Improving Consumer Engagement in Health Technology Assessment and Decision Making: Lessons from 'Values in Science'
Health technology assessments (HTA) and related decision making by regulators, payers and clinical policymakers (henceforth HTA-DM) are not simply technical processes, but normative ones, shaped by economic, moral, and scientific values. Since these value-laden decisions affect consumers, it is broadly accepted that their values need to be taken into consideration in HTA-DM and that they should be actively engaged in some aspects of these processes. This paper begins by examining current approaches to consumer engagement in HTA-DM. We then identify key challenges inherent in meaningful consumer participation, including procedural obstacles, representation issues, and difficulties in values interpretation. After briefly reviewing existing responses to these challenges, we demonstrate how insights from the philosophy of science-particularly scholarship on values in scientific inquiry-can provide new insights into the challenges of integrating consumers' values into HTA-DM and offer some strategies for more targeted and effective consumer involvement.
Cost-Utility Analysis of TAVI Versus Surgery in Low-Risk Patients with Severe Aortic Stenosis in the UK
European guidelines recommend transcatheter aortic valve implantation (TAVI; class IA) for symptomatic severe aortic stenosis (sSAS) in patients aged ≥ 75 years, if a transfemoral approach is possible. Recent cost-utility analyses based on the PARTNER 3 trial have suggested that TAVI with the SAPIEN 3 device is cost effective versus surgical aortic valve replacement (SAVR) in patients at low risk of surgical mortality in several European countries. This cost-utility analysis compares TAVI with SAPIEN 3 versus SAVR in patients with sSAS at low risk of surgical mortality from the UK healthcare system perspective, using 5-year PARTNER 3 trial data.
Data-Related and Methodological Challenges in Assessing the Cost-Effectiveness of Traditional and Genomic Newborn Screening Programs
As newborn screening (NBS) programs consider adding conditions, policymakers would benefit from evidence showing whether early screening offers greater economic and health benefits than standard clinical diagnosis and treatment. However, economic evaluations have not been consistently used to guide decisions on expansion of NBS programs globally. This narrative review discusses data-related and methodological challenges in conducting cost-effectiveness analyses (CEAs) of NBS programs including (1) difficulties in identifying appropriate comparators and related data, (2) gaps in understanding and limited data on health outcomes and costs of care among children with newborn-screened conditions who received effective treatment after clinical diagnosis without screening, (3) lack of data on short- and longer-term costs and outcomes for affected individuals identified through screening, (4) challenges in distinguishing the effects of screening from improvements in treatment over time, and (5) limited data on costs outside of the healthcare system, limiting the ability to conduct evaluations from the societal perspective. Advancements in genomic technologies, such as genomic sequencing and gene-targeted therapies, are expanding the number of conditions that are potential targets for NBS. Genomic NBS poses additional challenges for CEAs that stem from the ability to simultaneously identify hundreds or even thousands of conditions and limitations of knowledge of genotype-phenotype associations. The data-related challenges discussed herein may encourage cross-disciplinary collaboration to improve data collection. Addressing these methodological issues is imperative for generating reliable evidence on the costs and benefits of NBS program expansion, thereby supporting informed policymaking on adding new conditions to NBS programs.
Budget Impact Analysis of Implementing C-Reactive Protein Point-of-Care Testing to Aid Antibiotic Prescribing in Belgian Primary Care
The inappropriate use of antibiotics is a key driver of antimicrobial resistance (AMR) and can lead to various adverse side effects. C-reactive protein (CRP) point-of-care testing (POCT) in primary care has shown potential as an effective strategy to reduce inappropriate antibiotic prescribing. This study evaluates the budget impact of introducing CRP POCT in Belgian primary care over a 5-year time horizon.
Structured Expert Elicitation to Inform Long-Term Survival Extrapolations in Advanced Renal Cell Carcinoma
In the absence of long-term data, structured expert elicitation gathers expert judgments and associated uncertainties to assess the clinical plausibility of long-term extrapolations.
Preferences for Digital Mental Health Services in Urban China: A Discrete Choice Experiment
Despite the high prevalence of mental disorders in China, most patients do not utilize mental health services. Digital mental health services offer a potential solution, but their full potential remains untapped. Understanding consumer preferences is key to tailoring these services and boosting acceptance and adoption.
Correction: Therapy, Pills and Unmet Needs for Financial Reasons: Socioeconomic Inequalities and Inequities in Access to Mental Health Care in Spain 2014-2020
The Role of Price Variation in Economic Analyses for Cancer Screenings: A Rapid Review
Health care spending continues to rise, and recent policies have made prices more visible.
Opportunities for CMS to Further Specify and Broaden Its Approach to Assessing Unmet Medical Need in the Context of the Medicare Drug Price Negotiation Program
The United States Inflation Reduction Act of 2022 established the Medicare Drug Price Negotiation Program, directing the Centers for Medicare and Medicaid Services (CMS) to establish "maximum fair prices" for select drugs. In arriving at maximum fair prices, CMS is required to consider several criteria, including the extent to which a selected drug addresses unmet medical need (UMN). Through a targeted literature review of 48 original research studies, we identified and categorized 40 potential elements of UMN as they relate to pharmaceuticals, as well as treatment-related barriers to addressing this UMN, from the patient, caregiver, and societal perspectives. We synthesized these elements of UMN into seven domains: (1) traditional clinical effectiveness, (2) impacts on patient, caregiver, or family quality of life, (3) economic burden on the patient, caregiver, or family, (4) economic burden on society, (5) societal perspective elements, (6) elements of treatment administration, and (7) availability of other treatments. Comparing these elements with the United States Food and Drug Administration guidance regarding UMN reveals potential key gaps in the current CMS approach to assessing UMN in the Medicare Drug Price Negotiation Program, particularly regarding domains 2 through 6, including quality of life, economic burdens, societal elements, and treatment administration.
Health Technology Assessment Guidelines and Recommendations Across European Union Countries and the United Kingdom in Rare Disease and Paediatric Populations
Due to limited clinical data, high uncertainty, and outcome variability, assessing therapies for paediatric and rare disease populations poses specific challenges, often requiring adjustments to standard health technology assessment (HTA) frameworks. This study examines how national HTA guidelines and recommendations across Europe reflect these demands, identifying methodological adaptations and country-specific disparities.
Which Factors are Holding up the Integration of the Environmental Impact of Health Technologies in Health Technology Assessment? Insights from a Multi-Stakeholder Interview Study
The integration of environmental impact into health technology assessment (HTA) is increasingly discussed, but its practical implementation remains uncertain. This study explores multi-stakeholder perspectives on the progress, challenges, and equity implications of incorporating environmental considerations into HTA.
Aligning Indigenous and Western Concepts of Health Resource Decision Making in a Western Canadian First Nations Context
Western health economic evaluation tools often fail to reflect the relational, collective, and holistic perspectives that underpin Indigenous concepts of health. These limitations pose challenges when applying Western measures in Indigenous contexts. The individualistic foundation of the Western definition of health and the values that inform it are significantly different from the community-based values typically found in Canadian Indigenous communities. For health economics to effectively support Indigenous health decision making, a values-based approach should initially be undertaken to identify conceptual commonalities with Western perspectives.
A Mixed-Methods Assessment of India's Health Technology Assessment Ecosystem
This study aims to evaluate the technical quality of health technology assessment (HTA) studies conducted in India. Second, we aim to identify process-related challenges across the life cycle of an HTA from commissioning to policy translation.
A Systematic Review of Economic Evaluations on Interventions Targeting Insomnia or Hypersomnia
Insomnia and hypersomnia are sleep conditions associated with significant costs to the healthcare system and society. This study aimed to review the cost-effectiveness evidence of interventions for insomnia and hypersomnia, including psychotherapy, pharmacotherapy, and complementary and alternative medicine (CAM), across the age spectrum.
Willingness to Pay per QALY: A Systematic Review of Demand-Side Valuations with a Focus on Age and Disease Severity
Willingness-to-pay (WTP) studies offer a demand-side perspective on the monetary value of health gains, typically expressed as WTP per quality-adjusted life year (WTP/Q). These estimates can complement supply-side cost-effectiveness thresholds (CETs) and inform whether healthcare budgets align with public preferences. However, existing thresholds often overlook heterogeneity by condition or population characteristics.
Burden of Disease in Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS): A Scoping Review
Myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS) is a serious chronic and complex multi-system disease characterised by symptoms such as post-exertional malaise, fatigue, cognitive impairment and pain. Diagnosis is based on international consensus criteria, and no curative treatment is available. In the USA, its prevalence is estimated at 0.42% among adults, with women affected three times as often as men. Prevalence is expected to increase due to the COVID-19 pandemic. In addition to its severe symptoms, ME/CFS has a substantial economic impact. This scoping review aimed to systematically examine the global health, social and economic burden of ME/CFS.
The Economic Cost of Obesity: A Cost-of-Illness Study in Greece
Obesity represents a significant public health and economic problem worldwide. In Greece, where the prevalence of adult obesity is among the highest in Europe, no prior study has examined its economic impact among adults. This study estimates the total economic burden of obesity in Greece for 2024, adopting a societal perspective and considering both direct and indirect costs.
Mechanisms Considering Public Investment in Pricing and Reimbursement Decisions of Medicines and Other Health Technologies: A Scoping Review
Pricing and reimbursement (P&R) systems do not normally use public investments in research and development (R&D) as criteria when negotiating the prices and reimbursement of health technologies.
The Economics of Antibiotic Resistance: A Systematic Review and Meta-analysis Based on Global Research
Antibiotic resistance (ABR) is a growing global health threat; reliable evidence on its impact is crucial for prioritising public health interventions.
Healthcare Costs and Carbon Emissions of Stage III Melanoma Surveillance Imaging
The aim of this study was to estimate the health system cost and carbon emissions of diagnostic imaging tests undertaken by patients on different surveillance schedules for follow-up of stage III melanoma. We also aimed to demonstrate how different monetary valuations of carbon emissions affect overall cost.
A Systematic Review of Modelling Approaches in Economic Evaluations of Treatments for Inherited Bleeding Disorders
The aim of this review is to identify and assess modelling approaches in published model-based economic evaluations of treatments for individuals with inherited bleeding disorders.
