Opportunities and Challenges in Cross-Country Collaboration: Insights from the Beneluxa Initiative.

National pricing and reimbursement agencies face growing challenges with complex health technologies, prompting European policy advancements. Beneluxa is a cross-country collaboration involving Belgium, the Netherlands, Luxemburg, Austria, and Ireland that aims to address sustainable access to medicines. In view of the soon-to-be-implemented EU HTA Regulation, insights and experiences from stakeholders with Beneluxa cross-country collaboration could provide possible transferable learnings. Therefore, this research aims to (i) identify the opportunities and challenges faced by Beneluxa, (ii) gather insights from stakeholders, namely (possible) applicants and policymakers, within and beyond Beneluxa on the initiative and broader cross-country collaboration principles, and (iii) transfer these insights into learnings and recommendations in anticipation of the full implementation of the new HTA Regulation. Fifteen semi-structured interviews were conducted with industry and European HTA/policy stakeholders. The principal challenges discussed by stakeholders encompass hesitancy from the industry toward Beneluxa assessments, which were attributed to procedural and timeline uncertainties, legislative framework ambiguity, and challenges in terms of industry's internal organization. Another challenge highlighted is the resource-intensive nature of the procedure due to diverse approaches among member states. In addition, industry stakeholders mentioned limited communication and procedural complexity. Despite challenges, both stakeholder groups recognized important opportunities for cross-country collaboration. Transferable insights for future cross-country collaboration include transparent communication, clear legislative embedding, internal industry restructuring to facilitate joint HTAs, and member state support for conducting collaborative assessments. The study underscores diverging views among stakeholders on cross-country collaboration's potential to support HTA and the market access of complex health technologies. While acknowledging benefits, there still are challenges, including industry hesitancy, emphasizing the need for transparent communication and clear guidance in the evolving EU HTA landscape.

Creating win-win-win situations with managed entry agreements? Prioritizing gene and cell therapies within the window of opportunity.

Outcome-based reimbursement models are gaining attention for managing the clinical uncertainties and financial impact of gene and cell therapies. Little guidance exists on how such models can create win-win-win situations, benefiting health-care payers, health-technology developers and patients. Our innovative approach prospectively prioritizes therapies for which a 'window of opportunity' might occur through the analysis of health-technology assessments and product characteristics. Within this window, one size does not fit all, and depending on the extent of clinical uncertainty and potential added benefit levels, different win-win-win situations exist in the United States, the United Kingdom and the Netherlands. Dutch Horizon scanning data prioritized etranacogene dezaparvovec (Hemgenix) and mozafancogene autotemcel for their potential to benefit from outcome-based reimbursement models. These insights extend beyond gene and cell therapies, and could help to provide sustainable health care and patient access to innovative therapies.

[EMERGINCaRE, the horizon scanning system for anticancer drugs of the French National Cancer Institute]. / EMERGINCaRE, le dispositif d'horizon scanning dédié aux médicaments anticancéreux développé par l'Institut national du cancer.

INTRODUCTION: In a context of intensive clinical development and innovation in oncology, the French National Cancer Institute has developed a horizon scanning focused on emerging anticancer drugs since 2019. This tool aims to provide further insight to national authorities responsible of the access to medicines and policymakers. METHODS: EMERGINCaRE is based on an annual cycle initiated by the identification of clinical developments of interest from a database, one to three years prior European marketing authorization. Clinical developments are ranked and prioritized using a scoring approach. Scores are based on public information about developments collected by the Institute and on the evaluation carried out by clinicians who were asked to analyse and identify the most impacting drugs. A national steering committee prioritizes several high-score developments each year. RESULTS: Seventy-five developments were analysed during the 2023 cycle. Among these developments, 50 are related to drugs for solid tumors and 25 for hematological malignancies. At the end of this cycle, six developments, including two concerning Advanced Therapy Medicinal Products, were prioritized. Half of these prioritized developments evaluate a drug for a poor prognosis cancer. DISCUSSION: Among the developments evaluated with a high clinical impact score, some drugs were finally approved for the clinical situation concerned. As first public Horizon Scanning in France, the methodology of EMERGINCaRE has been refined and deadlines have been optimized to provide annually the information generated by this system to interested public institutions.

A Future European Scientific Dialogue Regulatory Framework: Connecting the Dots.

The regulatory framework of the European Union (EU) offers multiple and valuable options for Scientific Advice (SA). However, at a time of increasing scientific complexity and global competition, navigating the SA landscape may be challenging. Such challenges are related to the technicalities of the framework itself, as well as to fundamental changes in the development of promising therapeutics. This article provides an overview of these challenges and reflects on the ways in which the already available SA options could be consolidated and optimized for building an integrated, easy-to-navigate process. The key elements of the proposal are improved orientation and navigation support, a simplified process of managing parallel interactions with multiple bodies, competitive SA timelines, consistency and harmonization across stakeholders, a strengthened horizon scanning to increase network preparedness, and a mechanism for building an institutional memory. The article builds on ongoing dialogues driven by the European Medicines Agency and the European Medicines Regulatory Network, and contributes the viewpoint of the European Federation of Pharmaceutical Industries on the ways in which the EU SA framework needs to evolve to provide effective Scientific Dialogue throughout the medicine lifecycle. The article is timely because of the current discussion on the future Scientific Dialogue framework and may inform forthcoming legislative changes in the draft General Pharma Legislation revision and how they are practically implemented.

Mapping horizon scanning systems for medical devices: similarities, differences, and lessons learned.

OBJECTIVES: This article presents the mapping of horizons scanning systems (HSS) for medical devices, conducted by the Medical Devices Working Group of the International Horizon Scanning Initiative (IHSI MDWG). It provides an overview of the identified HSS, highlights similarities and differences between the systems, and lessons learned. METHODS: Potentially relevant HSS were identified through literature searches, scan of an overview of EuroScan members, and input from the IHSI MDWG members. Structured information was collected from organizations that confirmed having an HSS for medical devices. RESULTS: Sixteen initiatives could be identified, of which 11 are currently ongoing. The purposes of the HSS range from raising awareness of trends and new developments to managing informed decisions on innovative health services in hospitals. The time-horizon is most often 3 years up to a few months before market entry. Three models of identification of new technologies crystallized: a reactive (stakeholders outside HSS inform), a pro-active (actively searching multifold sources), and a hybrid model. Prioritization is often conducted by separate committees via scoring or debate. The outputs focus either on in-depth information of single technologies or on a class of technologies or on technologies in specific disease areas. CONCLUSIONS: The identified HSS share the common experience that horizon scanning (HS) for medical devices is a resource-intensive exercise that requires a dedicated and skilled team. Insights into the identified HSS and their experiences will be used in the continued work of the IHSI MDWG on its proposal for an IHSI HSS for medical devices.

Greening procurement: Exploring evolving practices in an Irish university.

Including green criteria in the public procurement of goods and services requires increased expertise, new methodologies, more significant monitoring efforts and more support towards innovation. These added complexities influence procurement professionals and their everyday practices. This article explores the under-researched issue of practitioner-led beliefs, attitudes, and their accounts of Green Public Procurement (GPP). We delve into a qualitative case study of University College Cork (UCC) in Ireland to explore the journey of procurement professionals in introducing GPP across the various sectors and departments of the university. We draw from interviews, a horizon scanning workshop, and secondary materials to capture and build on the expertise of a broad range of staff in UCC with experience in this area. We use this collective viewpoint to make sense of GPP and to position such views relative to ongoing policy priorities, looking at past, present, and future outlooks. The research shows that efforts have been made to introduce green criteria in new tenders. These gradually became wider opportunities to develop competencies, skills, and stimuli to implement more impactful strategies. The research also shows underdeveloped practices around supporting innovation, monitoring, and post-award evaluation. Overall, the paper offers a unique perspective based on the day-to-day practice of public procurement practitioners. While the case study is geographically bound and therefore presents difficulties in replicating findings, it provides a new lens for researching GPP adoption through interaction with practitioners.

Towards greater impact in health technology assessment: System dynamic approach for new and emerging technologies in Iran.

PURPOSE: As classical health technology assessment models fail to predict the complexities of related impacts, the application of modeling techniques such as systems dynamics simulation (SD) is essential. This study aimed to develop an SD model to predict the outcomes of access to a new medicine in Iran. METHODS: This study extracted the important and influential variables in providing access to new pharmaceutical technologies by comprehensively reviewing previous research and combining the technical knowledge of experts in this field. The variables were incorporated into the systems thinking framework and modeled using dynamic systems tools, followed by simulation and testing in VENSIM. The model was piloted for deferoxamine and deferasirox in thalassemia. Various tests were used to evaluate the validity and reliability of the model. The model was designed for a ten-year horizon (2018-2028) for medicines selected as the pilot. RESULTS: The variables extracted from the panel of experts encompassed the primary and short-term impacts of access to newly emerged medicine and long-term impacts regarding the economy, health, and society. After modeling, the leverage points presented for the problem with the greatest impact or effectiveness in access to new medicine included the policy determining the amount of medicine supply, the import and production of medicine, the prevalence and incidence of disease, insurance coverage, and treatment adherence. CONCLUSION: The SD models allow the researchers to evaluate the efficiency and health outcomes of a new pharmaceutical more precisely in the health system in Iran.

Closing the loop between horizon scanning and health technology assessment - an overview of topics submitted for appraisal in England.

OBJECTIVES: Horizon scanning for health technology appraisal (HTA) in England involves topic notification to the National Institute for Health and Care Excellence (NICE) via technology briefings. This activity is undertaken by the Innovation Observatory with submission timelines designed to ensure that HTA decisions align with regulatory approval time. In this paper, we aimed to track and assess the progression and current status of the topics notified for HTA and provide a descriptive analysis of these topics. METHODS: Technology briefings were mapped from submission to NICE technology appraisal/highly specialized technologies recommendations from April 2017 until October 2021. This was done using a combination of searches on Google and NICE website, searching a downloadable spreadsheet containing NICE topic selection decisions, and querying NICE Topic Selection team. Analysis was undertaken regarding type of indications and interventions of submitted topics and published guidance. RESULTS: Six-hundred and ninety-three topics entered the NICE scoping process, of which 94 percent were prioritized. As of November 2021, approximately 39 percent of prioritized topics were in scoping/in progress, 31 percent were proposed/completed, 20 percent were suspended/terminated, and 4 percent were referred back to Innovation Observatory (IO) for further monitoring. CONCLUSIONS: Our work demonstrates that horizon scanning for HTA is a complex and time-intensive process. Timelines and progress through HTA is challenging due to the growing number of innovative medicines, significant uncertainties, and limited transparency in clinical development and regulatory pathways. A better understanding of clinical trials and regulatory requirements may help eliminate some of this uncertainty and improve timely HTA.

Horizon scanning: what next for bovine TB control in England?

England is currently in year nine of its 25-year strategy to achieve TB freedom. This talk will speculate on what new tools and approaches could be introduced in the future to help us achieve our goal.Using Defra's response to the independent review of its TB programme as a starting point and building on the plenary talk by the UK CVO, I will look at some opportunities that could arise under the different aspects of the response.Firstly, how best to help farmers reduce their herd TB risk through better informed purchasing decisions will be considered, including looking at the recent publication of the health ratings for every cattle herd in England.Cattle vaccination, and its associated DIVA test could be the biggest change in Tb control in England in many years while the related development of a molecularly defined tuberculin which could become the default testing reagent.Advances in whole genome sequencing will allow us to sequence the genome of M.bovis isolated from most infected herds in England and these data could unlock a variety of opportunities from tracing the spread of infection to ground-truthing the efficacy of testing and epidemiological assessment of breakdowns.Finally, the move to vaccination as the primary way of controlling TB in badgers with culling used very sparingly will be considered using a case study of how a targeted badger cull successfully removed infection from an area in Cumbria and enabled the switch to vaccination.

The launch of the EU Health Emergency Preparedness and Response Authority (HERA): Improving global pandemic preparedness?

The crowded global health landscape has been joined by the European Union Health Emergency Preparedness and Response Authority (HERA). HERA will assume four broad areas of responsibility: horizon scanning for major health threats; research and development; support for capacity to manufacture drugs, vaccines, and equipment; and procuring and stockpiling key medical countermeasures. In this Health Reform Monitor article, we outline the reform process and describe HERA's structure and responsibilities, explore issues that arise from the creation of this new organisation, and suggest options for collaboration with existing bodies in Europe and beyond. The COVID-19 pandemic and other infectious disease outbreaks have shown the need to treat health as a cross-border issue, and there is now a broad consensus that greater direction and coordination at the European level is needed. This ambition has been matched with a considerable increase in EU funding to tackle cross-border health threats, and HERA can be used to deploy this funding in an effective manner. Yet this is contingent upon clearly defining its role and responsibilities vis-à-vis existing agencies to reduce redundancies.

Horizon Scanning in Tissue Engineering Using Citation Network Analysis.

BACKGROUND: Establishing a horizon scanning method is critical for identifying technologies that require new guidelines or regulations. We studied the application of bibliographic citation network analysis to horizon scanning. OBJECTIVE: The possibility of applying the proposed method to interdisciplinary fields was investigated with the emphasis on tissue engineering and its example, three-dimensional bio-printing. METHODOLOGY AND RESULTS: In all, 233,968 articles on tissue engineering, regenerative medicine, biofabrication, and additive manufacturing published between January 1, 1900 and November 3, 2021 were obtained from the Web of Science Core Collection. The citation network of the articles was analyzed for confirmation that the evolution of 3D bio-printing is reflected by tracking the key articles in the field. However, the results revealed that the major articles on the clinical application of 3D bio-printed products are located in clusters other than that of 3D bio-printers. We investigated the research trends in this field by analyzing the articles published between 2019 and 2021 and detected various basic technologies constituting tissue engineering, including microfluidics and scaffolds such as electrospinning and conductive polymers. The results suggested that the research trend of technologies required for product development and future clinical applications of the product are sometimes detected independently by bibliographic citation network analysis, particularly for interdisciplinary fields. CONCLUSION: This method can be applied to the horizon scanning of an interdisciplinary field. However, identifying basic technologies of the targeted field and following the progress of research and the integration process of each component of technology are critical.

A Horizon Scan to Support Chemical Pollution-Related Policymaking for Sustainable and Climate-Resilient Economies.

While chemicals are vital to modern society through materials, agriculture, textiles, new technology, medicines, and consumer goods, their use is not without risks. Unfortunately, our resources seem inadequate to address the breadth of chemical challenges to the environment and human health. Therefore, it is important we use our intelligence and knowledge wisely to prepare for what lies ahead. The present study used a Delphi-style approach to horizon-scan future chemical threats that need to be considered in the setting of chemicals and environmental policy, which involved a multidisciplinary, multisectoral, and multinational panel of 25 scientists and practitioners (mainly from the United Kingdom, Europe, and other industrialized nations) in a three-stage process. Fifteen issues were shortlisted (from a nominated list of 48), considered by the panel to hold global relevance. The issues span from the need for new chemical manufacturing (including transitioning to non-fossil-fuel feedstocks); challenges from novel materials, food imports, landfills, and tire wear; and opportunities from artificial intelligence, greater data transparency, and the weight-of-evidence approach. The 15 issues can be divided into three classes: new perspectives on historic but insufficiently appreciated chemicals/issues, new or relatively new products and their associated industries, and thinking through approaches we can use to meet these challenges. Chemicals are one threat among many that influence the environment and human health, and interlinkages with wider issues such as climate change and how we mitigate these were clear in this exercise. The horizon scan highlights the value of thinking broadly and consulting widely, considering systems approaches to ensure that interventions appreciate synergies and avoid harmful trade-offs in other areas. We recommend further collaboration between researchers, industry, regulators, and policymakers to perform horizon scanning to inform policymaking, to develop our ability to meet these challenges, and especially to extend the approach to consider also concerns from countries with developing economies. Environ Toxicol Chem 2023;42:1212-1228. © 2023 Crown copyright and The Authors. Environmental Toxicology and Chemistry published by Wiley Periodicals LLC on behalf of SETAC. This article is published with the permission of the Controller of HMSO and the King's Printer for Scotland.

Food fraud and mitigating strategies of UK food supply chain during COVID-19.

Covid-19 had shown the vulnerability of the food supply chain and fraudsters may take advantage of the pandemic whilst the population needed a continuous supply of safe and quality food. The lack of monitoring and policing in the food supply chain may encourage fraudsters to upscale their operations. Previous studies had warned of a surge in fraudulent products due to COVID-19. This raised the question on whether food fraud had increased during the pandemic? This study aims to investigate food fraud during COVID-19 and how the food supply chain develops mitigating strategies against fraudulent activities. A mixed-method approach including survey and semi-structured interviews were conducted among UK food businesses. Two hundred and two agri-food businesses responded to the survey and 15 semi-structured interviews were conducted. The majority of the food businesses did not experience an increase of food fraud activities during COVID-19. Two thematic domains and ten sub-themes were identified from the data set. There was a heightened sense of anticipation and preparation for increased fraudulent activities during the pandemic. The main risk mitigating strategies included horizon scanning; developing and maintaining supplier relationship and assurance; understanding product characteristics, testing capabilities, conducting vulnerability assessments and training. Practical and cost-effective strategies for small and medium food businesses were recommended. This is the first empirical study on food fraud and mitigating strategies of the UK food supply chain during the pandemic. Our findings provide evidence for informing the policies and practices of the food regulatory authorities as well as best practices to protect the UK food supply chain against food fraud during exogenous shocks like COVID-19.

A rapid priority setting exercise combining existing, emergent evidence with stakeholder knowledge identified broad topic uncertainties.

OBJECTIVES: The project aimed to rapidly identify priority topic uncertainties as a first step to identify future systematic review questions of pertinence to key international fecal incontinence (FI) stakeholders (patients, carers, health care professionals, policy makers and voluntary, community, or social enterprise representatives). The paper's aim is to share our methods, experience, and learning with other groups planning to deliver a rapid priority setting exercise. STUDY DESIGN AND SETTING: An evidence gap map incorporated three evidence streams: emerging evidence identified through horizon scanning; existing evidence identified through systematic searches of bibliographic databases; and FI stakeholder insights collected through an international survey. The evidence gap map was presented during an online workshop with stakeholders, where they shared their expertize to expand, refine, and rank topic uncertainties using ideation techniques, focus group discussions, consensus techniques, and online polling. RESULTS: The multistep methods used to deliver this priority setting exercise resulted in identification of broad priority topic uncertainties. The methods appear to have high acceptability and engagement with participants but await full evaluation. CONCLUSION: This project successfully followed robust methodology, building upon frameworks from published priority setting and evidence gap mapping projects while incorporating strong patient and public involvement components.

Implementing Horizon Scanning as a tool for the strategic development of regulatory guidelines for nanotechnology-enabled health products.

Strategic regulatory development is essential to ensure that new innovations in nanotechnology-enabled health products (NHPs) successfully reach the market and benefit patients. Currently, the lack of specific regulatory guidelines for NHPs is considered one of the primary causes of the so-called "valley of death" in these products, impacting both current and future advancements. In this study, we have implemented a methodology to anticipate key trends in NHP development and compare them with the current regulatory landscape applicable to NHPs. This methodology relies on Horizon Scanning, a tool commonly used by policymakers to foresee future needs and proactively shape a regulatory framework tailored to those needs. Through the application of this methodology, different trends in NHP have been identified, notably NHPs for drug delivery and dental applications. Furthermore, the most disruptive elements involve NHPs that are multicomposite and multifunctional, harnessing nano-scale properties to combine therapeutic and diagnostic purposes within a single product. When compared with the regulatory landscape, current regulations are gradually adapting to accommodate emerging trends, with specific guidelines being developed. However, for the most disruptive elements, multicomposite and multifunctional NHPs, their novelty still poses significant regulatory challenges, requiring a strategic development of guidelines by regulatory agencies to ensure their safe and effective integration into healthcare practices. This study underscores the importance of proactive regulatory planning to bridge the gap between NHP innovation and market implementation.

Health horizons: Future trends and technologies from the European Medicines Agency's horizon scanning collaborations.

In medicines development, the progress in science and technology is accelerating. Awareness of these developments and their associated challenges and opportunities is essential for medicines regulators and others to translate them into benefits for society. In this context, the European Medicines Agency uses horizon scanning to shine a light on early signals of relevant innovation and technological trends with impact on medicinal products. This article provides the results of systematic horizon scanning exercises conducted by the Agency, in collaboration with the World Health Organization (WHO) and the European Commission's Joint Research Centre's (DG JRC). These collaborative exercises aim to inform policy-makers of new trends and increase preparedness in responding to them. A subset of 25 technological trends, divided into three clusters were selected and reviewed from the perspective of medicines regulators. For each of these trends, the expected impact and challenges for their adoption are discussed, along with recommendations for developers, regulators and policy makers.

From adoration to damnation? Exploring role of media in shaping low-carbon economy in times of the COVID-19 pandemic.

The leverage of the public narrative created and maintained by the media as a highly influential social actor is decisive, but also sensitive, in bringing about the energy transition and advancing towards a low-carbon economy. The outbreak of the COVID-19 pandemic has potential to slow down and deform the public acceptance of the above-mentioned processes as it is likely that the priorities of individual governments will be revisited and tailored to tackle the ongoing health crisis. We are replying to such a threat with this study that aims to reflect on the immense role of media in shaping a low-carbon economy in transitional economies. We are using Poland as an illustrative example to demonstrate how wide, colourful, and sometimes even confusing the low-carbon narrative might be. By means of employing the horizon scanning of the diverse types of media, we detected that media overwhelmingly affect and deform the ongoing discussions about the nuances of energy transition and benefits a low-carbon economy. We argue that political preferences of individual journalists (and publishing houses) in Poland tend to influence the style, depth, extent, and quality how the topic is covered and narrated.

Application of Internet of Medical/Health Things to Decentralized Clinical Trials: Development Status and Regulatory Considerations.

Background: The need for a new style of clinical trials, called decentralized clinical trials (DCTs), has been increasing as they do not depend on physical visits to clinical sites. DCTs are expected to provide a new opportunity to patients who cannot participate in a clinical trial due to geographical and time limitations. For the adoption of DCTs, it is essential that medical devices with Internet of Medical Things (IoMT) and Internet of Health Things (IoHT) based technologies are developed and commercially adopted. In this study, we aimed to identify the regulatory considerations when IoMT/IoHT-based technologies are used in DCTs or products developed using DCTs. Method: To understand the study and development field of IoMT/IoHT comprehensively and panoramically, relevant papers published in Web of Science were searched online. Subsequently, a citation network was obtained and characterized as a cluster using a text mining method to identify IoMT/IoHT-based technologies expected to be utilized in DCTs or products developed using DCTs. Result and Discussion: Upon analysis of the top 15 clusters and subsequent 51 sub-clusters, we identified the therapeutic areas (psychology, neurology) and IoMT/IoHT-based technologies (telemedicine, remote monitoring, and virtual reality) that are expected to be used in DCTs. We also identified several considerations based on the current regulatory guidance. Conclusion: IoMT/IoHT-based technologies that are expected to be used or products developed using DCTs and key considerations made when they are used in DCTs were identified. The considerations could encourage conducting DCTs using IoMT/IoHT-based technologies.