Matthias Perleth

Gemeinsame Bundesausschuss (G-BA) · Medical Consultancy

Objectives: To clarify the concept of disruptive technologies in health care, provide examples and consider implications of potentially disruptive technologies for health technology assessment (HTA). Methods: We conducted a systematic review of conceptual and empirical papers on healthcare technologies that are described as "disruptive." We sear...

Part II: Factors that Enable or Inhibit HTA Impact Assessment Activities in HTA Agencies This report presents the results of an intensive qualitative investigation into the factors that enable or inhibit HTA agencies in their implementation of HTA impact assessment. In this study, a social cognitions lens is used to uncover the sources of support f...

Part I: HTA Impact Assessment Practices in INAHTA Member Agencies In this report, the findings of an environmental scan of INAHTA member agencies are presented. The detailed impact assessment practices, including the types of decisions informed by HTAs, the indicators of HTA impact that are assessed, and methods and tools used to assess impact are...

Introduction: The Federal Joint Committee (G-BA) is the highest decision-making body of the self-governing health care system in Germany [1]. Aside numerous responsibilities the G-BA performs an (additional) benefit assessment (see § 35a SGB V) for all newly approved drugs including drugs treating rare diseases (so-called orphan drugs [OD]). The e...

Objective Medical devices (MDs) encompass a broad and heterogeneous range of technologies. While practices vary considerably across countries, MDs often find application in patient care with little or no evaluation of their effectiveness and safety following market approval. A recently proposed taxonomy of MDs considered devices from the viewpoint...

Background The Federal Joint Committee (Gemeinsamer Bundesausschuss, G-BA) is the highest decision-making body of the joint self-government in Germany [1]. It is comprised of associations of office-based physicians, dentists, hospitals, health insurance funds and patients’ representatives. The G-BA issues legally binding directives and thus determ...

Health Technology Assessment is one of the standard instruments in support of the decision-making to define the public health services both internationally and in the German health care system. Besides systematic reviews, benefit-harm-analyses, health economic evaluations, and, decision-analytic modelling, especially epidemiological and biometrical...

Introduction Health technology assessment (HTA) agencies wish to ensure the impact of their HTAs. HTA impact assessment measures the influence of a HTA on decision-making and downstream to patient outcomes. Despite their potential to provide insights, the use of impact assessment frameworks by HTA agencies is limited. Understanding the underlying m...

This article examines the current status and most important changes over time to the legislative framework on the health technology assessment-informed decision-making process on diagnostic and therapeutic ‘methods’ in Germany. The relevant information was obtained through documentary analysis covering the period 1990 to 2017. The findings show tha...

Health Technology Assessments (HTAs) liefern für zahlreiche Entscheidungen im Gesundheitswesen relevante Informationen. Die Erstellung Matthias Perleth1 Petra Schnell-Inderst2,3 von HTA-Berichten erfordert gut ausgebildete, interdisziplinär arbeitende Alric Rüther4 Spezialisten, die angemessene Interpretation und Umsetzung in Entscheidungen erforde...

Background: The aim of this study was to examine the scientific evidence on clinical effectiveness and safety used in health technology assessments (HTAs) of high-risk medical devices (MDs) in Europe. Methods: We applied a systematic approach to identify European institutions involved in HTA and to select reports assessing MDs considered high-ri...

Hintergrund Der Gemeinsame Bundesausschuss (G-BA) führt für alle neu zugelassenen Arzneimittel eine (Zusatz)Nutzenbewertung durch (vgl. § 35a SGB V). Für Arzneimittel zur Behandlung seltener Erkrankungen (sogenannte Orphan Drugs [OD]) gelten einige Sonderregeln. Methodik Anhand öffentlich zugänglicher Dokumente auf der Homepage des G-BA sowie der F...

Background: The aim of this study was to assess the quality of reporting sample size calculation and underlying design assumptions in pivotal trials of high-risk medical devices (MDs) for neurological conditions. Methods: Systematic review of research protocols for publicly registered randomized controlled trials (RCTs). In the absence of a publish...

Objectives: Health Technology Assessment (HTA) of Medical devices (MDs) and MD-based procedures can be challenging due to the unique features and particularities of this group of technologies, such as device-operator interaction. The aim of this study was to (1) clarify, and supplement earlier findings on European HTA institutions' structural, pro...

Dieses Lehrbuch bietet eine Einführung ins Business Planning für die Gesundheitswirtschaft – einem wichtigen Baustein der erfolgreichen Übertragung neuer medizinischer Erkenntnisse in die klinische und Public-Health-Praxis. Dabei schlägt es eine Brücke von der Frage, ob eine Innovationsidee eine gesellschaftlich wünschenswerte Verwendung knapper Re...

Objectives: The suitability of general HTA methodology for medical devices is gaining interest as a topic of scientific discourse. Given the broad range of medical devices, there might be differences between groups of devices that impact both the necessity and the methods of their assessment. Our aim is to develop a taxonomy that provides research...

Background Incremental cost-effectiveness and cost-utility analyses [health economic evaluations (HEEs)] of vaccines are routinely considered in decision making on immunization in various industrialized countries. While guidelines advocating more standardization of such HEEs (mainly for curative drugs) exist, several immunization-specific aspects (...

Funding of diagnostic and therapeutic methods in Germany's statutory health insurance (SHI) follows a dichotomy: in outpatient care, only methods with proven benefit are reimbursed while in inpatient care, all methods may be provided unless they are excluded due to proven harm or lack of benefit. In January 2012, a new section 137e was added to the...

Unlabelled: Presenting continuous outcomes in Summary of Findings tables presents particular challenges to interpretation. When each study uses the same outcome measure, and the units of that measure are intuitively interpretable (e.g., duration of hospitalisation, duration of symptoms), presenting differences in means is usually desirable. When t...

Since 1989 a periodic health examination beginning at the age of 35 for the early detection of "common diseases" (especially cardiovascular and kidney diseases as well as diabetes) by means of history-taking, physical examination, blood and urine tests and counselling has been available in Germany. Altogether, the respective directive of the Federa...

In Summary-of-Findings-Tabellen wird für jeden der sieben (oder weniger) wichtigsten Endpunkte folgendes dargestellt: die Anzahl der Studien und die Anzahl der Teilnehmer, das Vertrauen in die Effektschätzer (Qualität der Evidenz) sowie die besten Schätzungen der absoluten und relativen Effekte. Möglicherweise schwierig gestaltet sich bei der Erste...

The aim of the present systematic review was to evaluate the evidence of a potential learning curve for laser treatment of benign prostatic hyperplasia. A systematic literature search was conducted in November 2011 using The Cochrane Library, EMBASE and MEDLINE. Overall, 18 sources (mostly case series) were included for further assessment. The majo...

Last September, the European Commission published proposals to update regulations for medical devices in order to improve patient safety.1 The proposals are being discussed by the European parliament where critical debate is being led by the Committee on Environment, Public Health and Food Safety. Medical devices range from bandages to life support...

GRADE requires guideline developers to make an overall rating of confidence in estimates of effect (quality of evidence—high, moderate, low, or very low) for each important or critical outcome. GRADE suggests, for each outcome, the initial separate consideration of five domains of reasons for rating down the confidence in effect estimates, thereby...

Summary of Findings (SoF) tables present, for each of the seven (or fewer) most important outcomes, the following: the number of studies and number of participants; the confidence in effect estimates (quality of evidence); and the best estimates of relative and absolute effects. Potentially challenging choices in preparing SoF tables include using...

In the GRADE approach, randomised trials start as high-quality evidence and observational studies as low-quality evidence, but both can be rated down if most of the relevant evidence comes from studies that suffer from a high risk of bias. Well-established limitations of randomised trials include failure to conceal allocation, failure to blind, los...

This article deals with inconsistency of relative, rather than absolute, treatment effects in binary/dichotomous outcomes. A body of evidence is not rated up in quality if studies yield consistent results, but may be rated down in quality if inconsistent. Criteria for evaluating consistency include similarity of point estimates, extent of overlap o...

Direct evidence comes from research that directly compares the interventions in which we are interested when applied to the populations in which we are interested and measures outcomes important to patients. Evidence can be indirect in one of four ways. First, patients may differ from those of interest (the term applicability is often used for this...

In the GRADE approach, randomized trials are classified as high quality evidence and observational studies as low quality evidence but both can be rated down if a body of evidence is associated with a high risk of publication bias. Even when individual studies included in best-evidence summaries have a low risk of bias, publication bias can result...

GRADE suggests that examination of 95% confidence intervals (CIs) provides the optimal primary approach to decisions regarding imprecision. For practice guidelines, rating down the quality of evidence (i.e., confidence in estimates of effect) is required when clinical action would differ if the upper versus the lower boundary of the CI represented...

Background: This is an update of a Cochrane Review first published in The Cochrane Library in Issue 1, 2005 and previously updated in 2007 and 2009.Idiopathic sudden sensorineural hearing loss (ISSHL) is common and has a significant effect on quality of life. Hyperbaric oxygen therapy (HBOT) may improve oxygen supply to the inner ear and result in...

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, predominantl...

This article is the first of a series providing guidance for the use of the GRADE system of rating quality of evidence and grading strength of recommendations in systematic reviews, health technology assessments, and clinical practice guidelines addressing alternative management options. The GRADE process begins with asking an explicit question, in...

GRADE requires a clear specification of the relevant setting, population, intervention, and comparator. It also requires specification of all important outcomes - whether evidence from research studies is, or is not, available. For a particular management question, the population, intervention, and outcome should be sufficiently similar across stud...

This article introduces the GRADE approach to rating the quality of evidence. GRADE specifies four categories (high, moderate, low, and very low) that are applied to a body of evidence, not to individual studies. In the context of a systematic review, quality reflects our confidence that the estimates of the effect are correct. In the context of re...

In the GRADE approach, randomised trials start as high-quality evidence and observational studies as low-quality evidence, but both can be rated down if most of the relevant evidence comes from studies that suffer from a high risk of bias. Well-established limitations of randomised trials include failure to conceal allocation, failure to blind, los...

The most common reason for rating up the quality of evidence is a large effect. GRADE suggests considering rating up quality of evidence one level when methodologically rigorous observational studies show at least a two-fold reduction or increase in risk, and rating up two levels for at least a five-fold reduction or increase in risk. Systematic re...

In this article we describe how to include considerations about resource utilisation when making recommendations according to the GRADE approach. We focus on challenges with rating the confidence in effect estimates (quality of evidence) and incorporating resource use into evidence profiles and Summary of Findings (SoF) tables. GRADE recommends tha...

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. Cohort of all newborns in Germany per year. In total, 3 screening strategi...

With the aim to study the level of evidence on which coverage decisions of the Federal Joint Committee (G-BA) were made and how the G-BA deals with missing or insufficient evidence, we analysed the final reports of coverage decisions after benefit assessments (outpatient care) between 1.1.1998 and 31.10.2008. A total of 36 decisions were analysed....

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...

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...

With the aim to study the level of evidence on which coverage decisions of the Federal Joint Committee (G-BA) were made and how the G-BA deals with missing or insufficient evidence, we analysed the final reports of coverage decisions after benefit assessments (outpatient care) between 1.1.1998 and 31.10.2008. A total of 36 decisions were analysed....

The assessment of the transferability, sometimes called external validity, of study results is subordinate to the assessment of the internal validity of clinical studies. Although internal and external validity seem to be related they actually are fundamentally different concepts. While assessing the transferability or generalisability of study res...

To provide an overview of the development of health technology assessment (HTA) in Germany since the 1990s. Analysis of key documents (e.g. literature, laws, and other official documentation) and personal experiences. Health technology assessment (HTA) entered the political agenda in Germany only in the mid-1990s, basically as the result of a top-d...

The assessment of the transferability, sometimes called external validity, of study results is subordinate to the assessment of the internal validity of clinical studies. Although internal and external validity seem to be related they actually are fundamentally different concepts. While assessing the transferability or generalisability of study res...

Compared to the assessment of efficacy, effectiveness, safety and costs, the analysis of the organisational impact of medical technologies has often been neglected. In addition, no international consensus has been reached so far about the definition of what is meant by the organisational impact of a medical technology. This article integrates diffe...

Legal, ethical, socio-cultural and organisational aspects may play a decisive role in HTA-supported decision-making and, therefore, should not be left unconsidered. However, these aspects are not regularly discussed in practice. In contrast to current practice, a systematic information basis for these aspects should be created which calls for suita...

Background Testosterone levels decline gradually with age in men. A number of manifestations such as reduced libido or depression have been attributed to subphysiological testosterone levels. Hence, testosterone supplementation has been proposed in these cases. Objective To assess the efficacy and safety of testosterone supplementation therapy in...

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...

Testosterone levels decline gradually with age in men. A number of manifestations such as reduced libido or depression have been attributed to subphysiological testosterone levels. Hence, testosterone supplementation has been proposed in these cases. To assess the efficacy and safety of testosterone supplementation therapy in hypogonadal healthy me...

The aim of this study was to identify research and development on genetic testing to find out if research addresses important disease areas, how far it is from potential clinical use, and what consequences might arise for the prioritization of health technology assessment (HTA) activities. Also a horizon scanning methodology developed in Germany is...

Die minimal-invasive Chirurgie (MIC) führt nach Ansicht ihrer Verfechter zu weniger Schmerzen, schnellerer Erholung bzw. Heilung, kleineren Narben und kürzerer Aufenthaltsdauer im Krankenhaus, idealerweise kombiniert mit geringeren Kosten. In diesem Beitrag wird dargestellt, welche Aspekte der ökonomischen Evaluation der MIC aus der Perspektive der...

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 t...

Zusammenfassung Das Deutsche Netzwerk Evidenzbasierte Medizin (DNEbM) wurde im Oktober 2000 gegründet. Wesentliche Impulse für die Entwicklung der evidenzbasierten Medizin (EbM) kamen bereits Mitte der 1990er-Jahre aus der deutschen Cochrane Initiative, die die Prinzipien und Aktivitäten der 1993 gegründeten internationalen Cochrane Collaboration a...

The following article contains six brief statements defining health technology assessment (HTA) and a commentary. It aims at clarifying the definition, backgrounds, tasks and importance of HTA.

Information derived from Health Technology Assessment (HTA) reports is increasingly used for decision-making in healthcare. The production of HTA reports requires well-qualified interdisciplinarily working specialists who are not yet available in sufficient number. The German HTA Society and the German Network for Evidence Based Medicine have now d...

Um die Perspektive der Kostenträger transparenter zu machen, werden in dem Beitrag zwei Fragenkomplexe erörtert: 1. Wie werden Entscheidungen in der Selbstverwaltung der Ärzte und Krankenkassen getroffen? 2. Wie würden die Kostenträger zu ihren Entscheidungen gelangen, wenn sie alleine entscheiden könnten? Zum 1. Fragekomplex: Der Gemeinsame Bundes...

Health Technology Assessment (HTA) is defined as a form of policy analysis which evaluates systematically short and long-term consequences of the application of a medical technology, a group of related technologies or technology-related aspects on the basis of published studies. The goal of HTA is the support of decisions in politics and practice....

[Executive Summary] The aim of Working Group 4 has been to develop and disseminate best practice in undertaking and reporting assessments, and to identify needs for methodologic development. Health technology assessment (HTA) is a multidisciplinary activity that systematically examines the technical performance, safety, clinical efficacy, and effe...

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) establis...

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.

Aims The purpose of this report is to define the role that health technology assessment (HTA; a discipline in which both health outcomes and associated economic variables are investigated) can play in determining the effects of various anticoagulation regimens after mechanical heart valve replacement. Method and results A review of literature, incl...

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 progr...

Die Bewertung medizinischer Technologien (Health Technology Assessment, kurz HTA) hat sich in vielen Gesundheitssystemen als wichtiges Instrument der Entscheidungsunterstützung etabliert. Oft handelt es sich dabei um eine systematische, mehrdimensionale Evaluation von Nutzen, Kosten und sonstigen Effekten der Anwendung medizinischer Technologien. E...

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. Epidemiologic data and German studies evaluating different aspects of these preventive measures were identified and analyzed. Only a f...

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...