Reducing overuse of care is smarter than forgoing progress

Our solution assumes that a significant portion of healthcare spending is due to overuse and can be reduced without diminishing the quality of care. However, this requires addressing unresolved challenges.

1. In any society with health care overuse, this problem will only be addressed if the risk of citizens being unable to finance health care outweighs the opportunity to make commercial gains from health care services. Without this threshold being exceeded, no society will be willing to discuss "reducing overuse".

2. Services must be identified that are to be classified as "overuse". Without this evidence, there is no formal justification to distinguish between necessary and dispensable health care services.

3. The identification of dispensable health services can be provided by the method of "comparative effectiveness research". Our group has developed this method named "Pragmatic Controlled Trial (PCT)" over the last 20 years. Until now, there has been no way to confirm the feasibility of this method in everyday care.

4. Once the dispensable care services are identified, the new care strategy can be defined and the reduction in care costs can be calculated.

5. After confirming the feasibility of this strategy in at least two different cultures, the idea of implementing primary healthcare spending by defining thresholds will be discussed again. 

We would like to start the project in Cameroon because there is an equal need to improve health care for the population as in the United States. In Cameroon, it is more likely than in the U.S. to implement the envisioned solution without major hurdles because the commercial aspect of health care is less important than optimizing health care.

As an ideal way to solve the problem, we would like to accept the offer of the administration and banks of the country of Cameroon to support us in increasing the efficiency of healthcare delivery in selected hospitals.

Our strategy will enable all healthcare providers in the selected hospitals to maintain "business as usual" care without external intervention. We adapt measurement to existing conditions, rather than adapting existing conditions to meet measurement requirements. This measurement adjustment is made possible through additional data collection. The effort for scientific performance (scientific staff positions and clinical staff time to communicate with the science team) should be funded separately to ensure that the required data can actually be collected. During this phase of data collection, the care team staff will not be consulted. Communication is limited solely to the scientific task to ensure complete and error-free data collection.

The analyzed results will be presented to the care teams for discussion of four aspects:

(a) significant discrepancies are expected to be identified in the care strategies used for comparable health problems and their costs, which are not associated with a corresponding increase in health care value measured as Real-World Effectiveness (RWE).

(b) Among the care strategies that have the lowest cost for the same added health value, preference shall be given to them. Expected savings (some strategies are expected to save up to 75% of costs) can be used to fund the compensation plan (see "d") during a transition period.

(c) The data collected will justify the introduction of a "threshold quartet," each consisting of a clinically defined threshold, the MINIMAL CLINICALLY IMPORTANT DIFFERENCE (MCID), and the mathematically defined threshold, the MINIMAL DETECTABLE CHANGE (MDC). For the application of a clinical economic justifiable health service, the exceeding of these two thresholds must be demonstrated under two conditions to avoid overuse: By matching the clinical and mathematical thresholds, it must be confirmed that there is a health problem requiring care (e.g., pandemic) and that evidence of a fit intervention has been provided to care for this health problem.   

(d) These RWE data will only be available successively for different health problems. By deriving economic decisions, transitional phases can be expected for the healthcare industry, which can be represented as gains or losses. Consideration should be given to whether the health care administration should consider a compensation plan to offset opportunities and risks by providing "economic certainty" that can be expected when the financing system changes.

Our project will initially only benefit the patients who are cared for in the clinics in Cameroon and the cooperating healthcare system in Cameroon that made available to us the hospital facilities and financial support to optimize healthcare (letters of intent is available). The experience we derive from the results in Cameroon will be published in international journals. It is likely that managers of hospitals in industrialized countries will also take up the published method for their management considerations. Since the cost reduction is due to an avoidance of cost-intensive measures, decreases in turnover desired by the system are to be expected for measures with little benefit. It would be useful to consider a compensation plan in the initial phase of the project to offset revenue declines until the new adjustment to revenue is established.

By resolving the terminology conflict, we were able to lay the groundwork for confirming the difference between experimental proof of "efficacy" (or proof of principle) and pragmatic proof of real-world effectiveness. 

Currently, we have been invited by a medical journal to guest edit a special volume on "Implementation of Evidence-Based Practices and Policies in Healthcare". We submitted a current four-part series discussing common clinical epidemiological problems for publication.
In implementing our scientific project in Cameroon, the following tasks were undertaken by our fellow members:

IPTASYS GmbH, ULM/Germany     https://www.iptasys.org

Dr. Ing. Guy Leonard Kouemou      Director IPTASYS GmbH, Ulm / Germany      (native Cameroon citizen)            Technical Director and Project Manager                                                           liaison and contact to the CAMEROON
                                                   government and finance institutions  

Dr. med. Ariane Hack                    General practitioner, homeopathy, Ulm /                                                       Germany                                                                                                       Liaison person science & practical medicine

Institute of Clinical Economics    https://www.clinical-economics.com
(ICE) e.V., ULM/Germany
Ringgold ID: 587744

Prof. Dr. med. Franz Porzsolt        Founder and president ICE e.V., Ulm /                                                           Germany                                                                                                       Scientific Director and Project Manager                                                         Team member “Pragmatic Controlled Trial"                                                     Cooperation with MIT-SOLVE                                                                           Liaison person science & practical medicine

Dr. med.Guy Oscar Kamga            Internal Medicine, Family Medicine, Berlin/
(native Cameroon citizen)            Germany  (subject to time conflict with
                                                   patient care in Berlin)                                                                                    Liaison person science & practical medicine  

Prof. Dr. Christel Weiss                Biomathematics, Statistics, Heidelberg /                                                        Germany                                                                                                        Team member “Pragmatic Controlled Trial”                                                    Evaluation of outcomes

Prof. Dr. med. Manfred Weiss       Anesthesiology, Intensive Care, MBA, Ulm /                                                    Germany                                                                                                        Team member “Pragmatic Controlled Trial”                                                    Liaison person science & practical medicine

Dr. med. Jörg M. Sigle                 General Medicine, Quality of Life recorder
                                                  http://www.ql-recorder.com
                                                  Data recording. Riehen / Switzerland   

Silvia Frankenhauser                   Liaison officer science & public affairs        

Porzsolt F, Wiedemann F, Kunhardt H, Weiss Ch, Legrum P, Weiss M. Methodological aspects of demonstrating the real-world effectiveness of health services. Int J Nursing Health Care Research 2023; in press.

Summary

The starting point of our considerations 40 years ago was the worrying perception of the difference between the care results we observed in our oncological patients and the results of the international literature. It was only years later that we considered that we were observing effects that occur under the conditions of everyday care, whereas the literature mostly describes effects that can be achieved under experimental conditions in selected subjects. At that time, we could not foresee that the development of a method suitable for describing the results of non-structured everyday care would require answers to a variety of different questions. About 80 years ago, Sir Archibald Cochrane (1909 - 1988) and Sir Austin Bradford Hill (1897 - 1991) recommended answers to the three questions "Can it work?", "Does it work?" and "Is it worth it?" to confirm the proof of principle (PoP), the real-world effectiveness (RWE) and the value of health services.

The aim of our review is to identify ten methodological aspects that were used to answer the Cochrane-Hill questions and to prove the suitability of health care services for daily use: 1) The description of functions and structures of the three different conditions of care. 2) The resolution of the terminology conflict between "efficacy (PoP)" and "effectiveness (RWE)". 3) The description of the five criteria to distinguish PoP, RWE, and value. 4) The description of formal and functional differences between inclusion and exclusion criteria in studies. 5) The required combination of non-structured care conditions with structured conditions to evaluate outcomes. 6) The description (clinical relevance) and confirmation (statistical significance) of thresholds, respectively, for evidence of need for treatment and for minimally desirable reduction of a health problem. 7) Difference of initial care goals/strategies and reasons for their change. 8) Expected recruitment and training of a new professional group as a link between health care and health services research. 9) Expected added value for society through the demonstration of RWE. 10) Difference between scientific and political tasks.

The presented results are necessary for all partners of a team who strive to maintain or restore health. Administrative staff and all professionals who provide care need to be involved for several reasons. Each professional group can only consider aspects of its own profession and may therefore overlook problems that are not within its own field of vision. Until now, the financing of healthcare systems has been based on the services provided. In the future, it will be based mainly on the generated added value instead of only the services provided. The generated value is derived from the constantly updated risk profiles of the patients receiving care. Changes in risk profiles are derived from objectively measurable data. Thus, a consensually defined and objectively measurable value of care can be financed. This paper offers all professions involved in the care of patients to learn about the proposal for the management of health care and to contribute to its optimization.

Introduction

The starting point for our considerations was the observation 40 years ago that the oncological care results observed in our own clinic were less satisfactory than the results in international journals. A decade passed before a plausible explanation for this observation was found: the effects perceived under the conditions of everyday care in practices and clinics were not comparable to the effects generated under strictly selected and standardized conditions in experimental studies and reported as objectively measurable effects in the literature [1]. We found this explanation sufficiently convincing to attempt to develop a robust method for demonstrating the everyday utility of health benefits.

We ultimately succeeded in developing this method by considering two recommendations to answer three questions: The first recommendation, "Form Follows Function (FFF)," originated from American designers and architects [2]. We got to know them through our cooperation with teachers and students of the former "hochschule für gestaltung (hfg) ulm" (ulm school of design). The second recommendation is a quote from Albert Einstein (*1879 in Ulm): "Problems cannot be solved by the way of thinking that caused them". Consistent consideration of these two recommendations to answer Sir Archibald Cochrane's and Sir Austin Bradford Hill's three questions (Can it work? Does it work? Is it worth it?) prior to the introduction of health care innovations [3] has been instrumental in the development of the Real-World Effectiveness (RWE) method for describing health care services.

The method of proof of principle and the proof of terminology conflict between PoP) and RWE were essential prerequisites for deriving a three-dimensional strategy from the three Cochrane-Hill questions with the proof of value [4-7]. In the present document, we describe ten aspects, which are considered from the idea to the applicability of the method for the proof of RWE in everyday care.

Methods

Ten aspects were considered for the standardized measurement of RWE in everyday care using a non-experimental Pragmatic Controlled Trial (PCT).

1) The description of functions and structures of the three different care conditions. Two functional and 12 formal/structural criteria were described to differentiate the three supply conditions. These 14 criteria allow clear differentiation of the conditions in an experimental Randomized Controlled Trial (RCT) from the conditions in a non-experimental Pragmatic Controlled Trial (PCT) for health services research and as a third health care condition the functional and formal criteria of the usual everyday health care, under which a systematic evaluation of the results is not possible [8].

2) The resolution of the terminology conflict between "Efficacy" and "Effectiveness". To identify and resolve the existing terminology conflict between the two objective outcome dimensions PoP and RWE, it had to be accepted that daily health care takes place under conditions of "Natural Chaos", because almost every patient is quite differentiated from another patient and because the care strategy of almost all therapists is different [7]. For an unbiased measurement of effects observed under these non-structured conditions of care, a method had to be developed that meets both of the following requirements. It must allow care under non-structured conditions and, on the other hand, ensure documentation of the results under strictly structured conditions. These two requirements could be solved by applying Bayes' principle [4 - 7].

3) Five criteria to distinguish "Efficacy" / "Proof of Principle", "Re-al-World Effectiveness", and "Value". RCTs are not suitable for the measurement and evaluation of effects, which have been generated under (non-experimental) everyday conditions, because RCTs should only be conducted in populations in which patients are excluded, who may distort the measured primary endpoint due to their risk profile or whose study participation appears problematic for ethical reasons. The populations in which high-risk patients were excluded cannot represent everyday care. Considering that all reliable data have been collected in RCTs, our healthcare decisions so far have been based on objective evidence of a theoretical principle of action (POP) but not on evidence of practical everyday use. Therefore, both a consensus and the establishment of a method to prove the effects actually achieved in everyday health care are necessary.

4) Formal and functional differences between inclusion and exclusion criteria in trials. In the international literature, formal and functional differences between inclusion and exclusion criteria in clinical trials have not been systematically considered. We performed formal analyses and formulated necessary conclusions because exclusion criteria are required in experimental studies to reduce potential confounders but may not be applied in studies describing RWE. This differentiated application and the discussion of the two selection criteria are only possible if a consensus has been reached to distinguish these criteria. This consensus has not yet been reached [8].

5) Combining non-structured care with structured evaluation. The necessary data to enable a comparative analysis of the results of almost all therapy options applied in non-structured care can be collected by the following steps. For each patient, the primary health problem to be solved must be defined. For this purpose, each society must either accept a superordinate nomenclature or define its own nomenclature. It should be borne in mind that the latter option involves the considerable disadvantage of not being able to use international experience to make its own decisions. The likelihood of occurrence of an adverse health event (e.g., death or morbidity or inadequate response to therapy) is influenced by preexisting risk factors, which may be different for each endpoint. Because there are different adverse problems to be avoided in each individual patient (in terms of main outcome criterion, side effects, and cost of care), the specific risk profiles for each of these problems (end-points of care study) must also be considered. A comparison of different patients with respect to a specific endpoint is only possible if each patient is assigned to an "endpoint-specific risk class" for each of the measured endpoints upon enrollment in a healthcare study. Since the definitions of these risk classes are pre-defined by the study management group of each PCT and stored in the PCT's IT system, a significant part of the workload and complexity of the process can be taken over by IT solutions. In addition to the existing baseline risks, the sum of all applied interventions influences the measured endpoints. In the future, systematic data collection will also make it possible to take into account interpersonal factors, e.g. effects of the doctor-patient relationship or interprofessional communication. Since in most health care studies, different interventions are to be compared with each other, it has to be considered which concrete effects are classified as relevant for decision-making and is therefore taken into account in a study. It often remains unnoticed that the majority of our individual and almost all policy decisions are influenced by objective criteria, but are almost always decided on the basis of subjective values [9 - 13]. These ultimately subjective decisions are an unavoidable cause of the observed variation in forms of care. Without a concrete description of this variation, it is also impossible to distinguish desirable from undesirable influencing factors. Many methods claim to describe RWE but don’t consider the effects of confounders. A method that claims to describe reliable data on RWE needs to meet three conditions:

- It should name the forms/structures and functions, which makes the proof of objective RWE different from the proof of objective PoP and from the proof of subjective value [4]. - It should include a solution that allows the provision of care under nonstructured conditions, but quantifies the outcomes of care under structured conditions [4- 7]. - It should consider two pairs of thresholds (threshold quartet) explained in the net paragraph.

6) Definition of the threshold quartet (two pairs of different thresholds). In contrast to clinical research, in which the definition and demonstration of a single threshold is sufficient to demonstrate PoP, demonstration of RWE requires the definition and demonstration of two thresholds: (1) demonstration of the existence of a health problem requiring care and (2) demonstration of an intervention that can alleviate or cure the existing health problem. As a reminder of a significant difference, evidence of PoP in an experimental study can only confirm the efficacy of an intervention in principle, not the ability of that intervention to induce a clinically meaningful effect. This difference between PoP and RWE has not been discussed before because no method to prove RWE was available until now and RWE did not exist in our thought model. About 20 years ago, the literature discussed the Minimal Clinically Important Difference (MCID) and the Minimal Detectable Change (MDC) as competing measurement methods in RCTs [14]. The idea that they might be complementary methods whose information could be combined was not supported by the thinking at the time, which was based on RCTs and consequently on the assessment of PoP but not RWE.

7) Differentiation of initial care goals/strategies and reasons for their change. The definition of goals of care has been one of the issues we have continuously pursued in the care of oncology patients since the mid-1980s [6, 15 - 22]. The need to define and subsequently review specific goals has been confirmed to justify diagnostic and therapeutic interventions. Because the goals of care are different for primary, secondary, and tertiary prevention, the risks of overtreatment were high without estimation and confirmation of realistically achievable goals. Interdisciplinary discussion of acute and chronic care problems highlighted the substantial differences that must be considered when planning and managing care approaches.

8) Required recruitment and training of a new professional group. Another indispensable prerequisite for proving RWE is the recruitment and training of a professional group that can represent the link between care and research by collecting data. Physicians and nurses will indeed be responsible for implementing this task. However, neither of these groups will be able to take on these additional tasks alongside patient care. Therefore, a training concept is needed to describe RWE. The core of this concept should be the competence to collect, document, and analyze data. In addition, this group of experts should be capable of communicating with treating physicians, scientists, nursing, and controlling in order to recognize the complex challenges of proving RWE and to solve them together with the cooperating partners.

9) Expected societal added value through proof of RWE. Proof of the suitability of health care services for daily use (RWE) reflects the expected social added value of the services provided because it can be concretely confirmed which of the intended goals can be achieved under which conditions, with what frequency and completeness. The most significant aspect of this is the satisfaction of appropriate basic needs of individual patients and society. This goal can only be achieved through consensus and trust. Contradictory, unrealistic, and disturbing information should be avoided because of its significant potential to induce fear and uncertainty [13].

10) Differentiate between scientific and policy tasks. The statements regarding thresholds lead to the consideration in health care that different tasks should be performed by science and policy. Over the past 50 years, academic teaching has given the impression that valid scientific evidence justifies the guidance for decisions in the health care system. We would like to put this statement into perspective by echoing the statement of a thought leader in the British health care system, Sir John Armstrong Muir Gray (b. 1944): "Scientists make decisions, policymakers take decisions" [9]. We, scientists, have to prepare and justify principled decisions, while policymakers have to make principled decisions. The individual decisions of the respective patient are unaffected by this because they can only be made and answered for by the treating therapist.

Results

1) The description of functions and structures of the three different conditions of care. When different forms of health care are conducted, the un-different functions of each should also be named. The function of the study should be consistent with the chosen form or structure of the study. Through the application of the FFF designer rule, it had been noticed that in several studies the expected function was either not described or was inconsistent with the presented form/structure of the study. In a recent manuscript, it is confirmed that the three forms of care - experimental care in an RCT, non-experimental but controlled care under everyday conditions in a PCT, and usual pragmatic everyday care without systematic evaluation - are differentiated by two functional and 12 formal/structural criteria [8]. The graphical representation confirms that experimental care conditions are differentiated from everyday care conditions outside of trials by two functional and 12 formal/structural criteria. A PCT is differentiated from the other two care conditions by only two formal criteria but by six criteria from experimental studies and by four criteria from everyday care conditions. Since there are no contradictions between functional and structural/formal criteria in any of the three care conditions, we assume that the described concept on the form and function of care conditions stands up to critical scrutiny. 

2) The terminology conflict between "efficacy" and "effectiveness." This conflict could be confirmed by the evidence of the synonymous use of the two terms "efficacy" and "effectiveness". Both terms are often used synonymously in the literature, although the scientific consensus is that the term "efficacy" refers to the proof of the principle of action of an intervention under (ideal) experimental study conditions, and the term "effectiveness" refers to the proof of the suitability of an intervention for everyday use. The logical consequence that everyday conditions cannot be observed in an experimental study and therefore can only be measurable if the care actually took place under non-structured everyday conditions has not been explicitly demanded so far, not least because no method has been offered so far with which valid measurements of the results can be carried out under the mentioned study conditions. By proving this conflict, it was possible to confirm its cause the lack of observance of the designers' and architects' rule "Form Follows Function (FFF)". The violation of the FFF rule inevitably leads to an incorrectly applied terminology and the lack of differentiation between "Efficacy" and "Effectiveness". The lack of differentiation prevents the consistent interpretation of clinical trial results. The inconsistent interpretation of clinical trials causes an unexpectedly high inconsistency of recommendations in clinical guidelines [23]. Applying the FFF designer rule to the design of the three-dimensional response recommended by Cochrane and Bradford Hill ('Can it work?', 'Does it work?' and 'Is it worth it?') avoids the terminology problem and the derivation of erroneous consequences if the mindset that caused the problem can be abandoned [3].

3) Five criteria to differentiate "Efficacy" / "Proof of Principle", "Re-al-World Effectiveness" and "Value". The combination of the two findings, the avoidance of the terminology conflict, and the combination of the supply and evaluation conditions finally resulted in the three-dimensional strategy with which the three Cochrane-Hill questions can be answered without overlapping in content. The result is shown in Table 1 [4 - 7].

This strategy is based on the consideration that only the objective proof of the principle of action is plausible under experimental conditions. Neither the objective proof of everyday suitability nor the subjective description of the value of a health care service can be done under experimental conditions because any kind of experimental intervention will distort the exact description of the effects obtained under everyday conditions of care. As a result, answering two of the three Cochrane-Hill questions is only possible under everyday conditions of care. Experimental study conditions are only suitable for demonstrating an effect principle. By using different strategies to prove an effect principle or everyday suitability, it is predictable that the interpretations of the results of RCTs and PCTs must also differentiate.

4) Formal and functional differences between inclusion and exclusion criteria in trials. Our traditional way of thinking requires the definition of inclusion and exclusion criteria for the design of a healthcare study. This assumption is correct for experimental studies but not for pragmatic studies. The function of exclusion criteria is to reduce potential confounders that could bias the analysis of a study. In experimental studies that aim to demonstrate an effect principle, the risk of not being able to provide the desired evidence would increase with the number of confounding factors present. Therefore, it makes sense to exclude confounding factors in experimental studies. However, this consideration does not apply to non-experimental studies to demonstrate suitability for everyday use, because the so-called confounding factors cannot be avoided under everyday conditions and their targeted avoidance in healthcare studies would distort the description of everyday health care.

The function of the inclusion criteria is to specify all criteria that a subject must fulfill in order to be included in a study (experimental or non-experimental / pragmatic). How narrowly or broadly the inclusion criteria are defined depends on the exact question of the study. As a conclusion of our current knowledge it can be deduced that the more precisely a precise research question matches the study population, the confounding factors considered and effective, and the interventions described and applied, the more reliable the results of any study will be. Any imprecision in the design of the study will be reflected in the inadequate precision of the results [8].

5) The combination of non-structured care with structured evaluation. Evidence of RWE can only succeed if effects generated under the non-structured conditions of everyday care are actually measured. However, this measurement can be done if the available data are transformed into a structured form. We argue that the combination of unstructured care conditions with structured evaluation conditions is only successful if the intended endpoints of care are defined as measurable (adverse) events and classified by each subject's risks of achieving one of these undesirable endpoints. This strategy is based on Bayes' principle. Application of this principle requires careful planning that involves the selection of the endpoints to be measured, identification and classification of the individual risks of each subject with respect to each of the measured endpoints, and categorization of all interventions to be included in a health care study [5 - 7]. This proposed solution may seem complex but is necessary to differentiate influential from low-impact factors using measurable criteria.

6) Definition of two thresholds of care. The definition of the four thresholds is in line with the requirements of evidence-based medicine and aims to provide guidance to decision-makers in everyday care to avoid overuse. Individual deviations from thresholds will occur in any care setting. If the recommended thresholds are systematically exceeded, the results should be discussed to rule out the possibility that the recommended thresholds do not meet the requirements of everyday care.

7) Differentiation of initial care goals/strategies and reasons for their change. We skip the discussion of widely known care goals and strategies and limit ourselves to the discussion of the experiences of colleagues in intensive care medicine. In principle, any development of a care strategy should be based on a realistic assessment of the patient's existing opportunities and risks. The aim of any intensive care strategy is to preserve the function of all impaired organ functions in the event of a temporary health risk by taking maximum measures that do more good than harm. To this end, two aspects must be taken into account. First, the measures cannot be offered without the patient's consent. Second, they are to be reduced after an initial phase of maximal care, as soon as either a successful bridging of the critical health phase has been achieved or a greater benefit can be achieved in another patient who could be helped by the locally available resources by discontinuing an individual maximal therapy that has not yet achieved its goal. This strategy is in line with the current definition of triage [24], which, however, has so far been based on subjective assessments of the chances and risks by the caring team, because standardized methods to prove fitness for purpose are not yet available. The principle experience and considerations gathered and weighed under the conditions of intensive care may apply to any form of care under the condition that primarily the concrete and realistically achievable goal of care has been documented and signed by the responsible team. This example illustrates the importance of evidence from RWE, whose data will be needed in the future to guide healthcare delivery.

8) Necessary recruitment and training of a new professional group (experts for applied health services research) as a link between health care and health services research. The primary task of the new professional group is to ensure IT competence in the care team. IT competence includes data literacy (basic understanding of data generation, care strategies, and the tasks of the care team, knowledge of data collection, use, and analysis, including digitization), basic knowledge of communication with the care team (basics of medical terminology, including its abbreviations) and basic knowledge of care and communication with controlling (ability to carry out plausibility checks). The establishment of new positions for applied health services research must ensure that the two task blocks of health services and health services research remain clearly separated by the personnel structure and personnel functions. The areas of tasks and responsibilities must be distinguished from one another because care and research pursue different goals and must apply different strategies and observe different preferences to achieve them. These principles must be observed by all team members in order to comply with ethical principles that protect patient health and data. From the perspective of the universities and colleges being trained, the further specific requirements for the training programs for this new activity will only emerge through practical experience in the field. Therefore, in the phase of development, the training centers will need teaching staff to develop programs at the interface between the practice of health services research and the training of applied health services researchers to solve challenges that arise from the everyday life of the new profession (practicality and efficiency). We want to avoid developing a theoretically conceived professional profile that can only partially fulfill the functions expected of it. As a goal of this training, new members of a care team should not only be trained but also shaped for dealing with patients with health problems and professionally stressed staff. No risks should be taken to cause problems in the cooperation between care and care research because of staff shortages. Only if care can be offered without restrictions, will care research be able to take its place and support progress in care. At TH Deggendorf (University of Applied Sciences Deggendorf), this path of inter- and multidisciplinary education and transfer of competence at the data interfaces is pursued and consolidated by joint lectures, e.g. in the Bachelor Physician Assistant or in the Bachelor Nursing and by joint internships in a teaching and transfer laboratory. For the other study programs in the field of management in health, social and rescue services, interdisciplinary seminars are offered to promote the professional exchange of different fields of study in joint case discussions.

9) Expected societal added value through the demonstration of RWE. Evidence of RWE will enable a redesign of the health care system (licensing procedures, pricing, care concepts, guidelines, liability issues, basis of medical law decisions, reimbursement issues) because decisions can be based on achievable outcomes (RWE and VAL) instead of PoP or services provided. Outcome-based assessment of the services provided by all healthcare professionals will reveal a new weighting of patients' needs: Patients will evaluate the quality of biomolecular and technical solution strategies for the relief of physical and psychosocial distress. A separation of responsibility for care and for research could become necessary in the field of health research to avoid conflicts of interest because the interests of care and science have to be weighed against each other and should therefore each be represented by the profession concerned. Experienced nurses could be ideally qualified, due to their professional background, to guarantee a uniformly organized evaluation of RWE by means of special additional training. This would provide nurses with management experience who no longer feel fully able to cope with the physical stresses of the nursing profession with an opportunity to fully preserve their experience for the health care system.

10) Differentiate between academic and policy roles. Specific examples of meaningful cooperation between science and policy emerged during the Covid-19 pandemic. The national and international press, as well as various public health policy institutions, justified the determination of the pandemic by the existing excess mortality. Rupert Kroemer from our team was able to obtain the necessary data on the number of inhabitants and the number of annual deaths at the end of the year for a review of excess mortality from the Federal Statistical Office [25]. Figure 1 shows that the relative mortality in Germany between the years 1950 and 2020 describes a sinusoidal course with a maximum between the years 1968 and 1975 and a minimum between the years 2001 and 2006. From these data, it can be deduced that the small annual differences in the mortality of a population are probably influenced by short-term factors, whereas the sinusoidal course over 70 years indicates long-term effects. This course also confirms the problem of defining existing excess mortality. Depending on the reference time point chosen, higher or lower mortality can be confirmed for 2019 than in the reference year.

As a second example, we had discussed with our scientific colleagues and with policymakers to interpret polymerase chain reaction (PCR) initially only as an amplification method, but not as a "test" without additional data. Since the risk of possible misinterpretation was already pointed out in the American patent specification for this method [26], we performed 59,853 examinations in the city of Foz do Iguaçu / Paraná in cooperation with Brazilian colleagues to test the assumption of whether a positive PCR result indicates a clinically significant risk. This assumption should be confirmed by more frequent hospitalization in individuals with positive than negative PCR results. The results of our study did not confirm this assumption (Table 2) [25].

The two examples of excess mortality and the use of PCR as a diagnostic tool confirm the need for cooperation between science and policy, with science having the task of proposing solutions that can be supported with robust data. Politics should initiate the cooperation between science and politics and decide on the basis of the scientific feedback which of the available scientific proposals can be implemented.

Discussion

This complex discussion of methodological aspects to prove the suitability of health services for everyday use can only be justified if society confirms the will to implement a useful solution. This confirmation can be achieved by a convincing concept for the optimization of health care, by different ways of thinking in health care, health science, and health policy that is oriented towards the respective goal, and by new opportunities that are opened up, among other things, by the proof of the suitability for daily use (RWE) of health care services.

The compelling concept of Optimizing Health Care. Our report reflects a subjective perspective on the potential optimization of healthcare delivery. Health services research could become the supreme discipline of health sciences for two reasons. It describes the strategy for implementing the three-dimensional Cochrane-Hill concept. Progress in health care will be promoted by shifting the perspective of observation. The focus of orientation will shift from experimental "efficacy" to pragmatic "effectiveness" (suitability for everyday use). This shift is graphically illustrated in Figure 2.

As our mindset changes, individual and societal evaluations could in the future be made through a subjective evaluation of the objectively measurable RWE rather than a subjective evaluation of the PoP. By presenting this real perceived value based on measured fitness for everyday use, the trade-off between health benefits and harms becomes quantifiable. The demonstration of the suitability for daily use is to be seen as progress because the high sensitivity of this measurement method makes it possible to demonstrate the differences between competing treatment strategies. This exposes the hitherto little addressed problem that previously unquantifiable effects may contribute more than expected to alleviate specific health problems. Also helpful in solving this problem may be Albert Einstein's statement that problems cannot be solved with the same mindset that caused them.

Goal-oriented ways of thinking about health care, science, and policy. Three considerations suggest that health care, health science, and health policy each have specific tasks to perform. Science is constantly on a journey toward truth; only in the case of accepted laws of nature can it be assumed with sufficient probability that the current state of knowledge reflects more than a "partial truth." The second consideration is described in the safety loop. It states that almost all our decisions are made not on the basis of objective risks, but on the basis of subjective perception of objective risks [10 - 13]. No one can escape the influence of risk communication by the media. Therefore, even in health care, principled decisions should be made by professional bodies with diverse perspectives. Practitioners, scientists, and decision-makers use different decision-making strategies, so it will hardly be possible for one person to apply practical, scientific, and political strategies to solve the same problem at the same time. It is also not possible to play chess against oneself if different strategies are to be used for this purpose. The third consideration arises at the interface between care and research. Every responsible physician knows the conflict when his own conviction contradicts a scientific recommendation. Providers, researchers, and policymakers are differentiated by their values, by their goals, and by their decisions. It is to be expected that the resolution of these functional conflicts will require structural solutions in the future. The healthcare professional (including “nurse practitioners”) providing care has to be convinced of a concept, the scientist (including “nurse scientists”) has to question a concept and correct possible errors, and the politician has to bring about compatible solutions to implement concepts.

New opportunities through proof of everyday suitability (RWE) of health services. Access to and affordability of essential health services should be a core task of every society. Without proof of RWE, however, this core task can hardly be guaranteed. The effort required for data collection and for planning, including consensus building, of a PCT will be justified by avoiding overprovision. Risk profiles, thresholds, and other details of the PCT should not be perceived as a restriction but as a guarantor of freedom of therapy. Thresholds would block their own function if they were conveyed as binding specifications. They should therefore be offered as orienting recommendations to make each person making decisions aware of the implications of their own decisions and the power of personal responsibility. Without co-decision by the patients concerned, it will not be possible to establish a consensus on threshold values. A significant task for academia, the media, and policymakers is to provide the necessary transparency of reporting and the knowledge needed to interpret reported data [27]. The considerable resources that must be expended to identify untrustworthy reports will continue to fizzle out ineffectively and influence public opinion and political decisions unhindered if the privately organized expert panels that already exist are not systematically used to assess far-reaching political decisions neutrally and professionally and to make them publicly perceptible. Adherence to these criteria avoids overprovision and thus the waste of valuable resources. If a majority can be won over to a change from an "Efficacy-Based Value" to an "Effectiveness-Based Value" (Figure 2), further opportunities for healthcare and healthcare financing will arise that have not been discussed so far. The majority of society will always decide on the basis of the prosperity achieved, not primarily on the basis of health care. Therefore, optimal health care can only be financed if the economy is flourishing. This link between health and the economy highlights the cooperation needed to successfully manage both challenges. This challenge will not succeed without taking ownership. Power is the reward of assuming responsibility because assumed responsibility goes hand in hand with an increase in desired characteristics, e.g. a mission statement, respect, and influence. In all decisions, two aspects must be considered. A decision always involves the allocation of available resources, e.g. one's own skills, instruments, time, or money, to achieve a specific goal. The principle of opportunity cost states that only a few resources are available without limits and therefore any allocation of resources reduces the possibility to use these resources for other goals. Therefore, triage will remain an unavoidable issue in health care. The second aspect concerns the basis of health care decisions, which are mostly based on studies. Clinically relevant studies probably underestimated the risk of health care without sound clinical experience and methodological knowledge. The increasing complexity, growing administrative constraints, and rising costs of experimental clinical trials have spurred the development of a new experimental trial design, a modified RCT as a 'joint option' [28]. It is also possible that the PCT could be considered a solution to the problem, because this design preserves the therapists' freedom of therapy, demonstrates the suitability of the interventions for everyday use, and saves considerable reinclude sources [29]. Table 3 outlines the implementation of a PCT in eleven steps. 

References

1. Porzsolt F, Gaus W. Wirksamkeit und Nutzen medizinischer Maßnahmen: Ein Beitrag zur Optimierung des Gesundheitssystems. Der Klinikarzt 1993; 12: 522-528.

2. Sullivan LH. The Tall Office Building Artistically Considered. Lippincott’s Magazine 1896;57: 403-409. Reprinted in Inland Architect and News Record 27 (May 1896), pp. 32-34; Western Architect 31 (January 1922), pp. 3-11; published as “Form and Function Artistically Considered” The Craftsman 8 (July 1905), pp. 453-58. https://www.giarts.org/article/tall-office-building-artistically-considered-excerpt. Last access oct 22, 2019.

3. Haynes B. Can it work? Does it work? Is it worth it? The testing of healthcare interventions is evolving. BMJ. 1999 Sep 11;319(7211):652-3. doi 10.1136/bmj.319.7211.652. PMID: 10480802; PMCID: PMC1116525.

4. Porzsolt F, Wiedemann F, Phlippen M, Weiss C, Weiss M, Schmaling K, Kaplan RM. The terminology conflict on efficacy and effectiveness in healthcare. J Comp Eff Res. 2020;9:1171-1178. doi: 10.2217/cer-2020-0149.

5. Porzsolt F, Eisemann M, Habs M, Wyer P. Form Follows Function: Pragmatic Controlled Trials (PCTs) have to answer different questions and require different designs than Randomized Controlled Trials (RCTs). J Publ Health 2013;21:307-313. DOI 10.1007/s10389. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3655212/

6. Porzsolt F, Rocha NG, Toledo-Arruda AC, Thomaz TG, Moraes C, Bessa-Guerra TR, Leão M, Migowski A, Araujo de Silva AR, Weiss C. Efficacy and Effectiveness Trials Have Different Goals, Use Different Tools, and Generate Different Messages. Pragmatic and Observational Research 2015;6:47-54. DOI http://dx.doi.org/10.2147/POR.S89946.

7. Porzsolt F, Weiss Ch, Weiss M. Covid-19: Hybridmethode zum Nachweis der Real-World Effectiveness unter Alltagsbedingungen [Covid-19: Twin method for demonstration of real-world effectiveness (RWE) under the conditions of day-to-day care]. Das Gesundheitswesen 2022;84:1-4. DOI: 10.1055/a-1819-6237. ISSN 0941-3790.

8. Porzsolt F, Phlippen MS, Legrum P, Balassone V, Kamga GO, Ghosh K, Ghosh A. Consistency of form and function of the building blocks of clinical trials. Submitted for publication.

9. Gray JAM. Evidence-based policy making. BMJ. 2004;329(7473):988-989. doi:10.1136/bmj.329.7473.988.

• Porzsolt F, Polianski I, Görgen A, Eisemann M. Safety and security: the valences of values. Journal of Applied Security Research 2011,6:4,483-490. http://dx.doi.org/10.1080/19361610.2011.604069.
• Porzsolt F. Safety means perception of risk. J Med Safety 2016; Oct:18-24. ISSN 1349-5232. http://www.iarmm.org/JMS/JMS2016Cover.pdf
• Porzsolt F. Clinical Economics – It is about Values not about Money. Brazil J Medicine Human Health 2016;4 (3).  DOI: http://dx.doi.org/10.17267/2317-3386bjmhh.v4i3.1052
• Porzsolt F, Pfuhl G, Kaplan RM, Eisemann M. Covid-19 pandemic lessons: Uncritical communication of test results can induce more harm than benefit and raises questions on standardized quality criteria for communication and liability. Health Psychology and Behavioral Medicine 2021;9:818-829. DOI 10.1080/21642850.2021.1979407.
• Porzsolt F, Legrum P, Weiss Ch, Weiss M. "Proof of Principle (PoP)" or "Efficacy" is the one-dimensional counterpart to the two-dimensional "Real-World Effectiveness (RWE)".
• Kreuser ED, Anger B, Porzsolt F. Ziele, Organisation und Akzeptanz des Interdisziplinären Onkologischen Konzils am Tumorzentrum Ulm. ATO 1986;5:56-62.
• Porzsolt F. Ziele der palliativen Krebstherapie. Dtsch Arztebl 1992;89:2974-2976.
• Porzsolt F, Tannock I. Goals of palliative cancer therapy. J. Clin. Oncol. 1993;11:378-381.
• Porzsolt F, Wirth A, Mayer-Steinacker R, Voigt W, Heymer B, Link KH, Schulte M, Röttinger E. Qualitätssicherung in der Onkologie: Zieldefinition und Zielerreichung am Beispiel von Therapieempfehlungen des Interdisziplinären Onkologischen Konsils des Tumorzentrums Ulm. Münch. med. Wschr. 1994;136:371-376.
• Porzsolt F, Wirth A, Mayer-Steinacker R, Schulte M, Negri G, Suhr P, Link KH, Gaus W, Röttinger EM. Quality assurance by specification and achievement of goals in palliative cancer treatment. Cancer Treatment Reviews 1996;22:41-50.
• Porzsolt F. Neues Ziel. Von der Wirkung zum Nutzen. In: Frickhofen, Porzsolt, Fetzer (Hrsg.). Die Ausbildung zum Arzt. Rahmenbedingungen, Ziele, Elemente. Bericht über ein Symposium aus Anlaß der Emeritierung von Prof. Dr. Hermann Heimpel. Stuttgart, New York: Schattauer; 1998. p. 23-28.
• Porzsolt F, Zimmermann T.  Optimierung der Gesundheitsversorgung am Beispiel der Rehabilitation: Angestrebte Ziele sind zu definieren und erreichte Ziele zu bestätigen. Med Klin 2010;105:345-350 DOI 10.1007/s00063-010-1063-1
• Porzsolt F. Bedeutung einer zielgerichteten Versorgungsstrategie. In: Porzsolt F (Hrsg). Grundlagen der Klinischen Ökonomik. Schriftenreihe PVS Verband Band 11, 1.Auflage. Berlin 2011, pp 25-33.
• Porzsolt F, Wiedemann F, Schmaling K, Kaplan RM. The risk of imprecise terminology: incongruent results of clinical recommendations in clinical trials and incongruent recommendations in clinical guidelines. 10.1136/bmjebm  2019;24(Suppl 1):A17-A18.
• Savioli G, Ceresa IF, Gri N, Bavestrello Piccini G, Longhitano Y, Zanza C, Piccioni A, Esposito C, Ricevuti G, Bressan MA. Emergency Department Overcrowding: Understanding the Factors to Find Corresponding Solutions. J Pers Med. 2022 Feb 14;12(2):279. doi: 10.3390/jpm12020279. PMID: 35207769; PMCID: PMC8877301.
• Porzsolt F. Kroemer R, Silva-Sobrinho RA. The two sides of the value medal – confirmed or not confirmed by Real-Word Effectiveness? Value-Based Health Care Thinkers Magazine Nov. 2022: 32-35.
• Patent US4683202A: Process for amplifying nucleic acid sequences. Angemeldet am 25. Oktober 1985, veröffentlicht am 28. Juli 1987, Anmelder: Cetus Corp, Erfinder: Kary B. Mullis.
• Doi SA, Furuya-Kanamori L, Xu C, Lin L, Chivese T, Thalib L. Controversy and Debate: Questionable utility of the relative risk in clinical research: Paper 1: A call for change to practice. J Clin Epidemiol. 2022 Feb;142:271-279. doi 10.1016/j.jclinepi.2020.08.019. Epub 2020 Nov 7. PMID: 33171273.
• Bowman L, Weidinger F, Albert MA, Fry ETA, Pinto FJ. Randomized Trials Fit for the 21st Century. A Joint Opinion from the European Society of Cardiology, American Heart Association, American College of Cardiology, and the World Heart Federation. Global Heart. 2022; 17(1): 85. DOI: https://doi.org/10.5334/gh.1178.
• Porzsolt F, Weiss Ch, Silva-Sobrinho RA, Mayer PCM, Herzberger BA, Weiss M. "Joint Option" and the Management of Complexity in Everyday Healthcare. Submitted for publication.
  • A precise description of the health problems focused on in this project 
  • Objective of care (e.g. to prevent hospitalization in chronic diseases)
  • Hypothesis #1… (e.g. substitution of … reduces the 1-year hospitalization rate due ..)
  • Threshold (need of care is considered) (e.g. patients with a high risk of hip fracture) Threshold (success of care is assumed) (e.g. reduction of fractions/year by 15%)   
  • Definition of endpoint-specific risk profile in study participants at study entry
  • Definition of endpoint-specific risk classes (high, low, and intermedial) by risk factors 
  • Definition of single (or categories of) interventions

Once the feasibility is proven, every health system will take up this solution. It increases the efficiency of health care because all decisions can be measured against the added value achieved for the individual patient and for society through the demonstration of Real-World Effectiveness (RWE) and the introduction of thresholds (to demonstrate a health problem in need of treatment and to demonstrate a beneficial intervention).

I am applying to SOLVE in the hope of getting in touch with partners who are interested in combining medical and economic aspects without violating ethical issues.
A healthcare system that wants to use the progress of medicine cannot be financed without a flourishing economic system. This marriage of convenience between medicine and business carries the risk of a conflict of interest or unethical actions. That is why I like to use the slogan "Making money without dizziness and deceit" (in German "schwindelfrei Geldverdienen")  to remind my colleagues of the opportunities and risks of our profession.    

Our innovation consists in developing a method to answer the second Cochane-Hill question "Does it work?". To confirm the validity of this evidence, we described a three-dimensional strategy. It confirms that the answers (Proof of Principle, Real-World Efficacy, Value) to the three Cochrane-Hill questions also differ based on the conditions (experimental or pragmatic), perspectives (experimental research or health services research or economic research), study types (explanatory study or observational study), and tools (Randomized Controlled Trial or Pragmatic Controlled Trial or Complete Economic Analysis). 
Answering the second Cochrane-Hill question is the central key of health services research because it can be used to prove the functioning of a health care system. So far, randomized trials have only been able to demonstrate effectiveness under experimental conditions (analogous to diesel exhaust tests under laboratory conditions). Although it was suspected that there were significant differences between the results generated under experimental conditions or under everyday conditions, the methods to prove the suspected difference were lacking. This evidence can now be readily provided.         

Over the next year, we will support local staff in implementing local standards of care while ensuring that the necessary infrastructure is in place to confirm that the system is working by continuously documenting the outcomes achieved. To do this, we need staff to provide ongoing guidance through site visits and regular video conferencing.

At the end of the year, we expect to have collected the data that will allow us to demonstrate the health value added, so that we can no longer judge the financing of health services by the services provided but by the value added achieved.

If our proposal is accepted by MIT-SOLVE, we will start in parallel with an IT company to prepare the knowledge and the necessary technical requirements to offer the implementation of the system in interested countries.

A secret wish would be to set up an office right next to the newly emerging "Albert Einstein Discovery Center" (an initiative of an American physicist who lives in Ulm) as a nucleus for preserving the "Ulm heritage": the "Ulm heritage combines three elements to find innovative solutions:
- Albert Einstein's warning "A problem cannot be solved with the way of thinking that caused the problem".       
- The rule of designers and architects "Form Follows Function" whose general validity we are currently trying to prove in a separate project to distinguish between cause and effect, and
- The claim of the former "hochschule für gestaltung ulm (academy of design ulm, 1953 - 1968) to develop not only specific but generally valid solutions with socio-political relevance. 
To this end, we are currently submitting a four-part series of scientific publications
the "Quadriga of Clinical Trials" for publication.

We measure the frequency of achieved goals, according to a previously defined threshold (temporal, quantitative, qualitative). Targets are considered to have been achieved if a desired health goal can be reached with less stress than before. This can be financial, physical, psychological, and social burdens.
We use the Declaration of Alma-Ata 1978 as an international definition of the care goals to strive for    

The quality of current health care is affected by existing conflicts of interest. 
Healthcare services without added value are avoided and replaced by healthcare services with added value. 
Raise the awareness of health care providers to orient success to the goal of the patient, but not to the goal of the health care provider. 
Capture the actual added value per patient group under everyday conditions to optimize endpoint-specific risks. 

The core technologies of this solution were 

Based on the three parts of the "Ulm Heritage"

- The rule of American architects and designers:
   "Form Follows Function (FFF)"  

- Albert Einstein's warning:
  a problem cannot be solved with the same mindset that caused it.

- The philosophy of the hochschule für gestaltung (academy of design; 1953 -    1968): designing products not only for a specific purpose but for general     
   applicability with societal significance 

The ideas that 

 - "real-world conditions" represent a "natural chaos"

- a "natural chaos" cannot be analyzed in an artificial, well-structured study

- the endpoint-specific risk profile enables the analysis of a "natural chaos" 

The solution follows the designer rule "Form follows function (FFF rule)". It has been shown that concepts and products that follow the FFF rule are more successful than concepts and products that do not follow this rule. 

Public Paper: Form Follows Function

We, the project team, have always been committed to creating the conditions for equality and tolerance towards all persons, countries, strata of the population in the field of health services.

Disadvantages of individual nations, genders, as well as socially disadvantaged groups are thus reduced in the first step and eliminated in the long term and a balance is created.  

The business model consists in the reduction of inferior healthcare services in order to lower the costs of care while maintaining the quality of care. 

This increases the efficiency of the applied healthcare services without producing additional costs for the healthcare provider or the patient. 

The work is financed in the long term through a combination of several services. These include grants, services, IT products, but also the individual governments. 

Research accomplishments to date over the past 20 years have been made without financial income to the research group. 
The Institute of Clinical-Economics e.V. is a non-profit association.