[Decisions of the German Federal Joint Committee in situations with insufficient data : Decision-making options for new diagnostic and treatment methods]. / Entscheidungen des Gemeinsamen Bundesausschusses bei unzureichender Datenlage : Entscheidungsoptionen bei neuen Untersuchungs- und Behandlungsmethoden.

BACKGROUND: In accordance with legal requirements, the Federal Joint Committee (German: Gemeinsamer Bundesausschuss, G­BA) decides based on the best available evidence which new diagnostic and treatment methods are reimbursed by statutory health insurance. If the benefit is proven with sufficient certainty, statutory health insurance providers pay for the new method, otherwise a trial study must be conducted. OBJECTIVES: To present the G­BA's decision-making options even in the case of insufficient evidence in the field of urology. MATERIALS AND METHODS: A document search was conducted on the homepage of the G­BA for the decisions about method evaluation and quality assurance in the field of urology of the last 10 years. The respective decisions are presented in the light of the decision options available in each case. RESULTS: Using the example of the debate on low-dose rate brachytherapy for localised prostate cancer, the years-long, ultimately futile, effort to increase the evidence base for an innovative method is presented. CONCLUSION: Compared to the development of guidelines, for example, the G­BA can only make dichotomous yes/no decisions and has to accept the often insufficient evidence situation, or (has to) try to increase the evidence base by initiating its own study. The latter is particularly difficult when specific methods are already established in routine care. A particular challenge is posed by new, especially invasive methods in the hospital sector, which has to be evaluated (benefit assessment) and, if necessary, tested by the G­BA with a trial study. To what extent this will succeed in the future is not yet foreseeable.

[10 years of assessing prevention and early diagnosis programmes by the Federal Joint Committee of Germany - a methodological analysis]. / Entscheidungen des Gemeinsamen Bundesausschusses zu Früherkennungsuntersuchungen - methodische Analyse der Beschlüsse im Zeitraum 2000 bis 2009.

Medical benefit is the crucial factor in the evidence-based decision-making process of the Federal Joint Committee (G-BA) of Germany. To review the decisions on population-based screening methods from January 2000 to December 2009, we analysed the coverage decisions. During this period of time, the G-BA decided on 14 screening methods, predominantly dealing with the early diagnoses of serious diseases. In 8 cases a positive assessment led to reimbursement by the statutory health insurance funds, in 5 cases the assessment was negative and in one case the conditions were changed. For 7 assessments, a fully reported review was available, 4 of this were positively evaluated. In 2 of these cases, controlled screening studies documented the benefit of the screening method. In another case there was only a case series without a control group. For the fourth method, the question for the benefit of the screening method was divided into 3 sub questions, investigated separately in different studies. The methods of benefit assessments have changed very much over time. A revision of the rules of procedure for the assessment of medical screening methods is advisable.

[A short-term diagnostic and economic impact model of neonatal screening for cystic fibrosis]. / Modellierung der kurzfristigen diagnostischen und ökonomischen Auswirkungen eines Neugeborenen-screenings auf Mukoviszidose.

BACKGROUND: Screening for cystic fibrosis (CF) is currently not a part of the neonatal screening program in Germany, but its implementation is being debated. The aim of this study was to model the short-term diagnostic and economic consequences of the implementation of such a screening. PATIENTS: Cohort of all newborns in Germany per year. METHODS: In total, 3 screening strategies were evaluated by decision modelling. The 3 screening protocols compared were alternative combinations of IRT- and DNA-testing with a final sweat test. RESULTS: All modelled screening strategies show a comparable diagnostic yield (171-175 CF-cases per year). There is a tendency of slightly higher sensitivity and shorter time to diagnosis for the "IRT-DNA with failsafe" protocol. In comparison to other strategies, the total number of screening-related visits for this protocol is lower, whereas the amount of sweat tests is the highest. All screening strategies are comparable in terms of avoided cases of extremely low height- or weight-for-age (37-38 and 13-14 cases, respectively). The incremental annual diagnostic costs (with deducted diagnostic costs without screening) are 1.13 to 1.16 million EUR. After 3 years of model runtime there is a nearly neutral total budget impact of screening (diagnostic and treatment costs) per cohort. CONCLUSION: All screening strategies lead to a considerable reduction of time to diagnosis at acceptable financial expenditures. The findings of this study broaden the current basis for discussion regarding the implementation of cystic fibrosis screening in Germany.

[Assessment of benefit and efficiency of innovative medical devices]. / Nutzen- und Wirtschaftlichkeitsbewertung der biomedizinischen Technik.

Medical devices cover a wide spectrum of products with very different diagnostic and therapeutic applications. However, for market licensing, uniform rules apply. Uniform rules are also valid for coverage decisions in German health care. In this article, the criteria for the assessment of benefit and efficiency of innovative biomedical technologies are described from the perspective of the statutory health insurance system. The key concepts relevant in the mandatory health insurance' "innovation"' "benefit", and "economic efficiency" are characterized. Only measurable effects of an intervention which lead to a more than marginal improvement in prognosis, symptoms, or quality of life as compared to a standard treatment are considered as beneficial. An innovative device is, therefore, subject to a benefit assessment if it is not yet reimbursed (or not yet part of the benefit package), when it is relevant to the health care system and a high public interest exists. In addition, it is important to consider a positive benefit assessment as a part of the value added chain to avoid conflicts of interest. Within the scope of early technology assessment, some conclusions can already be drawn in the early developmental stage of a device.

[Minimally invasive surgery. Economic analyses from the view point of the sickness funds]. / Minimal-invasive Chirurgie. Okonomische Analysen aus der Sicht der Kostenträger.

Minimally invasive surgery (MIS) claims to lead to less pain, faster recovery and/or healing, smaller scars and a shorter hospital stay, ideally in combination with lower costs. This article discusses which aspects of the economic evaluation of MIS, from the perspective of the statutory health insurance (SHI), are relevant. The economic perspective of the statutory health insurance primarily comprises the expenditures for illness (e.g. medical treatment, drugs, rehabilitation, sick-pay), i.e. direct costs only. Indirect costs (i.e. productivity losses) remain unconsidered. Thus, the perspective of the SHI describes only a reduced view of reality. However, on the basis of proven efficacy, cost-minimisation and cost-benefit analyses for certain questions are conceivable from the perspective of the SHI. Results from such analyses can generate important information for the calculation of tariffs and negotiation of contracts.

[The German Network for Evidence-based Medicine -- DNEbM]. / Das Deutsche Netzwerk Evidenzbasierte Medizin -- DNEbM.

The German Network for Evidence-based Medicine (DNEbM) was founded in October 2000. A major impetus for the development of evidence-based medicine (EBM) in Germany came in the mid-1990s from the German Cochrane Initiative, which referred to the principles and activities of the international Cochrane Collaboration. The Network's primary mission is to establish a competence and reference centre for all aspects of evidence-based medicine. Central to this mission is to encourage projects that are concerned with scientific and methodological problems, aspects of professional development and the question of how to translate EBM into clinical practice. With currently more than 600 individual members the DNEbM is the most important society concerned with EBM in the German-speaking region in Europe. Until now, the DNEbM has initiated the development of curricula for EBM and health technology assessment (HTA) for undergraduate and continuing medical education. In addition, position papers regarding hot topics such as prostate cancer screening with PSA or registration of randomised controlled trials were published. The Network's HTA section comprises experts from universities, institutes, health care administration and industry. Currently, the HTA section is developing an Internet-based course for users and doers of HTA reports in Germany.

Evaluation of preventive technologies in Germany: case studies of mammography, prostate cancer screening, and fetal ultrasound.

OBJECTIVE: In this article, three preventive strategies-mammography screening for breast cancer, PSA screening for prostate cancer, and routine ultrasound in normal pregnancy-are discussed in the context of German health care. METHODS: Epidemiologic data and German studies evaluating different aspects of these preventive measures were identified and analyzed. RESULTS: Only a few studies could be identified that investigate these preventive measures. Despite sufficient evidence, in part derived from a German study, there is not yet a mammography screening program. In contrast, ultrasound in pregnancy is offered routinely, although there are controversies regarding the benefit of this practice. PSA screening is not offered as part of the screening program for prostate cancer. However, PSA tests as well as mammographies are done in large numbers in German ambulatory care-a practice that could be considered wild or opportunistic screening. CONCLUSIONS: These case studies show that preventive programs and practices in Germany are not sufficiently based on sound evidence. The paucity of evaluation activities related to prevention in Germany is probably due to the low threshold to introduce new preventive programs into the German healthcare system in the past.

What is 'best practice' in health care? State of the art and perspectives in improving the effectiveness and efficiency of the European health care systems.

A framework for the classification of information on maintaining or improving effectiveness and efficiency in health care systems is proposed. Activities, disciplines and methods that are available to identify, implement and monitor the available evidence in health care are called 'best practice'. We reviewed the literature in order to (1) establish a definition for 'best practice' in the health sector, (2) develop a framework to classify relevant information, and (3) synthesise the literature on activities, disciplines and methods pertinent to the concept. Health care, public health activities and health policy should be advised by the best available evidence. Currently, the concept can be broken down into three activities (Health Technology Assessment (HTA), Evidence-Based Medicine (EBM), Clinical Practice Guidelines (CPGs)) by which evidence is synthesised either as an evidence base (EBM and most HTA) or in the form of recommendations (CPGs and some HTA) for different decision purposes in health care. These activities gain input mainly through four disciplines: clinical research, clinical epidemiology, health economics and health services research. The different disciplines are related to each other in three 'domains': (a) input, (b) dissemination/implementation and (c) monitoring/outcome. These provide evidence on (a) the (potential) effects of health care interventions and policies; (b) on ways to implement them; and (c) on ways to monitor their actual outcome. None of these separate approaches and activities exclusively forms a successful and all-embracing strategy to ascertain 'best practice'. A collective approach in the management of information is expected to add value to individual efforts. Resources should be devoted to increase quality and quantity of both primary and secondary research as well as the establishment of networks to synthesise, disseminate, implement and monitor 'best practice'.

[Rapid assessments"--quick evaluation of medical technology]. / "Rapid Assessments"--schnelle Bewertung medizinischer Technologien.

Assessing the clinical, economical and other consequences of medical technologies (health technology assessment, HTA) is an important instrument to support decisions in many health care systems. A comprehensive HTA, however, is time-consuming and costly while, on the other hand, decisions have to be made quickly. A number of international HTA programmes established rapid assessment tracks although they are variable in scope and methods. In the first part of this paper, these programmes were compared with respect to scope, methods and time to complete assessments. It can be shown from this comparison that there is no common definition of "rapid assessments". In the second part of the paper, a model for processing rapid assessment in the German context is introduced and discussed. The model aims at rapid assessments serving the needs of German decision-makers and ensuring high scientific quality at the same time. The model consists of a modular system that is tailored to the actual demand of the decision-maker. Modules are obligatory (such as a systematic literature search) or optional (such as meta-analysis). All modules are subject to standardisation as far as possible. However, it should be kept in mind that a close collaboration between commissioners and executives of an HTA is necessary to focus on the question and work out the HTA accordingly.

[Not Available]. / Health Technology Assessment (HTA), evidenzbasierte Medizin (EbM) Alter Wein in neuen Schläuchen? : Alter Wein in neuen Schläuchen?

Health Technology Assessment (HTA) defines the systematic analysis of short and long-term consequences of the application of medical technologies with the aim of supporting decisions in policy-making and practice. The aim of directly supporting decision-making at a political level is the major difference to evidence-based medicine and guideline development. In Germany, HTA is being established only since the nineties. In this period, however, important steps for a permanent establishment of HTA in the German health service have been undertaken. One of these steps was the German HTA project, which was funded from 1995 to 2001 by the Federal Ministry of Health. Beginning in 2001, this initiative will be relaunched by the German Institute for Medical Documentation and Information (DIMDI) as a regular HTA program. The bodies managing the German health care system rely increasingly on HTA reports when deciding on coverage of health technologies. HTA thus proves to be an instrument, which could be of benefit in the optimisation of the health care system.

[Not Available]. / HTA des biochemischen Screenings für fetale Chromosomenanomalien und Neuralrohrdefekte.

Biochemical screening during pregnancy - used since the early nineties in different combinations to detect fetal numerical chromosomal abnormalities (e.g. Down Syndrome) and open neural tube defects (spina bifida) - still remains controversial for a variety of reasons. Therefore a health technology assessment (HTA) was carried out to determine the medical effectiveness of the screening method. Systematic searches were performed in publication and project lists of international technology assessment institutions and in different biomedical and HTA-specific databases. The publications had to accomplish predefined criteria and were reviewed by check lists. Eighteen systematic reviews, meta-analyses and decision analyses as well as nine primary studies were used for the study. The data analyzed are highly consistent with regard to sensitivity and specificity of different combinations of biochemical and sonographical markers and stages of pregnancy. The detection rate (sensitivity) in second trimester screening averages 60% to 65% (false positive rate: 5%) when a screening covers maternal age, AFP, hCG, and uE3. Sensitivity stays the same when the screening includes Inhibin A as a fourth parameter. The detection rate in first trimester is 70% to 75% (false positive rate: 5%) on average when a combinatorial screening on maternal age, fetal nuchal translucency, PAPP-A, and free ß-hCG is performed. The more common second trimester screening may cause ethical problems, because termination of pregnancy, which may be asked for by women in case of an existing fetal chromosomal abnormality, has to be performed at an advanced gestational age. Actually there are new molecular technologies which promise a significant higher screening sensitivity and will allow faster results to be obtained.

["Best practice" in health care--or why we need evidence-based medicine, guidelines and health technology assessment]. / "Best Practice" im Gesundheitswesen--oder warum wir evidenzbasierte Medizin, Leitlinien und Health Technology Assessment brauchen.

In this article we describe an interdisciplinary approach that aims at developing a framework for the definition of best practice in health care. Its focus is on Health Technology Assessment (HTA), Evidence-based Medicine (EBM) and Clinical Practice Guidelines (CPG), each of which being approaches that increasingly rely on systematic reviews of the best available evidence. The concept is based on an extensive literature search comprising key articles on HTA, EBM and CPG and all relevant related disciplines (e.g. clinical epidemiology, outcomes research). The origin of the term "best practice" is also traced. The framework for the definition of best practice in health care is an iterative loop. It consists of the overriding concept of health care based on reliable evidence. In this concept, HTA, EBM and CPG are related to each other in three "domains", input, implementation and outcome. Information that constitutes evidence is delivered through disciplines and evaluation methods in health care to the input domain. The implementation domain refers to methods to effectively translate evidence into practice as well as to implementation barriers. The outcome domain considers methods to monitor best practice. This implies that indicators of performance at different levels of health care have to be identified which could serve as measures of success. The outcome domain may serve as information source for the input domain (iterative loop). Research recommendations are centered around improving availability and use of evidence (including systematic reviews), and promoting existing activities in best practice.

Health technology assessment in Germany. Status, challenges, and development.

The objectives of this paper are to describe the status and development of health technology regulation with regard to coverage decisions and utilization, and to analyze the current situation of health technology assessment (HTA) in Germany. The relevant literature for controlling health technologies was identified by searching the literature and databases and through personal contacts. The literature was analyzed with regard to the different sectors in the healthcare system. For the analysis of the current state of HTA in Germany, a national survey was carried out. In addition, the names of topics under assessment were collected. The results show that coverage decisions in the ambulatory sector appear to be much more regulated than those in the inpatient sector. The same is true for diffusion and usage of technologies. The strict separation of the hospital and the ambulatory care sector in Germany constitutes a barrier to regulation and to making HTA an effective instrument in Germany. Until recently, HTA in Germany focused on biotechnology, such as gene technology. Recently the German Scientific Working Group of Technology Assessment in Health Care has adopted a systematic methodology to undertake HTAs. It can be concluded that regulation of health technologies in Germany is characterized by inconsistencies ranging from strict regulation in the ambulatory sector to almost no regulation in the rehabilitation sector. Increasing interest in HTA, in conjunction with a high priority assigned to evaluation of health technologies by the newly elected government, is likely to improve this situation in the future.

[Determining priorities in the development of medical guidelines. 1: Criteria, procedures and actors: a methodological review of international experiences]. / Prioritätensetzung bei der Entwicklung medizinischer Leitlinien. Teil 1: Kriterien, Verfahren und Akteure: eine methodische Bestandsaufnahme internationaler Erfahrungen.

Setting priorities for the development of clinical practice guidelines has--similar to other decision-making procedures in health care--as much a political as a scientific component. Prioritizing guidelines aims to allocate resources to those health problems likely to maximize medical, social and economic outcomes associated with the use of these guidelines. This is a review and critical appraisal of international initiatives of systematically setting priorities for the development of clinical practice guidelines. Priority-setting criteria, both quantitative and qualitative methods as well as participation by relevant stakeholders will be discussed. This review provides possible decision-makers with an information basis which may assist in the development of concepts for setting priorities in a given context.

Regulation of health-related technologies in Germany.

Against the background of a financial crisis and supposed inefficiencies due to inappropriate use or the use of ineffective technologies in the German health care system, increasing awareness of the role of coverage decisions and the use of health technologies has stimulated interest in the regulation of health technologies. A systematic analysis of the decision processes at the levels of licensing/market admission, coverage by statutory health insurance and steering of diffusion and usage reveals considerable inconsistencies in different health care sectors. With regard to different types of technology, an explicit licensing procedure conforming to international standards is required for drugs and medical devices. Concerning coverage decisions, the ambulatory sector appears to be much more regulated than the in-hospital sector. This applies also to diffusion and usage of technologies. However, steering of usage of health related technologies is generally weak in Germany since only non-binding guidelines are in place.

[Health technology assessment in the international context: where does Germany stand? For the "German Scientific Working Group Technology Assessment for Health care"]. / Health Technology Assessment im internationalen Kontext: Wo steht Deutschland? Für die "German Scientific Working Group Technology Assessment for Health Care".

The evaluation of medical technologies, Health Technology Assessment (HTA), is gaining more and more importance in Germany. Starting from a comprehensive definition of the term medical technology, this article first describes the development of HTA since the seventies as an instrument for improving decision-making in health policy. HTA is presented as a repetitive cycle of a sequence of steps starting with the prioritization of topics to be evaluated to the analysis of the impact of the results. Current trends in the further improvement of HTA methods are described, such as the formalization of the topic selection. The current situation in Germany is presented with regard to the regulation of licensing/market entry, coverage by the statutory health insurance, and the utilization of medical technologies. Finally, current activities in the field of HTA in Germany as well as the role of the German Scientific Working Group Technology Assessment for Health Care are discussed.

Cardiac catheterization in Germany. Diffusion and utilization from 1984 to 1996.

OBJECTIVE: To describe the diffusion of cardiac catheterization technologies and time trends of their use according to setting and geographic region in Germany during a 13-year period. It is hypothesized that the cardiac catheterization technology has matured from an experimental state to a broadly accepted technology. METHODS: Data come from the annual survey of the German Society for Cardiovascular Research. All German cardiac catheterization units are requested to provide data on volume and type of catheterization procedures. Data are available from 1984 to 1996. Number and type of procedure, type of unit, diagnoses, and complications are all recorded. The overall response rate is 90%, on average. RESULTS: The total number of catheterization units was 324 in 1996, or an average of 3.69 units per 1 million population. In 1996, all of the East German Länder and districts were below average. Utilization of cardiac catheterization procedures increased exponentially during the study period. The number of angiographies rose from about 45,000 in 1984 to more than 450,000 in 1996; the number of angioplasties increased almost by a factor of 50 to 125,000 procedures in 1996. Inverse correlations between the rates per million population of either coronary angiographies or PTCAs and mortality rates from ischemic heart disease were observed at the level of the German Länder. CONCLUSION: Further studies taking patient characteristics, long-term outcomes, and other factors in account are necessary to clarify the large geographic variations and the negative relationship between utilization rates and coronary heart disease mortality found in this study.

[Methodological quality of clinical practice guidelines in Germany: results of a systemic assessment of guidelines presented on the Internet]. / Methodische Qualität ärztlicher Leitlinien in Deutschland. Ergebnisse einer systematischen Untersuchung deutscher Leitlinienberichte im Internet

Critics claim that most of the German clinical practice guidelines are of poor quality having produced by informal ad hoc methodologies without a rigorous approach. This paper reports on the systematic appraisal of 329 guidelines published online by the Association of the Scientific Medical Societies (AWMF) in Germany. The results of this study suggest that most of the guidelines presented in Internet do not meet internationally recognised criteria for quality. Proposals are offered how to enhance the methodological quality of future guidelines.