[CF Lung Disease - a German S3 Guideline: Pseudomonas aeruginosa]. / S3-Leitlinie: Lungenerkrankung bei Mukoviszidose ­ Pseudomonas aeruginosa.

Cystic Fibrosis (CF) is the most common autosomal recessive genetic multisystemic disease. In Germany, it affects at least 8000 people. The disease is caused by mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene leading to dysfunction of CFTR, a transmembrane chloride channel. This defect causes insufficient hydration of the airway epithelial lining fluid which leads to reduction of the mucociliary clearance.Even if highly effective, CFTR modulator therapy has been available for some years and people with CF are getting much older than before, recurrent and chronic infections of the airways as well as pulmonary exacerbations still occur. In adult CF life, Pseudomonas aeruginosa (PA) is the most relevant pathogen in colonisation and chronic infection of the lung, leading to further loss of lung function. There are many possibilities to treat PA-infection.This is a S3-clinical guideline which implements a definition for chronic PA-infection and demonstrates evidence-based diagnostic methods and medical treatment in order to give guidance for individual treatment options.

[The potentials of patient surveys for quality assurance in medical care]. / Die Potenziale von Patientenbefragungen für die Qualitätssicherung in der medizinischen Versorgung.

What constitutes "quality" in medical care and the means by which it is controlled, secured, or (re-)established has largely been assigned to the joint self-administration of service providers and health insurers in Germany. The current implementation uses an understanding of quality that does not sufficiently take into account important patient-centered quality dimensions. One of the reasons for this might be that patient surveys are not yet recognized as an equivalent data source for quality assessment in the German healthcare context and are not yet established nationwide. Where patient survey instruments are used, they are often reduced to the survey of so-called patient satisfaction. In the future, survey instruments must be viewed and used in a much more differentiated manner.Indication-specific patient surveys on treatment results (patient-reported outcome measures; PROMs) can provide information on the success of treatment in certain diseases. With the targeted collection of patient experiences (patient-reported experience measures; PREMs), the process quality can also be determined. The routine use of corresponding patient surveys is provided according to §§ 136 ff. and § 299 Social Security Code Book V (SGB V) for the purposes of legally binding external quality assurance. Their use is intended to support the alignment of the health system towards value and benefit for patients. To this extent, there is an urgent need for implementation and considerable further development.

[Reliable health information for patients with rare diseases : Quality demands defined by the National Action Plan and how they are put into practice]. / Verlässliche Gesundheitsinformationen zu seltenen Erkrankungen : Die Anforderungen des Nationalen Aktionsplans und deren praktische Umsetzung.

Information for patients with rare diseases has to adhere to strict quality criteria in order to support individual treatment decisions or coping strategies. However, developers are facing specific challenges: For example, the evidence is often insufficient or of very low quality. In the context of the National Action League for People with Rare Diseases (NAMSE), criteria have been developed that assure high-quality information on rare diseases. Core criteria comprise the involvement of patients or their advocates in all stages of the development process, the systematic search and assessment of the evidence, systematic collection of patient experience, transparency in terms of people involved and funding, and nondirective and neutral formulation of content and documentation of the process. In a joint project between the Alliance for Chronic Rare Diseases (ACHSE e. V.) and the German Agency for Quality in Medicine (ÄZQ), ten short information leaflets on different rare diseases have been developed in the past three years, conceived to show the applicability of these criteria. First experiences with this format show that the criteria are adaptive to a broad range of diverse rare diseases and settings. Involving patients and their advocates throughout the whole development process - from prioritization to development of methods and provision of patient experience and coping strategies - is crucial. Insufficient evidence remains a challenge. The examples show that in the absence of proven findings, information that matters to patients and reflects this uncertainty is feasible.

[The guidelines of the Federal Joint Committee on acute pain management : Background and consequences for the practice in hospitals]. / Die Vorgaben des Gemeinsamen Bundesausschusses zum Akutschmerzmanagement : Hintergründe und Konsequenzen für die Praxis im Krankenhaus.

The quality of postoperative pain therapy in Germany shows a heterogeneous treatment practice and large differences in quality between individual institutions, The patient representatives in the Federal Joint Committee (G-BA) have therefore decisively campaigned for many years that instruments of non-legislative standards are employed in order to noticeably improve the quality of perioperative pain therapy for patients in Germany. As a result of these efforts, in October 2020 a binding specification for internal quality management was included in the quality management guidelines (QM-RL) by the G­BA. This describes in concrete terms the structural and procedural requirements for an internal quality management of acute pain for all institutions in which operations and comparable potentially painful interventions are carried out. This article describes the content of this regulation and the resulting consequences for the institutions, the medical and administrative management and especially the role of anesthesia.

[White Paper - Improving the care of patients with impairments following sepsis and infections]. / White Paper ­ Verbesserung der Versorgungs- und Behandlungsangebote für Menschen mit Sepsis- und Infektionsfolgen.

Hundreds of thousands of individuals who experience lasting sequelae after sepsis and infections in Germany do not receive optimal care. In this White Paper we present measures for improvement, which were developed by a multidisciplinary expect panel as part of the SEPFROK project. Improved care rests on four pillars: 1. cross-sectoral assessment of sequelae and a structured discharge and transition management, 2. interdisciplinary rehabilitation and aftercare with structural support, 3. strengthening the specific health literacy of patients and families, and 4. increased research into causes, prevention and treatment of sequelae. To achieve this, appropriate cross-sectoral care structures and legal frameworks must be created.

[Patient orientation and patient participation in research: Deficits, modes and expectations from a patient representative's point of view]. / Patientenorientierung und Patientenbeteiligung in der Forschung ­ Mängel, Formen und Erwartungen aus Sicht eines Patientenvertreters.

The article addresses deficits in patient orientation and patient involvement in medical research from a patient's perspective and provides recommendations for their further development. Researchers often practice patient orientation as well as patient involvement in an unstructured and inconsistent manner. The decision if and how patient involvement takes place is, to a considerable extent, the researchers' responsibility. This decision should always be part of a patient-oriented alignment of the research project, which researchers pursue regardless of the existence, initiative and participation of a patient organization. Possible modes of involvement are assigned to different phases of the research process. In this article, patient orientation and the choice of the form of involvement is considered to constitute an obligation of researchers and decision makers early in the process. Involvement should take place on a fair basis. Decision makers should consider determining specific requirements for patient orientation, e. g. in their calls for proposals or selection/award criteria.

[Participation of patients in the program for national disease management guidelines--current state and implications]. / Patientenbeteiligung am Programm für Nationale VersorgungsLeitlinien - Stand und Konsequenzen.

Patient involvement has been implemented in the Program for National Disease Management Guidelines since 2005. Currently patient/consumer participation is being incorporated in terms of patients' comments of consultation papers on National Disease Management Guidelines (NDMG) and in the development of NDMG-based patient guidelines (PG). The editorial activities in patient guideline development from the beginnings to its publication are conducted in close cooperation with the patient representatives appointed by the Patient Forum. Between June 2005 and September 2006, three NDMG and three patient guidelines on asthma, chronic obstructive pulmonary disease (COPD) and chronic coronary artery disease (CAD) were produced by including patients in the guideline development process. The information provided in these guidelines is freely accessible at http://www.versorgungsleitlinien.de. The present contribution focuses on the development of patient guidelines. It describes the current state of patient involvement and joint work and indicates the implications that can be derived from patient participation in the NDMG Program. Accompanying the involvement procedures, experiences resulting from previous NDMG and PG development activities are continuously investigated for the possibility of further methodological development of consumer participation by a work group of the Patient Forum in coordination with the patient organizations involved. In particular, the procedures resulting from more intensive patient participation in patient guideline development are to be examined as to their relevance for the expansion of patient involvement in NDMG development.

[Shared decision-making based on equal information. Patient guidelines as a tool for patient counseling]. / Auf einheitlicher Basis entscheiden: die Rolle von NVL-basierten PatientenLeitlinien.

In discussions on the quality of cross-sectorial health-care services high importance is attributed to patient education and patient counseling, with guideline-based patient information being considered a crucial tool. Guideline-based patient information is supposed to serve patients as a decision-making basis and, in addition, to also support the implementation of the guidelines themselves. The article highlights how patient guidelines for National Disease Management Guidelines in Germany--within the scope of patient education and patient counseling--may provide a uniform information platform for physicians and patients aiming to promote shared decision-making. The authors will also address the issue which contents should be included in patient guidelines in order to meet these requirements and which measures are required to review their quality. The present paper continues the series of articles on the Program for German National Disease Management Guidelines.

Gene therapy for choroideremia: in vitro rescue mediated by recombinant adenovirus.

Choroideremia (CHM) is an X-linked retinal degenerative disease resulting from a lack of functional Rab Escort Protein-1 (REP-1). As a first step in developing gene-based therapies for this disease, we evaluated the feasibility of delivering functional REP-1 to defective lymphocytes and fibroblasts isolated from individuals with CHM. A recombinant adenovirus delivering the full-length human cDNA encoding REP-1 under the control of a cytomegalovirus promoter was generated. Adenovirus-mediated delivery of REP-1 rescued the defective cells as assessed through protein and enzymatic assays. Ultimately, it may be possible to use virus-mediated delivery of REP-1 to evaluate disease intervention in vivo.

[Patient involvement in clinical guideline development: are patient organisations prepared for this task?]. / Patientenbeteiligung an der Leitlinienentwick- lung--sind die Patientenorganisationen für diese Aufgabe gerüstet?

The involvement of patients in the development of clinical guidelines essentially aims at ensuring and improving the quality of patient-centred care. Hence, it becomes an important tool for quality management in medicine since patients are learning the hard way where clinical care is lacking. This may include the inappropriate consideration of current medical knowledge, the unintelligible or insufficient information and education of the patient or information gaps at the interface between care settings. These experiences from the patients' perspective can be purposefully integrated in quality assurance measures, for example, by including the patient perspective in clinical guidelines and patient guidelines. Suitable procedures for the collection and presentation of the experiences of patient organisations are essential for the successful involvement of patients in guideline programmes. Patient organisations collect data on the experiences and attitudes of their members for different purposes. A systematic approach has been sought but hardly practised so far. This is the result demonstrated in a survey among 112 member organisations of the "Bundesarbeitsgemeinschaft SELBSTHILFE von Menschen mit Behinderung und chronischer Erkrankung und ihren Angehörigen e.V." (BAG SELBSTHILFE), a federal German self-help association of disabled and chronically ill people and their relatives. Patient participation in the "Programm for Nationale VersorgungsLeitlinien" ["Programme for National Disease Management Guidelines"] has been practised at the Agency for Quality in Medicine (AEZQ) since 2005. The experiences that have been made by those involved with integrating the concerns of patient representatives provide a starting point for a practical handout for patients and consumers participating in guideline programmes. The "Handbuch Patientenbeteiligung-Beteiligung am Programm für Nationale VersorgungsLeitlinien" [Handbook of Patient Participation in the Programme for National Disease Management Guidelines] is intended to make the process of patient participation in guideline programmes transparent and practicable. It describes the background, the organisation and the process of patient participation and offers useful advice to participating patient organisations and their representatives. In this context the question of how patient experiences might be introduced in the guideline and patient guideline development process in a structured and purposeful manner is of special importance. But so far there is too little experience, both nationally and internationally. A survey among self-help organisations shall provide an initial overview.

[From uncertainty to solidarity--promoting evidence in rare diseases through physician-patient interaction]. / Von der Unsicherheit zum solidarischen Handeln--Evidenz-Förderung in der Arzt-Patient-Interaktion am Beispiel Seltener Erkrankungen.

With rare diseases the evidence base is often poor. The reasons are, among other things: (a) strict separation of evidence users (i.e. physicians and patients) and evidence producers (i.e. scientists); (b) lack of methods and procedures for the inclusion of unused evidence sources in medical care (e.g. documentation of individual disease development, subjective patient experiences); (c) low level of referral to specialized physicians; (d) lack of rewards for the disclosure of unused but relevant evidence sources. The development of suitable methods, aids and incentives should also focus on the individual physician-patient interaction. Even today medical care suppliers can positively influence the patients' motivation for participating in the continuous documentation of the disease process. Adequate management of the medical records is not only useful for individual care, but--with suitable procedures like registers being available--on a collective level also. It should be checked which alterations in incentive systems and supporting programmes might be able to improve evidence-promoting documentation and analysis.