To promote robust prevention strategies and reflect broader economic impacts, we developed the productivity-adjusted life years (PALYs) metric, which captures a more comprehensive view of health’s societal value. This systematic review explores the use of PALYs across various disease contexts, illustrating its application as a novel outcome measure in health economic evaluations.

We conducted a comprehensive review of studies utilizing PALYs to identify effective methods for decision-making and to illustrate their applications. Using a snowball search, we selected studies that applied PALYs to quantify societal health burdens in specific diseases or contexts. Extracted data included health conditions, country setting, time frame, model type, outcomes, data type (incidence or prevalence), discounting, time horizon, prevention strategies, working-age population, productivity index components, PALY economic value, gross domestic product (GDP), and sensitivity analysis details. Income-level classifications were based on World Bank standards, and findings were summarized through narrative synthesis.

We reviewed 34 studies published from 2018 to 2024, covering conditions like cardiovascular and kidney disease, and environmental factors such as PM2.5 exposure. Most were conducted in high-income countries (n=20). Life table models (n=23) and dynamic models (n=8) were predominant, with other methods including mathematical projections, questionnaires, and cross-sectional designs. Studies focused more on prevention (n=18) than on chronic disease management (n=14). Diabetes, hypertension, sleep apnea, and non-optimal temperatures had the greatest societal impact, while epilepsy led to significant losses in life years and PALYs. Productivity indices varied, with losses ranging from USD1,137 to USD217,983 per full-time worker.

The diversity of health conditions and economic contexts covered in these studies highlighted a broad interest in the determinants of health as measured by the PALY. This diversity is essential, as it provides a comprehensive understanding of the economic impacts and health challenges. Moreover, the emphasis on prevention over chronic disease management suggests a strategic shift toward averting disease onset.

Geriatric assessment and management (GAM) is a guideline-recommended strategy for optimizing cancer management among older adults. In a recent cost-utility analysis of the Canadian 5C randomized controlled trial (RCT), GAM appeared cost effective only in selective patients. We assessed the cost-utility of GAM plus usual care (UC) versus UC alone in older adults with cancer using a decision model and best available evidence from four international RCTs: GAIN, GAP70, INTEGERATE, and 5C.

We conducted a model-based economic evaluation using pooled data from four international RCTs (GAIN, GAP70, INTEGERATE, and 5C), supplemented by additional evidence from the literature. Deterministic and probabilistic analyses were performed from the healthcare payer perspective, applying a six-month time horizon. The base case and main analyses used Canadian cost data. Sensitivity analyses included per-trial scenario analyses, one-year time horizon, and the use of USA costs. We reported healthcare costs per quality-adjusted life year (QALY) and the incremental net monetary benefit (INMB) using a CAD50,000(USD36,277) per QALY threshold.

The base case analysis using Canadian costs indicated that GAM was cost effective with an INMB of CAD1,117 (USD819) (95% credibility interval [CrI]: −CAD2,450 [−USD1,796], CAD5,035 [USD3,692]) and 70.7 percent probability of GAM being cost effective at a cost-effectiveness threshold of CAD50,000 (USD36,666) per QALY. Trial-specific results varied, with the GAP70 and INTEGERATE trials yielding positive INMB values (CAD2,635 [USD1932] and CAD2,886 [USD2,116], respectively), while 5C and GAIN resulted in negative INMB values (−CAD642 [−USD471] and −CAD268 [−USD196], respectively). Sensitivity analyses revealed that chemotherapy costs were the main driver of costs in both GAM and UC strategies.

Evidence showed that GAM is generally cost effective. However, GAM’s cost effectiveness varied across trial scenarios, driven primarily by differences in chemotherapy costs. Future research should focus on identifying key GAM components that most effectively reduce severe toxicity, hospitalizations, and chemotherapy-related costs to optimize overall cost effectiveness.

Equity-informed economic evaluations require the baseline distribution of health across equity subgroups upon which the equity impact of interventions is evaluated. The distribution of health status, measured as quality-adjusted life expectancy (QALE), by socioeconomic status (SES) is unknown for Australia. We aimed to estimate QALE across SES groups, stratified by sex and year of age, for the Australian population.

SES was measured with the Socio-Economic Indexes for Areas Index of Relative Socio-Economic Disadvantage, from quintile one (Q1) most socioeconomically disadvantaged, to quintile five (Q5) least disadvantaged, and remoteness categories from the Australian Statistical Geography Standard: major cities, inner regional, and outer regional to very remote. Life expectancy (LE) was estimated from 2022 Australian Bureau of Statistics mortality data. Mean short form (SF-6D) utility by age, sex, and SES was estimated from the Household, Income and Labour Dynamics in Australia Survey (2022) using linear regression. Person-years were multiplied by utility to determine the QALE for each sex-SES group.

At birth, LE for all individuals in Q1 was 78.7 years, compared with 86.3 years for those in Q5. Incorporating health-related quality of life amplified disparities, with those in Q1 experiencing a QALE of 43.9 (95% confidence interval [CI]: 42.6, 45.2) years, compared with 55.6 (95% CI: 54.1, 57.1) years for Q5, a 27 percent relative difference. There were modest disparities by remoteness. Individuals in major cities had a LE of 83.1 years and a QALE of 50.8 (95% CI: 49.8, 51.7) years, while those in outer regional to remote areas had a LE of 80.5 years and a QALE of 47.0 (95% CI: 44.2, 49.7) years.

There is clear disparity in QALE by SES in Australia, whereby both the quantity and quality of life decrease with increasing socioeconomic disadvantage and geographical remoteness. These findings highlight the need for targeted interventions to address health inequalities. These detailed QALE estimates can be applied to future equity-informed economic evaluations.

The assessment of technology in hospital settings is a crucial step towards ensuring the delivery of efficient, effective, and safe healthcare.

This study conducts a Hospital-Based Health Technology Assessment to evaluate the efficacy of a screening rapid test for mild Traumatic Brain Injury (mild TBI) utilizing blood biomarkers, specifically Glial Fibrillary Acidic Protein (GFAP) and Ubiquitin C-terminal Hydrolase L1 (UCH-L1). The assessment focuses on the clinical utility and performance characteristics of the proposed rapid test within a hospital setting.

The screening model was meticulously examined for its ability to accurately detect mild TBI, considering the sensitivity and specificity of GFAP and UCH-L1 as blood biomarkers. The study involved a thorough evaluation of the test’s diagnostic accuracy, comparing its outcomes with established standards for mild TBI diagnosis.

Results from the Hospital-Based Health Technology Assessment highlight the potential of the GFAP and UCH-L1 blood biomarker-based rapid test as an efficient screening tool for mild TBI within a hospital environment. The evidence results show that the test is highly sensitive (91 percent to 100 percent) for the prediction of acute traumatic intracranial lesions, which helps rule out injury when the result is negative. When used within 12 hours of injury in adult patients with mild TBI, this test holds promise in reducing the utilization of CT.

The findings contribute valuable insights into the feasibility and reliability of implementing this technology for timely and accurate identification of mild TBI, enhancing clinical decision making and patient care in hospital settings.

Incorporating patient preferences into health technology assessment represents a promising avenue for its enhancement. However, the complexity and diversity of methods for evaluating preferences, coupled with uncertainties regarding their impact on decision-making processes, present significant challenges to their effective integration. Consequently, the objective of this study was to evaluate the application of the MaxDiff analysis methodology for determining patient preferences, with a focus on pharmaceutical services as a case study.

The experimental design for scaling MaxDiff was developed using Sawtooth Lighthouse software. The design parameters included 12 pharmaceutical services. Descriptive statistics, hierarchical Bayesian analysis, latent class analysis, and logistic regression methods were employed to generate preference results.

The most preferred services were identified as follows: the creation of a personalized list of safe medications to address patient needs, with a quantitative contribution of 0.5; and the provision of rapid tests (for influenza and Helicobacter) and stroke risk assessment (quantitative contribution of 0.31). In contrast, patients did not prioritize services related to contraception selection or weight control and weight loss program development. Descriptive statistics, hierarchical Bayesian analysis, and logistic regression methods sequentially identified the most preferred services. Latent class analysis revealed two distinct consumer segments: Segment 1 (39%) and Segment 2 (60%), which differed in their sociodemographic characteristics and preferred services.

The obtained results can be utilized as patient-based evidence to understand patients’ unmet needs in the assessment of technologies such as pharmaceutical services, taking into account the local context. Despite the simplicity of the MaxDiff analysis methodology, the resource intensity and the level of knowledge among specialists and patients regarding preference collection methods remain significant barriers to its implementation.

Constraints on surgical capacity due to budgetary and workforce shortages necessitate prioritization. Lessons learned from the COVID-19 pandemic emphasize the societal debate around these decisions and stress the need to align decisions with societal preferences. This study examined societal preferences for prioritizing patients with three different conditions—breast cancer, deafness, or knee arthrosis—for scarce surgical capacity.

We conducted a labeled discrete choice experiment among 1,046 members of the Dutch public. Respondents completed 14 choice tasks in which they prioritized patients for surgery, based on condition, age, health-related quality of life (HRQoL) before and after surgery, and waiting time until surgery.

Respondents were more likely to prioritize patients suffering from breast cancer over patients suffering from knee arthrosis or deafness. Respondents were also more likely to prioritize patients with lower levels of HRQoL before surgery, larger surgery-related increases in HRQoL, and longer waiting times until surgery. They were less likely to prioritize patients who were relatively older, although the opposite held true for patients suffering from deafness. Observed preference heterogeneity largely resulted from differences in preference strength, rather than preference direction.

Our results provided insight into societal preferences for prioritizing patients with different conditions for surgery. This insight aids in understanding public outcry that may follow decisions deviating from societal preferences. Aligning prioritization decisions with societal preferences may increase their legitimacy. Further research may examine the relevance of these preferences for physicians and their willingness to be guided by this.

The aim of this study is to propose and validate a value assessment framework for Health Technology Assessment (HTA) for rare diseases drugs in Brazil.

A scoping review was performed to identify criteria used by HTA agencies in countries with public healthcare systems when evaluating orphan drugs. Based on the findings, a criteria framework for rare disease drugs was proposed for Brazil. Content validity was conducted over three rounds using Delphi technique and content validity ratio (CVR) approach was employed to evaluate the ratings from the eighteen stakeholders (experts and patients).

Twenty-nine HTA criteria for rare disease drugs were identified to compose the Brazilian framework. After three Delphi rounds, the final value framework comprised fifteen criteria categorized into four domains: disease-related factors, treatment-related factors, social and political factors, and economic factors. Among the most well-rated criteria by the CVR, considering the relevance attribute, were “relevance of outcomes for a rare disease,” “impact on patient’s quality of life,” “price negotiation,” and “adjusted cost-effectiveness threshold.” On the other hand, “budget impact threshold,” “innovative nature of treatment,” and “willingness to accept greater uncertainty in clinical evidence” received negative evaluations and were excluded from the final framework.

A value assessment framework validated by key stakeholders of rare diseases in Brazil could contribute to improve HTA transparency, decision making, and efficiency of the healthcare system, and inspire the development of a local guidance for rare-disease HTA.

Decisions to not reimburse or discontinue reimbursement of health technologies from public funding are considered increasingly necessary. However, such decisions remain politically sensitive and often provoke public opposition. Such opposition may be considerable and puts pressure on decision-makers to revoke or revise a decision; however, the specifics of this opposition remain largely unclear. Our aim was to obtain insight into these specifics by mapping the scientific literature on this topic.

We performed a systematic review following PRISMA guidelines. The electronic search was performed in Embase and using the Google Scholar, Google, and Startpage search engines and supplemented with a hand search in 2021. Both the electronic searches and the hand search were updated in 2022 and 2024.

Based on 81 articles, we developed a framework of 27 categories grouped under the ‘who, what, when, where, and why’ of public opposition to negative reimbursement decisions. Our findings indicated that patient representatives, physicians, and citizens are the primary actors opposing such decisions—often influenced by industry and politicians. These actors typically challenge the outcomes and arguments underlying decisions, driven by unrealistically high expectations of technology effectiveness and the assumption that decision-makers prioritize cost containment above all else. Additionally, our findings indicate increasing distrust among these actors toward decision-makers and evidence-based processes, leaving them vulnerable to commercial exploitation and misinformation.

Understanding the dynamics between actors is crucial for understanding public opposition to negative reimbursement decisions in health care. Aligning decision-making processes with public values, addressing misconceptions, and countering misinformation may enhance the legitimacy of reimbursement decisions. Further research should explore strategies to mitigate distrust and foster evidence-based engagement among actors to ensure more informed and equitable healthcare decision-making.

The Shared Learning model redefines patient engagement in health technology assessment (HTA) by combining immersion, knowledge sharing, and network expansion. Through flexible programs like Patient Voice Initiative (PVI) Patient Partners, it empowers advocates to deepen their understanding of HTA processes and disseminate insights broadly. This innovative, scalable approach ensures patient voices are central to decision-making, fostering informed contributions and addressing the challenges of rapidly evolving health technologies.

The Shared Learning model piloted by the PVI supported three patient advocates through a structured immersion in HTA processes. Advocates engaged directly in HTA discussions, meetings, and consultations, attending the HTAi Annual Meeting 2024 for deeper insights and networking. The model emphasized peer knowledge dissemination through webinars, workshops, and community forums, creating a ripple effect of shared learning. Advocates reflected on their experiences, identifying insights to benefit broader patient networks. This approach combined immersive learning, knowledge-building, and capacity expansion, fostering both vertical engagement with HTA stakeholders and horizontal knowledge-sharing within patient communities.

The Shared Learning pilot demonstrated success in enhancing patient engagement within HTA. Advocates deepened their understanding of HTA processes through direct participation and shared insights with wider patient networks, fostering a multiplier effect. Peer-to-peer knowledge dissemination expanded patient capacity, strengthening advocacy communities. Advocates utilized their experiences to host webinars, workshops, and community forums, broadening the reach of HTA knowledge. The initiative also improved the ability of patient networks to contribute meaningfully to decision-making processes. These outcomes highlight the model’s potential for creating sustainable, informed, and resilient advocacy communities equipped to address the challenges of rapidly evolving health technologies.

The Shared Learning model revolutionized patient engagement in HTA by emphasizing immersion, knowledge sharing, and network expansion. Through flexible programs like PVI Patient Partners, the model deepened engagement while enabling broader dissemination of insights. As health technologies rapidly evolve, this scalable approach ensures patient voices remain central to HTA decision-making, fostering informed, sustainable contributions within patient communities.

Left ventricular assist devices (LVAD) are a therapeutic option in patients with advanced heart failure (HF) not a candidate for cardiac transplant as destination therapy (DT). However, important uncertainties remain regarding the use of LVAD in the long-term in real practice settings. When planning registries, it is important to choose the appropriate outcomes that ensure comparability and reduce the possibility of bias.

The purpose of this study was to establish a minimum dataset (MDS) that should be collected in all LVAD for DT registries to meet the needs and demands of Health Technology Assessment (HTA) doers and health professionals.

To design the MDS for LVAD, a preliminary list of outcome domains and data items were developed attending to the gaps and research needs derived from existing evidence coming from HTA carried out at the European Network of Health Technology Assessment (EUnetHTA) level. The list of data items and domains was agreed upon by all involved HTA organizations and a three-round Delphi was conducted among an experienced panel of cardiologists to rate the importance of the items for measuring uncertainty gaps.

After the three-round Delphi process, the expert panel reached a consensus regarding eighteen outcomes and forty-seven variables divided into seven main domains (safety, effectiveness, patient acceptability, satisfaction, healthcare system impact, pharmaceutical management, and technique-related factors).

The MDS of outcomes and measures, developed based on research gaps and needs, can allow for standardizing data collection and improving the quality of data for decision making and practice.

Restricted access to orphan drugs significantly challenges rare disease patients, impairing health outcomes and quality of life. In Türkiye, systemic barriers such as regulatory inefficiencies, economic limitations, and inadequate stakeholder collaboration persist. This study evaluated these issues through stakeholder perspectives, proposing a collaborative framework to improve access, ensure equity, and enhance systemic efficiency in addressing rare disease challenges in Türkiye.

This qualitative study employed semi-structured focus group discussions with 19 patients and caregivers and 13 in-depth interviews with healthcare providers, policymakers, industry representatives, and regulatory bodies. Thematic analysis of 1,996 coded excerpts identified key barriers to orphan drug access, categorized as direct or indirect impacts. Grounded theory was applied to synthesize these findings into a cohesive framework addressing economic, regulatory, and systemic challenges. Data triangulation enhanced validity, ensuring the perspectives of diverse stakeholders were accurately represented. Stakeholder feedback on the framework was incorporated iteratively, ensuring practicality and adaptability for policy and decision-making in Türkiye. Supported by TUBITAK 2224-A Program for participation in HTAi 2025; it had no role in the study conduct.

The study revealed multifaceted barriers to orphan drug access in Türkiye, including fragmented regulatory processes, high costs, and inadequate inter-stakeholder collaboration. Indirect factors, such as limited public awareness and insufficient professional education, exacerbated these challenges. The proposed framework emphasizes centralized regulatory mechanisms to streamline approvals, public–private partnerships to mitigate financial constraints, and enhanced communication to ensure transparency and accountability. Stakeholder feedback highlights the framework’s adaptability and potential impact, particularly in addressing disparities within Türkiye. Success metrics, such as improved regulatory efficiency and patient outcomes, were defined to support future implementation and continuous improvement.

The proposed framework addresses critical systemic barriers to orphan drug access in Türkiye by fostering collaboration, streamlining regulatory pathways, and enhancing equity. It emphasizes the importance of stakeholder alignment and sustainable policy solutions for rare disease patients. Future research may focus on piloting the framework, adapting it for broader contexts, and developing evaluation tools to measure its long-term impact.

The EUnetHTA Core Model® is well-established in the HTA community. Some recommendations of corresponding guidance documents leave room for alternative methodological choices. Considering the new HTA regulation (HTAR), we aimed to identify needs for concretization (NCs) in EUnetHTA guidance and provide indicative methodological options.

We carried out a qualitative document analysis and structured group discussion. Twenty-two EUnetHTA documents were screened using transparent criteria. Identified NCs were classified into topics according to the PRISMA statement and presented to Austrian HTA practitioners (n = 11) during a structured group discussion. Participants rated NC’s importance. To identify potential solutions, selected key handbooks for generic (Cochrane) and HTA-specific (IQWIG/NICE) evidence synthesis were systematically reviewed and matching content was charted against the NCs.

Thirty-two topics with varying numbers of NCs were identified, twenty-six during the screening process, and six from the group discussion. Most of the topics related to evidence synthesis methods (nine topics), evidence eligibility criteria (nine topics), risk of bias (three topics), and certainty assessment (three topics). Other topics related to information sources, search strategy, data collection process, data items, effect measures, and reporting bias. One or more methodological approaches and recommendations could be identified for each identified topic from the included methodological handbooks.

Our analysis identified a need for concretization in some EUnetHTA guidelines. The structured overview of methodological options may support HTA doers in adapting and applying the guidelines to the national and local practical context.

Generic medicines form the backbone of affordable health care in low- and middle-income countries (LMICs), constituting a significant portion of pharmaceutical consumption. However, issues such as fragmented pricing policies and insufficient regulatory frameworks hinder equitable access and affordability. This study evaluated pricing models that address these challenges by integrating best practices, including tiered pricing mechanisms and enhanced transparency. The aim was to guide policymakers toward sustainable solutions that improve medicine affordability, equity, and market resilience.

A comparative analysis was conducted across 20 countries, chosen for their diverse healthcare profiles and potential to be best practice examples for LMIC contexts. Key elements analyzed included pricing mechanisms (e.g., tiered pricing, external reference pricing, internal reference pricing), regulatory frameworks, and incentives for local production. Data were gathered from government reports, healthcare provider feedback, and industry insights. A policy evaluation framework was applied to assess each model’s effectiveness in addressing affordability, accessibility, and equity. The analysis prioritized generalizability to LMICs while accounting for contextual adaptations such as regional economic challenges and varying levels of regulatory capacity.

Supported by TUBITAK 2224-A Program for participation in HTAi 2025; it had no role in the study conduct.

The findings revealed that tiered pricing structures significantly enhanced affordability by incentivizing market competition and reducing prices as generics enter. Transparent regulatory practices, including centralized databases and consistent price reviews, mitigated regional price disparities and increased trust among stakeholders. Countries fostering domestic production reduced import dependencies, stabilizing supply chains and ensuring medicine availability. These strategies, when applied collectively, have great potential to improve equitable access to generics in resource-constrained settings. The results highlighted the importance of adapting global best practices to the economic and healthcare realities of LMICs, emphasizing sustainability and resilience in pharmaceutical markets.

Integrating tiered pricing frameworks, regulatory transparency, and local production incentives provided a pragmatic pathway to improve generic medicine access in LMICs. These approaches align with global best practices and address affordability and equity challenges. Policymakers should prioritize such strategies while leveraging stakeholder collaboration. Future research should examine dynamic pricing models and their long-term impacts on market stability and healthcare outcomes.

Following acquired brain injury (ABI), individuals often experience anxiety and/or depressive symptoms. BrainACT is an adapted form of Acceptance and Commitment Therapy (ACT) tailored to this target group. The current study is a trial-based health-economic evaluation comparing BrainACT to a psychoeducation and relaxation control treatment.

An economic evaluation from a societal perspective was conducted in the Netherlands alongside a multicenter randomized controlled two-armed parallel trial including 72 participants. A cost-utility and cost-effectiveness analysis was conducted where incremental costs, quality-adjusted life-years (QALYs), and anxiety/depression (Hospital Anxiety and Depression Scale (HADS) score) were collected and presented over a 1-year follow-up period. Bootstrapping, scenario, and subgroup analyses were performed to test the robustness of the results.

The BrainACT arm reported non-significant lower total costs (incremental difference of €−4,881; bootstrap interval €−12,139 to €2,330) combined with significantly decreased anxiety/depression (HADS) (3.2; bootstrap intervals 0.7–5.7). However, the total QALYs were non-significantly lower (−0.008; bootstrap interval −0.060 to 0.042) for BrainACT. The probability of the intervention being cost-effective was 86 percent at a willingness-to-accept threshold of €50,000/QALY. The scenario and subgroup analyses confirmed the robustness of the results.

BrainACT may be a more cost-effective alternative to a psychoeducation and relaxation intervention for anxiety and/or depressive symptoms following ABI. Despite limitations, BrainACT appears to be a promising addition to treatment options in the Netherlands. Further research is needed to validate these findings, and consideration should be given to implementing BrainACT in Dutch clinical settings with ongoing monitoring.