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.

Development of transferability guidance for integrated care models with special focus on Central and Eastern European countries.

AIM: To develop pragmatic recommendations for Central and Eastern European (CEE) policymakers about transferability assessment of integrated care models established in higher income European Union (EU) countries. METHODS: Draft recommendations were developed based on Horizon 2020-funded SELFIE project deliverables related to 17 promising integrated care models for multimorbid patients throughout Europe, as well as on an online survey among CEE stakeholders on the relevance of implementation barriers. Draft recommendations were discussed at the SELFIE transferability workshop and finalized together with 22 experts from 12 CEE countries. RESULTS: Thirteen transferability recommendations are provided in three areas. Feasibility of local implementation covers the identification and prioritization of implementation barriers and proposals for potential solutions. Performance measurement of potentially transferable models focuses on the selection of models with proven benefits and assurance of performance monitoring. Transferability of financing methods for integrated care explores the relevance of financing methodologies and planning of adequate initial and long-term financing. CONCLUSIONS: Implementation of international integrated care models cannot be recommended without evidence on its local feasibility or scientifically sound and locally relevant performance assessment in the country of origin. However, if the original financing method is not transferable to the target region, development of a locally relevant alternative financing method can be considered.

Identifying the Need for Good Practices in Health Technology Assessment: Summary of the ISPOR HTA Council Working Group Report on Good Practices in HTA.

The systematic use of evidence to inform healthcare decisions, particularly health technology assessment (HTA), has gained increased recognition. HTA has become a standard policy tool for informing decision makers who must manage the entry and use of pharmaceuticals, medical devices, and other technologies (including complex interventions) within health systems, for example, through reimbursement and pricing. Despite increasing attention to HTA activities, there has been no attempt to comprehensively synthesize good practices or emerging good practices to support population-based decision-making in recent years. After the identification of some good practices through the release of the ISPOR Guidelines Index in 2013, the ISPOR HTA Council identified a need to more thoroughly review existing guidance. The purpose of this effort was to create a basis for capacity building, education, and improved consistency in approaches to HTA-informed decision-making. Our findings suggest that although many good practices have been developed in areas of assessment and some other key aspects of defining HTA processes, there are also many areas where good practices are lacking. This includes good practices in defining the organizational aspects of HTA, the use of deliberative processes, and measuring the impact of HTA. The extent to which these good practices are used and applied by HTA bodies is beyond the scope of this report, but may be of interest to future researchers.

Strengthening the evidence-base of integrated care for people with multi-morbidity in Europe using Multi-Criteria Decision Analysis (MCDA).

BACKGROUND: Evaluation of integrated care programmes for individuals with multi-morbidity requires a broader evaluation framework and a broader definition of added value than is common in cost-utility analysis. This is possible through the use of Multi-Criteria Decision Analysis (MCDA). METHODS AND RESULTS: This paper presents the seven steps of an MCDA to evaluate 17 different integrated care programmes for individuals with multi-morbidity in 8 European countries participating in the 4-year, EU-funded SELFIE project. In step one, qualitative research was undertaken to better understand the decision-context of these programmes. The programmes faced decisions related to their sustainability in terms of reimbursement, continuation, extension, and/or wider implementation. In step two, a uniform set of decision criteria was defined in terms of outcomes measured across the 17 programmes: physical functioning, psychological well-being, social relationships and participation, enjoyment of life, resilience, person-centeredness, continuity of care, and total health and social care costs. These were supplemented by programme-type specific outcomes. Step three presents the quasi-experimental studies designed to measure the performance of the programmes on the decision criteria. Step four gives details of the methods (Discrete Choice Experiment, Swing Weighting) to determine the relative importance of the decision criteria among five stakeholder groups per country. An example in step five illustrates the value-based method of MCDA by which the performance of the programmes on each decision criterion is combined with the weight of the respective criterion to derive an overall value score. Step six describes how we deal with uncertainty and introduces the Conditional Multi-Attribute Acceptability Curve. Step seven addresses the interpretation of results in stakeholder workshops. DISCUSSION: By discussing our solutions to the challenges involved in creating a uniform MCDA approach for the evaluation of different programmes, this paper provides guidance to future evaluations and stimulates debate on how to evaluate integrated care for multi-morbidity.

HEALTH TECHNOLOGY ASSESSMENT IN CENTRAL, EASTERN, AND SOUTH EUROPEAN COUNTRIES: CROATIA.

OBJECTIVES: The aim of this study was to provide a brief, 7-year history of health technology assessment (HTA) implementation in Croatia through national and international activities. METHODS: We used retrospective descriptive analysis of key documents related to the legal framework, process of decision making, and HTA. Analysis of the Agency's plan for and experience with the implementation of a transparent HTA process in Croatia was performed by addressing seven key components of the HTA implementation scorecard framework. The main challenges and facilitating factors were also assessed. RESULTS: HTA is not yet fully implemented in Croatia. The main challenges are the insufficient legal framework, limited human and financial resources, and limited stakeholder involvement. Facilitating factors are active international collaboration and education through EUnetHTA and the International Society for Pharmacoeconomics and Outcomes Research and production of national and international HTA reports. CONCLUSIONS: The HTA process is not yet sustainable in Croatia and HTA reports are still not mandatory for reimbursement/investment or disinvestment decision processes. There are still barriers to overcome.

HTA Implementation Roadmap in Central and Eastern European Countries.

The opportunity cost of inappropriate health policy decisions is greater in Central and Eastern European (CEE) compared with Western European (WE) countries because of poorer population health and more limited healthcare resources. Application of health technology assessment (HTA) prior to healthcare financing decisions can improve the allocative efficiency of scarce resources. However, few CEE countries have a clear roadmap for HTA implementation. Examples from high-income countries may not be directly relevant, as CEE countries cannot allocate so much financial and human resources for substantiating policy decisions with evidence. Our objective was to describe the main HTA implementation scenarios in CEE countries and summarize the most important questions related to capacity building, financing HTA research, process and organizational structure for HTA, standardization of HTA methodology, use of local data, scope of mandatory HTA, decision criteria, and international collaboration in HTA. Although HTA implementation strategies from the region can be relevant examples for other CEE countries with similar cultural environment and economic status, HTA roadmaps are not still fully transferable without taking into account country-specific aspects, such as country size, gross domestic product per capita, major social values, public health priorities, and fragmentation of healthcare financing.

Collaboration in health technology assessment (EUnetHTA joint action, 2010-2012): four case studies.

OBJECTIVES: The aim of this study was to present the first four collaborative health technology assessment (HTA) processes on health technologies of different types and life cycles targeted toward diverse HTA users and facilitators, as well as the barriers of these collaborations. METHODS: Retrospective analysis, through four case studies, was performed on the first four collaboration experiences of agencies participating in the EUnetHTA Joint Action project (2010-12), comprising different types and life cycles of health technologies for a diverse target audience, and different types of collaboration. The methods used to initiate collaboration, partner contributions, the assessment methodology, report structure, time frame, and factors acting as possible barriers to and facilitators of this collaboration were described. RESULTS: Two ways were used to initiate collaboration in the first four collaborative HTA processes: active brokering of information, so-called "calls for collaboration," and individual contact between agencies after identifying a topic common to two agencies in the Planned and Ongoing Projects database. Several success factors are recognized: predefined project management, high degree of commitment to the project; adherence to timelines; high relevance of technology; a common understanding of the methods applied and advanced experience in HTA; finally, acceptance of English-written reports by decision makers in non-English-speaking countries. Barriers like late identification of collaborative partners, nonacceptance of English language and different methodology of assessment should be overcome. CONCLUSIONS: Timely and efficient, different collaborative HTA processes on relative efficacy/effectiveness and safety on different types and life cycles of health technologies, targeted toward diverse HTA users in Europe are possible. There are still barriers to overcome.

Pre-market clinical evaluations of innovative high-risk medical devices in Europe.

OBJECTIVES: High-quality clinical evidence is most often lacking when novel high-risk devices enter the European market. At the same time, a randomized controlled trial (RCT) is often initiated as a requirement for obtaining market access in the US. Should coverage in Europe be postponed until RCT data are available? We studied the premarket clinical evaluation of innovative high-risk medical devices in Europe compared with the US, and with medicines, where appropriate. METHODS: The literature and regulatory documents were checked. Representatives from industry, Competent Authorities, Notified Bodies, Ethics Committees, and HTA agencies were consulted. We also discuss patient safety and the transparency of information. RESULTS: In contrast to the US, there is no requirement in Europe to demonstrate the clinical efficacy of high-risk devices in the premarket phase. Patients in Europe can thus have earlier access to a potentially lifesaving device, but at the risk of insufficiently documented efficacy and safety. Variations in the stringency of clinical reviews, both at the level of Notified Bodies and Competent Authorities, do not guarantee patient safety. We tried to document the design of premarket trials in Europe and number of patients exposed, but failed as this information is not made public. Furthermore, the Helsinki Declaration is not followed with respect to the registration and publication of premarket trials. CONCLUSIONS: For innovative high-risk devices, new EU legislation should require the premarket demonstration of clinical efficacy and safety, using an RCT if possible, and a transparent clinical review, preferably centralized.

Prioritization of implementation barriers related to integrated care models in Central and Eastern European countries.

The importance of integrated care will increase in future health systems due to aging populations and patients with chronic multimorbidity, however, such complex healthcare interventions are often developed and implemented in higher income countries. For Central and Eastern European (CEE) countries it is important to investigate which integrated care models are transferable to their setting and facilitate the implementation of relevant models by identifying barriers to their implementation. This study investigates the relative importance of integrated care models and the most critical barriers for their implementation in CEE countries. Experts from Croatia, Hungary, Poland, Romania and Serbia were invited to complete an online survey within the SELFIE H2020 project. 81 respondents completed the survey. Although experts indicated that some integrated care models were already being implemented in CEE countries, the survey revealed a great need for further improvement in the integration of care, especially the managed care of oncology patients, coordinated palliative care of terminally ill patients, and nursing care of elderly with multimorbidity. Lack of long-term financial sustainability as well as of dedicated financing schemes were seen the most critical implementation barriers, followed by the lack of integration between health and social care providers and insufficient availability of human resources. These insights can guide future policy making on integrated care in CEE countries.

Drivers of successful implementation of integrated care for multi-morbidity: Mechanisms identified in 17 case studies from 8 European countries.

This paper provides a deeper understanding of the mechanisms underlying implementation strategies for integrated care. As part of the SELFIE project, 17 integrated care programmes addressing multi-morbidity from eight European countries were selected and studied. Data was extracted from 'thick descriptions' of the 17 programmes and analysed both inductively and deductively using implementation theory. The following ten mechanisms for successful implementation of integrated care were identified. With regards to service delivery, successful implementers (1) commonly adopted an incremental growth model rather than a disruptive innovation approach, and found (2) a balance between flexibility and formal structures of integration. For leadership & governance, they (3) applied collaborative governance by engaging all stakeholders, and (4) distributed leadership throughout all levels of the system. For the workforce, these implementers (5) were able to build a multidisciplinary team culture with mutual recognition of each other's roles, and (6) stimulated the development of new roles and competencies for integrated care. With respect to financing, (7) secured long-term funding and innovative payments were applied as means to overcome fragmented financing of health and social care. Implementers emphasised (8) the implementation of ICT that was specifically developed to support collaboration and communication rather than administrative procedures (technology & medical devices), and (9) created feedback loops and a continuous monitoring system (information & research). The overarching mechanism was that implementers (10) engaged in alignment work across the different components and levels of the health and social care system. These evidence-based mechanisms for implementation are applicable in different local, regional and national contexts.

[Quality of health care, accreditation, and health technology assessment in Croatia: role of agency for quality and accreditation in health]. / Kvaliteta zdravstvene zastite, akreditacija nositelja zdravstvene djelatnosti i procjena zdravstvenih tehnologija u Hrvatskoj: uloga agencije za kv alitetu i akreditaciju u zdravstvu.

Avedis Donabedian defined the quality of care as the kind of care, which is expected to maximize an inclusive measure of patient welfare, after taking into account the balance of expected gains and losses associated with the process of care in all its segments. According to the World Medical Assembly, physicians and health care institutions have an ethical and professional obligation to strive for continuous quality improvement of services and patient safety with the ultimate goal to improve both individual patient outcomes as well as population health. Health technology assessment (HTA) is a multidisciplinary process that summarizes information about the medical, social, economic and ethical issues related to the use of a health technology in a systematic, transparent, unbiased, robust manner, with the aim to formulate safe and effective health policies that are patient focused and seek to achieve the highest value. The Agency for Quality and Accreditation in Health was established in 2007 as a legal, public, independent, nonprofit institution under the Act on Quality of Health Care. The Agency has three departments: Department of Quality and Education, Department of Accreditation, and Department of Development, Research, and Health Technology Assessment. According to the Act, the Agency should provide the procedure of granting, renewal and cancellation of accreditation of healthcare providers; proposing to the Minister, in cooperation with professional associations, the plan and program for healthcare quality assurance, improvement, promotion and monitoring; proposing the healthcare quality standards as well as the accreditation standards to the Minister; keeping a register of accreditations and providing a database related to accreditation, healthcare quality improvement, and education; providing education in the field of healthcare quality assurance, improvement and promotion; providing the HTA procedure and HTA database, supervising the healthcare insurance standards, and providing other services in the field of healthcare quality assurance, improvement, promotion and monitoring, according to the Act. Formal activities of the Agency in the field of HTA actually began in summer 2009. In the field of quality and accreditation, the plan and program of healthcare quality assurance, improvement, promotion and monitoring was finished and published in October 2010; preparation of the healthcare quality standards as well as the accreditation standards is still in process, with the aim to start accreditation process at 10 hospitals in 2011. Education in the field of healthcare quality assurance, improvement and promotion has been established as a continuous process from the beginning. The Agency is member of the International Society for Quality in Health Care (ISQua) and participates in the work of the European Accreditation Network (EAN). In the field of HTA, the Agency has established international collaboration and support, which resulted in its appointment and participation in the European network for Health Technology Assessment (EUnetHTA) Joint Action Project as a EUnetHTA Partner, as well as its membership in the international society, HTAi. TAIEX project has been approved as a two-day workshop in December 2010. The Croatian HTA Guidelines have been issued with the aim to start the HTA process and reports that should serve as recommendations, as a support to policy-makers at the national level, in particular the Croatian Ministry of Health and Social Welfare, and Croatian Institute of Health Insurance, in making evidence-informed decisions on the strategic planning, investment, management and implementation of technologies in health care, on funding (reimbursement) and coverage of health technologies, and at hospital level on the request from hospital directors and policy teams. In conclusion, establishment of all these measures in Croatia is by no means an easy and quick process, however, we do believe that it is feasible through continuous and close collaboration of all those involved.

The transferability of health technology assessment: the European perspective with focus on central and Eastern European countries.

INTRODUCTION: Lower-income European countries have a worse health status and less funds for health care compared to Western Europe. Despite their limited human and financial capacities for conducting Health Technology Assessment (HTA), the need for evidence-based decision-making is growing. Two main approaches emerged as potential solutions: joint clinical assessments on the European level, and simplified procedures relying on the judgments of well-established HTA agencies of Western countries. AREAS COVERED: Based on considerations of transferability, the European Network for Health Technology Assessment (EUnetHTA) was built up to harmonize HTA methodologies across the European Union, and to develop an HTA Core Model by focusing on joint production of relative effectiveness assessment, which can be used as a basis for national value assessments. The second approach has been suggested in various forms without considering transferability issues. EXPERT OPINION: Joint clinical assessments reduce duplication of efforts based on appropriate scientific rationale. On the other hand, recent examples show that relying on judgments of HTA agencies from wealthier countries with potentially different health-care priorities can lead to suboptimal allocation decisions. In the short term, some stakeholders may benefit from ignoring transferability, but it will ultimately lead to limited access in other disease areas.

Quo Vadis HTA for Medical Devices in Central and Eastern Europe? Recommendations to Address Methodological Challenges.

Objectives: Methodological challenges in the evaluation of medical devices (MDs) may be different for early and late technology adopter countries, as well as the potential health technology assessment (HTA) solutions to tackle them. This study aims to provide guidance to Central and Eastern European (CEE) countries on how to address key challenges of HTA for MDs with special focus on the transferability of scientific evidence. Methods: As part of the COMED Horizon 2020 project, a comprehensive list of issues related to MD HTA were identified based on a targeted literature review. Health technology assessment issues which pose a greater challenge or require different solutions in late technology adopter countries were selected. Draught recommendations to address these issues were developed and discussed in a focus group. The recommendations were then validated with a wider group of experts, including HTA and reimbursement decision makers from CEE countries in May and June 2020. Results: A consolidated list of 11 recommendations were developed in 3 major areas: (1) clinical value assessment, focusing on the use of joint EU work, relying on real-world evidence, use of coverage with evidence development schemes, transferring evidence from foreign countries and addressing the challenges of learning curve and centre effect; (2) economic value assessment, covering cost calculation of complex medical devices and transferability of economic evaluations of MDs; (3) HTA processes, related to the frequent product modifications and various indications of MDs. Conclusions: Central and Eastern European countries with limited resources for conducting HTA, can benefit from HTA methods and evidence generated in early technology adopter countries. Considering the appropriate reuse of international HTA materials, late technology adopter countries can still implement HTA, even for MDs, which have a more limited evidence base compared with pharmaceuticals.

Citation analysis of the Croatian Medical Journal: the first 15 years.

The Croatian Medical Journal (CMJ) is a bimonthly scientific journal, that publishes mostly original articles. It is indexed in the Index Medicus/MEDLINE, Current Contents/Clinical Medicine, and the Science Citation Index Expanded. Since the CMJ's 15th anniversary in 2007, our aim has been to assess the importance of the Journal through its impact factor (IF) and immediacy index, with a particular focus placed on the proportion of self-citations. According the Web of Knowledge database, the current official IF for the CMJ is 0.825, ranking it 62nd out of 103 journals within the Thomson Scientific category "Medicine - General and Internal." The exclusion of self-citations resulted in a small decrease in the journal's rank - to 66th place. According to the Web of Science database, the predicted CMJ IF in 2007 is between 1.024 and 1.125, showing a clear increase. The immediacy index of the CMJ is continuously low, with a high contribution of self-citations, implying that articles published in the CMJ require more time to be cited, and that their topics are of particular interest to the journal's readers and contributors. Self-citations contributed significantly to the IF in the first few years after the journal was established. The proportion of independent citations progressively increased, and of all the citations included in the IF in 2007, almost 70% were fully independent. Some of these citations were from articles published in journals with IF higher than 5. Taken together, our data suggest that the CMJ has significantly improved its citation ratings during the last 15 years, confirming that a quality-oriented editorial policy in a small peripheral journal may result in a truly increased international visibility.

Impact of aminoglycoside cycling in six tertiary intensive care units: prospective longitudinal interventional study.

AIM: To determine the effect of aminoglycoside cycling in six tertiary intensive care units (ICU) on the rates of sepsis, aminoglycoside resistance patterns, antibiotic consumption, and costs. METHODS: This was a prospective longitudinal interventional study that measured the effect of change from first-line gentamicin usage (February 2002-February 2003) to amikacin usage (February 2003-February 2004) on the aminoglycoside resistance patterns, number of patients with gram-negative bacteremia, consumption of antibiotics, and the cost of antimicrobial drugs in 6 tertiary care ICUs in Zagreb, Croatia. RESULTS: The change from first-line gentamicin to amikacin usage led to a decrease in the overall gentamicin resistance of gram-negative bacteria (GNB) from 42% to 26% (P<0.001; z-test of proportions) and netilmicin resistance from 33% to 20% (P<0.001), but amikacin resistance did not change significantly (P=0.462), except for Acinetobacter baumanni (P=0.014). Sepsis rate in ICUs was reduced from 3.6% to 2.2% (P<0.001; chi(2) test), with a decline in the number of nosocomial bloodstream infections from 55/100 patient-days to 26/100 patient-days (P=0.001, chi(2) test). Furthermore, amikacin use led to a 16% decrease in the overall antibiotic consumption and 0.1 euro/patient/d cost reduction. CONCLUSION: Exclusive use of amikacin significantly reduced the resistance of GNB isolates to gentamicin and netilmicin, the number of GNB nosocomial bacteremias, and the cost of total antibiotic usage in ICUs.

A utility of model input uncertainty analysis in transferring tobacco control-related economic evidence to countries with scarce resources: results from the EQUIPT study.

AIMS: To inform the transferability of tobacco control-related economic evidence to resource-poor countries. METHODS: We ran a univariate sensitivity analysis on a return on investment (ROI) model, the European study on Quantifying Utility of Investment in Protection from Tobacco model (EQUIPTMOD), to identify key input values to which the ROI estimates were sensitive. The EQUIPTMOD used a Markov-based state transition model to estimate the ROI of several tobacco control interventions in five European countries (England, Germany, Spain, Hungary and the Netherlands). Base case ROI estimates were obtained through average values of model inputs (throughout the five countries), which were then replaced one at a time with country-specific values. Tornado diagrams were used to evaluate the significance of sensitivity, defined as a ≥ 10% difference in ROI estimates from the base case estimates. RESULTS: The ROI estimates were sensitive to 18 (of 46) input values. Examples of model inputs to which ROI estimates were sensitive included: smoking rate, costs of smoking-related diseases (e.g. lung cancer) and general population attributes. CONCLUSION: Countries that have limited research time and other resources can adapt EQUIPTMOD to their own settings by choosing to collect data on a small number of model inputs. EQUIPTMOD can therefore facilitate transfer of tobacco control related economic evidence to new jurisdictions.

Defining good health and care from the perspective of persons with multimorbidity: results from a qualitative study of focus groups in eight European countries.

OBJECTIVES: The prevalence of multimorbidity is increasing in many Western countries. Persons with multimorbidity often experience a lack of alignment in the care that multiple health and social care organisations provide. As a response, integrated care programmes are appearing. It is a challenge to evaluate these and to choose appropriate outcome measures. Focus groups were held with persons with multimorbidity in eight European countries to better understand what good health and a good care process mean to them and to identify what they find most important in each. METHODS: In 2016, eight focus groups were organised with persons with multimorbidity in: Austria, Croatia, Germany, Hungary, the Netherlands, Norway, Spain and the UK (total n=58). Each focus group followed the same two-part procedure: (1) defining (A) good health and well-being and (B) a good care process, and (2) group discussion on prioritising the most important concepts derived from part one and from a list extracted from the literature. Inductive and deductive analyses were done. RESULTS: Overall, the participants in all focus groups concentrated more on the care process than on health. Persons with multimorbidity defined good health as being able to conduct and plan normal daily activities, having meaningful social relationships and accepting the current situation. Absence of shame, fear and/or stigma, being able to enjoy life and overall psychological well-being were also important facets of good health. Being approached holistically by care professionals was said to be vital to a good care process. Continuity of care and trusting professionals were also described as important. Across countries, little variation in health definitions were found, but variation in defining a good care process was seen. CONCLUSION: A variety of health outcomes that entail well-being, social and psychological facets and especially experience with care outcomes should be included when evaluating integrated care programmes for persons with multimorbidity.

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.

European collaboration on relative effectiveness assessments: What is needed to be successful?

OBJECTIVE: The objective of this study is to identify the possible barriers and critical success factors for the implementation of European collaboration in the field of relative effectiveness assessment (REA) of drugs. METHODS: Data were gathered through semi-structured interviews with representatives from eight European health technology assessment (HTA) organisations involved in assessment of drugs for coverage decision-making (AAZ, AIFA, AHTAPol, HAS, HVB, IQWIG, NICE and ZiN). RESULTS: Potential barriers identified mainly relate to methodology, resources and challenges with implementation in the respective national processes (e.g. legal restrictions). The most critical success factors for production of cross-border assessments were the continuous cooperation of competent partners, and the quality and timely availability of the assessment. CONCLUSION: Further adaptation of the process and methods is required for optimal collaboration. In the near future it can be expected that cross-border assessments will meet in particular the needs of smaller/middle-sized European countries and also European countries with less developed HTA systems as the potential efficiency/quality gains are the highest for these countries. Therefore, national implementation of cross-border assessments is especially likely in these countries in the coming years. Once more experience is gained with cross-border assessments, and successes become more evident, efficiency/quality gains may also be likely for some larger countries with well established processes.