Open research questions in Health Systems, Economic Evaluations, Quality of Life
616 gap statements mined from Health Systems, Economic Evaluations, Quality of Life papers in our 7.9M-paper local library, which holds 4,612 papers on the topic — drawn mostly from each paper's own stated research gap, future-work, challenge and limitation notes, and its abstract. The ones listed below are a selection still marked open; each names the study that raised it, with a DOI link where the paper has one.
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What the literature leaves open
If a goal of a research design is to distinguish between mechanisms or detect a posited mechanism, outcomes for which Assumptions 1 and 2 are plausible should be prioritized in HTE analysis.
While many of the factors identified in our study are considered by HTA decision makers already, whether a greater consideration of broader factors is required for gene therapies, compared to existing therapies, is needed, remains an open question.
Patient, consumer and societal values, perceptions and preferences on high-cost gene therapies · 2026 · DOIWhether the framework can evolve to address these challenges while maintaining scientific rigor will be a defining question for the next phase of implementation.
Rigorous, transparent and inconclusive: what Europe’s first joint clinical assessment reveals about a framework trying to serve everyone · 2026 · DOIHowever, certain psychometric properties remain to be established in future work. Another limitation is that the current study utilized a cross-sectional database from a secondary analysis; consequently, the tool’s longitudinal sensitivity to capture clinically meaningful shifts in patient status could not be evaluated.
Development and psychometric validation of the short-form thai version of the spine oncology study group outcomes questionnaire (TH-SOSGOQ-8D) · 2026 · DOIFuture work should focus on establishing observational data on CAR-T outcomes in Colombia and on constructing stratified models that include age, performance status, and prior therapies. A limitation of this Study is that the cost of sourcing CAR-T remains uncertain.
Cost-utility analysis of CAR-T therapy compared to standard of care in patients with relapsed/refractory diffuse large B-cell lymphoma in Colombia, 2025 · 2026 · DOIThe study used a limited sample size of 226 interviews. The study was based on a computer-assisted, on-line interviewer-assisted interview, which may have limitations.
Ordinal relationship of composite time trade-off and discrete choice experiment preference data · 2026 · DOILimited, outdated, or geographically narrow existing studies on the economic burden of neurological disorders. Lack of comprehensive and comparable estimates of the economic burden of neurological disorders across Europe. Need for a standardized framework to assess the economic burden of neurological disorders.
The economic burden of neurological disorders in Europe – COIN-Eu: Study design and analytic framework · 2026 · DOIThe lack of established willingness-to-pay (WTP) thresholds in Sweden. The need to develop a methodological framework for the cost-utility analysis during the trial. The challenge of estimating the incremental cost of scaling up ELR in routine practice.
Evaluating the cost-effectiveness of Everyday Life Rehabilitation (ELR) for individuals with serious mental illness in supported accommodation · 2026 · DOIThe absence of a detailed health economic analysis plan required that the methodological framework be developed during the trial. The lack of quality-of-life data for those living at the housing unit but who did not participate in the intervention. Sweden lacks formally established willingness-to-pay (WTP) thresholds.
Evaluating the cost-effectiveness of Everyday Life Rehabilitation (ELR) for individuals with serious mental illness in supported accommodation · 2026 · DOIOne of the challenges is the uncertainty in the survival data and the assumptions used in the model. Another challenge is the limited availability of Japan-specific clinical trial data and real-world cost estimates. The study also faces the challenge of selecting the most appropriate methodology for the cost-effectiveness analysis.
Cost-effectiveness analysis of immune checkpoint inhibitors versus platinum-based doublet chemotherapy in the first-line treatment of advanced non-small-cell lung cancer with High PD-L1 expression in Japan · 2026 · DOIThere is a need for a comprehensive evaluation of the cost-effectiveness of immune checkpoint inhibitors versus platinum-based doublet chemotherapy in Japan. Prior studies may not have incorporated recent Japan-specific clinical trial data and real-world cost estimates.
Cost-effectiveness analysis of immune checkpoint inhibitors versus platinum-based doublet chemotherapy in the first-line treatment of advanced non-small-cell lung cancer with High PD-L1 expression in Japan · 2026 · DOIFurther outcomes are not known (yet) and cannot be considered in this analysis. The effect of the intervention on employment, i.e. sickness days and early retirement, is uncertain and should be studied further.
Complex intervention for MS patients in Germany – can it be cost-saving for the society? · 2026 · DOIThere is a lack of studies assessing the efficiency of interventions from a life-time perspective.
Complex intervention for MS patients in Germany – can it be cost-saving for the society? · 2026 · DOIThere is a lack of understanding about the appropriateness of the EQ-5D-5L and EQ-HWB measures in capturing loneliness. There is a need to develop interventions to address loneliness and evaluate their cost-effectiveness. The study aims to address this gap by examining the relationship between loneliness and utility scores.
Measuring outcomes for loneliness research: examining the appropriateness of the EQ-5D-5 L and EQ-HWB · 2026 · DOIThe gap between the normative focus on maximizing life-years in HTA and public preferences is not well understood. There is a need to investigate public preferences for prioritising lives versus life-years.
Public preferences for saving lives versus life-years: evidence from a person-trade-off experiment in 12 countries during the COVID-19 pandemic · 2026 · DOILack of country-specific value sets for the QLU-C10D for several countries. Need for accurate health economic evaluations in cancer patients. Limited generalizability of existing value sets to different countries and populations.
Cancer specific health utilities - QLU-C10D country value sets for Belgium, Hungary, Portugal and the Republic of Korea – an international valuation study · 2026 · DOIThe study identifies challenges in comparing the psychometric performance of the EQ-5D-3L and EQ-5D-5L, including the need for rigorous cost-utility analyses. The study also identifies challenges in ensuring the interchangeability of the two versions, including differences in mean utility scores.
Is EQ-5D-5L better than EQ-5D-3L? A comparison of descriptive systems and utility scores in patients with kidney chronic disease · 2026 · DOIOne challenge is the high acquisition cost of the Oncotype DX test. Another challenge is the need to determine the optimal threshold for guiding treatment decisions. The paper also mentions the challenge of balancing the benefits of the test with its costs.
Budget impact analysis of Oncotype DX® multigenic prognostic test in patients with early breast cancer in Italy · 2026 · DOIThe gap in the literature is the lack of studies on the budget impact of the Oncotype DX test in Italy. There is a need to assess the cost-effectiveness of the test in the Italian healthcare context.
Budget impact analysis of Oncotype DX® multigenic prognostic test in patients with early breast cancer in Italy · 2026 · DOIThis is further supported by the stability analysis, which suggested more late-decline and persistent adverse trajectories in Institution B and more late improvement in Institution A, even though formal statistical separation of stability classes remained limited by the smaller longitudinally evaluable subset.
Locked-Window EQ-5D-5L (Index and VAS) Benchmarking in Sarcoma Care: Rule-Based Traffic-Light Classification Across Two Institutions · 2026 · DOIFinally, the analysis does not account for specific organizational aspects such as unsuccessful interventions, extended follow-up pathways, or alternative remuneration models, which may influence cost structures in different settings. In particular, the economic model did not incorporate health outcomes and was therefore limited to a cost analysis rather than a full cost-effectiveness evaluation.
How Much Does a Home Care Nursing Visit Cost? A National Micro-Costing Study from the AIDOMUS-IT Project · 2026 · DOIQALE norms are not available for the Canadian population. Conventional cost-utility analyses may not capture the full magnitude of health losses associated with more severe conditions.
Quality-adjusted life expectancy norms for the Canadian population using EQ-5D-5L and SF-6Dv2 · 2026 · DOIThe sample size was limited to 170 patients. The study was conducted in a Spanish cohort of patients with chronic kidney disease. The study used a cross-sectional design.
Is EQ-5D-5L better than EQ-5D-3L? A comparison of descriptive systems and utility scores in patients with kidney chronic disease · 2026 · DOIA 2003 study of the costs of treating pneumonia in an NH estimated the cost at $458 but noted that the costs varied widely depending on clinical presentation.
Economic Evaluation of an Intervention to Improve Mouth Care and Reduce Pneumonia Incidence in Nursing Homes: An Exploratory Analysis · 2026 · DOIIdentifying and addressing data gaps and inconsistencies in the literature. Ensuring the accuracy and reliability of the estimates through expert validation. Developing a standardized framework to assess the economic burden of neurological disorders across 47 European countries.
The economic burden of neurological disorders in Europe – COIN-Eu: Study design and analytic framework · 2026 · DOI
Most-cited papers in Health Systems, Economic Evaluations, Quality of Life
- Dissecting racial bias in an algorithm used to manage the health of populations · Science · 2019 · 6,070 citations
- Recommendations for Conduct, Methodological Practices, and Reporting of Cost-effectiveness Analyses · JAMA · 2016 · 2,904 citations
- Recommendations of the Panel on Cost-Effectiveness in Health and Medicine · JAMA · 1996 · 2,204 citations
- Updating Cost-Effectiveness — The Curious Resilience of the $50,000-per-QALY Threshold · New England Journal of Medicine · 2014 · 2,059 citations
- Foundations of Cost-Effectiveness Analysis for Health and Medical Practices · New England Journal of Medicine · 1977 · 1,552 citations
- Discrimination and Calibration of Clinical Prediction Models · JAMA · 2017 · 1,442 citations
- The Role of Cost-effectiveness Analysis in Health and Medicine · JAMA · 1996 · 1,244 citations
- Recommendations for Reporting Cost-effectiveness Analyses · JAMA · 1996 · 1,179 citations
- Recommendations for reporting cost-effectiveness analyses. Panel on Cost-Effectiveness in Health and Medicine · JAMA · 1996 · 1,040 citations
- The role of cost-effectiveness analysis in health and medicine. Panel on Cost-Effectiveness in Health and Medicine · JAMA · 1996 · 970 citations
Most recent work
- One Pivotal Trial, the New Default Option for FDA Approval — Ending the Two-Trial Dogma · New England Journal of Medicine · 2026
- A United Kingdom Value Set for the EQ-5D-5L · Value in Health · 2026
- Measuring Public Preferences for Statin Therapy Using the Smallest Worthwhile Difference · JAMA Internal Medicine · 2026
- Estimating the costs of informal care for individuals with brain health disorders from 2000 to 2021: a modelling study · The Lancet Public Health · 2026
- Implications of Incorporating Environmental Sustainability Into Health Technology Assessment for Digital Health Technologies · Value in Health · 2026
- Cost-Effectiveness Analysis of 177 Lu-PSMA-617 Versus Cabazitaxel in Metastatic Castration-Resistant Prostate Cancer from a U.S. Health Care Perspective · Journal of Nuclear Medicine · 2026
- Global, regional, and national impact of the Expanded Programme on Immunization against 14 pathogens from 1974 to 2024: an economic evaluation · The Lancet Global Health · 2026
- Politicised Changes to the NICE Threshold Risk Making Cost-Effectiveness Analysis Performative, Not Informative · Applied Health Economics and Health Policy · 2026
- Advancing the use of artificial intelligence in health technology assessment activities: insights and next steps from the 2025 HTAi Global Policy Forum · International Journal of Technology Assessment in Health Care · 2026
- Operator and Institutional Recommendations and Requirements for Tricuspid Interventions: 2026 ACC/AHA/ASE/HRS/STS Expert Consensus Systems of Care Document · Journal of the American College of Cardiology · 2026
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