Cytomegalovirus (CMV), a prevalent human herpes virus, has long periods of latency. It can reactivate following immunosuppression causing diverse clinical manifestations. In immunocompetent individuals, CMV infections are usually asymptomatic. However, in immunosuppressed individuals, such as patients with human immunodeficiency virus, CMV infections can cause serious complications. Antiviral treatment is crucial for minimizing health risks and preventing severe outcomes, such as retinitis and colitis, in this population.

A cost-effectiveness analysis was conducted using a decision tree model to estimate the incremental cost-effectiveness ratio (ICER) of ganciclovir (1 mg/mL) for people with acquired immunodeficiency syndrome (AIDS) affected by CMV-related retinitis or colitis. The model compared ganciclovir therapy to no ganciclovir, with the outcome being progression of CMV disease. Budget impact was considered over a five-year time frame with 100 percent diffusion among patients with AIDS and CMV-related colitis.

In patients with AIDS and CMV-related retinitis, use of ganciclovir incurred an incremental cost of BRL29,378.95 (USD5,188.70) with an incremental effectiveness of 0.51 in the therapeutic response rate and an ICER of BRL57,605.79 (USD10,173.93). For those with AIDS and CMV-related colitis, ganciclovir incurred an incremental cost of BRL10,040.71 (USD1,773.32), with an incremental effectiveness of 0.26 and an ICER of BRL38,618.11 (USD6,820.45). The estimated cumulative budget impact, considering 100 percent ganciclovir diffusion over five years, was BRL1,124,800,751.17 (USD198,654,342.23) and BRL571,223,309.75 (USD100,885,415.26) for patients with AIDS CMV-related retinitis and colitis, respectively.

The application of economic evaluation and budget impact tools is crucial for maintaining health system sustainability. Introducing treatments with high ICERs and budget impact can jeopardize the health system and access to treatments. Upholding criteria, such as the willingness-to-pay threshold, is essential for ensuring the sustainability and continuity of health care.

Implementation of polygenic risk scores (PRS) may improve disease prevention and management, but its benefits and costs are still to be quantified. The review aimed to summarize evidence on the economic evaluation of PRS, exploring how their use impacts healthcare resource utilization, cost outcomes, and overall cost-effectiveness in order to help guide healthcare decision-making and resource allocation.

The PubMed, Scopus, and Web of Science databases were searched. The search terms were constructed to identify full economic evaluations of healthcare programs containing PRS aimed at assessing the risk of diseases or other health outcomes in any target population. The search strategy was adapted to fit each database’s research criteria. The literature search was supplemented by scanning the reference lists of retrieved articles. No language or date restriction was applied. The database search was rerun just before the final analysis. This research was supported by the European Commission and the Ministry for Universities and Research under the National Recovery and Resilience Plan (M4C2-I1.3 Project PE_00000019 “HEAL ITALIA”).

Of the 1,891 articles screened, seven economic evaluations were included. They were conducted between 2020 and 2022 in Asia (n=1), Australia (n=1), Europe (n=2), and the USA (n=3). The field of application was ophthalmology (n=2), oncology (n=1), cardiovascular (n=2), nephrology (n=1), and endocrinology (n=1). The time horizon was lifetime (57%) or between five and 40 years (43%). The most frequent viewpoint was the health system perspective (n=5). The target populations included healthy people or individuals at risk for a specific disease. Performing PRS tests was found to be a dominant strategy, with lower costs and better health outcomes in all studies.

Although the integration of PRS in clinical practice seems to be a cost-effective strategy, the small number of studies available and the heterogeneity in the field of application and the target populations limits the generalizability of results. A health technology assessment approach could be useful for summarizing the evidence on all domains of PRS implementation.

Patients with hematological malignancies are at high risk of infections due to both the disease and the associated treatments. The use of immunoglobulin (Ig) to prevent infections is increasing in this population, but its cost effectiveness is unknown. This trial-based economic evaluation aimed to compare the cost effectiveness of prophylactic Ig with prophylactic antibiotics in patients with hematological malignancies.

The economic evaluation used individual patient data from the RATIONAL feasibility trial, which randomly assigned 63 adults with chronic lymphocytic leukemia, multiple myeloma, or lymphoma to prophylactic Ig or prophylactic antibiotics. The following two analyses were conducted to estimate the cost effectiveness of the two treatments over the 12-month trial period from the perspective of the Australian health system:

(i) a cost-utility analysis (CUA) to assess the incremental cost per quality-adjusted life-year (QALY) gained using data collected with the EuroQol 5D-5L questionnaire; and

(ii) a cost-effectiveness analysis (CEA) to assess the incremental cost per serious infection prevented (grade ≥3) and per infection prevented (any grade).

The total cost per patient was significantly higher in the Ig arm than in the antibiotic arm (difference AUD29,140 [USD19,000]). There were non-significant differences in health outcomes between the treatment arms: patients treated with Ig had fewer QALYs (difference −0.072) and serious infections (difference −0.26) than those given antibiotics, but more overall infections (difference 0.76). The incremental cost-effectiveness from the CUA indicated that Ig was more costly than antibiotics and associated with fewer QALYs. In the CEA, Ig costed an additional AUD111,262 (USD73,000) per serious infection prevented, but it was more costly than antibiotics and associated with more infections when all infections were included.

These results indicate that, on average, Ig prophylactic treatment may not be cost effective compared with prophylactic antibiotics for the group of patients with hematological malignancies recruited to the RATIONAL feasibility trial. Further research is needed to confirm these findings in a larger population and over the longer term.

Innovation is needed for the growing number of patients with chronic obstructive pulmonary disease (COPD). Pulmonary rehabilitation (PR) is effective in improving exercise tolerance and quality of life, but these benefits do not appear to be sustained. This highlights the need for cost effective methods to maintain benefits on completion of therapy. The findings of a large trial from the UK are reported.

A two-center randomized controlled trial of patients discharged from PR compared the costs and benefits of PR maintenance with standard care. National Health Service (NHS) resource use, personal expenditure, and societal costs were recorded over one year, and bottom-up costing was undertaken for the PR maintenance program. Changes in health-related quality of life were recorded using the EQ-5D-5L, and differences were compared with the level identified as significant for COPD. A cost utility analysis was undertaken from an NHS perspective; uncertainties in cost and outcome data were incorporated into a sensitivity analysis. Cost-effectiveness ratios and cost-effectiveness acceptability curves (CEACs) were computed.

The study included 116 patients who had finished PR within the last four weeks. The economic analysis showed that mean healthcare costs per patient for PR maintenance were approximately GBP139.72 (EUR165.57) lower than for usual care. The observed 0.118 advantage in mean quality-adjusted life-years (QALYs) (p<0.05) was above the threshold (0.051) for COPD significance. CEACs indicated there was a 97 percent chance of achieving GBP20,000 (EUR23,699.80) per QALY (NICE acceptance level ≤GBP30,000 (EUR35,549.70). Patient and societal costs increased this percentage. It was estimated that if patients with COPD completed a maintenance program following PR, the NHS could save up to GBP28.6 million (EUR33.89 million).

Our findings confirm that a structured PR maintenance program is highly cost effective in extending the benefits of short-term PR. The trial, undertaken during COVID, also signals the potential for emerging digital innovations to provide future transformative change in delivering self-management programs to sustain health and reduce NHS costs for people living with chronic conditions.

The incidence of facial palsy has been rising worldwide, with recent evidence emerging of links to COVID-19 infection. To date, guidance on cost-effective treatments is limited to medication (prednisolone). In terms of physical therapy, neuromuscular retraining (NMR) to restore balanced facial function has been most widely evaluated, but not in terms of cost effectiveness. The added value of telerehabilitation is unknown.

A multistage technology assessment was conducted, which included the following:

• • a national survey of current therapy pathways in the UK and patients’ and clinicians’ views on the benefits and challenges of telerehabilitation;

• • a systematic review of clinical effectiveness trials evaluating facial NMR therapy;

• • calculation of long-term morbidity costs (national economic burden) based on incidence, patient recovery profiles, health-related quality of life, and national facial palsy treatment costs (valuation of clinical improvements in monetary terms was provided by a national Delphi panel); and

• • evaluation of the cost effectiveness of telerehabilitation (remote monitoring wearables) added to current face-to-face NMR delivery.

Nationally, approximately five percent of patients with facial palsy (17% of unresolved cases) are referred for facial NMR. The long-term economic burden associated with unresolved cases is estimated to range from GBP351 (EUR417) to GBP584 (EUR692) million, indicating substantial savings if long-term recovery can be improved. Medical treatment costs are GBP86.34 (EUR102) million per annual cohort, and physical and psychological therapy costs are GBP643,292 (EUR762,561). Economic modeling showed that telerehabilitation was cost effective, producing a health gain and a cost-saving of GBP468 (EUR555) per patient. If scaled to the national level for all patients who do not recover fully, an annual saving of GBP3.075 (EUR3.65) million is possible.

Economic modeling indicates that NMR could improve patient outcomes and reduce costs. The national survey demonstrated that access to NMR therapy services is limited, so introduction of telerehabilitation could improve access for currently underserved populations. Future clinical trials need to incorporate economic evaluations to help inform decision-making.

Pembrolizumab is used as a monotherapy or in addition to chemotherapy for patients with non-small cell lung cancer (NSCLC) as an alternative to platinum-based chemotherapy. The objective was to conduct a cost-utility analysis for subgroups of patients with NSCLC using the Brazilian Unified Health System (SUS) threshold of BRL120,000 (USD24,458) per quality-adjusted life-year (QALY).

We built a partitioned survival model with three health states based on overall survival (OS) and progression-free survival (PFS) curves for three scenarios according to programed death ligand-1 (PD-L1) expression as follows: (i) pembrolizumab monotherapy for at least 50 percent PD-L1; (ii) pembrolizumab monotherapy for at least one percent PD-L1; and (iii) pembrolizumab plus chemotherapy for one to 49 percent PD-L1. The outcome of interest was QALYs, so utility values were derived from pre- and post-progression states according to the treatment received. Survival curves were extrapolated for 20 years using different distributions. The best fitted distribution was selected by visual inspection, clinical plausibility, and Akaike and Bayesian information criterion tests. Direct costs were also considered.

Pembrolizumab provided incremental gains of 1.23, 0.35, and 1.10 QALYs when compared with platinum-based chemotherapy for scenarios one, two, and three, respectively. The subgroup with the best incremental cost-utility ratio (ICUR) was pembrolizumab monotherapy for at least 50 percent PD-1 (BRL201.366 [USD41,041] per QALY gained), followed by pembrolizumab with chemotherapy for one to 49 percent PD-L1 (BRL267.216 [USD54,463] per QALY gained). When assessed for all PD-L1 positive patients, the ICUR reached BRL571.425 (USD116,465), which was 4.5 times the cost-effectiveness threshold. Sensitivity analysis showed that pembrolizumab must cost a maximum of BRL4.761 (USD970) per vial to be cost effective for the SUS, which is a 40.7 percent reduction in the base case price.

Evidence shows that pembrolizumab alone or in combination with chemotherapy is more effective than platinum-based chemotherapy for treating patients with NSCLC, especially those with a high expression of PD-L1. However, price reduction is essential for pembrolizumab to be cost effective for treating these patients in the SUS.

Cardiac rehabilitation (CR) with physical exercise is crucial for the secondary prevention of myocardial infarction and heart failure. However, according to published studies there are differences in access to hospital-based CR depending on sex, age, ethnicity, and geographical region. An alternative is CR in non-hospital settings such as primary care, the patient’s home, or another place by means of telerehabilitation.

We conducted a systematic review of full economic evaluations where non-hospital CR was compared with hospital CR in patients with ischemic heart disease or heart failure. Other eligibility criteria were model-based or clinical trial-based evaluations; studies reporting quality-adjusted life-years, years gained, or other clinical outcomes relevant to CR; and studies published in English or Spanish. Searches were conducted in June 2023 in various literature databases, including MEDLINE, Embase, CINAHL, Web of Science, INAHTA, PEDro, the Cost-Effectiveness Analysis Registry, and others. Study selection, data extraction, quality assessment, and evidence synthesis were conducted by one economist and checked by a second reviewer.

Nine studies were selected from the 673 references identified. Another study was identified through previous systematic reviews. Ten randomized clinical trials were included in the review. None of the studies found differences in effectiveness between hospital CR and non-hospital CR. Two studies found that non-hospital CR was less costly than hospital CR, whereas the remainder did not find any differences in costs between the two groups or were unable to demonstrate the statistical significance of any differences observed. The best conducted studies concluded that non-hospital CR was as effective as and less costly than hospital CR.

Non-hospital CR was as cost effective as hospital CR for low- to-moderate risk patients. Based on the evidence, CR can be recommended in non-hospital settings. However, any form of CR should be evaluated after implementation because its complexity limits the generalizability of results across regions.

Partitioned survival analysis (PtSA) is a useful modeling technique, especially in oncology, in which different parametric distributions can be used to extrapolate survival data. Many studies lack justification for their chosen distributions, neglecting exploration of uncertainty in extrapolated estimates. We evaluated how different distributions impact the incremental cost-utility ratio (ICUR) for docetaxel, compared with abiraterone plus docetaxel (AA+DTX), for prostate cancer.

A three-state PtSA was constructed using overall survival (OS) and progression-free survival (PFS) curves from a docetaxel trial extrapolated over a 20-year horizon with exponential, Weibull, and Gompertz distributions. Log-normal, log-logistic, and generalized gamma were not considered because they are not compatible with the proportional hazards assumption. Curves for AA+DTX were adjusted using hazard ratios (HRs) from indirect comparisons for OS (HR 0.75, 95% confidence interval [CI]: 0.59, 0.95) and PFS (HR 0.50, 95% CI: 0.35, 0.72). Assessment included visual inspection, clinical plausibility, and Akaike and Bayesian information criterion (AIC/BIC) statistics. Utility values, medication costs, and disease monitoring costs were considered based on health state (pre- or post-progression).

For docetaxel PFS, visual inspection showed no significant differences between the three distributions used for the proportional hazards model, while the exponential model showed the longest tail for OS. When choosing by the lowest AIC/BIC (OS: Weibull; PFS: exponential), cost-effectiveness analysis resulted in an ICUR of BRL79,224 (USD16,139) per quality-adjusted life-year (QALY). The maximum and minimum ICUR was BRL81,559 (USD16,615) (OS: Gompertz; PFS: Weibull) and BRL70,136 (USD14,288) (OS: exponential; PFS: Weibull), respectively, which represented an important variation in the base case scenario (AIC/BIC) ICUR.

Using the Brazilian cost-effectiveness threshold (BRL120,000 [USD24,446] per QALY gained) and choosing any of the three distributions of the proportional hazards model, AA+DTX would be considered cost effective, which would not change the direction of the recommendation. However, despite the few recommendations in the literature regarding the adoption of parametric models for economic analyses in health, it is important to explore scenarios with different distributions.

In 2020, the World Health Organization (WHO) released guidance for climate-resistant and environmentally sustainable healthcare facilities. Implementation of sustainability into healthcare policy is at varying stages, and its impact on technology appraisal and procurement is expected to differ across countries. The objective of this research was to review healthcare sustainability policy and implementation progress across the EU5.

Healthcare sustainability policies in the EU5 (France, Germany, Italy, Spain, and the UK) were reviewed. Policies were identified via government websites (e.g., the Ministere de la sante et de la prevention) and through review of climate and sustainability policy news on the WHO website. Common goals featured in the policies included carbon reduction plans, reduction of single use plastics, ethical and environmental considerations, and self-sustainability. These four goals were selected to form sustainability categories for comparisons across markets. The policies were reviewed for each of the categories with respect to level of inclusion, targets, and anticipated implementation dates.

The UK had clear policies focusing on carbon and emission reduction. Implementation of these policies began in April 2023 and will become a requirement for all new procurements from April 2024. France, Italy, and Spain mentioned carbon emission reduction and environmental impact in policies, but targets and guidelines have yet to be set. Italy and the UK had policies relating to reduction of single-use plastics, whereas the other markets did not. Germany had limited policies mandating changes geared toward increased environmental sustainability, focusing instead on self-sustainability and resilience in the market.

As expected, incorporation of sustainability plans into healthcare policy is variable. Within markets with established policies, such as the UK, there is progress toward mandating the inclusion of sustainability policy in procurement, the impact of which may be seen in early 2024. Although the progress of implementing sustainability in other markets is slower, countries are moving toward environmentally sustainable healthcare policies.

Chronic kidney disease (CKD) is a progressively worsening condition that is often overlooked in its early stages. In Brazil, factors such as population aging and rising comorbidities are expected to shift CKD prevalence toward more advanced stages, leading to greater socioeconomic and environmental impacts. The significant burden of renal replacement therapy (RRT) suggests the need to prioritize preventive and early detection strategies.

We developed a patient-level simulation model to estimate the impact of CKD in Brazil over 10 years (from 2023 to 2032) on clinical, patient, health system, environmental, productivity, and societal outcomes. Validation was conducted against Brazilian demographic data and cross-validated with the Inside CKD model. We estimated productivity losses by multiplying CKD-related workdays missed by daily costs for patients and caregivers.

The number of Brazilians with CKD was projected to increase by 7.2 percent (approximately 27.7 million) over the next 10 years, mainly among patients with late-stage disease, while the number of patients undergoing dialysis was projected to increase by 170.8 percent (approximately 233,000) over the same period. CKD was projected to result in BRL198 billion (USD 38 billion) of lost income. From an environmental standpoint, freshwater consumption, fossil fuel depletion, and carbon dioxide emissions due to patients with CKD were projected to increase by 40 percent by 2032. RRT was projected to require the equivalent annual water usage of approximately 370,000 households and the annual power of approximately 11 million lightbulbs and will produce annual carbon dioxide emissions equivalent to approximately 1.5 million cars.

While the overall number of patients with CKD will increase by 7 percent (from 25.8 million in 2022 to 27.7 million in 2032), the distribution toward later stages of CKD will cause significant impacts in terms of the healthcare system (resource use and costs), patients and caregivers, society, and the environment.

A scoping review was conducted to understand which environmental factors are currently being discussed as candidates for inclusion in health technology assessments (HTAs). By mapping the current literature, the results of the scoping review will inform future work to assess the importance policymakers place on aspects of environmental concern.

The scoping review involved literature searches across three electronic databases (MEDLINE, CINAHL, and PsycINFO), encompassing articles published up to the end of 2023, to identify a starting set of articles. Backward and forward citation searching of relevant studies was then employed to expand the scoping review, with inclusion criteria established to select studies based on the specific focus on HTA. Screening was conducted independently by two reviewers in two stages: title and abstract and then full-text screening. Data extraction involved a structured approach, collating relevant information for thematic analysis.

While the scoping review is ongoing, emerging issues included: methods for incorporating environmental pollution externalities into HTAs; the impact of climate change on health outcomes; intergenerational equity of health outcomes and definitions of sustainability; environmental epidemiology; and the potential for technologies to mitigate the impact of climate change. The ongoing thematic analysis will synthesize the results of the scoping review using mapping and categorization to identify knowledge gaps.

The threat of climate change is the greatest issue faced by HTA in a generation, but this challenge is intersectoral and demands an intersectoral response. This paper identifies environmental concerns that might, in principle, be included within the HTA process. Ultimately, the HTA community must do its part to address the environmental crisis we face and move toward a more sustainable future.

The National Health Service (NHS) in England aims to be carbon neutral by 2045, acknowledging the link between planetary and human health. By 2028, NHS suppliers will need to report carbon footprint data for their products, including the medicines and technologies that the National Institute for Health and Care Excellence (NICE) appraises. Additionally, company level data are already being reported. This type of data may provide NICE with an opportunity to support system environmental sustainability goals.

The NICE Science Policy and Research team conducted an options appraisal to consider the feasibility and acceptability of different ways NICE might engage with environmental impact data (EID). We held discussions with NICE teams that could be affected by the collection and use of EID. Discussions examined the array of potential options for NICE—from being a simple conduit for EID to incorporating EID into its decision-making—and sought to identify those that were practical for further consideration. We then discussed these options with key external stakeholders (NHS England, industry, and commissioners) to understand their expected usefulness and practicality.

Several options for NICE to use EID were identified as suitable for further consideration. Using company level data, NICE could encourage companies to engage with NHS sustainability goals by citing their carbon reduction plans alongside guidance or using them to prioritize its activities. Using product level data, NICE could pilot an evaluation comparing the environmental outcomes of competing health technologies that have no differential direct health outcomes. NICE could also publish environmental impact assessment tools to help commissioners consider EID in procurement decisions. Better data and methodological standards are needed before NICE might consider embedding product-level EID in its usual decision-making frameworks.

Our options appraisal has identified several ways that NICE might start to engage with EID to have a positive impact on NHS sustainability goals and wider planetary health. Future work will scope out how these activities should proceed. The identified options are not necessarily mutually exclusive and may evolve as the data and methods around sustainable health care continue to advance.

Medical devices cause significant spending in healthcare systems and provide methodological challenges for health technology assessment (HTA). This poster presents the results of a dissertation on the HTA of prosthetic limb technology and care, with a focus on the ethical implications of this aspect of health care. It is also a case study for ethics in HTA more generally, which will contribute to methodological discussions in this field.

The methodology was based on Hofmann´s Socratic approach and empirical ethics. Literature reviews of HTA reports and ethics and social science literature on limb prosthetics and care were supplemented by semi-structured interviews with 16 prosthesis wearers and 18 stakeholders (e.g., insurance payers and orthopedic technicians) from Germany. This material served to identify and prioritize ethical issues and dive deeper into the causes of unequal access to prosthetic limbs. Beauchamp and Childress´ principlism and Norman Daniels’ theory of just health were used to describe ethical requirements and conflicts and to discuss the limits of normative recommendations that HTA can provide for decision-makers.

There were 42 ethical aspects related to limb prosthetic technology and care identified, reflecting also general challenges for HTA like artificial intelligence in health and resource scarcity in times of multiple crises. The perspectives of patients and stakeholders provided evidence for unequal access to limb prosthetics that was dependent on socioeconomic status, age, and living region. This is due to a combination of legal framework conditions (not supporting evidence-based reimbursement), socioeconomic success factors in interaction with gatekeepers, non-optimal quality of care (due to lack of data use and scarcity of professionals), and political non-willingness to address rationing–as well as the lack of HTAs.

Access issues in prosthetic care and their implications for patient wellbeing, efficiency, and sustainability may be generalizable to a certain extent to other medical device types and healthcare systems. Governments should provide resources, and synergies with health services research could be leveraged to enable HTA to address the challenges of medical device and ethical assessment.

To ensure accurate and objective comparisons between interventions, the best available clinical evidence must be used. This updated analysis sought to review the quality of clinical evidence submitted by applicant companies to a national health technology assessment (HTA) agency in Ireland. This analysis aimed to assess the association between the quality of clinical evidence and the reimbursement recommendation.

A retrospective analysis of the clinical evidence supporting all oncology HTAs submitted to the National Centre for Pharmacoeconomics (NCPE) from May 2012 to December 2023 was conducted. NCPE recommendations were classed as “conditional-positive/positive” or “conditional-negative/negative” for the purposes of this analysis. Data extraction of key clinical evidence characteristics relating to the main clinical trial and indirect treatment comparison (ITC), if applicable, was informed by EUnetHTA guidance. Trends in clinical evidence characteristics were assessed over time. Potential associations between the quality of clinical evidence and reimbursement recommendations were investigated using a hierarchy of chi-squared tests and multivariate regression analysis.

This analysis included 101 completed HTAs, 84 of which were supported by traditional randomized controlled trials (RCTs). The proportion of HTAs supported by single-arm trials (SATs) increased to 55.6 percent in 2023. HTAs supported by RCTs were more likely to receive a “conditional-positive/positive” recommendation, compared with those supported by SATs (42.8% versus 17.6%). Most of the RCTs were open-label studies (n=49), however recommendations were similar among HTAs supported by blinded and open-label studies. The overall survival data maturity was variable. ITCs were required in 76 HTAs, mostly supported by anchored ITCs. SATs supported by unanchored ITCs were more likely to receive a “conditional-positive/positive” recommendation than naïve comparisons.

Reliance on SATs and open-label studies has increased over the past decade. This analysis proposes that HTAs supported by RCTs remain the gold standard for HTA. A key limitation of this study is that the quality of evidence submitted is only one domain of the HTA evaluation process. No tests were statistically significant, which was partly explained by the small sample sizes.

Existing guidelines on overviews of reviews and umbrella reviews recommend an assessment of the certainty of evidence but provide limited guidance on how to apply GRADE to such a complex evidence synthesis. We present one approach to applying GRADE to an overview of reviews developed using general principles derived from current GRADE guidelines.

The methods were developed in an iterative and exploratory fashion following discussion with 11 methodologists and health services researchers. Key principles were distilled on the five GRADE domains (risk of bias, inconsistency, imprecision, indirectness, and publication bias) from the relevant GRADE guidelines, particularly those on test accuracy.

A ‘general principles’ approach of applying the five domains of GRADE to an overview of reviews and arriving at an overall summary judgment for outcomes was developed. These methods were successfully applied to an overview of reviews on 18F-prostate specific membrane antigen positron emission tomography and computed tomography in the staging of patients with high-risk or recurrent prostate cancer.

Our approach distilled key principles from relevant GRADE guidelines and allowed us to apply GRADE to a complex body of evidence. Such an approach may be of interest to other researchers working on overviews of reviews or umbrella reviews.

Health economic analyses compare the necessary investments and health outcomes for two or more technologies, assisting in resource allocation. How these analyses are conducted directly affects the results obtained. Therefore, it is essential to consider their quality during decision-making. The aim of this study was to develop a domain-based tool for the critical assessment of cost-effectiveness and cost-utility studies.

We conducted a scoping review to identify tools available for the critical assessment of health economic analyses and extracted their recommendations. Based on the tools’ items and the discussions of a working group, we identified domains related to the methodological quality of health economic analyses for inclusion in the new tool. The items extracted during the scoping review were classified according to the previously defined domains and were used to identify complementary aspects that should be included in the new tool.

We identified 21 tools, all of which were checklists containing seven to 80 items. The following four quality domains were established for the new tool: (i) applicability of the research question; (ii) model structure; (iii) model parameters; and (iv) precision of the results. Assessment of each domain was guided by signaling questions. The first domain assessed the applicability of the research question to the desired setting; the second evaluated whether the model adequately represents the complexity of the clinical condition; the third assessed the quality (certainty) of the key parameters used in the model; and the fourth evaluated the certainty of the incremental cost-effectiveness or cost-utility ratio.

The tool was developed to integrate critical aspects that affect the methodological quality of health economic analyses, which are often missing in other tools. The quality of reporting was not included as a domain because it is already covered by existing tools. A multidisciplinary panel with different key stakeholders is being organized to review and refine the first version of the tool.

Assurance that supporting evidence is based on valid and unbiased assessments, evaluated using rigorously developed risk of bias (validity assessment) tools, is fundamental to good decision-making. Among those available, selecting and correctly using the best tool that is fit-for-purpose is challenging. Collaboration across the global evidence synthesis and health technology assessment (HTA) communities promotes best practices and harmonizes tool use across jurisdictions.

We have established the LATITUDES Network (https://www.latitudes-network.org/), a publicly available website library of validity assessment tools and resources to guide decision-makers in selecting and applying tools appropriate for particular contexts, including informing HTA reimbursement decisions, clinical guideline development, and stand-alone evidence synthesis projects. The internationally representative leadership team comprises five evidence synthesis experts who have been supported by a competitively awarded academic innovations grant. The 23-member advisory panel representing five continents provided expertise to finalizing criteria for tool inclusion, identifying key tools, and suggesting inclusion of additional tools. Formal launch took place at the 2023 Cochrane Colloquium.

Officially launched in September 2023, the LATITUDES Network indexes validity assessment tools developed for healthcare studies in an online library. To date, 10 key tools are featured to help reviewers identify the optimal tool for their use. Nineteen additional tools have met all screening criteria and are also recommended. Information characterizing each tool (e.g., citation and training materials) is provided. Seven tools are currently under development. A mechanism for users to suggest new tools is provided. Additional tools and information on toolkits and online training materials, as well as links to courses and events, will be added over time.

LATITUDES aims to be the primary resource that provides key information to reviewers conducting validity assessments for evidence synthesis, clinical guideline development, and HTA decision-making. It is intended to increase the robustness of evidence synthesis by improving the process of validity assessment, helping scientists use tools more effectively and efficiently, promoting best practices, and harmonizing validity assessment across the globe.

When indirectly compared trials are too heterogeneous to provide a reliable estimate, matching-adjusted indirect comparison (MAIC) studies can be employed. This technique is commonly used for oncology treatments. MAIC is an indirect comparison that adjusts effect-modifying variables through propensity score methods. The objective of this study was to map the characteristics of MAIC studies in oncology.

We performed a scoping review of the characteristics of MAIC studies that applied MAIC to compare active treatments in oncology. The literature search was last updated in August 2023 in PubMed, Embase, and the Cochrane Library. We extracted sources of funding, outcomes reported, and whether the results were significantly in favor of the trial for which individual patient data (IPD) were available or for the aggregate data. We then calculated the relative risk (RR) and confidence interval (CI) of an outcome favoring the IPD trial technology that was also funded by industry.

A total of 90 studies were included in the review. The pharmaceutical industry was the most frequent funder (n=78; 87%); the source of the IPD data was not reported in 68 studies (76%). In total, 391 efficacy outcome estimates were reported in base case analyses. The risk of favoring IPD while being funded by industry was 93 percent, while the risk of favoring IPD while having other sources of funding was 61 percent (RR 1.520, 95% CI: 1.146, 2.016; p=0.004). Specifically, the RR was 1.246 (95% CI: 0.891, 1.743) for overall survival and 1.426 (95% CI: 0.959, 2.120) for progression-free survival.

MAIC results are influenced by the choice and number of effect-modifying variables used for matching the population. National Institute for Health and Care Excellence guidelines consider it necessary to provide evidence that the matched estimate will be less biased than the unmatched one. We have concluded that industry funded MAIC studies may be more likely to report results favoring IPD than studies with another funding source.

The scarcity of high quality evidence is a common constraint on the willingness to publicly fund innovative technologies. Our aim was to prepare a Methods and Process Guide to support the development of pilot healthcare programs in Poland. Such guides play a pivotal role in enhancing the quality of pilot programs and confidence in public funding decisions in health care.

We reviewed guidelines for pilot healthcare programs published by the World Health Organization (WHO) and other healthcare organizations and analyzed the pilot healthcare programs in Poland. The Ministry of Health in Poland and the general public will be invited to provide feedback on the Guide.

Pilot programs serve as valuable testing grounds for healthcare solutions in low risk, small-scale clinical practice settings. A pilot program may be considered for interventions with proven safety and effectiveness, and when the intervention is complex, its implementation requires testing, or the intervention is considered high cost. Our Methods and Process Guide defines key elements of pilot healthcare programs, including objectives, starting criteria, conducting conditions, and monitoring rules. Public consultation on the Guide is underway.

The publicly available Methods and Process Guide should enhance the methodological rigor of pilot healthcare programs in Poland. Well-designed pilot programs are expected to provide high quality real-world data that will facilitate public funding decisions for innovative technologies.

Diagnostic test accuracy (DTA) systematic reviews (SR) provide acknowledged challenges for health technology assessment (HTA) due to insufficiency of trials and a paucity of methodological frameworks (compared with interventional SRs). Additionally, research in neglected tropical diseases (NTDs) is scarce. Assessing the methodological compliance of a SR protocol in this context is of utmost importance for developing robust HTA in NTD.

A search strategy was conducted in PROSPERO in November 2023 to identify protocols of SRs on diagnostic test accuracy using the following terms: “diagnostic accuracy test”, “diagnostic test”, and “diagnostic accuracy”. Deduplication was performed using Excel’s “remove duplicates” functionality. Eligible studies were SRs with predicted meta-analyses on DTA in any NTD (as per the World Health Organization list). in vitro studies, those with non-human populations, and methodological studies were ineligible. The variables of interest included registry characteristics (author, year, country), protocol status, and methodological characteristics (pre-defined statistical analyses, sensitivity analyses, and heterogeneity analyses). The results were presented using descriptive statistics and narrative analysis of methodological issues.

From 7,931 registries, 106 protocols were selected that anticipated conducting a meta-analysis of DTA in the context of NTDs. The number of registry entries has grown steadily from one protocol in 2012 to 17 in 2023. Twenty-three NTDs were identified, the three most common being dengue (n=23; 22%), leishmaniasis (n=18; 17%), and syphilis (n=12; 11%). Only 14 protocols were reported as published. Regarding quality of reporting and methods, half (n=54) of the reports explicitly stated a meta-analytical approach in their title. Only 16 mentioned a sensitivity analysis and 32 did not mention an analysis of heterogeneity.

The meager number of protocols reported as published could mean a lack of updates or publications, or both. The absence of essential methodological descriptions in protocol titles and body text is worrisome and may ultimately be reflected in the number of publications. Further analysis should assess the specifics of methodological quality based on well-established methodological frameworks for quality of reporting and methods.