scholarly journals Systematic review of health state utility values used in pharmacoeconomic evaluations for chronic hepatitis C: impact on cost-effectiveness results

Author(s):  
Ru Han ◽  
Clément François ◽  
Mondher Toumi

AbstractBackgroundHealth state utility values (HSUVs) identified from utility elicitation studies are widely used in pharmacoeconomic evaluations for chronic hepatitis C (CHC) and are particularly instrumental in health technology assessment (HTA) evaluation like the National Institute for Health and Clinical Excellence (NICE).ObjectiveThe objective of this study is to identify HSUVs used in cost-utility analyses (CUAs) for CHC in Europe and evaluate the impact of HSUVs selection on cost-effectiveness results in terms of incremental cost per quality-adjusted life-year (QALY) gained (ICER).MethodsA systematic search of pharmacoeconomic evaluations for CHC was updated in Medline and Embase from the period of 2012-2017 to the period of 2017-2020. Data on health states, HSUVs and utility elicitation studies were extracted. The difference in HSUVs of the same health state in different CUAs and the difference between HSUVs of one health state and of the interlink health state in the same CUAs were calculated. A quality assessment was performed to evaluate the selection of HSUVs in CUAs. Sets of HSUVs identified were used in a re-constructed CUA model to assess the impact on ICER.ResultsTwenty-six CUAs conducted in European countries and referring to 17 utility elicitation studies were included. The difference in HSUVs of the same health states in different CUAs ranged from 0.021 (liver transplant) to 0.468 (decompensated cirrhosis). The difference between HSUVs of one health state and of the interlink health state of next disease severity level was calculated between health state of F0-F1/mild and F2-F3/moderate (n=11, 0.040 to 0.110), F2-F3/moderate and F4/compensated cirrhosis (n=18, 0.027 to 0.130), compensated cirrhosis and decompensated cirrhosis (n=22, 0.020 to 0.100), decompensated cirrhosis and hepatocellular carcinoma (n=24, 0.000 to 0.200), hepatocellular carcinoma and liver transplant in the first year (n=17, −0.329 to 0.170) and liver transplant in the first year and in subsequent years (n=17, −0.340 to 0.000). The utility elicitation study selected by most CUAs (n=11)was recommended as the source of HSUVs, as least for the CUAs conducted in the UK, based on the results of quality assessment. Seven sets of HSUVs were generated to fit the re-constructed model and changed the results of incremental analysis from being cost-effective to not cost-effective (ICER raging from £2,460 to £24,954 per QALY gained), and to dominated in the UK setting.ConclusionsThe CUAs for CHC were found to apply various HSUVs from different utility elicitation studies in the same health state. This variability of HSUVs has the potential to significantly affect ICER and ICER-based reimbursement decision. A rigorous selection of HSUVs in CUAs to inform healthcare resource allocation is suggested for future studies of CUAs and guideline development.

2020 ◽  
Vol 41 (1) ◽  
pp. 89-99
Author(s):  
Aureliano Paolo Finch ◽  
John Brazier ◽  
Clara Mukuria

Background Generic preference-based measures (GPBMs) such as the EQ-5D are valid across many conditions, but in some cases, “bolting on” additional dimensions may improve validity. The selection of “bolt-ons” has been based on the psychometric impact of individual dimensions, but preferences provide another important way to select them. This study aims to test the potential of using pairwise choices to inform the selection of bolt-ons for the EQ-5D-5L. Methods General population preferences were collected using an online survey of 1040 UK residents. Three EQ-5D-5L health state pairs were selected based on pairs that had a 50:50 split in respondent preferences from a previous pairwise survey. Participants were presented with pairwise choices of EQ-5D-5L health states without and with bolt-ons of hearing, sleep, cognition, energy, and relationships, each added individually. Logistic models were used to assess the impact of bolt-ons, as well as bolt-ons at different severity levels, on the log odds of responders choosing between health states. Results Preferences varied according to the bolt-ons and their severity level (only levels 1, 3, and 5 were used). Additions of bolt-ons at level 1 generally resulted in nonstatistically significant differences while additions of bolt-ons at level 3 and level 5 produced a negative and statistically significant impact on preferences for the health state with the bolt-on. At level 5, hearing had the largest impact, followed by cognition, relationships, energy, and sleep. At level 3, cognition produced the largest impact, followed by hearing and sleep with similar impacts, energy, and relationships. This ordering offers information for bolt-on selection, with hearing and cognition appearing as the most important. The weight placed on the different health problems is not constant across severity levels between bolt-ons. Conclusions Pairwise choices provide a cost-effective approach of generating information on preferences to support bolt-on selection.


Author(s):  
Morteza Arab-Zozani ◽  
Hossein Safari ◽  
Zoha Dori ◽  
Somayeh Afshari ◽  
Hosein Ameri ◽  
...  

Health-state utility values of diabetic foot ulcer (DFU) patients are necessary for clinical praxis and economic modeling. The purpose of this study was to estimate utility values in DFU patients using the EuroQol-5-dimension-5-level (EQ-5D-5L) and composite time trade-off (cTTO). The EQ-5D-5L and cTTO were used for estimating utility values. Data were collected from 228 patients referred to the largest governmental diabetes center in the South of Iran, Yazd province. When appropriate, independent sample t-test or analysis of variance test was used to test the difference in the utility values in each of the demographic and clinical characteristics of the patients. Finally, the BetaMix was used to identify predictors of the utility values. The means of EQ-5D-5L and cTTO values were 0.55( SD 0.21) and 0.67( SD 0.23), respectively. Anxiety and pain were the most common problems reported by the patients. The difference between the mean EQ-5D-5L values was significant for age, grade of ulcer, number of comorbidities, and having complications. In addition, variables of gender, age, grade of ulcer, and having complications were significant predictors of the EQ-5D-5L. The difference between the mean cTTO values was significant for age, employment status, grade of ulcer, number of comorbidities, and having complications. Moreover, variables of gender, age, grade of ulcer, number of comorbidities, and developing complications were significant predictors of cTTO. The current study provided estimates of utility values for DFU patients for clinical praxis and economic modeling. These estimates, similar to utilities reported in other studies, were low. Identifying strategies to decrease anxiety/depression and pain in patients is important to improve the utility values.


Sarcoma ◽  
2013 ◽  
Vol 2013 ◽  
pp. 1-9 ◽  
Author(s):  
Julian F. Guest ◽  
Erikas Sladkevicius ◽  
Nicholas Gough ◽  
Mark Linch ◽  
Robert Grimer

Soft tissue sarcomas are a rare type of cancer generally treated with palliative chemotherapy when in the advanced stage. There is a lack of published health utility data for locally advanced “inoperable”/metastatic disease (ASTS), essential for calculating the cost-effectiveness of current and future treatments. This study estimated time trade-off (TTO) and standard gamble (SG) preference values associated with four ASTS health states (progressive disease, stable disease, partial response, complete response) among members of the general public in the UK (n=207). The four health states were associated with decreases in preference values from full health. Complete response was the most preferred health state (mean utility of 0.60 using TTO). The second most preferred health state was partial response followed by stable disease (mean utilities were 0.51 and 0.43, respectively, using TTO). The least preferred health state was progressive disease (mean utility of 0.30 using TTO). The utility value for each state was significantly different from one another (P<0.001). This study demonstrated and quantified the impact that different treatment responses may have on the health-related quality of life of patients with ASTS.


2012 ◽  
Vol 26 (7) ◽  
pp. 445-451 ◽  
Author(s):  
Gloria Woo ◽  
George Tomlinson ◽  
Colina Yim ◽  
Les Lilly ◽  
George Therapondos ◽  
...  

BACKGROUND: The effect of chronic hepatitis B (CHB) infection on health-related quality of life (HRQoL) and health state utilities has not been well characterized.OBJECTIVE: To measure utility scores and HRQoL across disease states associated with CHB infection.METHODS: Patients attending four tertiary care clinics for CHB were approached between July 2007 and March 2009. Respondents completed version 2 of the Short-Form 36 Health Survey, the EQ5D, a visual analogue scale, the Health Utilities Index Mark 3, standard gamble, and demographics and risk factor surveys in English, Cantonese or Mandarin. Charts were reviewed to determine disease stage and comorbidities.RESULTS: A total of 433 patients were studied: 294 had no cirrhosis; 79 had compensated cirrhosis; seven had decompensated cirrhosis; 23 had hepatocellular carcinoma; and 30 had received a liver transplant. The mean standard gamble utilities for these disease states were 0.89, 0.87, 0.82, 0.84 and 0.86, respectively. HRQoL scores in noncirrhotic patients were similar to those of the general population. Scores of patients with compensated cirrhosis were not significantly lower; however, patients with decompensated cirrhosis and hepatocellular carcinoma had significantly lower HRQoL scores compared with noncirrhotic patients (P<0.05). Similar scores were observed among patients on and off oral antiviral treatment. Post-liver transplant patients had a higher HRQoL than patients with decompensated cirrhosis. Age, number of comorbidities and relationship status were significantly associated with HRQoL scores.CONCLUSIONS: HRQoL in CHB patients is only impaired in the later stages of liver disease. Neither CHB infection nor antiviral treatment is associated with a lower quality of life.


2004 ◽  
Vol 18 (6) ◽  
pp. 411-412
Author(s):  
Kevork M Peltekian

Chong and colleagues (1) used gold-standard methods to assess health-state utilities in 193 outpatients at various stages of liver disease due to chronic hepatitis C (HCV) infection. They showed worsening of health-state utility scores with progression of disease from mild to moderate chronic hepatitis, to compensated cirrhosis, to decompensated cirrhosis and to hepatocellular carcinoma. They also confirmed improvement in health-state utility scores after liver transplantation and with sustained virological response (SVR) to treatment of HCV infection. Patients with HCV infection had lower health-state utility scores than the general Canadian population (PÃ0.001) and significantly poorer quality of life compared with population norms in the United States (PÃ0.005).


Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 3427-3427 ◽  
Author(s):  
Simone Critchlow ◽  
Miranda Cooper ◽  
Ilse van Oostrum ◽  
Verna L Welch ◽  
T. Alexander Russell-Smith

Introduction: Inotuzumab ozogamicin (InO), is a novel anti-CD22 antibody-calicheamicin conjugate approved in R/R B-ALL due to its high hematologic remission rate (81%) based on the phase 3 INO-VATE trial comparing to investigators choice (IC). The TOWER trial demonstrated the efficacy and safety of blinatumomab (Blina) for treatment of Ph- B-ALL versus IC. The relative effectiveness of InO versus Blina was investigated by applying indirect treatment comparison (ITC) methods. A UK-based cost-effectiveness model (CEM) submitted to the Scottish Medicines Consortium (SMC) explored the impact of treatment differences with regard to mean life years (LY) gained and quality-adjusted life years (QALY). Methods: As R/R ALL is a terminal disease if left untreated, achievement of complete response/complete response with incomplete count recovery (CR/CRi) in conjunction with stem cell transplant (SCT) is essential for long-term survival. The three most important outcomes related to treatment are thus the level of response determined by CR/CRi, the rate of SCT, and overall survival (OS). Without potentially curative therapy such as SCT, there is no evidence to suggest long-term survival is possible. Therefore, to compare InO to Blina, comparisons of these outcomes were explored using patient-level data from the INO-VATE ALL trial and aggregate data from the TOWER trial. The CEM structure contained four health states categorising patients based on 'No CR/CRi & no SCT', 'CR/CRi and no SCT' and patients receiving SCT ('SCT/Post SCT') - with progression-free survival (PFS) and OS modelled within these states. States were clinically validated as relevant to treatment of the disease. Death was the fourth health state. Different methods were incorporated to allocate Blina patients to the respective health-states. For levels of response (CR/CRi) and SCT a matching-adjusted indirect comparison (MAIC) and a Bucher ITC were explored. As CR/CRi and SCT rates are not mutually exclusive, a multinomial ITC was also conducted. Once allocated into respective health states, OS and PFS were modelled. Three ITC methods were used to compare OS; a simulated treatment comparison (STC), MAIC and a standard network meta-analysis. In the absence of PFS data for Blina, PFS was assumed to have the same relative treatment effect as OS. Quality of life data within the model for the 'No CR/CRi & no SCT' and 'CR/CRi and no SCT' were informed from InO trial data, while SCT quality of life was informed from the literature with time-varying utilities. Costs were incorporated from a UK perspective using 2017 sources and were those submitted to the SMC. Results were annually discounted at 3.5%. Results: Health state proportions for Ph- InO patients were used as the basis to estimate corresponding Blina proportions and show 49.3% of patients treated with InO reach SCT. With higher odds for CR/CRi and SCT for InO, the ITC results consistently indicate Blina leads to lower proportions of patients receiving SCT (19.1-22.5%) and CR/CRi (25.2-33.3%). ITCs comparing OS outcomes for InO versus blinatumomab show negligible differences between treatments, consistently across the three methods. All combinations of the various methods were explored using the list price for both treatments. The results of the CEM ranged from 0.91-1.14 incremental QALYs for InO versus Blina, while LYs ranged from 2.03-2.59 resulting from higher rates of SCT. The incremental cost-effectiveness ratio (ICER) ranged from £3,700 to £7,010 for InO versus Blina. Extensive scenario analysis indicates that InO is a cost-effective option compared to Blina at a willingness to pay threshold of £20,000 per QALY. The SMC recommended InO as a cost-effective use of resources citing an ICER of £6,754 in the CEM when using the MAIC; InO was associated with a mean survival gain of &gt;29 months over Blina corresponding to this ICER. Conclusions: Outcomes from the ITC indicate that InO provides patients with a greater probability of achieving CR/CRi and/or receiving a subsequent SCT versus Blina. As CR/CRi followed by SCT are essential for long-term survival and potential cure, the mean OS gain in the model cited in the SMC recommendation is intuitive as it aligns with the superior CR/CRi and SCT odds ratios associated with InO. Further research is required to determine the long-term PFS and OS following SCT in R/R B-ALL, beyond what can be reliably captured within clinical trials. Disclosures Critchlow: BresMed Health Solutions Ltd.: Consultancy. Cooper:BresMed Health Solutions Ltd.: Consultancy. van Oostrum:Ingress Health: Employment; Pfizer: Consultancy; Merck: Consultancy; Janssen: Consultancy; AstraZeneca: Consultancy. Welch:Pfizer Inc: Employment, Equity Ownership. Russell-Smith:Pfizer: Employment, Equity Ownership.


2019 ◽  
Vol 3 (Supplement_1) ◽  
pp. S636-S636
Author(s):  
Matthew Pichiello ◽  
Meghan McDarby ◽  
Elissa K Kozlov ◽  
Brian Carpenter

Abstract Adult children often help older parents make medical decisions when their health is compromised. To do so in a way that respects parent values requires children to know how their parent views health states and consequent quality of life. The current study compared older parent and adult child valuations of quality of life in different health contexts. Families consisted of older parents (n = 37) and their adult children (n = 66). Parents rated perceived quality of life in 14 compromised health states on a scale from 1 (difficult but acceptable) to 5 (not worth living). Children estimated how their parent responded to each health state, yielding an index of their knowledge of parent perceptions. Overall, parents described all compromised health states as less acceptable than adult children thought they would, t(99) = 2.19, p &lt; .05. Notably, parents believed situations that caused financial or emotional burden to their family were much less acceptable than their children estimated. Children were more knowledgeable about parent valuations for more extreme circumstances, such as living with a feeding tube. Within families, children demonstrated only slight knowledge about parent quality of life valuations (kappa = .081). Across the entire sample of families, there was a broad range of knowledge (kappas = -.181 – .351), but at best, knowledge was still only fair. Results from this study suggest that adult children may underestimate the impact of compromised health states on parent estimations of quality of life, which could affect collaborations on healthcare decisions.


2019 ◽  
Vol 39 (4) ◽  
pp. 393-404 ◽  
Author(s):  
Martine Hoogendoorn ◽  
Mark Oppe ◽  
Melinde R. S. Boland ◽  
Lucas M. A. Goossens ◽  
Elly A. Stolk ◽  
...  

Objectives. To evaluate the impact of adding a respiratory dimension (a bolt-on dimension) to the EQ-5D-5L health state valuations. Methods. Based on extensive regression and principal component analyses, 2 respiratory bolt-on candidates were formulated: R1, limitations in physical activities due to shortness of breath, and R2, breathing problems. Valuation interviews for the selected bolt-ons were performed with a representative sample from the Dutch general public using the standardized interview protocol and software of the EuroQol group. Hybrid models based on the combined time-tradeoff (TTO) and discrete choice experiment (DCE) data were estimated to assess whether the 5 levels of the respiratory bolt-on led to significant changes in utility values. Results. For each bolt-on candidate, slightly more than 200 valuation interviews were conducted. Mean TTO values and DCE choice probabilities for health states with a level 4 or 5 for the respiratory dimension were significantly lower compared with the same health states in the Dutch EQ-5D-5L valuation study without the respiratory dimension. Results of hybrid models showed that for the bolt-on “limitations in physical activities,” the utility decrements were significant for level 3 (–0.055), level 4 (–0.087), and level 5 (–0.135). For “breathing problems,” the utility decrements for the same levels were greater (–0.086, –0.219, and –0.327, respectively). Conclusions. The addition of each of the 2 respiratory bolt-ons to the EQ-5D-5L had a significant effect on the valuation of health states with severe levels for the bolt-on. The bolt-on dimension “breathing problems” showed the greatest utility decrements and therefore seems the most appropriate respiratory bolt-on dimension.


2019 ◽  
Vol 29 (3) ◽  
pp. 593-605 ◽  
Author(s):  
Shelagh M. Szabo ◽  
Ivana F. Audhya ◽  
Daniel C. Malone ◽  
David Feeny ◽  
Katherine L. Gooch

Abstract Background Preferences for health states for Duchenne muscular dystrophy (DMD) are necessary to assess costs and benefits of novel therapies. Because DMD progression begins in childhood, the impact of DMD on health-related quality-of-life (HRQoL) affects preferences of both DMD patients and their families. The objective of this review was to synthesize published evidence for health state utility from the DMD patient and caregiver perspectives. Methods A systematic review was performed using MEDLINE and Embase, according to best practices. Data were extracted from studies reporting DMD patient or caregiver utilities; these included study and patient characteristics, health states considered, and utility estimates. Quality appraisal of studies was performed. Results From 888 abstracts, eight publications describing five studies were identified. DMD utility estimates were from preference-based measures presented stratified by ambulatory status, ventilation, and age. Patient (or patient–proxy) utility estimates ranged from 0.75 (early ambulatory DMD) to 0.05 (day-and-night ventilation). Caregiver utilities ranged from 0.87 (for caregivers of adults with DMD) to 0.71 (for caregivers of predominantly childhood patients). Both patient and caregiver utilities trended lower with higher disease severity. Variability in utilities was observed based on instrument, respondent type, and country. Utility estimates for health states within non-ambulatory DMD are under reported; nor were utilities for DMD-related health states such as scoliosis or preserved upper limb function identified. Conclusion Published health state utilities document the substantial HRQoL impacts of DMD, particularly with disease progression. Additional research in patient utilities for additional health states, particularly in non-ambulatory DMD patients, is warranted.


2008 ◽  
Vol 3 ◽  
pp. BMI.S639 ◽  
Author(s):  
Seren Ozenirler ◽  
Banu Sancak ◽  
Ugur Coskun

Serum and ascitic fluid superoxide dismutase (SOD) and malondialdehyde (MDA) levels were measured in 43 patients with cirrhosis and in a 10 healthy control group. Compensated cirrhotic patients had no clinically detectable ascites, but decompensated patients had massive ascites. Cirrhotic patients were divided into three groups: patients with compensated cirrhosis (n = 16), patients with decompensated cirrhosis with Spontaneous bacterial peritonitis (SBP) (n = 14), and patients with decompensated cirrhosis without SBP (n = 13). All cirrhotic patients in the experimental group had significantly higher serum SOD (p < 0.001) and MDA levels (p < 0.01) than those in the control group. There were no significant differences with respect to serum SOD and MDA levels among the three different groups of patients. There was no remarkable difference in ascitic fluid SOD and MDA levels between decompensated cirrhotic patients with and without SBP (p > 0.05). These results suggest that the increase in serum SOD and MDA levels are not related to the presence of SBP and the status of liver cirrhosis. To sum up, clarifying the impact of increased serum SOD and MDA levels in cirrhotic patients needs further investigation.


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