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.

Digital Health Applications (DiHA): Approaches to develop a reimbursement process for the statutory health insurance in Austria.

Purpose: To elaborate a concept for implementing digital health applications (DiHA), including prioritisation criteria (PC) for the Austrian context and an overview of available prioritised DiHAs. Methods: Based on European DiHA-listings and input by Austrian experts, a categorised meta-directory of DiHAs was created. PC were developed to reflect, inter alia, the provisions of the Austrian General Insurance Act, and were applied to the meta-directory to identify DiHAs potentially relevant for the Austrian statutory health insurance. An iterative process with expert involvement was used to tailor an existing reimbursement framework to the Austrian setting. Results: The meta-directory comprised 132 DiHAs. Developed PC focused on plausibility (German language) and legal aspects (treatment/monitoring of chronic conditions), while other criteria (e.g. interoperability standards) were considered optional. After applying the PC, 38 DiHAs were potentially relevant in the Austrian setting. Of these, only seven supported current health record integration. Most of the prioritised DiHAs reported on CE marking (29/38) and data protection (35/38), while reporting on risk class (10/38) and technical algorithms (0/38) was sparse. For DiHA reimbursement, a four-step process is proposed: identification (ideally based on needs assessment); filtering based on PC; review of technical, regulatory and evidentiary requirements; and health technology assessment. Conclusion: The proposed concept can offer guidance for policy makers (e.g., on prioritising available DiHAs) and may further foster scientific debate with regard to DiHA implementation. Further discussion on how to fully incorporate regulatory, technical, and evidentiary criteria is needed. Attention should be given to national implementation requirements, re-assessment criteria, and appropriate remuneration schemes.

EUnetHTA's contribution to the new legal framework for health technology assessment cooperation in Europe.

The history of the European cooperation on health technology assessment (HTA) has reached an important milestone with the adoption of the European HTA Regulation (HTA R) 2021/2282 in Dec 2021 (1). Its publication in the Official Journal of the European Union means a lot to those of us who wish to give HTA a stronger role in supporting health policy in favor of sustainable healthcare systems in Europe. The HTA R was prepared well by the European Commission with an impact assessment on policy options for enhanced EU cooperation on HTA (2) in 2017, followed by 3 years of negotiations with Member States. Now the ratified document stipulates that the European Cooperation on HTA will be based on a legal mandate and no longer stay a voluntary project-based initiative. It also means sustainability, since a permanent Secretariat at the European Commission will be set-up under the HTA R.

European Network for Health Technology Assessment's Response to COVID-19: Rapid Collaborative Reviews on Diagnostic Tests and Rolling and Rapid Collaborative Reviews on Therapeutics.

OBJECTIVES: In spring 2020, The European network for Health Technology Assessment (EUnetHTA) decided to join forces to produce best evidence to inform health policy in the COVID-19 pandemic. The objective of this paper is to describe the process and output of the coordinated and collaborative activities of EUnetHTA. METHODS: Relevant published and internal documents were retrieved for a descriptive analysis of EUnetHTA processes, methods, and outputs related to EUnetHTA's response to the pandemic. RESULTS: Process: In April 2020, a COVID-19 task force was set up and a survey collected pressing health policy questions across Europe. Two coordinating agencies for diagnostic tests and therapeutics were assigned. A process for prioritization and selection was set up for therapeutics, as well as explicit starting and stopping rules. Methodology: To increase a timely response, it was agreed that the rapid collaborative reviews (rapid CRs) would not require the consultation of manufacturers and the involvement of external experts, but would not differ in the methods and conduct of the systematic search, review, and synthesis of all available evidence, nor in the requirement for reviewing by EUnetHTA partners. Final reports: The joint effort resulted in the production of two rapid CRs on diagnostic tests, nineteen collaborative rolling reviews on therapeutics, three of which later moved to rapid CRs. CONCLUSIONS: During COVID-19 pandemic, the EUnetHTA partners proved capable of prompt collaboration, which allowed speeding up the production and release of high-quality EUnetHTA outputs, while the relationships with the other European institutions facilitated their quick dissemination.

Reimbursement decisions for medical services in Austria: an analysis of influencing factors for the hospital individual services catalogue between 2008 and 2020.

OBJECTIVES: To (1) describe the (evidence-based) reimbursement process of hospital individual services, (2) evaluate the accordance between evidence-based recommendations and reimbursement decision of individual services and (3) elaborate potential aspects that play a role in the decision-making process in Austria. METHOD: The reimbursement process is described based on selected relevant sources such as official documents. Evidence-based recommendations and subsequent reimbursement decisions for the annual maintenance of the hospital individual service catalogue in Austria between 2008 and 2020 were analysed using a mixed methods approach, encompassing descriptive statistics and a focus group with Austrian decision makers. RESULTS: 118 evidence-based recommendations were analysed. There were 93 (78.8%) negative and 25 (21.2%) positive evidence-based recommendations. In total, 107 out of 118 evidence-based recommendations (90.1%) did not lead to a deviating reimbursement decision. We identified six aspects that may have played a role in the decision-making process for the annual maintenance of the hospital individual service catalogue, with clinical evidence being the most notable. Further aspects included quality assurance/organisational aspects (i.e., structural quality assurance), costs (if comparable to already existing medical services, not: cost-effectiveness), procedural aspects (e.g., if certain criteria for adoption have not been met formally through the proposals), "other countries" (i.e., taking into account how other countries decided) and situational aspects (such as the COVID-19 pandemic). CONCLUSIONS: There is good accordance between evidence-based recommendations and reimbursement decisions regarding hospital individual services in Austria. Beyond clinical evidence, organisational aspects seem to be considered often with regard to quality assurance but costs do not appear to play a major role. The Austrian system has mechanisms in place that can restrict widespread adoption of novel hospital individual services with uncertain clinical benefits. Future studies could investigate how well these mechanisms work and how they compare to other health systems in Europe.

[Technology assessment of digital health applications for reimbursement decisions]. / Technologiebewertung digitaler Gesundheitsanwendungen für Refundierungsentscheidungen.

BACKGROUND: For most digital health applications (DiGA) only limited evidence of benefit is available. Currently available assessment frameworks do not cover all domains of a full health technology assessment (HTA). Additionally, technology-specific aspects are required for the evaluation of DiGA. This work aimed to analyze the available assessment frameworks and design an evaluation process for DiGA. METHODS: By a systematic literature search six assessment frameworks for DiGA were selected and analyzed. A hand search for strategies on DiGA of selected countries was conducted. RESULTS: Of the analyzed assessment frameworks four described study designs. One assessment framework proposed a risk classification of DiGA. Aspects of artificial intelligence were assessed by one assessment framework. The analyzed countries have differing strategies for reimbursement of DiGA. CONCLUSION: Assessment frameworks for DiGA are very heterogeneous. There are efforts to find regulations for DiGA on a national level. When evaluating DiGA, a staged approach considering risk classes with subsequent evaluation of relevant HTA aspects is recommended.

Health technology assessment applied to emergency preparedness: a new perspective.

Emergency preparedness is a continuous quality improvement process through which roles and responsibilities are defined to effectively anticipate, respond to, and recover from the impact of emergencies. This process results in documented plans that provide a backbone structure for developing the core capacities to address health threats. Nevertheless, several barriers can impair an effective preparedness planning, as it needs a 360° perspective to address each component according to the best evidence and practice. Preparedness planning shares common principles with health technology assessment (HTA) as both encompass a multidisciplinary and multistakeholder approach, follow an iterative cycle, adopt a 360° perspective on the impact of intervention measures, and conclude with decision-making support. Our "Perspective" illustrates how each HTA domain can address different component(s) of a preparedness plan that can indeed be seen as a container of multiple HTAs, which can then be used to populate the entire plan itself. This approach can allow one to overcome preparedness barriers, providing an independent, systematic, and robust tool to address the components and ensuring a comprehensive evaluation of their value in the mitigation of the impact of emergencies.

Extracorporeal Cytokine Adsorption Therapy As a Preventive Measure in Cardiac Surgery and As a Therapeutic Add-On Treatment in Sepsis: An Updated Systematic Review of Comparative Efficacy and Safety.

OBJECTIVES: Evaluating whether there is a clinical benefit of using extracorporeal cytokine adsorption therapy in two indications. DESIGN: Systematic review. SETTING: Search on four databases, Medline, Embase, The Cochrane Library, and the European Network for Health Technology Assessment planned and ongoing projects database. PATIENTS: Patients with sepsis/septic shock; patients undergoing cardiac surgery. INTERVENTIONS: Cytokine adsorption. MEASUREMENTS AND MAIN RESULTS: Randomized controlled trials and prospective studies with concurrent control were eligible for the evidence synthesis. The quality of the individual studies and the strength of the available evidence were assessed using the Cochrane risk of bias tool and the Grading of Recommendations, Assessment, Development, and Evaluation approach, respectively. For the preventive treatment of extracorporeal cytokine adsorption therapy in patients undergoing cardiac surgery, we found very low-quality inconclusive evidence for mortality (five randomized controlled trials, n = 163), length of stay in the ICU (five randomized controlled trials, n = 163), and length of hospitalization (three randomized controlled trials, n = 101). Very low-quality inconclusive evidence was found for (serious) adverse events (four randomized controlled trials, n = 148). For the therapeutic treatment of extracorporeal cytokine adsorption therapy in patients with sepsis/septic shock, we found very low-quality inconclusive evidence for mortality up to 60-day follow-up (two randomized controlled trials, n = 117), organ function (two randomized controlled trials, n = 117) and length of stay in the ICU (one randomized controlled trial, n = 20). Very low-quality inconclusive evidence was found for (serious) adverse events (two randomized controlled trials, n = 117). CONCLUSIONS: Given the available evidence, the efficacy and safety of extracorporeal cytokine adsorption therapy in combination with standard care in the investigated indications was not established. We strongly recommend considering well-powered studies with patient-relevant endpoints instead of investing further research funds on studies that may not shed light on the clinical benefit of extracorporeal cytokine adsorption therapy.

Evaluating options for decision making on costly hospital drugs in Austria.

OBJECTIVES: The aim of this study was to suggest options for a national and standardized process for the reimbursement of costly drugs provided in Austrian hospitals. METHODS: For answering the research questions, reimbursement processes of ten countries were investigated and the strengths and weaknesses of elaborated options of actions were analyzed, resulting in suggestions for solutions in the Austrian reimbursement processes for hospital drugs. RESULTS: Based on the information derived from the international analysis and the deliberation of the strengths and weaknesses on optional approaches, as well as, on the consideration of the existing reimbursement processes in Austria, three options to reorganize the current decentralized inpatient reimbursement process in Austria were suggested. The first option presents a process following the established processes of the decision making for outpatient drugs. The second option suggests stronger coordination of and cooperation across the existing processes of the nine regional "Pharmaceutical and Therapeutics Committees". The third option proposes to expand the already established reimbursement process for non-drug interventions. CONCLUSIONS: Evidence-based, transparent, fair and efficient resource allocations are needed for priority setting decisions. However, a decision process can be based on the best available evidence, can be fair and transparent, although it might be substantially more time-consuming. Thus, a pragmatic balance between quality, transparency and timeliness is crucial.

Publicly accessible evidence of health-related quality of life benefits associated with cancer drugs approved by the European Medicines Agency between 2009 and 2015.

OBJECTIVE: Health-related quality of life (HRQoL) is one of the most important patient-relevant study end-points for the direct measurement of the benefit of cancer drugs. Therefore, our aim is to detect cancer indications with no published information on HRQoL at the time of European Medicines Agency (EMA) approval and monitor any reported HRQoL evidence updates after at least three years of follow-up. METHODS: We included all cancer indications that were approved by the EMA between January 2009 and October 2015. Our main sources of information were the EMA website, clinicaltrials.gov and a systematic literature search in PubMed. Information on HRQoL outcomes was extracted alongside evidence on median overall survival. RESULTS: In total, we identified 110 indications, of which more than half (n = 58, 53%) were lacking available information on HRQoL assessments at the time of EMA approval. After a monitoring period of at least three years, 24 updates were identified, resulting in 34 (31%) therapies where information on HRQoL was still not available. For the 76 therapies with reported information on HRQoL, cancer-specific instruments were mostly used (n = 49/76). Regarding cumulative evidence on median overall survival and HRQoL, 33 (n = 33/110, 30%) as well as 15 (n = 15/110, 14%) cancer drugs were lacking information on both study end-points at the time of approval and after monitoring, respectively. CONCLUSION: Our results demonstrate that there is an urgent need of routine re-evaluation of reimbursed cancer drugs with initially missing information on major outcomes. Standardisation of the typology and quality of HRQoL assessments need to be improved to allow better comparability of results.

Health Technology Assessment of Carbon-ion Beam Radiotherapy: A Systematic Review of Clinical Effectiveness and Safety for 54 Oncological Indications in 12 Tumour Regions.

BACKGROUND/AIM: Due to the unique physical dose distribution of carbon-ion radiotherapy (CIRT), CIRT can be regarded as a novel tumour irradiation technique - potentially advantageous for various tumour types. Yet it is unclear in how far, superiority or inferiority can be claimed when comparing CIRT to standard irradiation. This study aimed to assess the scientific evidence regarding the effectiveness and safety of CIRT. MATERIALS AND METHODS: A systematic literature review was performed using the European Network for Health Technology Assessment (EUnetHTA) Core Model® for rapid relative effectiveness assessment. The literature search for clinical outcome studies on CIRT was performed using four databases [Cochrane (Central), Centre for Research and Dissemination (CRD), Embase and OVID MEDLINE]. The Cochrane Risk of Bias Tool (for randomised controlled trials) and the Institute of Health Economics (IHE-18) Checklist (for observational studies) were used to assess the risk of bias of the included studies. The evidence synthesis was restricted to 54 oncological indications in 12 broad tumour regions and studies with a low or moderate risk of bias, published between 2005 and 2017. RESULTS: Twenty-seven studies were eligible for the qualitative synthesis of the evidence regarding the effectiveness and safety of CIRT: One randomised controlled trial that primarily focused on the feasibility of CIRT, three case-control studies, three before- and after- studies with a focus on quality of life, and 20 further studies of case series. Overall, insufficient scientific evidence was found for superiority or inferiority of CIRT when compared to standard irradiation for 13/54 oncologicaI indications in 7/12 tumour regions (skull base tumours, brain cancer, cancer in the ear-nose-throat region, bone and soft-tissue tumours, lung cancer, prostate cancer, gastrointestinal tumours). No scientific evidence was found for the remaining 41/54 oncological indications. CONCLUSION: CIRT is undoubtedly, theoretically, a promising cancer treatment. To date, however, it lacks randomised controlled trials assessing the long-term effectiveness and harms associated with the use of CIRT. CIRT must be considered as an experimental treatment due to the lack of high-quality clinical research.

European Collaboration in Health Technology Assessment (HTA): goals, methods and outcomes with specific focus on medical devices.

The European Network for Health Technology Assessment (EUnetHTA) was founded to support efficient production and use of health technology assessments (HTAs) across Europe by reducing redundancies through collaboration. To facilitate collaboration, a range of practical tools, methods and process definitions were developed. The article describes when and how these tools and methods are used along the HTA process with specific focus on "other technologies", that is medical devices and non-pharmaceutical procedures. EUnetHTA was able to deliver tangible achievements complying with its goals. The practical tools and the developed methods formed a basis for close collaboration among over 70 agencies at a European level. The activities of EUnetHTA laid a strong foundation for sustainable cooperation. In the long run, jointly produced assessments could realise economies of scale with improved quality, consistency and transparency for the health systems in Europe.

Pre-reimbursement: early assessment for coverage decisions.

BACKGROUND: In the past decade, the Ludwig Boltzmann Institute for Health Technology Assessment (LBI-HTA) has introduced two programs: "Horizon Scanning in Oncology" (HSO) and extra medical services ("MELs"), which are to facilitate coverage decisions based on early assessments. This article aims to outline the general process and methods within these two programs. METHODS: A narrative-descriptive synthesis of the literature was performed to outline the general and LBI-HTA-specific processes and methods of early assessments. RESULTS: In total, 79 HSO assessments (2009-2018) and 95 MELs (2008-2018) have been conducted by the LBI-HTA. Recently, additional methods that contribute to European applicability have been introduced into these programs. CONCLUSIONS: Overall, pre-coverage decisions based on early assessment reports are dependent on the existing evidence. However, the organisation of the health care system and the cross-linking between decision-makers and HTA institutions can have an impact.

TOWARD A CONTINGENCY MODEL FOR HOSPITAL-BASED HEALTH TECHNOLOGY ASSESSMENT: EVIDENCE FROM ADHOPHTA PROJECT.

OBJECTIVES: Hospital-based health technology assessment (HB-HTA) is becoming increasingly relevant because of its role in managing the introduction and withdrawal of health technologies. The organizational arrangement in which HB-HTA activities are conducted depends on several contextual factors, although the dominant models have several similarities. The aims of this study were to explore, describe, interpret, and explain seven cases of the application of HB-HTA logic and to propose a classification for HB-HTA organizational models which may be beneficial for policy makers and HTA professionals. METHODS: The study was part of the AdHopHTA Project, granted under the European 7th Framework Research Programme. A case study methodology was applied to analyze seven HB-HTA initiatives in seven countries, with collection of qualitative and quantitative data. Cross-case analysis was performed within the framework of contingent organizational theory. RESULTS: Evidence showed that some organizational or "structural" variables, namely the level of procedure formalization/structuration and the level of integration with other HTA bodies at the national, regional, and provincial levels, predominantly shape the HB-HTA approach, determining a contingency model of HB-HTA. Crossing the two variables, four options have emerged: integrated specialized HTA unit, stand-alone HTA unit, integrated-essential HTA, independent group unit. CONCLUSIONS: No one-best-way approach can be used for HTA at the hospital level. Rather, the characteristics of HTA models depend on many contextual factors. Such conceptualization may aid the diffusion of HB-HTA to inform managerial decision making and clinical practice.

Individual and Institutional Corruption in European and US Healthcare: Overview and Link of Various Corruption Typologies.

In recent years, the fight against healthcare corruption has intensified. Estimates from the European Healthcare Fraud and Corruption Network calculate an approximate €56 billion annual loss to Europe as a result of corruption. To promote understanding of the complexity and interconnection of corrupt activities, we aim to present healthcare-related corruption typologies of the European Union and European Healthcare Fraud and Corruption Network. We subsequently link them to the typology of individual and institutional corruption introduced by Dennis Thompson in the context of investigating misconduct of US Congressional members. According to Thompson, individual corruption is the personal gain of individuals performing duties within an institution in exchange for nurturing private interests, while institutional corruption pertains to the failure of the institution in directing the individual's behaviour towards the achievement of the institution's primary purpose because the institutional design promotes the pursuit of individual goals. Effective anti-corruption activities not only require the enactment of anti-corruption laws but also the monitoring and, where appropriate, revision of institutional frameworks to prevent the undermining of the primary purposes of health systems or institutions. To gain further understanding of the similarities and differences of the three typologies, prime examples of corrupt activities in the health sector in the European Union and USA (along with their potential remedies) are provided. Linking corruption cases to Thompson's typology revealed that many corrupt activities may show elements of both individual and institutional corruption because they are intertwined, partly overlap and may occur jointly. Hence, sanctioning individual actors only does not target the problem.

ANALYSIS OF DUPLICATION AND TIMING OF HEALTH TECHNOLOGY ASSESSMENTS ON MEDICAL DEVICES IN EUROPE.

OBJECTIVES: Strengthening efforts toward better collaboration plays a pivotal role in the assessment of medical devices to reduce overlap and save resources. This study explored the level of duplication in health technology assessments (HTA) of medical devices in Europe and their respective timing in order to identify areas for better collaboration. METHODS: An analysis of European HTA reports of medical devices regarding overlaps in topics and timing in relation to market authorization was performed. We conducted a systematic search in the ADVANCE, Centre for Reviews and Dissemination, Syngerus, and POP databases, complemented by hand searching, to identify HTA reports published between 01/2003 and 07/2016 for a preselected cohort group of ten technologies. We analyzed the number of annual assessments per technology and evaluated activity patterns and timing in undertaking the HTA of the different institutes in Europe. RESULTS: The results revealed the amount of duplication in the European HTA production: the number of reports per technology ranged from minimum seven to maximum twenty-two over a time-span of 13.5 years. HTA institutes perform assessments at a similar time range within 5 to 10 years following market authorization. The timing of the initial assessment in relation to the granting of the CE-mark varies according to the particular technology. CONCLUSION: The findings suggest that efficient collaboration may help to save scarce resources and time of HTA institutes in Europe. Efficient collaboration as such needs to shift the focus beyond the time span of 1 year, and build on each's others work from previous assessments.

Wearable cardioverter defibrillators for the prevention of sudden cardiac arrest: a health technology assessment and patient focus group study.

AIM: To summarize the evidence on clinical effectiveness and safety of wearable cardioverter defibrillator (WCD) therapy for primary and secondary prevention of sudden cardiac arrest in patients at risk. METHODS: We performed a systematic literature search in databases including MEDLINE via OVID, Embase, the Cochrane Library, and CRD (DARE, NHS-EED, HTA). The evidence obtained was summarized according to GRADE methodology. A health technology assessment (HTA) was conducted using the HTA Core Model® for rapid relative effectiveness assessment. Primary outcomes for the clinical effectiveness domain were all-cause and disease-specific mortality. Outcomes for the safety domain were adverse events (AEs) and serious adverse events (SAEs). A focus group with cardiac disease patients was conducted to evaluate ethical, organizational, patient, social, and legal aspects of the WCD use. RESULTS: No randomized- or non-randomized controlled trials were identified. Non-comparative studies (n=5) reported AEs including skin rash/itching (6%), false alarms (14%), and palpitations/light-headedness/fainting (9%) and discontinuation due to comfort/lifestyle issues (16-22%), and SAEs including inappropriate shocks (0-2%), unsuccessful shocks (0-0.7%), and death (0-0.3%). The focus group results reported that experiencing a sense of security is crucial to patients and that the WCD is not considered an option for weeks or even months due to expected restrictions in living a "normal" life. CONCLUSION: The WCD appears to be relatively safe for short-to-medium term, but the quality of existing evidence is very low. AEs and SAEs need to be more appropriately reported in order to further evaluate the safety of the device. High-quality comparative evidence and well-described disease groups are required to assess the effectiveness of the WCD and to determine which patient groups may benefit most from the intervention.