To save content items to your account,
please confirm that you agree to abide by our usage policies.
If this is the first time you use this feature, you will be asked to authorise Cambridge Core to connect with your account.
Find out more about saving content to .
To save content items to your Kindle, first ensure no-reply@cambridge.org
is added to your Approved Personal Document E-mail List under your Personal Document Settings
on the Manage Your Content and Devices page of your Amazon account. Then enter the ‘name’ part
of your Kindle email address below.
Find out more about saving to your Kindle.
Note you can select to save to either the @free.kindle.com or @kindle.com variations.
‘@free.kindle.com’ emails are free but can only be saved to your device when it is connected to wi-fi.
‘@kindle.com’ emails can be delivered even when you are not connected to wi-fi, but note that service fees apply.
Geriatric assessment and management (GAM) is a guideline-recommended strategy for optimizing cancer management among older adults. In a recent cost-utility analysis of the Canadian 5C randomized controlled trial (RCT), GAM appeared cost effective only in selective patients. We assessed the cost-utility of GAM plus usual care (UC) versus UC alone in older adults with cancer using a decision model and best available evidence from four international RCTs: GAIN, GAP70, INTEGERATE, and 5C.
Methods
We conducted a model-based economic evaluation using pooled data from four international RCTs (GAIN, GAP70, INTEGERATE, and 5C), supplemented by additional evidence from the literature. Deterministic and probabilistic analyses were performed from the healthcare payer perspective, applying a six-month time horizon. The base case and main analyses used Canadian cost data. Sensitivity analyses included per-trial scenario analyses, one-year time horizon, and the use of USA costs. We reported healthcare costs per quality-adjusted life year (QALY) and the incremental net monetary benefit (INMB) using a CAD50,000(USD36,277) per QALY threshold.
Results
The base case analysis using Canadian costs indicated that GAM was cost effective with an INMB of CAD1,117 (USD819) (95% credibility interval [CrI]: −CAD2,450 [−USD1,796], CAD5,035 [USD3,692]) and 70.7 percent probability of GAM being cost effective at a cost-effectiveness threshold of CAD50,000 (USD36,666) per QALY. Trial-specific results varied, with the GAP70 and INTEGERATE trials yielding positive INMB values (CAD2,635 [USD1932] and CAD2,886 [USD2,116], respectively), while 5C and GAIN resulted in negative INMB values (−CAD642 [−USD471] and −CAD268 [−USD196], respectively). Sensitivity analyses revealed that chemotherapy costs were the main driver of costs in both GAM and UC strategies.
Conclusions
Evidence showed that GAM is generally cost effective. However, GAM’s cost effectiveness varied across trial scenarios, driven primarily by differences in chemotherapy costs. Future research should focus on identifying key GAM components that most effectively reduce severe toxicity, hospitalizations, and chemotherapy-related costs to optimize overall cost effectiveness.
Although randomized controlled trials (RCTs) are the gold standard for evaluating health interventions, their methodology is challenging for arthroplasties. An international network of arthroplasty registries (AR) provides real-world data to generate evidence. However, the extent to which AR data contribute to scientific evidence remains unclear. This scoping review aimed to analyze the use of AR data in scientific publications.
Methods
All methodological stages of a scoping review were conducted in accordance with the Joanna Briggs Institute manual. The database search utilized official Medical Subject Heading and Health Science Descriptors terms, retrieving 25,846 studies. Study selection was performed in two stages by pairs of reviewers to ensure accuracy and reliability. The report followed the PRISMA Extension for Scoping Reviews. The analysis of the results was carried out using descriptive categories to classify and interpret the data comprehensively. No funding was provided for this study.
Results
After selection, 140 studies were included. Studies on national AR were most prevalent in Australia and the UK, followed by studies on institutional and regional AR in the USA. Retrospective cohort designs dominated, involving large and heterogeneous populations. Total and unicompartmental arthroplasties were the primary subjects, with surgical revision being the most reported outcome. Authors identified limitations related to AR data application and observational study methodologies, impacting evidence reliability.
Conclusions
When properly implemented, AR are crucial data repositories that enable the generation of scientific evidence on arthroplasty technologies, predictive failure models, and post-sale prosthesis monitoring. This demands institutional efforts, collaborations, and researcher training for the proper use of observational studies, alongside paradigm shifts in confidence toward the evidence produced to support decision-making.
There is limited research on the association between soda consumption and the risk of metabolic syndrome (MetS), particularly during the COVID-19 pandemic. This study investigated the relationship between soda consumption and MetS in Korean adults, stratified by sex, and compared differences before and after the pandemic using data from the Korea National Health and Nutrition Examination Survey (2017–2021). A total of 13,051 adults aged 19–64 years were included. Soda consumption was assessed using 24-hour recall and categorized into five groups (nondrinkers and four quartiles). Multivariable logistic regression models were used to estimate odds ratios (OR) and 95% confidence intervals (CI) for MetS and its components. After adjusting for multiple covariates, no significant association was found between soda consumption and MetS overall. However, adults in the highest quartile of soda consumption (≥373g/day) had higher risks of abdominal obesity (P-trend=0.006) and hypertriglyceridemia (P-trend=0.003), compared to nondrinkers. When analyzed by gender, women in the highest quartile of soda consumption (≥315g/day) had significantly increased risks: MetS by 70% (OR=1.70; 95% CI: 1.08–2.68), abdominal obesity by 63% (OR=1.63; 95% CI:1.12–2.38), hypertriglyceridemia by 83%(OR=1.83; 95% CI:1.23–2.74), and low HDL cholesterol by 46%(OR=1.46; 95% CI:1.06–2.01), whereas no significant associations were observed in men. Post-pandemic analysis revealed a significant association between high soda consumption (≥ 416g/day) and MetS (OR=1.56; 95% CI:1.04–2.34), which was not significant in the pre-pandemic period (P-interaction=0.031). These findings suggest that high soda consumption may increase the risk of MetS, particularly among Korean women.
To design healthcare system strategies to inform policy decisions in prevention and early diagnosis of melanoma in children, adolescents, and young adults (CAYA) in Europe, a patient perspective is needed. The MELCAYA project addresses this issue, since this disease lacks preventive and diagnostic strategies. A focus session was conducted to identify challenges and potential improvements in current policies on rare cancers.
Methods
The focus session involved 15 participants, including patients, caregivers, patient advocates, and healthcare professionals from seven European countries. This session explored, through open discussions, challenges in melanoma prevention and early diagnosis as well as the role of patient advocates in policy development and implementation. Furthermore, a group dynamic was carried out to collect patients’ insights on 20 policy strategies from National Cancer Control Plans (NCCP), which were categorized into three areas: (i) early diagnosis and symptom awareness; (ii) health promotion and ultraviolet radiation prevention; and (iii) precision medicine and genetic counseling. Participants provided insights by identifying lacking policies and proposing actions for their implementation.
Results
Key challenges in melanoma prevention and early diagnosis include: (i) low awareness and education among the general population and a misconception about its severity; (ii) fragmented care in the patient journey from diagnosis to treatment; (iii) ineffective communication with younger generations; and (iv) difficulties on how to navigate through the healthcare systems. Participants proposed 18 additional potential strategies to the NCCP and 22 actions for policy integration into healthcare systems. The current ad hoc approach fails to fully leverage contributions from advocates and healthcare professionals, often resulting in policies misaligned with clinical realities and patient needs.
Conclusions
This focus session provided patient ideas for policy initiatives, identified gaps in current policies from the NCPP, and emphasized the need for improved awareness, education, and communication strategies. Patient involvement in policy development, in a more structured way, is crucial for creating effective strategies, and its integration into healthcare systems could enhance melanoma prevention and early diagnosis in CAYA populations.
Spina bifida (myelomeningocele [MMC]) is the most common congenital anomaly with permanent disability. Traditional treatment consists of repairing the defect itself and correcting hydrocephalus (via shunt surgery), when present, within the first 48 hours after birth. Since 2011, fetal surgery to correct MMC has become an impactful treatment alternative, with better short-term results, both neurological and cognitive. However, cost remains a limiting factor for guidelines and health policy changes.
Methods
Fetal surgery (the alternative) was compared with neonatal surgery (the comparator) in a cost-utility analysis. The perspective of the study was from supplementary health, with direct cost data extracted from the public database of the National Supplementary Health Agency (Agência Nacional de Saúde Suplementar). The time horizon considered was 10 years, with a discount rate of five percent. The effectiveness measure was quality-adjusted life years (QALYs). Eligibility, utility, and outcome data were obtained from medical literature. The model used was Markov, with coupled decision tree. Subsequently, probabilistic and deterministic sensitivity analyses were carried out, and the cost-effectiveness acceptability curve (CEAC) was calculated.
Results
The results indicated that the incremental cost-effectiveness ratio (ICER) of fetal surgery for MMC was dominant, with savings of BRL162,956 (USD26,972.80) per QALY gained. Probabilistic sensitivity analysis showed uncertainty, with 87.3 percent of points agreeing with base case results, but with some dispersion. CEAC showed that fetal surgery is more likely to be cost effective, regardless of the willingness-to-pay threshold. Through deterministic univariate sensitivity analysis, the variation in the cost of the shunt and complications due to prematurity by ±20 percent presented the greatest variations in the ICER, showing the possibility of an increase in cost.
Conclusions
This study demonstrated that prenatal repair generated cost savings, being cost effective, with an ICER of minus BRL162,956 (USD26,972.80) per QALY gained. This is the first study conducted at national level comparing fetal and neonatal myelomeningocele surgery. Although this new approach has been associated with higher costs, these additional costs translate into better long-term results, which could justify changes in health policies.
Integrating large language models (LLMs) into horizon scanning workflows requires understanding of baseline features, like the ability to extract data and handle noisy data, and contextual understanding to inform considerations for LLM use. We evaluated 25 LLMs to assess their applicability for horizon scanning methods in general and to inform the design and integration strategy of our unit’s advanced horizon scanning system.
Methods
We developed a comprehensive framework detailing 32 features across 10 categories for 25 LLMs. To build this framework, we used ChatGPT-4 to generate a preliminary list of categories, features, and LLMs relevant to HS. We supplemented this with parameters from the 2024 LeewayHertz assessment and finalized it through team consensus. Next, we employed a human-in-the-loop approach utilizing a recursive prover-verifier-chain: Microsoft Copilot>Claude 3.5>ChatGPT-4. Each LLM was assessed for variations in baseline features impacting their applicability in horizon scanning methods and potential integration into our horizon scanning system.
Results
We identified six variable features (19%) across five categories. Nineteen of the LLMs support on-premises or self-hosted deployment. Regarding integration flexibility, only seven LLMs were open source and four lacked strong vendor support. Eighteen models offered a usage-based pricing system, allowing budget tailoring. Five LLMs excelled in handling noisy data, beneficial for horizon scanning methods dealing with diverse information sources. Seventeen models had multimodal capabilities.
Conclusions
Variations in key features among the 25 candidate LLMs affected their suitability for integration into horizon scanning workflows. Units must consider the trade-offs between deployment options, open-source availability, vendor support, pricing models, data handling capabilities, and multimodal features. This extensive framework supports assessment and selection of appropriate LLMs for horizon scanning workflows by filtering models according to these key features.
Differentiated thyroid cancer (DTC) treatment involves thyroidectomy and radioiodine ablation. Some patients have contraindications to induced endogenous hypothyroidism or cannot produce endogenous thyroid-stimulating hormone (TSH), preventing ablation unless recombinant TSH is used. This study aimed to evaluate the cost effectiveness of recombinant TSH followed by ablation in patients with DTC from the Brazilian Unified Health System perspective.
Methods
An economic evaluation was conducted to estimate the incremental cost-effectiveness ratio of radioiodine ablation (with prior recombinant TSH), compared with the absence of these procedures in patients who, after thyroidectomy, are unable to induce endogenous hypothyroidism. A Markov model was built with three health states: no recurrence, recurrence, and death. The outcomes evaluated were quality-adjusted life years (QALYs) and life years (LYs) gained. Direct medical costs were included over a 30-year time horizon, with a five percent discount rate applied to both costs and outcomes. A deterministic and probabilistic sensitivity analysis was also performed.
Results
In general, the combination of recombinant TSH with radioiodine ablation proved to be dominant compared with not performing ablation, resulting in lower costs (BRL25,062 versus BRL26,941 [USD10,271 versus USD11,041]) and better clinical outcomes (QALYs 9.69 versus 9.55 and LYs 10.82 versus 10.57). This result was mainly due to a statistically significant difference in recurrence rates. In the probabilistic sensitivity analysis, most simulations showed greater clinical benefit with recombinant TSH and ablation than no ablation. Additionally, the deterministic analysis indicated that the correction factor applied to costs and recurrence risk were the most impactful parameters in the economic model.
Conclusions
The combination of recombinant TSH with radioiodine ablation was considered dominant in terms of QALYs and LYs gained, compared with no ablation, meaning the evaluated technology resulted in greater clinical benefit and lower cost.
The Epidemiology and Health Services: Journal of the Brazilian National Health System (RESS; Epidemiologia e Serviços de Saúde: revista do SUS) is a journal edited by the Brazilian Ministry of Health focused on disseminating scientific evidence relevant to the Brazilian Unified Health System. We aimed to assess the patterns of publication relevant to health technology assessment (HTA) and appraise the journal’s contribution to advancing knowledge and decision-making in the field.
Methods
We performed a cluster analysis of RESS publications from 2016 to 2024. Abstracts were imported from PubMed to EndNote and exported to Excel. Text preprocessing (tokenization, removal of punctuation, numbers, stop words, and stemming) was performed. Abstracts with terms related to diseases and technologies were regarded as HTA relevant. Corpuses were converted into term frequency–inverse document frequency, and dimensionality was reduced using principal component analysis. K-means clustering with silhouette coefficient for consistency were calculated. Thematic groups were analyzed based on frequent terms, document distribution, and intra-cluster similarity and compared to HTA Core Model®. Python 3.13 was used for analyses.
Results
From 674 abstracts, 409 were deemed relevant for HTA. Six clusters were identified: risk factors (22.2% of abstracts; cohesion 95.5%), health services (33.5%; cohesion 0.8%), infections and clinical services (18.8%; cohesion 86.4%), mortality trends (14.2%; cohesion 95.6%), causes of death (8.8%; cohesion 86.9%), and congenital syphilis (2.4%; cohesion 94.2%). The silhouette coefficient was 0.4, indicating moderate clustering quality. Most clusters displayed high internal cohesion, except for health services, and were related to HTA domains of current use, safety, organization, and patient and social. No clusterization of technical, clinical effectiveness, cost and economic, ethical analysis, or legal domains was observed.
Conclusions
Main patterns of HTA relevant research were identified, with thematic clusters such as risk factors, mortality, health services, and infections. Other HTA relevant domains—like economic evaluations and patient-reported outcomes—were not detected in the clusters, suggesting a gap in RESS focus. To improve its relevance for the Brazilian HTA field, RESS should prioritize less represented HTA domains.
Cutaneous leishmaniasis (CL) is a neglected infectious disease with a known negative impact on health-related quality of life (HRQoL). However, no studies were found that quantitatively defined the health utility of CL. Our aim was to evaluate the impact of CL on HRQoL in patients attending a Brazilian reference center using the EQ-5D-3L visual analogue scale questionnaire to generate utility measures for CL.
Methods
This cross-sectional study included patients with CL from a referral center for the disease in Minas Gerais, Brazil between 2020 and 2023. Patients were interviewed during active disease and retrospectively before the onset of disease symptoms. Each patient’s health status was assessed using the EQ-5D-3L questionnaire. The losses in HRQoL were estimated by comparing the proportion of responses before CL onset with those during its active phase. Socioeconomic data were collected using a standardized questionnaire, and sociodemographic and clinical data were collected directly from the medical records. This study was approved by the Fiocruz Minas ethics committee.
Results
A total of 143 patients with a mean age of 52 years (standard deviation 17) were included, 73 percent of whom were male. The mean utility score before the onset of CL symptoms was 0.858. A comparison of the responses related to health status before and after the illness revealed significant losses (p<0.05) in all dimensions of the EQ-5D, especially those related to pain, malaise, and usual activities, which were reduced to 50 percent and 27 percent, respectively. CL also affected the median visual analogue scale score and utility scores, which were 87 and 70 and 0.858 and 0.716, respectively.
Conclusions
These results confirmed the negative impact of CL on all dimensions assessed by the EQ-5D-3L visual analogue scale questionnaire, revealing another mechanism perpetuating the cycle of suffering and poverty surrounding neglected tropical diseases. These findings are essential for conducting future cost-utility studies, enabling comparisons of different treatments for this disease.
Implementation of the Health Technology Assessment (HTA) Regulation (HTAR) marks a shift in the assessment of high-risk medical devices (MDs) and in vitro diagnostics (IVDs) across the European Union (EU) and European Economic Area (EEA). Understanding current decision-making pathways for reimbursement of these technologies is crucial to inform HTAR integration within national frameworks. This review aimed to understand how comparative safety and effectiveness evidence is used in reimbursement decisions.
Methods
A scoping review was conducted in accordance with a registered protocol (osf.io/65bdk). Relevant documents were identified through searches of websites of HTA bodies and national health authorities across the EU, EEA, and UK. Relevant publications were identified from key databases (MEDLINE Complete, Embase, and the Cochrane Library) and supplemented by a comprehensive gray literature search. Data were extracted and checked by a second reviewer. National health officials further validated the extracted data.
Results
The searches of official websites resulted in the retrieval of 369 relevant records. Database and gray literature searches resulted in the retrieval of 953 records, with 134 of these being included after full-text screening. The results revealed diverse reimbursement pathways and HTA processes across the EU, EEA, and UK for high-risk devices. Findings highlighted the variability in reimbursement processes. Some countries rely on centralized decision-making bodies, while others rely on regional or hospital-specific approaches. The findings also emphasized the limited integration of comparative safety and effectiveness data in the reimbursement frameworks of some countries and underscored the complexity of reimbursement processes across the European region.
Conclusions
This review identified key variations in reimbursement pathways for high-risk MDs and IVDs across the EU, EEA, and UK. The review of the European reimbursement decision-making landscape and the role of HTA underscores the importance of sharing experiences to facilitate the efficient use of HTAR-generated evidence in decision-making and to support unified patient access to safe and effective technologies.
The HI-PRIX European project aims to accelerate access to high value, affordable health innovations. This Delphi exercise aimed to build a consensus on new payment schemes that could facilitate the adoption or uptake of innovative medical devices, including digital health technologies, in an integrated care (IC) context.
Methods
A two-round Delphi exercise was performed, which included 29 statements distributed into four sections: (i) principles for innovation to qualify for a new payment scheme; (ii) payment schemes to support the adoption of innovative technologies by healthcare providers in an IC context; (iii) additional payment schemes for digital health technologies; and (iv) targeted funding sources. The Delphi was sent to 180 thematic-knowledgeable professionals. A six-point Likert scale was used to quantify agreement and consensus rules were established. Round two focused only on statements with moderate consensus.
Results
Round one received 92 responses, with around 65 percent of the statements achieving moderate consensus, 28 percent high consensus, and seven percent low consensus. High consensus statements emphasized the importance of incorporating health technology assessment processes to inform pricing negotiations, establishing clear criteria for qualifying innovations into an innovative payment scheme, and incorporating performance-based payments to incentivize their adoption. The findings also highlighted the need to align payment schemes with broader healthcare objectives, such as improving patient pathways and ensuring equitable access, while addressing implementation barriers. Participants stressed the importance of tailoring payment schemes to specific healthcare contexts and ensuring financial sustainability.
Conclusions
The Delphi exercise highlighted both the need and the feasibility of implementing new payment schemes to support the adoption of innovative technologies, specifically in IC contexts. Combining different payment schemes is advisable to leverage the benefits of innovative technologies, while minimizing potential perverse incentives and ensuring alignment with broader healthcare objectives.
Most treatment regimens for people living with HIV (PLHIV) require the intake of multiple daily pills, which can pose challenges to adherence. As an alternative, long-acting (LA) dual regimens have gained attention for their potential to simplify treatment. Given the importance of evaluating the long-term effects of LA regimens, this study aimed to synthesize evidence on their effectiveness and safety.
Methods
A systematic review was performed following the Cochrane Collaboration recommendations and the Meta-Analysis of Observational Studies in Epidemiology (MOOSE) guidelines, with searches in PubMed, Scopus, and Web of Science (July 2024) (PROSPERO-CRD42024570754). Longitudinal studies evaluating any dual-therapy antiretroviral regimen with LA agents (islatravir, cabotegravir, lenacapavir, rilpivirine, albuvirtide) for PLHIV were included. For each outcome of interest, data were pooled using bivariate meta-analysis (discontinuations due to adverse events) and network meta-analysis (NMA) (virologic failure [VF]), with results presented as risk ratios (RR) with 95 percent credibility intervals (CrIs); heterogeneity was assessed using the I-squared statistic. The surface under the cumulative rating curve analysis (SUCRA) was also conducted for the NMA.
Results
Thirty-two studies (2016 to 2024) evaluating 11 treatments were included. Four cohorts (n=2,570) were assessed in the NMA of VF, where the rilpivirine/dolutegravir (RPV/DTG) regimen was superior to non-LA regimens dolutegravir/lamivudine (RR −1.60, 95% CrI: −4.08, 0.91) and dolutegravir/doravirine (RR −0.42, 95% CrI: −1.68, 0.68), with SUCRA probabilities of 9.6 percent, 31 percent, and 59.5 percent, respectively. The triplet regimens dolutegravir plus tenofovir/emtricitabine and dolutegravir plus abacavir/lamivudine had the highest probabilities of leading to CD4+ count increases at six to 24 months (65.6 to 85.7% and 65.8 to 84.4%, respectively), followed by RPV/DTG (59 to 60%) and dolutegravir/lamivudine (46 to 53%). However, RPV/DTG regimens were considered safer than dolutegravir/lamivudine (RR 0.53, 95% CrI: 0.32, 0.89) regarding discontinuation due to adverse events.
Conclusions
This updated synthetized evidence suggested that the RPV/DTG regimen is a promising alternative for simplifying the treatment of PLHIV, demonstrating comparable effectiveness to the dolutegravir/lamivudine and dolutegravir/doravirine regimens, while offering better tolerability. However, considerations of drug accessibility and cost must be carefully weighed in the decision-making process in different settings.
A legislation, regulation, and policy review of limited South African legislative framework of health technology assessment (HTA) was undertaken to identify opportunities to advocate for required legislation reform or development to entrench principles and processes for patient and citizen involvement (PCI). We reviewed and mapped the capacity, knowledge, and skills of South African patient and citizen advocacy actors to actively advocate in developing regulations and policy in the National Health Insurance landscape.
Methods
We used a literature scan and analysis of relevant national and international documentation, including reports of primary and secondary data analysis to aid in the identification of current gaps in, and opportunities for, PCI in HTA in South Africa. Data sources included resolutions, legislation, regulations, government policy, and technical reports gleaned from websites of relevant agencies and experts. An electronic survey was distributed to 213 patient and citizen actors. The survey aimed to establish the capacity, knowledge, and skills of South African patient and citizen advocacy actors and organizations across multiple disease areas, predominately non-communicable diseases.
Results
Legislation and policy documents indicated engagement initiatives are located at “involvement” or “consultation” stages of the engagement continuum. Patient advocacy groups participated in raising awareness of disease burden, advocating for adoption of specific technology, or utilizing legal channels to compel coverage arrangements for specific therapies.
The barriers and challenges observed were:
• lack of skills, training, and education;
• insufficient information regarding roles and responsibilities;
• lack of logistical or financial support; and
• lack of integrated strategy.
Possible dimensions for capacity building include select starting points that build on research partners’ strengths, meeting the immediate needs of governments, and contributing to longer-term goals.
Conclusions
Results indicated non-involvement of key actors in crucial healthcare processes. Actors were adamant that they would have greater influence at the provincial or national level to make meaningful contributions. Existing legislative and policy frameworks do not include PCI capacity building strategies. We can only conclude that once healthcare processes are inclusive, PCI will become mandatory.
Restricted access to orphan drugs significantly challenges rare disease patients, impairing health outcomes and quality of life. In Türkiye, systemic barriers such as regulatory inefficiencies, economic limitations, and inadequate stakeholder collaboration persist. This study evaluated these issues through stakeholder perspectives, proposing a collaborative framework to improve access, ensure equity, and enhance systemic efficiency in addressing rare disease challenges in Türkiye.
Methods
This qualitative study employed semi-structured focus group discussions with 19 patients and caregivers and 13 in-depth interviews with healthcare providers, policymakers, industry representatives, and regulatory bodies. Thematic analysis of 1,996 coded excerpts identified key barriers to orphan drug access, categorized as direct or indirect impacts. Grounded theory was applied to synthesize these findings into a cohesive framework addressing economic, regulatory, and systemic challenges. Data triangulation enhanced validity, ensuring the perspectives of diverse stakeholders were accurately represented. Stakeholder feedback on the framework was incorporated iteratively, ensuring practicality and adaptability for policy and decision-making in Türkiye. Supported by TUBITAK 2224-A Program for participation in HTAi 2025; it had no role in the study conduct.
Results
The study revealed multifaceted barriers to orphan drug access in Türkiye, including fragmented regulatory processes, high costs, and inadequate inter-stakeholder collaboration. Indirect factors, such as limited public awareness and insufficient professional education, exacerbated these challenges. The proposed framework emphasizes centralized regulatory mechanisms to streamline approvals, public–private partnerships to mitigate financial constraints, and enhanced communication to ensure transparency and accountability. Stakeholder feedback highlights the framework’s adaptability and potential impact, particularly in addressing disparities within Türkiye. Success metrics, such as improved regulatory efficiency and patient outcomes, were defined to support future implementation and continuous improvement.
Conclusions
The proposed framework addresses critical systemic barriers to orphan drug access in Türkiye by fostering collaboration, streamlining regulatory pathways, and enhancing equity. It emphasizes the importance of stakeholder alignment and sustainable policy solutions for rare disease patients. Future research may focus on piloting the framework, adapting it for broader contexts, and developing evaluation tools to measure its long-term impact.
Globally, healthcare services contribute five percent of total greenhouse gas (GHG) emissions. Advances in biomedical research have led to innovative technologies, but the use of fossil fuel reliant products is a critical environmental issue. The burden is even greater in low resource settings (LRS) with increasing waste management issues. Real world studies can shed light on the environmental footprint of health care and help prioritize assessments.
Methods
Various policies and guidance are being developed on environmental sustainability of healthcare. However, it is imperative that health technology at different levels of care is studied empirically, taking a holistic approach to the environmental impact from a planetary health perspective. By presenting research from LRS, we can understand how use of devices and services have different environmental consequences depending on established regulatory frameworks. We conducted a rapid review with snowballing to understand how LRS have adapted to unmet needs and tried to solve these using a circular economy approach.
Results
The footprint of a typical cataract operation in a high resource setting was 182 kg of carbon dioxide equivalent. Efficient systems and equipment reuse reduces this to 6 kg in a LRS, a 30-fold reduction. Despite both having positive clinical outcomes, one phacoemulsification surgery in the UK emits 20 times more GHG than the equivalent in India. Resource scarcity necessitated circular economy practices like “repair” and “reuse” to keep products in circulation. An electrosurgical unit, typically lacking in LRS, is designed to be durable, repairable, upgradable, and reusable even during electrical failures. Planetary and human health across all settings require resource optimization, innovative materials engineering, and reusable solutions.
Conclusions
Urgent research is needed to understand the potential impacts of more sustainable health technology innovation across eye care in different settings. A broader range of environmental impacts beyond GHG emissions studied across the life cycle of devices is necessary. Routine inclusion of environmental outcomes in clinical studies and explicit identification, testing, and promotion of sustainable practices will help promote sustainable decisions.
Over twenty years ago, an editorial in this journal called for further studies of the epidemiological and psychopathological characteristics of ‘recurrent brief depression’ in clinical samples in primary and secondary care settings. At that time, relatively little was known about the epidemiology or potential neuropsychobiology of the condition, and no evidence-based treatments had been identified. Two decades have passed, but there have been no substantial developments in understanding in the last ten years. The seeming withdrawal from research into recurrent brief depression is regrettable, given widespread concerns about the burden of depressive symptoms in young people. It seems reasonable to call once again for further investigations in clinical samples, this time with a focus on younger individuals.
Saúde e Educação (SeE) is an extension project of the Federal University of Minas Gerais (UFMG), Brazil, which aims to disseminate information and research related to health in a simplified way for the public. Based on the perception of existing knowledge gaps in society, the SeE created a thematic series to disseminate accessible information about health technology assessment (HTA).
Methods
A partnership was established between the Collaborating Centre for Health Technology Assessment and Excellence (CCATES) and the UFMG. Members of CCATES were invited by the SeE to voluntarily produce informative texts about HTA to be published and disseminated by the SeE. The SeE editors reviewed the texts and formatted them to have a layout that motivates reading and facilitates understanding. The materials were published on the SeE website and disseminated on the project’s social networks. Strategic days were selected for publication. Partnerships with patient associations, for example, were formed to facilitate dissemination. The project had no funding.
Results
In 2024, five texts by seven collaborators were produced, published, and disseminated. Three are in the process of preparation or review. To date, the texts have received 154 views on the website, and the advertisements have received more than 1,622 views on social media. Challenges encountered include problems with knowledge translation and reaching the target audience.
Conclusions
The SeE has been disseminating knowledge about HTA through digital means. This is important to ensure that information is accessible to the public. It can promote more inclusive and participatory decision-making, contribute to equity in access to health innovations, strengthen social control, and improve the implementation of technologies that meet the population’s needs.
The Australian Government Department of Health and Aged Care (Department) and Medicines Australia (the peak body for the pharmaceutical industry in Australia) have entered into a five-year strategic agreement. A core objective of this agreement was to develop a consumer-led, co-designed process to enhance consumer engagement earlier in the Australian health technology assessment (HTA) system.
Methods
A co-design proposal was developed by a consumer reference group and included guidance on “consumer-led” processes such as establishing a Co-design Working Group (Group) of consumer, industry, and Department members weighted toward a greater number of consumers. Selection of consumer members was by a consumer-peer selection panel. An independent facilitator led the co-design process, founded on a work schedule and design principles developed by the Group. Recent consultation reports on consumer engagement in HTA processes guided the Group and enabled it to be “solution focused.” The Group sought open consultation on draft recommendations prior to finalizing a report of recommendations.
Results
The Enhance HTA report describes recommendations for consideration by the Australian Minister for Health and Aged Care. The Group established a vision where “Australians’ diverse health care experiences and needs are understood, and consumer engagement is integral in HTA decision-making.” Enhance HTA includes 10 recommendations related to themes of partnerships, transparency, and evidence and input. The report notes the need for alignment with reformative HTA policies and methods, investment in systemic change, and development of a framework to embed the proposed recommendations along the health technology pathway. The co-design process and outcomes will be discussed if accepted for presentation.
Conclusions
The consumer-led, co-designed process is a strong signal of the desire to engage consumers earlier in Australian HTA processes and in a more meaningful way. The expectations are high that the recommendations emerging from this process may be implemented to further embed consumer engagement into HTA processes, ensuring the diverse healthcare needs of Australians are integral to HTA decision-making.
Health systems in Australia and globally struggle to keep up with disruptive health technologies like gene therapies, gene editing, regenerative medicine, genomics, and artificial intelligence. These technologies promise better health outcomes but pose challenges due to rapid advancements, information gaps, and lack of a unified national approach. Solutions require new legal frameworks, workforce training, infrastructure investments, and government funding, supported by horizon scanning (HS) to anticipate future needs.
Methods
We will provide a review of the history of HS efforts to date in Australia and outline our proposal for the establishment of a new HS function to transform Australia’s approach to new technologies from reactive to proactive. This function will integrate with post-reform health technology assessment (HTA) processes to support planning and priority setting across all levels of government, service providers, product developers, and consumers by identifying emerging technologies and outlining their benefits, risks, and operational implications over the short and long term.
Results
Australia has a 17-year history of HS devices and diagnostic health technologies for HTA, investment, implementation, and disinvestment purposes, although such activity ceased during the pandemic. There is growing support for re-establishing HS, although stakeholders have diverse views on its use. A proposed HS approach aims to support planning and priority setting across federal and state/territory governments, service providers, product developers, and consumers by identifying emerging technologies and articulating the likely benefits, risks, and operational implications, including resource requirements, over near- and longer-term horizons.
Conclusions
With respect to HTA, following the completion of the HTA Policy and Methods Review, the Australian government established an Implementation Advisory Group to develop a roadmap in 2025 for the government’s response to the review recommendations. We will discuss how our proposed HS function can integrate with the HS-related reforms that have been proposed under this review.