Implementing economic principles in medicine while maintaining medical professionalism

Introduction: Various forms of the managed-care concept have been conceived to reconcile the medical professionalism with necessary cost-cutting measures in health-care provision. A plethora of bureaucratic regulations and required paperwork result in increasing resignation among physicians and withdrawal from patient care. Options: An appropriate option would be to focus not on structures and processes, but primarily on patient-related outcomes. We describe suitable options, their possible consequences, possible developments, and conclusions that can be derived. Prognoses: Neglecting a trustful doctor-patient relationship risks forfeiting fundamental parts of essential health-care provision. Patients’ preferences have to be respected, while unnecessary risks and expenses have to be avoided. Further development: At least 12 dimensions influence the balance between medical professionalism and economics. The success of our health-care systems will depend on the best possible mix. Conclusion: An optimal health-care system is characterized neither by the structures nor processes of health-care delivery, but by the quality of patient outcomes. Our patients must be involved in the description of the goals they want to achieve so that physicians can select the best possible ways to attain these goals

Spontaneous remissions in breast cancer underline the need of more evidence: screening should not detect more cancer but earlier cancer

Aim: Analysis of the hypothesis that 22% of breast cancer cases detected by screening mammography would disappear spontaneously. Methods: Critical appraisal of the relevant scientific literature using established methods. Results: The hypothesis—although it is unusual—seems to be very well supported by data from several independent research groups and justifies three lessons. Conclusions: First, it is necessary to complete the last step of the concept of traditional evidence-based medicine (EBM), i.e., to confirm that the outcome that is predicted by scientific evidence will also be observed in daily clinical practice. Second, the focus of medical teaching should be directed to final clinical outcomes and patient benefit rather than surrogate parameters, such as completed procedures or test results. Third, the availability of data to answer unsolved questions may become a more important indicator for high-quality health-care systems than the performance of services of unknown incremental value

Form follows function: pragmatic controlled trials (PCTs) have to answer different questions and require different designs than randomized controlled trials (RCTs).

Aim Rising concern for demonstrated real world comparative effectiveness has heightened interest in “pragmatic trials” design. Pragmatic trials investigate whether the efficacy, presumed or found in explanatory trials under ideal conditions, can also be detected under real world conditions, i.e. effectiveness. It is also recognized that ‘real world’ effects which are usually addressed in public health research gain growing interest in confirming the ‘road capability’ of results obtained under ideal study conditions. This paper demonstrates that studies under ideal or real world conditions use different methods, generate different information and cannot replace each other. Subjects and methods The PCT design meets four requirements of public health and of effectiveness research. It includes all individuals who presented with the selected condition. It classifies the included individuals according to baseline risks. It enables plausibility controls. Finally, it compares the outcomes resulting from specified and notspecified interventions or treatments. Proposal We propose a pragmatic controlled trial (PCT) design in which patient preference and other co-factors crucial in determining the actual effectiveness of interventional options will not be neutralized by concealed randomization and blinding. This design is applicable to record the selected interventions and generated outcomes in day-to-day health care and is capable of incorporating preference and other participative factors into assessment of effectiveness. Conclusions The PCT design is useful for public health research, e.g. the effectiveness of interventions to change smoking habits or to prevent death frombreast cancer, as well as for comparative effectiveness research where it will supplement the traditional randomized controlled trial (RCT)

Preferences of diabetes patients and physicians: A feasibility study to identify the key indicators for appraisal of health care values

<p>Abstract</p> <p>Background</p> <p>Evidence-based medicine, the Institute of Medicine (IOM) and the German Institute for Quality and Efficiency in Health Care (IQWiG), support the inclusion of patients' preferences in health care decisions. In fact there are not many trials which include an assessment of patient's preferences. The aim of this study is to demonstrate that preferences of physicians and of patients can be assessed and that this information may be helpful for medical decision making.</p> <p>Method</p> <p>One of the established methods for assessment of preferences is the conjoint analysis. Conjoint analysis, in combination with a computer assisted telephone interview (CATI), was used to collect data from 827 diabetes patients and 60 physicians, which describe the preferences expressed as levels of four factors in the management and outcome of the disease. The first factor described the main treatment effect (reduction of elevated Hb_A1c, improved well-being, absence of side effects, and no limitations of daily life). The second factor described the effect on the body weight (gain, no change, reduction). The third factor analyzed the mode of application (linked to meals or flexible application). The fourth factor addressed the type of product (original brand or generic product). Utility values were scaled and normalized in a way that the sum of utility points across all levels is equal to the number of attributes (factors) times 100.</p> <p>Results</p> <p>The preference weights confirm that the reduction of body weight is at least as important for patients - especially obese patients - and physicians as the reduction of an elevated Hb_A1c. Original products were preferred by patients while general practitioners preferred generic products.</p> <p>Conclusion</p> <p>Using the example of diabetes, the difference between patients' and physicians' preferences can be assessed. The use of a conjoint analysis in combination with CATI seems to be an effective approach for generation of data which are needed for policy and medical decision making in health care.</p

Advantages and limitations of Twin Assessment of Clinical Trials (TACT)

Background: Considerable time and energy are expended in the scientific community to discuss the validity, importance, and applicability of the results of clinical trials. Depending on the goals, perspectives, and other motivating factors, protagonists and skeptics come to different conclusions, even when using the same methods and tools for critical appraisal. The aim of this study was to complement existing methods and tools with minor modifications to provide a prototype instrument that generates commonly accepted versions of critical appraisals. Methods: As a pilot experiment, one university-based and one industry-based referee independently completed the twin assessment of five trials published in well-recognized journals. They identified the study questions, defined the simplest, i.e., ideal, study designs to answer these questions, and checked eight validity criteria. Identical positive or negative answers of both referees increased or decreased the validity score. A maximum of two disagreements (0 score) was allowed. This procedure, which had been tested by two referees in a pilot experiment, was repeated with 19 third-year medical students and their supervisor at the Universidade Federal Fluminense, Niterói/RJ, Brasil. Four students each played the roles of the industry-based and university-based referees and finally recorded their consensus. Results: The two referees of the pilot experiment agreed in all but one answer to the five investigated publications. The points of criticism differed in various papers. The consensus reached by the students considerably differed from the consensus reached by the referees. Conclusions: A consensus score generated by two referees or by two groups of students is feasible, but the achieved result is not necessarily reproducible. The critical appraisal of the study question in connection with the applied study design deserves special attention. It is time consuming but possible to identify and describe the possible flaws in the design conduct and report of clinical trials, but it is unlikely to reach a reproducible interpretation. These data indicate the problems with even evidence-based assessments and appraisals: the assessments may well be reproducible, but not the appraisals. Quality scores that include also the appraisal may therefore be interpreted with caution. Appraisals or quality scores may be used for interim decisions until data are available that confirm under real-world conditions what was predicted by the results generated under ideal but artificial conditions of a clinical trial

Soluble Tumor Necrosis Factor Receptors Correlate with Parasitemia andDisease Severity in Human Malaria

Elevated serum or plasma concentration of immunoreactive tumor necrosis factor (TNF) is consistently detected in patients with malaria. TNF levels correlate with high parasitemia and clinical severity but not always with outcome. Since the effects of TNF may be neutralized by soluble TNF receptors, sera of 30 nonimmune patients with malaria were analyzed before and during antimalarial therapy. High concentrations of receptors R1 (55 kDa) and R2 (75 kDa) were detected immunologically in all sera of untreated patients. Levels of immunoreactive TNF correlated closely with levels of soluble TNF R1 and R2 (r = .75 and .59, respectively). In contrast, sera lacked cytotoxic activity against target cells in the TNF bioassays. Soluble TNF receptor levels remained elevated for days after treatment. These results suggest that excessive release of TNF induced by the asexual stage of malaria parasites is controlled by a subsequent shedding of soluble TNF receptors that may bind and deactivate biologically functional TN

Gesundheit - Markt - Gerechtigkeit

"The three elements of the title intersect at three points which will be discussed from the perspective of three professions – medicine, economics, and ethics. The discussion of the nine emerging topics illustrates the chances and risks in this field of conflicting principles. As cryptonormative definitions are one of the most frequent sources of error, we try to be as transparent as possible in the use of different terms and thus to come to resilient conclusions." (author's abstract

A new instrument to describe indicators of well-being in old-old patients with severe dementia – The Vienna List

BACKGROUND: In patients with very severe dementia self-rating of quality of life usually is not possible and appropriate instruments for proxy-ratings are not available. The aim of this project is to develop an instrument of clinical proxy-ratings for this population. METHODS: Using electronic instruments, physicians and nurses recorded patient behaviour and changes of behaviour over a period of one year. Based on these data a list of 65 items was generated and subsequently allocated to 14 categories. This list was tested in 217 patients (61–105 yrs) with dementia diagnosed according to ICD-10 by both physicians and nurses. The severity of dementia was assessed by means of the Global Deterioration Scale (GDS) and the Brief Cognitive Rating Scale (BCRS). The Spitzer-Index (proxy-rating) was used as a global quality of life measure. Activity of daily living was rated using the Barthel Index. RESULTS: A factor analysis of the original 65 items revealed 5 factors (communication, negative affect, bodily contact, aggression, and mobility). By stepwise removing items we obtained satisfactory internal consistencies of the factors both for nurses' and physicians' ratings. The factors were generally unrelated. The validity of the instrument was proven by correlations of the factors communication and mobility with the Brief Cognitive Rating Scale (BCRS) and the Barthel-Index. CONCLUSION: The results demonstrate the reliability and validity of the Vienna List as a proxy rating measurement of quality of life in patients with severe dementia. The psychometric properties of the scale have to be proved in further studies

Appraisal of health care: from patient value to societal benefit

Aim: This paper summarizes the deficiencies and weaknesses of the most frequently used methods for the allocation of health-care resources. New, more transparent and practical methods for optimizing the allocation of these resources are proposed. Method: The examples of quality-adjusted life years (QALYs) and efficiency frontier (EF) are analyzed to describe weaknesses and problems in decisions regulating health-care provision. After conducting a literature search and discussions with an international group of professionals, three groups of professionals were formed to discuss the assessment and appraisal of health-care services and allocation of available resources. Results: At least seven essential variables were identified that should be heeded when applying the concept of QALYs for decisions concerning health-care provision. The efficiency frontier (EF) concept can be used to set a ceiling price and perform a cost-benefit analysis of provision, but different stakeholders—a biostatistician (efficacy), an economist (costs), a clinician (effectiveness), and the patient (value)—could provide a fairer appraisal of health-care services. Efficacy and costs are often based on falsifiable data. Effectiveness and value depend on the success with which a particular clinical problem has been solved. These data cannot be falsified. The societal perspective is generated by an informal cost-benefit analysis including appraisals by the above-mentioned stakeholders and carried out by an authorized institution. Conclusion: Our analysis suggests that study results expressed in QALYs or as EF cannot be compared unless the variables included in the calculation are specified. It would be far more objective and comprehensive if an authorized institution made an informal decision based on formal assessments of the effectiveness of health-care services evaluated by health-care providers, of the value assessed by consumers, of efficacy described by biostatisticians, and of costs calculated by economists