In health technology assessment (HTA), “value for money” is frequently conceptualized as incremental cost-effectiveness (CE), with effectiveness measured as (quality-adjusted) life years gained (LYG). Commonly used CE thresholds have been subject to controversial debate. We reviewed and analyzed the worldwide economic literature on the value of a statistical life (VSL), which reported empirical estimates during the last 25 years.

We conducted an extended systematic literature search in the EconBiz and EconLit databases, spanning the period from January 1995 to December 2020. We transformed VSL data into the implied values of a statistical life year (VSLY) and analyzed the results by regional origin, elicitation method, and annual gross domestic product (GDP) per capita. Then, we performed a regression analysis using the ordinary least squares (OLS) model after log-transforming the VSLY estimates.

We identified 156 empirical economic studies with 169 unique VSL estimates. Overall, the median VSLY was EUR168,367 (mean, EUR256,701) or 6.3 times annual GDP/capita. The median VSLY (expressed as multiples of GDP/capita) differed by regional origin (North America: EUR288,994; 7.2; Europe: EUR168,367, 5.2; Asia: EUR45,260, 4.5) and by elicitation method (revealed preference/wage risk studies: EUR274,625, 9.1; stated preference/contingent valuation studies: EUR113,246, 4.4; stated preference/discrete choice experiments: EUR178,130, 5.2). Regression analyses confirmed that studies with data originating from North America resulted in significantly higher VSLY estimates. The difference remained statistically significant after adjusting for GDP/capita.

The empirical economic literature, while showing heterogeneity by elicitation method, suggests that the adoption of a preference-based or “demand-side” perspective leads to willingness-to-pay estimates for a LYG that exceed commonly used CE thresholds in the context of HTA.

Rheumatoid arthritis (RA) is a chronic inflammatory disease. Treatment aims to improve functional capacity and quality of life. Biological disease-modifying antirheumatic drugs (DMARDs) and oral inhibitor of Janus kinases (JAKs) used in treatment are high cost. The objective of this study was to analyze the profile of use and expenses of these medicines used in Brazil’s Unified Health System (SUS).

This is a retrospective cohort of users who received at least one biological DMARDs or oral inhibitor of Janus kinases (JAKs) registered in the Open Room on Health Intelligence—Sabeis/Datasus. The valuation of the 11 drugs standardized by the Clinical Protocols and Therapeutic Guidelines (PCDT) was carried out based on public purchases registered in the Price Bank in Health and an active search with the Ministry of Health, between 2017 and 2021. Expenses were calculated based on the number of High Complexity Procedure Authorizations (APACs) registered in the SIA/SUS database.

Evaluation of 195,163 patient dispensing records was undertaken. Spending on medicines was BRL408,146,527 (USD79,012,759) in 2017, BRL237,615,290 (USD46,002,748) in 2018, BRL213,790,222 (USD41,350,815) in 2019, BRL195,626,575 (USD37,837,644) in 2020, and BRL140,096,142 (USD27,109,717) in 2021. It was found that in 2017, adalimumab (33%), etanercept (28%), and golimumab (14%) were the most used biological DMARDs. It is worth noting that in 2017, adalimumab and etanercept represented more than 50 percent of the costs of treating RA in the SUS. In 2021, it was found that 61 percent of total expenses were distributed between the drugs etanercept (23%), golimumab (18%), certolizumab (14%), adalimumab (11%), and tofacitinib (11%).

In the period from 2017 to 2021, changes can be observed in the profile of use and expenditure of biological DMARDs and oral inhibitor JAKs, resulting from the incorporation of new drugs and the entry of biosimilars in the Brazilian market. However, adalimumab, one of the first biological DMARDs incorporated, is the most consumed and least expensive medicine.

Many cultures across the world have varying conceptions about death and dying. Perceptions about health states considered “worse than death” also vary based on sociocultural norms as well as health system capacity. We explore worse-than-death health states in Ghana as well as reasons for opting for death in those health states.

We interviewed 28 participants from three regions in Ghana to understand the contextual “value of life” in Ghana and factors influencing respondents’ decision to opt to die rather than live in a particular health state. Written consents were sought from all participants to partake in the study. Interviews were conducted in either Twi or English based on each participant’s preference and lasted for an average of 30 to 35 minutes. Interviews were transcribed verbatim and stored in NVivo software. Data were analyzed thematically.

We identified that health states perceived as worse than death were those associated with impairment in mobility, anxiety/depression, and pain/discomfort. Participants preferred death under these circumstances because they wanted to avoid the financial burden on themselves and family, time spent in caregiving by family, loss of personhood, and loss of social status. Decisions regarding health states worse than death hold considerable importance, particularly in a context where culture and societal norms play a role in shaping how quality of life is assessed.

An understanding of the value Ghanaians attach to health states perceived as worse than death provides useful information for patient-centered care. Findings from the study can provide evidence on healthcare resource allocation and aid policymakers and clinicians in making informed decisions on which treatments to prioritize, and how to maximize the overall health and well-being of individuals.

Dengue is an arbovirus that causes an acute febrile illness transmitted by the Aedes aegypti mosquito. The TAK-003 vaccine produced with Vero cells using recombinant DNA technology from the live and attenuated virus was approved by the Brazilian regulatory agency for preventing dengue infection, regardless of previous infection.

A cost-effectiveness assessment comparing vaccinating specific age groups versus not vaccinating was conducted through a microsimulation model that tracked all epidemic rates over 20 years. Transitions between five health states (susceptible, asymptomatic, ambulatory, hospitalized, and death) were considered. Direct costs included vaccine costs and dengue healthcare (inpatient and outpatient). Quality-adjusted life years (QALY) were the primary outcome. Deterministic and probabilistic analyses explored key model uncertainties.

At a price of USD34.00 per dose, the average incremental cost-effectiveness ratio (ICER) was USD11,724.40/QALY. In the probabilistic analysis, no simulation was below the threshold of USD8,000.00/QALY. In an alternative scenario, in which the price of the vaccine would not be subject to taxes in the case of direct import, the average ICER was USD10,378,09/QALY, with one percent of the simulations below the standard cost-effectiveness threshold.

At both price scenarios, dengue vaccine did not prove to be a cost-effective technology to be funded for patients between four and 60 years old. Sensitivity analyses showed that vaccine price is the most sensitive variable. This study can support price negotiations to improve access to the vaccine.

Recently, there has been considerable emphasis on survival curves for data extrapolation, especially in the field of economic evaluation in oncology. Common methods for adjusting survival curves are complex and heavily reliant on individual patient data (IPD), which may not be feasible for health technology assessment (HTA). We propose an alternative method for survival curve extrapolation with direct adjustment to aggregated data.

Common parametric survival analysis models were tested: exponential, Weibull, log-normal, log-logistic, generalized gamma, and Gompertz. We had access to the IPD from a published randomized clinical trial (n=694) testing therapies (anastrozole and fulvestrant) for metastatic breast cancer with 10 years of follow-up on progression-free survival (PFS) and overall survival (OS) outcomes. After adjusting the original IPD, we sought to fit models to published aggregated data (Kaplan–Meier curves) using nonlinear regressions and optimization algorithms. Both methods were compared in terms of visual inspection and statistical fit quality (Akaike information criterion [AIC] and Bayesian information criterion [BIC]).

Survival curves directly adjusted to aggregated data showed a visually similar profile compared to IPD adjustments. According to AIC/BIC values, Weibull and generalized gamma distributions best fit OS data, both in individualized and aggregated approaches. For PFS, log-logistic and log-normal curves were the best choices for the anastrozole arm, and for fulvestrant, the best choices were log-normal and generalized gamma for individualized data, and Gompertz and generalized gamma for the aggregated method. The proposed R language code proved to be reproducible and amenable to automation in future HTA applications.

Directly adjusting survival curves to aggregated data is a simple and useful alternative in situations where access to IPD is not feasible.

Therapies for early-stage cancers demonstrate clinical benefit with early endpoints that measure delayed or avoided disease recurrence, and survival benefits take years to confirm. Economic evaluations for health technology assessment (HTA) require assumptions about long-term benefits to project lifetime disease trajectories. We examined economic modeling approaches used in HTA for adjuvant/neo-adjuvant therapies to understand challenges, explore patterns, and identify opportunities for methodological improvements.

We included drug indications with Canadian Agency for Drugs and Technologies in Health (CADTH) reimbursement recommendations as of November 2023 for adjuvant/neoadjuvant treatment of early-stage solid tumors. We collected recommendation outcomes and details of submitted clinical and economic evidence. Adapting prior work and focusing on threats to validity arising from equivocal methodological challenges, we classified issues raised in the economic review, particularly those related to surrogacy, treatment pathways, and long-term benefit assumptions (extrapolation, duration of benefit, and cure). We made comparisons with respect to the approaches to long-term benefit assumptions and treatment pathways for drug indications that also had appraisals by the National Institute for Health and Care Excellence (NICE, UK) and the Pharmaceutical Benefits Advisory Committee (PBAC, Australia).

Seventeen drug indications for adjuvant/neoadjuvant treatment of solid tumors were included. Reimbursement was recommended in Canada for 83 percent of drug indications, all were reviewed and recommended in the UK, and 11/15 (73%) have been recommended in Australia. Assessments described overall survival (OS) as immature, but interim OS data appeared to be supportive. There was considerable variability in approaches to long-term benefit assumptions by both submitters and reviewers. Modifications to the assumptions were made in two-thirds of reviews before acceptance by HTA. There was notable inconsistency in approaches to handling treatment-waning, while cure assessment time greater than or equal to five years from initiation was consistently considered appropriate by reviewers.

While all assessments recognized immature OS data, positive reimbursement recommendations were common. There was important variability in application of methods to estimate long-term treatment effectiveness, particularly determining appropriate, evidence-informed assumptions for duration of benefit and cure. This research can support guidance, methodological advancements, and consensus-building to appropriately capture benefits and assess uncertainties for treatments in early-stage cancers with more consistency.

Health technology assessment (HTA) studies focused on equipment have particularities. The evaluation of orthoses (ORT) and prostheses (PRT) is even more challenging and tends to present more difficulties when compared to technologies such as medicines. Furthermore, in Brazil there are different manufacturing and dispensing processes for these technologies throughout the country; this heterogeneity could also affect the sustainability of the system itself.

(i) evaluate and disseminate the elements necessary to carry out adequate and sustainable assessments of non-implantable ORT and PRT and; (ii) produce a guideline on HTA assessment for non-implantable ORT and PRT.

Visits were made to ORT and PRT centers throughout Brazil to collect data on the production chain, dispensing, adaptation, and evaluation of the patient’s clinical results. At the same time, systematic literature reviews were carried out on two topics: (i) elements that must be considered in the manufacture, dispensation, and adaptation of non-implantable ORT and PRT; and (ii) HTA guidelines/examples focused on ORT and PRT.

Eight of a total of 12 centers throughout different Brazilian regions were visited. A scoping review was carried out on body-sizing methods for manufacturing non-implantable ORT and PRT, and the differences and particularities of each method. Another systematic review on existing HTA guidelines and the HTA assessment of ORT and PRT is ongoing. The data of the three phases will be analyzed and translated in a Brazilian guideline of HTA for non-implantable ORT and PRT.

With the network of regional and national centers and the sum of different actors (such as government agencies, universities, and health centers), working together for common ground and the sustainability of the health system in complex areas such as HTA of ORT and PRT would be viable. Even with this effort, the work to be done and the challenges are significant.

Suicide poses a severe public health challenge worldwide, impacting individuals, families, work, and society. The multifaceted nature of suicide demands a complex approach involving psychological, biological, social, cultural, and environmental factors. Recognizing suicide’s status as the leading external cause of death in Spain, prevention increasingly incorporates technology, specifically mobile and software applications.

A systematic review of the effectiveness and safety of mobile and software applications was conducted (MEDLINE, Embase, CINAHL, and PsycINFO databases). Outcome variables included: suicide; suicidal behavior; suicidal intent; suicidal ideation/thinking; self-perceived suicide risk; using/seeking mental health services; associated mental symptoms; mental health-related quality of life; satisfaction of the user and the health professional; adverse events related to the app, as defined in the included studies. Studies that do not include suicidal behavior, intention, or ideation were excluded. Where available data allowed, a meta-analysis was conducted for each outcome variable.

One systematic review and 13 randomized controlled trials (n=2,952) were analyzed. No significant differences were found in deaths by suicide or suicide attempts. At post-intervention, small but significant reductions were observed in suicidal ideation, hopelessness, depression, and worry, with anxiety reduction slightly above statistical significance. At follow-up (8 to 52 weeks), these variables also obtained significant results, except depression and suicidal ideation. Regarding safety, there was no significant difference in safety phone calls for participants with suicidal ideation.

The evidence on suicide prevention app effectiveness is of low quality, precluding conclusive findings. Attempt reduction is suggested at 21 percent, but the confidence interval includes a potential 60 percent increase. Evidence on suicide-related psychological variables (suicide ideation, depression, hopelessness, and anxiety) is of higher quality (low–moderate), but effects are small and clinically uncertain. Safety findings are uncertain, impacting risk/benefit balance.

The evolution of technology in medicine and information and communication technology (ICT) has been an important step to introduce innovative/resilient specialized health services. The emergence of telemedicine offers opportunities to enhance specialized diagnosis services. This study has evaluated the feasibility of using tele-Holter and tele-ABPM (ambulatory blood pressure monitoring) to build a resilient diagnosis network for cardiac pathologies in Paraguayan remote public hospitals.

This observational descriptive multicenter feasibility study is based on a telemedicine-driven approach for specialized cardiac diagnostic services using Holter and ABPM devices in remote and underserved public hospitals in Paraguay. A telemedicine platform was used to send records of Holter and ABPM devices from the remote hospitals to the cardiologist to screen cardiac pathologies.

During the pilot study, 52 cardiac diagnostic tests were carried out using the tele-Holter and tele-ABPM approach in 10 regional hospitals countrywide. Cardiac diagnosis was performed in 24 patients using Holter and 28 patients using ABPM. The most frequent findings using tele-Holter were normal (91.6%), not sustained ventricular tachycardia (4.2%), and atrial fibrillation (4.2%). Regarding tele-ABPM, the diagnoses performed were arterial hypertension (50.0%), uncontrolled arterial hypertension (40.0%), and normal (10.0%). Overall, an average of 90.0 percent of diagnosed patients suffered high blood pressure and 8.4 percent suffered heart disorder.

According to our results, the use of a telemedicine-driven approach to build a resilient diagnosis network for cardiac pathologies in remote underserved public hospitals in Paraguay is feasible. A widespread use-assessment should be analyzed before this tool is adopted.

The healthcare digital landscape has evolved and is crucial in shaping strategies for fortifying the health system, specifically in building a network for the future of health services. There is also considerable interest in digital health to facilitate innovation and access to health services in underserved hospitals. This study has evaluated the results of a telemedicine-driven diagnostic network in remote Paraguayan hospitals.

This is a descriptive study, where the results using a digital telemedicine-driven diagnosis innovation in remote public hospitals were evaluated as a tool to improve equity and accessibility of specialized diagnostic services countrywide. For these purposes, the type and frequency of diagnosis performed through a digital telemedicine platform was determined.

During the study, a futuristic telemedicine-driven diagnostic innovation was implemented in 67 hospitals countrywide. The digital telediagnosis network facilitated tele-electrocardiography (ECG), teletomography, tele-electroencephalography (EEG), tele-Holter and tele-ABPM (ambulatory blood pressure monitoring). The implemented digital telemedicine network has performed 828,073 tests in total between 2013 and 2023. The most performed diagnoses were ECG (543,815 tests) followed by teletomography (266,750 tests), EEG (17,418 tests), Holter (43 tests), and ABPM (28 tests).

According to our results, the telemedicine-driven diagnostic innovation network facilitates faster and more equitable access to tertiary-level diagnostic services for patients in remote underserved public hospitals in Paraguay. A widespread use-assessment is necessary before this platform is implemented.

As the most internally rigorous design, randomized controlled trials (RCTs) are the gold standard for assessing the efficacy and safety profile of interventions. Increasingly, health technology assessment (HTA) considers evidence from non-randomized studies. Guidance recommends synthesizing different study designs separately due to their different inherent biases/limitations. But when authors or reviewers misclassify studies, this could affect which studies are included and therefore have an impact on review results.

We are conducting a methods project to (i) identify a clear study design classification system, (ii) explore whether its use produces consistent study design categorizations among reviewers, and (iii) iteratively improve the classification system. We performed a pragmatic web-based search for study design categorization tools and used the resulting schemas to develop a clear algorithm for use by reviewers of all levels of experience, specifically in reviews of treatment interventions. Next, we tested tool consistency and user experience by web-based survey in a small internal sample of reviewers, each independently using the system to categorize 18 published studies.

A median of seven reviewers (range four to eight) categorized each study. Rater agreement using the chart varied widely, with 100 percent agreement on the designs of three studies (17%), and at least 75 percent of reviewers agreeing on one design for nine studies (50%). The most common agreement was reached on RCTs and non-randomized controlled trials. The most common sources of disagreement were between different types of cohort studies and between case series and controlled cohort studies, largely due to inconsistent reporting. We also identified several improvements: the wording of prompt questions, the ordering of designs, and the addition of new elements.

The classification system as initially designed led to too much variation in study design categorization to be useful. Consequently, we present a revised version that we now aim to evaluate in a larger sample of reviewers. Further research will also investigate whether using the tool would change the results of systematic reviews, using a small sample of published reviews.

Data visualization is a powerful communication tool to facilitate the understanding of a complex process or data. Data visualization has been used in health technology assessment (HTA) reports for a long period (e.g., PRISMA flow diagrams, critical assessments, or GRADE). This study aims to investigate the number and manners in which HTA reports have been using descriptive data visualization for the years 2021 to 2023.

The international HTA database was used to identify and download the HTA reports from the years 2021 to 2023. We applied the database limits: full HTA and completed reports. The records were imported into a Microsoft Excel spreadsheet and screened by eight independent researchers applying the inclusion criteria: full HTA (according to methodological definition), access to the full text, and use of data visualization with a descriptive purpose (we excluded PRISMA flow diagrams, forest plots, and others). The data were observed with the software Power BI. Our analysis included variables such as agency name, country, section, type of visualization, and software.

From the international HTA database, 1,128 records were exported: 89 records were directly excluded from this set as they were tagged as ongoing or other types of reports in the database; 1,039 records were screened. Around 30 percent of records were included for the data visualization screening criteria after fulfilling our inclusion criteria (full HTA and available full text). Finally, 12 percent of the reports included data visualizations for a descriptive purpose of their results or conclusions. The countries with a higher number of records included in this analysis were Germany (28%), Canada (18%), the UK (16%), and Spain (11%).

In our sample, we observed that data visualization is not widely used so far to describe outcomes and/or conclusions from HTA reports. An additional finding was the number of records tagged as full HTA in the international HTA database that were excluded as non-full HTA from our study.

A diverse range of services often supplements procedures that involve medical technologies and adds value along patient care pathways. However, these novel elements of value are often not captured in traditional assessment frameworks. ExpertLink is one such example. ExpertLink uses digital solutions to connect clinical experts worldwide, enabling remote training and collaboration, while maintaining the highest standard of patient care.

Rezum™ is a minimally invasive therapy for patients with symptomatic benign prostatic hyperplasia (BPH). It is a quick day procedure with proven safety, effectiveness, and durability in clinical outcomes. Leveraging ExpertLink, an expert in Sydney, Australia, remotely proctored 11 Rezum™ procedures in Malaysia in November 2022, supporting five urologists in five hospitals across five states within five hours. Efficient and straightforward procedures such as Rezum™ are well suited to remote proctorship. Through this case study, we quantify the sustainability, equity, and access benefits, illustrating the additional value ExpertLink brings across the healthcare continuum and beyond.

For a proctor traveling from Australia to Malaysia, over 6,500 kilometers and 17 hours travel time is saved, equating to an estimated 1,700-kilogram reduction in CO₂ emissions. Without ExpertLink, a proctor may be away from practice for up to a week. ExpertLink allows for continuity of practice, including consultations and procedures, during this time. For five doctors traveling from Malaysia to Australia for training, an estimated 7,400-kilogram reduction in CO₂ emissions and approximately 85 hours travel time is saved. ExpertLink provided 11 geographically dispersed patients with timely access to treatment and expedited the physician learning curve.

This case study illustrates the value for just one technology on one day. ExpertLink embodies novel elements of value that are not captured in traditional value assessment frameworks. Collaborative effort between stakeholders is needed to broaden the view of value in healthcare, incorporate additional elements of value in existing assessment frameworks, and appropriately recognize this often-uncounted value in decision-making.

The use of artificial intelligence (AI) in health care has the potential to improve clinical and patient outcomes and to reduce rising costs. There is an exponential increase in health technologies that use AI. We present a health technology assessment (HTA) case study demonstrating that the rapid rise in publications presents challenges for HTA bodies seeking to provide robust, timely assessments.

We conducted an HTA of AI-assisted endoscopy in the detection and characterization of lower gastrointestinal (GI) cancer. Searches were conducted up to October 2023. Unusually, the search targeted only the intervention: artificial intelligence and lower-GI-tract endoscopy (including colonoscopy, proctoscopy, etc.). The search strategy was peer reviewed. MEDLINE, Embase, KSR Evidence, CINAHL, Cochrane Library, and the INAHTA HTA database were searched, as well as ongoing trial registers and key websites. A date limit of 2010 onwards was applied, as Xbox Kinect launched in 2010 and was the first mainstream device used for healthcare imaging.

Two network meta-analyses, 15 meta-analyses, one systematic review of meta-analyses, and three systematic reviews published since 2020 were identified. One review conducted searches to January 2020, identifying three randomized controlled trials (RCTs); a review that searched up to February 2023 identified 21 RCTs. There was substantial overlap regarding included primary studies, but not all reviews included the same outcomes. An additional seven RCTs were published in 2023. We also identified 12 cohort studies published between 2021 and 2023. We prioritized higher quality meta-analyses of clinical RCTs to include all outcomes of interest and updated the meta-analyses for primary outcomes.

This case study of an HTA of an AI-related technology demonstrates how rapidly the field is moving. It is necessary to use well targeted but not overly exclusive search strategies, limit by date, and prioritize inclusion of identified evidence according to quality and availability of outcomes. Time should be allowed to update existing meta-analyses.

Patient engagement in the health technology assessment (HTA) field is crucial but faces significant challenges. This study focuses on identifying the barriers and facilitators surrounding the incorporation of patients in HTA while proposing solutions from the perspective of the Patient Interest Group (GIP) within the Spanish Network of Agencies for Health Technology Assessment and Services of the National Health System (RedETS).

An ad hoc questionnaire with four open-ended questions about barriers, facilitators, and proposals was developed and distributed among the GIP members through an email survey methodology. The obtained results were discussed in an open in-person meeting, and a thematic analysis of the data was conducted.

Fifteen people answered the questionnaire (at least one HTA researcher from each agency), and forty people participated in the face-to-face discussion. Main barriers included the perception of a lack of institutional support, insufficient participative culture, lack of time and experience, and difficulties in planning. Facilitators included activities organized within the GIP and methodological procedures and documents published. The main proposals for improvement were focused on the training and capacity building of HTA researchers and patients, institutional commitment to patient involvement, promotion of forums and activities with patient entities, and improvement of communication channels with patients and dissemination.

Despite current challenges in patient involvement, this study highlights facilitators and proposals for improvement. Specialized training and education, along with the promotion of a participative culture, emerge as relevant strategies.

Patient participation in health technology assessment (HTA) plays an increasingly relevant role due to increased recognition of its essential contribution to addressing uncertainties in evidence and its real-world application. The objective is to analyze and describe how patients with chronic obstructive pulmonary disease (COPD) participate in the evaluation of transcutaneous capnography (TC) in the primary care setting.

The Spanish Association of Patients with Chronic Obstructive Pulmonary Disease facilitated contact with three COPD expert patients. A face-to-face video interview was conducted with each patient to know about their knowledge of the technology, real-life experiences, and expectations. Patients were informed of the objective of the evaluation and signed confidentiality and conflict of interest forms. All interviews were conducted in April 2023 by two researchers. Expert patients with COPD were able to participate in the review of the protocol and in the final version of the report. Literature searches were also conducted on patient perceptions of TC compared to arterial blood CO2 measurement.

All patients were male, older than 60 years and were ex-smokers with greater than 30 years of tobacco consumption. Patients highlighted the relevance of early detection of COPD to facilitate the planning and organization of treatment as their clinical situation progresses. Likewise, they also emphasized the importance of the implementation of less invasive tests, and the proximity and accessibility of primary care. Two studies reported greater patient satisfaction and less pain with TC than with arterial blood gases.

Expert patients show high expectations for the technology, as it is less invasive than arterial blood gases; they also acknowledged its proximity in primary care settings and the potential for detecting complications of oxygen therapy. Patient participation in HTA adds intangible value, as they provide “disease-specific knowledge” and real-life applicability of the technology.

Patients diagnosed with low- to intermediate-1-risk myelodysplastic syndrome (LR-MDS) encounter symptom burden that diminishes their health-related quality of life (HRQoL). Erythropoiesis-stimulating agents (ESAs) remain an option to alleviate anemia-related symptoms. However, existing HRQoL studies show limited evidential support. This study assesses the impact of ESAs on LR-MDS patients’ EuroQol 5-dimension questionnaire (EQ-5D) scores compared to not using ESA as initial therapy.

The European MDS Registry (EUMDS) collects information including ESA usage, covariates, and EQ-5D scores at six-month intervals. Estimating average treatment effect (ATE) from observational data requires adjusting for several sources of bias. Our study controls for baseline and time-varying confounding by using inverse probability of treatment weights. Employing a methodology based on marginal structural models, we are able to estimate robust ATEs. A two-part mixed-effects beta model was used to calculate ATE during a four-year follow-up period. We compare ESA therapy every six months versus clinical management not involving the use of ESAs.

Our results show an overall positive ESA effect on EQ-5D over the four-year follow-up period. The majority of time points have a positive ESA effect after adjustment, though a few time points show no effect. The estimated ATE at four years is small: 0.046 (−0.031, 0.114).

We found that use of ESAs over a four-year follow-up period produces mostly positive treatment effect estimates after adjusting for time-varying variables and confounders. Our robust results can be used to inform more reliable treatment decision-making.

Electroanatomic mapping (EAM) has been shown to be an alternative procedure to fluoroscopy for the implantation of conduction system pacing (His-bundle pacing [HBP] and left bundle branch pacing [LBBP]) in patients with severe bradyarrhythmias, mainly those vulnerable to ionizing radiation. However, the evidence of its beneficial and harmful effects has not been assessed in a systematic review (SR).

An SR of the available scientific literature was conducted on the safety, effectiveness, and cost-effectiveness of implantation of the HBP and LBBP using EAM system versus fluoroscopy in patients with bradycardia with an indication for pacing therapy. Cochrane methodology and PRISMA statement for reporting were followed. A partial economic evaluation was carried out to compare the costs of both pacemaker implantation strategies from the perspective of the Spanish National Health System. A budget impact analysis was also conducted with a five-year horizon.

Seven comparative observational studies (N=259) analyzing the use of EAM versus fluoroscopy were selected. Statistically significant differences were observed in total fluoroscopy time: −9.87 minutes (95% confidence interval [CI]: −14.20, −5.53; p<0.01; I2=95%; k=7; n=231); His-lead fluoroscopic time: −8.08 minutes (95% CI: −10.36, −5.81; p<0.01; I2=0%; k=2; n=50); and His-lead radiation dose: −17.21 mGy (95% CI: −24.08, −10.34; p<0.01; k=1; n=20). No differences in total radiation dose, procedural success, immediate procedure-related complications, electrode revision, or device infection were found. The use of EAM represents an increase of EUR1,397.81 (USD1,513.88) per patient and a net budget impact of EUR1.63 million (USD1.77 million).

No differences between EAM and fluoroscopy in terms of procedure success and safety were found. Therefore, EAM is a valuable alternative for patients who should not be exposed to ionizing radiation. The inclusion of EAM systems, for the indication under study, in routine clinical practice would mean an increase in costs for the Spanish National Health System.

Health-related quality of Life (HRQoL) is reported as a strong predictor of survival. However, little is known about the relationship between HRQoL and influencing factors of gastric cancer among Korean patients. This study aims to investigate the HRQoL of Korean patients with advanced gastric cancer and identify factors influencing their HRQoL.

A total of 156 patients with advanced gastric cancer were 1:1 matched for demographics and clinical characteristics between a ramucirumab/paclitaxel combination group and a chemotherapy group. The objective response of the subjects was divided into four categories: partial response (PR), stable disease (SD), progressive disease (PD), and no complete response/no progressive disease (non-CR/non-PD). The six-month changes in HRQoL measured by EuroQol 5-dimension 5-level questionnaire (EQ-5D-5L) were estimated and further investigated for associations with demographic and clinical factors.

The average changes in HRQoL were highest in PR (0.086±0.057), followed by non-CR/non-PD (0.078±0.076), SD (0.035±0.097), and PD (no change). The HRQoL by age group was 0.176±0.088 in subjects 40 years or younger, 0.035±0.071 in the 50s, 0.067±0.072 in the 60s, and −0.003±0.029 in those who were 70 years or older. The HRQoL of females was 0.145±0.136, and for males it was 0.047±0.066. The HRQoL of the non-diabetic group was 0.072±0.095, and for the diabetic group was 0.044±0.057. The HRQoL of the non-hypertensive group was 0.097±0.097, and for the hypertension group was 0.020±0.048.

For advanced gastric cancer patients, the increase of HRQoL was diminished as cancer progresses, age increases, for males, and when comorbidity of diabetes or hypertension exists. The reported HRQoL data is valuable for health technology assessment of advanced gastric cancer agents such as ramucirumab, as this study is a publicly funded neutral study.

Herpes zoster and post-herpetic neuralgia (PHN) can cause significant pain and morbidity, impacting on the health-related quality of life of those affected. This research aimed to identify primary studies that elicited health state utility values (HSUVs) relating to both acute herpes zoster and PHN, and to assess their suitability for an economic model of herpes zoster vaccination.

Cost-effectiveness analyses of herpes zoster vaccination were identified from (i) a recently published systematic review of economic evaluations of herpes zoster vaccination and (ii) an independently conducted rapid review that sought to update the results of the identified systematic review. The evaluations were hand-searched to identify original studies that elicited HSUVs of interest. Narrative synthesis of the original study characteristics was conducted and possible sources of variation in the studies identified. Appraisal of the methodological quality of the studies was undertaken using a published open response tool that outlined key quality assessment criteria for HSUV studies.

Twenty-one studies that elicited HSUVs were identified. Values related either to changes in utility resulting from increased pain severity, or to changes with time since disease onset. Heterogeneity was observed, potentially caused by differences in population size and characteristics, methods of elicitation, and preference weights used to value utility. Methodological quality varied across studies, with concerns around low sample sizes for populations with PHN, and poor reporting of both missing data and uncertainty around the HSUVs obtained. Differences in study population, time of participant recruitment, and methods of measuring utility could limit the applicability of HSUV study data.

This study highlights both the heterogeneity in HSUVs obtained for herpes zoster and the challenges associated with selecting HSUV data suitable for an economic model. An evidence base from which to select values for an economic evaluation has been created, though poor methodological quality and reporting of the primary studies may compromise the validity of point estimates obtained.