Applied Health Economics and Health Policy最新文献

Introduction

Consumers may purchase commercial diagnostic tests (CDT) without prior doctor consultation. This paper analyzes three CDT markets—commercial cholesterol tests (CCT), direct-to-consumer genetic health tests (DGT) and total body scans (TBS)—in the context of the universal, collectively financed health care system of the Netherlands.

Methods

An online willingness-to-pay (WTP) questionnaire was sent to a representative sample of 1500 Dutch consumers. Using contingent valuation (CV) methodology, an array of bids for three self-tests were presented to the respondents. The results were extrapolated to the Dutch population and compared to current prices and follow-up medical utilization, allowing analysis from a societal perspective.

Results

Overall, 880 of 1500 respondents completed the questionnaire (response rate 59%). Of the respondents, 26–44% were willing to pay a positive amount for the CDT. Willingness-to-pay was correlated to age and household income, but not to health status or prior experience with these tests. At mean current prices of €29 for CCT, €229 for DGT and €1,650 for TBS, 3.3%, 2.5%, and 1.1%, were willing to purchase a CCT, DGT, and TBS, respectively. All three CDT resulted in net costs to the health system, estimated at €5, €16, and €44 per test, respectively. Reducing volumes by 90,000 CCTs (19%), 19,000 DGTs (5%) and 4,000 TBSs (2.5%) in 2019 would optimize welfare.

Conclusion

Most respondents were unwilling to consume CDT at any price or only if the CDT were provided for free. However, for a small group of consumers, societal costs exceed private benefits. Therefore, CDT regulation could provide small welfare gains.

Background and Objective

Deep brain stimulation (DBS) is an established treatment for Parkinson’s disease (PD) in patients with advanced motor symptoms with an inadequate response to pharmacotherapies. Despite its effectiveness, the cost effectiveness of DBS remains a subject of debate. This systematic review aims to update and synthesize evidence on the cost effectiveness of DBS for PD.

Methods

To identify full economic evaluations that compared the cost effectiveness of DBS with other best medical treatments, a comprehensive search was conducted of the PubMed, Embase, Scopus, and Tufts Cost-Effective Analysis registry databases. The selected papers were systematically reviewed, and the results were summarized. For the quality appraisal, we used the modified economic evaluations bias checklist. The review protocol was a priori registered with PROSPERO, CRD42022345508.

Results

Sixteen identified cost-utility analyses that reported 19 comparisons on the use of DBS for PD were systematically reviewed. The studies were primarily conducted in high-income countries and employed Markov models. The costs considered were direct costs: surgical expenses, calibration, pulse generator replacement, and annual drug expenses. The majority of studies used country-specific thresholds. Fourteen comparisons from 12 studies reported on the cost effectiveness of DBS compared to best medical treatments. Eleven comparisons reported DBS as cost effective based on incremental cost-utility ratio results.

Conclusions

The cost effectiveness of DBS for PD varies by time horizon, costs considered, threshold utilized, and stage of PD progression. Standardizing approaches and comparing DBS with other treatments are needed for future research on effective PD management.

Background and Objective

Adding gene expression profiles (GEPs) to the current diagnostic work-up of aggressive large B-cell lymphomas may lead to the reclassification of patients, treatment changes and improved outcomes. A GEP test is in development using TempO-Seq^® technology to distinguish Burkitt lymphoma (BL) and primary mediastinal large B-cell lymphoma (PMBCL) from diffuse large B-cell lymphoma (DLBCL), and to classify patients with DLBLC and to predict the benefit of (e.g.) adding bortezomib to R-CHOP therapy (RB-CHOP). This study aims to estimate the potential impact of a GEP test on costs and health outcomes to inform pricing and evidence generation strategies.

Methods

Three decision models were developed comparing diagnostic strategies with and without GEP signatures over a lifetime horizon using a UK health and social care perspective. Inputs were taken from a recent clinical trial, literature and expert opinion. We estimated the maximum price of the test using a threshold of Great Britain Pound (GBP) 30,000 per quality-adjusted life-year (QALY). Sensitivity analyses were conducted.

Results

The estimated maximum threshold price for a combined test to be cost effective is GBP 15,352. At base-case values, the BL signature delivers QALY gains of 0.054 at an additional cost of GBP 275. This results in a net monetary benefit at a threshold of GBP 30,000 per QALY of GBP 1345. For PMBCL, the QALY gain was 0.0011 at a cost saving of GBP 406 and the net monetary benefit was GBP 437. The hazard ratio for the impact of treating BL less intensively must be at least 1.2 for a positive net monetary benefit. For identifying patients with the DLBCL subtype responsive to bortezomib, QALY gain was 0.2465 at a cost saving of GBP 6175, resulting in a net monetary benefit of GBP 13,570. In a probabilistic sensitivity analysis using 1000 simulations, a testing strategy was superior to a treat all with R-CHOP strategy in 81% of the simulations and with a cost saving in 92% assuming a cost price of zero.

Conclusions

Our estimates show that the combined test has a high probability of being cost effective. There is good quality evidence for the benefit of subtyping DLBCL but the evidence on the number of patients reclassified to or from BL and PMBCL and the impact of a more precise diagnosis and the cost of treatment is weak. The developers can use the price estimate to inform a return on investment calculations. Evidence will be required of how well the TempO-Seq^® technology performs compared to the testing GEP technology used for subtyping in the recent clinical trial. For BL and PMBCL elements of the test, evidence would be required of the number of patients reclassified and improved costing information would be useful. The diagnostic and therapeutic environment in haematological malignancies is fast moving, which increases the risk for developers of diagnostic tests.

Background

Self-reported service use informs resource utilisation and cost estimates, though its validity for use within economic evaluations is uncertain.

Objective

The aim of this study is to assess agreement in health resource-use measurement between self-reported and administrative data across different resource categories, over time and between different recall periods by subgroups among Australians living with psychosis.

Methods

Data were obtained for 104 participants with psychotic disorders from a randomised controlled trial. Agreement between self-reported resource-use questionnaires and administrative data on community-based services and medication use was assessed through estimating differences of group mean number of visits and medications used and intraclass correlation coefficients (ICC) over multiple time periods.

Results

ICC showed moderate agreement across most time periods for general practitioners, psychiatrists and mental health medications. No clear trends were discernible over time, between varying lengths of recall periods nor across participant subgroups.

Conclusion

Despite poor agreement, when measuring visits to psychologists and other health professionals, small overall differences in group mean number of visits indicate that self-reported data may still be valid for use in economic evaluations in people living with psychosis.

In Australia, local health services with allocated budgets manage public hospital services for defined geographical areas. The authors were embedded in a local health service for around 2 years and undertook a range of local level economic evaluations for which three decision contexts were defined: intervention development, post-implementation and prioritisation. Despite difficulties in estimating opportunity costs and in the relevance of portfolio-based prioritisation approaches, economic evaluation added value to local decision-making. Development-focused (ex ante) economic evaluations used expert elicitation and calibration methods to synthesise published evidence with local health systems data to evaluate interventions to prevent hospital acquired complications. The use of economic evaluation facilitated the implementation of interventions with additional resource requirements. Decision analytic models were used alongside the implementation of larger scale, more complex service interventions to estimate counterfactual patient pathways, costs and outcomes, providing a transparent alternative to the statistical analyses of intervention effects, which were subject to high risk of bias. Economic evaluations of more established services had less impact due to data limitations and lesser executive interest. Prioritisation-focused economic evaluations compared costs, outcomes and processes of care for defined patient populations across alternative local health services to identify, understand and quantify the effects of unwarranted variation to inform priority areas for improvement within individual local health services. The sustained use of local level economic evaluation could be supported by embedding health economists in local continuous improvement units, perhaps with an initial focus on supporting the development and evaluation of prioritised new service interventions. Shared resources and critical mass are important, which could be facilitated through groups of embedded economists with joint appointments between different local health services and the same academic institution.

Objective

This study aimed to estimate the societal cost of racial disparities in pneumococcal disease among US adults aged ≥  50 years.

Methods

In a model-based analysis, societal costs of invasive pneumococcal disease (IPD) and hospitalized nonbacteremic pneumococcal pneumonia (NBP) were estimated using (1) direct medical costs plus indirect costs of acute illness; (2) indirect costs of pneumococcal mortality; and (3) direct and indirect costs of related disability. Disparities costs were calculated as differences in average per-person pneumococcal disease cost between Black and non-Black adults aged ≥  50 years multiplied by the Black population aged ≥  50 years. Costs were in 2019 US dollars (US$), with future costs discounted at 3% per year.

Results

Total direct and indirect costs per IPD case were US$186,791 in Black populations and US$182,689 in non-Black populations; total hospitalized NBP costs per case were US$100,632 (Black) and US$96,781 (non-Black). The difference in population per-person total pneumococcal disease costs between Black and non-Black adults was US$47.85. Combined societal costs of disparities for IPD and hospitalized NBP totaled US$673.2 million for Black adults aged ≥  50 years. Disease and disability risks, life expectancy, and case-fatality rates were influential in one-way sensitivity analyses, but the lowest cost across all analyses was US$194 million. The 95% probability range of racial disparity costs were US$227.2–US$1156.9 million in a probabilistic sensitivity analysis.

Conclusions

US societal cost of racial pneumococcal disease disparities in persons aged ≥ 50 years is substantial. Successful pneumococcal vaccination policy and programmatic interventions to mitigate these disparities could decrease costs and improve health.

Introduction

Funding decisions for many health technologies occur without undergoing health technology assessment (HTA), in particular, without assessment of cost effectiveness (CE). Immunoglobulins in Australia are an interesting case study because they have been used for a long time for various rare disorders and their price is publicly available. Undertaking an HTA enables us to assess CE for an intervention for which there is limited clinical and economic evidence. This study presents a post-market review to assess the CE of immunoglobulins for the treatment of multifocal motor neuropathy (MMN) compared with best supportive care.

Methods

A Markov model was used to estimate costs and quality-adjusted life-years (QALYs). Input sources included randomised controlled trials, single-arm studies, the Australian clinical criteria for MMN, clinical guidelines, previous Medical Services Advisory Committee (MSAC) reports and inputs from clinical experts. Sensitivity analyses were conducted to assess the uncertainty and robustness of the CE results.

Results

The cost per patient of treating MMN with immunoglobulin was AU$275,853 versus AU$26,191when no treatment was provided, with accrued QALYs of 6.83 versus 6.04, respectively. The latter translated into a high incremental cost-effectiveness ratio (ICER) of AU$317,552/QALY. The ICER was most sensitive to the utility weights and the price of immunoglobulins. MSAC advised to continue funding of immunoglobulins on the grounds of efficacy, despite the high and uncertain ICER.

Conclusions

Beyond the ICER framework, other factors were acknowledged, including the high clinical need in a patient population for which there are no other active treatments available. This case study highlights the challenges of conducting HTA for already funded interventions, and the efficiency trade-offs required to fund effective high-cost therapies in rare conditions.