Applied Health Economics and Health Policy最新文献

Background

Women’s preferences for time allocation reveal how they would like to prioritise market work, family life, and other competing activities. Whilst preferences may not always directly translate to behaviour, they are an important determinant of intention to act.

Objective

We present the first study to apply a discrete choice experiment (DCE) to investigate time allocation preferences among women diagnosed with breast cancer and women without a cancer diagnosis.

Methods

Time attributes were paid work, household work, caregiving, passive leisure and physical leisure. An income attribute was included to estimate the monetary value of time. The study took place in the UK and the DCE was completed by 191 women diagnosed with breast cancer and 347 women without a cancer diagnosis. Responses were analysed using a mixed logit model.

Results

Women diagnosed with breast cancer have stronger positive preferences for daily activities compared to women without a cancer diagnosis. They require less compensation (not significant) for an additional hour of paid work (£5.58), household work (£7.92), and caregiving (£8.53). They are willing to pay more for an additional hour of passive leisure (£1.70, not significant) and physical leisure (£13.66, significant).

Conclusion

The heterogeneous preferences for time allocation among women have policy implications and are significant for welfare analysis.

Objective

This article reviews the assessment pathways that have been implemented worldwide to facilitate access to drugs for patients with rare diseases.

Methods

The Preferred Reporting Items for Systematic reviews and Meta-Analyses (PRISMA) guidelines were used to conduct a systematic literature review. The Ovid (Embase/MEDLINE), Cochrane, Web of Science, Econlit, National Institute of Health Research, Centre for Reviews and Dissemination, and International Network of Agencies for Health Technology Assessment databases were searched. Two independent reviewers screened all titles and abstracts; one reviewer did the full-text review and data extraction. Data were extracted on study general characteristics, general aspects of rare diseases, source of funding, allocation of public resources (e.g., use of health technology assessment), and pricing strategies. Assessment pathways were classified as: (1) separate processes; (2) exception to standard process; (3) standard process with no change; and (4) alternative process. Each assessment pathway was characterized based on its unique characteristics specific to rare diseases focusing on whether they targeted specific aspects of the process, utilized particular methodologies during the evaluation of the evidence, or considered specific attributes in the recommendation.

Results

A total of 5604 unique citations were screened and 158 were included for data extraction. Sixty-one assessment pathways were identified in 43 countries, categorized as separate processes (37%), exceptions to standard processes (32%), standard processes with no changes (26%), and alternative processes (5%). Some countries (10/43; 23%) have more than one assessment pathway available. Assessment pathways varied in their inclusion of a health technology assessment, source of funding, consideration of uncertainty, and pricing strategies.

Conclusions

The diversity of assessment pathways reflects the complexity of addressing access to treatments for rare diseases. Furthermore, most assessment pathways are from high-income countries; therefore, there is less clarity on what is happening in low- and middle-income countries.

Background and Objective

Rising healthcare costs challenge the financial sustainability of healthcare systems. Interventional pharmacoeconomics has emerged as a vital discipline to improve the cost-effective and sustainable use of drugs in clinical practice. However, current efforts are often fragmented, highlighting the need for an integrated hospital-wide approach. This study aimed to develop a scalable framework to systematically identify and implement cost-effective and sustainable drug use practices in hospitals.

Methods

This study was conducted at the Erasmus University Medical Centre in Rotterdam between December 2022 and July 2023. A novel ‘8-Step Efficiency Model’ was designed to systematically identify and evaluate strategies for cost-effective and sustainable drug use. The process involved identifying high-expenditure drugs, systematically assessing these drugs using the Efficiency Model, and conducting a multi-disciplinary evaluation of the proposed cost-effectiveness strategies.

Results

The study assessed 39 high-cost drugs, representing 57% of the Dutch national expensive drug expenditure in 2021. Initiatives for enhancing cost-effectiveness and sustainability were identified or developed for 27 out of the 39 assessed drugs (51% of the national drug expenditure in 2021). Case examples of infliximab (e.g., wastage prevention) and intravenous immunoglobulins (e.g., lean body weight dosing) illustrate practical applications of the framework, resulting in substantial cost savings and improved sustainability.

Conclusions

This study presents a systematic scalable model for enhancing the cost-effectiveness of high-expenditure drugs in hospital settings. This approach not only addresses financial sustainability but also promotes the quality of patient care and sustainable drug use. This model could serve as a generic blueprint for other institutions to identify and implement cost-effective and sustainable drug use strategies.

Objectives

Quality-adjusted life expectancy (QALE) norms reflect the normative profiles or reference data of QALE of the general population and provide a meaningful anchor for comparison to inform healthcare decision-making. This study aimed to develop the QALE norms for the Chinese population by using a representative dataset of health utility values collected using the EQ-5D-5L and short-form 6-dimension version 2 (SF-6Dv2) instruments.

Methods

Age-specific population norms of health utility values calculated using the EQ-5D-5L and SF-6Dv2 were used. Both utility norms were combined with the latest version of the National Life Tables of China published in 2021 to calculate QALE estimates on the basis of age, sex, and urban/rural residence area. QALE estimates were further discounted using 1.5%, 3.5%, 5.0%, and 8.0% discount rates.

Results

When using the health utility values evaluated by the SF-6Dv2, the QALE at age 0 years was 66.34 years at the discount rate of 0% and 16.65 years at the discount rate of 5%. For the EQ-5D-5L, the QALE at age 0 years was 76.50 years at the discount rate of 0% and 19.45 years at the discount rate of 5%. At birth, females exhibited a higher QALE, while the difference between females and males initially increased before subsequently declining overtime, ultimately resulting in females having a lower QALE. Rural population had a monotonically lower QALE than urban population.

Conclusion

This study constructed age-stratified QALE norms for the Chinese population categorized by sex and residence area using mortality data alongside corresponding health utility values derived from the EQ-5D-5L and SF-6Dv2.

Economic evaluations play a crucial role in health resource allocation by assessing the costs and effects of various interventions. However, existing methodologies often overlook significant differences related to sex and gender, leading to a ‘blind spot’ in understanding patient heterogeneity. This paper highlights how biological and social factors influence costs and health outcomes differently for women, emphasising the need for a more explicit consideration of these differences in economic evaluations to ensure efficient and equitable resource allocation. The paper is structured to first outline how sex and gender factors impact costs and outcomes. It then identifies biases in current economic evaluation methods and practices, using real-world examples to illustrate the implications of these biases on policymaking and health equity. Notably, we argue that neglecting gender considerations can lead to inefficiencies and inequities in healthcare resource distribution. Key areas of gender bias include the estimation of productivity losses, quality of life variations and the secondary household effects of interventions. The analysis reveals that women often face higher healthcare costs and experience different health outcomes due to systemic biases in treatment and care. The paper concludes with practical recommendations for analysts, decision makers and research funders, advocating for the integration of sex and gender-responsive methodologies in health economic evaluations. Ultimately, this work calls for a paradigm shift in health economics to better reflect the complexities of sex and gender and improve health outcomes for all.

Background

The dead state can affect the value sets derived from discrete choice experiments (DCEs). Our aim was to empirically assess the direct impact of the immediate death state on health utilities using discrete choice experiment with time (DCE_TTO).

Methods

A sample of the general population in Quebec, Canada, completed two approaches: DCE_TTO followed by a best-worst scaling with time (BWS_TTO) (hereafter referred to as DCE_BWS), versus DCE_TTO followed by the dominated option and the immediate death state (hereafter referred to as DCE_DOD), both designed with the SF-6Dv2. In DCE_BWS, all participants first completed 10 DCE_TTO choices (i.e., option A vs B), followed by 3 BWS_TTO. In DCE_DOD, the same participants first completed the same 10 DCE_TTO choices, followed by a repeated choice between the dominated option (i.e., A or B) and the immediate death state. A conditional logit model was used to estimate value sets. The performance of models was assessed using goodness of fit using Bayesian information criterion, parameters’ logical consistency, and levels’ significance. The direct impact of the death state on DCE latent utilities was evaluated by examining the magnitude of coefficients, assessing the agreement among the value sets estimated by DCE_TTO with DCE_BWS and with DCE_DOD using Bland-Altman plots, the proportion of worst-than-dead (WTD) health states, and analyzing the range of estimated values.

Results

From 398 participants, a total of 348 participants were included for final analysis. The number of parameters with illogical consistency and non-significant coefficients was lower in DCE_BWS. The observed consistency in the relative importance of dimensions across all approaches suggests a stable and reliable ranking. The utility range for DCE_DOD (− 0.921 to 1) was narrower than for DCE_TTO (− 1.578 to 1) and DCE_BWS (− 1.150 to 1). The DCE_DOD estimated a lower percentage of WTD health states (20.01 %) compared to DCE_TTO (47.19 %) and DCE_BWS (33.73 %). The agreement between DCE_TTO and DCE_BWS was slightly stronger than between DCE_TTO and DCE_DOD, and the mean utility values were higher in DCE_DOD than in DCE_BWS.

Conclusions

The inclusion of the immediate death state directly within DCE increased utility values. This increase was higher when the immediate death was included in a sequence within a DCE_TTO (i.e., DCE_DOD) than when it was included in a continuum of DCE_TTO (i.e., DCE_BWS). The use of DCE_DOD was potentially better suited to incorporate the dead state into a DCE.

Background

Hemophilia B, a severe genetic disorder, involves substantial treatment costs and frequent interventions. Etranacogene dezaparvovec (EDZ) is a recently approved gene therapy for hemophilia B.

Objective

This study evaluates the cost-effectiveness of EDZ compared with conventional factor IX (FIX) prophylaxis.

Methods

A semi-Markov model simulated a cohort of adult males with severe hemophilia B to assess the economic impact of EDZ versus FIX prophylaxis over a lifetime horizon from a health system perspective in the USA. Inputs derived from clinical trials included therapy durability and transition probabilities based on Pettersson Scores. Scenario analyses incorporated frameworks suggested by the Institute for Clinical and Economic Review for single or short-term transformative therapies.

Results

Base-case analysis showed that at a cost of US$3.5 million, EDZ led to lifetime cost savings of US$11 million and an additional 0.64 quality-adjusted life years (QALYs) compared with FIX. However, FIX has extremely high annual costs. When annual cost offsets attributed to EDZ were capped at US$150,000, EDZ was found to have a threshold price of US$3.1 million at a willingness-to-pay of US$150,000 per QALY.

Conclusion

EDZ proved to be a dominant strategy over FIX prophylaxis in the base-case scenario, providing large cost savings and slightly better outcomes. The substantial costs associated with FIX are a primary driver behind these results. The introduction of cost-offset caps significantly affects the value-based price of EDZ. Using caps on cost offsets in considering price can help to balance affordability and value in the health system.

Objectives

The UK has recently established subscription-payment agreements for two antimicrobials: cefiderocol and ceftazidime-avibactam. This article summarises the novel value assessments that informed this process and lessons learned for future pricing and funding decisions.

Methods

The evaluations used decision modelling to predict population incremental net health effects (INHEs), informed by systematic reviews, evidence syntheses, national surveillance data and structured expert elicitation.

Results

Significant challenges faced during the development of the evaluations led to profound uncertainty in the estimates of INHEs. The value assessment required definition of the population expected to receive the new antimicrobials; estimating value within this heterogenous population; assessing comparative efficacy using antimicrobial susceptibility data due to the absence of relevant clinical data; and predicting population-level benefits despite poor data on current numbers of drug-resistant infections and uncertainties around emerging resistance. Though both antimicrobials offer the potential to treat multi-drug resistant infections, the benefits estimated were modest due to the rarity of true pan-resistance, low life expectancy of the patient population and difficulty of identifying and quantifying additional sources of value.

Conclusions

Assessing the population INHEs of new antimicrobials was complex and resource intensive. Future evaluations should continue to assemble evidence relating to areas of expected usage, patient numbers over time and comparative effectiveness and safety. Projections of patient numbers could be greatly enhanced by the development of national level linked clinical, prescribing and laboratory data. A practical approach to synthesising these data would be to combine expert assessments of key parameters with a simple generic decision model.

Background

The majority of cost-effectiveness analyses of pharmaceuticals do not incorporate future price changes that are expected once generic competition enters the market. A common rationale for not doing so is the uncertainty around what postloss of exclusivity price to model. The objective of this study is to assess if a drug’s price postloss of exclusivity can be estimated on the basis of its cost of goods sold (COGS).

Methods

First, stakeholders were engaged to understand pricing practices for generic drugs. Then peer-reviewed literature, gray literature, and manufacturer financial statements were reviewed to estimate the typical manufacturer profit margin over COGS. Using pricing data from the Mark Cuban CostPlus Drug Company (MCCPDC), estimates of COGS were calculated by drug form. Finally, a COGS-based approach to estimating a drug’s price postloss of exclusivity was tested.

Results

COGS were estimated for 2168 unique National Drug Codes (NDCs) reported in the MCCPDC price list by removing the typical manufacturer profit margin (50%) from the manufacturer prices (net of any markup or fees from the MCCPDC). A tablet/capsule, the most common form in the price list, had a median COGS of $0.10 per tablet/capsule. Estimating a drug’s price postloss of exclusivity as the median COGS for that drug form times two (to reflect a 50% profit margin), produces estimates of postloss of exclusivity prices that account for drug-specific attributes.

Conclusions

This study provides an evidence-based approach to estimate a drug’s price postloss of exclusivity as a function of its COGS for incorporation into cost-effectiveness analyses.