Applied Health Economics and Health Policy最新文献

Background

Economic evaluation of one-time therapies during reimbursement decision-making is challenging due to uncertain long-term outcomes. The availability of 5-year outcome data from the ELIANA trial and real-world evidence of tisagenlecleucel, the first chimeric antigen receptor T-cell (CAR-T) therapy, presents an opportunity to re-evaluate the predictions of prior cost-effectiveness analyses (CEAs).

Objective

To conduct a systematic literature review (SLR) of prior CEAs of tisagenlecleucel for pediatric/young adult relapsed or refractory acute lymphoblastic leukemia (r/r ALL) and evaluate the impact of recently available 5-year efficacy data from ELIANA and advances in CAR-T manufacturing in an updated CEA model.

Methods

OVID MEDLINE/Embase and health technology assessment (HTA) databases were searched for full-text economic evaluations in English reporting cost-effectiveness results for tisagenlecleucel for r/r ALL. Evaluations with publicly reported incremental cost-effectiveness ratios (ICERs) were included in the SLR. Study screening and data abstraction were conducted following PRISMA guidelines. Data extracted included the country/currency, perspective, clinical trial evidence, model structures, long-term efficacy extrapolation approaches (i.e., overall survival [OS]), time horizon, discount rates, and outcomes (i.e., life years [LY], quality-adjusted LY [QALY], and ICERs). The CEA model reported in Wakase et al. was updated using 5-year OS data from ELIANA and the CAR-T infusion rate informed by real-world practice.

Results

Sixteen records corresponding to 15 unique studies were included in the SLR (11 publications and 5 HTA reports); all were conducted from the health care system perspective of the respective countries. Most studies found tisagenlecleucel to be cost effective, but all studies’ projected 3- and 5-year OS rates for tisagenlecleucel were lower than the observed 3- and 5-year rates, respectively, derived from 5-year ELIANA data. When applying updated OS projections from the most recent ELIANA data cut and higher infusion rates of 92.5% (per the real-world infusion rate)—96.0% (per the manufacturer success rate) to the CEA of Wakase et al., the associated QALYs for tisagenlecleucel increased from 11.6 to 14.6–15.0, and LYs increased from 13.3 to 17.0–17.5. Accordingly, the ICERs for tisagenlecleucel decreased from ¥2,035,071 to ¥1,787,988–¥1,789,048 versus blinatumomab and from ¥2,644,702 to ¥2,257,837–¥2,275,181 versus clofarabine combination therapy in the updated CEA model.

Conclusions and Relevance

Projections at launch of the likely cost effectiveness of tisagenlecleucel appear to have underestimated its ultimate economic value given more recent trial and real-world data. To balanc

Multiple, accelerating and interacting ecological crises are increasingly understood as constituting a major threat to human health and well-being. Unconstrained economic growth is strongly implicated in these growing crises, and it has been argued that this growth has now become “uneconomic growth”, which is a situation where the size of the economy is still expanding, but this expansion is causing more harm than benefit. This article summarises the multiple pathways by which uneconomic growth can be expected to harm human health. It describes how health care systems—especially through overuse, low value and poor quality care—can themselves drive uneconomic growth. Health economists need to understand not only the consequences of environmental impacts on health care, but also the significance of uneconomic growth, and pay closer attention to the growing body of work by heterodox economists, especially in the fields of ecological and feminist economics. This will involve paying closer heed to the existence and consequences of diminishing marginal returns to health care consumption at high levels; the central importance of inequalities and injustice in health; and the need to remedy health economists’ currently limited ability to deal effectively with low value care, overdiagnosis and overtreatment.

Objective: We compared two generic, preference-based health-outcome measures: the novel patient-centered Château-Santé Base (CS-Base), entailing a multi-attribute preference response framework, and the widely used EQ-5D-5L, regarding effects of different measurement frameworks and different descriptive systems.

Methods: We conducted a cross-sectional study using a random sample of patients (3019 reached, 1988 included) in the USA with various health conditions. The CS-Base (12 attributes, each with four levels), EQ-5D-5L and the 5D-4L (an ad hoc, multi-attribute preference response-based measure that includes five attributes similar to the EQ-5D-5L, but with four levels) were used as health-outcome measures. We compared the proportions of problems reported on health attributes, statistical robustness and face validity of coefficients, attribute importance, differentiation between health states based on health-state values obtained with these measures, and user experience.

Results: All the CS-Base and 5D-4L coefficients had logical orders and significant differences from the reference level (p < 0.001). Substantial differences were observed in the CS-Base and 5D-4L coefficients between all levels on all attributes, while subtle differences were seen in those of the EQ-5D-5L. Attribute importance of usual (daily) activities were lowest or second lowest in all the three health-outcome measures. Attributes with the highest importance in the CS-Base, 5D-4L, and EQ-5D-5L were respectively mobility, anxiety/depression, and pain/discomfort. Four attributes are similar between the CS-Base and EQ-5D-5L, eight are exclusive to CS-Base. Of the eight, vision and hearing had the highest importance. Health-state values showed a smoother distribution with minimal discontinuity in the CS-Base and EQ-5D-5L than in the 5D-4L. In user experience evaluation, both CS-Base and the 5D-4L showed mean scores above 50 (indicating positive evaluation) in terms of the description of health and ease of understanding.

Conclusions: This study demonstrated that CS-Base and 5D-4L, which are grounded in the multi-attribute preference response framework, produced statistically robust coefficients, with better face validity than those for the EQ-5D-5L. CS-Base and the EQ-5D-5L outperformed the 5D-4L in differentiating between health states. Notwithstanding differences in content, measurement frameworks, and estimated coefficients, the computed health-state values were similar between CS-Base and EQ-5D-5L.

Conflicts over pharmaceutical pricing are driven by the patients' need for affordable medicines and the producer's reward for the investments in developing innovative medicines. A single price cannot achieve both goals, as it will either obstruct access by patients or provide too low a return to investors. This has led to calls to "delink" the payment for innovation from the price paid for drugs, so that both goals can be met efficiently and without conflict. However, the details of how best to do that are unclear. This paper proposes a specific implementation for delinking the Optional Delinked Reward System (ODRS), which integrates ideas from numerous pharmaceutical reimbursement systems. The ODRS would allow firms to choose either to negotiate a sales price for a drug (as is the current practice in most countries) or to sell their drug at a low "generic" price with a supplementary "delinked" reward based on assessed health benefit. This model builds on recent innovations in drug reimbursement including the UK's Antibiotic Subscription Pilot and the Pneumococcal Vaccine Advanced Market Commitment. The ODRS would ensure affordable and immediate access for patients and a fair reward for innovators.

Community-based health promotion (CBHP) interventions are promising approaches to address public health problems; however, their economic evaluation presents unique challenges. This review aims to explore the opportunities and limitations of evaluating economic aspects of CBHP, focusing on the assessment of intervention costs and outcomes, and the consideration of political-level changes and health equity. A systematic search of the PubMed, Web of Science and PsycInfo databases identified 24 CBHP interventions, the majority of which targeted disadvantaged communities. Only five interventions included a detailed cost/resource assessment. Outcomes at the operational level were mainly quantitative, related to sociodemographics and environment or health status, while outcomes at the political level were often qualitative, related to public policy, capacity building or networks/collaboration. The study highlights the limitations of traditional health economic evaluation methods in capturing the complexity of CBHP interventions. It proposes the use of cost-consequence analysis (CCA) as a more comprehensive approach, offering a flexible and multifaceted assessment of costs and outcomes. However, challenges remain in the measurement and valuation of outcomes, equity considerations, intersectoral costs and attribution of effects. While CCA is a promising starting point, further research and methodological advancements are needed to refine its application and improve decision making in CBHP.

Objectives: To develop preliminary good practice recommendations for synthesising and linking evidence of treatment effectiveness when modelling the cost-effectiveness of diagnostic tests.

Methods: We conducted a targeted review of guidance from key Health Technology Assessment (HTA) bodies to summarise current recommendations on synthesis and linkage of treatment effectiveness evidence within economic evaluations of diagnostic tests. We then focused on a specific case study, the cost-effectiveness of troponin for the diagnosis of myocardial infarction, and reviewed the approach taken to synthesise and link treatment effectiveness evidence in different modelling studies.

Results: The Australian and UK HTA bodies provided advice for synthesising and linking treatment effectiveness in diagnostic models, acknowledging that linking test results to treatment options and their outcomes is common. Across all reviewed models for the case study, uniform test-directed treatment decision making was assumed, i.e., all those who tested positive were treated. Treatment outcome data from a variety of sources, including expert opinion, were utilised for linked clinical outcomes. Preliminary good practice recommendations for data identification, integration and description are proposed.

Conclusion: Modelling the cost-effectiveness of diagnostic tests poses unique challenges in linking evidence on test accuracy to treatment effectiveness data to understand how a test impacts patient outcomes and costs. Upfront consideration of how a test and its results will likely be incorporated into patient diagnostic pathways is key to exploring the optimal design of such models. We propose some preliminary good practice recommendations to improve the quality of cost-effectiveness evaluations of diagnostics tests going forward.