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Circulating tumor DNA (ctDNA) detection has multiple promising applications in oncology, but the road toward implementation in clinical practice is unclear. We aimed to support the implementation process by exploring potential future pathways of ctDNA testing. To do so, we studied four ctDNA‐testing applications in two cancer types and elicited opinions from 30 ctDNA experts in the Netherlands. Our results showed that the current available evidence differed per application and cancer type. Tumor profiling and monitoring treatment response were found most likely to be implemented in non‐small cell lung cancer (NSCLC) within 5 years. For colorectal cancer, applications of ctDNA testing were found to be at an early stage in the implementation process. Demonstrating clinical utility was found a key aspect for successful implementation, but there was no consensus regarding the evidence requirements. The next step toward implementation is to define how clinical utility of biomarkers should be evaluated. Finally, these data indicate that specific challenges for each clinical application and tumor type should be appropriately addressed in a deliberative process involving all stakeholders to ensure implementation of ctDNA testing and timely access for patients.

Emanuela Foglia,1 Federica Asperti,1 Grazia Antonacci,2,3 Yogini H Jani,4 Elisabetta Garagiola,1 Daniele Bellavia,1 Lucrezia Ferrario1 1LIUC - University Cattaneo, Healthcare Datascience LAB, Castellanza, Varese, 21053, Italy; 2Department of Primary Care and Public Health, Imperial College London, National Institute of Health Research (NIHR) Applied Research Collaboration (ARC) Northwest London, London, UK; 3Business School, Centre for Health Economics and Policy Innovation (CHEPI), Imperial College London, London, UK; 4Centre for Medicines Optimisation Research and Education, University College London Hospitals NHS Foundation Trust, London, UKCorrespondence: Elisabetta Garagiola, Healthcare Datascience Laboratory - HD LAB, LIUC-Università Cattaneo, Corso G. Matteotti, 22, Castellanza, VA, 21053, Italy, Tel +39.0331.572.467, Email egaragiola@liuc.itBackground: Automated Drug Dispensing (ADD) systems are considered to be strategic hospital assets used to reduce errors and enhance economic and organizational sustainability. With regards to efficacy and safety, the literature evidence demonstrates the incremental benefits of centralised or decentralised systems compared to manual dispensing. Analyses about organisational and economic sustainability are still lacking and the present study aims to perform a Health Technology Assessment (HTA), producing multidimensional evidence on the use of ADD systems within hospitals.Methods: In 2023, a comprehensive HTA draws insights from healthcare professionals across six European nations: Italy, France, Germany, the Netherlands, the United Kingdom, and Belgium. This appraisal juxtaposed four drug dispensing scenarios: manual methods, centralized ADD systems, decentralized ADD systems, and integrated solutions employing cutting-edge technologies in both central pharmacies and wards. The study deployed an Activity-Based Costing approach that was combined with a cost-effectiveness and Budget Impact Analysis to evaluate economic impacts. Qualitative questionnaires were implemented to assess ethical, legal, organizational, safety, and efficacy aspects.Results: From a multidimensional perspective, healthcare professionals acknowledged ADD manifold advantages of ADD systems. From an organizational perspective and within a 12-month timeframe, transitioning to automation may face initial challenges that are attributed to potential resistance from professionals and significant investments. However, 36 months past its adoption, automation’s superiority over manual methods was recognized. Economically, savings burgeoned from +17.9% in UK to +26.6% in Belgian hospitals that adopted integrated systems in comparison to traditional manual approaches.Conclusion: Compared to traditional methods, implementing ADD systems could improve the logistic management of drug in the hospital setting, thereby enhancing safety and efficacy, streamlining the healthcare professionals’ workflow, and bolstering financial stability.Keywords: automated drug dispensing systems, HTA, economic impact, drug logistic management, efficiency

Abstract Background Moldova strives for universal health coverage (UHC) and considers health technology assessment (HTA) an important policy instrument to inform the choice of services that should be covered to progressively realize UHC. It plays a key role in determining which technologies are coved, considering various dimensions often including cost-effectiveness, budget impact, and feasibility. This paper reports on work undertaken to develop a roadmap for HTA implementation, using evidence-informed deliberative processes (EDPs) as the guiding framework. Methods Between 2020 and 2022, we undertook several activities that informed the roadmap. We conducted a needs assessment and an assessment of European HTA best practices using a combination of desk research, interviews and surveys. We then conducted a document review of six selected HTA systems, complimented by expert interviews from three neighbouring countries. Results The roadmap provided a suggested HTA process, which included instructions on how to execute the different steps of the EDP framework to enhance the legitimacy of HTA-informed decision-making. The roadmap encompasses several aspects such as how to organize stakeholder involvement, how to identify and operationalize decision criteria, and how to make the decision process transparent. Guidance was given to the country on establishing a legal framework for HTA; a communication strategy; strengthening capacity and leveraging HTA expertise. The country was also given tailored advice on the positioning of the HTA-agency, first as an entity within the Ministry of Health or the National Agency of Public Health but with the long-term vision for it to be independent of any state institution. Conclusions Despite several challenges, including a partial disruption because of the Covid-19 pandemic, the development of the roadmap for HTA implementation was completed and agreed upon by the Ministry of Health in Moldova. This work has helped motivate and support the country in HTA institutionalization.

Aim: Regulatory and health technology assessment (HTA) agencies have increasingly published frameworks, guidelines, and recommendations for the use of real-world evidence (RWE) in healthcare decision-making. Variations in the scope and content of these documents, with updates running in parallel, may create challenges for their implementation especially during the market authorization and reimbursement phases of a medicine’s life cycle. This environmental scan aimed to comprehensively identify and summarize the guidance documents for RWE developed by most well-established regulatory and reimbursement agencies, as well as other organizations focused on healthcare decision-making, and present their similarities and differences. Methods: RWE guidance documents, including white papers from regulatory and HTA agencies, were reviewed in March 2024. Data on scope and recommendations from each body were extracted by two reviewers and similarities and differences were summarized across four topics: study planning, choosing fit-for-purpose data, study conduct, and reporting. Post-authorization or non-pharmacological guidance was excluded. Results: Forty-six documents were identified across multiple agencies; US FDA produced the most RWE-related guidance. All agencies addressed specific and often similar methodological issues related to study design, data fitness-for-purpose, reliability, and reproducibility, although inconsistency in terminologies on these topics was noted. Two HTA bodies (National Institute for Health and Care Excellence [NICE] and Canada’s Drug Agency) each centralized all related RWE guidance under a unified framework. RWE quality tools and checklists were not consistently named and some differences in preferences were noted. European Medicines Agency, NICE, Haute Autorit ´e de Sant´ e, and the Institute for Quality and Efficiency in Health Care included specific recommendations on the use of analytical approaches to address RWE complexities and increase trust in its findings. Conclusion: Similarities in agencies’ expectations on RWE studies design, quality elements, and reporting will facilitate evidence generation strategy and activities for manufacturers facing multiple, including global, regulatory and reimbursement submissions and re-submissions. A strong preference by decision-making bodies for local real-world data generation may hinder opportunities for data sharing and outputs from international federated data networks. Closer collaboration between decision-making agencies towards a harmonized RWE roadmap, which can be centrally preserved in a living mode, will provide manufacturers and researchers clarity on minimum acceptance requirements and expectations, especially as novel methodologies for RWE generation are rapidly emerging.

Abstract Background There is limited evidence-informed guidance on TISP processes for countries where health technology assessment (HTA) is in a nascent phase. We aimed to explore the range of topic identification, selection and prioritization (TISP) processes and practices for HTA in selected countries and identify aspects relevant to emerging HTA systems. Methods This mixed design study included a systematic literature review, an electronic survey, and individual interviews. We conducted a systematic literature review with criteria that were developed a priori to identify countries deemed to have a recently formalized HTA system. Based on the literature review, a twenty-three item online survey was shared with the identified countries, we completed follow-up interviews with ten participants who have experience with HTA. We analyzed documents, survey responses and interview transcripts thematically to identify lessons related to TISP processes and practices. Results The literature review identified 29 nine candidate countries as having a “potential” recently formalized HTA system. Twenty-one survey responses were analyzed and supplemented with ten individual interviews. We found variation in countries’ approaches to TISP — particularly between pharmaceutical and non-pharmaceutical interventions. Results indicate that TISP is heavily driven by policy makers, expert involvement, and to a lesser extent, relevant stakeholders. The use of horizon-scanning and early warning systems is uncommon. Interviewee participants provided further insight to the survey data, reporting that political awareness and an institutional framework were important to support TISP. TISP can be optimized by stronger national regulations and legislative structures, in addition to education and advocacy about HTA among politicians and decision-makers. In some settings regional networks have been useful, particularly in the development of TISP guidelines and methodologies. Additionally, the technical capacity to conduct TISP, and access to relevant local data were factors limiting TISP in national settings. Increased network collaboration and capacity building were reported as future needs. Conclusions This study provides current insights into a topic where there is limited published peer reviewed literature. TISP is an important first step of HTA, and topics should be selected and prioritized based on local need and relevance. The limited capacity for TISP in settings where HTA is emerging may be supported by local and international collaboration to increase capacity and knowledge. To succeed, both TISP and HTA need to be embedded within national health care priority setting and decision-making. More in-depth understanding of where countries are situtated in formalizing the TISP process may help others to overcome factors that facilitate or hinder progress.

Since late 2020, the Canadian Agency of Drugs and Technologies in Health (CADTH) has been using a threshold of $50,000 (CAD) per quality-adjusted life-year (QALY) for both oncology and non-oncology drugs. When used for oncology products, this threshold is hypothesized to have a higher impact on the time to access these drugs in Canada. We studied the impact of price reductions on time to engagement and negotiation with the pan-Canadian Pharmaceutical Alliance for oncology drugs reviewed by CADTH between January 2020 and December 2022. Overall, 103 assessments reported data on price reductions recommended by CADTH to meet the cost–effectiveness threshold for reimbursement. Of these assessments, 57% (59/103) recommendations included a price reduction of greater than 70% off the list price. Eight percent (8/103) were not cost-effective even at a 100% price reduction. Of the 47 assessments that had a clear benefit, in 21 (45%) CADTH recommended a price reduction of at least 70%. The median time to price negotiation (not including time to engagement) for assessments that received at least 70% vs >70% price reduction was 2.6 vs 4.8 months. This study showed that there is a divergence between drug sponsor’s incremental cost–effectiveness ratio (ICER) and CADTH revised ICER leading to a price reduction to meet the $50,000/QALY threshold. For the submissions with clear clinical benefit the median length of engagement (2.5 vs 3.3 months) and median length of negotiation (3.1 vs 3.6 months) were slightly shorter compared with the submissions where uncertainties were noted in the clinical benefit according to CADTH. This study shows that using a $50,000 per QALY threshold for oncology products potentially impacts timely access to life saving medications.

Oortwijn et al continue their guide to good practice in the use of deliberative processes in health technology assessment (HTA) based on a survey of international practice. This is useful, and I applaud their care in maintaining objectivity, especially regarding the treatment of moral and politically controversial issues, in reporting how jurisdictions have handled such matters in designing HTA procedures and in their execution. To their suggestions for future research, I add: the historical development of deliberation in healthcare decision-making and in other fields of public choice, with comparisons of methods, successes and failures; development of guidance on the design and use of deliberative processes that enhance decision-making when there is no consensus amongst the decision-makers; ways of identifying and managing context-free and context-sensitive evidence; and a review of high-level capacity building to raise awareness of HTA and the use of knowledge translation and exchange (KTE) and deliberation amongst policy makers, especially in low and middle-income countries.

Background: According to scientific literature, some 99% of patients affected by Alzheimer’s disease (AD) suffer from behavioral and psychological symptoms of dementia (BPSD), also known as neuropsychiatric symptoms (NPSs). In particular, agitation is one of the most difficult disorders to treat. States of agitation represent a very serious problem as they make these subjects dangerous for themselves and others and worsen as the disease advances. To date, there are no specific solutions for treating agitation. The only authorized drug is risperidone (as well as brexpiprazole, approved by the FDA on 11 May 2023), which can be used for no longer than 6–12 weeks because it increases the risk of death—owing to cardiocerebrovascular accidents—by 1.6–1.7 times. Methods: In order to address the latter noteworthy unmet medical need, NanoBEO was produced. The aim of the present work is to generate the health technology assessment (HTA) of this nanotechnological device. The latter consists of a controlled release system, based on solid lipid nanoparticles loaded with bergamot essential oil (BEO). Results: The results of the present research assessed the current evidence in the field of non-pharmacological treatments for this condition, including relevant primary preclinical and clinical data studies supporting the use of this device and the production of the operative plan for its launch on the market. The findings offer recommendations for decision-making on its implementation in dementia. Conclusions: NanoBEO represents a public-worth innovation in this neglected area, marking a significant advancement in the history of dementia, moving from academic research to product development.

Background/Objectives. Many vaccines have been developed in recent decades, and many more will be available in the future. When new safe and effective vaccines are available, decision-makers must extensively assess them before including them in the national immunization plan and issuing recommendations. The Health Technology Assessment (HTA) could be an objective, transparent, and comprehensive approach to guiding the decision-making process for the use of vaccines. Objectives and Methods. The aim of this study was to review the indications for HTA use contained in Italian institutional documents on vaccination, namely the National Immunization Plans (NIPs) and available full Italian HTA reports on vaccines, assessing their availability at the time of national recommendations’ introductions. Results. HTA has been recognised as an eligible approach to deciding upon the introduction of vaccines through the NIPs of 2012–2014 and 2017–2019, and the last NIP, of 2023–2025, highlights the lack of funding dedicated to the production of independent HTA reports that can be used for issuing recommendations. In 2007–2023, twenty full HTA reports on vaccines were published in Italy: eight reports on influenza vaccines, five on Human Papilloma Virus (HPV), three each on meningococcal and pneumococcal vaccines, and one on rotavirus vaccine. HTA was applied with different purposes, namely the evaluation of new vaccines or their re-assessment, but it was not always timely with respect to both the marketing authorisation and the issuing of national recommendations for use. Conclusions. As HTA can be considered the best tool to disentangle the overall value of vaccines, it would be desirable for it to be used more and more to provide the evidence for efficient resource use. This calls for action to improve the transfer of HTA results to decision-makers, to try to fill the gap between research and decision and foster evidence-based recommendations.

Objectives: The aim of this study was to characterize the reimbursement policy for orphan drugs (ODs) in Central and Eastern European (CEE) countries in relation to the availability and impact of clinical evidence, health technology assessment (HTA) procedure, selected economic indicators, and the drug type according to indications.Materials and methods: A list of authorized medicines with orphan designation and information about active substance, Anatomical Therapeutic Chemical (ATC) classification, and therapeutic area was extracted from the web-based register of the European Medicines Agency (EMA). A country-based questionnaire survey was performed between September 2021 and January 2022 in a group of selected experts from nine CEE countries (an invitation was sent to 11 countries). A descriptive and statistical analysis was conducted to determine statistical significance, correlations, between the drug or country characteristic and the positive recommendation or reimbursement of ODs.Results: The proportion of reimbursed orphan drugs differed between countries, ranging from 17.7% in Estonia to 49.6% in Hungary (p < 0.001). The odds that ODs were reimbursed were reduced in countries with a “strong” level of impact of drug safety and efficacy on reimbursement decisions (p=0.018), the presence of other additional specific clinical aspects (e.g., genomic data) considered in the reimbursement decision (p < 0.001) and mandatory (without exception) safety assessments (p=0.004). The probability that ODs were reimbursed was increased in countries with a “moderate” level of impact of drug safety and efficacy on reimbursement decisions (p=0.018), when reimbursement decisions are dependent on the EMA registration status and orphan drug designation (p < 0.001), the presence of the “positive HTA recommendation guarantees reimbursement” policy (p < 0.001), higher GDP per inhabitant (p=0.003), and higher healthcare expenditure (p < 0.001).Conclusion: We found that there are differences among CEE countries in the reimbursement of orphan drugs, and we identified aspects that may influence these differences. Safety, efficacy, and specific clinical aspect issues significantly influenced reimbursement decisions. Antineoplastic and immunomodulating agents drugs were the largest group of ODs and increased the chance of getting a positive recommendation. The higher GDP per inhabitant and healthcare expenditures per inhabitant were positively linked to the chance that an OD receives reimbursement.

Amidst competing priorities for allocating finite health resources, using evidence-informed priority setting is a valuable tool for achieving population-level health goals. The paper by Baltussen et al comprehensively reports on the development of practical guidance for evidence-informed deliberative processes (EDPs) which will help with sustainability of programs aimed at universal health coverage (UHC). The authors’ experience with the Joint Learning Network for UHC’s (JLN) peer-to-peer learning platform on evidence-informed priority setting offers insights on the practical challenges faced by countries in health benefits package (HBP) design, especially to draw in actors to advocate for the priorities and values across the health system. Lessons harvested from JLN countries that have established such advisory committees can provide practical insights for countries in earlier stages of establishing a systematic process for HBP design. Peer-to-peer learning modalities among countries offer viable and effective approaches to institutionalizing EDPs and systematic priority setting.

Abstract Introduction Patient and public involvement (PPI) is an expectation when conducting research, including Health Technology Assessment (HTA), but practical guidance for those wishing to embed PPI into the grant application process is not always easily accessible. We wanted to ensure that PPI was central when preparing a proposal for an investigator‐led evidence synthesis HTA investigating nonsurgical interventions for pelvic organ prolapse (POP) in women. Here, we describe our methods. Methods We recruited two patient co‐applicants separately through an open process to help ensure that patient voice was present within our proposal's management and direction. We invited co‐applicants to attend research team meetings and comment on the full proposal. We designed, recruited to and facilitated a scoping workshop, as well as undertook its subsequent evaluation. The insight shared within the workshop for patients with a lived experience of POP, including our patient co‐applicants, helped us develop the scope and rationale behind our HTA proposal. We particularly considered the interventions to include within the evidence synthesis. We also considered the outcome measures for both the evidence synthesis and economic evaluation. We elicited ideas about where and how results could be disseminated. Feedback suggested the workshop was as valuable for the attendees as it was for the researchers, making them feel valued and listened to. The time spent by researchers working on the activity was substantial and not directly funded but a necessary and valuable activity in developing our potential HTA. Our work was informed using the UK Standards for Public Involvement and the Authors and Consumers Together Impacting on eVidencE (ACTIVE) framework. Conclusions PPI can be enormously valuable in both developing and strengthening research proposals. However, further guidance is needed to help researchers recognise the level and type of involvement to use at this early stage, particularly given the large time investment needed to embed meaningful PPI. Patient and Public Contribution Women with a lived experience of POP were involved at every stage of the grant application process; their involvement is documented in full throughout this work.

Pelabuhan Perikanan Pantai (PPP) Lempasing merupakan pelabuhan perikanan terbesar di Lampung. Salah satu kapal yang paling banyak dioperasikan di Pelabuhan Lempasing yaitu mini purse seine. Terdapat berbagai aktivitas di Pelabuhan Lempasing, yaitu pendaratan (bongkar muat) dan pemasaran ikan. Aktivitas bongkar muat di Pelabuhan Perikanan Lempasing merupakan aktivitas utama setelah kapal berlabuh di dermaga. Kecelakaan yang terjadi pada aktivitas bongkar muat dapat menyebabkan kerugian berupa terhambatnya kegiatan. Tujuan dari penelitian ini adalah mengidentifikasi kegiatan dan potensi bahaya aktivitas bongkar muat kapal mini purse seine, menganalisis tingkat kecelakaan dan peluang risiko yang mungkin dapat terjadi, memberikan rekomendasi/alternatif solusi, serta menentukan tindak pengendalian kecelakaan kerja. Metode survei deskriptif HTA (Hierachy Task Analysis) digunakan dalam penelitian ini. Hasil penelitian menunjukkan terdapat 47 risiko kecelakaan pada aktivitas bongkar muat, kegiatan berisiko ringan 36 (77%), menengah terdapat 5 (11%) kegiatan, 2 (4%) kegiatan berisiko berat, dan 4 (9%) kegiatan berisiko fatal. Risiko kecelakaan kerja terbesar terjadi pada kegiatan bongkar, hal ini disebabkan karena individu yang terlibat cukup banyak (sekitar 20 orang) dan aktivitas yang dilakukan cukup berat.

New therapies in a publicly funded healthcare system are first appraised by health technology assessment agencies that provide funding recommendations to the payers. Treatment with Chimeric Antigen Receptor-T cell (CAR-T) therapy is revolutionizing the management of patients with relapsed/refractory aggressive B-cell lymphoma by providing an effective alternative to the standard of care. Yet, the implementation of CAR-T treatment has a substantial impact on the healthcare system due to its high cost, complex manufacturing process, and requirement for highly specialized services and expertise. CAR-T Cells, as a “living drug”, are fundamentally different from usual medications, and their approvals and funding recommendations pose unique challenges to the health technology agency. In this paper, we explore the specific challenges that face the health technology agencies in reviewing reimbursement recommendations for CAR-T therapy. We take a Canadian perspective and use CAR-T treatment of relapse/refractory aggressive B-cell lymphoma as an example.

Artificial intelligence is a new field that uses computers to act like human intelligence. AI is going to make big changes in healthcare in the next few years and will become much bigger in the next decades. Artificial intelligence is expected to help medical centers work better and efficiently, diagnose diseases accurately, and create treatment plans. Artificial Intelligence has been used in health care fields with some success (1). Even though we have made a lot of progress using artificial intelligence for medical research, there are still differences between what the computers can do and what we need for real-life health care situations (2). The fast growth of AI technology can be used in hospitals to improve how they treat patients. This could make a big difference in how healthcare is delivered. It is important to write down and share information about how AI is used in hospitals, so that healthcare workers have the right tools and know-how to use it properly (3). To make better decisions, we need to evaluate how AI can change our healthcare system. Considering how important AI could be in many areas, we should look at more than just how well it works and how much it costs. We need to think about its overall value in the world and how it can help people in their daily lives (4). Due to the increasing importance of the use and application of artificial intelligence in the field of health technology assessment and evidence-based policymaking, especially after Covid-19 pandemic, “health technology assessment (HTA) in action journal” decided to invite researchers and experts to help to develop this knowledge by publishing specialized papers in this field. It is hoped that the published articles can provide up-to-date results in this field. The editor wants to thank all the authors who submitted their work to the special issue of the "HTA in Action journal" Also, thanks to all the reviewers for working hard to review the articles and help the editors make the final decision.

Introduction. La revue annuelle de la qualité des produits (RAQP), exigence internationale pour les formes finies et les substances actives, est particulièrement scrutée lors d’inspections et d’audits. Elle s'apparente à une validation rétrospective continue permettant de détecter toute dérive et de s’assurer de la fiabilité des procédés et de la qualité des produits. La RAQP du Gardénal 50mg (Phénobarbital) fabrique sur le site de Dakar en boite de 30 comprimes a porté sur cinq (5) lots Matériels et méthodes. L’évaluation des OOS/OOT a suivi la procédure CQ PRO 015. Les fiches de déclaration des résultats hors tendance ou non conformes obtenus lors de l’analyse au laboratoire, la description et la cause d’anomalie, le numéro de la déviation correspondante et de suivi des plans d’actions correctives et préventives (CAPA) ont été renseignées. Résultats. Les rendements, de 82,22-88,05%, étaient inférieurs à 98%. L’évaluation du poids moyen a montré des résultats conformes : 86.695-88.245 mg, pour une valeur cible de 87.500 mg. Les temps de désagrégation étaient conformes (60-637 sec), en dessous de la limite maximale de 900 sec. Il en a été de même pour le titre en Phénobarbital avec des résultats conformes de 47,80-50,80 mg/cp (moyenne 49,21 mg/cp). Deux déviations impactant deux lots ont été notées, respectivement, sur le dosage en principe actif pour le lot 2, le temps de désagrégation in process et le test de dissolution pour le lot 3 occasionnant son rejet après retraitement. Le lot 2 a fait l’objet d’une fiche OOT. Discussion. Quatre lots ont été libérés dans les 19 à 50 jours suivants leur fabrication. Huit actions correctives et préventives ont été clôturées à 100 % dans les délais. Seul le lot 3 a été rejeté après retraitement, suite à une dissolution non conforme 61% (limite 75%). Aucun lot n’a fait l’objet de réclamation, de rappel ou de retour. L’étude permet d’affirmer que le produit détient les qualités attribuées : l’évaluation du dosage en principe actif, des paramètres critiques in process, des paramètres de libération disponibles a donné des résultats conformes. Le process de fabrication a été validé.

Introduction. L’étude entre dans le cadre de valorisation des ressources de la pharmacopée africaine. Elle visait le recensement de plantes utilisées dans de la région de Thiès, centre du Sénégal, pour traiter le diabète et l'hypertension artérielle. Matériel et méthodes. Soixante-quinze (75) tradipraticiens, 78,66% d’hommes et 21,34% de femmes également répartis dans les trois départements de la région ont été interrogés sur un questionnaire et les données analysées quantitativement. Résultats. Sur 49 plantes recensées, 36,73% servaient au traitement du diabète, 26,53% à l’HTA et 36,73% pour les deux pathologies. Les plus citées furent : Sclerocarya birrea (18,05%), Combretum micrantum (16,9%), Rauwolfia vomitoria (9,98%), Catharantus roseus (7,32%), Balanites aegyptiaca (7,14%), Lonchocarpus sepium (7,35%) et Combretum glutinosum (6,62%). Les plus fréquemment prescrites étaient les mêmes : Sclerocarya birrea (15,46%), Combretum micrantum (14,9%), Rauwolfia vomitoria (8,39%), Balanites aegyptiaca (7,14%, Catharantus roseus (6%), Lonchocarpus sepium (5,84%) et Combretum glutinosum (5,84%). L’exploitation des feuilles (48,67%), racines (26,22%), fruits (15,44%), écorces (13,33%), graines (10,88%), fleurs (5,92%), bulbes (5,33%) et plantes entières (4,08%) permettait la préparation de décoctés (58,39%), d’infusés (44,28%), de poudres (18,53%), d’huiles (10,74%) essentiellement. Discussion. La position géographique de la région de Thiès, au centre du Sénégal, a permis d’enrôler des interlocuteurs de diverses ethnies pour une plus grande diversité de remèdes. Bien que nettement minoritaires, les tradipraticiennes avaient un important taux de fréquentation. Une convergence des pratiques est à noter en ce qui concerne les ressources utilisées et les préparations. La standardisation des procédures de prélèvement, de transformations et des doses administrées restent des écueils pour l’exploitation rationnelle de la Pharmacopée traditionnelle.

Introdução: Access to drugs for rare diseases constitutes a challenge to healthcare systems, especially those with public funding. The difficulty of conducting robust clinical trials limits the quality of evidence and elevates the cost of development, later translated into the drug’s prices. Thus, it is necessary for the Health Technology Assessment (HTA) agencies to have differentiated criteria for the evaluation of reimbursement recommendations when dealing with such drugs. Objetivos: The objective of this research is to identify and summarize the specific criteria used when evaluating reimbursement recommendations for orphan drugs that are adopted by HTA agencies in countries with different models of public healthcare systems. Material e Método: A comprehensive literature search was performed on the databases PubMed, LILACS, Scopus and Embase up to March 2022. We included any publication type (opinion articles, commentaries, editorials, original articles and reviews) that addressed the criteria used by HTA agencies in countries with public healthcare systems when evaluating reimbursement recommendations for orphan drugs. Resultados: This scoping review summarizes the identified criteria for 18 countries and ranks them within three models of healthcare systems (NHS, NHI and SHI). We identified that NHS countries, such as the UK, Sweden, and Italy, lean towards innovation, the collection of real-world data, and the impact on organizational aspects of the system. Meanwhile, SHI countries, such as Germany, France and the Netherlands, often employ budget ceilings and expedited evaluation processes. All models shared concern over unmet need and disease nature. The 16 included studies range from 2015 to 2022 and the majority consists of reviews of HTA reports and original articles. Discussão e Conclusões: This review provides a good basis for the understanding of each model’s classification and general tendencies when creating differentiated criteria to accommodate and compensate for the lack of evidence and investment around rare diseases.

Objectives: Assess how relevant precedent within the EMA (a key regulator) and NICE (a key HTA body) can inform how such novel medicines targeting asymptomatic conditions can be brought to market from a regulatory and payor perspective. Methods: All phase 2 or phase 3 clinical trials with "asymptomatic", "presymptomatic", "subclinical", "premanifest", or "preclinical" as a condition key word were downloaded from clinicaltrials.gov. Trials were cleaned to identify those for novel medicines with a primary target to treat asymptomatic conditions. EMA and NICE reports for such drugs were downloaded online in December 2021 and analysed to understand how they assessed the evidence. Results: 278 clinical trials were identified. Of those, only 8 had relevant matching reports from EMA and/or NICE. 7 of these 8 received a positive review from EMA, and 4 of these 8 received a positive recommendation from NICE. Those receiving positive recommendations showed or were assumed to show statistically significant survival benefit given a short life expectancy of untreated patients. Conclusions: There is no precedent within EMA or NICE for approving or funding new medicines for asymptomatic patients where survival benefit cannot be established. Additional research is warranted to understand how regulatory and payor agencies can prepare for future generations of innovative medicines.

For many reasons randomized clinical trials today can»t cope with the tasks set by modern health care, which was clearly demonstrated by the COVID-19 pandemic. This gave new impetus to the development of the RWD / RWE. It became obviously that in cases of impossibility, unethical and other reasons, new methods for obtaining high-quality evidence may be effectively used, and later can be used by decision-makers and organizations engaged in the assessment of health technologies.

Abstract Background Few antibiotics have entered the market in recent years despite the need for new treatment options. Some of the challenges of bringing new antibiotics to market are linked to the marketing authorization and health technology assessment (HTA) processes. Research shows great variation in geographic availability of new antibiotics, suggesting that market introduction of new antibiotics is unpredictable. We aimed to investigate regulatory authorities’ and HTA agencies’ role in developing non-financial incentives to stimulate antibiotic research and development (R&D). Methods We conducted individual, semi-structured, stakeholder interviews. Participants were recruited from regulatory authorities (EMA and FDA) and HTA agencies in Europe. Participants had to be experienced with assessment of antibiotics. The data were analyzed using a deductive and inductive approach to develop codes and identify key themes. Data were analyzed using thematic analysis including the constant comparison method to define concepts, and rival thinking to identify alternative explanations. Results We found that (1) interpretation of key concepts guiding the understanding of what type of antibiotics are needed vary (2) lack of a shared approach on how to deal with limited clinical data in the marketing authorization and HTA processes is causing barriers to getting new antibiotics to market (3) necessary adaptations to the marketing authorization process causes uncertainties that transmit to other key stakeholders involved in delivering antibiotics to patients. Conclusions A shared understanding of limited clinical data and how to deal with this issue is needed amongst stakeholders involved in antibiotic R&D, marketing authorization, and market introduction to ensure antibiotics reach the market before resistance levels are out of control. Regulatory authorities and HTA agencies could play an active role in aligning the view of what constitutes an unmet medical need, and direct new economic models towards stimulating greater diversity in the antibiotic armamentarium.

Abstract Background New medicines are increasingly being identified as efficacious across multiple indications. The impact of current pricing and reimbursement policies on launch decisions across these indications remains unclear. Objective This paper, first, maps marketing authorisation and HTA coverage recommendation sequences of multi-indication medicines across Germany, France, England, Scotland, Canada, Australia, and the USA, and, second, evaluates the clinical characteristics, clinical development time and coverage recommendation time of multi-indication medicines, drawing comparisons between the first and subsequent indications of an approved molecule. Methods Medicine approvals by the Food and Drug Administration between 2009–2019 were screened to identify multi-indication products with approved oncology indications. Data on clinical trial characteristics, clinical performance and HTA outcomes were extracted from publicly available regulatory approval and HTA reports. Results Relative to subsequent indications, first indications were more likely to receive conditional marketing authorisation, have an orphan designation, have a single arm phase II pivotal trial and lower MCBS score. Subsequent indications had faster HTA coverage recommendation times in England and Canada. While the majority of first indications received HTA coverage recommendations across all settings, the proportion of subsequent indications with HTA coverage recommendations was lower and uptake varied considerably across settings. Conclusions Discordance in the value of first versus subsequent indications can pose major challenges in systems that define price based on the initial indication. Current pricing and reimbursement systems generate significant fragmentation in the approval and availability of multi-indication products across settings.

Abstract Objectives Dealing with uncertainty is one of the critical topics in health technology assessment. The greater decision uncertainty in appraisals, the less clear the clinical- and cost-effectiveness of the health technology. Although the development of targeted cancer therapies (TCTs) has improved patient health care, additional complexity has been introduced in drug appraisals due to targeting more specific populations. Real-world data (RWD) are expected to provide helpful information to fill the evidence gaps in appraisals. This study compared appraisals of TCTs with those of non-targeted cancer therapies (non-TCTs) regarding sources of uncertainty and reviewed how RWD have been used to supplement the information in these appraisals. Methods This study reviews single technology appraisals (STAs) of oncology medicines performed by the National Institute for Health and Care Excellence (NICE) over 11 years up to December 2021. Three key sources of uncertainty were identified for comparison (generalisability of clinical trials, availability of direct treatment comparison, maturity of survival data in clinical trials). To measure the intensity of use of RWD in appraisals, three components were identified (overall survival, volume of treatment, and choice of comparators). Results TCTs received more recommendations for provision through the Cancer Drugs Fund (27.7, 23.6% for non-TCT), whereas similar proportions were recommended for routine commissioning. With respect to sources of uncertainty, the external validity of clinical trials was greater in TCT appraisals (p = 0.026), whereas mature survival data were available in fewer TCT appraisals (p = 0.027). Both groups showed similar patterns of use of RWD. There was no clear evidence that RWD have been used more intensively in appraisals of TCT. Conclusions Some differences in uncertainty were found between TCT and non-TCT appraisals. The appraisal of TCT is generally challenging, but these challenges are neither new nor distinctive. The same sources of uncertainty were often found in the non-TCT appraisals. The uncertainty when appraising TCT stems from insufficient data rather than the characteristics of the drugs. Although RWD might be expected to play a more active role in appraisals of TCT, the use of RWD has generally been limited.

Abstract Background/objective Sexually transmitted infections (STIs) not only have an impact on the health sector but also the private resources of those affected, their families and other sectors of society (i.e. labour, education). This study aimed to i) review and identify economic evaluations of interventions relating to STIs, which aimed to include a societal perspective; ii) analyse the intersectoral costs (i.e. costs broader than healthcare) included; iii) categorise these costs by sector; and iv) assess the impact of intersectoral costs on the overall study results. Methods Seven databases were searched: MEDLINE (PubMed), EMBASE (Ovid), Web of Science, CINAHL, PsycINFO, EconLit and NHS EED. Key search terms included terms for economic evaluation, STIs and specific infections. This review considered trial- and model-based economic evaluations conducted in an OECD member country. Studies were included that assessed intersectoral costs. Intersectoral costs were extracted and categorised by sector using Drummond’s cost classification scheme (i.e. patient/family, productivity, costs in other sectors). A narrative synthesis was performed. Results Twenty-nine studies qualified for data extraction and narrative synthesis. Twenty-eight studies applied a societal perspective of which 8 additionally adopted a healthcare or payer perspective, or both. One study used a modified payer perspective. The following sectors were identified: patient/family, informal care, paid labour (productivity), non-paid opportunity costs, education, and consumption. Patient/family costs were captured in 11 studies and included patient time, travel expenses, out-of-pocket costs and premature burial costs. Informal caregiver support (non-family) and unpaid help by family/friends was captured in three studies. Paid labour losses were assessed in all but three studies. Three studies also captured the costs and inability to perform non-paid work. Educational costs and future non-health consumption costs were each captured in one study. The inclusion of intersectoral costs resulted in more favourable cost estimates. Conclusions This systematic review suggests that economic evaluations of interventions relating to STIs that adopt a societal perspective tend to be limited in scope. There is an urgent need for economic evaluations to be more comprehensive in order to allow policy/decision-makers to make better-informed decisions.

Real world data (RWD) and real world evidence (RWE) is one of the most rapidly developing areas in the modern health care systems, which is caused by the development of technologies for both collecting and analyzing data, the emergence of new data sources and the formation of needs both by regulatory bodies, health technology assessment (HTA) agencies, and by the clinical and scientific communities. However, at the moment, there is no universal approach to RWD/E generation and application in the context of HTA. Over the past few years, the rate of including RWD/E in the HTA dossier has noticeably increased, but the nature of the existing guidelines and recommendations is rather fragmentary, which might create obstacles in the way of RWD/E study development and submission. The publication is aimed to describe and summarize the main and most rapidly developing spheres of RWD/E applications and analyze the possibilities of using RWD/E in various HTA areas in the world and in the Russian Federation considering the main trends in the further HTA development.

BackgroundEvaluating and integrating digital health technologies is a critical component of a national healthcare ecosystem in the 2020s and is expected to even increase in significance.DesignThe paper gives an overview of international practices on public financing and health technology assessment of digital health technologies (DHTs) in five European Union (EU) countries and outlines recommendations for country-level action that relevant stakeholders can consider in order to support uptake of digital health solutions in Hungary. A scoping review was carried out to identify and gather country-specific classifications and international practices on the financing DHTs in five pioneering EU countries: Germany, France, Belgium, the United Kingdom and Finland.ResultsSeveral frameworks have been developed for DHTs, however there is no single, unified framework or method for classification, evaluation, and financing of digital health technologies in European context. European countries apply different taxonomy, use different assessment domains and regulations for the reimbursement of DHTs. The Working Group of the Hungarian Health Economic Society recommends eight specific points for stakeholders, importantly taking active role in shaping common clinical evidence standards and technical quality criteria across in order for common standards to be developed in the European Union single market.ConclusionSpecificities of national healthcare contexts must be taken into account in decisions to allocate public funds to certain therapies rather than others.

To ensure that limited domestic resources are invested in the most effective interventions, immunization programs in low- and middle-income countries (LMICs) must prioritize a growing number of new vaccines while considering opportunities to optimize the vaccine portfolio, as well as other components of the health system. There is a strong impetus for immunization decision-making to engage and coordinate various stakeholders across the health system in prioritization. To address this, national immunization program decision-makers in LMICs collaborated with WHO to structure deliberation among stakeholders and document an evidence-based, context-specific, and transparent process for prioritization or selection among multiple vaccination products, services, or strategies. The output of this effort is the Country-led Assessment for Prioritization on Immunization (CAPACITI) decision-support tool, which supports using multiple criteria and stakeholder perspectives to evaluate trade-offs affecting health interventions, taking into account variable data quality. Here, we describe the user feedback from Indonesia and Ethiopia, two initial countries that piloted the CAPACITI decision-support tool, highlighting enabling and constraining factors. Potential immunization program benefits and lessons learned are also summarized for consideration in other settings.

Abstract Introduction Drug reimbursement decisions are often made based on a price set by the manufacturer. In some cases, this price leads to public and scientific debates about whether its level can be justified in relation to its costs, including those related to research and development (R&D) and manufacturing. Such considerations could enter the decision process in collectively financed health care systems. This paper investigates whether manufacturers’ costs in relation to drug prices, or profit margins, are explicitly mentioned and considered by health technology assessment (HTA) organisations. Method An analysis of reimbursement reports for cancer drugs was performed. All relevant Dutch HTA-reports, published between 2017 and 2019, were selected and matched with HTA-reports from three other jurisdictions (England, Canada, Australia). Information was extracted. Additionally, reimbursement reports for three cases of expensive non-oncolytic orphan drugs prominent in pricing debates in the Netherlands were investigated in depth to examine consideration of profit margins. Results A total of 66 HTA-reports concerning 15 cancer drugs were included. None of these reports contained information on manufacturer’s costs or profit margins. Some reports contained general considerations of the HTA organisation which related prices to manufacturers’ costs: six contained a statement on the lack of price setting transparency, one mentioned recouping R&D costs as a potential argument to justify a high price. For the case studies, 21 HTA-reports were selected. One contained a cost-based price justification provided by the manufacturer. None of the other reports contained information on manufacturer’s costs or profit margins. Six reports contained a discussion about lack of transparency. Reports from two jurisdictions contained invitations to justify high prices by demonstrating high costs. Conclusion Despite the attention given to manufacturers’ costs in relation to price in public debates and in the literature, this issue does not seem to get explicit systematic consideration in the reimbursement reports of expensive drugs.

In India, there is a renewed emphasis on Universal Health Coverage (UHC). Alongside this, Health Technology Assessment (HTA) is an important tool for advancing UHC. The development and application of HTA in India, including capacity building and establishing institutional mechanisms. We emphasized using the HTA approach within two components of the Ayushman Bharat programme, and the section concludes with lessons learned and the next steps. The UHC has increased the importance of selecting and implementing effective technologies and interventions within national health systems, particularly in the context of limited resources. To maximize the use of limited resources and produce reliable scientific assessments, developing and enhancing national capacity must be based on established best practices, information exchange between different sectors, and collaborative approaches. A more potent mechanism and capacity for HTA in India would accelerate the country’s progress toward UHC.

Introducción: la presión arterial elevada es el principal factor de riesgo de enfermedad global. En la República Dominicana el 31 % de la población padece de hipertensión arterial (HTA) y de este, un 60 % lleva tratamiento médico. Objetivo: determinar los niveles tensionales en individuos no hipertensos entre 18-65 años en la comunidad de Pizarrete Abajo, durante el período agosto-septiembre 2018. Metodología: estudio prospectivo, descriptivo y de corte longitudinal, en el que se analizaron 85 individuos que residen en la comunidad de Pizarrete Abajo, de los cuales 44 resultaron con niveles tensionales alterados. Resultados: el 51.7 % de los encuestados presentó niveles de tensión arterial alterados, sin haber sido anteriormente diagnosticado con hipertensión arterial. Un 24 % de las personas mostró niveles alterados una semana después de la primera toma de presión arterial. Conclusiones: en una muestra de 85 personas se encontraron niveles tensionales alterados en 44 individuos. Se procedió a una segunda toma de presión arterial una semana después de la primera y se observó que un 56.8 % correspondió a valores tensionales elevados, un 20.4 % a valores de HTA estadio 1 y un 18.1 % a valores de HTA estadio 2.

IntroductionNew medicines for the management of type 2 diabetes became available internationally in 2005, yet only in 2018 did the first of these become available in New Zealand. Access to these new medicines in New Zealand is largely dependent on decisions made by the Pharmaceutical Management Agency (PHARMAC). AimThis study sought to describe the decision-making processes to better understand access to new medicines in New Zealand. MethodsWe conducted an analysis of publicly accessible information on therapeutic committee deliberations, prices of medicines and registration and formulary listing dates. ResultsPrices for the new diabetes medicines in New Zealand are lower than comparator countries, but access to them takes longer. DiscussionGiven that knowledge on efficacy, safety and quality is widely available to support decision-making on new medicines, differences in access to them between nations appears to depend on the fourth hurdle of cost. However, we suggest that a rush to market is the norm, that activities of the pharmaceutical industry and regulatory agencies are less transparent than desirable, and that greater focus on availability of safety data is required. Deliberations of PHARMAC therapeutic committees are robust yet protracted. Opportunities to expedite decision-making, as well as resolving inequities, may be worthy of examination.

Introduction: Industrial accidents are among the major problems of today's societies. Studies on accidents indicated that the most important cause of their occurrence was the human factor. Therefore, the purpose of this study was to identify and evaluate human error in the operation of furnace using the human event analysis technique in a petrochemical industry. Methods: This was a qualitative and cross-sectional study which was analyzed by directly observing the activities, interviewing the analysis, and reviewing the documents and instructions, tasks and sub-tasks by Hierarchical task analysis (HTA) method, and the results were provided in HTA charts. In the next step, according to Technique for Human Error Analysis (ATHEANA) instructions, the relevant worksheet was completed. Results: The results indicated that coil breakage or precision instrument connections, improper bypassing of pressure in the paths to rotate the furnace to the atmosphere, or process and leakage of gas lines, flanges and valves whose probability of defect was 0.20, 0.14 and 0.13, respectively, had the highest probability of error. The probability of human error in the operation of the furnace was calculated to be 0.63. Conclusion: Applying corrective measures such as: selecting skilled people, holding training courses, routine monitoring of temperature, pressure measuring equipment, etc. for proper operation of the apparatus and visual monitoring of all furnace parts, including valves, are some of the things with which the likelihood of human error in the operation of the furnaces was reduced.

Banyaknya teknologi kesehatan yang tersedia saat ini, menyebabkan adanya kebutuhan terkait penilaian pada berbagai aspek. Penilaian tersebut biasa disebut dengan Health Technology Assessment (HTA). HTA adalah evaluasi secara sistematis, efek, dan/atau dampak dari teknologi kesehatan yang bertujuan untuk memberikan informasi kepada pembuat kebijakan yang berhubungan dengan teknologi kesehatan, dengan demikian dapat meminimalkan biaya dan mencegah penggunaan teknologi yang diragukan. HTA telah banyak digunakan dalam melakukan penilaian terhadap teknologi kesehatan. Teknologi kesehatan yang dimaksud dapat berupa obat-obatan, terapi, prosedur tindakan medis, maupun alat kesehatan. Proses dan metodologi HTA untuk obat-obatan telah banyak dilakukan sedangkan untuk alat kesehatan masih jarang. Dari beberapa literatur review yang dilakukan sebelumnya, penggunaan kriteria HTA berbeda-beda meskipun objeknya alat kesehatan. Penelitian ini bertujuan untuk mengetahui mengenai kriteria apa saja yang sering digunakan pada HTA untuk alat kesehatan medis. Metode penelitian scoping review pada publikasi HTA sebelumnya (n=392) dilakukan untuk membahas tujuan tersebut dengan menggunakan database Pubmed dan Science Direct. Berdasarkan hasil pencarian literatur dengan menggunakan PRISMA, didapatkan 7 artikel sesuai dengan kriteria inklusi yang berhubungan dengan HTA untuk alat kesehatan dengan subjek yang berbeda, yaitu: alat kesehatan dan HTA berbasis rumah sakit. Kesimpulan dari hasil temuan tersebut, diketahui bahwa kriteria yang sering digunakan dalam HTA untuk alat kesehatan yaitu, ekonomi, keamanan, efektivitas, dan organisasi. Saran penelitian ini adalah hasil kriteria yang didapatkan divalidasi terlebih dahulu oleh ahlinya atau stakeholder.

Abstract Background The Health Technology Assessment (HTA) has encountered different issues and challenges over the last two decades. The main purpose of this research is to review the issues and challenges in high- and middle-income countries through reviewing studies related to the HTA. Methods The HTA area literature of different countries was collected from 2009 to 2020 and analyzed using scoping review, based on Scopus and WoS databases. Results Given the fact that the HTA is practically done in high- and middle-income countries, the results of reviewing the studies and articles of countries reveal that high-income countries seek to increase the participation of stakeholders and enhance the transparency of processes, policy-making, and regulation of the HTA, as well as the systematization of various participant institutions in this area. Middle-income countries, on the other hand, are mostly involved in raising awareness, training and skill development of HTA-related staff, institutionalizing the concept of HTA, and allocating appropriate resources for effective and safe decision-making in their health system. Conclusion The problem of incoordination between stakeholders (participant institutions) in the HTA, and thereby, problems in decision-making were found in many of the studied reports and articles. Thus, one of the useful efforts to be made by different countries to maintain the integrity of this system would be the process of involving all members of this system and the formation of a healthy ecosystem in the HTA.

Introduction: Human error plays a significant role in the occurrence of industrial accidents. Displacement and unloading operations are operations in which the occurrence of human error may lead to plenty of human and financial losses. The present study aimed to investigate the possibility of human error occurrence in overhead crane operators in a Steel Company in Hormozgan, Iran. Material & Methods: This cross-sectional and descriptive study was conducted using the Standardized Plant Analysis Risk-Human Reliability Analysis (SPAR-H) technique. In this study, the job tasks of overhead crane operators were firstly analyzed using the hierarchical task analysis (HTA) technique. Subsequently, the probability of human error in job tasks was assessed using the SPAR-H technique. (Ethic code: IR.ACECR.JDM.REC.1399.005) Findings: Generally, five main tasks and 16 sub-tasks have been analyzed in this study. The highest probability of error was related to the three sub-tasks of longitudinal motion, transverse motion, and high and low motion (0.3975). The lowest probability of error was under the duty of recording shift reports, transmitting information orally, and writing (0.05). The results of this study clearly showed the effect of interdependence on increasing the probability of error occurrence. Discussion & Conclusion: Based on the results of the present study, some preventive measures were proposed to reduce the possibility of human error, including identifying and controlling job stressors, correcting the ergonomic status of crane cabins, preparing work instructions, as well as training and monitoring their proper implementation.

Objectives: The aim of this study was to determine the role of the patient’s point of view when assessing health technologies as well as the patients’ willingness to participate in this process in our country.

In this latest update we discuss the transportability of comparative effectiveness evidence across countries. We highlight results of a survey indicating that European HTA agencies are reluctant to accept real-world data from other countries, review recent benefit assessments indicating a potential softening of a requirement for the use of local real-world data in Germany, and outline a recent review presenting approaches that can correct for a lack of transportability.

OBJECTIVES The most promising medical innovations appear to take a long time to be used in healthcare practice. This research has two main objectives: to understand the factors that limit or promote the adoption of Emerging Technologies (ET) in healthcare and to provide a model for understanding, codifying and improving the current situation. METHODOLOGY A case study is used to shed light on the process of adopting ET in healthcare between a teaching hospital (adopter) and a manufacturer of innovation (producer). The primary data, investigated through thematic analysis and literary evidence, consists of nine semi-structured interviews from ET experts on the side of the adopter and the producer. The Attitude, Decision, and Implementation (ADI) decision-making model is used as the theoretical lens for this study. This framework is a modification of the process described in the theory of Diffusion of Innovation (DoI) (Rogers, 2003) adapted to the peculiarities of the healthcare environment. RESULTS From the analysis of the interviews, it is clear that innovation in medicine has several limitations (financial resources, free time of healthcare professionals, correct identification and know-how of the professionals involved, communication, healthcare resistance) against few facilitators (Innovation Champions, appropriate marketing, medical conferences). This study also highlights the absence of a shared and codified model for the adoption of innovation in medicine. DISCUSSION The impediments and the facilitating factors that emerged in the analysis of the primary data have been confirmed in literature. Furthermore, the primary data analysis with literary evidence supports the ADI model as a possible decision-making process for the adoption of ET in healthcare. Its three cyclical phases: the creation of an attitude (A) towards the ET, the decision to continue the collaboration between the parties (D), and the implementation of innovation (I) are sustained by primary data and literary evidence. CONCLUSIONS The ADI model could improve the efficiency and pace of adoption of ET in healthcare by limiting barriers, exploiting facilitating factors and building a beneficial relationship between users and producers of ET.

BackgroundThe Egyptian healthcare system is currently in the early phase of health technology assessment (HTA) implementation. The aim of this study is to propose an implementation roadmap based on the national healthcare system status.MethodsA survey was conducted among Egyptian healthcare sector decision-makers to assess the current and future (preferred) HTA implementation status in Egypt based on a widely used international scorecard methodology. Subsequently, interviews were conducted with experts representing middle- and top-tier management in the Egyptian healthcare system to interpret the survey results and recommend specific actions.ResultsExperts recommended more capacity-building programs for HTA and health economics. Additionally, they proposed establishing HTA units in separate healthcare authorities and merging them into a single central HTA unit in the long term. Regarding the scope of implementation, experts recommended commencing with the assessment of innovative pharmaceuticals, and thereafter, expanding the scope to cover all health technologies in the long term. Additionally, they recommended using innovative tools such as “multi-criteria decision analysis (MCDA)” for tendering, and “managed entry agreements” for reimbursement decisions. Local burden of diseases and costing studies were also recommended to facilitate the implementation of HTA.ConclusionExperts agreed that several actions are required for successful HTA implementation in Egypt, including coordination between HTA bodies, application of an explicit MCDA framework, and strengthening of local evidence generation. To implement these actions, investment in technical capacity-building is indispensable. Most experts favored using multiple and soft cost-effectiveness thresholds. Efforts should be made to publish HTA submission guidelines and timelines of the processes.

Pharmaceutical industry investments in Italy must be supported with a reform process that promotes simplifications to generate an attractive ecosystem that can enhance innovation. Key actions include facilitating the start-up of clinical trials, promoting public-private partnerships to support technology transfer, integrating data infrastructures to overcome the logic of silos, expanding programs for prevention and early identification of diseases, simultaneous reimbursement for therapy and diagnostic testing, and launching a structural program for early access to therapies. The total contribution of the sector in the last 10 years to the Italian economy was € 315 bn. Looking at employment (67 thousand people employed in the sector in 2020), it is possible to estimate about 280 thousand jobs activated in Italy just in 2020. An Altems study quantified a leverage effect of 2.77 in terms of benefits to the Italian NHS from direct investment in clinical research, which means that for every € 1 invested by companies in clinical trials, € 1.77 of additional savings were generated for the NHS. Applying the multiplier on R&D investments of the entire pharmaceutical sector in 2020 (€ 1.6 bn), we could estimate approximately € 4.4 bn in benefits for the NHS. In addition to these benefits there are the noneconomic benefits of clinical research, including early access for patients to innovative therapies, resulting in improved clinical outcomes and quality of life for patients and caregivers in general.

ABSTRACT Three directly acting antivirals (DAAs) demonstrated substantial reduction in COVID-19 hospitalizations and deaths in clinical trials. However, these agents did not completely prevent severe illness and are associated with cases of rebound illness and viral shedding. Combination regimens can enhance antiviral potency, reduce the emergence of drug-resistant variants, and lower the dose of each component in the combination. Concurrently targeting virus entry and virus replication offers opportunities to discover synergistic drug combinations. While combination antiviral drug treatments are standard for chronic RNA virus infections, no antiviral combination therapy has been approved for SARS-CoV-2. Here, we demonstrate that combining host-targeting antivirals (HTAs) that target TMPRSS2 and hence SARS-CoV-2 entry, with the DAA molnupiravir, which targets SARS-CoV-2 replication, synergistically suppresses SARS-CoV-2 infection in Calu-3 lung epithelial cells. Strong synergy was observed when molnupiravir, an oral drug, was combined with three TMPRSS2 (HTA) oral or inhaled inhibitors: camostat, avoralstat, or nafamostat. The combination of camostat plus molnupiravir was also effective against the beta and delta variants of concern. The pyrimidine biosynthesis inhibitor brequinar combined with molnupiravir also conferred robust synergistic inhibition. These HTA+DAA combinations had similar potency to the synergistic all-DAA combination of molnupiravir plus nirmatrelvir, the protease inhibitor found in paxlovid. Pharmacodynamic modeling allowed estimates of antiviral potency at all possible concentrations of each agent within plausible therapeutic ranges, suggesting possible in vivo efficacy. The triple combination of camostat, brequinar, and molnupiravir further increased antiviral potency. These findings support the development of HTA+DAA combinations for pandemic response and preparedness. IMPORTANCE Imagine a future viral pandemic where if you test positive for the new virus, you can quickly take some medicines at home for a few days so that you do not get too sick. To date, only single drugs have been approved for outpatient use against SARS-CoV-2, and we are learning that these have some limitations and may succumb to drug resistance. Here, we show that combinations of two oral drugs are better than the single ones in blocking SARS-CoV-2, and we use mathematical modeling to show that these drug combinations are likely to work in people. We also show that a combination of three oral drugs works even better at eradicating the virus. Our findings therefore bode well for the development of oral drug cocktails for at home use at the first sign of an infection by a coronavirus or other emerging viral pathogens.

Background: Requiring access to sensitive data can be a significant obstacle for the development of health models in the Health Economics & Outcomes Research (HEOR) setting. We demonstrate how health economic evaluation can be conducted with minimal transfer of data between parties, while automating reporting as new information becomes available. Methods: We developed an automated analysis and reporting pipeline for health economic modelling and made the source code openly available on a GitHub repository. The pipeline consists of three parts: An economic model is constructed by the consultant using pseudo data. On the data-owner side, an application programming interface (API) is hosted on a server. This API hosts all sensitive data, so that data does not have to be provided to the consultant. An automated workflow is created, which calls the API, retrieves results, and generates a report. Results: The application of modern data science tools and practices allows analyses of data without the need for direct access – negating the need to send sensitive data. In addition, the entire workflow can be largely automated: the analysis can be scheduled to run at defined time points (e.g. monthly), or when triggered by an event (e.g. an update to the underlying data or model code); results can be generated automatically and then be exported into a report. Documents no longer need to be revised manually. Conclusions: This example demonstrates that it is possible, within a HEOR setting, to separate the health economic model from the data, and automate the main steps of the analysis pipeline.

Abstract The goal of this work is to present i* framework as alternative to support design decisions in the Human Computer Interaction Area (HCI), mainly, in the process of task analysis. We show i* framework a modeling language suitable for an early phase of software system modeling to understand the problem domain, also, that provides methods to represent tasks, as well as support design decisions the development that satisfies the process requirements from the beginning until the end. To achieve our goal, approaches addressed to analyze notations aimed at task analysis and their representation. Finally, we present suggestions for improvement in the i* framework through a proposed extension for refining task model, instantiating examples and justifying our proposal through peer review.

Although the access to innovative medicines is usually informed by health technology assessment reports, underlying uncertainties in HTA may delay such access. Managed Entry Agreements may provide a promising solution to handle such uncertainty through focusing on value for information. Notably, majority of MEA designs include volume-based pricing and not outcome-based pricing strategies – which is an optimal access pathway for rare disease drugs. This is attributed to transparency issues regarding MEA content. This article proposes a framework which may be helpful which considering MEA within the context of low-and-middle-income countries using an interdisciplinary approach. This included needs assessment, value framework, governance, and choice of outcomes.

Abstract Background Health technology assessment (HTA) agencies have an important role in the evaluation and approval of new technologies. They determine their value within a health system so to promote equitable, quality care with available healthcare resources. Many HTA agencies have some mechanism for involving patients in their processes, but there is great variability and an absence of comprehensive, robust practices for involvement. The accelerating pace of medical innovation creates a need to improve the depth and breadth of patient involvement in the HTA process. Main body In this ‘Call to action’, we present ideas from three HTA expert commentaries calling for collaborative learning and to share innovative ideas for changes in HTA. We also draw on examples of HTA agencies creatively pursuing this goal. We propose a ‘Call to action’ for HTA stakeholders to undertake serious dialogue with patient advocates aimed at creating shared goals. HTA agencies can use these goals to ensure meaningful patient involvement at every step of the HTA process. Five elements are explored. In ‘Recognizing the value of shared purpose’, we highlight examples of HTA agencies that have patients working in partnership with medical practitioners and HTA staff. Results include improved processes that instil confidence. ‘Committing to patient involvement as part of HTA culture’ highlights several initiatives aimed at changes in HTA organisational culture to be more inclusive of patients. In ‘Aligning patient and HTA goals’ we cite work in Belgium and New Zealand which places a greater emphasis on quality of life rather than life expectancy and cost-effectiveness. By ‘Integrating patient involvement at every step of the HTA process’ patients can make vital contributions at every stage of the HTA process. We provide two examples of where HTA agencies have successfully involved patients early in the process in order to broaden the scope of evaluations. ‘Developing a common language and working together’ can support transformative dialogue through ‘unified language’. Conclusion The authors of this commentary ask that agencies and stakeholders involved in HTA take up this call to work together for visionary and transformative elevation of the voice of patients in HTA worldwide.

A health technology assessment (HTA) is commonly defined as a multidisciplinary approach used to evaluate medical, social, economic, and ethical issues related to the use of a health technology in a systematic, transparent, unbiased, robust manner. To help inform HTA recommendations, the surveillance of social media platforms can provide important insights to the clinical community and to decision makers on the effectiveness and safety of the use of health technologies on a patient. A scoping review of the published literature was performed to gain some insight on the accuracy and automation of sentiment analysis (SA) used to assess public opinion on the use of health technologies. A literature search of major databases was conducted. The main search concepts were SA, social media, and patient perspective. Among the 1,776 unique citations identified, 12 studies that described the use of SA methods to evaluate public opinion on or experiences with the use of health technologies as posted on social media platforms were included. The SA methods used were either lexicon- or machine learning-based. Two studies focused on medical devices, three examined HPV vaccination, and the remaining studies targeted drug therapies. Due to the limitations and inherent differences among SA tools, the outcomes of these applications should be considered exploratory. The results of our study can initiate discussions on how the automation of algorithms to interpret public opinion of health technologies should be further developed to optimize the use of data available on social media.

Objective: Core outcome sets (COS) are an agreed standardised collection of outcomes created with representation from all key stakeholders (such as patients, clinicians, researchers), which should be reported as a minimum for all trials in that corresponding clinical area. There has been little research investigating the use of core outcomes in Health technology assessments (HTAs) and none in non-oncology HTAs. This study aimed to assess the similarity between COS and HTA outcomes. Methods: Ten COS published between 2015 and 2019 were selected, with patient participation taken as a proxy measure for a high quality COS. The INAHTA database was used as a source to identify relevant HTAs, which were accessed through the hyperlinks provided. Outcomes selected for these assessments were categorised as either a specific, partial or no match compared to the COS. An additional cohort of non-oncology HTAs published between 2019 and 2021 were identified from the NICE website and compared against a relevant COS. Results: Six hundred and fifty-one HTAs were matched to the ten COS areas, of which 119 were reviewed. Of a possible 1318 core outcome matches, there were 562 (43%) matches, 413 (31%) specific and 149 (11%) partial. NICE HTA matches against corresponding COS ranged from 44% to 100%, with a total of 78% (73/94) matches, 57 (61%) specific and 16 (17%) partial. Conclusion: Further work is required to promote the awareness and implementation of COS within HTAs. The degree of matching between COS and NICE HTA outcomes is encouraging, demonstrating acceptance of COS by HTA producers.

INTRODUCTION: In this study, it was aimed to evaluate the views and opinions of the health administrators, who are working in the private hospitals in Konya province centre about health technology assessment and applications. METHODS: Qualitative research methods have been used in research, and phenomologic method is preferred. The research was conducted using face-to-face interviews with health administrators working in private hospitals serving in the centre of Konya and accepting to participate in the research. A semi-structured interview form was used as a data collection tool in the survey. RESULTS: As a result of the analysis, sub-themes were created under the headings of health technologies, health technology assessment, health technology assessment applications in Turkey, health technology assessment approach of private hospitals, establishment of health technology assessment department, health technology assessment applications of the respondents' hospitals and future position of health technology assessment. And the findings under the created themes are interpreted. DISCUSSION AND CONCLUSION: The managers had various information about health technology assessment applications however it was limited. In this respect, it would be beneficial to provide a general education about health technology assessment in order to increase awareness and application of health technology assessment practices.