Published and unpublished evidence in coverage decision-making for pharmaceuticals in Europe: existing approaches and way forward.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

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.

Evidence-informed recommendations to reduce dissemination bias in clinical research: conclusions from the OPEN (Overcome failure to Publish nEgative fiNdings) project based on an international consensus meeting.

Background: Dissemination bias in clinical research severely impedes informed decision-making not only for healthcare professionals and patients, but also for funders, research ethics committees, regulatory bodies and other stakeholder groups that make health-related decisions. Decisions based on incomplete and biased evidence cannot only harm people, but may also have huge financial implications by wasting resources on ineffective or harmful diagnostic and therapeutic measures, and unnecessary research. Owing to involvement of multiple stakeholders, it remains easy for any single group to assign responsibility for resolving the problem to others.

[GRADE guidelines: 6. Rating the quality of evidence: imprecision].

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.

High-dose chemotherapy followed by autologous stem cell transplantation for metastatic rhabdomyosarcoma--a systematic review.

Introduction: Patients with metastatic rhabdomyosarcoma (RMS) have a poor prognosis. The aim of this systematic review is to investigate whether high-dose chemotherapy (HDCT) followed by autologous hematopoietic stem cell transplantation (HSCT) in patients with metastatic RMS has additional benefit or harm compared to standard chemotherapy.

Methods: Systematic literature searches were performed in MEDLINE, EMBASE, and The Cochrane Library.

Autologous hematopoietic stem cell transplantation following high-dose chemotherapy for non-rhabdomyosarcoma soft tissue sarcomas.

Background: Soft tissue sarcomas (STS) are a highly heterogeneous group of rare malignant solid tumors. Non-rhabdomyosarcoma soft tissue sarcomas (NRSTS) comprise all STS except rhabdomyosarcoma. In patients with advanced local or metastatic disease, autologous hematopoietic stem cell transplantation (HSCT) applied after high-dose chemotherapy (HDCT) is a planned rescue therapy for HDCT-related severe hematologic toxicity.

CoCanCPG. Coordination of cancer clinical practice in Europe.

All European countries are facing common challenges for delivering appropriate, evidence-based care to patients with cancer. Despite tangible improvements in diagnosis and treatment, marked differences in cancer survival exist throughout Europe. The reliable translation of new research evidence into consistent patient-oriented strategies is a key endeavour to overcome inequalities in healthcare.

How pre- and postnatal risk factors modify the effect of rapid weight gain in infancy and early childhood on subsequent fat mass development: results from the Multicenter Allergy Study 90.

Background: It is unclear which exposures may cause or modify the adverse effect of rapid weight gain on fat mass development in term children whose birth weight is appropriate-for-gestational age (AGA).

Objective: To determine which intrauterine or postnatal exposures increase the risk of or modify the effect of rapid weight gain on body fat percentage (BF%) and body mass index (BMI) trajectories between 2 and 6 y of age.

Design: Term AGA singletons (n = 370) from the German Multicenter Allergy Study (MAS-90), a longitudinal birth cohort study, with repeated anthropometric measurements until 6 y, and data on breastfeeding status, exposure to smoking during pregnancy, and maternal anthropometric and socioeconomic characteristics were included in this analysis.

History of respiratory infections in the first 12 yr among children from a birth cohort.

Respiratory infections are the most frequent health problem in childhood. There is little precise information on how many respiratory illness episodes can be expected in a normal child. This study was designed to create reference values for the frequency of respiratory infections as recordable by history.

Gastro esophageal reflux disease is associated with absence from work: results from a prospective cohort study.

Aim: To study the association of gastro-esophageal reflux disease (GERD) with the absence from work and to estimate the extent of loss in gross domestic product due to inability to work.

Methods: Analysis was based on the prospectively gathered data of a large European cohort study involving 6 215 symptomatic GERD patients (ProGERD). Among these patients, 2 871 were initially employed.

Regional variation in medication following coronary events in Germany.

Background: Mortality rates from ischaemic heart disease have consistently been higher in East compared to West Germany both prior to and since reunification. Coronary care is inversely related to mortality from ischaemic heart disease. The objective of the present study was, therefore, to compare cardiovascular medication in East and West German patients following cardiac rehabilitation.

European survey on circadian variation of angina pectoris (ESCVA) in treated patients.

Background And Purpose: Previous reports of a circadian variation of angina pectoris were based primarily on selected patients of clinical studies. The present ESCVA Study (European Survey on Circadian Variation of Angina Pectoris) was designed to determine the timing of angina pectoris attacks in outpatients, the association of wake time and possible external triggers with angina attacks, and the influence of cardiac medication on the circadian pattern.

Patients And Methods: Inclusion criteria were stable angina pectoris for at least 3 months, average frequency of two or more attacks per week, and treatment with on-demand nitrates.

Risk factors for erosive esophagitis: a multivariate analysis based on the ProGERD study initiative.

Objectives: Gastroesophageal reflux disease can be divided into three categories: nonerosive GERD (NERD), erosive GERD (ERD), and Barrett's esophagus. A shift among these categories rarely occurs. The aim of the present study was to elucidate potential patient-associated risk factors associated with ERD.

[Return to work after cardiologic rehabilitation].

Objectives: The objectives of the present study were to determine prospectively return to work and its predictors in patients after cardiac rehabilitation.

Methods: Patients were enrolled at admission to inpatient cardiac rehabilitation centres (n = 18). Primary indications for admission were myocardial infarction, coronary artery bypass grafting or percutaneous transluminal coronary angioplasty.

Regional trends in cerebrovascular mortality in Germany after unification (1990-1999).

Background And Purpose: After the unification in 1990 two different health and political systems merged in Germany. Our aim was to analyze trends in mortality from cerebrovascular diseases in the formerly divided western and eastern parts of Germany since the unification.

Methods: Trends in mortality were determined by analyzing age-adjusted vital statistics data obtained from the Federal Statistics Office.

Comparing methodological quality and consistency of international guidelines for the management of patients with chronic heart failure.

Background: Guidelines (GLs) for the management of heart failure (HF) are of great importance in order to define and disseminate therapeutic recommendations based on scientific evidence. The aim was to analyse and to compare the methodological quality of HF GLs as well as to evaluate the consistency of therapeutic recommendations.

Methods: Eleven international GLs for the management of chronic HF were identified by search of the internet, electronic databases and references of published literature.

Messages from the German Multicentre Allergy Study.

Several birth cohort studies have been initiated during the past two decades to study environmental and genetic risk factors for atopic dermatitis, asthma and allergic rhinitis. This article summarizes results from the German Multicentre Allergy Study (MAS), which has followed children (initially 1,314) from birth (in 1990) to the present time. The effects of immunizations, allergen exposure, early sensitization patterns as well as upper airway infections on the subsequent development of asthma and atopy at school age are described.

The development of childhood asthma: lessons from the German Multicentre Allergy Study (MAS).

Epidemiological surveys have indicated that there has been a notable increase in the prevalence of both asthma and other allergic symptoms in children and young adults. Since it seems unlikely that genetic factors would contribute to the rising trend, environmental factors might play a major part in the development of childhood asthma. In a prospective birth-cohort study, we assessed the relevance of different exposures such as mite and cat allergen exposure, environmental tobacco smoke (ETS) exposure, early infectious diseases and vaccinations for the development of childhood asthma up to the age of 10 years.

How should a birth cohort study be organised? Experience from the German MAS cohort study.

Birth cohort studies offer the opportunity to study average risks, rates and occurrence times of disease longitudinally from birth. The effect of genetic and environmental factors and their interactions can be studied. Furthermore, quantity and duration of exposure to environmental agents can be evaluated prospectively.