Applied Health Economics and Health Policy最新文献

Objective

This study leveraged data from 11 independent international diabetes models to evaluate the impact of unrelated future medical costs on the outcomes of health economic evaluations in diabetes mellitus.

Methods

Eleven models simulated the progression of diabetes and occurrence of its complications in hypothetical cohorts of individuals with type 1 (T1D) or type 2 (T2D) diabetes over the remaining lifetime of the patients to evaluate the cost effectiveness of three hypothetical glucose improvement interventions versus a hypothetical control intervention. All models used the same set of costs associated with diabetes complications and interventions, using a United Kingdom healthcare system perspective. Standard utility/disutility values associated with diabetes-related complications were used. Unrelated future medical costs were assumed equal for all interventions and control arms. The statistical significance of changes on the total lifetime costs, incremental costs and incremental cost-effectiveness ratios (ICERs) before and after adding the unrelated future medical costs were analysed using t-test and summarized in incremental cost-effectiveness diagrams by type of diabetes.

Results

The inclusion of unrelated costs increased mean total lifetime costs substantially. However, there were no significant differences between the mean incremental costs and ICERs before and after adding unrelated future medical costs. Unrelated future medical cost inclusion did not alter the original conclusions of the diabetes modelling evaluations.

Conclusions

For diabetes, with many costly noncommunicable diseases already explicitly modelled as complications, and with many interventions having predominantly an effect on the improvement of quality of life, unrelated future medical costs have a small impact on the outcomes of health economic evaluations.

Background and Objective

Patients may get more treatment options with off-label use of drugs while exposed to unknown risks of adverse events. Little is known about the public or demand-side perspective on off-label drug use, which is important to understand how to use off-label treatment and devise financial assistance. This study aimed to quantify public preference for off-label cancer treatment outcomes, process, and costs, and perceived importance of associated adverse events.

Methods

A discrete choice experiment and a best-worst scaling were conducted in Hong Kong in December 2022. Quota sampling was used to randomly select the study sample from a territory-wide panel of working-age adults. Preferences and willingness to pay (WTP) for treatment effectiveness, risk of adverse events, mode of drug administration, and availability of off-label treatment guidelines were estimated using a random parameter logit model and latent class model. The relative importance of different adverse events was elicited using Case 1 best-worst scaling.

Results

A total of 435 respondents provided valid responses. In the discrete choice experiment, the respondents indicated that extra overall survival as treatment effectiveness (WTP: HK$448,000/US$57,400 for 12-month vs 3-month extra survival) was the most important attribute for off-label drugs, followed by the risk of adverse events (WTP: HK$318,000/US$40,800 for 10% chance to have adverse event vs 55%), mode of drug administration (WTP: HK$42,000/US$5300 for oral intake vs injection), and availability of guidelines (WTP: HK$31,000/US$4000 for available versus not available). Four groups with distinct preferences were identified, including effectiveness oriented, off-label use refusal, oral intake oriented, and adverse event risk aversion. In the best-worse scaling, hypothyroidism, nausea/vomiting, and arthralgia/joint pain were the three most important adverse events based on the perceptions of respondents. Risk-averse respondents, who were identified from the discrete choice experiment, had different perceived importance of the adverse events compared with those with other preferences.

Conclusions

Knowing the preference and WTP for cancer treatment-related characteristics from a societal perspective facilitates doctors’ communications with patients on decision making and treatment goal-setting for off-label treatment, and enables devising financial assistance for related treatments. This study also provides important insight to inform evaluations of public acceptance and information dissemination in drug development as well as future economic evaluations.

Objective

The aim of this study was to examine the association between characteristics of novel drugs and incremental health gains relative to standard of care, in terms of quality-adjusted life-years (QALYs).

Methods

This study’s unit of analysis is the drug–indication pair. For pairs approved by the US FDA from 1999 to 2018, we quantified incremental health gains using QALYs from the published literature and characterized each pair’s novelty in terms of a series of six binary (yes/no) characteristics of novel drugs given special consideration by Health Technology Assessment agencies: Novel mechanism of action, Indicated for a rare disease, Indicated for a pediatric population, Treats a serious condition, Offers meaningful improvement over available therapies, and Potential to address unmet clinical needs. We analyzed measures of bivariate association (Mann-Whitney U and Kolmogorov-Smirnov tests) and multivariable regression, accounting for the influence of multiple novelty characteristics simultaneously.

Results

Our sample of 146 drugs represents 21% of drugs approved the FDA in the time period (1999–2018). Median and mean QALY gains for ‘novel’ drug–indication pairs exceeded corresponding QALY gains for non-novel drug–indication pairs. For most comparisons, the bivariate relationships between QALY gains and novelty characteristics were significant at p < 0.05 except for novel mechanism of action (Kolmogorov-Smirnov test) and pediatric indication (both bivariate tests). Multivariable models revealed an independent association between novelty characteristics and QALY gain except for unmet clinical need and indicated for a rare disease.

Conclusions

Drugs with novelty characteristics conferred larger health gains than drugs without these characteristics in bivariate analysis, multivariable models, or both. Future research should examine other aspects of drug novelty, such as patient and health system costs and equitable access.

Background

With its clear focus on financial protection, government-funded health insurance (GFHI) stands out among the strategies for universal health coverage (UHC) implemented by low-to-middle income countries globally. Since 2018, India has implemented a GFHI programme called the Ayushman Bharat Pradhan Mantri Jan Arogya Yojana (AB-PMJAY), which covers 500 million individuals. The current study aims to evaluate the performance of GFHI in meeting its key objectives of improving access, quality and financial protection for hospital-based care in two large central Indian states: Madhya Pradesh and Maharashtra.

Methods

The study measures access in terms of utilisation of inpatient care. Financial protection was measured in terms of catastrophic health expenditure which was defined as the incidence of out-of-pocket expenditure (OOPE) above thresholds of 10% and 25% of annual household expenditure. Patient-satisfaction with care was taken as an indicator of quality. A household survey was conducted in 2023, covering a multi-stage sample of 11,569 and 12,384 individuals in Madhya Pradesh and Maharashtra, respectively. Multi-variate analyses were conducted to find the effect of GFHI-enrolment on the desired outcomes. The instrumental variable method was applied to address potential endogeneity in insurance enrolment. Additionally, propensity score matching was done to ensure robustness.

Results

Around 71% and 63% of surveyed individuals were enrolled under GFHI in Madhya Pradesh and Maharashtra, respectively. The hospitalisation rate did not differ much between the GFHI-enrolled and non-enrolled population. The average OOPE on hospitalisation was similar for the GFHI-enrolled and non-enrolled patients. The OOPE and catastrophic health expenditure in private hospitals remained very high, irrespective of GFHI enrolment. The pattern was similar in both states. Multi-variate adjusted models showed that GFHI had no significant effect on utilisation, quality, OOPE and catastrophic health expenditure. The above results were confirmed by propensity score matching.

Conclusions

Coverage by GFHI enrolment was ineffective in improving access, quality or financial protection for inpatient hospital care despite 5 years of implementation of the programme. Long-standing supply-side gaps and poor regulation of private providers continue to hamper the effectiveness of GFHI in India.

Background

Clinical healthcare is not the only way to improve an individual’s health. Community-based interventions can have health and wellbeing impacts as well; however, the nature of these interventions, which have public good characteristics, poses challenges for the typical ways in which we value outcomes for use in (health) economic evaluations. The approaches to valuation of these type of interventions should allow for the incorporation of all types of values including option value, externalities and individual use-value.

Objective

This is a feasibility study with the objective to re-consider the importance of health externalities when valuing public health interventions that are treated as public goods from an economic perspective.

Methods

A contingent valuation (CV) survey was designed to elicit individual willingness to pay (WTP) for the public piano programme (PPP). Five different scenarios were designed; three scenarios focussed on individual use–value, while the other two (scenarios 4 and 5) covered option values and externalities. An online survey was conducted with a sample of 105 people.

Results

Preferences differed across the different scenarios. The mean WTP for scenario 1 was £0.81, for scenario 2 £3.65, for scenario 3 £3.07, for scenario 4 £7.26 and for scenario 5 £6.02. The WTP results for each scenario are presented and discussed regarding the nature of the good, user and non-user perspectives, payment vehicles and individual characteristics.

Conclusion

This study provides evidence that all types of use are necessary for inclusion in an economic evaluation, especially when the good in question is a public good where its benefits can be obtained from all community members.

The Health Service Executive, responsible for operating the Irish health service, has introduced health technology management (HTM) initiatives to manage expenditure on medicines. One such approach is managed access protocols (MAPs) to support access to high-cost medicines, while providing oversight, governance and budgetary certainty to the payer. Herein we describe the development and operation of MAPs, using case studies of liraglutide (Saxenda^®), dupilumab (Dupixent^®) and calcitonin gene-related peptide monoclonal antibodies. A MAP imposes the eligibility criteria attached to reimbursement support of a medicine. Criteria applied include controls on prescribing authority, clinical diagnostic and severity criteria, previous lines of treatment, concomitant treatments, outcome data collection, and validations within the reimbursement claims system. The choice of criteria are specific to each medicine, dictated by the areas of uncertainty highlighted in the health technology assessment report, such as the place in treatment, population, duration of treatment, etc., the commercial arrangements reached with the marketing authorisation holder, and specific recommendations made by the decision maker. By December 2023, there were 28 medicines reimbursed subject to a MAP in Ireland. Across the three case studies outlined, over 3000 patients were accessing novel treatments for chronic illnesses in September 2023. Managed access protocols can provide some cost certainty for the payer by aligning utilisation and expenditure with committed funds, while enabling access where unmet need is highest. Managed access protocols are now established in the drug reimbursement process in Ireland, meeting the needs of both payers, patients and industry, and are likely to remain a feature of the reimbursement landscape.

Background

Increasing healthcare costs require evidence-based resource use allocation for which assessing costs rigorously and comparably is crucial. Harmonized cross-country costing methods for evaluating interventions from a societal perspective are lacking. This study presents the development process and content of the service costing templates developed as part of the European project PECUNIA.

Methods

The six developmental steps towards technological readiness of the templates included (1) a common conceptual costing framework and review of methodological costing issues, (2) harmonization strategy formulation, (3) proof-of-concept with expert feedback, (4) piloting, (5) validation, and (6) demonstration in six European countries.

Results

The PECUNIA Reference Unit Cost (RUC) Templates for service costing are three new self-completion tools to be used with secondary or primary data for top-down micro-costing or top-down gross-costing approaches. Complementary data collection and unit cost aggregation/weighting templates are available. The applications leading to the final versions including (4) piloting through calculation of 15-unit costs, (5) validation within a Health Technology Assessment framework, and (6) RUC calculations mostly based on secondary data demonstrated the templates’ general feasibility, with feedback for improved usability incorporated and a supplementary user guide developed.

Conclusion

The validated PECUNIA RUC Templates for multi-sectoral and multi-country service costing allow for harmonized RUC development while incorporating flexibility and transparency in the choice of costing approaches, data sources and magnitude of remaining heterogeneity. The templates are expected to significantly improve the quality, comparability and availability of unit costs for economic evaluations, and promote the transferability of service cost information across Europe.