Health State Utility Values Research Articles

Health State Utility Values Associated with Cochlear Implants in Adults: A Systematic Review and Network Meta-Analysis.

The cost-effectiveness of bilateral cochlear implants in adults remains uncertain despite established clinical benefits. In cost-effectiveness studies, benefit is often measured by change in health state utility value (HSUV), a single number summary of health-related quality of life anchored at 0 (state of being dead) and 1 (perfect health). Small differences in bilateral cochlear implant HSUV change conclusions of published models, and invalid estimates can therefore mislead policy and funding decisions. As such, we aimed to review and synthesize published HSUV estimates associated with cochlear implants. We included observational or experimental studies reporting HSUV for adult patients (age ≥18 years) with at least moderate-profound sensorineural hearing loss in both ears who received unilateral or bilateral cochlear implants. We searched MEDLINE, EMBASE, PsycINFO, and Cochrane Library databases up to May 1, 2021. Study and participant characteristics and HSUV outcomes were extracted. Narrative synthesis is reported for all studies. A Bayesian network meta-analysis was conducted to generate pooled estimates for the mean difference in HSUV for three comparisons: (1) unilateral cochlear implant versus preimplant, (2) bilateral cochlear implants versus preimplant, (3) bilateral versus unilateral cochlear implants. Our principal measure was pooled mean difference in HSUV. Thirty-six studies reporting unique patient cohorts were identified. Health Utilities Index, 3 (HUI-3) was the most common HSUV elicitation method. HSUV from 19 preimplant mean estimates (1402 patients), 19 unilateral cochlear implant mean estimates (1701 patients), and 5 bilateral cochlear implants mean estimates (83 patients) were pooled to estimate mean differences in HUI-3 HSUV by network meta-analysis. Compared with preimplant, a unilateral cochlear implant was associated with a mean change in HSUV of +0.17 (95% credible interval [CrI] +0.12 to +0.23) and bilateral cochlear implants were associated with a mean change of +0.25 (95% CrI +0.12 to +0.37). No significant difference in HSUV was detected for bilateral compared with unilateral cochlear implants (+0.08 [95% CrI -0.06 to +0.21]). Overall study quality was moderate. The findings of this review and network meta-analysis comprise the best-available resource for parameterization of cost-utility models of cochlear implantation in adults and highlight the need to critically evaluate the validity of available HSUV instruments for bilateral cochlear implant populations.Protocol registration: PROSPERO (CRD42018091838).

Improving Interpretation of Evidence Relating to Quality of Life in Health Technology Assessments of Rare Disease Treatments.

Rare diseases are often severe, debilitating, life-limiting conditions, many of which occur in childhood. These complex conditions have a wide range of clinical manifestations that have a substantial impact on the lives of patients, carers and families and often produce heterogeneous clinical outcomes. Therefore, the evaluation of quality-of-life (QoL) impacts is important. In health technology assessment (HTA), patient-reported outcome measures (PROMs) and/or health state utility values (HSUVs) are used to determine QoL impacts of new treatments, but their use in rare diseases is challenging due to small and heterogeneous populations and limited disease knowledge. This paper describes challenges associated with the use of patient-reported outcomes (PROs)/HSUVs to evaluate QoL in HTA of rare disease treatments (RDTs) and identifies five recommendations to ensure appropriate interpretation of QoL impacts. These were derived from mixed methods research (literature reviews, appraisal document analyses, appraisal committee observations and interviews) examining the use of PROs/HSUVs in HTA of RDTs. They highlight that HTAs of RDTs must (1) understand the QoL impacts of the disease and of treatments; (2) critically assess PRO data, recognising the nuances in development and administration of PROMs/HSUVs, considering what is feasible and what matters most to the patient population; (3) recognise that lack of significant effect on a PRO does not imply no QoL benefit; (4) use different forms of evidence to understand QoL impacts, such as patient input; and (5) provide methodological guidance to capture QoL impacts on patients/carers.

Quality of life (QoL) in patients with heart failure with preserved ejection fraction (HFpEF): a systematic review

Abstract Introduction Heart failure with preserved ejection fraction (HFpEF) is associated with significant clinical unmet need as mortality and morbidity rates are high despite current treatments. Patient-reported quality of life (QoL) is an important and clinically relevant endpoint in patients with HF so understanding the impact of HFpEF on QoL is essential. Purpose The aim of this systematic review was to identify and summarize data on QoL in HFpEF. Methods Systematic searches of Medline, Medline Epub Ahead of Print (In-Process & Other Non-Indexed Citations), Embase, and EBM Reviews were conducted in October 2021. Congress proceedings from the past 3 years and reference lists of included publications were also searched. Publications were screened against eligibility criteria by title/abstract and then by full text. Eligibility criteria included clinical studies of any design reporting baseline QoL in adults with HFpEF published from 2016 onwards. Results The electronic database search identified 6,403 articles on HFpEF. After screening, 35 full publications reporting HFpEF and QoL were included. Study designs included observational cohort (n=17), cross-sectional (n=3), and post-hoc analyses of interventional studies (n=15). Tools used to measure QoL included KCCQ (n=17), MLHFQ (n=12), EQ-5D-VAS (n=8), SF-12 (n=2), SF-36 (n=2), and EHFSQ-1 (n=1). Health-state utility values (HSUVs) were captured using EQ-5D in four studies. HSUVs in HFpEF ranged from 0.67–0.74, indicating a substantial QoL burden. Eleven studies using various instruments compared QoL in patients with HFpEF vs non-HFpEF (Table 1). Of these, five reported statistically significantly poorer QOL in HFpEF vs non-HFpEF, one reported statistically significantly poorer QOL in non-HFpEF vs HFpEF and five found no statistically significant difference between HF subtypes. Four studies compared QoL in men vs women with three reporting statistically significantly worse QoL in women. Three studies examined the impact of comorbidities, and all reported statistically significantly poorer QoL in patients with comorbid atrial fibrillation, diabetes, or metabolic syndrome. Two studies examined the effect of age; one reported that, vs elderly patients (age ≥85 years), younger patients (age ≤55 years) with HFpEF had statistically significantly worse QoL despite having fewer comorbidities; in contrast, the second study reported that younger patients (age ≤55 years) had statistically significantly better QoL than elderly patients (age ≥75 years). Two studies reported on hospitalizations, with one reporting no difference in QoL between patients who had previously been hospitalized and those who had not and the other reporting that readmission for HF correlated with QoL. Conclusions QoL burden among HFpEF patients is substantial. QoL may be affected by age, comorbidities, gender, and prior hospitalization, and may be associated with poorer outcomes. QoL in HFpEF is generally poorer than in non-HFpEF. Funding Acknowledgement Type of funding sources: Private company. Main funding source(s): AstraZeneca

Value-Based Analysis of Therapies in Refractory Metastatic Colorectal Cancer in US

BackgroundRecent phase 2 trials have provided data supporting regorafenib dose optimization (ReDO) and trifluridine/tipiracil (TAS-102) with bevacizumab (TAS-BEV) as treatment options in refractory metastatic colorectal cancer (mCRC). Historically, regorafenib standard dose (RSD) and TAS-102 have been utilized as third-line options in mCRC. Given the incorporation of ReDO and TAS-BEV as treatment options, we sought to evaluate relative cost-effectiveness of ReDO vs. RSD, TAS-102, and TAS-BEV for mCRC from a payer perspective. MethodsA Markov model was constructed to estimate total costs and quality-adjusted life-years (QALYs) for ReDO, RSD, TAS-102, and TAS-BEV. Clinical parameters were obtained from phase 2 and 3 trials for comparators. Health state utility values were from the RSD phase 3 clinical trial. Incremental cost-effectiveness ratios (ICERs) were utilized to compare treatments. Model robustness was checked with one-way and probabilistic sensitivity analyses. ResultsIn the base case, ReDO was dominant over TAS-BEV (ie provided a higher QALY at a lower cost). ReDO produced an ICER of $104,308 per QALY relative to RSD and $37,966 relative to TAS-102. In one-way sensitivity analyses, monthly drug cost of TAS-BEV was the most influential parameter determining relative cost-effectiveness between TAS-BEV and ReDO. When TAS-102 and RSD were independently compared to ReDO, the most influential parameters were related to duration of OS and PFS and costs of managing AEs. ConclusionsThe optimum dosing strategy for regorafenib has improved its benefit-to-toxicity ratio and relative cost-effectiveness compared to RSD, TAS-102, and TAS-BEV.

Effect of Digital Medication Event Reminder and Monitor-Observed Therapy vs Standard Directly Observed Therapy on Health-Related Quality of Life and Catastrophic Costs in Patients With Tuberculosis

Little is known about whether digital adherence technologies are economical for patients with tuberculosis (TB) in resource-constrained settings. To test the hypothesis that for patients with TB, a digital medication event reminder monitor (MERM)-observed therapy provides higher health-related quality of life (HRQoL) and lower catastrophic costs compared with standard directly observed therapy (DOT). This study was a secondary analysis of a randomized, 2-arm, open-label trial conducted in 10 health care facilities in Ethiopia. Eligible participants were adults with new or previously treated, bacteriologically confirmed, drug-sensitive pulmonary TB who were eligible to start first-line anti-TB therapy. Participants were enrolled between June 2, 2020, and June 15, 2021, with the last participant completing follow-up on August 15, 2021. Participants were randomly assigned (1:1) to receive a 15-day TB medication supply dispensed with a MERM device to self-administer and return every 15 days (intervention arm) or the standard in-person DOT (control arm). Both groups were observed throughout the standard 2-month intensive treatment phase. Prespecified secondary end points of the original trial were HRQoL using the EuroQoL 5-dimension 5-level (EQ-5D-5L) tool and catastrophic costs, direct (out-of-pocket) and indirect (guardian and coping) costs from the individual patient perspective using the World Health Organization's Tool to Estimate Patient Costs, and common factors associated with lower HRQoL and higher catastrophic costs. Among 337 patients screened for eligibility, 114 were randomly assigned, and 109 were included in the final complete-case intention-to-treat analysis (57 control and 52 intervention participants). The mean (SD) age was 33.1 (11.1) years; 72 participants (66.1%) were men, and 15 (13.9%) had HIV coinfection. EQ-5D-5L overall median (IQR) index value was 0.964 (0.907-1). The median (IQR) value was significantly higher in intervention (1 [0.974-1]) vs control (.908 [0.891-0.964]) (P < .001). EQ-5D-5L minimum and maximum health state utility values in intervention were 0.906 and 1 vs 0.832 and 1 in control. Patients' overall median (IQR) postdiagnosis cost was Ethiopian birr (ETB) 80 (ETB 16-ETB 480) (US $1.53). The median cost was significantly lower in intervention (ETB 24 [ETB 16-ETB 48]) vs control (ETB 432 [ETB 210-ETB 1980]) (P < .001), with median possible cost savings of ETB 336 (ETB 156-ETB 1339) (US $6.44) vs the control arm. Overall, 42 participants (38.5%; 95% CI, 29.4%-48.3%) faced catastrophic costs, and this was significantly lower in the intervention group (11 participants [21.2%]; 95% CI, 11.1%-34.7%) vs control (31 participants [54.4%]; 95% CI, 40.7%-67.6%) (P < .001). Trial arm was the single most important factor in low HRQoL (adjusted risk ratio [ARR], 1.49; 95% CI, 1.35-1.65; P < .001), while trial arm (ARR, 2.55; 95% CI, 1.58-4.13; P < .001), occupation (ARR, 2.58; 95% CI, 1.68-3.97; P < .001), number of cohabitants (ARR, 0.64; 95% CI, 0.43-0.95; P = .03), and smoking (ARR, 2.71; 95% CI, 1.01-7.28; P = .048) were the most important factors in catastrophic cost. In patients with TB, MERM-observed therapy was associated with higher HRQoL and lower catastrophic costs compared with standard DOT. Patient-centered digital health technologies could have the potential overcoming structural barriers to anti-TB therapy. ClinicalTrials.gov Identifier: NCT04216420.

Assessment of health-related quality of life in Australian patients with idiopathic pulmonary fibrosis: a comparison of the EQ-5D-5L and the AQoL-8D

PurposeIdiopathic pulmonary fibrosis (IPF) is a progressive and debilitating chronic lung disease with a high symptom burden, which has a substantial impact on health-related quality of life (HRQoL). Our study aimed to assess the suitability of the EuroQol five-dimension (EQ-5D-5L) and the Assessment of Quality of Life- eight-dimension (AQoL-8D) questionnaires in measuring HRQoL as health state utility values (HSUVs) in an Australian IPF cohort.MethodsData for estimation of health state utility values (HSUVs) were collected from participants of the Australian IPF Registry (AIPFR) using self-administered surveys which included the EQ-5D-5L and the AQoL-8D. Data on lung function and disease specific HRQoL instruments were collected from the AIPFR. Performance of the two instruments was evaluated based on questionnaire practicality, agreement between the two instruments and test performance (internal and construct validity).ResultsOverall completion rates for the EQ-5D-5L and AQoL-8D were 96% and 85%, respectively. Mean (median) HSUVs were 0.65 (0.70) and 0.69 (0.72) for the EQ-5D-5L and AQoL-8D, respectively. There was reasonable agreement between the two instruments based on the Bland–Altman plot mean difference (−0.04) and intraclass correlation coefficient (0.84), however there were some fundamental differences. A larger range of values was observed with the EQ-5D-5L (−0.57–1.00 vs 0.16–1.00). The EQ-5D-5L had a greater divergent sensitivity and efficacy in relation to assessing HSUVs between clinical groupings. The AQoL-8D ,however, had a higher sensitivity to measure psychosocial aspects of HRQoL in IPF.ConclusionThe EQ-5D-5L demonstrated superior performance when compared to AQoL-8D in persons with IPF. This may be attributable to the high symptom burden which is physically debilitating to which the EQ-5D-5L may be more sensitive.

Cost-Utility Analysis of Sacroiliac Joint Fusion in High-Risk Patients Undergoing Multi-Level Lumbar Fusion to the Sacrum.

PurposeMulti-level lumbar fusion to the sacrum (MLF) can lead to increased stress and angular motion across the sacroiliac joint (SIJ), with an incidence of post-operative SIJ pain estimated at 26–32%. SIJ fusion (SIJF) can help obviate the need for revisions by reducing range of motion and screw stresses. We aimed to evaluate the cost-utility of MLF + SIJF compared to MLF alone among high-risk patients from a payer perspective, where high risk is defined as high body mass index and high pelvic incidence.MethodsA Markov process decision-analysis model was developed to evaluate cumulative 5-year costs, quality-adjusted life years (QALYs), and incremental cost-effectiveness ratio (ICER) of MLF + SIJF compared to MLF alone using published data; costs from Medicare claims data analyses and health state utility values (derived from EQ-5D) informed by three prospective, multicenter, clinical trials. The base case assumed a reduction in post-operative SIJ pain from 30% to 10% (relative risk reduction [RRR] of 67%). Costs and utilities were discounted 3% annually. The ICER is reported in 2020 US dollars. One-way, multi-way, and probabilistic sensitivity analyses were performed.ResultsWith an assumed 30% incidence of SIJ pain after MLF alone, stabilizing with SIJF was associated with an additional 5-year cost of $2421 and a gain of 0.14 QALYs, resulting in an ICER of $17,293 per QALY gained (similar to total knee arthroplasty and more favorable than open discectomy). ICERs were most sensitive to the RRR of post-operative SIJ pain conferred by SIJF, time horizon, and probability of successful treatment with MLF alone. At a willingness-to-pay threshold of $50,000/QALY gained, MLF + SIJF has a 97.7% probability of being cost-effective in the target patient population.ConclusionFusing the SIJ in high-risk patients undergoing MLF was cost-effective when the incidence of post-operative SIJ pain after MLF alone exceeds approximately 25%, providing value-based healthcare from a payer perspective.

The importance of preference interactions in joint health-state utility values applied to decision analyses for shared decision-making

The direct elicitation of health-state utility values (HSUVs) is difficult, and inconsistent HSUVs are a prevalent problem. Joint health conditions (JHCs) affect people’s quality of life in different ways. They can be preference substitutes, preference complements, or mutually utility independent. This article develops a novel model called the correlated bivariate Bernoulli health-utility (CBBHU) model, for estimating joint HSUVs (JHSUVs) from known single HSUVs and a small subset of elicited JHSUVs. A bivariate health utility function (HUF) is developed for the dependence of JHSUVs on severity. It consists of the product of the constituent single HUFs and a bivariate function with two parameters that vary with health conditions and patients’ preferences. These parameters can be fitted to as few as two severity levels and the parametrized HUF used to estimate HSUVs for different severity levels. A bootstrap method that requires a significantly reduced number of elicited HSUVs is proposed for estimating JHSUVs for three or more JHCs. The CBBHU functions satisfy the Fréchet bounds and provide internally consistent HSUVs. Preference interactions can have a substantial impact on patients’ medical decisions. CBBHU values are appropriate for shared decision-making applications. HIGHLIGHTS The CBBHU model is a novel theoretical model for estimating JHSUVs. The CBBHU model provides a practical approach to model and predict reliable JHSUVs. The JHSUVs satisfy the Fréchet inequalities, utility theory, and prospect theory. The CBBHU models JHCs that are preference complements and preference substitutes. A practical bootstrap method extends the CBBHU model to multimorbidities.

HTA6 Evaluation of Health State Utility Values (HSUV) Used in Health Technology Assessment (HTA) Submissions for Rare Diseases

Health state utility values (HSUVs) are a measure of preference for a health state on a scale of 0 to 1, with 0 indicating death and 1 representing perfect health. Obtaining HSUVs may be difficult in rare diseases due to inherent challenges such as small sample size, higher HSUVs with patient experiencing the condition, and absence of valid mapping algorithms for specific health-related quality of life measures (HRQoL). We aimed to investigate the range of sources of HSUVs and their appropriateness per health agencies.

PCR176 Time Trade-Off Utility Values for Health States Characterizing Progressive Muscular Degeneration in Duchenne Muscular Dystrophy (DMD)

The progression of DMD is characterized by loss of ambulation (LOA) and upper limb function, cardiorespiratory impairment, and early mortality. Published utility values are limited, and generally restricted to health states defined solely by ambulatory status. The objective of this study was to estimate societal utility values for health states describing the progression of DMD.

PCR72 Health State Utility Values for Patients with Chemotherapy-Induced Myelosuppression: A Systematic Review

Cytotoxic chemotherapy can induce myelosuppression and have an influence on a cancer patient’s quality of life (QOL). Two newer products developed to prevent chemotherapy-induced myelosuppression, plinabulin and trilaciclib, have primarily been studied in early-stage breast and advanced lung cancer. This systematic literature review sought to characterize empirically derived health state utility values (HSUVs) of chemotherapy-induced myelosuppression at any stage of breast cancer (BC), small cell lung cancer (SCLC), and non-small cell lung cancer (NSCLC).

PCR104 Improvements in Health Utility Scores after Administration of Ferric Derisomaltose Versus Ferric Carboxymaltose in Patients with Inflammatory Bowel Disease and Iron Deficiency Anemia

Inflammatory bowel disease (IBD) is a chronic inflammatory disease of the gastrointestinal tract that commonly result in iron deficiency anemia (IDA) due to impaired iron absorption and chronic blood loss. Patients with IBD experience reduced health-related quality of life, with fatigue playing a key role. The aim of the present analysis was to calculate health state utility values (HSUVs) from a recent randomized controlled trial (RCT) of intravenous iron in the treatment of patients with IBD and IDA.

Health State Utility Values in People With Stroke: A Systematic Review and Meta-Analysis.

BackgroundHealth state utility values are commonly used to provide summary measures of health‐related quality of life in studies of stroke. Contemporaneous summaries are needed as a benchmark to contextualize future observational studies and inform the effectiveness of interventions aimed at improving post‐stroke quality of life.Methods and ResultsWe conducted a systematic search of the literature using Medline, EMBASE, and Web of Science from January 1995 until October 2020 using search terms for stroke, health‐related quality of life, and indirect health utility metrics. We calculated pooled estimates of health utility values for EQ‐5D‐3L, EQ‐5D‐5L, AQoL, HUI2, HUI3, 15D, and SF‐6D using random effects models. For the EQ‐5D‐3L we conducted stratified meta‐analyses and meta‐regression by key subgroups. We screened 14 251 abstracts and 111 studies met our inclusion criteria (sample size range 11 to 12 447). EQ‐5D‐3L was reported in 78% of studies (study n=87; patient n=56 976). The pooled estimate for EQ‐5D‐3L at ≥3 months following stroke was 0.65 (95% CI, 0.63–0.67), which was ≈20% below population norms. There was high heterogeneity (I2>90%) between studies, and estimates differed by study size, case definition of stroke, and country of study. Women, older individuals, those with hemorrhagic stroke, and patients prior to discharge had lower pooled EQ‐5D‐3L estimates.ConclusionsPooled estimates of health utility for stroke survivors were substantially below population averages. We provide reference values for health utility in stroke to support future clinical and economic studies and identify subgroups with lower healthy utility.RegistrationURL: https://www.crd.york.ac.uk/prospero/. Unique Identifier: CRD42020215942.

Application of validated mapping algorithms between generic PedsQL scores and utility values to individuals with sickle cell disease.

There is a paucity of empirically estimated health state utility (HSU) values to estimate health-related quality of life among individuals with sickle cell disease (SCD). This study aims to map the Pediatric Quality of Life Inventory generic core scales (PedsQL GCS) to HSUs for children and adolescents with SCD in the United States, using published algorithms, and to assess the construct validity of these HSUs against SCD-specific PedsQL scores. We used the published mapping algorithms identified in four published articles, in which the PedsQL GCS was mapped to either the EuroQol-5 Dimension 3-Level, Youth Version or the Child Health Utility 9-Dimension to obtain HSUs. We employed the algorithms to calculate HSUs for a sample of children and adolescents from the Sickle Cell Clinical Research and Intervention Program. To assess the construct validity of the mapped HSUs in SCD patients, we computed Spearman's correlation coefficient comparing the HSUs with the PedsQL SCD total score and separately with each PedsQL SCD dimension-specific score. The mean mapped HSU across published algorithms was 0.792 (95% CI: 0.782-0.801). It was significantly higher among children aged 5-12years than children aged 13-17years. The Spearman's correlation coefficient for HSUs versus PedsQL SCD total scores was 0.64 (95% CI: 0.57-0.71). Correlations ranged from 0.40 (95% CI: 0.32-0.48) to 0.60 (95% CI: 0.54-0.66) for HSUs versus PedsQL SCD dimension-specific scores. The existing mapping algorithms show acceptable construct validity in children and adolescents with SCD. Additional algorithms are needed for adults and for specific SCD comorbidities.

An examination of machine learning to map non-preference based patient reported outcome measures to health state utility values.

Non‐preference‐based patient‐reported outcome measures (PROMs) are popular in health outcomes research. These measures, however, cannot be used to estimate health state utilities, limiting their usefulness for economic evaluations. Mapping PROMs to a multi‐attribute utility instrument is one solution. While mapping is commonly conducted using econometric techniques, failing to specify the complex interactions between variables may lead to inaccurate prediction of utilities, resulting in inaccurate estimates of cost‐effectiveness and suboptimal funding decisions. These issues can be addressed using machine learning. This paper evaluates the use of machine learning as a mapping tool. We adopt a comprehensive approach to compare six machine learning techniques with eight econometric techniques to map the Patient‐Reported Outcomes Measurement Information System Global Health 10 (PROMIS‐GH10) to the EuroQol five dimensions (EQ‐5D‐5L). Using data collected from 2015 Australians, we find the least absolute shrinkage and selection operator (LASSO) model out‐performed all machine learning techniques and the adjusted limited dependent variable mixture model (ALDVMM) out‐performed all econometric techniques, with the LASSO performing better than ALDVMM. The variable selection feature of LASSO was then used to enhance the performance of the ALDVMM in a hybrid model. Our analysis identifies the potential benefits and challenges of using machine learning techniques for mapping and offers important insights for future research.

Exploring the impact of excessive daytime sleepiness caused by obstructive sleep apnea on patient and partner quality of life: a time trade-off utility study in the UK general public.

This study aimed to quantify the impact of excessive daytime sleepiness (EDS) on patient and patient's partner health-related quality of life in the form of utility values typically used in health economic evaluations. A time trade-off study was conducted in a UK general population sample (representing a societal perspective) to elicit utility values, measured on a 0 to 1 scale, for health states with varying obstructive sleep apnea-associated EDS severity. In a time trade-off study, health states are described, and participants "trade off" time in a specific higher severity state for a shorter amount of time in full health. Overall, the sample consisted of 104 participants, who were interviewed and took part in the time trade-off exercise to elicit utility values for patient and partner residual EDS health states. The average utility score declined with increasing obstructive sleep apnea-associated EDS severity for both patient (no EDS, 0.926; mild EDS, 0.794; moderate EDS, 0.614; severe EDS, 0.546) and partner (no EDS, 0.955; mild EDS, 0.882; moderate EDS, 0.751; severe EDS, 0.670) health states. These results demonstrate the high impact that EDS in obstructive sleep apnea is estimated to have on patient and partner health-related quality of life. Tolley K, Noble-Longster J, Mettam S, etal. Exploring the impact of excessive daytime sleepiness caused by obstructive sleep apnea on patient and partner quality of life: a time trade-off utility study in the UK general public. J Clin Sleep Med. 2022;18(9):2237-2246.

Cost-effectiveness analysis of aprepitant-based anti-emetic regimen for children receiving highly emetogenic chemotherapy: Individual patient data analysis of a randomized trial.

Aprepitant has been shown to reduce chemotherapy-induced nausea and vomiting in children receiving highly emetogenic chemotherapy (HEC). In this study, we assessed the cost-effectiveness of aprepitant for children receiving HEC in India, United Kingdom, and the United States. We utilized individual patient-level outcome data from a pediatric randomized trial, which demonstrated the superiority of an aprepitant-based anti-emetic prophylaxis over standard ondansetron and dexamethasone for HEC. Health state for each day of follow-up was analyzed and quality-adjusted life years (QALYs) were estimated. The incremental cost-utility ratio (ICUR), incremental cost-effectiveness ratio (ICER), and net monetary benefit (NMB) for each country were estimated. Sensitivity analyses by varying cost of aprepitant, hospitalization, and health state utility values by ±25% were conducted. Use of the aprepitant-based regimen resulted in gain of 0.0019 QALY per chemotherapy cycle along with cost savings of $22.25, $1335.52, and $6612.10 for India, United Kingdom, and the United States, respectively. The cost savings per QALY was estimated to be $12,355.84 for India, $734,282.90 for the United Kingdom, and $3,567,564.11 for the United States. The cost savings for 50% gain in the percentage of days without grade 3 vomiting was $124.18 for India, $7451.63 for the United Kingdom, and $36,892.76 for the United States. The NMB for gain in QALY was $33.62, $1418.60, and $6727.01 for India, United Kingdom, and the United States, respectively. The estimates remained cost-effective across all scenarios of the sensitivity analyses. Aprepitant-based anti-emetic regimen is cost-effective for children receiving HEC. It results in overall cost savings and reduced healthcare-resource utilization.

Stick or twist? Cost-effectiveness of siponimod compared with continuing existing disease-modifying therapies in the treatment of active secondary progressive multiple sclerosis in the UK

Objective Identification of the phenotypic transition from relapsing-remitting multiple sclerosis (RRMS) to secondary progressive multiple sclerosis (SPMS) is often delayed due to disease complexity and an unwillingness to withdraw RRMS disease-modifying therapies (DMTs), driven by limited SPMS treatment options. Despite the paucity of clinical evidence for efficacy in patients with SPMS, DMTs licensed for RRMS are frequently continued into the early stages of SPMS. The cost-effectiveness of oral siponimod, an active SPMS DMT, versus continued oral or infused RRMS DMTs for patients with active SPMS, was evaluated. Methods A cohort Markov model based on disease progression through Expanded Disability Status Scale health states, with annual cycles and lifetime horizon, was employed to determine the cost-effectiveness of siponimod from a UK National Health Service (NHS) perspective for patients with active SPMS. Baseline characteristics, health state utility values, hazard ratios for time to 6-month confirmed disability progression, annualized relapse rate ratios and adverse events for siponimod were obtained from the phase 3 EXPAND clinical trial, supplemented by published literature. Published costs, resource use data and comparator efficacy data were obtained from the literature and, in the absence of data, reasonable assumptions were made. Results Quality-adjusted life years (QALYs) were greater for siponimod versus all comparators (3.45 versus 2.69–2.83). Incremental cost-effectiveness ratios (ICERs), calculated as cost per QALY, for siponimod versus natalizumab (dominant), ocrelizumab (£4,760), fingolimod (£10,033) and dimethyl fumarate (£15,837) indicated that siponimod was cost-effective at the commonly accepted willingness-to-pay threshold of £30,000/QALY. Conclusions Recognition of active SPMS and treatment of this phenotype with siponimod offers a cost-effective and clinically beneficial treatment approach compared with the continuation of oral or infused RRMS DMTs.

Utility values and its time to deterioration in breast cancer patients after diagnosis and during treatments.

The potential effects of breast cancer (BC) on health-related quality of life (HRQoL) should be considered in clinical and policy decision-making, as the economic burden of BC management is currently assessed. In the last decades, time-to-HRQoL score deterioration (TTD) has been proposed as an approach to the analysis of longitudinal HRQoL in oncology. The main objectives of the current study were to investigate the evolution of the utility values in BC patients after diagnosis and during follow-ups and to evaluate the TTD in utility values among women in all stages of BC. Health-state utility values (HSUV) were assessed using the EuroQol 5-Dimension 3-Level at diagnosis, at the end of the first hospitalization and 3 and 6months after the first hospitalization. For a given baseline score, HSUV was considered to have deteriorated if this score decreased by ≥ 0.08 points of the EQ-5D utility index score and ≥ 7 points of the EQ visual analogue scale. TTD curves were calculated using the Kaplan-Meier estimation method. Overall 381 patients were enrolled between February 2006 and February 2008. The highest proportions of respondents at the baseline and all follow-ups reporting some and extreme problems were in pain discomfort and anxiety/depression dimensions; more than 80% of patients experienced a deterioration in EQ-5D utility index score and EQ VAS score with a median TTD of 3.15months and 6.24 Months, respectively. BC patients undergoing therapy need psychological support to cope with their discomfort, pain, depression, anxiety, and fear during the process of diagnosis and treatment to improve their QoL.

Cost-effectiveness analysis of the Oncotype DX Breast Recurrence Score test in node-positive early breast cancer

Aims Given the high rate of adverse events and high cost of adjuvant chemotherapy, it is optimal to avoid its use when endocrine therapy is equally effective at preventing distant recurrence of early breast cancer. The Oncotype DX test is a predictive and prognostic multigene assay used to guide adjuvant chemotherapy decisions in early breast cancer based on a Recurrence Score (RS) result. A model-based cost-effectiveness analysis compared the Oncotype DX test to clinical risk tools alone for HR+/HER2– node-positive (1–3 axillary lymph nodes) early breast cancer patients based on results from the RxPONDER trial. Materials and methods A decision-tree and Markov model was developed in Microsoft Excel. Distributions of patients and distant recurrence probabilities with endocrine and chemo-endocrine therapy were derived from the RxPONDER trial, TransATAC and SWOG-8814. Chemotherapy assignment data were obtained from the Clalit registry. The cost of adjuvant chemotherapy was based on the distribution of treatments used in the UK combined with published drug unit costs in the UK. The cost of distant recurrence and health state utility values were obtained from literature. Results The Oncotype DX test was found to be more effective (with an estimated 0.02 additional QALYs) at a lower estimated cost (–£989) compared to clinical risk tools alone. The results did not substantially change with more conservative clinical and cost scenarios. The RxPONDER trial was restricted to RS 0–25, and data synthesis with other studies was required to inform the analysis, which increased uncertainty. Conclusions The Oncotype DX test is highly likely to be cost-effective in node-positive early breast cancer. The results were driven by reduction in the use of chemotherapy with consequence avoidance of the costs and harmful effects of chemotherapy. Targeted treatment of a minority (11%) of women with RS 26–100 who benefit from chemotherapy reduced cost and improved survival.