Background and objective: Companion diagnostics (CDx) are critical to precision medicine. Developing and commercializing new CDx faces regulatory and economic challenges. This study aims to illustrate the utility of an early health technology assessment in quantifying the unmet clinical need and commercial opportunity created by the limited accuracy of existing programmed cell death ligand 1 CDx.
Methods: The study uses an early health technology assessment and market sizing to assess the potential value of a novel programmed cell death ligand 1 CDx for non-small cell lung cancer (NSCLC). Decision tree-based cost-effectiveness models were used to evaluate clinical and economic outcomes for improved programmed cell death ligand 1 testing in atezolizumab-treated patients with stage II-IIIA and metastatic NSCLC from a US payer perspective in 2023 US Dollars. Three strategies were examined: standard care, new CDx for cytology specimens only, and new CDx for all patients. Commercial opportunities from the perspectives of diagnostics and pharmaceutical manufacturers were assessed using headroom and threshold analyses.
Results: Headroom analyses indicated that a new CDx is not cost effective for metastatic NSCLC but holds significant value for stage II-IIIA NSCLC. Assuming perfect sensitivity and specificity, the incremental cost-effectiveness ratio for the new CDx in stage II-IIIA NSCLC was $57,650/quality-adjusted life-year and $54,950/quality-adjusted life-year for cytology specimens only and all patients, respectively. A threshold analysis showed that at a $500 price point, the new CDx is cost effective at sensitivity levels of 0.9 for all patients and 0.8 for cytology only. The total addressable US market for the CDx manufacturer was estimated at $2.6 million per year with a $500/test kit price.
Conclusions: A novel, highly accurate CDx for stage II-IIIA NSCLC could provide significant value to patients, payers, and manufacturers.
Purpose: There has been growing interest in understanding the economic impacts of loneliness and social isolation. This study updates a previous review on the economic costs of loneliness and social isolation and the cost effectiveness of related interventions.
Methods: We conducted a systematic search in the MEDLINE, PsycInfo, CINAHL, and Embase databases from 2018 to 13 August 2024, supplemented by a search of the grey literature. Studies included cost-of-illness studies, economic evaluations, and social return on investment (SROI) analyses published in the English language. All studies were evaluated for quality and summarised using a narrative approach. Costs reported were converted into US$, year 2024 values.
Results: In total, 15 studies were included: six cost-of-illness studies, four economic evaluations, and five SROI studies. Cost-of-illness studies primarily examined healthcare and productivity costs. All but one study reported excess costs linked to loneliness and social isolation, ranging from US$2 billion to US$25.2 billion per annum. Among four economic evaluations, three were model-based cost-utility or cost-effectiveness analyses (targeting older adults and the general population), and one was trial based (focusing on low-income individuals with health issues). One study found an intervention cost effective, whereas cost-effectiveness probabilities in others ranged from 54% to 68%. One study concluded that an intervention to reduce severe loneliness in older adults was cost effective but unlikely to be cost saving. All SROI studies reported positive returns, with SROI ratios ranging from US$2.28 to US$13.72.
Conclusion: This review highlights additional evidence on the economic burden of loneliness and social isolation. Future research should explore broader cost impacts beyond healthcare and expand cost-effectiveness studies to younger populations.
Background and aims: Many state Medicaid programs implemented sobriety restrictions that delay timely initiation of direct-acting antivirals (DAAs) for patients with hepatitis C virus (HCV) infections. This claims database study examined the economic impact of sobriety restrictions on DAAs among Medicaid-insured patients with HCV.
Methods: A retrospective database analysis of the Anlitiks All Payor Claims data (APCD) during the period January 1, 2020 to June 30, 2022 was conducted. Continuously enrolled adult (aged 18-64 years) Medicaid-insured patients with HCV who initiated DAAs (i.e., index date) during the period January 1, 2021 to December 31, 2021 with ≥ 12 months pre-index and ≥ 6 months post-index follow-up were categorized into two cohorts (states with sobriety restriction [SR] and states with no sobriety restriction [NSR]) based on the sobriety restriction status in the state of residence on the index date. Measures analyzed were the proportion of patients with one or more all-cause medical health care resource utilization (HCRU) (inpatient hospitalization [IP], emergency department [ED], outpatient [OP], professional office [PV], and other [OV] visits) and mean per-patient medical, pharmacy, and overall costs. HCRU and cost differences were compared using adjusted multivariable logistic and gamma-log link regression models, respectively.
Results: Patients in the SR (n = 2,295) versus NSR (n = 4,623) cohort had a higher mean age (45 ± 12.02 vs. 43 ± 11.51 years), fewer males (50.28% vs. 58.1%), and they had lower substance use rates (44.10% vs. 59.68%), all significant at p < 0.05. The SR vs. NSR cohort had higher rates of patients with all-cause HCRU by type (IP 22.0% vs.18.1%; ED 42.3% vs. 37.4; OP 62.5% vs. 55.4%; PV 76.4% vs. 69.1%; other visits 47.4% vs. 46.5%). The SR vs. NSR cohort had a significantly higher adjusted odds ratio (95% confidence interval) for IP (2.09; 1.59-2.73) and OP (1.52; 1.28-1.82). Similarly, the SR versus NSR cohort had a significantly higher all-cause adjusted least squares mean cost per patient for IP ($42,616 vs. $15,063), ED ($982 vs. $420), OP ($715 vs. $349), PV ($840 vs. $621), medical ($11,845 vs. $3,850), pharmacy ($53,453 vs. $38,298), and overall ($63,935 vs. $41,524).
Conclusion: Patients who initiated DAAs with SR versus NSR had 2 times and 1.5 times greater likelihood of IP and OP visits, respectively. Similarly, the SR versus NSR cohort had 3 times greater medical costs. Restricting DAA access among patients with HCV increases HCRU and cost burden, potentially impeding World Health Organization (WHO) 2030 HCV global elimination goals.
Background and objectives: Task shifting between different cadres of health worker has been proposed as an approach to address workforce shortages. Whether such reallocation is a useful strategy for a health system depends on the potential costs and consequences. Too narrow a focus has implications for population health as resources could be incorrectly directed towards inefficient activities owing to important costs and/or benefits being omitted from the evaluation. We aim to identify the key issues when evaluating the value for money of task shifting and review the applied literature to determine whether it is fit for purpose.
Methods: We developed an a priori logic model of task shifting and searched five databases (MEDLINE, Embase, EconLit, Social Sciences Citation Index and CEA Registry) for economic evaluations of task shifting published between 2014 and 2024. We performed forwards and backwards citation searching. We considered the scope of the evaluations with respect to the ability to capture key costs and outcomes of task shifting from the logic model. Reporting quality was assessed using the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) checklist.
Results: The rapid review identified 26 studies for inclusion covering 16 countries. Studies evaluated task shifting to community health workers and lay health workers as well as from doctors to radiographers, non-physician clinicians and nurse-midwives. The studies included health costs and outcomes but few included changes in the capacity of the workforce to undertake tasks, access, waiting times, productivity, burden on other staff, patient satisfaction, patient productivity and health equity concerns. There was a predominance for cost-effectiveness analysis to be used to assess the value for money of task shifting but the literature did include a cost-benefit analysis, a cost-consequence analysis and an extended cost-effectiveness analysis.
Conclusions: The majority of studies identified a range of costs and consequences that may only be appropriate for resource allocation under the strong assumption that all longer term costs and consequences would be unaffected by the task shift.
Introduction: Summary measures such as quality-adjusted life expectancy (QALE) are increasingly used to monitor health inequalities. Socioeconomic inequalities in health are well documented in Australia, including inequalities by education. However, estimates for QALE by level of education are lacking for Australia. We aimed to provide QALE stratified by age and sex across levels of educational attainment for the Australian population aged 25 years and above.
Methods: We categorized educational attainment as low (completed year 11 or below), intermediate (completed year 12 and/or other non-tertiary or vocational qualification) or high (completed a bachelor's degree or above). Mean Short-Form Six-Dimension health utility was estimated for sex- and education-specific subgroups from the Household, Income and Labour Dynamics in Australia survey (2022). We constructed life tables using age-sex-education-specific mortality rates for 2019 obtained from linked 2016 Census and Death Registrations data. Health utility was incorporated into the life tables to derive age- and sex-specific QALE across education levels.
Results: At age 25 years, males with high education had 7.3 years greater life expectancy than those with low education (61.0 versus 53.7 years undiscounted) and larger QALE (39.9 versus 28.8 years undiscounted), a gap of 11.1 years (39% relative difference). Females aged 25 years with a high level of education experienced 3.9 years greater life expectancy (LE; 63.1 versus 59.2 years, undiscounted) and an additional 7.6 years of QALE (36.9 versus 29.3 years, undiscounted), compared with those with low education, a 26% relative difference in QALE.
Conclusions: Significant disparities in QALE by educational attainment exist in Australia. These findings can inform policies aimed at reducing health inequity by guiding resource allocation and supporting future equity-informative economic evaluations.
Background and objective: The Centers for Medicare and Medicaid Services increasingly rely on real-world evidence to inform drug price negotiations under the Inflation Reduction Act. This study aims to evaluate methodological decisions that impact real-world comparative effectiveness outcomes using a case example of first-line pembrolizumab versus therapeutic alternatives in advanced non-small cell lung cancer among the Medicare population.
Methods: This study used a deidentified, electronic health record-derived, advanced non-small cell lung cancer dataset (2011-23) to analyze Medicare-eligible stage IV patients in three indications: (1) non-squamous, epidermal growth factor receptor, and anaplastic lymphoma kinase negative; (2) squamous; and (3) epidermal growth factor receptor and anaplastic lymphoma kinase negative with programmed death ligand-1 expression ≥1%. Indications (1)-(2) involved pembrolizumab combinations, while (3) referred to pembrolizumab monotherapy. Comparators included common non-platinum-based chemotherapy regimens. Propensity score-based inverse probability weighting was applied. The primary outcomes were real-world progression-free survival and overall survival. Scenario analyses examined the influence of time period selection, programmed death ligand-1 inclusion, therapeutic alternatives, and treatment switching on comparative effectiveness estimates.
Results: In the non-squamous cohort (1), overall survival benefits of pembrolizumab therapies compared to alternatives varied from a non-significant difference to an improvement of 2.7 months (95% confidence interval 1.2, 4.8), depending on analytical choices. In the squamous cohort (2), pembrolizumab combinations consistently demonstrated overall survival benefits, which ranged from 1.4 months (95% confidence interval 0.1, 3.0) to up to 3.6 months (95% confidence interval 0.1, 5.9). However, for pembrolizumab monotherapy (3), overall survival differences were statistically non-significant. Scenario analyses indicated substantial variability in outcomes based on methodological choices.
Conclusions: This study underscores the importance of transparent reporting and scenario analyses in real-world evidence to support Centers for Medicare & Medicaid Services decision making during drug price negotiations. Findings highlight the need for rigorous methodological standards to ensure the external validity of real-world evidence and its alignment with clinical practice.
Background and objective: A growing number of health technology assessment agencies recommend inclusion of informal carer outcomes in health economic evaluations. While generic preference-based measures (GPBMs) are favoured, the evidence regarding their performance in measuring the health-related quality of life of informal carers has not been synthesised. The aim of this systematic review was to synthesise the psychometric evidence of GPBMs in informal carers.
Methods: Following Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines, a literature search (indexed through October 2024) was conducted in CINAHL, PsycInfo, Embase and MEDLINE databases, supplemented with forward and backward citation searches. Publications were included that reported the psychometric performance of GPBMs in informal carers, regardless of care recipients' condition. Narrative synthesis was used to summarise the evidence. Quality of studies was evaluated using the COSMIN risk of bias checklist. International Prospective Register of Systematic Reviews (PROSPERO) registration is CRD42023434651.
Results: Twenty-one studies (published between 2001 and 2024) were identified, with nine evaluating multiple GPBMs (head-to-head comparisons). The EQ-5D 3-level (EQ-5D-3L) [n = 9] and EQ-5D 5-level (EQ-5D-5L) [n = 7] were the most frequently evaluated, followed by the Short-form 6-Dimension version 1 (SF-6Dv1) [n = 4], EuroQol Health and Wellbeing Short Form (EQ-HWB-9) [n = 4], Health Utilities Index (HUI) marks 2/3 (n = 3), Health-related Quality of Life Instrument with 8 Items (HINT-8) [n = 1] and Quality of Well Being Self-Administered (QWB-SA) [n = 1]. Studies were conducted in the USA (n = 6), UK (n = 4), China (n = 4), Australia (n = 3), Italy (n = 1), Iran (n = 1) and South Korea (n = 1), including a multi-country study (UK, Germany and France) study (n = 1). Care recipient conditions included carers of unspecified conditions, adults using long-term care, Alzheimer's disease or dementia, autism, cancer, leukaemia, craniofacial malformations, meningitis and multiple sclerosis. The EQ-5D-3L and EQ-5D-5L had evidence of ceiling effects at the index level. The EQ-5D-3L, EQ-5D-5L and EQ-HWB-9 demonstrated at least 'good' (intraclass correlation coefficient > 0.60) test-retest reliability. Known-group validity evidence was available for the EQ-5D-3L, EQ-5D-5L, EQ-HWB-9, HUI3 and SF-6Dv1 where each GPBM was able to discriminate over 60% of the groups (known or exploratory). Convergent validity studies reported that the EQ-5D-3L, EQ-5D-5L, EQ-HWB-9, HUI3, SF-6Dv1 and QWB-SA had moderate correlations with at least one care-specific preference-based measure (Adult Social Care Outcomes Toolkit for Carers [ASCOT-Carer], Care-Related Quality of Life [CarerQol] and Carer Experience Scale [CES]). Responsiveness was evaluated for the EQ-5D-5L, EQ-HWB-9 and SF-6Dv1 where mixed evidence was repor
Background: The Quality of Life Utility - Core 10 Dimensions (QLU-C10D) is a disease-specific preference-based measure (PBM) designed to obtain health state utility values from patients with cancer. Previously, satisfactory psychometric properties were established from retrospective trial analyses using clinical anchors. This study aimed to validate the QLU-C10D against two generic PBMs in a prospective sample of Austrian and French patients with cancer using patient-reported anchors.
Methods: Patients completed the European Organisation of Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire - Core 30 (QLQ-C30), EQ-5D-5L and Short Form 36 (SF-36) at study baseline (any time during anti-cancer treatment) plus a follow-up assessment 3-6 months later. Sociodemographic and clinical characteristics were assessed. QLU-C10D and SF-6Dv2 utilities were calculated from QLQ-C30 and SF-36 data, respectively. German, French and UK value sets were applied for all three PBMs. Floor and ceiling effects were assessed. Known-group validity (independent t-test) and responsiveness (paired t-tests) were assessed respectively by ability to detect health status differences and changes over time according to patient-rated overall quality of life/health perception assessed by the QLQ-C30 Global Health Status scale, the EQ-5D-5L VAS and the SF-36 General Health scale.
Results: A total of 465 patients were included in the analysis. QLU-C10D index scores (intra-class correlation) and domains (Pearson) were correlated with EQ-5D-5L and Short-Form Six Dimensions (SF-6Dv2) conceptual counterparts. Correlation coefficients for the index scores of QLU-C10D and the generic PBMs ranged from 0.63 to 0.81. The QLU-C10D detected statistically significant differences between groups at baseline in 100% of tests performed (n = 27). For changes over time, QLU-C10D detected expected effects in 68% of cases (n = 29). In comparison with the generic PBMs, QLU-C10D detected differences and changes with a higher statistical efficiency in 76% of cases (77 of 102).
Conclusions: The QLU-C10D is a fit-for-purpose ready-to-use PBM to estimate health state utilities of patients with cancer. This study adds to evidence that QLU-C10D has appropriate psychometric properties and appears to have higher statistical efficiency than generic PBMs in cancer.
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