Value in Health最新文献

Background: Evidence that a healthcare intervention is clinically effective and cost-effective, and subsequently funded, does not guarantee adoption into routine clinical practice. Implementation science can improve adoption, although it also involves valuable resources. This study assessed the cost-effectiveness of a theory-based compared to an intuition-based implementation approach, using genetic testing for Lynch syndrome (LS) as a case-study.

Methods: A pragmatic two-arm cluster randomised controlled trial (the Hide and Seek Project, HaSP) compared the approaches across seven large Australian hospitals (N=3,321 patients). Costs associated with the implementation approaches and the strategies were collected during the trial, including initial once-off and ongoing strategy costs. Results were incorporated into a published microsimulation model (Policy1-Lynch). Costs were reported in 2021 Australian Dollars (AUD).

Results: Compared with the intuition-based approach, the theory-based approach was previously shown to significantly improve genetics services referral in high LS risk patients (p<0.0001). The within-trial analysis found that a theory-based approach reduced once-off plus ongoing implementation costs by AUD$0.75 per patient versus an intuition-based approach over five years. The modelled analysis estimated that a theory-based approach increased health system costs by AUD$103.74 per colorectal cancer patient, but resulted in fewer colorectal cancer deaths and more life years gained (LYG), and thus cost AUD$37,679 more per LYG than an intuition-based approach.

Conclusions: The theory-based approach resulted in more effective strategies and was cost-effective versus an intuition-based approach. Further research is needed to confirm these results in larger trials, other settings and conditions.

Objectives: We developed the methodology of welfare-based healthcare planning. For proof of concept, we empirically identified welfare-optimal hospital locations for thoracic surgical treatment of lung cancer (TSTLC) in Germany.

Methods: We used statutory health insurance data to estimate a volume-outcome model capturing the case-volume elasticity of the 1-year survival odds in patients with TSTLC. We conducted a discrete choice experiment to estimate the willingness to travel of representative (potential) patients for increases in the 1-year survival probability after TSTLC. Combining these results with a gravity model fitted to observed locations of patients and hospitals, we simulated different health planning scenarios (HPS) in 2035. For each HPS, we applied a Nash social welfare function to derive social welfare.

Results: Using data on 1449 patients with TSTLC treated in 189 hospitals, we estimated a case-volume elasticity of 0.27 (95% confidence interval [CI] = 0.07;0.46). The discrete choice experiment revealed that, for an increase in the 1-year survival probability from 90% to 91%, representative individuals would be willing to travel additional 66 minutes (95% CI = 45;93 minutes) when traveling 60 minutes and additional 23 minutes (95% CI = 18;33 minutes) when traveling 240 minutes. The top 1000 HPS according to welfare included between 15 and 22 hospitals. The welfare-optimal HPS included 19 hospitals with an average travel time of 54 minutes (status-quo HPS: 40 minutes) and a 1-year survival probability between 90.5% and 93.6% (status-quo HPS: 89.1%).

Conclusions: Our findings highlight the potential of welfare-based healthcare planning to increase the welfare of patients in Germany due to centralization of TSTLC.

Objectives: Existing personal health agency (ability to manage health on one's own) measures focus on adherence to disease-modifying treatments rather than accommodation of disease on one's own with optional visits, tests, and treatments as effective health strategy. This study aimed to develop a personal health agency measure with themes more relevant for discretionary care (taking control in self-management rather than adherence).

Methods: In this three-stage, cross-sectional study among 801 people seeking discretionary musculoskeletal specialty care, we performed sequential exploratory and confirmatory factor analyses starting with 33 items derived from legacy measures. We retained items that contributed to model fit (root mean square error of approximation < 0.05) accounting for favorable item discrimination, item difficulty, internal consistency, local dependencies, and differential item functioning. The findings were internally validated using 2 cohorts. We also assessed external validity (correlation with Patient Activation Measure [PAM]-13) and consistency (factors associated with new measure vs PAM-13).

Results: We identified 1 underlying theme and selected 9 items to represent the new Agency for Navigating Challenges, Health Ownership, and Resiliency (ANCHOR) measure. The 3-item, 6-item, and 9-item versions displayed excellent model fit (root mean square error of approximation < 0.001-0.035) and internal consistency (Cronbach alpha = 0.82-0.93) in internal validation. The floor effects were 2% to 3% and ceiling effects were 9% to 17%. The ANCHOR measures had moderate-to-strong correlations with PAM-13 (rho = 0.61-0.73) with similar external correlates, supporting external validity and consistency.

Conclusions: Enhancement of personal health agency-an important element of effective accommodation of symptoms over a lifetime-can be facilitated by routine measurement using ANCHOR. Agency-enhancing health strategies have the potential to support health while limiting discretionary use of resources.

Objectives: The diagnostic evaluation for abdominal and pelvic cancers without routine screening programs, such as pancreatic, liver, ovarian, and bladder cancers, can vary significantly in pathway and timing. Efficient and timely diagnosis is desirable in patients with a suspicion of cancer. We characterized real-world diagnostic pathways and examined the association of first imaging modality with measures of diagnostic efficiency in these cancers.

Methods: We conducted a retrospective cohort study using Surveillance, Epidemiology and End Results cancer registry data linked with Medicare claims from 2010 to 2019. Patients aged ≥65 years diagnosed with incident pancreatic, liver, ovarian, or bladder cancer were included. We estimated unadjusted timing, cost, and healthcare resource utilization from first imaging to diagnosis, and from diagnosis to treatment initiation by cancer type. Using negative binomial count models, we evaluated the association between computed tomography (CT) scan as the first imaging procedure and both time to diagnosis and number of imaging procedures used in the diagnostic pathway.

Results: Among 169 781 patients (48 751 pancreatic; 24 493 liver; 16 537 ovarian; and 80 000 bladder), CT scan was the most common initial imaging test (51%-65%). Patients who received a CT scan first experienced significantly shorter time to diagnosis (29-42 days faster) and required fewer imaging procedures (0.25-0.70 fewer) compared with other imaging modalities (P < .001). Higher comorbidity scores were associated with longer, more complex diagnostic pathways.

Conclusions: CT scans as the first imaging test were associated with greater diagnostic efficiency in abdominal and pelvic cancers. These findings provide a benchmark for evaluating emerging diagnostic technologies and optimizing cancer diagnostic pathways.

Objectives: The EU Health Technology Assessment Regulation (HTAR) introduces joint clinical assessments (JCAs) to harmonize clinical evidence evaluation across Member States. JCAs are commencing in 2026 for high-risk medical devices (MD) and in vitro diagnostics (IVD), but little is known about how national health technology assessment (HTA) bodies are preparing for implementation. This study aims to explore how selected European Union (EU)/European Economic Area HTA bodies are planning for JCAs integration and HTAR implementation for high-risk MD/IVD.

Methods: Semistructured interviews were conducted with 15 participants from 11 different HTA bodies across 11 EU/European Economic Area Member States. Interviews were recorded and transcribed verbatim before being anonymized and coded using NVivo Software. Data were analyzed using thematic analysis.

Results: All participants recognized JCAs as a potential tool for improving evidence quality for high-risk devices and highlighted their willingness to use JCA-generated evidence in their clinical assessment. Some HTA bodies have initiated procedural or legislative adaptations; however, many remain in an observational position, waiting for further implementation documents. Key opportunities identified included work sharing, improved evidence standards, and capacity building, particularly in smaller HTA systems that might not have access to high-quality assessments without JCAs. However, substantial challenges were reported, including regulatory uncertainty, timing misalignments, and limited manufacturer preparedness for JCA evidence demands.

Conclusions: HTAR is viewed as an opportunity to strengthen HTA practices for high-risk MD/IVD. However, realizing its goals will require further collaboration, alignment of procedural timelines, and investment in national capacity. Further studies are needed after full HTAR implementation for high-risk MD/IVD to capture ongoing experiences.

Objectives: This study aims to reperform cost-effective analyses (CEAs) that informed American Heart Association (AHA) guidelines between 2014 and 2022 using the health years in total (HYT) instead of the quality-adjusted life-year (QALY) to quantify the extent to which value assessment conclusions would change using a different metric.

Methods: We reanalyzed 21 CEAs that were referenced in clinical practice recommendations in AHA guidelines between 2014 and 2022. We then compared value conclusions based on cost-per-HYT with cost-per-QALY CEA results and assessed whether the change in metric affected value assessment classifications within the AHA-identified categories of high-, intermediate-, and low-value care (using $50 000/QALY and $150 000/QALY thresholds). In a sensitivity analysis, we applied an additional $100 000/QALY threshold to further subdivide intermediate-value care into intermediate-high and intermediate-low subcategories.

Results: There were 48 incremental cost-effectiveness ratios (ICERs) used to inform the AHA guidelines across the 21 CEAs we reanalyzed. We found that 96% of the value assessments classifications (46/48 ICERs) used in AHA clinical practice guidelines in 2014 to 2022 remained unchanged whether using QALY or HYT, with 90% (43/48 ICERs) remaining unchanged in the sensitivity analysis using the $100 000/QALY threshold.

Conclusions: We found a strong correlation between value assessments based on CEAs using the QALY versus the same CEA reanalyzed using the HYT. These findings suggest that current AHA recommendations would remain largely unaffected if they were informed by the HYT, a QALY alternative that does not discriminate against individuals at lower baseline health.

Objectives: Climate change has intensified heatwaves, posing significant health risks to older adults; yet, their impact on health-related quality of life (HRQoL) in China's aging population remains underexplored. This study investigates the impact of heatwaves on HRQoL among older adults using the EuroQol 5-Dimension 5-Level (EQ-5D-5L).

Methods: A cohort of community-dwelling Chinese adults aged ≥60 years were followed up from spring to fall in 2023. Univariate regression and restricted cubic spline models analyzed linear and nonlinear relationships between meteorological/demographic factors and EQ-5D-5L outcomes (utility values and EuroQol Visual Analog Scale [EQ VAS] scores). Machine learning models identified key predictors of heatwave-related HRQoL changes.

Results: Among the 627 participants, both EQ-5D-5L utility values and EQ VAS scores followed a rise-and-fall pattern, initially increasing during the early heatwave period (T1) but subsequently declining significantly during the peak heatwave period (T2) (P < .001). Underweight individuals had lower utility values (β = -0.394, P = .006), whereas obese individuals had lower EQ VAS scores (β = -0.385, P = .040). Age was negatively associated with both utility values (β = -0.003, P < .001) and EQ VAS scores (β = -0.228, P < .001). Meteorological factors, such as daily average relative humidity and temperature significantly affected both EQ-5D-5L utility values and EQ VAS scores (P < .05). The Support Vector Machine model outperformed the other machine learning models, identifying body mass index as the most influential variable in predicting the EQ-5D-5L utility values, followed by age, preretirement occupation, education level, and daily average relative humidity.

Conclusions: Heatwaves dynamically affect HRQoL in older adults in China, with an initial increase followed by a significant decline. Older, underweight, and less-educated adults are more vulnerable to the adverse impacts of heatwaves on HRQoL.

Objectives: Randomized controlled trials provide high internal validity but often have limited generalizability. Benchmarking real-world data against randomized controlled trial findings helps evaluate external validity. The objectives were to assess whether the results of the IXORA-S trial comparing ixekizumab and ustekinumab could be replicated using real-world data from the Shanghai Psoriasis Effectiveness Evaluation Cohort (SPEECH) and to explore potential reasons for differences between trial and observational findings.

Methods: We conducted a prospective cohort study of adults with moderate-to-severe plaque psoriasis enrolled in SPEECH (2021-2023) and treated with ixekizumab or ustekinumab. Propensity-score methods and multiple imputation were applied to address confounding and missing data. Outcomes were benchmarked against IXORA-S using agreement metrics and sensitivity analyses, including population trimming. The primary endpoint was the proportion of patients achieving a ≥90% reduction in the Psoriasis Area and Severity Index (PASI 90) at week 12.

Results: Among 367 patients (ixekizumab n = 203; ustekinumab n = 164), ixekizumab achieved higher PASI 90 response rates at week 12 compared with ustekinumab. Effect estimates were consistent with IXORA-S in direction, magnitude, and statistical significance. Differences in baseline eligibility criteria and disease severity explained part of the efficacy-effectiveness gap.

Conclusions: The results of IXORA-S were successfully replicated using real-world data from SPEECH. This demonstrates that registry-based observational data can yield reliable estimates of treatment effects and may be used to investigate effectiveness questions not feasible to study in randomized trials, such as long-term outcomes and effects in underrepresented populations.