Value in Health最新文献

Objectives: Health-related quality-of-life instruments for cardiovascular diseases (CVDs) have been commonly used to measure important patient-reported outcomes in clinical trials and practices. This study aimed to systematically identify and evaluate the psychometric properties of CVD-specific health-related quality-of-life instruments.

Methods: We searched cumulative index to nursing and allied health literature, Embase, and PubMed from inception to January 20, 2022. Studies that reported psychometric properties of CVD-specific instruments were included. Two reviewers independently assessed the methodological quality using the Consensus-based Standards for the Selection of Health Measurement Instruments methods for evaluating measurement properties and quality of evidence. Seven psychometric properties, including structural validity, internal consistency, test-retest reliability, convergent validity, divergent validity, discriminative validity, and responsiveness, were evaluated.

Results: We identified 142 studies reporting psychometric properties of 40 instruments. Five (12.5%) instruments demonstrated measurement properties with sufficient or inconsistent ratings; 16 (40.0%) instruments did not report any responsiveness evidence. Of the 40 instruments, 15 (37.5%) instruments were rated sufficient with high quality of evidence on internal consistency; 4 (10.0%) on structural validity, convergent validity and divergent validity; and 3 (7.5%) on discriminative validity.

Conclusions: When measuring patient-reported outcomes in clinical trials or routine practice, it is important to choose instruments with established psychometric properties.

Objectives: This study aimed to evaluate the cost-effectiveness of an extended-role general practitioner symptoms clinic (SC), added to usual care (UC) for patients with multiple persistent physical symptoms (sometimes known as medically unexplained symptoms).

Methods: This was a 52-week within-trial cost-utility analysis of a pragmatic multicenter randomized controlled trial comparing SC + UC (n = 178) with UC alone (n = 176), conducted from the primary perspective of the UK National Health Service and personal and social services (PSS). Base-case quality-adjusted life-years (QALYs) were measured using EQ-5D-5L. Missing data were imputed using multiple imputation. Cost-effectiveness results were presented as incremental cost-effectiveness ratios and incremental net monetary benefits. Uncertainty was explored using cost-effectiveness acceptability curves (using 1000 nonparametric bootstrapped samples) and sensitivity analysis (including societal costs, using SF-6D and ICECAP-A capability measure for adults outcomes to estimate QALYs and years of full capability, respectively, varying intervention costs, missing data mechanism assumptions).

Results: Multiple imputation analysis showed that compared with UC alone, SC + UC was more expensive (adjusted mean cost difference: 704; 95% CI £605-£807) and more effective (adjusted mean QALY difference: 0.0447; 95% CI 0.0067-0.0826), yielding an incremental cost-effectiveness ratio of £15 765/QALY, incremental net monetary benefit of £189.22 (95% CI -£573.62 to £948.28) and a 69% probability of the SC + UC intervention arm being cost-effective at a threshold of £20 000 per QALY. Results were robust to most sensitivity analyses but sensitive to missing data assumptions (2 of the 8 scenarios investigated), SF-6D, and ICECAP_A capability measure for adults quality-of-life outcomes.

Conclusions: A symptoms clinic is likely to be a potentially cost-effective treatment for patients with persistent physical symptoms.

Objectives: Rising out-of-pocket (OOP) costs paid by healthcare consumers can inhibit access to necessary healthcare. Yet, it is unclear if higher OOP payments are associated with better care quality. This study aimed to identify the individual and socio-contextual predictors of OOP costs and to explore the association between OOP costs and quality of care outcomes for 4 surgical procedures.

Methods: A retrospective cohort analysis was conducted using data from Medibank Private health insurance members aged ≥18 years who underwent hip replacement, knee replacement, cholecystectomy, and radical prostatectomy during 2015 to 2020 across >300 hospitals in Australia. Healthcare quality outcomes investigated were hospital-acquired complications, unplanned intensive care unit admissions, prolonged length of stay, and readmissions within 28 days. Socio-contextual determinants of OOP costs examined were patient demographics, socioeconomic status, health insurance, and procedure complexity. Generalized linear mixed modeling examined the risk of each outcome, adjusting for covariates and considering patient clustering within surgeons and hospitals.

Results: Patients were more likely to pay OOP costs if they were aged 65 to 74 years compared with aged 18 to 44 years for all 4 surgical procedures. No association between OOP payments and the risk of hospital-acquired complications, intensive care unit admission, or hospital readmission was identified. Patients who paid OOP costs were less likely to have a prolonged length of stay for all 4 procedure types.

Conclusions: Higher OOP payments were not linked to improved care quality except for shorter hospital stays. Greater transparency on OOP costs is needed to inform consumer decisions.

Objectives: Engaging patients with patient-reported outcome measures (PROMs) is a widely recognized and pressing challenge, yet our understanding of how to achieve this is limited. This study investigated strategies implemented by a Dutch university hospital aimed at enhancing response rates among outpatients from nearly 70 subdepartments. Response rates improved, but remained below desired levels. To deepen understanding and inform future strategies, we identified patient and consultation characteristics associated with response behavior.

Methods: We investigated strategies and their underlying rationales through a document analysis of internal hospital documentation (2020-2023) using the COM-B model. We exploited electronic health record data to identify patient and consultation characteristics associated with PROMs completion, estimating a multivariate logistic regression model (n = 46 468 outpatient consultations).

Results: Thirteen strategies targeted outpatients' capability, opportunity, and motivation to complete PROMs. In 2023, PROMs were completed in more than half of the 46 468 unique consultations (56%) for which a PROM was sent. Challenges persisted in establishing effective feedback mechanisms and accommodating non-Dutch-speaking patients. The multivariate analysis showed a significantly higher response among patients of high or middle socioeconomic status and those with an in-person consultation, ie, not using telehealth. Women, patients attending a follow-up visit, or those having their consultation on a Friday were slightly less likely to complete PROMs.

Conclusions: Response rates to PROMs improved but remained below desired levels, despite multiple strategies. Hospitals may benefit from effective patient feedback on PROMs and tailoring strategies to engage specific patient groups. These approaches can enhance successful implementation and promote equity in value-based healthcare.

Objectives: This study aimed to quantify the burden of 8 noncommunicable conditions on productivity-adjusted life-years (PALYs) at work and within the household among the Finnish general adult population.

Methods: Survey data on 18- to 79-year-old Finnish respondents collected in 2022 were used to calculate age- and sex-specific productivity indices at work and within the household using 0- and 1-inflated beta regression for individuals with and without a certain condition (asthma or chronic obstructive pulmonary disease, cardiovascular disease, depression or other mental health problem, diabetes, gastrointestinal disease, hypothyroidism or other thyroid disease, migraine or other chronic headache, and musculoskeletal disease). Age and sex distributions of the Finnish population obtained from Statistics Finland together with the prevalence of the condition and the estimated productivity indices were used to produce the population-level 1-year losses in PALYs at work and within the household.

Results: Among 8 conditions, depression and other mental health problems had the highest PALY losses (99 570 PALY loss burden at work, 256 086 PALY loss at home, and 250 980 PALY loss in general adult populations), with diabetes having the lowest (3666 PALY loss burden at work, 46 344 PALY loss at home, and 43 443 PALY loss in general adult populations). All the examined conditions were as significant in affecting both the productivity at work and the within-household productivity.

Conclusions: Depression and other mental health problems have a major effect on self-reported work ability and productivity compared with other chronic conditions.

Objectives: The overdose epidemic continues to be one of the leading causes of death in North America and continues to contribute to high healthcare costs. Although harm reduction initiatives have significantly reduced the aforementioned costs, there is a dearth of evidence regarding overdose response hotlines and applications. We aim to evaluate the social return on investment from a payer perspective of one such overdose response hotline, Canada's National Overdose Response Service, and its implications for service users, service operators, the Canadian healthcare system, and program funders.

Methods: Outcome variables determined from theory of change models were developed in consultation with the aforementioned vested interest groups. Proxy values were attributed to each variable identified through values present within existing literature and databases. These values were then compared with operational costs accounting for deadweight, attribution, and displacement to determine a final social return on investment ratio. A discount rate was then applied based on the influence of risk on the outcome achieved.

Results: The ratio illustrating the value created for all stakeholders, resulting from the $1 592 000 investment made over 2 years, is $15.84 per single dollar invested. The value generated stems primarily from overdose prevention, mental health support, staff employment, reductions in emergency service utilization, service referrals, and volunteer well-being, which outweigh costs including operational funding, work-related stressors, compassion fatigue, and false calls.

Conclusions: The results of our study demonstrate that the National Overdose Response Service provides a social value that far outweighs the costs attributed to the program's operation.

Objectives: This study aimed to assess the practicality, validity, and responsiveness of the proxy Child Health Utility-9 Dimensions (CHU9D) in children aged 2 to 5 years.

Methods: We used data from the Barrier Enhancement for Eczema Prevention trial, a UK randomized controlled trial testing whether daily emollients in infancy could prevent eczema in high-risk infants. The main parent/carer completed the proxy CHU9D using developers' additional guidance for completion in those younger than 5 years and the Patient-Oriented Eczema Measure (POEM) at ages 2, 3, 4, and 5 years. Practicality was assessed by completion rates. Construct validity assessed whether CHU9D could discriminate between those with/without eczema and between eczema severity levels on POEM. Responsiveness was determined by ability to discriminate between 3 groups: (1) those whose POEM score deteriorated ≥3 points, (2) those whose change was not clinically important (-2.9 to 2.9 points), and (3) those whose POEM score improved ≥3 points. Analysis was conducted in Stata 17.

Results: Of 1394 children participating in the Barrier Enhancement for Eczema Prevention trial, study questionnaires were completed by 1212 (87%), 981 (70%), 990 (71%), and 976 (70%) at 2, 3, 4, and 5 years. Of these the CHU9D was completed by 1066 (88.0%), 685 (69.8%), 925 (93.4%), and 923 (94.6%), respectively. Mean utility at all time points was approximately 0.934 (range 0.443-1). For construct validity, very small differences in the CHU9D between known groups were observed (P < .01). A total of 801 participants had responsiveness data: 13% deteriorated, 72% had nonclinically important change, and 15% improved. Mean utility change (standardized response mean) for these groups was -0.0198 (0.21), 0.0041 (0.05), and 0.0175 (0.21) showing small change and small responsiveness.

Conclusions: Proxy CHU9D in 2- to 5-year-old children shows potential but further research is needed.

Objectives: There is growing recognition of the consequences of a person's health and illness experience for the health and wider welfare of those close to them. However, estimation of these health spillovers is challenging. This study adopts a longitudinal approach to examine maternal mental health spillovers associated with various forms of child illness and disability.

Methods: Dynamic panel models are used in estimating maternal mental health spillovers related to 7 subcategories of chronic child illness and disability. In particular, we use longitudinal data from the Growing Up in Ireland study and a system generalized method of moments approach. We also consider heterogeneity in these spillovers by the severity of the child's illness/disability and by household deprivation.

Results: We find that a child's experience of chronic nervous system conditions and chronic mental and behavioral disorders are associated with 10.8 and 5.1 percentage point increases in the probability of maternal depression, respectively. Similar associations were not observed for other health conditions. Spillover magnitude is also found to be strongly related to illness/disability severity. Finally, subsample analyses reveal a larger association between severe child illness and maternal depression among deprived households.

Conclusions: This analysis, in observing health spillovers related to certain disease categories but not others, draws further attention to their context specificity. Our findings also further corroborate calls for inclusion of caregiver and family member outcomes in the economic evaluation of child health services and support consensus guidelines for collection of these outcomes alongside patient outcomes in clinical trials.

Objectives: Real-world evidence (RWE) is valuable in supporting regulatory and health technology assessment (HTA) decisions; however, the actual contribution to approvals remains elusive. This study aimed to review RWE approaches and use in oncology medicine approvals in Europe and understand cohesion and discrepancy in the acceptance of the RWE by the European Medicines Agency (EMA) and European HTA bodies.

Methods: This scoping review involved a search of the EMA database, National Institute for Health and Care Excellence (NICE), Gemeinsamer Bundesausschuss (G-BA), and Haute Autorité de Santé (HAS) websites to identify final reports and appraisals for oncology medicines with references to RWE. The selection was guided by research terms associated with RWE study designs, data sources, and outcomes. Qualitative analysis was used to systemize the data. Case studies assessed by more than one agency were selected for comparative assessment of RWE approach, use, and acceptability.

Results: RWE was mainly leveraged as an external control for indirect treatment comparisons or contextualization to support clinical trial results by the EMA, NICE, G-BA, and HAS. However, this approach was mostly rejected due to methodology biases. Comparative assessment of RWE acceptability for the same oncology medicines across agencies suggests discrepancies between EMA and European HTA bodies and among NICE, G-BA, and HAS.

Conclusions: There is diverging acceptance of RWE in EMA and European HTA bodies with no clear consensus on the most effective way to leverage RWE in approvals. With the introduction of the joint European Union Joint Clinical Assessment in 2025, it is crucial for European HTA bodies and EMA to develop synergetic standards for the use of RWE to ensure equitable and timely access to medicines.