minesh Jobanputra ◽  
...  

Abstract Objectives Transfusion-dependent β-thalassemia (TDT) is a genetic disease that affects production of red blood cells. Conventional treatment involves regular red blood cell transfusions and iron chelation, which has a substantial impact on quality of life. While potentially curative, allogeneic hematopoietic stem cell transplantation (allo-HSCT) is associated with risk of complications, including graft-versus-host disease (GvHD). Gene addition therapy, a novel treatment approach, involves autologous transplantation of the patient’s own genetically modified hematopoietic stem cells. The purpose of this study was to estimate utilities associated with treatment approaches for TDT. Methods General population respondents in England valued eight health state vignettes (developed with clinician, patient, and parent input) in time trade-off interviews. Results A total of 207 participants completed interviews (49.8% female; mean age = 43.2 years). Mean (SD) utilities for the pre-transplant health states were 0.73 (0.25) with oral chelation and 0.63 (0.32) with subcutaneous chelation. Mean utilities for the transplant year were 0.62 (0.35) for gene addition therapy, 0.47 (0.39) for allo-HSCT, and 0.39 (0.39) for allo-HSCT with acute GvHD. Post-transplant utilities were 0.93 (0.15) for transfusion independent, 0.75 (0.25) for 60% transfusion reduction, and 0.51 (0.38) for chronic GvHD. Acute and chronic GvHD were associated with significant disutility (acute = − 0.09, p < 0.0001; chronic = − 0.42, p < 0.0001). Conclusions Utilities followed expected patterns, with logical differences between treatment options for TDT and substantially greater utility for transfusion independence than for ongoing treatment involving transfusion and chelation. These utilities may be useful in cost-utility models estimating the value of treatments for TDT.


2018 ◽  
Vol 38 (6) ◽  
pp. 635-645 ◽  
Author(s):  
Franz Ombler ◽  
Michael Albert ◽  
Paul Hansen

The calculation of quality-adjusted life years, as used for cost–utility analysis, depends on the availability of value sets representing people’s preferences with respect to health-related quality of life (HRQoL). A value set consists of HRQoL index values for all health states representable by the particular descriptive system used, of which the EQ-5D (EuroQoL, 5 Dimensions) is by far the most widely used. High correlation coefficients for EQ-5D value sets derived from different samples—across countries and/or using different valuation techniques—are conventionally interpreted as evidence that the people in the respective samples have similar HRQoL preferences. However, EQ-5D value sets—for both versions of the system (EQ-5D-3L and EQ-5D-5L)—contain many inherent rankings of health state values by design. By calculating correlation coefficients for value sets created from random data, we demonstrate that “high” coefficients are artifacts of these inherent rankings, such as median Pearson’s r = 0.783 for the EQ-5D-3L and 0.850 for the EQ-5D-5L instead of zero. Therefore, high correlation coefficients do not necessarily constitute evidence of meaningful associations in terms of similar HRQoL preferences. After calculating significance levels based on our simulations—available as an online resource for other researchers—we find that many high coefficients are not as significant as conventionally interpreted, whereas other coefficients are not significant. These “high” but insignificant correlations are in fact spurious.


2016 ◽  
Vol 37 (3) ◽  
pp. 285-297 ◽  
Author(s):  
Brendan Mulhern ◽  
Nick Bansback ◽  
Arne Risa Hole ◽  
Aki Tsuchiya

Background: Discrete choice experiments incorporating duration can be used to derive health state values for EQ-5D-5L. Yet, methodological issues relating to the duration attribute and the optimal way to select health states remain. The aims of this study were to: test increasing the number of duration levels and choice sets where duration varies (aim 1); compare designs with zero and non-zero prior values (aim 2); and investigate a novel, two-stage design to incorporate prior values (aim 3). Methods: Informed by zero and non-zero prior values, two efficient designs were developed, each consisting of 120 EQ-5D-5L health profile pairs with one of six duration levels (aims 1 and 2). Another 120 health state pairs were selected, with one of six duration levels allocated in a second stage based on existing estimated utility of the states (aim 3). An online sample of 2,002 members of the UK general population completed 10 choice sets each. Differences across the regression coefficients from the three designs were assessed. Results: The zero prior value design produced a model with coefficients that were generally logically ordered, but the non-zero prior value design resulted in a set of less ordered coefficients where some differed significantly. The two-stage design resulted in ordered and significant coefficients. The non-zero prior value design may include more “difficult” choice sets, based on the proportions choosing each profile. Conclusions: There is some indication of compromised “respondent efficiency”, suggesting that the use of non-zero prior values will not necessarily result in better overall precision. It is feasible to design discrete choice experiments in two stages by allocating duration values to EQ-5D-5L health state pairs based on estimates from prior studies.


Blood ◽  
2013 ◽  
Vol 122 (21) ◽  
pp. 1735-1735 ◽  
Author(s):  
Tom Kouroukis ◽  
Darrell White ◽  
Morgan Kruse ◽  
Donna Lawrence ◽  
Cristina Trambitas ◽  
...  

Abstract Introduction The effectiveness of bortezomib for induction treatment prior to ASCT in multiple myeloma (MM) patients has been demonstrated in a number of randomized, open-label phase III trials, including the IFM 2005-01 trial (Harousseau et al., J Clin Oncol 2010;28(30):4621-9). This trial showed that the addition of bortezomib as part of an induction treatment prior to ASCT resulted in statistically significant improvements in post-induction response rates and longer progression-free survival (PFS) compared to a non-bortezomib containing regimen (NBCR). The objective of this study was to assess the cost-utility of a bortezomib-containing regimen (BCR) vs. a NBCR for induction treatment in previously untreated MM patients prior to ASCT from a Canadian public payer perspective, based on the results of the IFM 2005-01 study. Methods A Markov model was developed to estimate the cost-utility over a lifetime horizon (50 years) in previously untreated MM patients undergoing induction and ASCT. The model simulated disease progression of patients with previously untreated MM through three health states: “progression-free”, “progression” and “death”, with all patients beginning in the progression-free state. The PFS and overall survival (OS) curves from the IFM 2005-01 trial were extrapolated beyond the study follow-up period to estimate the timeframe spent in each health state. Each health state was associated with a utility value and direct medical costs. Utilities for the progression-free and progression health states were derived from a previous cost-utility analysis for bortezomib and were 0.81 and 0.645, respectively (Hornberger et al., Eur J Haematol 2010;85(6):484-91). Transition probabilities between health states were estimated by calibrating the model to the PFS and OS curves from the IFM 2005-01 trial. In the base case, transition probabilities beyond the trial follow-up period were conservatively assumed to be equal for both treatment groups. Medical resource utilization was estimated using the IFM 2005-01 trial, and supplemented by published literature and clinical advisors. Clinical advisors also provided input on management of adverse events (> grade 3) and treatment of patients who progressed after induction and ASCT. Resource costs were estimated using Canadian sources ($CAN 2012) and costs and outcomes were discounted at 5% annually. Because patients in each group incurred similar costs (i.e. cost of an ASCT), only incremental costs between the two arms were included in the analysis. One-way sensitivity analyses and probabilistic sensitivity analyses were performed to test the robustness of the model. Results The mean total MM-related cost over the lifetime analysis in the model was $68,800 per patient treated with a BCR and $47,000 per patient treated with a NBCR. Addition of bortezomib to the induction regimen increased costs by $21,700 (see table). Over the model lifetime, a delay in progression with a BCR led to 0.25 years of additional survival compared to a NBCR and a quality-adjusted life-year (QALY) gain of 0.22 years. The incremental cost-utility ratio for induction using a BCR compared to a NBCR approach was $99,200/QALY. Sensitivity analyses identified the major factors impacting the cost-utility ratio as: transition probabilities beyond the trial follow-up period, discounting, utilities and bortezomib costs. The probability of a BCR being cost-effective compared to a NBCR was 43.9% at a threshold of $100,000/QALY. Conclusions A number of phase 3 trials have demonstrated the effectiveness of bortezomib as part of an induction regimen prior to ASCT. This analysis indicates that, from a Canadian perspective, induction treatment with a BCR in previously untreated MM patients prior to ASCT can be cost-effective at conventional decision thresholds with a cost-utility ratio of $99,200/QALY. Disclosures: Kouroukis: Janssen Inc.: Honoraria. White:Janssen Inc.: Consultancy, Honoraria. Kruse:OptumInsight: Employment. Lawrence:OptumInsight: Employment. Trambitas:Janssen Inc.: Employment.


Blood ◽  
2004 ◽  
Vol 104 (11) ◽  
pp. 2205-2205
Author(s):  
Joan E. Wasserman ◽  
William K. Hoots

Abstract Research has shown that disease-specific health-related quality of life (HRQoL) instruments are more responsive than generic instruments to particular disease conditions. However, only a few studies have used disease-specific instruments to measure HRQoL in hemophilia. The goal of this project was to develop a disease-specific utility instrument that measures patient preferences for various hemophilia health states. The visual analog scale (VAS), a ranking method, and the standard gamble (SG), a choice-based method incorporating risk, were used to measure patient preferences. Study participants (n=128) were recruited from the UT/Gulf States Hemophilia and Thrombophilia Center and stratified by age: 0 – 18 years and 19 +. Test retest reliability was demonstrated for both VAS and SG instruments, overall within-subject correlation coefficients were 0.91 and 0.79, respectively. Results showed statistically significant differences in responses between pediatric and adult participants when using the SG (p=.045). However, no significant differences were shown between these groups when using the VAS (p=.636). When responses to VAS and SG instruments were compared, statistically significant differences in both pediatric (p<.0001) and adult (p<.0001) groups were observed. Stratification of the study sample into mild, moderate and severe disease categories yielded no statistically significant differences in patient preference values among the three groups for both instruments (VAS (p =0.578 for mild vs. moderate, p =0.590 for mild vs. severe and p= 0.920 for moderate vs. severe and SG (p= 0.578 for mild vs. moderate, p = 0.590 for mild vs. severe and p=0.920 for moderate vs. severe). The utility measures obtained from this study can be applied in economic evaluations and decision models that analyze the cost/utility of alternative hemophilia treatments. Results derived from the SG indicate that age can influence patients’ preferences regarding their state of health. This may have implications for considering treatment options based on the mean age of the population under consideration. Data from this study also demonstrated that persons with varying severity of disease were able to objectively evaluate a wide range of health states for hemophilia. When assessing HRQoL for a rare disease such as hemophilia, it is important to establish that HRQoL measurements can be applied broadly across the population. Although the VAS and SG independently demonstrated reliability and validity, results indicate that the two measures may not be interchangeable.


Blood ◽  
2014 ◽  
Vol 124 (21) ◽  
pp. 1288-1288 ◽  
Author(s):  
Heather Cameron ◽  
Melissa Thompson ◽  
John-Paul Marino ◽  
Michael Duong ◽  
Ursula Becker ◽  
...  

Abstract BACKGROUND: In Canada, treatment options are limited for patients with chronic lymphocytic leukemia (CLL) where fludarabine-based regimens are considered inappropriate. For these patients, chlorambucil monotherapy is considered a standard treatment option. Obinutuzumab is a novel recombinant humanized and glycoengineered Type II anti-CD20 monoclonal antibody of the IgG1 isotype. Clinical data demonstrate that first line therapy with obinutuzumab + chlorambucil can improve progression-free survival (PFS) compared with chlorambucil alone in CLL patients ineligible for fludarabine-based chemotherapy (29.9 vs. 11.1 months; HR 0.18 (95% CI [0.14; 0.24]), p<0.0001). (Goede et al., 2014; Roche. Data on file; May 2014). Obinutuzumab + chlorambucil also demonstrated an overall survival (OS) benefit versus chlorambucil alone (HR for death, 0.47; 95% CI, 0.29 to 0.76; P=0.0014). (Goede et al., 2014; Roche. Data on file; May 2014). We conducted a cost-effectiveness and cost-utility analysis of obinutuzumab + chlorambucil versus chlorambucil monotherapy from a Canadian healthcare perspective. METHODS: A Markov model was created to estimate the cost-utility of the treatment with obinutuzumab + chlorambucil versus chlorambucil monotherapy over a ten-year time horizon in previously untreated CLL patients ineligible for fludarabine-based chemotherapy. The model simulated patients moving through three health states: “progression-free”, “progression”, and “death”, with all patients beginning in the progression-free state. The progression-free state was divided into sub health states; progression-free with therapy, and progression-free without therapy. Each health state was associated with a utility value and direct medical costs. (Roche. Data on file; April 2014) Transition probabilities from the progression-free health state to the progression state were determined by PFS collected in the CLL11 trial for obinutuzumab + chlorambucil and chlorambucil monotherapy arms (Roche. Data on file; May 2014). Patients who experienced disease progression transitioned to the progression health state where they received second-line therapy and ongoing supportive care. Transition probabilities from the progression-free health state to death were determined based on the treatment specific death rates observed in CLL11. Due to the lack of mature OS data from CLL11 the transition probabilities from the progressed health state to death were determined based on data from the CLL5 trial (Eichhorst et al., 2009). Resource use and costs were estimated using Canadian sources ($CAD 2014), and both costs and outcomes were discounted at 5% annually. The stability of model results was tested using one-way and probabilistic sensitivity analyses. RESULTS: Treatment with obinutuzumab + chlorambucil produced more life years and quality adjusted life years (QALYs) than treatment with chlorambucil alone. The incremental cost was $35,330 for an incremental life years gain (LYG) of 1.038 and an incremental QALY gain of 0.975 (Table 1). These result in an incremental cost per LYG ratio of $34,028 and an incremental cost per QALY gained of $36,246. The results of one-way sensitivity analyses indicated that the model was robust to changes in model inputs, with the most impactful parameters being time horizon, assumptions regarding survival, treatment duration, and exclusion of second-line therapies. A probabilistic sensitivity analysis resulted in a mean ICER of $35,370, with obinutuzumab + chlorambucil having a 94.3% chance of being cost-effective at a willingness to pay threshold of $50,000/QALY, and a 100% chance of being cost-effective at a willingness to pay threshold of $100,000/QALY and $150,000/QALY. Abstract 1288. Table 1. Ten year cost-effectiveness results Treatment Total Costs Total LYs Total QALYs Incremental Costs Incremental LYs Incremental QALYs Cost per LYG Cost per QALY Chlorambucil $22,417 3.971 2.546 Obinutuzumab + Chlorambucil $57,747 5.009 3.521 $35,330 1.038 0.975 $34,028 $36,246 CONCLUSIONS: The results of this analysis demonstrated that improvements in PFS and OS with obinutuzumab + chlorambucil translate into longer term gains in LYs and QALYs. From a Canadian healthcare perspective, first line treatment of CLL patients ineligible for fludarabine based therapies with obinutuzumab + chlorambucil is cost-effective with a cost-utility ratio of $36,246/QALY. Disclosures Cameron: Cornerstone Research Group: Employment. Thompson:Cornerstone Research Group: Employment. Marino:2Hoffmann-La Roche Limited : Employment. Duong:2Hoffmann-La Roche Limited : Employment. Becker:Roche: Employment. Wiesner:4Genentech, Inc. A Member of the Roche Group: Employment.


1993 ◽  
Vol 9 (4) ◽  
pp. 463-478 ◽  
Author(s):  
Erik Nord ◽  
Jeff Richardson ◽  
Kelly Macarounas-Kirchmann

AbstractIn most of the cost-utility literature, quality-adjusted life-year (QALY) gains are interpreted as a measure of social value. Given this interpretation, the validity of different multi-attribute health-state scaling instruments may be tested by comparing the values they provide on the 0–1 QALY scale with directly elicited preferences for person trade-offs between different treatments (equivalence of numbers of different patients treated). Norwegian and Australian public preferences as measured by the person trade-off suggest that the EuroQol Instrument assigns excessively low values to health states. This seems to be even more true of the McMaster Health Classification System. The Quality of Well-being Scale appears to compress states toward the middle of the 0–1 scale. By contrast, the Rosser/Kind index fits reasonably well with directly measured person trade-off data.


2021 ◽  
Author(s):  
Stana Pačarić ◽  
Želimir Orkić ◽  
Ivan Erić ◽  
Tajana Turk ◽  
Goran Kondža ◽  
...  

Background: Breast cancer is the most common cancer in women which affects them emotionally and psychologically. The aim of this research was to examine emotional functioning and self-perception in post mastectomy women. Subjects and methods: This cross-sectional single-center study included 101 women with breast cancer one month and one year after mastectomy. It was conducted using anonymous questionnaires developed by the European Organization for Research and Treatment of Cancer (EORTC): EORTC Quality of Life questionnaire (QLO) - C 30 (version 3), questionnaire with breast cancer module EORTC QLQ BR-23 and a sociodemographic questionnaire.Results: Compared to results one month after mastectomy, in women one year after mastectomy there was significantly less tension (Mann-Whitney U test, p=0.011) and emotional irritability (Mann- Whitney U test, p=0.013), also the memory problems declined (Mann-Whitney U test, p=0.008). Discomfort with hair loss affected all parameters except concentration problems. The participants felt less physical attractive (p=0.647), were worried (p=0.645) and less feminine due to illness (p=0.638). A year after surgery there was no connection between anxiety and observed parameters. Conclusion: Breast cancer affects emotional functioning and self-perception of women especially in early postoperative period and during cancer treatment. A year after surgery there was no more connection between anxiety and hair loss discomfort. Patients need medical, social and psychological support during and after breast cancer treatment.


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