Importance of hemodialysis-related outcomes: comparison of ratings by a self-help group, clinicians, and health technology assessment authors with those by a large reference group of patients

Janssen IM, Scheibler F, Gerhardus A. Importance of hemodialysis-related outcomes: comparison of ratings by a self-help group, clinicians, and health technology assessment authors with those by a large reference group of patients. Patient Preference and Adherence. 2016;10:2491-2500.Background: The selection of important outcomes is a crucial decision for clinical research and health technology assessment (HTA), and there is ongoing debate about which stakeholders should be involved. Hemodialysis is a complex treatment for chronic kidney disease (CKD) and affects many outcomes. Apart from obvious outcomes, such as mortality, morbidity and health-related quality of life (HRQoL), others such as, concerning daily living or health care provision, may also be important. The aim of our study was to analyze to what extent the preferences for patient-relevant outcomes differed between various stakeholders. We compared preferences of stakeholders normally or occasionally involved in outcome prioritization (patients from a self-help group, clinicians and HTA authors) with those of a large reference group of patients. Participants and methods: The reference group consisted of 4,518 CKD patients investigated previously. We additionally recruited CKD patients via a regional self-help group, nephrologists via an online search and HTA authors via an expert database or personal contacts. All groups assessed the relative importance of the 23 outcomes by means of a discrete visual analog scale. We used descriptive statistics to rank outcomes and compare the results between groups. Results: We received completed questionnaires from 49 self-help group patients, 19 nephrologists and 18 HTA authors. Only the following 3 outcomes were ranked within the top 7 outcomes by all 4 groups: safety, HRQoL and emotional state. The ratings by the self-help group were generally more concordant with the reference group ratings than those by nephrologists, while HTA authors showed the least concordance. Conclusion: Preferences of CKD patients from a self-help group, nephrologists and HTA authors differ to a varying extent from those of a large reference group of patients with CKD. The preferences of all stakeholders should form the basis of a transparent approach so as to generate a valid list of important outcomes

Health technology assessment of public health interventions: an analysis of characteristics and comparison of methods-study protocol

Mathes T, Willms G, Polus S, et al. Health technology assessment of public health interventions: an analysis of characteristics and comparison of methods-study protocol. SYSTEMATIC REVIEWS. 2018;7(1): 79.Background: Conducting a health technology assessment (HTA) of public health interventions (PHIs) poses some challenges. PHIs are often complex interventions, which affect the number and degree of interactions of the aspects to be assessed. Randomized controlled trials on PHIs are rare as they are difficult to conduct because of ethical or feasibility issues. The aim of this study is to provide an overview of the methodological characteristics and to compare the applied assessment methods in HTAs on PHIs. Methods: We will systematically search HTA agencies for HTAs on PHIs published between 2012 and 2016. We will identify the HTAs by screening the webpages of members of international HTA organizations. One reviewer will screen the list of HTAs on the webpages of members of international HTA organization, and a second review will double-check the excluded records. For this methodological review, we define a PHI as a population-based intervention on health promotion or for primary prevention of chronic or non-chronic diseases. Only full HTA reports will be included. At maximum, we will include a sample of 100 HTAs. In the case that we identify more than 100 relevant HTAs, we will perform a random selection. We will extract data on effectiveness, safety and economic as well as on social, cultural, ethical and legal aspects in a priori piloted standardized tables. We will not assess the risk of bias as we focus on exploring methodological features. Data extraction will be performed by one reviewer and verified by a second. We will synthesize data using tables and in a structured narrative way. Discussion: Our analysis will provide a comprehensive and current overview of methods applied in HTAs on PHIs. We will discuss approaches that may be promising to overcome the challenges of evaluating PHIs

Ethical analysis in HTA of complex health interventions

Background: In the field of health technology assessment (HTA), there are several approaches that can be used for ethical analysis. However, there is a scarcity of literature that critically evaluates and compares the strength and weaknesses of these approaches when they are applied in practice. In this paper, we analyse the applicability of some selected approaches for addressing ethical issues in HTA in the field of complex health interventions. Complex health interventions have been the focus of methodological attention in HTA. However, the potential methodological challenges for ethical analysis are as yet unknown. Methods: Six of the most frequently described and applied ethical approaches in HTA were critically assessed against a set of five characteristics of complex health interventions: multiple and changing perspectives, indeterminate phenomena, uncertain causality, unpredictable outcomes, and ethical complexity. The assessments are based on literature and the authors’ experiences of developing, applying and assessing the approaches. Results: The Interactive, participatory HTA approach is by its nature and flexibility, applicable across most complexity characteristics. Wide Reflective Equilibrium is also flexible and its openness to different perspectives makes it better suited for complex health interventions than more rigid conventional approaches, such as Principlism and Casuistry. Approaches developed for HTA purposes are fairly applicable for complex health interventions, which one could expect because they include various ethical perspectives, such as the HTA Core Model® and the Socratic approach. Conclusion: This study shows how the applicability for addressing ethical issues in HTA of complex health interventions differs between the selected ethical approaches. Knowledge about these differences may be helpful when choosing and applying an approach for ethical analyses in HTA. We believe that the study contributes to increasing awareness and interest of the ethical aspects of complex health interventions in general

Preferences of patients undergoing hemodialysis - results from a questionnaire-based study with 4,518 patients

Janssen IM, Gerhardus A, von Gersdorff G, et al. Preferences of patients undergoing hemodialysis - results from a questionnaire-based study with 4,518 patients. Patient Preference and Adherence. 2015;2015(9):847-855.Background: Chronic kidney disease is an increasing health problem worldwide and in its final stage (stage V) can only be treated by renal replacement therapy, mostly hemodialysis. Hemodialysis has a major influence on the everyday life of patients and many patients report dissatisfaction with treatment. Little is known about which aspects of treatment are considered important by hemodialysis patients. The objective of this study was to rate the relative importance of different outcomes for hemodialysis patients and to analyze whether the relative importance differed among subgroups of patients. Patients and methods: Within the framework of a yearly questionnaire which is distributed among patients receiving hemodialysis by the largest hemodialysis provider in Germany, we assessed the relative importance of 23 outcomes as rated on a discrete visual analog scale. Descriptive statistics were used to rank the outcomes. Subgroup analyses were performed using Mann–Whitney U or Kruskal–Wallis tests. Results: Questionnaires of 4,518 hemodialysis patients were included in the analysis. The three most important outcomes were safety of treatment, health-related quality of life, and satisfaction with care. Further important outcomes were hospital stays, accompanying symptoms, hemodialysis duration, and the improvement or preservation of a good emotional state. Age, profession, and education had the strongest influence on relevant differences of preferences for outcomes; no relevant influence of sex or comorbidity was observed. Conclusion: Outcomes concerning the delivery or provision of care and aspects influencing quality of life are rated by patients to be at least as important as clinical outcomes. Many of the outcomes judged to be important by the patients are not regularly considered in research, evaluation studies, or quality programs

Why is it so difficult to integrate ethics in Health Technology Assessment (HTA)? The epistemological viewpoint

Ethics has been identified as a key element in Health Technology Assessment (HTA) since its conception. However, ethical issues are still not frequently addressed explicitly in HTA. Several valuable reasons have been identified. The basis of the article is the claim that ethics is often not part of HTA for “epistemological reasons”. Hence, the main aim of the contribution is to explore in more details and emphasize them by using the fact/value dichotomy. Our conclusion is that current HTA configuration is dominantly based on the comparison among objective and empirically testable “facts”, whilst ethics is not empirically testable. In this sense, there is a sort of “epistemological gap”, which can explain why it is so difficult to integrate ethics in HTA. We suggest that the epistemological differences among the various domains of HTA are addressed more explicitly

Applying for, reviewing and funding public health research in Germany and beyond

Gerhardus A, Becher H, Groenewegen P, et al. Applying for, reviewing and funding public health research in Germany and beyond. HEALTH RESEARCH POLICY AND SYSTEMS. 2016;14(1): 43.Public health research is complex, involves various disciplines, epistemological perspectives and methods, and is rarely conducted in a controlled setting. Often, the added value of a research project lies in its inter- or trans-disciplinary interaction, reflecting the complexity of the research questions at hand. This creates specific challenges when writing and reviewing public health research grant applications. Therefore, the German Research Foundation (DFG), the largest independent research funding organization in Germany, organized a round table to discuss the process of writing, reviewing and funding public health research. The aim was to analyse the challenges of writing, reviewing and granting scientific public health projects and to improve the situation by offering guidance to applicants, reviewers and funding organizations. The DFG round table discussion brought together national and international public health researchers and representatives of funding organizations. Based on their presentations and discussions, a core group of the participants (the authors) wrote a first draft on the challenges of writing and reviewing public health research proposals and on possible solutions. Comments were discussed in the group of authors until consensus was reached. Public health research demands an epistemological openness and the integration of a broad range of specific skills and expertise. Applicants need to explicitly refer to theories as well as to methodological and ethical standards and elaborate on why certain combinations of theories and methods are required. Simultaneously, they must acknowledge and meet the practical and ethical challenges of conducting research in complex real life settings. Reviewers need to make the rationale for their judgments transparent, refer to the corresponding standards and be explicit about any limitations in their expertise towards the review boards. Grant review boards, funding organizations and research ethics committees need to be aware of the specific conditions of public health research, provide adequate guidance to applicants and reviewers, and ensure that processes and the expertise involved adequately reflect the topic under review

Implications of a complexity perspective for systematic reviews and guideline development in health decision making.

There is growing interest in the potential for complex systems perspectives in evaluation. This reflects a move away from interest in linear chains of cause-and-effect, towards considering health as an outcome of interlinked elements within a connected whole. Although systems-based approaches have a long history, their concrete implications for health decisions are still being assessed. Similarly, the implications of systems perspectives for the conduct of systematic reviews require further consideration. Such reviews underpin decisions about the implementation of effective interventions, and are a crucial part of the development of guidelines. Although they are tried and tested as a means of synthesising evidence on the effectiveness of interventions, their applicability to the synthesis of evidence about complex interventions and complex systems requires further investigation. This paper, one of a series of papers commissioned by the WHO, sets out the concrete methodological implications of a complexity perspective for the conduct of systematic reviews. It focuses on how review questions can be framed within a complexity perspective, and on the implications for the evidence that is reviewed. It proposes criteria which can be used to determine whether or not a complexity perspective will add value to a review or an evidence-based guideline, and describes how to operationalise key aspects of complexity as concrete research questions. Finally, it shows how these questions map onto specific types of evidence, with a focus on the role of qualitative and quantitative evidence, and other types of information

The development of CHAMP : a checklist for the appraisal of moderators and predictors

BACKGROUND: Personalized healthcare relies on the identification of factors explaining why individuals respond differently to the same intervention. Analyses identifying such factors, so called predictors and moderators, have their own set of assumptions and limitations which, when violated, can result in misleading claims, and incorrect actions. The aim of this study was to develop a checklist for critically appraising the results of predictor and moderator analyses by combining recommendations from published guidelines and experts in the field. METHODS: Candidate criteria for the checklist were retrieved through systematic searches of the literature. These criteria were evaluated for appropriateness using a Delphi procedure. Two Delphi rounds yielded a pilot checklist, which was tested on a set of papers included in a systematic review on reinforced home-based palliative care. The results of the pilot informed a third Delphi round, which served to finalize the checklist. RESULTS: Forty-nine appraisal criteria were identified in the literature. Feedback was obtained from fourteen experts from (bio)statistics, epidemiology and other associated fields elicited via three Delphi rounds. Additional feedback from other researchers was collected in a pilot test. The final version of our checklist included seventeen criteria, covering the design (e.g. a priori plausibility), analysis (e.g. use of interaction tests) and results (e.g. complete reporting) of moderator and predictor analysis, together with the transferability of the results (e.g. clinical importance). There are criteria both for individual papers and for bodies of evidence. CONCLUSIONS: The proposed checklist can be used for critical appraisal of reported moderator and predictor effects, as assessed in randomized or non-randomized studies using individual participant or aggregate data. This checklist is accompanied by a user's guide to facilitate implementation. Its future use across a wide variety of research domains and study types will provide insights about its usability and feasibilit

Harry Potter und der Schein des Weisen. Erkenntnisse und Entscheidungen zur HPV-Impfung in Deutschland. [Editorial]

Gerhardus A. Harry Potter und der Schein des Weisen. Erkenntnisse und Entscheidungen zur HPV-Impfung in Deutschland. [Editorial]. HTA-Newsletter. 2009;73:1