Contrasting clinical evidence for market authorisation of cardio-vascular devices in Europe and the USA: a systematic analysis of 10 devices based on Austrian pre-reimbursement assessments.

BACKGROUND: European medical device regulation is under scrutiny and will be re-regulated with stricter rules concerning requirements for clinical evidence for high-risk medical devices. It is the aim of this study to analyse the differences between Europe and USA in dealing with risks and benefits of new cardio-vascular devices. METHODS: Since no information is available on clinical data used by the Notified Body for CE-marking, data from Austrian pre-reimbursement assessments close to European market approval were used as proxy and compared with clinical data available at time of market approval by FDA in the USA. RESULTS: 10 cardio-vascular interventions with 27 newly CE approved medical devices were analysed. The time lag between market authorisation in Europe and in the USA is 3 to 7 years. Only 7 CE-marked devices also hold a FDA market approval, 7 further devices are in FDA approved ongoing efficacy trials. For 4 of the CE-marked devices the FDA market application or the approval-trial was either suspended due to efficacy or safety concerns or the approval was denied. Evidence available at time of CE-marking are most often case-series or small feasibility RCTs, while large RCTs and only in rare cases prospective cohort studies are the basis of FDA approvals. Additionally, the FDA often requires post-approval studies for high-risk devices. CONCLUSIONS: Market authorisation based on mature clinical data deriving from larger RCTs and longer follow-ups do not only change the perspective on the risk-benefit ratio, but also secures real patient benefit and safety and assures payers of investing only in truly innovative devices.

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.

Assessing the impact of health technology assessment on the Austrian healthcare system.

OBJECTIVES: In Austria, research in health technology assessment (HTA) has been conducted since the 1990s. The aim of this study is to analyze whether the HTA research program of the Institute of Technology Assessment (ITA) and the Ludwig Boltzmann Institute for HTA (LBI-HTA) have had an impact on the Austrian healthcare system. METHODS: We applied qualitative and quantitative empirical research methods, such as interviewing, download analysis, questionnaire, retrospective routine data analysis, and media analysis. Data were analyzed according to a conceptual framework, considering seven impact categories (awareness, acceptance, process, decision, practice, final outcomes, enlightenment) and different target groups. RESULTS: A rising number of downloads and single HTA reports with high media interest were identified. Interviews showed that HTA reports have increasingly been used for investment and reimbursement decisions, as well as for the preparation of negotiations. Economic impact was indicated by decreased expenditures due to HTA recommendations. Overall, knowledge about evidence-based medicine increased and, in places, an "HTA culture" can be recognized. Yet, several decision-making processes occur at all levels without the use of HTA. CONCLUSIONS: The analysis demonstrated an impact within all predefined categories; however, it depends on the system level and its target groups. HTA reports are primarily used by hospital management, (social) insurances, and the Austrian Ministry of Health. Nevertheless, there is still potential to increase the impact of HTA. Therefore, the inclusion of HTA in decision-making processes in Austria needs to move from a voluntary basis to a mandatory one.

The impact of health technology assessment reports on decision making in Austria.

OBJECTIVES: Health technology assessment (HTA) was established in Austria in the 1990s and, since then, it has gained considerable importance. In this study, we aim to analyze whether the HTA reports that have been produced at the Institute for Technology Assessment (ITA) and at the Ludwig Boltzmann Institute for HTA (LBI-HTA) have had an impact on decision making within the Austrian health care system. METHODS: We selected all reports that were intended for supporting (i) reimbursement/investment or (ii) disinvestment decisions. Eleven full HTA reports and fifty-eight rapid assessments fulfilled the inclusion criteria. We used interview data and administrative data on volumes, tariffs and expenditure of products/services to analyze whether and how reports were in reality used in decision making and what the consequences for health care expenditure and resource distribution have been. RESULTS: Five full HTA reports and fifty-six rapid technology assessments were used for reimbursement decisions. Four full HTA reports and two rapid assessments were used for disinvestment decisions and resulted in reduced volumes and expenditure. Two full HTA reports showed no impact on decision making. Impact was most evident for hospital technologies. CONCLUSIONS: HTA has played some role in reducing volumes of over-supplied hospital technologies, resulting in reduced expenditure for several hospital providers. Additionally, it has been increasingly included in prospective planning and reimbursement decisions of late, indicating re-distribution of resources toward evidence-based technologies. However, further factors may have influenced the decisions, and the impact could be considerably increased by systematically incorporating HTA into the decision-making process in Austria.

Artificial total disc replacement versus fusion for the cervical spine: a systematic review.

Cervical total disc replacement (CTDR) has been increasingly used as an alternative to fusion surgery in patients with pain or neurological symptoms in the cervical spine who do not respond to non-surgical treatment. A systematic literature review has been conducted to evaluate whether CTDR is more efficacious and safer than fusion or non-surgical treatment. Published evidence up to date is summarised qualitatively according to the GRADE methodology. After 2 years of follow-up, studies demonstrated statistically significant non-inferiority of CTDR versus fusion with respect to the composite outcome 'overall success'. Single patient relevant endpoints such as pain, disability or quality of life improved in both groups with no superiority of CTDR. Both technologies showed similar complication rates. No evidence is available for the comparison between CTDR and non-surgical treatment. In the long run improvement of health outcomes seems to be similar in CTDR and fusion, however, the study quality is often severely limited. After both interventions, many patients still face problems. A difficulty per se is the correct diagnosis and indication for surgical interventions in the cervical spine. CTDR is no better than fusion in alleviating symptoms related to disc degeneration in the cervical spine. In the context of limited resources, a net cost comparison may be sensible. So far, CTDR is not recommended for routine use. As many trials are ongoing, re-evaluation at a later date will be required. Future research needs to address the relative effectiveness between CTDR and conservative treatment.

Cost-effectiveness analysis of human papillomavirus-vaccination programs to prevent cervical cancer in Austria.

OBJECTIVE: The study predicts long-term cervical cancer related population health and economic effects of introducing the HPV-vaccination for 12-year-old girls (and boys) in addition to current screening compared with screening only. METHOD: Health effects are predicted by a dynamic transmission model. Model results are used to calculate incremental cost-effectiveness ratios (ICER) in euro per life year gained (LYG) for a time-horizon between 2008 and 2060 from a public payer and a societal perspective. RESULTS: Vaccination of girls results a discounted ICER of euro 64,000/LYG and euro 50,000/LYG from a payer's and societal perspectives respectively. The additional vaccination of boys increases the ICER to euro 311,000 and euro 299,000/LYG respectively. Results were most sensitive to vaccination price, discount rate and time-horizon. CONCLUSION: HPV-vaccination for girls should be cost-effective when adopting a longer time-horizon and a societal perspective. Applying a shorter time frame and a payer's perspective or vaccinating boys may not be cost-effective without reducing the vaccine price.

[Economic evaluation in health care]. / Gesundheitsökonomische Evaluation.

Economic evaluation of healthcare programmes helps us to assure the efficient use of healthcare resources. Cost-minimisation analysis, cost-effectiveness analysis, cost-utility analysis and cost-benefit analysis are the most commonly applied evaluation types. They differ in how they measure outcomes of healthcare interventions. Several guidelines for conducting economic evaluations exist. Furthermore, checklists have been developed for systematic critical appraisal of economic evaluations. Even though economic evaluations increase the transparency of resource allocation decisions and make decision criteria more explicit, they do not replace public discussions on the availability of overall resources for the public health care system.

[LDL apheresis for the treatment of familial hypercholesterolemia. A systematic review]. / Low-Density-Lipoprotein-Apherese bei familiärer Hypercholesterinämie. Eine systematische Ubersicht.

BACKGROUND AND PURPOSE: Low-density lipoprotein (LDL) apheresis is indicated in patients with familial hypercholesterolemia who are resistant to drug therapy. The study evaluates whether the various apheresis techniques available result in relevant improvement of clinical endpoints (cardiovascular morbidity and mortality, quality of life). MATERIAL AND METHODS: A systematic literature review in standard databases (Medline, EMBASE, CRD York, Cochrane, and others) was conducted. Extraction of core data, assessment of study quality and evidence rating according to the GRADE method were undertaken in an internal peer-review process. RESULTS: Evidence from the ten studies included weakly indicates an improvement in angina symptoms and reduced mortality. However, due to limited study quality, no reliable answers are possible concerning the question whether LDL apheresis results in reduced cardiovascular morbidity/mortality or improved quality of life. CONCLUSION: The available evidence indicates a possible net benefit of LDL apheresis. However, since the quality of the evidence is very low, treatment should be strictly restricted to patients with severe familial hypercholesterolemia who are resistant to standard care.

Is it worth investing in mental health promotion and prevention of mental illness? A systematic review of the evidence from economic evaluations.

BACKGROUND: While evidence on the cost of mental illness is growing, little is known about the cost-effectiveness of programmes in the areas of mental health promotion (MHP) and mental disorder prevention (MDP). The paper aims at identifying and assessing economic evaluations in both these areas to support evidence based prioritisation of resource allocation. METHODS: A systematic review of health and non health related bibliographic databases, complemented by a hand search of key journals and analysis of grey literature has been carried out. Study characteristics and results were qualitatively summarised. Economic evaluations of programmes that address mental health outcome parameters directly, those that address relevant risk factors of mental illness, as well as suicide prevention interventions were included, while evaluations of drug therapies were excluded. RESULTS: 14 studies fulfilled the inclusion criteria. They varied in terms of topic addressed, intervention used and study quality. Robust evidence on cost-effectiveness is still limited to a very small number of interventions with restricted scope for generalisability and transferability. The most favourable results are related to early childhood development programmes. CONCLUSION: Prioritisation between MHP and MDP interventions requires more country and population-specific economic evaluations. There is also scope to retrospectively add economic analyses to existing effectiveness studies. The nature of promotion and prevention suggests that innovative approaches to economic evaluation that augment this with information on the challenges of implementation and uptake of interventions need further development.

[Informal care of people with mental disorders: does the Austrian long-term care system provide adequate support?]. / Informelle Betreuung psychisch erkrankter Menschen: Schafft das osterreichische Pflege- vorsorgesystem adäquate Voraussetzungen?

OBJECTIVE: The Austrian long-term care system covers all types of long-term chronic diseases and handicaps and is based on a payment for care scheme. The benefit is directed to care recipients, who are - in the outpatient sector - largely free in how to use it. Herewith, the payment for care scheme also has a significant impact on the provision of informal care. The paper studies this impact for the particular case of informal care provided for mentally ill people. METHODS: The analysis is based on a questionnaire survey of persons that are providing informal care to mentally ill people in Austria. The data is confronted with results from other studies on informal care provision in Austria. RESULTS: The study shows specific characteristics of informal long-term care for people with mental illness which is associated with specific burdens for the informal carers. Carers and care recipients benefit only to a moderate extent from public long-term care benefits and bear comparably high material and social costs. CONCLUSIONS: The cash-oriented long-term care system in Austria offers only limited support for the particular case of informal care provided for mentally ill people. The long-term care system needs to be tailored to the special needs of both carer and care recipient in order to achieve the stated aim of self-determination and freedom of choice.

Distributional impacts of mental health care financing arrangements: a comparison of the UK, Germany and Austria.

BACKGROUND: Although mental health care has undergone substantial re-structuring processes, little attention has been paid to financing issues during these processes. AIMS OF THE STUDY: In this paper the authors seek to examine distributional effects of systems of mental health care financing in the UK, Germany and Austria against the backdrop of ongoing reforms and broader welfare state transformations. METHOD: The article is based on secondary data on mental health care reform processes and financing arrangements. Distributional issues are studied a) on the macro-level related to the sectors 'state', 'market', 'family' and 'voluntary/community' and b) on the individual level. RESULTS AND DISCUSSION: In all of the three countries, shifts towards community care have resulted in a new division of financing responsibilities with a tendency to rising responsibilities for the 'family' and the 'voluntary/community sector'. In addition, strengthening market principles often increases financial burdens for affected individuals and/or their relatives. The study has been limited by the lack of precise data on resource allocation and expenditure for mental health care. IMPLICATIONS FOR HEALTH POLICIES, HEALTH CARE PROVISION AND USE: An increasing focus on the relationship between financing and service provision is required in order to prevent new forms of social exclusion of people with mental disorders. IMPLICATIONS FOR FURTHER RESEARCH: Further research needs to be carried out to increase transparency concerning the complex relationship between provision and the finance of mental health care. In terms of distributional impacts, it needs to be analysed in more detail which persons are affected in which ways. This particularly includes users as well as carers in the formal and informal sector.

["Suddenly, no more money was left". The role of financing in the Austrian mental health care reform]. / "Dann war auf einmal kein Geld mehr da!". Zur Rolle der Finanzierung in der österreichischen Psychiatriereform.

OBJECTIVE: The article studies the role of financing mental health care for the mental health care service structure in the process of moving towards a patient-oriented and decentralised mental health care system. METHOD: The analysis is based on a description of the Austrian mental health care financing system and a discourse-analytical examination of reform documents and interviews with key actors in this country. RESULTS: Existing structures of mental health care services are a reflection of mental health care financing structures. Reform goals are in various forms linked to financing issues. However, an explicit discussion of the finance issues in reform documents is widely missing. CONCLUSIONS: Adapting the finance of mental health care to new paradigms of mental health care provision requires not just technical modifications, but also improved transparency of processes and implications involved.

Incentives in financing mental health care in Austria.

BACKGROUND: In Austria, financing health care -and even more so mental health care- is characterized by a mix of federal and provincial responsibilities, lack of uniformity in service provision and service providers, and diverse funding arrangements. The division between financing structures for health care and social care makes the situation even more complex. This state of affairs results in various, partly counterproductive and sometimes paradoxical financial incentives and disincentives for the providers, recipients and financiers of mental health services. In several provinces of Austria, recent reform plans in mental health care have focused strongly on establishing community-based and patient-oriented mental health care. One of the main challenges in implementing this new policy is the re-allocation of resources. AIMS OF THE STUDY: The authors hypothesize that the existing structure of mental health care financing, with its incentives and disincentives, constitutes an obstacle to patient-oriented community-based mental health care. Analyzing the characteristics of the overall mental health care financing system in one Austrian province, Lower Austria, will provide a better understanding of actor-relationships and inherent incentives and highlight implications for the process of deinstitutionalization. METHOD: The authors used an analytical framework based on the principal-agent theory, empirical evidence, and information on financial, organizational and legal structures to identify the characteristics of actor-relationships and the position of single actors within the system. RESULTS: The article shows how incentives are linked to existing constellations of actors involved in mental health care financing and identifies significant power relations. As a consequence, incentives and disincentives within the financing system result in hospital- centered and supply-oriented mental health care in Lower Austria. DISCUSSION: The current system of financing mental health care provides an obstacle to the provision of patient-oriented and community-based mental care. This is due to existing constellations and power relations among the actors where, most importantly, patients are the weakest party in the patient-payer-provider triangle. Balancing power relations will be a significant prerequisite for alternative financing systems. IMPLICATIONS FOR HEALTH POLICIES AND FURTHER RESEARCH: If a community and needs-based mental health care system is to be established in Austria, the financing structures have to be changed accordingly. Applying a principal-agent framework is useful for identifying key aspects in mental health care financing in relation to the provision of services. Further research is needed to help develop alternative financing mechanisms that support community-based and patient-oriented mental health care systems.