Transcatheter aortic valve implantation (TAVI) is an established alternative to surgical aortic valve replacement (SAVR) for patients with severe aortic stenosis. While procedural advancements have reduced the risk of stroke, stroke remains a serious complication of TAVI. To date, no study has investigated post-TAVI stroke costs in Korea. This study compared medical costs between patients with and without stroke after TAVI.

This was a retrospective study using claims data from the Korean Health Insurance Review and Assessment Service. Patients who underwent TAVI in certified hospitals between June 2015 and December 2020 were included; patients with SAVR prior to TAVI were excluded. Patients with postoperative stroke within 30 days of TAVI formed the “Stroke” group; remaining patients formed the “Non-Stroke” group. A generalized linear model with adjustment was used to compare mean medical costs in the first year after TAVI between the two groups. Exchange rate from xe.com (5 December 2022) was applied.

In total, 3,046 TAVI patients were included for analysis (47% male, 85% aged ≥ 75 years). There were 61 (2%) patients in the “Stroke” group and 2,985 (98%) in the “Non-Stroke” group. Compared to the “Non-Stroke” group, the “Stroke” group had significantly higher adjusted mean total first-year medical costs (KRW 25,453,725 (95% confidence interval (CI):15,215,439-42,581,231) (USD 19,640 (95% CI:11,740-32,856)) vs. KRW 19,169,447 (95% CI:11,818,973-31,091,340) (USD 14,791 (95% CI:9,120-23,990)), p < 0.01). Of these costs, 90 percent (“Stroke”) and 84 percent (“Non-Stroke”) were hospitalization-related (“Stroke” vs. “Non-Stroke”: KRW 6,847,975 (USD 5,284); p < 0.01); the remainder were outpatient costs. Predictors of total medical costs were gender; hospital type; prior chronic obstructive pulmonary disease; prior diabetes; prior stroke; and postoperative stroke.

In Korea, TAVI patients with stroke had higher first-year medical costs compared to those without stroke, driven by hospitalization costs. Stroke poses an immediate, heavy economic burden on healthcare systems. Longer-term (e.g., caregiver, rehabilitation) costs were not captured in this analysis; future studies are needed to provide supplementary evidence on the total economic burden of stroke.

Type 2 diabetes mellitus (T2DM) is a major public health problem. Evidence suggests an association between diabetes and cancer, but this may be distorted by confounding. This research aimed to identify and assess the evidence suggesting a causal association between T2DM and cancer.

A systematic review was conducted in Pubmed, Embase, CINAHL, Web of Science, and the Cochrane Library for literature on the association between T2DM and cancer, from inception to 7 May 2021. Case-control and cohort studies published in any language were considered. Based on a targeted literature review, a directed acyclic graph for each type of cancer was developed to test whether the causal effects were adequately controlled for potential confounding.

A total of 131 studies with a low risk of bias were selected that reported 415 effect estimates of the association of T2DM with 57 types of cancer. Breast, colorectal, pancreas, prostate, and lung were the cancer sites with the highest number of studies. Causality was claimed in 57 studies, but only 34 percent of the outcomes were adequately controlled for confounders. Of the studies assessing a causal relationship for prostate and pancreatic cancer, 87 and 70 percent adequately controlled for confounding. In contrast, only 29 percent of lung cancer, 27 percent of colorectal cancer, and 17 percent of breast cancer results considered the minimal sufficient adjustment set. Lifestyle variables were identified as key potential confounders in more than 20 types of cancer, but they were not included in the analyses.

Many studies simply reported an association between diabetes and cancer. The policy implications of such studies are unclear. Of the studies claiming a causal link between diabetes and cancer, a large proportion did not adequately control for confounding. It is critical that studies take a systematic approach to identifying potential confounding factors, such as targeted reviews and the development of directed acyclic graph approaches, to estimate causal effects that may be useful in informing health policy.

The latest clinical practice guidelines for non-small cell lung cancer (NSCLC) published by the National Comprehensive Cancer Network in 2022 recommend that patients with NSCLC (>1 and ≤2 cm) should be diagnosed as T1b. Segmentectomy and lobectomy are equally effective in treating patients with NSCLC no bigger than 2 cm, and especially for tumors no bigger than 1 cm. However, the effectiveness of these treatments for NSCLC tumors between 1 and 2 cm is unknown. We conducted a systemic review and meta- analysis to assess the efficacy of these two surgical treatments in patients with T1b stage NSCLC.

We searched for randomized controlled trials (RCTs) and cohort studies investigating the efficacy of lobectomy and segmentectomy for patients with T1b stage NSCLC. Study quality was assessed with the Cochrane Quality in Prognosis Studies tool. We used random effects models to analyze overall survival (OS) and lung cancer-specific survival (LCSS), expressed as hazard ratios (HR) and 95% confidence intervals (CIs). The effect of covariates was assessed using subgroup analysis. All procedures were performed according to the PRISMA guidelines.

We identified ten cohort studies that matched our selection criteria, with general low risk of quality, including a total of 37,691 patients. No publication bias was found. Compared to lobectomy, segmentectomy had lower OS (HR 1.31, 95% CI: 1.16, 1.47; p=0.026) and LCSS (HR 1.21, 95% CI: 1.03, 1.42, p=0.015) before Cox regression. After multivariable Cox regression, adjusted by age, sex, histological type, and lymph node section, segmentectomy had similar OS (HR 1.17, 95% CI: 1.00, 1.37; p=0.2) and LCSS (HR 1.10, 95% CI: 0.89, 1.36; p=0.8).

Segmentectomy can be used to treat patients with T1b stage NSCLC. Patients who undergo segmentectomy have survival outcomes that are the same as those of patients who received lobectomy. This evidence-based observation provides a reference for surgical choice in the treatment of patients with T1b stage NSCLC, which should be further confirmed through RCTs.

Patient and public involvement (PPI) is a core element of the health technology assessment (HTA) process. Since its creation in 2011, the National Committee for Health Technology Incorporation (Conitec) has promoted initiatives to include stakeholders in HTA for the Brazilian Public Health System (SUS). This work aimed to present a report on the advances and challenges related to PPI in 11 years of Conitec.

A retrospective analysis of PPI actions carried out at Conitec was conducted, based on an analysis of minutes and records of meetings and discussions held internally and documents published on Conitec´s website.

Events and meetings were held over the years with different actors interested in the HTA process. Since 2015, a plain-language version of the technical report has been made available to the public during public consultations for each HTA topic. Recently, a register of patients, specialists, and SUS managers was created to form a database and establish a network with the stakeholders. Since 2020, SUS users have been allocated time to speak at Conitec´s meetings. Qualitative analysis of public consultation documents started in 2021 and a pilot qualitative evidence synthesis was carried out in 2022. These initiatives, although not directly focused on PPI, increase the consideration of the perspectives of patients, family members, and caregivers in the HTA process.

PPI actions implemented at Conitec have significantly promoted inclusiveness and exchanges among stakeholders, contributing to a greater transparency regarding Conitec’s actions. Nonetheless, we have important challenges on our horizon, such as strengthening connections with primary healthcare managers and professionals and social movements. It is also strategic to expand the technical and scientific discussion on PPI and qualitative approaches with HTA researchers and the voting members of Conitec. Finally, another aim is to improve knowledge of HTA and public health policy among law professionals and the pharmaceutical industry in Brazil.

The number of studies on digital health technologies (DHTs) for remote treatment and patient self-management is increasing. Existing health technology assessment (HTA) frameworks for DHTs, which guide researchers in generating evidence suitable for HTA, do not cover all domains of the commonly used EUnetHTA Core Model, and DHT-specific considerations have not been informed by a large stakeholder preference study. Our aim was to develop a stakeholder prioritized, literature-informed checklist of DHT-specific considerations that aligns with the EUnetHTA model.

We conducted two systematic reviews to identify: (i) DHT evaluation frameworks published to March 2020 for content; and (ii) primary research on DHTs published from 1 January 2015 to 20 March 2020.

Stakeholder prioritization of issues was performed using a best-worst scaling preference study among a broad cross-section of patients, carers, health professionals, and the general population in Australia, Canada, New Zealand, and the UK. Systematic review issues were prioritized and adapted for use as a practical checklist.

DHT evaluation content was recommended by the 44 identified frameworks for 28 of the 145 issues in the EUnetHTA model and for 22 new DHT-specific issues. A coverage assessment of 112 clinical studies of remote treatment and self-management DHTs for patients with cardiovascular disease or diabetes revealed that less than half covered DHT-specific content in all but one domain, or traditional HTA content in clinical effectiveness and ethical analysis. The preference survey of 1,251 stakeholders identified broad agreement on the 12 most important DHT attributes, six of which were related to safety. The most important attribute was “helps health professionals respond quickly when changes in patient care are needed”, which is not a focus of existing DHT HTA frameworks.

The review identified mismatches in the content generated by DHT clinical studies and that required for DHT-specific HTAs. These findings informed the development of an extended checklist comprising 22 stakeholder-prioritized DHT-specific considerations, which are aligned with the EUnetHTA model and will help ensure the planning of DHT-specific research generates evidence suitable for HTA.

The French National Authority for Health’s (HAS) 2019-2024 strategic workplan called for “making public involvement a priority.” This project was designed within the roadmap validated in 2021; “Strengthening public involvement in health technology assessment (HTA) at the HAS”, including an action of building a means of training patient organizations for their contribution. The project’s overall objective was to develop with patients and consumers a first training tool, representing an online knowledge base targeting French patients and consumers to explain the HAS HTA process and assist their participation in these assessments.

Three stages were designed to meet this objective. Firstly, an international benchmark was performed of available online HTA training tools for patients and consumers, notably those used by HTA bodies and European and international patient and consumer groups (PCGs). Secondly, an internal HAS search for e-learning tools, was conducted to identify whether they could meet the training needs of French patients and consumers. Finally, the training tool was developed via a working group composed of patients and HAS scientific officers.

The benchmark identified 82 online training tools selected according to the specified inclusion criteria. Sixteen international HTA agencies, nine European and international PCGs and 13 other bodies provided online HTA training tools for patients and consumers during the research period, but no journal articles identified such tools. Eleven formats and 12 key themes, divided into two main categories were identified: important content related to HTA, and important content related to public involvement in HTA. The HAS search for e-learning tools resulted in internal e-learning tools offered for clinical experts not meeting the needs and preferences of patients and consumers. Finally, HAS based its training tool development on these preferences and needs to create a PowerPoint in two blocks of modules covering the two main categories above (six modules in total).

French patients and consumers preferences and needs for a HTA training tool were inspired by the international benchmark, dividing key themes into two main categories: important content related to HTA, and important content related to public involvement in HTA. This resulted in HAS development of two blocks of modules in PowerPoint format covering these two categories.

The objective was to conduct a benchmarking study of available online health technology assessment (HTA) training tools for patients, specifically those used by HTA agencies and major European and international patient and consumer groups (PCGs). We compared existing online training tools on this topic in order to develop in-house HTA training tools for French patients and consumers.

A literature search and a scoping review of websites was conducted by including the websites of HTA agencies, European and international PCGs, and other bodies. This was supplemented with videoconference interviews with selected HTA agencies and patient groups. The inclusion criterion was the existence of content describing HTA and patient and public involvement (PPI) in HTA that PCGs could use (regardless of its format).

Eighty-two online training tools were selected according to the specified inclusion criterion. Sixteen international HTA bodies, nine European and international PCGs, and 13 other bodies provided online HTA training tools available for patients and consumers. No journal articles matching the inclusion criterion were found. Two broad categories of content were identified: the first relating to HTA and the second relating to PPI in HTA. Moreover, the formats of these tools ranged from interactive to non-interactive, with varying accessibility (freely available or with a paywall) and assessment methods.

These results should be considered together with budget requirements, project time constraints, human resources, and the preferences of HAS and patients when developing HTA training tools to improve the participation of patients and consumers in the HTA process at HAS.

There is currently no standardized way to share information about health technology assessment (HTA). Standardised Data on Initiatives (STARDIT) addresses current limitations and inconsistencies in sharing data about HTA processes by providing a way to report these data, including which stakeholders have been involved, their tasks, what methods and data sources were used, and any impacts or outcomes observed.

STARDIT development began in 2019 and was guided by participatory action research paradigms. A multidisciplinary international team of over 100 citizens, experts, and data users was involved in co-creating STARDIT. These co-creators include cancer patients, people affected by rare diseases, Indigenous peoples from multiple countries, representatives involved in HTA processes, health researchers, environmental researchers, economists, librarians, and academic publishers. Methods of involving people included public events, online discussions, and a public consultation process. STARDIT is free to use, and data can be submitted by anyone. Report authors can be verified to improve trust and transparency, and data can be checked for quality.

STARDIT can help create high-quality standardized information about HTA processes that can be accessed and edited by anyone. STARDIT enables data reporting at all stages of the HTA process and works in multiple languages. This allows stakeholders involved in or affected by HTA processes (including patients, the public, Indigenous peoples, and people from industry) to appraise and edit information and to self-identify the labels and terminology used to describe them. Organizations such as the Cochrane Collaboration, Australian Genomics, and multiple universities have created STARDIT reports. A link to the working beta version can be found at scienceforall.world/STARDIT.

STARDIT offers those conducting HTA access to standardized information that enables well-founded comparisons of the effectiveness of different HTA methods, including the most effective methods of involving stakeholders. STARDIT allows anyone to access data about HTA processes, which can support participatory ways of working and help improve the equity and quality of HTA processes worldwide.

Treatment with atezolizumab plus standard chemotherapy can prolong the overall survival of patients with metastatic non-squamous non-small cell lung cancer (NSCLC). However, the economic value of this treatment regimen is unknown. This study aimed to estimate the cost effectiveness of atezolizumab plus chemotherapy in the first-line treatment of metastatic non-squamous NSCLC from a healthcare system perspective in China.

A partitioned survival model consisting of three discrete health states was developed to estimate the cost and effectiveness of atezolizumab plus carboplatin or cisplatin plus pemetrexed (APP) versus carboplatin or cisplatin plus pemetrexed (PP) in the first-line treatment of metastatic non-squamous NSCLC over a 12-year lifetime horizon. Key clinical data were generated from the IMpower132 trial. Local direct medical and non-medical costs were used and health preference data were collected from patients with NSCLC in 13 tertiary hospitals across five provinces in China. Costs, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios (ICERs) were measured. One-way and probabilistic sensitivity analyses were performed to assess the robustness of the model.

Compared with the PP regimen, APP therapy yielded a gain of 0.21 QALYs at an increased cost of CNY145,602 (USD22,574), resulting in an ICER of CNY684,894 (USD106,185) per QALY gained. The ICER was significantly higher than three times the gross domestic product per capita for China in 2021 (USD37,663). One-way sensitivity analyses revealed that one of the most influential factors in this model was the cost of atezolizumab. Probabilistic sensitivity analysis showed that there was 14.7% probability that atezolizumab plus chemotherapy was cost effective at a willingness-to-pay value of CNY242,928 (USD37,663) per QALY gained.

The APP regimen could prolong survival and improve health benefits over standard chemotherapy in the first-line treatment of patients with metastatic non-squamous NSCLC, but it is unlikely to be a cost-effective treatment option in China.

Sintilimab is an IgG4 anti-programmed cell death protein 1 (PD-1) antibody that has a high-affinity blocking interaction with PD-1 and its ligands. The updated ORIENT-11 study showed that sintilimab plus chemotherapy significantly prolonged progression-free and overall survival, compared with chemotherapy alone, in the first-line treatment of non-squamous non-small cell lung cancer (NSCLC). In China, it is uncertain whether sintilimab is a cost-effective alternative to standard immunotherapy.

A partitioned survival model with three health states (including progression-free survival, disease progression, and death) was constructed from the Chinese societal perspective using a three-week cycle with a lifetime horizon (16 years). Individual patient data were captured from the updated ORIENT-11 study, and high-risk and clinically severe adverse events were specifically added to the states. Quality-adjusted life-years (QALYs) and incremental cost-effectiveness ratios (ICERs) were the primary outcomes. Costs, health productivity losses, and utilities were derived from questionnaires and supplemented by expert opinion and literature review. All costs were expressed in 2021 USD, and costs and QALYs were discounted at an annual rate of five percent. Sensitivity analyses and scenario analyses considering the healthcare system perspective were performed to explore model uncertainty.

Patients receiving sintilimab plus chemotherapy incurred a mean total cost of USD67,727 and gained 2.5 QALYs during the lifetime period, compared with USD40,530 and 1.5 QALYs for patients receiving standard chemotherapy. The corresponding ICER was USD27,665 per QALY in China. At a willingness-to-pay threshold of three times the gross domestic product per capita in China (USD37,663), sintilimab plus chemotherapy was the optimal treatment in 84 percent of replications. Deterministic sensitivity analysis showed that the most significant driving determinant was the discount rate of costs and QALYs. An ICER of USD21,020 per QALY was obtained from the Chinese healthcare system, validating the robustness of the cost-effectiveness analysis.

Compared with standard chemotherapy, sintilimab plus chemotherapy is a cost-effective treatment regimen for non-squamous NSCLC in China. Thus, sintilimab may benefit Chinese patients and should be promoted by decision makers.

In the RESORCE trial, regorafenib was shown to provide overall survival (OS) benefit for patients with hepatocellular carcinoma (HCC) that has progressed on sorafenib treatment. Subsequently, it was approved by the Therapeutic Goods Administration for the treatment of patients with HCC who were previously treated with sorafenib; however, regorafenib is still not recommended by the Pharmaceutical Benefits Advisory Committee in Australia. We aimed to assess the cost effectiveness of regorafenib as a second-line therapy for patients with HCC who progressed on sorafenib from an Australian healthcare perspective.

We developed a Markov model to compare the cost effectiveness of regorafenib with best supportive care (BSC) as a second-line therapy for HCC after treatment with sorafenib. The health outcomes of life-years and quality-adjusted life-years (QALYs) were derived from the RESORCE trial. Survival benefits sourced from the RESORCE trial were fitted with the parametric model to estimate survival beyond the follow-up period. Drug costs and costs associated with adverse events (AEs) were sourced from published literature and the Independent Health and Aged Care Pricing Authority cost report. Model validity was verified using probabilistic sensitivity analyses.

The incremental monthly cost of treatment with regorafenib was AUD19,273 (USD13,374), with an incremental life-year gain of 0.38, compared with BSC. The incremental QALYs gained with regorafenib were 0.24, resulting in a base-case incremental cost-effectiveness ratio (ICER) of AUD80,511 (USD55,872) per QALY. In the probabilistic sensitivity analyses across scenarios, the ICER remained above the conventional threshold of AUD50,000 (USD34,698) per QALY, with a zero probability of being cost effective at this willingness-to-pay threshold.

At the current price, second-line treatment with regorafenib in patients with HCC that has progressed on sorafenib was not cost effective at the conventional willingness-to-pay threshold from an Australian health-system perspective.

Since 2017, health technology assessment (HTA) has been included in the Ukrainian Health Law fundamentals and its implementation has accelerated since it became mandatory in 2020. SAFEMed has been supporting the Ministry of Health in integrating HTA into the decision-making ecosystem and building capacity in HTA. In this 2022 to 2023 project, we aimed to create and conduct HTA training for doers, users, and trainers based on a developed model curriculum for an HTA master’s program, and to identify sets of criteria for successful training and training centers.

First, we reviewed websites and documents of current academic HTA master’s and advanced programs worldwide. Second, we performed an assessment of the training needs of HTA doers, users, and trainers in Ukraine using an online survey that captured level of experience and knowledge gaps. Third, we reviewed the capacity and quality requirements of existing academic centers that provide HTA training.

We identified seven HTA master’s programs globally, which covered five HTA domains: (i) health problem and current use of the technology; (ii) description and technical characteristics; (iii) safety; (iv) clinical effectiveness; and (v) costs and economic evaluations. Other aspects of HTA, such as ethical, legal, social, and cultural aspects were also covered, but not in all programs. The needs assessment was completed by 40 doers (53%), users (43%), and potential trainers (5%) of HTA in Ukraine. Specific knowledge gaps included: comparative effectiveness, health economics, qualitative evidence synthesis, patient and public involvement, and ethical issues. The proposed program addresses these gaps and includes an introduction to HTA that is in line with the new HTA definition. We also generated a minimum set of quality assurance criteria to ensure successful training and to develop efficient training centers for delivering HTA programs.

Our study provides a strong foundation for planning and conducting sustainable HTA training for current and future doers, users, and trainers in Ukraine, which can be an example for other countries wishing to increase HTA capacity.

Several overarching health policy reform processes are currently underway in South Africa (SA), providing an opportunity to establish health technology assessment (HTA) and value-based assessment (VBA) frameworks that foster patient and citizen involvement (PCI). A mapping of the capacity, knowledge, and skill of SA PCI advocacy actors and understanding of the ‘middle-ground’ and influencing relationships that influence advocacy strategies for PCI in HTA, will allow us to determine the needs of PCI actors to entrench PCI principles in the emerging institutionalization of HTA in SA.

An analysis of national and international legislative and policy frameworks indicates current gaps and opportunities for PCI institutionalization in HTA in SA. A survey was conducted to determine SA patient and citizen advocacy actors’ capacity, knowledge, and skill across multiple disease areas. An analysis of decision maker’s opinions and positions about PCI in HTA and VBA policy, and their potential influence on the PCI process was undertaken.

The legislation and policy review indicate that engagement initiatives are positioned at the ‘involvement’ or ‘consultation’ stages of the engagement continuum, rather than higher-level engagement. Five percent of patient advocacy groups (PAGs) interviewed have formalized PCI HTA advocacy strategies. Few PAGs indicated employing processes to actively monitor the HTA and PCI-related activities of decision-makers.

The majority of PAGs stated that collaborative efforts within larger networks would generate more success, if they engaged in PCI in HTA advocacy. Over eighty percent of civil society stakeholders face capacity constraints, such as lack of knowledge of the legislative framework and theory of HTA, funding and manpower to engage in PCI. The majority of HTA processes undertaken by funders in SA do not actively include PAGs or formalized PCI.

Existing legislative and policy frameworks do not include PCI capacity-building strategies. This is impacted by the lack of coordination amongst patient and consumer groups, the willingness of existing HTA structures to formalize PCI, and the resources of the country’s PCI advocate actors to influence existing HTA processes.

The methodology for explicitly incorporating patient preferences by expert committees engaged in deliberative health technology assessment (HTA) processes for drug reimbursement recommendations is a relatively unexplored area despite the growing emphasis on patient-reported outcomes and patient engagement. The Patient Values Project (PVP) aims to improve patient input to expert review committees and promote a better understanding of the patient perspective using quantitative data to support the rationale in assessing new cancer drugs. Using colorectal cancer as a starting point, the PVP aims to develop a framework to objectively incorporate quantitative patient values and preferences into Canada’s cancer drug HTA decision-making process. We report on results from the first phase.

In the first phase, we developed a bilingual survey informed by qualitative focus groups, literature review and feedback from clinicians, patients and experts. The survey includes background questions, general and cancer specific quality-of-life tools, two discrete choice experiments (DCE) and a best worst scaling (BWS) experiment. After pre-testing and pilot testing, the survey was administered across Canada to metastatic and non-metastatic colorectal cancer patients and caregivers, in addition to adults from the general population. In the next phases, we will use vignettes to explore how patient preferences could be incorporated explicitly into decision-making, and what approach to use in HTA submissions.

DCE1 survey results (˜n=1,000) reflect trade-offs between health-related quality-of-life and survival; DCE2 results reflect trade-offs between treatment regimens, side effects and survival/risk of recurrence; BWS results ranked and weighted the tolerability of 25 possible side effects of treatment. We observed differences in preferences amongst the general population, patients with metastatic cancer, non-metastatic cancer and caregivers.

Patients have unique perspectives and preferences about what is important and of value to them, which may impact patient adherence to treatment. In the next phases, we will explore how this evidence from patient preferences can be translated into values that could potentially be incorporated as an explicit element of the deliberative process for HTA decision-making.

Patient-reported outcome measures are being increasingly considered both in clinical practice and in the field of health technology assessment. Although emotional distress is currently recognized as the sixth vital sign in cancer care, its early detection and screening is not yet included in routine clinical practice in Spain. The main objective of this study was to assess the psychometric properties and diagnostic accuracy of validated tools for the early detection of distress among adults with cancer in the Spanish context, at the request of the Spanish National Health System (NHS) Cancer Strategy.

A systematic review was carried out to analyze development and validation studies. The Quality Assessment of Diagnostic Accuracy Studies tool (QUADAS-2) was used for the risk of bias assessment, and a multicriteria global assessment was used for the tests. Ethical and organizational aspects were also addressed.

Fifteen validation studies were included, corresponding to seven tests. The tools considered were the Distress Thermometer (DT), the Brief Symptom Inventory-18 (BSI-18), the Edmonton Symptom Assessment System-revised (ESAS-r), the Hospital Anxiety and Depression Scale (HADS), the Visual Analog Scale for Anxiety and Depression (VAS-AD), the Detection of Emotional Distress (DED) scale, and the Psychosocial and Spiritual Needs Evaluation (ENP-E) scale. Evidence of validity, reliability (internal consistency), and diagnostic accuracy (sensitivity, specificity, and area under the receiver operating characteristic curve) were summarized. Three scales were rated as poor (VAS-AD, BSI-18, and ESAS-r), the ENP-E scale was rated as acceptable, and three scales were rated as moderate (DT, DED, and HADS).

The DT (single-item measure) stands out as an appropriate tool for early detection of emotional distress in the Spanish NHS. The use of this scale could be considered a first stage, to be combined later with a longer scale to improve screening specificity. The HADS scale could be utilized for this purpose. The use of these tools should be framed within a structured screening program that ensures further evaluation and subsequent psychological care when needed.

Pediatric cancers are rare tumors, heterogeneous in location and biologically very different from adult cancers. Documented survival variation across European countries and Italian regions shows that there is still room for further improvement by reducing inequalities. We aim to understand why there are differences in survival. The BENCHISTA-ITA project (National Benchmarking of Childhood Cancer Survival by Stage at diagnosis), that is the Italian twin project of the International BENCHISTA, collects stage at diagnosis of solid pediatric tumors, according to the Toronto Guidelines. We will compare how far the cancer has spread at diagnosis and test if differences in tumor stage explain any survival differences between Italian regions.

The project study involved the stage distribution and the survival of 9 pediatric solid tumors diagnosed between 2013 and 2017 in Italy. All patients therefore had at least 3 years of follow-up in 2021 for life-stage definition. The study involves the identification of all new diagnoses of cancer, evaluation of the clinical documentation of cases eligible for research, and international classification and coding. Analyses of stage distribution and survival rates for each tumor type will be described.

Data from 35 population-based cancer registries from 18 out of 20 Italian regions were collected covering about 84 percent of the Italian child population. In particular, data on: imaging/examination performed before any treatment; source used for staging; primary treatment defined as given within one year from diagnosis; relapse/ recurrence/ progression; follow up and status of life. The study tested the applicability of the Toronto Guidelines as a tool to obtain population-level comparable stage information for childhood cancers. There were 1,343 cases collected (242 Neuroblastoma, 124 Wilms Tumour, 145 Medulloblastoma, 148 Osteosarcoma, 135 Ewing sarcoma, 115 Rhabdomyososarcoma, 54 Ependymoma, 47 Retinoblastoma, 333 Astrocytoma). Toronto stage could be assigned in more than 90 percent in the majority of tumors. Tumors in which it was more difficult to assign the stage using the Toronto staging guidelines were ependymoma, astrocytoma, and retinoblastoma. It was easier to retrieve data for patients in the 0-14 years of age range than adolescents (14-18 years). Differences in stage distribution and survival differences between regional grouping were presented.

The Italian BENCHISTA project, improving the connection between pediatric cancer registries, aims to improve care of children with cancer across the nation, reducing possible disparities.

The wide adoption of the Toronto Guidelines will facilitate international comparative incidence studies, strengthen the interpretation of survival data, and contribute to more appropriate solutions to improve childhood cancer outcomes.

Antidepressants are one of the main treatment approaches for depression, and previous evidence suggests that consideration of patient preferences can improve their adherence to medication regimens. The objective was, therefore, to evaluate the preferences of depressed and depression-prone groups in China with respect to antidepressant medications.

An online survey with best-worst scaling choices was administered in depressed and depression-prone patients. The balanced independent block design generated 13 choice task profiles for each participant to answer, with each choice set consisting of four alternatives out of 13 antidepressant-specific attributes. Count analysis and a conditional logit model were used to estimate the relative importance of the 13 attributes and preference heterogeneity.

The analytical sample included 210 participants, comprising 49 individuals who had previous experience with depression and 161 who were depression prone. Participants in both groups preferred medications with a low risk of liver or kidney damage, headache or dizziness, and recurrence. There were significant differences in both groups regarding out-of-pocket costs and duration of medication. The K-means clustering further proved preference heterogeneity among the patients.

Our study revealed patient preferences for antidepressant medication choices in China. Healthcare decision makers should consider and discuss patient preferences in the treatment decision-making process to improve patient adherence to and satisfaction with medications, and to ultimately improve patient outcomes.

Policy makers are keen to introduce cost containment measures in medicine spending due to aging populations and fiscal pressure. A major price reform was applied to the Australian Pharmaceutical Benefits Scheme (PBS) in 2014. This aimed to stimulate price reductions by increasing competition among generics, amidst growing evidence at the time of unnecessarily high generic medicine prices in Australia. The aim of this study was to estimate the effect of patent expiration and generic competition on drug prices, while controlling for other determinants of drug prices in Australia.

A dataset from publicly available sources was constructed using monthly data on the price of drugs listed on the PBS from October 2014 to July 2022. The information included the generic drug name, item code, date, approved ex-manufacturer price, dispensed price per maximum quantity, and brand names. This was supplemented with monthly government spending and number of prescriptions filled per item code. A fixed effects regression model was used to estimate the effect of patent expiration and generic competition on dispensed drug prices.

The model estimated that introducing generics in Australia led to an 18 percent decrease in prices, excluding further decreases resulting from other controlled variables. The price elasticity of total prescriptions filled was estimated to be -0.6, suggesting that a one percent increase in prescriptions filled resulted in drug prices being lowered by 0.6 percent. This reflects the fact that, on average, firms reacted by reducing prices to increase market share when faced with an increase in quantity demanded. Each extra competitor was estimated to result in a reduction in prices of roughly 1.8 percent.

These results show that entry of generics into the Australian pharmaceutical market resulted in a significant reduction in drug prices. However, this alone does not provide empirical support for the effectiveness of these price reforms in generating savings by inducing generic competition, especially over other forms of pharmaceutical regulation.

Most drugs have data only from clinical trials focused on a specific population at the time of first registration, so their indications for use are restricted to this population. In Brazil, the prices of new drugs for clinical conditions with no therapeutic alternatives are relatively high. When these drugs expand to other indications their prices are not reviewed, which can have a major financial impact. This study aimed to evaluate the financial impact of expanding the indication for trastuzumab deruxtecan.

We calculated the annual cost to treat all Brazilian patients with the indications listed for trastuzumab deruxtecan at first registration, and then all additional indications. Populations were estimated from epidemiological data from National Committee for Health Technology Incorporation reimbursement documents and a clinical trial comparing trastuzumab deruxtecan with trastuzumab emtansine. Costs were calculated using the ANVISA Câmara de Regulação do Mercado de Medicamentos price value and a patient weight of 70 kg.

In January 2022, trastuzumab deruxtecan was introduced in Brazil for patients with human epidermal growth factor receptor (HER2) positive metastatic or unresectable breast cancer who had received two or more anti-HER2 treatment regimens. In June 2022, the indication was expanded to patients with HER2-positive metastatic or unresectable breast cancer who had received one anti-HER2 treatment regimen. In November 2022, the indication was further expanded to patients with metastatic or unresectable low-expression HER2 breast cancer who had received prior systemic therapy. The number of patients estimated to be eligible for the drug increased from 383 to 23,000, with an increased total cost from BRL467,970,786 (USD90,621,763) to BRL26,048,234,160 (USD5,044,197,164).

The expansion of indications for trastuzumab deruxtecan may substantially increase its financial impact and compromise the sustainability of health systems. In Brazil, the lack of monitoring of drug prices means that the only change in prices occurs due to regulated annual inflation adjustments. Regulation is needed to reduce drug prices according to new indications, changes in therapeutic options for the same condition, and obsolescence.