Open research questions in Health Systems, Economic Evaluations, Quality of Life
192 unresolved questions extracted from the limitations and future-work sections of 1,108 Health Systems, Economic Evaluations, Quality of Life papers in our library. Each links back to the study that raised it.
What the literature leaves open
Identifying barriers and facilitators for future PrEP scale-up in Indonesia - Fostering positive attitudes toward PrEP for provider comfort in offering it - Increasing empowerment for community workers as the main drivers of the PrEP program
Knowledge, Attitudes, and Experiences among PrEP Providers in the Indonesian Pilot PrEP Program: A Cross-Sectional Survey · 2026 · DOINo incentive or compensation was given for participants in this survey, - The survey used convenience sampling, - The study was limited to participants working at facilities involved in the pilot program
Knowledge, Attitudes, and Experiences among PrEP Providers in the Indonesian Pilot PrEP Program: A Cross-Sectional Survey · 2026 · DOIThe tool can therefore be considered a promising instrument, although further evaluation in a larger number of medical organizations is warranted.
Improving cost assessment and health economic evaluation of a new medical technology during clinical approbation: the role of a structured costing tool · 2026 · DOIConclusion: Despite the interest, the effective implementation of OBSP falls behind due to a lack of consensus on how this new reimbursement method can be a sustainable solution.
Towards implementing new payment models for the reimbursement of high-cost, curative therapies in Europe: insights from semi-structured interviews · 2025 · DOIAlthough steps have been taken in recent years, efforts remain insufficient, and structural and political barriers continue to hinder broader implementation.
HTAs undertaken on complex interventions introduced unique domains like politics, implementation, early stakeholder engagement, outcome uncertainty, adaptive methods and real-world data, with expert opinion recommended when data were insufficient.
Methods for the health technology assessment of complex interventions: A scoping review · 2025 · DOIThe relationship between sex, gender, and frequent healthcare utilization remains underexplored among individuals living with CP.
A sex- and gender-based analysis plus of frequent healthcare utilization among individuals living with chronic pain: a cohort study · 2025 · DOIBACKGROUND: The possibility of adverse effects of medical treatment (AEMT) is increasing worldwide, but little is known about AEMT in China.
Global trends and partial forecast of adverse effects of medical treatment from 1990 to 2019: an epidemiological analysis based on the global burden of disease study 2019 · 2024 · DOIResults: Numerous steps, such as the development of implementing acts and procedural guidelines remain to be taken.
Implementing the EU HTA regulation: Insights from semi-structured interviews on patient expectations, Belgian and European institutional perspectives, and industry outlooks · 2024 · DOIAlthough LLMs have shown remarkable potential in various areas, their performance in the dynamic environment of daily clinical practice remains uncertain.
However, no consensus exists on how biomarkers of aging should be validated before their translation to the clinic.
The major limitation of the approach adopted here is that it involves a case-by-case approach. Further studies of this kind are necessary before a definitive conclusion can be reached on the connection between process innovations in medicine and health expenditures.
Some countries with already expedited national review processes were initially resistant to participate. Different regulatory packages were needed for different countries. Regulatory authorities often hesitate to adopt digital platforms for reliance initiatives due to concerns about data security, continuous access, and potential conflicts of interest.
Accelerating Global Access for Indication Extension Through Regulatory Reliance Pilot: The First Indication Extension Pilot in Oncology · 2026 · DOIThere has been limited application of regulatory reliance to labelling variations, including indication extensions. Regulatory reliance for indication extensions remains a relatively unexplored area.
Accelerating Global Access for Indication Extension Through Regulatory Reliance Pilot: The First Indication Extension Pilot in Oncology · 2026 · DOIThere is a need for further development of MCDA in Italy. The study highlights the importance of considering multiple dimensions of value in healthcare decision making.
Multicriteria decision analysis in the Italian healthcare context: a review of applications and methodological approaches · 2026 · DOIFuture studies should evaluate the test-retest reliability and responsiveness of the PROMIS-16 and EQ-5D-5L. Future studies should compare the measurement properties of the PROMIS-16 and EQ-5D-5L in different populations and settings.
A direct comparison of the measurement properties of the PROMIS-16 and EQ-5D-5L in the U.S. general population · 2026 · DOIThere is a need for a comprehensive comparison of the measurement properties of the PROMIS-16 and EQ-5D-5L. There is a lack of understanding of the psychometric properties of the PROMIS-16 and EQ-5D-5L in the U.S. general population.
A direct comparison of the measurement properties of the PROMIS-16 and EQ-5D-5L in the U.S. general population · 2026 · DOIOur study encountered several strengths. First, we used the cTTO data from the Jordanian EQ-5D-3L valuation study[25]. Second, our study was representative of the Jor- danian population, and high-quality assurance standards were applied in accordance with the standardized EuroQol EQ-VT v2.1 valuation protocol [15, 16]. Furthermore, we collected not only socioeconomic characteristics but also information on health-related indicators, such as smoking and exercise. On the other hand, we have encountered several limita- tions: representativeness is tied to what is known and mea- surable. Our quota sampling did not include employment, education, and marital status as stratification factors in addi- tion to geographical region, age, and gender. In addition, we agree with van Nooten et al. (2018) that there is a need to systematically investigate the impact of respondents’ socio- demographics on cTTO health state valuation by designing a focused study with larger sample sizes rather than using by-product data from valuation studies [19]. Despite this Quality of Life Research (2026) 35:154 1 3 154 Page 10 of 12 limitation, future valuation studies in Jordan can benefit from this study in sampling planning. Another limitation may be the respondent’s understand- ing of exercise. We kept it a general question, without dif- ferentiating between going to the gym or engaging in any physical activity, such as walking. We did not ask about the number of family members in a household or the number of children per family, even though previous studies have shown these variables to be predictors of health state valu- ations [19, 64].
Factors associated with health state valuations: a secondary analysis of an EQ-5D-3L valuation study from Jordan · 2026 · DOIStructural barriers in topic selection, including a lack of institutionalised, participatory processes. The underrepresentation of rare disease communities in HTA decision-making. The need for policy and cultural change to achieve equity in HTA topic selection.
Integrating Equity in Topic Selection for Orphan Medicinal Products: Policy Implications for Health Technology Assessment in Rare Diseases · 2026 · DOIThe topic selection phase in HTA often lacks fairness and transparency, particularly disadvantaging orphan medicinal products for rare diseases. There is a need for conceptual frameworks and policy strategies to promote fairness in HTA topic selection.
Integrating Equity in Topic Selection for Orphan Medicinal Products: Policy Implications for Health Technology Assessment in Rare Diseases · 2026 · DOIDifferences in population characteristics, healthcare systems, and data availability may present significant challenges to the transportability of comparative effectiveness research evidence. The lack of broader, real-world population representation in randomized controlled trials limits the external validity of findings. The need for careful planning, transparent reporting, and early discussions with decision-makers like regulators and health technology assessors.
Transportability Analyses in Comparative Effectiveness Research: A Conceptual Framework and Methodological Principles Endorsed by the I nternational S ociety for P harmacoepidemiology · 2026 · DOIThe lack of a structured framework for considering transportability exercises in comparative effectiveness research analyses. The limited understanding of methodological principles for transportability analyses. The need for empirical testing and validation of transportability methods targeting different questions across the product lifecycle.
Transportability Analyses in Comparative Effectiveness Research: A Conceptual Framework and Methodological Principles Endorsed by the I nternational S ociety for P harmacoepidemiology · 2026 · DOIThe integration of estimand specification, measurement validation, and RWE phenotype development. The need for a structured approach to address intercurrent events and missing data. The requirement for a high-level conceptual synthesis of rapidly evolving regulatory, methodological, and technological paradigms.
From Concept to Claim: An Integrated Lifecycle Roadmap (ILR) for Patient-Centric Endpoints in Clinical Trials and Real-World Evidence · 2026 · DOIThe lack of an integrated framework linking components across the evidence lifecycle. The need for a coherent lifecycle perspective for endpoint development. The requirement for a structured approach to estimand specification, measurement validation, and RWE phenotype development.
From Concept to Claim: An Integrated Lifecycle Roadmap (ILR) for Patient-Centric Endpoints in Clinical Trials and Real-World Evidence · 2026 · DOIThe practical value of AI-enabled HTA is not guaranteed by technology deployment alone. There is a need to understand how AI-enabled HTA improves decision-making effectiveness in Saudi private healthcare.
AI-ENABLED HEALTH TECHNOLOGY ASSESSMENT AS AN ORGANISATIONAL CAPABILITY SYSTEM: MIXED-METHOD EVIDENCE FROM SAUDI PRIVATE HEALTHCARE · 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
- Five‐Hundred Life‐Saving Interventions and Their Cost‐Effectiveness · Risk Analysis · 1995 · 883 citations
- Consolidated Health Economic Evaluation Reporting Standards (CHEERS) 2022 Explanation and Elaboration: A Report of the ISPOR CHEERS II Good Practices Task Force · Value in Health · 2022 · 745 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 precision health approach to medication management in neurodevelopmental conditions: a model development and validation study using four international cohorts · medRxiv · 2026
- The EAT–Lancet Commission: issues and responses · The Lancet · 2026
- Is Progression-Free Survival 2 Ready for Prime Time in US Food and Drug Administration Regulatory Decision Making? · Journal of Clinical Oncology · 2026
- Risk adjustment to improve fairness and efficiency of resource allocation: a case from the Emilia Romagna region in the decentralized Italian SSN · Social Science & Medicine · 2026
- Social value of a childhood QALY - Revealed preferences from pharmaceutical funding decisions in Australia 2005–2023 · Social Science & Medicine · 2026
- Valuing health system resilience: The limits of conventional economic evaluation under catastrophic risk · Health Policy · 2026
- Comment on Schumacher et al. Shared Decision-Making Feasibility Gap in Obesity and Type 2 Diabetes Care · Diabetes Care · 2026
- Cost-Effectiveness of Pharmacologic Treatments in Adults With Overweight or Obesity: A Systematic Review for the American College of Physicians · Annals of Internal Medicine · 2026
- 2026 ESAIC Consensus Document on Mitigation Strategies in Intensive Care Medicine · European Journal of Anaesthesiology · 2026
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