[Extrapolation of data to inform system-level decisions of the Federal Joint Committee]. / Extrapolation bei Systementscheidungen im G-BA.

The evaluation procedures of the Federal Joint Committee (G-BA) often raise the question of whether and under what conditions findings can be transferred to other areas of application or interventions. Additional information is required in each case to test transferability. On the basis of selected examples from the decision-making practice of the G-BA, the complex weighing processes are outlined and the need for transparent illustration of the reasons for decisions is pointed out. The criteria, which in any case must be examined, cannot be described conclusively. Depending on the concrete question - method, pharmaceutical - further criteria may be relevant. A systematic approach still has to be developed so that the complex weighing processes could be simplified by transfer assumptions with sufficient certainty.

Evidence-based decision-making for diagnostic and therapeutic methods: the changing landscape of assessment approaches in Germany.

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 that, even if the outpatient care sector appears to be much more regulated than the inpatient sector (based on a strict separation of the two care settings), developments in Germany have led to a more tightened assessment framework, making the use of evidence a firm component in the decision-making process. Nevertheless, a comprehensive approach for a systematic assessment of diagnostic and therapeutic 'methods' still does not exist. Readjustments of current regulations in Germany, such as the existing 'Verbotsvorbehalt' (i.e. provision of a diagnostic and therapeutic 'method' possible unless actively delisted) in the inpatient care setting, as well as further developments at the European level are needed in order to create a system that ensures early access to innovation under controlled study conditions.

Published and unpublished evidence in coverage decision-making for pharmaceuticals in Europe: existing approaches and way forward.

BACKGROUND: Dissemination bias occurs when only some results emerging from clinical research reach their intended audience in the knowledge translation process. Given that coverage decisions increasingly rely on evidence, it is important to explore the types of evidence considered. This paper aimed to examine the evidence base used by regulatory institutions involved in pricing and reimbursement of pharmaceuticals in a broad range of European countries, as well as their awareness of and approach towards dissemination bias. METHODS: A mixed methods approach was adopted. Regulatory documents and published literature were identified in systematic searches and relevant documents were analysed. An online survey was carried out to verify and expand insights. RESULTS: Forty-two relevant regulatory documents and 10 publications were included. The survey had a 35% response rate, yielding valid responses for 13 countries. A fragmented impression was obtained for most countries indicating a general lack of transparency regarding both processes of decision-making and approaches towards unpublished information. Dissemination bias was rarely consistently considered. Practices for the identification and inclusion of all available evidence varied considerably, as did the influence of missing evidence on decision-making. Differences were often attributable to the regulatory context and/or institutional principles. CONCLUSIONS: Best practice is difficult to generalize given the identified variations. Individual exemplary practices support the necessity for institutional exchange at international level. Increased institutional commitment to transparency of methods and processes should be advocated.

From market access to patient access: overview of evidence-based approaches for the reimbursement and pricing of pharmaceuticals in 36 European countries.

BACKGROUND: Coverage decisions determining the benefit baskets of health systems have been increasingly relying on evidence regarding patient benefit and costs. Relevant structures, methodologies, and processes have especially been established for pharmaceuticals but approaches differ. The objective of this work was thus to identify institutions in a broad range of European countries (n = 36) in charge of determining the value of pharmaceuticals for pricing and reimbursement purposes and to map their decision-making process; to examine the different approaches and consider national and supranational possibilities for best practice. METHODS: Institutions were identified through websites of international networks, ministries, and published literature. Details on institutional practices were supplemented with information from institution websites and linked online sources. RESULTS: The type and extent of information available varied considerably across countries. Different types of public regulatory bodies are involved in pharmaceutical coverage decisions, assuming a range of responsibilities. As a rule, the assessment of scientific evidence is kept structurally separate from its appraisal. Recommendations on value are uniformly issued by specific committees within or commissioned by responsible institutions; these institutions often also act as decision-makers on reimbursement status and level or market price. While effectiveness and costs are important criteria in all countries, the latter are often considered on a case-by-case basis. In all countries, manufacturer applications, including relevant evidence, are used as one of the main sources of information for the assessment. CONCLUSION: Transparency of evidence-based coverage decisions should be enhanced. International collaboration can facilitate knowledge exchange, improve efficiency of information production, and strengthen new or developing systems.

[Training courses in Evidence-based Medicine at the Federal Joint Committee]. / Fortbildungskurse zu Themen der Evidenzbasierten Medizin im Gemeinsamen Bundesausschuss.

Since 2006 the office of the Federal Joint Committee (FJC) has been offering training courses in Evidence-based Medicine (EbM) to its delegates. The courses were initiated due to a comprehensive and far-reaching restructuring of the FJC. For example, in 2004 it was decided to involve patient representatives in the discussions. Furthermore, the enactment of the cross-sectoral Code of Procedure in 2005 led to the removal of sectoral differences in the evaluation of the benefit of diagnostic and therapeutic methods. The main aim of the training courses is to build a common understanding of EbM in the interdisciplinary boards of the FJC. From the first course in 2006 until the middle of 2012, 385 people participated in these courses. Since many of them attended more than one course, the total number of participants is 663. In 2011, the courses were certified as the "Basic EbM-Course" by the German Network for Evidence-Based Medicine. The results of the course feedback and the continuing demand for further courses confirm their successful implementation and demonstrate that teaching EbM to groups with a fairly heterogeneous academic and professional background is advantageous.

[GRADE guidelines: 5. Rating the quality of evidence: publication bias]. / GRADE Leitlinien: 5. Einschätzung der Qualität der Evidenz - Publikationsbias.

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 in substantial overestimates of effect. Authors should suspect publication bias when available evidence comes from a number of small studies most of which have been commercially funded. A number of approaches based on examination of the pattern of data are available to help assess publication bias. The most popular of these is the funnel plot; all, however, have substantial limitations. Publication bias is likely frequent, and caution in the face of early results, particularly with small sample size and number of events, is warranted.