Background: Quality of life is recognized as an important additional outcome measure in clinical trials and health economic evaluations. The EQ-5D is an important generic health outcome instrument often used for economic evaluations as a complement with disease-specific outcome measures. In this study quality of life data was assessed using the EQ-5D-Y (new EQ-5D version for children and adolescents) and the Cystic Fibrosis Questionnaire (CFQ). The objective of the study is to evaluate the cross-sectional validity of the EQ-5D-Y as a generic health outcome instrument in children and adolescents with cystic fibrosis in Germany.

Costs of outpatient medication for CF patients significantly depend on age, co-morbidities and other clinical parameters. Hence, non-optimal treatment could lead to a significantly higher burden for the healthcare system.

In March 2010 a National Action League for People with Rare Diseases (NAMSE) was founded. The NAMSE created a national plan of action for people with rare diseases for improving medical care in the field of rare diseases which was approved by the Federal Government in August 2013. Thus, two important areas of the research report have already been implemented. In a comparison of the areas of activity of the research report with those of the National Action Plan it becomes clear that priorities will be in the context of health services research in rare diseases, for example the introduction of centres of reference for rare diseases, measures to accelerate the diagnostic process and the promotion of research and information management in the future.

Citizen science projects in health-related research usually follow a crowdsourcing approach where laypersons primarily have a supplying role in data collection. By contrast, this article presents an approach on a much higher engagement level (co-creation) where a team of professional and citizen scientists jointly plans, implements, and evaluates a scientific study on a chronic disease from which the citizen (patient) scientists themselves suffer. We call this approach patient science; it systematically makes use of the patients’ expertise of living with the disease. This article describes the pilot project and conceptual differences compared to other participatory approaches in medical and health research. It elaborates on the implications of involving chronically ill people as co-researchers and, finally, reflects on the benefits and challenges of patient science.

International audienceObjective Objective of this study is to assess and evaluate resource use in outpatient treatment in Germany and to compare it with remuneration. Methods Outpatient treatment was evaluated in seven different centers for pediatric and adult CF patients. Data was recorded during one representative month in 2006. A micro-costing approach was used to value resource use data. Results For outpatient treatment mean costs (excluding drugs) of 488 € per patient per quarter occurred. Correlation analysis identified significant cost drivers including age and co-morbidities (pancreatic insufficiency, hepatobiliary complications, lung function capacity or bacterial lung colonization). Remuneration covered only 51% of the total costs (252 € per patient/quarter). Conclusions As the human resources available to these centres today are already below the requirements set by the European consensus for standards of CF care it will be important for of a high level of patient care to reach a cost-covering remuneration scheme