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Informative value of Patient Reported Outcomes (PRO) in Health Technology Assessment (HTA)Background: “Patient-Reported Outcome” (PRO) is used as an umbrella term for different concepts for measuring subjectively perceived health status e. g. as treatment effects. Their common characteristic is, that the appraisal of the health status is reported by the patient himself. In order to describe the informative value of PRO in Health Technology Assessment (HTA) first an overview of concepts, classifications and methods of measurement is given. The overview is complemented by an empirical analysis of clinical trials and HTA-reports on rheumatoid arthritis and breast cancer in order to report on type, frequency and consequences of PRO used in these documents. Methods: For both issues systematic reviews of the literature have been performed. The search for methodological literature covers the publication period from 1990 to 2009, the search for clinical trials of rheumatoid arthritis and breast cancer covers the period 2005 to 2009. Both searches were performed in the medical databases of the German Institute of Medical Documentation and Information (DIMDI). The search for HTA-reports and methodological papers of HTA-agencies was performed in the CRD-Databases (CRD = Centre for Reviews and Dissemination) and by handsearching the websites of INAHTA member agencies (INAHTA = International Network of Agencies for Health Technology Assessment). For all issues specific inclusion and exclusion criteria were defined. The methodological quality of randomized controlled trials (RCT) was assessed by a modified version of the Cochrane Risk of Bias Tool. For the methodological part information extraction from the literature is structured by the report’s chapters, for the empirical part data extraction sheets were constructed. All information is summarized in a qualitative manner. Results: Concerning the methodological issues the literature search retrieved 158 documents (87 documents related to definition or classification, 125 documents related to operationalisation of PRO). For the empirical analyses 225 RCT (rheumatoid arthritis: 77; breast cancer: 148) and 40 HTA-reports and method papers were found. The analysis of the methodological literature confirms the role of PRO as an umbrella term for a variety of different concepts. The newest classification system facilitates the description of PRO measures by construct, target population and the method of measurement. Steps of operationalisation involve defining a conceptual framework, instrument development, exploration of measurement properties or, possibly, the modification of existing instruments. Seven out of 59 RCT analysing the effects of antibody therapy for rheumatoid arthritis define PRO as the primary endpoint, 38 trials utilize composite measures (ACR, DAS) and ten trials report clinical or radiological parameters as the primary endpoint. Six out of 123 chemotherapy trials for breast cancer define PRO as the primary endpoint, while 98 trials report clinical endpoints (survival, tumour response, progression) in their primary analyses. Discrepancies in the number of trials result from inaccurate specifications of endpoints in the publications. This distribution is reflected in the HTA-reports: while almost all reports on rheumatoid arthritis refer to PRO, this is only the case in about half of the reports on breast cancer. Conclusions: As definition and classification of PRO are concerned, coherent concepts are found in the literature. Their operationalisation and implementation must be guided by scientific principles. The type and frequency of PRO used in clinical trials largely depend on the disease analysed. The HTA-community seems to pursue the utilization of PRO proactively – in case of missing data the need for further research is stated.
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Prevention of alcohol misuse among children, youths and young adultsBackground: Despite many activities to prevent risky alcohol consumption among adolescents and young adults there is an increase of alcohol intoxications in the group of ten to twenty year old juveniles. Objectives: This report gives an overview about the recent literature as well as the German federal prevention system regarding activities concerning behavioral and policy prevention of risky alcohol consumption among children, adolescents and young adults. Furthermore, effective components of prevention activities are identified and the efficiency and efficacy of ongoing prevention programs is evaluated. Methods: A systematic literature review is done in 34 databases using Bool’sche combinations of the key words alcohol, prevention, treatment, children, adolescents and young adults. Results: 401 studies were found and 59 studies were selected for the health technology assessment (HTA). Most of the studies are done in USA, nine in Germany. A family strengthening program, personalized computer based intervention at schools, colleges and universities, brief motivational interventions and policy elements like increase of prices and taxes proved effective. Discussion: Among the 59 studies there are three meta-analyses, 15 reviews, 17 randomized controlled trials (RCT) and 18 cohort studies. Despite the overall high quality of the study design, many of them have methodological weaknesses (missing randomization, missing or too short follow-ups, not clearly defined measurement parameters). The transferability of US-results to the German context is problematic. Only a few prevention activities reach a sustainable reduction of frequency and/or amount of alcohol consumption. Conclusion: The HTA-report shows the need to develop specific and target group focused prevention activities for the German situation. Essential for that is the definition of target goals (reduction of consumption, change of behaviour) as well as the definition and empirical validation of risky alcohol consumption. The efficacy of prevention activities should be proven before they are launched. At present activities for the reduction or prevention of risky alcohol consumption are not sufficiently evaluated in Germany concerning their sustainable efficacy.
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Informative value of Patient Reported Outcomes (PRO) in Health Technology Assessment (HTA)Hintergrund und Zielsetzung Patient-Reported Outcome (PRO) wird als Oberbegriff für unterschiedliche Konzepte zur Messung subjektiv empfundener Gesundheitszustände verwendet. Sie sind dadurch gekennzeichnet, dass der Patient selbst seine Einschätzung berichtet. Um den Stellenwert von PRO im Kontext von HTA-Verfahren (HTA = Health Technology Assessment) zu beschreiben, wird zunächst eine Übersicht über Konzepte, Klassifikationen und methodische Messansätze erstellt. Diese Übersicht wird ergänzt um eine empirische Analyse von klinischen Studien und HTA-Berichten mit dem Ziel, Art, Häufigkeit und Konsequenzen der verwendeten PRO zu dokumentieren. Methodik Beide Fragestellungen werden mithilfe von systematischen Literaturübersichten bearbeitet. Für den methodischen Teil wird in den medizinnahen Datenbanken des Deutschen Instituts für Medizinische Dokumentation und Information (DIMDI) mit einer Recherchestrategie aus drei Modulen im Zeitraum von 1990 bis 2009 gesucht. Die Recherche nach randomisierten klinischen Studien (RCT) zu den Krankheitsbildern rheumatoide Arthritis (RA) und Mammakarzinom erfolgt ebenfalls in den Datenbanken des DIMDI, für den Zeitraum von 2005 bis 2009. Die Recherche nach HTA-Berichten und -Methodenpapieren umfasst die Datenbanken des Centre for Reviews and Dissemination (CRD) und Handsuchen. Für alle Fragestellungen werden spezifische Ein- und Ausschlusskriterien zur Literaturselektion definiert. Die methodische Qualität der RCT wird mithilfe eines in Anlehnung an das "Risk of Bias Tool" der Cochrane Collaboration konzipierten Instruments bewertet. Die Informationsextraktion erfolgt für den methodischen Teil strukturiert durch die Kapitelgliederung, für den empirischen Teil in Extraktionsbögen. Alle Informationen werden qualitativ beschreibend zusammengefasst. Ergebnisse Aus den Recherchen können 158 Dokumente zur Bearbeitung der methodischen Fragestellungen (87 Dokumente zu Definition/Klassifikation; 125 Dokumente zur Operationalisierung) und 225 RCT (77 RA, 148 Mammakarzinom) sowie 40 HTA-Berichte zur Bearbeitung der empirischen Fragestellungen gewonnen werden. Die Analysen zu Definitionen bestätigen PRO als Oberbegriff für eine Vielzahl von patientenberichteten Endpunkten. Das neueste Klassifikationssystem ermöglicht die Beschreibung der PRO nach dem Konstrukt, der Zielpopulation und der Messmethode. Ausführungen zur Operationalisierung beziehen sich auf den Konzeptrahmen, die Instrumentenentwicklung, -eigenschaften und -modifikationsmöglichkeiten. Von 59 Studien zur Antikörpertherapie der RA verwenden sieben ausschließlich PRO, 38 gemischte Zielgrößen (American College of Rheumatology [ACR], Disease Activity Score [DAS]) und zehn rein klinische bzw. radiologische Parameter als primäre Outcomes. Von 123 Studien zur Chemotherapie des Mammakarzinoms stützen sich nur sechs auf PRO als primäre Zielgröße; 98 Studien gebrauchen klinische Parameter (Überlebenszeit, Tumoransprechen, Progression). Abweichungen von der Gesamtzahl resultieren aus ungenauen Angaben der Zielgrößen. Diese Verteilung spiegelt sich auch in den analysierten HTA-Berichten wieder. In den Berichten zur RA werden durchweg PRO-Zielgrößen berichtet, während in den Berichten zum Mammakarzinom dies in knapp der Hälfte der Publikationen der Fall ist. Zusammenhänge zwischen Studienqualität und der Verwendung von PRO sind nicht erkennbar. Schlussfolgerungen Für die Definition und die Klassifikation von PRO existieren inzwischen schlüssige Konzepte, deren Umsetzung wissenschaftlichen Kriterien genügen muss. Die Häufigkeit und die Art der in klinischen Studien verwendeten PRO variieren abhängig vom untersuchten Krankheitsbild. Im Kontext von HTA wird die Notwendigkeit zur Erfassung von PRO wahrgenommen, bei fehlenden Daten wird Forschungsbedarf formuliert.