European Journal of Health Economics最新文献

Objective: To assess the 12-month cost-effectiveness of the nurse-led transitional care program "Cardiolotse" (CL) for patients with cardiovascular diseases compared to usual care (UC).

Methods: A cost-effectiveness analysis (CEA) and cost-utility analysis (CUA) were conducted from the perspective of statutory health insurance (SHI), covering a time horizon of 12 months. Analyzed outcomes included the number of rehospitalizations and health-related quality of life (HRQoL). Total costs comprised program costs and the utilization of healthcare resources. Point estimates are presented as incremental cost-effectiveness ratios (ICERs) and incremental cost-utility ratios (ICURs). Sensitivity and subgroup analyses were conducted to illustrate uncertainty and provide insights into the impact mechanisms of the CL program.

Results: The study population consisted of 2550 patients, with 1256 allocated to the intervention group and 1294 to the control group. Patients who received support from CLs experienced fewer rehospitalizations and lower inpatient costs from an SHI perspective, compared to the UC group. HRQoL assessments indicated higher utility values for CL patients at the 12-month follow-up. Total program costs amounted to €1454.65 per patient. The CEA and CUA demonstrate that the CL program is dominant compared to UC from the SHI perspective.

Conclusion: Our study shows that the CL program not only reduces the number of rehospitalizations and costs but increases HRQoL, resulting in a dominant ICER and ICUR. Further research is necessary to evaluate longer periods of time, different levels of care intensity, and perspectives of different healthcare stakeholders.

Trial registration: German Clinical Trial Register DRKS00020424, 2020-06-18, retrospectively registered.

Access to new medicines is crucial for patients but increasingly sparks discussion due to high prices. Simultaneously, the growing emphasis on specialized products and uncertainty surrounding the long-term effectiveness of new drug classes brought to the market underscore the need for innovative pricing approaches. A systematic literature review of pharmaceutical pricing models, accompanied by a critical appraisal, was conducted to offer insights contributing to novel approaches balancing sustainable pharmaceutical innovation with affordability and accessibility for patients. Six different pricing models are identified: value based pricing, basic cost-based pricing, and four more comprehensive pricing models incorporating numerous elements: the cancer-drug-pricing model, AIM model, (Nuijtens) discounted cash flow, and the real-option rate of return method. Although there are many similarities among the models, each has unique assumptions for implementation. For instance, all models except for the standard incremental cost-effectiveness ratio and basic cost-based pricing consider the number of eligible patients and the remaining patent period. Only the AIM model and the Nuijtens discounted cash flow model use lump sums. Both the latter and the real-option rate of return method explicitly include the cost of capital as a major cost-based component. Recognizing the diverse applications of each model highlights the need for more differential and dynamic pricing tailored to the characteristics and therapeutic areas of each drug. Additionally, the study underscores the importance of cost transparency in achieving this goal. Consequently, these findings can help stakeholders develop sustainable and affordable drug pricing mechanisms that address the complexities of the ever-changing pharmaceutical landscape.

Introduction: Acid sphingomyelinase deficiency (ASMD) type B is a rare genetic disorder leading to enlargement of the spleen and liver, pulmonary dysfunction, and other symptoms. Cost-utility analyses are often conducted to quantify the value of new treatments, and these analyses require health state utilities. Therefore, the purpose of this study was to estimate utilities associated with varying levels of severity of adult and pediatric ASMD type B.

Methods: Seven adult and seven child health state vignettes describing ASMD were developed based on published literature, clinical trial results, and interviews with clinicians, patients with ASMD, and parents of children with ASMD. The health states were valued in time trade-off interviews with adult general population respondents in the UK.

Results: Interviews were completed with 202 participants (50.0% female; mean age = 41.3 years). The health state representing ASMD without impairment had the highest mean utility for both the adult and child health states (0.92/0.94), and severe ASMD had the lowest mean utility (0.33/0.45). Every child health state had a significantly greater utility than the corresponding adult health state. Differences between adult/child paired states ranged from 0.02 to 0.13. Subgroup analyses explored the impact of parenting status on valuation of child health states.

Discussion: Greater severity of ASMD was associated with lower mean utility. Results have implications for valuation of pediatric health states. The resulting utilities may be useful in cost-utility modeling estimating the value of treatment for ASMD.

Our objective was to compare AOTMiT (Polish: Agencja Oceny Technologii Medycznych i Taryfikacji) recommendations to other HTA (Health Technology Assessment) agencies for newly registered drugs and new registration indications issued by the European Medicines Agency between 2014 and 2019. The study aims to assess the consistency and justifications of AOTMiT recommendations compared to that of other HTA agencies in 11 countries. A total of 2496 reimbursement recommendations published by 12 HTA agencies for 464 medicinal products and 525 indications were analyzed. Our analysis confirmed that the Polish AOTMiT agency seems to bear the closest resemblance to the corresponding HTA agencies from Canada (CADTH) and New Zealand (PHARMAC), when it comes to the outcome of HTA recommendations (positive or negative). Poland had a general scheme for justifying recommendations, similar to that of Ireland-four aspects (i.e., clinical efficacy, safety profile, cost-effectiveness, and impact on the payer's budget) are important for Poland when formulating the final decision. Compared to other countries, Poland shows a noticeably different pattern of justifying reimbursement recommendations, as revealed primarily in terms of budget impact and somewhat less so for cost-effectiveness rationales.

Government employees in Spain are covered by public Mutual Funds that purchase a uniform basket of benefits, equal to the ones served to the general population, from private companies. Companies apply as private bidders for a fixed per capita premium hardly adjusted by age. Our hypothesis is that this premium does not cover risks, and companies have incentives for risk selection, which are more visible in high-cost patients. We focus on a particularly costly disease, cancer, whose prevalence is similar among government employees and the general population. We compare hospitalisations in the public hospitals of the government employees that have chosen public provision and the general population. We analysed a database of hospital discharges in the Valencian Community from 2010 to 2015 (3 million episodes). Using exact matching and logistic models, we find significant risk selection; thus, in hospitalised government employees, the likelihood for a solid metastatic carcinoma and non-metastatic cancer to appear in the registry is 31% higher than in the general population. Lymphoma shows the highest odds ratio of 2.64. We found quantitatively important effects. This research provides indirect evidence of risk selection within Spanish Mutual Funds for government employees, prompting action to reduce incentives for such a practice. More research is needed to figure out if what we have observed with cancer patients occurs in other conditions.

Introduction: The Individualized Neuromuscular Quality of Life Questionnaire (INQoL) is used to measure quality of life in neuromuscular disorders such as non-dystrophic myotonia (NDM). Here we report methods to estimate utilities, with a focus on NDM, from this questionnaire based on two preference elicitation exercises.

Methods: Eight items from the INQoL were selected with input from three neuromuscular disorder clinical experts with expertise in treating NDM. A discrete choice experiment (DCE) survey of UK general public respondents (n = 508) described outcomes defined by the INQoL items. The same 8 items were also valued using time trade-off (TTO) face-to-face interviews (n = 200). A hybrid regression modelling approach combined both datasets to inform the utility weights.

Results: Hybrid modelling of DCE and TTO data in conjunction improved out-of-sample predictive accuracy. The selected INQoL utility model indicates substantial disutility associated with all eight dimensions of health, with the greatest losses associated with subjective items such as pain and depression.

Discussion: The hybrid modelling approach allows us to combine data from the two methodologies and maximize the information from each to inform the utility weights for the INQoL. The TTO is the more conventional valuation method, but combined with the larger DCE study produced better descriptive coverage. This is a relatively novel method for estimating weights which we think is particularly well suited to economic evaluations of orphan drugs.

The US Inflation Reduction Act (IRA) prohibits the Centers for Medicare and Medicaid Services (CMS) from using standard quality-adjusted life-years or other value assessment methods that discriminate against the aged, terminally ill, or disabled when setting maximum fair prices for prescription drugs. This policy has reignited interest in methods for assessing value without discrimination. Equal value of life-years gained (EVL), healthy years in total (HYT), and Generalized Risk-Adjusted Cost-Effectiveness (GRACE) have emerged as proposals. Neither EVL nor HYT rests on well-articulated microeconomic foundations. We show that they produce decisions that are inconsistent over time in a variety of ways, including: (1) failure to support additivity and indirect comparison in cases where the standard-of-care therapy changes over time; (2) strictly negative value of survival gains that accrue from a new, better standard-of-care, particularly for the disabled themselves; (3) unbounded average value of survival gains; and (4) non-convex survival preferences. We propose an alternative method that relies on GRACE and its microeconomic foundations.

Health authorities using cost-effectiveness analysis (CEA) for informing reimbursement decisions on health technologies increasingly require economic evaluations encompassing both CEA and budget impact analysis (BIA). Good Research Practices advocate that the economic and clinical assumptions underlying these analyses are aligned and consistently applied. Nonetheless, CEAs and BIAs often are stand-alone analyses used in different stages of the decision-making process. This article used policy reports and Ministerial correspondence to discuss and elucidate the role of budget impact and its relationship with cost-effectiveness in reimbursement decisions in the Netherlands. The results indicate that CEAs and BIAs are both considered important for informing these decisions. While the requirements regarding CEAs-and application of the associated decision rule-are consistent across the different stages, the same does not hold for BIAs. Importantly, the definition of and evidence on budget impact differs between stages. Some important aspects (e.g. substitution and saving effects) typically are considered in the assessment and appraisal stages but are seemingly not considered in price negotiations and the final reimbursement decision. Further research is warranted to better understand why BIAs are not aligned with CEAs (e.g. in terms of underlying assumptions), vary in form and importance between stages, and do not have a clear relationship with the results of CEAs in the decision-making framework. Improving the understanding of the circumstances under which decision-makers attach a relatively larger or smaller weight to (different aspects of) budget impact may contribute to increasing the transparency, consistency, and optimality of reimbursement decisions in the Netherlands.

The psychometric properties of the EQ-5D-Y have not been widely tested in severely ill children. The aim of this study was to assess and compare the validity and responsiveness of the EQ-5D-Y-3L and EQ-5D-Y-5L in paediatric inpatients with haematological malignancies and caregivers. Respondents completed the interviewer-administered self-complete or proxy version of the EQ-5D-Y-3L and EQ-5D-Y-5L and an overall health assessment twice on different days. Known-groups validity was assessed by comparing patients who differed in overall health and Eastern Cooperative Oncology Group (ECOG) performance. Responsiveness to worsened health was assessed using standardised effect size (SES) for patients with worsened ECOG grade, self-reported rating, or chemotherapy initiation. Ninety-six dyads completed the baseline questionnaires. A smaller proportion of patients reported "no problems" on the EQ-5D-Y-5L compared to EQ-5D-Y-3L for most of the five dimensions. Patients in poor health reported more problems in all dimensions and had higher EQ-5D-Y-5L level sum score, lower EQ VAS and EQ-5D-Y-3L index scores (Cohen's d ES: 0.32-1.38 for patients; 0.50-2.05 for caregivers). There was a mild to good responsiveness to worsened health condition based on ECOG (SES: 0.14-0.61 for patients; 0.40-0.96 for caregivers), suggesting the proxy version was slightly responsive than the self-complete version of both instruments. The results demonstrated validity and responsiveness for both the self-complete and proxy versions of the EQ-5D-Y-3L and EQ-5D-Y-5L. The proxy and 5-level versions of the instrument were more sensitive than the self-complete and 3-level versions in this patient group.