Norbert Schmacke

EVITA: a tool for the early evaluation of pharmaceutical innovations with regard to therapeutic advantage.

BackgroundNew drugs are generally claimed to represent a therapeutic innovation. However, scientific evidence of a substantial clinical advantage is often lacking. This may be the result of using inadequate control groups or surrogate outcomes only in the clinical trials. In view of this, EVITA was developed as a user-friendly transparent tool for the early evaluation of the additional therapeutic value of a new drug.MethodsEVITA does not evaluate a new compound per se but in an approved indication in comparison with existing therapeutic strategies. Placebo as a comparator is accepted only in the absence of an established therapy or if employed in an add-on strategy on top. The evaluation attributes rating points to the drug in question, taking into consideration both therapeutic benefit and risk profile. The compound scores positive points for superiority in efficiency and/or adverse effects as demonstrated in randomized controlled trials (RCTs), whilst negative points are awarded for inferiority and/or an unfavorable risk profile. The evaluation follows an algorithm considering the clinical relevance of the outcomes, the strength of the therapeutic effect and the number of RCTs performed. Categories for therapeutic aim and disease severity, although essential parts of the EVITA assessment, are attributed but do not influence the EVITA score which is presented as a color-coded bar graph. In case the available data were unsuitable for an EVITA calculation, a traffic-type yield sign is assigned instead to criticize such practice. The results are presented online http://www.evita-report.de together with all RCTs considered as well as the reasons for excluding a given RCT from the evaluation. This allows for immediate revision in response to justified criticism and simplifies the inclusion of new data.ResultsAs examples, four compounds which received approval within the last years were evaluated for one of their clinical indications: lenalidomide, pioglitazone, bupropion and zoledronic acid. Only the first achieved an EVITA score above zero indicating therapeutic advantage.ConclusionsThe strength of EVITA appears to lie in its speedy assessment of the potential therapeutic advantage of a new drug for a given indication. At the same time, this approach draws attention to the typical deficits of data used for drug approval. EVITA is not intended to replace classical health technology assessment reports but rather serves as a screening tool in the sense of horizon scanning.

Attitudes Toward Postmenopausal Long-Term Hormone Therapy

In this article we address the question of why postmenopausal women undergo hormone therapy. Thirty-five women aged 46 to 75 years living in Bremen (Germany) and taking postmenopausal hormones for at least 12 months were interviewed. Following Fritz Schütze, the interviews were analyzed according to a reconstructive analytical procedure. Five different types of users were identified. They differed from each other in terms of their reasons for using hormones, their expectations of this type of therapy, and their personal habits and circumstances, including an integrity-preserving attitude, a performance-oriented attitude, a searching attitude, a faith-in-medicine attitude, and a benefit-generalizing attitude. The interviews show that there is a need for target-oriented counseling, taking into account the individual attitudes toward menopause and postmenopausal hormone therapy.

Palliativmedizin unter Betrachtung des demographischen Wandels : Was kann sich die Gesellschaft leisten?

gen, welche unwirksamen oder mit nur geringem Zusatznutzen versehenen Investitionen im Bereich der Versorgung alter und hochbetagter Menschen getätigt werden bzw. geplant sind. Um es etwas pointiert zu formulieren: Wenn das Alter unsinnigerweise medikalisiert wird und Unsummen in fragwürdige Diagnostik und Therapie gesteckt werden, dann findet de facto eine besonders starke Rationierung in denjenigen Bereichen statt, deren Durchsetzungskraft im Kampf um die Ressourcen relativ schwach ist. Hierzu gehört sicher heute noch die Palliativversorgung. Man spricht in der Systemforschung geradezu von „Cinderella“-Feldern der Versorgung.

CRISTOPH - A cluster-randomised intervention study to optimise the treatment of patients with hypertension in General Practice

BackgroundRecent guidelines for the management of hypertension focus on treating patients according to their global cardiovascular risk (CVR), rather than strictly keeping blood pressure, or other risk factors, below set limit values. The objective of this study is to compare the effect of a simple versus a complex educational intervention implementing this new concept among General Practitioners (GPs).Methods/designA prospective longitudinal cluster-randomised intervention trial with 94 German GPs consecutively enroling 40 patients each with known hypertension. All GPs then received a written manual specifically developed to transfer the global concept of CVR into daily General Practice. After cluster-randomisation, half of the GPs additionally received a clinical outreach visit, with a trained peer discussing with them the concept of global CVR referring to example study patients from the respective GP. Main outcome measure is the improvement of calculated CVR six months after intervention in the subgroup of patients with high CVR (but no history of cardiovascular disease), defined as 10-year-mortality ≥ 5% employing the European SCORE formula. Secondary outcome measures include the interventions effect on single risk factors, and on prescription rates of drugs targeting CVR. All outcome measures are separately studied in the three subgroups of patients with 1. high CVR (defined as above), 2. low CVR (SCORE < 5%), and 3. a history of cardiovascular disease. The influence of age, sex, social status, and the perceived quality of the respective doctor-patient-relation on the effects will be examined.DiscussionTo our knowledge, no other published intervention study has yet evaluated the impact of educating GPs with the goal to treat patients with hypertension according to their global cardiovascular risk.Trial registrationISRCTN44478543

Palliativmedizin unter Betrachtung des demographischen Wandels

gen, welche unwirksamen oder mit nur geringem Zusatznutzen versehenen Investitionen im Bereich der Versorgung alter und hochbetagter Menschen getätigt werden bzw. geplant sind. Um es etwas pointiert zu formulieren: Wenn das Alter unsinnigerweise medikalisiert wird und Unsummen in fragwürdige Diagnostik und Therapie gesteckt werden, dann findet de facto eine besonders starke Rationierung in denjenigen Bereichen statt, deren Durchsetzungskraft im Kampf um die Ressourcen relativ schwach ist. Hierzu gehört sicher heute noch die Palliativversorgung. Man spricht in der Systemforschung geradezu von „Cinderella“-Feldern der Versorgung.

The Role of End-of-Life Issues in the Design and Reporting of Cancer Clinical Trials: A Structured Literature Review

Background Randomized controlled trials (RCTs) are important sources of information on the benefits and harms patients may expect from treatment options. The aim of this structured literature review by the German Institute for Quality and Efficiency in Health Care was to explore whether and how the end-of-life (EoL) situation of patients with advanced cancer is considered in RCTs investigating anti-cancer treatments. Methods Our journal pool comprised 19 medical journals, namely five preselected key general medical journals as well as 14 specialist journals (mainly cancer) identified via a scoping search. We systematically searched these journals in MEDLINE to identify RCTs investigating anti-cancer treatments for the following four cancer types: glioblastoma, lung cancer (stage IIIb-IV), malignant melanoma (stage IV), and pancreatic cancer (search via OVID; November 2012). We selected a representative sample of 100 publications, that is, the 25 most recent publications for each cancer type. EoL was defined as a life expectancy of ≤ two years. We assessed the information provided on (1) the descriptions of the terminal stage of the disease, (2) the therapeutic goal (i.e. the intended therapeutic benefit of the intervention studied), (3) the study endpoints assessed, (4) the authors’ concluding appraisal of the intervention’s effects, and (5) the terminology referring to the patients’ EoL situation. Results Median survival was ≤ one year for each of the four cancer types. Descriptions of the terminal stage of the disease were ambiguous or lacking in 29/100 publications. One or more therapeutic goals were mentioned in 51/100 publications; these goals were patient-relevant in 38 publications (survival alone: 30/38; health-related quality of life (HRQoL) or HRQoL and survival: 6/38; symptom control or symptom control and survival: 2/38). Primary endpoints included survival (50%), surrogates (44%), and safety (3%). Patient-reported outcomes (PROs) were assessed in 36/100 RCTs. The implications of treatment-related harms for the patients were discussed in 22/100 appraisals. Terminology referring to the patients’ EoL situation (e.g. “terminal”) was scarce, whereas terms suggesting control of the disease (e.g. “cancer control“) were common. Conclusions The EoL situation of patients with advanced cancer should be more carefully considered in clinical trials. Although the investigation and robust reporting of PROs is a prerequisite for informed decision-making in healthcare, they are rarely defined as endpoints and HRQoL is rarely mentioned as a therapeutic goal. Suggestions for improving standards for study design and reporting are presented.

The Long Way Toward Cooperation Nurses and Family Physicians in Northern Germany

To better understand why cooperation between health care professionals is still often problematic, we carried out 25 semistructured face-to-face expert interviews with physicians and nurses in different rural and urban areas in northern Germany. Using Mayring’s qualitative content analysis method to analyze the data collected, we found that doctors and nurses interpreted interprofessional conflicts differently. Nursing seems to be caught in a paradoxical situation: An increasing emphasis is placed on achieving interprofessional cooperation but the core areas of nursing practice are subject to increasing rationalization in the current climate of health care marketization. The subsequent and systematic devaluation of nursing work makes it difficult for physicians to acknowledge nurses’ expertise. We suggest that to ameliorate interprofessional cooperation, nursing must insist on its own logic of action thereby promoting its professionalization; interprofessional cooperation cannot take place until nursing work is valued by all members of the health care system.

Krankheitsarbeit von Patienten und Patientinnen mit rheumatoider Arthritis - Ein ungesehenes Thema in der Interaktion

Welche Perspektive haben Patienten und Patientinnen mit rheumatoider Arthritis auf ihren Krankheitsverlauf? Diese Frage steht im Mittelpunkt einer qualitativen Studie1 zum Verstandnis von patientenseitigen Haltungen und Entscheidungen an der Schnittstelle von therapeutischem Fortschritt und Krankheitserleben im Kontext von rheumatoider Arthritis (RhA). Ausgangspunkt der Studie bildet die Annahme, dass sich vor allem fur den gravierend betroffenen Teil der Patientinnen und Patienten das Bild der RhA durch medizinischen Fortschritt grundlegend gewandelt habe, insbesondere durch Innovationen im pharmakologischen Bereich. Aus dieser Perspektive ist RhA als chronische Erkrankung zu beschreiben, die prinzipiell gut behandelbar ist. Anknupfend an diese Uberlegungen sind qualitative Leitfadeninterviews mit an RhA erkrankten Menschen gefuhrt worden, um ihre Perspektive auf den Krankheitsverlauf zu ergrunden und um zu erfassen, worin fur sie – trotz bzw. jenseits des pharmakologischen Fortschrittes – Versorgungsdefizite bestehen. Zu fragen bleibt, welche Schlussfolgerungen sich aus dem Krankheitserleben von Menschen mit RhA ziehen lassen fur die Gestalt medizinischer Versorgungsprozesse, explizit fur Begegnungen zwischen Arzten/Arztinnen und Patienten/Patientinnen.

Die Formung professionellen Handelns in Zeiten entschiedener Qualitätspolitik

Gute Qualitat im kurativen Gesundheitswesen schien lange Zeit wie selbstverstandlich durch autonome Entscheidungen der Arzteschaft garantiert zu werden. Der darin zum Ausdruck kommende gesellschaftlich weithin vorhandene Vertrauensvorschuss geriet im auslaufenden 20. Jahrhundert ins Wanken, nicht zuletzt festzumachen am Bericht „To Err is Human“ des Institute of Medicine. In den 80er Jahren war zuvor der Qualitatsbegriff in seinen Dimensionen Struktur, Prozess und Outcome durch die Publikation von Avedis Donabedian sprichwortlich geworden. Schrittweise ubernahm die Politik die Forderung nach Messung und Kontrolle der Qualitat bis hin zu neuzeitlichen Ansatzen der Kopplung der Vergutung medizinischer Leistungen an Qualitatsindikatoren. Eindeutige patientenrelevante Erfolge dieser Politik stehen aus, wahrend sich bei den Professionellen die Reaktionen zwischen innerer Abkehr vom Qualitatsdiskurs und einem Ringen um moglichst grose Teilhabe an den okonomischen Potenzialen dieser neuen Qualitatspolitik einzupendeln scheinen.

Münsteraner Memorandum Heilpraktiker. Die Thesen des „Münsteraner Kreises“ zu einer Neuregelung des Heilpraktikerwesens

Dieser Artikel ist ein Auszug aus „Münsteraner Memorandum Heilpraktiker. Ein Statement der interdisziplinären Expertengruppe ,Münsteraner Kreis‘ zu einer Neuregelung des Heilpraktikerwesens“, elektronisch veröffentlicht durch den Münsteraner Kreis in 2017 unter http://daebl.de/BB36. Die federführenden Hauptautoren dieses Artikels sind D.R. Friedrich, J.-O. Reichardt, B. Schöne-Seifert und C. Weymayr.