Health Economics Research Research Articles

Quality of Life Based Health Economics Research on Pharmaceutical Intervention in Cancer Chemotherapy

The adverse chapter of cancer chemotherapy negatively impact QOL, and pharmacists play a key role in improving QOL by providing optimal drug therapy through pharmaceutical interventions. Although outpatient cancer chemotherapy is now common, the impact of pharmaceutical interventions from a QOL perspective has not been thoroughly studied. Therefore, this study investigated the impact and cost-effectiveness of pharmaceutical interventions on QOL using the EuroQol 5 Dimension (EQ-5D) and Quality of Life Questionnaire for Cancer Patients Treated with Anticancer Drugs (QOL-ACD). The study was conducted between 2013 and 2015 on 39 patients who underwent their first outpatient chemotherapy for breast cancer at Gifu Municipal Hospital. The results showed that pharmaceutical interventions improved social relationship QOL in patients experiencing fatigue during the first cycle and enhanced psychological QOL in patients with adverse events of nausea during the second cycle. Furthermore, the maximum incremental cost-effectiveness ratio (ICER) was found to be 1.3 million yen per quality-adjusted life years (QALY) according to cost utility analysis. The pharmaceutical interventions by pharmacists in outpatient cancer chemotherapy improve QOL, and the ICER remains well below the Japanese threshold, signifying clear medical and economic benefits.

Health economics and services research in symbiosis: STREAMLINEing mental health services in Austria

Abstract Objectives The cost of mental diseases exceeds 4% of GDP in Europe. In Austria, every 5th person lives with mental ill-health (MIH). Ongoing epidemiological, demographic and societal changes increase the need for easily accessible, better integrated and good value services. In STREAMLINE, we combine, harmonize and implement the latest methods in human service mapping and economic costing to optimize future service provision and financing for MIH in Austria. Methods We comprehensively assess the availability, activity and affordability of human services targeting MIH in Vienna within the health and social care, education, labour and justice sectors based on a conceptual mind map. We identify services through extensive literature, document and web searches plus expert consultations. Services are mapped and costed according to the European PECUNIA methods: classification of the main service types using the international DESDE taxonomy of human services; development of Reference Unit Costs using the PECUNIA RUC templates with primary data; validation of the results with secondary data; compilation of a comprehensive multi-sectoral service catalogue; and synthesis for spatial and policy analyses. Results We identified 756 sources, 75% individual organisations, 5% multi-organisations and 20% existing provider lists. 84% relate to health and social care, 10% labour, and 3% education and justice sectors each. 58% deliver care to adults, 37% to children & adolescents, and 5% support carers. Further service mapping and RUCs will be presented. Discussion STREAMLINE pioneers in coordinated public mental health services and economics research at scale. Assessed levels of fragmentation call for better integration, improved transition, reduced spatial inequity and increased support for carers. Results increase the feasibility of service evaluations from a societal perspective. Planned public service atlas facilitates information and access for service users and carers. Funding: WWTF Key messages • New methods for harmonized health services and economics research allow comprehensive information for optimized future mental health service planning and financing. • Assessed levels of fragmentation highlight the need for better integration, improved transition, reduced spatial inequity and increased support for carers.

External validation and calibration of risk equations for prediction of diabetic kidney diseases among patients with type 2 diabetes in Taiwan

BackgroundMost existing risk equations for predicting/stratifying individual diabetic kidney disease (DKD) risks were developed using relatively dated data from selective and homogeneous trial populations comprising predominately Caucasian type 2 diabetes (T2D) patients. We seek to adapt risk equations for prediction of DKD progression (microalbuminuria, macroalbuminuria, and renal failure) using empiric data from a real-world population with T2D in Taiwan.MethodsRisk equations from three well-known simulation models: UKPDS-OM2, RECODe, and CHIME models, were adapted. Discrimination and calibration were determined using the area under the receiver operating characteristic curve (AUROC), a calibration plot (slope and intercept), and the Greenwood-Nam-D’Agostino (GND) test. Recalibration was performed for unsatisfactory calibration (p-value of GND test < 0.05) by adjusting the baseline hazards of risk equations to address risk variations among patients.ResultsThe RECODe equations for microalbuminuria and macroalbuminuria showed moderate discrimination (AUROC: 0.62 and 0.76) but underestimated the event risks (calibration slope > 1). The CHIME equation had the best discrimination for renal failure (AUROCs from CHIME, UKPDS-OM2 and RECODe: 0.77, 0.60 and 0.64, respectively). All three equations overestimated renal failure risk (calibration slope < 1). After rigorous updating, the calibration slope/intercept of the recalibrated RECODe for predicting microalbuminuria (0.87/0.0459) and macroalbuminuria (1.10/0.0004) risks as well as the recalibrated CHIME equation for predicting renal failure risk (0.95/-0.0014) were improved.ConclusionsRisk equations for prediction of DKD progression in real-world Taiwanese T2D patients were established, which can be incorporated into a multi-state simulation model to project and differentiate individual DKD risks for supporting timely interventions and health economic research.

Swedish Translation and Cultural Adaptation of the Head and Neck Patient Symptom Checklist: An Instrument to Screen for Nutrition Impact Symptoms in Clinical Practice and Research.

The Head and Neck Patient Symptom Checklist (HNSC) is a validated 2-part instrument used to ask patients with head and neck cancer about the nutrition impact symptoms they experience (part 1) and how these interfere with their eating (part 2). The purpose of this work was to translate and culturally adapt the HNSC into Swedish in accordance with the guidelines of the International Society for Health Economics and Outcomes Research (ISPOR). The ISPOR guidelines include 10 steps, and these were thoroughly followed. In step 7, 9 health care professionals from the field of head and neck cancer assessed the perceived relevance (content validity) of each item in the HNSC, as well as the full HNSC. A total of 522 participants with head and neck cancer were included and followed up on 7 occasions using the HNSC to assess internal consistency. The HNSC was translated from English into Swedish, ensuring accuracy through forward and backward translation and harmonization in the research team. Content validity for each part of the HNSC was rated excellent (scale content validity index 0.96). Internal consistency demonstrated a good Cronbach's alpha score (>0.8) across the 7 follow-up time points (from baseline [before the start of treatment] and up to 24 months posttreatment). The HNSC has been successfully translated and culturally adapted into Swedish. The HNSC can be used in both clinical practice and research to screen for nutrition impact symptoms and symptoms that interfere with eating in patients with head and neck cancer. ClinicalTrials.gov NCT03343236 (date of registration: November 17, 2017).

Epidemiology of Wilson disease in Germany – real-world insights from a claims data study

BackgroundWilson disease (WD) is a rare disorder of copper metabolism, causing copper accumulation mainly in the liver and the brain. The prevalence of WD was previously estimated around 20 to 33.3 patients per million for the United States, Europe, and Asia, but data on the prevalence of WD in Germany are limited.ObjectivesTo describe patient characteristics and to assess prevalence of WD in Germany using a representative claims database.MethodsWD patients were identified in the WIG2 (Wissenschaftliches Institut für Gesundheitsökonomie und Gesundheitssystemforschung; Scientific Institute for Health Economics and Health Systems Research) benchmark database of 4.5 million insured Germans by combining ICD-10-coding with WD-specific lab tests and treatments. The study period ranged from 2013 to 2016 for assessing patient characteristics, and to 2018 for prevalence, respectively.ResultsSeventy unique patients were identified. Most patients (86%) were between 18 and 64 years of age and more often male (60%) than female. Two patients (3%) younger than 18 years were included, as well as 8 patients (11%) older than 64 years. Most common WD subtypes were hepatic (57%), psychiatric (49%), and neurologic (44%). Average prevalence was 20.3 patients per million (range: 17.8–24.4), with similar results for two-year prevalence. Generally, prevalence increased steadily over the study period. Observed mortality was low, with only one death during the study period.ConclusionsThis study adds valuable real-world data on the prevalence and patient characteristics of WD in Germany. Generally, our findings align with other reports and contribute to the global understanding of WD epidemiology. Still, regional and temporal trends remain to be investigated more thoroughly to further the understanding of the natural history and epidemiology of this rare disease.

Budget impact models for lung cancer interventions: A systematic literature review.

Budget impact models (BIMs) forecast the financial implications of adopting new technologies and the potential need for budget reallocation, thus playing a crucial role in reimbursement decisions. Despite the importance of accurate forecasts, studies indicate large discrepancies between estimates and reality. We are developing an artificial intelligence-based clinical decision tool to identify patients with non-small cell lung cancer who are most likely to benefit from immunotherapy. To evaluate the budgetary implications and describe a systematic literature review of published lung cancer BIMs. We searched PubMed and EMBASE for studies published between 2010 and 2023 that include BIMs that describe lung cancer interventions. Forward and backward reference searches were performed for all qualifying studies. We extracted author and publication year, country, interventions, disease stages, time horizon, analytical perspective, modeling methods used, types of costs included, sensitivity analyses conducted, and data sources used. We then evaluated adherence to the Professional Society for Health Economics and Pharmacoeconomics Research best-practice guidelines. A total of 25 BIMs were identified, spanning 14 different countries. Model structure could not be ascertained definitively for nearly half of the models. The cost calculator approach was most common among the others. Time horizons ranged from 1 to 5 years, in line with recommendations. Most models compared drugs, 4 compared nondrug interventions, and 7 compared diagnostic technologies. Assumptions about market uptake were poorly documented and poorly motivated. Inclusion of cancer-related costs was rare. Adherence to best practices was variable and did not appear to improve over time. The number of published BIMs for lung cancer exceeded expectations. There were modest trends toward publication frequency and model quality over time. Our analysis revealed variability across the models, as well as their adherence to best practices, indicating substantial room for improvement. Although none of the models were individually suitable for the purpose of evaluating an artificial intelligence-based treatment selection tool, some models provided valuable insights.

Spatial Competition Models in Health Care Markets: A Review

AbstractThrough time, spatial competition has become the natural environment for studying a number of problems, ranging from product differentiation to mail order versus traditional business, to service provision under various assumptions about the institutional setting. Health economics research has greatly benefited from the intuitions that underlie the Hotelling model and several authors have contributed to the literature on the architecture of the market for health care by proposing models within this framework. In this paper we explore how spatial competition has been used in health economics to improve the knowledge of the mechanisms of the market for health care through the derivation of significant policy implications. One of the most important messages is that in spite of competition, the regulator still should have a central role in the architecture of the market and policies have to be carefully tailored in order to avoid the onset of perverse, undesired effects on quality and welfare.

Using a Patient-Centered Multicriteria Decision Analysis to Assess the Value of Multiple Sclerosis Treatments in the US: A Study Protocol.

The engagement of patients and family caregivers in value assessment is pivotal since they provide valuable contributions to assessment acceptability and relevance. The proposed study aims to use patient-centered techniques and multicriteria decision analysis (MCDA) to evaluate the values of disease-modifying therapies (DMTs) for multiple sclerosis (MS) from the perspectives of patients and family caregivers living in three 'Deep South' States of the US-Alabama, Louisiana, and Mississippi. This study will follow guidance from the Patient-Centered Outcomes Research Institute (PCORI) for patient engagement and two best practice reports for MCDA from the Professional Society for Health Economics and Outcomes Research (ISPOR) to complete value assessment. Throughout the study, we will engage multiple stakeholders, including patients, family caregivers,healthcare providers, and payers. Forty patients with MS and their family caregivers from Alabama, Louisiana, and Mississippi will be invited to participate in this study. We will intensively train them for value assessment knowledge and MCDA before we engage them in MCDA to determine the value of DMTs for MS. Our approach differs from common MCDA since we incorporated a patient-centered framework in this study. Unlikeprevious studies only briefly inform or prepare participants before the MCDA process, in this study, we will provide basicvalue assessment trainings forpatients and family caregivers to ensure they can effectively engage throughout the patient-centeredMCDAprocess. We expect this study will demonstrate that the patient-centered MCDA approach is feasible and likely leads to improved patients' and family caregivers' engagementin value assessment.

Impacts of X-linked Retinitis Pigmentosa and Patient Pathways in European Countries: Results from the Cross-sectional EXPLORE XLRP-1 Physician Survey.

X-linked retinitis pigmentosa (XLRP) is a rare, incurable, vision-threatening, genetic disease. In this study, we aimed to reveal the real-world burden of this disease from the viewpoint of retina specialists and geneticists involved directly in XLRP care and to identify unique insights that may not otherwise be available through typical clinical studies or health economic research. In this exploratory, cross-sectional study (EXPLORE XLRP-1), retina specialists (n = 20) and geneticists (n = 5) in France, Germany, Italy, Spain, and the UK provided anonymized insights on their experiences managing patients with XLRP (n = 80) via an online survey and 60-min telephone interview. Survey respondents reported that patient independence decreased over time, where 37% of patients were considered "completely autonomous" at diagnosis versus 23% at the last consultation. At their last visit, 45% of patients were active in the workforce; 67% (12/18) of "completely autonomous" patients had active working status compared with 13% (1/8) of "completely dependent" patients. The average time from onset of symptoms to diagnosis was 4years and varied among countries. In 78% of patients, XLRP was confirmed by genetic testing, the rate of which varied among countries (range, 50-94%), taking up to 6months to receive results. Specialists identified unmet needs in XLRP management including more standardized assessments of quality of life (QoL) as well as easier and earlier access to specialists, genetic testing, patient support programs, and effective treatment options. The diagnosis, genetic testing, and management pathways among patients with XLRP can vary considerably. There is a need for more standardized diagnosis and management pathways, and QoL assessments, due to the major impact that XLRP has on patients' lives.

German Translation and Linguistic Validation of the LIMB‑Q: A Patient-reported Outcome Measure for Lower Extremity Trauma.

Lower extremity trauma can have a significant impact on a patient's quality of life. The LIMB-Q is a recently developed and validated patient-reported outcome measure that assesses patient-specific outcomes and experience of health care. The aim of this study was to translate and linguistically validate the LIMB-Q from English to German. The translation was performed by combining World Health Organization and Professional Society for Health Economics and Outcomes Research guidelines. The process consisted of forward translations, a backward translation, expert panel meetings, cognitive debriefing interviews with patients, and several rounds of discussion and reconciliation with the creators of LIMB-Q. The goal was to obtain a culturally and conceptually accurate translation of LIMB-Q into German for use in Switzerland. From the two forward translations, there was one primary discrepancy between the two translators that was discussed to determine the most conceptually accurate translation. From the backward translations, there were 63 items that required discussion and re-translation. Nine patients participated in the cognitive debriefing interviews, which led to three items being modified. The translation process led to a linguistically validated and conceptually equivalent German version of the LIMB-Q. The German (Switzerland) version of LIMB-Q is now available. This will offer a valuable tool for lower extremity trauma research and clinical care in German-speaking populations.

Public preferences and willingness to pay for a net zero NHS: a protocol for a discrete choice experiment in England and Scotland

IntroductionClimate change poses a major threat to our health, livelihoods and the planet. In 2020, the UK National Health Service (NHS) committed to reducing its Scope 1, 2 and 3...

OP12 Exploring Preferences in Selecting Dissertation Topics Among Ph.D. Students in Health Economics and Outcomes Research (HEOR) in the United States

OP12 Exploring Preferences in Selecting Dissertation Topics Among Ph.D. Students in Health Economics and Outcomes Research (HEOR) in the United States

Investigating the Application of Regression Discontinuity in the Field of International Health Economics

As one of the most significant quasi-experimental research methods, with over eight decades of development, Regression Discontinuity Design (RDD) has garnered extensive attention within the field of international health economics. It has provided robust methodological support for causal inference in research conducted within this domain. This paper provides a comprehensive review and synthesis of the foundational concepts, origins, and evolutionary trajectory of the RDD. It systematically examines the relevant studies in the domain of international health economics, discusses the strengths and limitations of the RDD, and anticipates its future applications in the same field. The aim is to support the utilization of RDD in international health economic research. On a theoretical level, it has effectively enhanced the flexibility and applicability of research designs within the domain of international health economics, augmenting the economic efficiency and external validity of studies, and offering a versatile tool for related research on a global scale. On a practical level, RD accommodates the diverse policy needs of programs across various countries and regions. It provides a more robust and reliable empirical foundation for the formulation of pertinent health policies, aiming to improve the efficiency of health resource allocation and promote equity in health outcomes.

The Effect of Unemployment on Health in Russia: a Systematic Literature Review (2015‑2023)

The influence of unemployment on health is widely discussed in the economic literature. However, the question about the negative direct effect or the deterioration of health of the unemployed population remains open in the context of the existence of a reverse effect — the influence of health on employment. This study aims to assess the statistical associations between unemployment and health status, as well as to identify direct and reverse effects in this relationship in the Russian studies. The authors use a systematic literature review as the main research method. The search is conducted in Elibrary, Cyberleninka and Google Scholar databases to find published works on the research topic in 2015-2023 for men and women of working age. The full-text search identified fifteen publications that were included in the systematic review. The selected papers used self-rated health, mental health, cardiovascular health, and general well-being as health indicators. All studies confirmed the presence of statistically significant negative associations between the “unemployed” status and health. At the same time, employed respondents of both sexes reported good health one and a half times more often than unemployed individuals. Nine studies presented econometric models linking the labor market status variables to health. Of these, only one paper estimated direct and reverse effects for a mental health variable. There was no direct effect estimate for the self-rated health. This gap in the literature will be addressed by the authors in their next article using the matching method. If the assessment of the direct effect is negative, the authors plan to develop a series of policy recommendations to improve the health of unemployed and economically inactive individuals at the regional and federal levels. Due to the innovative approach, this study is of a great value to the research in health economics.

Swedish translation and cultural adaptation of the scored Patient-Generated Subjective Global Assessment (PG-SGA©)-A validated tool for screening and assessing malnutrition in clinical practice and research.

The scored Patient-Generated Subjective Global Assessment (PG-SGA©) is a validated tool for the screening, assessment and monitoring of malnutrition, and triaging of interventions. It contains a patient-generated component and a healthcare professional (HCP)-generated component. To translate the PG-SGA into Swedish, assess the linguistic and content validity of the Swedish version, and ensure conceptional, semantic and operational equivalence to the original English PG-SGA. In line with the methodology used in previously translated and culturally adapted versions, the standardised 10-step process suggested by the International Society for Health Economics and Outcomes Research (ISPOR) was followed. In step 7, a cross-sectional study targeting patients n = 51 and HCPs n = 52 was performed at a university hospital in Sweden. Using separate questionnaires, patients assessed the patient component and HCPs, the professional component regarding perceived comprehensibility and difficulty (linguistic validity). The HCPs also assessed perceived relevance (content validity) of all items on the PG-SGA. Item indices for comprehensibility (I-CI), difficulty (I-DI) and content validity (I-CVI) were calculated and averaged into scale indices (S-CI, S-DI and S-CVI). Cut-off standards for item and scale indices were used as reference. The Swedish version of the PG-SGA rated excellent for comprehensibility (S-CI 0.96) and difficulty (S-DI 0.93) for the patient component. The professional component rated acceptable for comprehensibility (S-CI 0.89) and below acceptable for difficulty (S-DI 0.70), with the physical examination rated most difficult (I-DI 0.39 to 0.69). Content validity for the full Swedish PG-SGA was rated excellent (S-CVI 0.94). The patient component was considered clear and easy to complete. The full Swedish PG-SGA was considered relevant by HCPs for screening and assessment of malnutrition. Due to perceived difficulty with the physical examination, training of Swedish HCPs in using the PG-SGA is essential before implementing the professional component into clinical practice or research.

The impact of health on labour market outcomes: A rapid systematic review

The relationship between an individual's health and their labour market outcomes has long been a subject of health economics research. This review aims to provide an up-to-date, global review of the substantive findings in the existing literature. We pay particular attention to causal effects, acknowledging the methodological complexities that have long challenged the research and emphasizing the importance of overcoming them to present robust, policy-relevant evidence. The recent literature shows a notable advancement in addressing these methodological issues compared to previous work.The evidence reviewed suggests that individuals with better health overwhelmingly exhibit higher earnings and often enhanced labour supply. These findings extend beyond geographical boundaries, as evidence from diverse regions underscores the global significance of this association. The review covers evidence from a wide range of health indicators and conditions – including e.g. self-reported health, chronic diseases, disability, nutritional health, infections, mental health, addictions and others. Within and across the different health domains, the health-related factors exert varying degrees of influence on labour market outcomes, highlighting the multifaceted nature of the health-labour relationship and its potentially profound implications for individuals, communities, and economies.

Translation, Cultural Adaption, and Linguistic Validation of the Norwegian Wound-QoL Questionnaire

Background and Purpose:A sound translation is complex but necessary to provide a valid patient-reported questionnaire. The aim of this study was therefore to translate, culturally adapt, and linguistically validate the 17-item Wound-Quality of Life (Wound-QoL) questionnaire from English to Norwegian.Methods:For translation and cultural adaption of the Wound-QoL, we followed the methods described by the ISPOR—The Professional Society for Health Economics and Outcomes Research (formerly, the International Society for Pharmacoeconomics and Outcome Research), Messaging Application Programming Interface Research Institute, and recommendations from literature reviews.Results:Differences in the language semantics and syntax caused challenges in the translation. The cognitive debriefing interviews revealed some irrelevant items related to personal preferences.Conclusions:This study provides a questionnaire that can improve the quality of clinical care and promote research and valuable knowledge to other researchers translating the Wound-QoL and similar questionnaires.

Markov modeling for cost-effectiveness using federated health data network.

To introduce 2 R-packages that facilitate conducting health economics research on OMOP-based data networks, aiming to standardize and improve the reproducibility, transparency, and transferability of health economic models. We developed the software tools and demonstrated their utility by replicating a UK-based heart failure data analysis across 5 different international databases from Estonia, Spain, Serbia, and the United States. We examined treatment trajectories of 47 163 patients. The overall incremental cost-effectiveness ratio (ICER) for telemonitoring relative to standard of care was 57 472 €/QALY. Country-specific ICERs were 60 312 €/QALY in Estonia, 58 096 €/QALY in Spain, 40 372 €/QALY in Serbia, and 90 893 €/QALY in the US, which surpassed the established willingness-to-pay thresholds. Currently, the cost-effectiveness analysis lacks standard tools, is performed in ad-hoc manner, and relies heavily on published information that might not be specific for local circumstances. Published results often exhibit a narrow focus, central to a single site, and provide only partial decision criteria, limiting their generalizability and comprehensive utility. We created 2 R-packages to pioneer cost-effectiveness analysis in OMOP CDM data networks. The first manages state definitions and database interaction, while the second focuses on Markov model learning and profile synthesis. We demonstrated their utility in a multisite heart failure study, comparing telemonitoring and standard care, finding telemonitoring not cost-effective.

Current global status of male reproductive health.

Current global status of male reproductive health.

Estimating quality-adjusted life expectancy (QALE) for local authorities in Great Britain and its association with indicators of the inclusive economy: a cross-sectional study

ObjectivesQuantifying area-level inequalities in population health can help to inform policy responses. We describe an approach for estimating quality-adjusted life expectancy (QALE), a comprehensive health expectancy measure, for local authorities...