The balance between safety and freedom: ‘we will cope’

Franz Porzsolt, Ulm

In our previous article "Shots in the Olympia Shopping Centre Munich" we discussed the concept of ‘perceived safety’, the risk cycle and its importance for our decision making. This article discusses the issue of the refugee crisis and is intended to show that the quality of safety competes with other factors such as freedom and that there will always be a trade-off between competing factors. 

Last year, we in Germany (80 million residents) accepted 1 million refugees. The discussion about this immigration is referred to as the ‘refugee crisis’. The example of the ‘refugee crisis’ in Germany confirms that the concept of ‘perceived safety’ is also important for political decision making. These political decisions affect the safety and freedom of both the citizens of the host country and the refugees themselves. A lack of safety leads to a considerable reduction in the quality of life and freedom of action. Because our focus is on health care provision, we are interested to know what we can take away from the current discussion that is relevant for health care.

We Will Cope

In September 2015, Angela Merkel expressed clear support for Germany’s refugee intake with her statement ‘We will cope’. Whether this statement was justified became the subject of a heated debate. Realists raised the valid question of whether the statement could be justified if there is no concrete solution for implementing the intake process. For others, the moral obligation to provide assistance has greater priority regardless of whether there is an existing solution. 

If the consequences of both decisions are considered, at first glance the issue is off the table for the host country if the response to opening the borders is NO. The search for a solution to this international problem becomes of secondary importance to us. If the response to opening the borders is YES, however, a series of problems that are difficult to foresee emerge for the host country. For the refugees, at least one initial problem is resolved and a new outlook on life opens up.

Considering whether NO or YES is appropriate when all the advantages and disadvantages are weighed up can be answered if underlying value judgements are taken into account, and we consider making decisions that we would contemplate in exceptional circumstances.

Numerous examples demonstrate that our society feels obliged to help those in need based on our own value judgements. As one of the most affluent countries in the world, we should remember that we were only able to take the first steps towards reconstructing our country after 1945 with external support. Even if self-serving motives are assumed for those providing the help, the alternative of not supporting those in need and leaving them to their fate is not an acceptable alternative from a moral perspective. From a moral point of view that is applicable to Germany, the statement ‘We will cope’ is therefore the correct response. Saying NO to opening the borders is hardly justifiable from this point of view.

Implementing the Commitment

The second question relates to implementing our commitment. It requires us to weigh up our values. Greater safety does not lead necessarily to more freedom. The opposite can also apply, with too much safety resulting in a considerable restriction of freedom. A person can secure their home and land with a fence, bars on the windows, an alarm system and security patrols. Each of these safety-related measures is associated with a reduction in that person’s freedom of action. The greater a person’s or society’s need for safety, the more freedom must be sacrificed. 

This correlation and the balance between safety and freedom that is required can also be applied to the refugee crisis. Some of the rules that have been defined to ensure our social safety are perceived by the public as bureaucratic hurdles to resolving the refugee crisis.

Actions to Resolve the Refugee Crisis

Even though stigma of being an economic refugee is problematic, we have to define criteria for acceptance as a refugee in our country. Managing the refugee crisis is an international problem. Every country that can contribute to resolving the problem is subject to this shared obligation.

Most of the refugees currently have a low potential for development. Well-educated refugees will not have any major problems after overcoming the language barrier in terms of their participation in the job market and their social integration. For most of the refugees, however, new programs will have to be developed to familiarise the people, most of whom are young, with their future roles in our society. As part of this, values and qualities must be taught that are essential for a successful work life and social integration. It is difficult to explain to refugees that the months of waiting must go by unused because no solutions have yet been found to start taking useful steps towards immediate integration.

An impressive number of volunteers have gathered to take on the tasks that will fall to our society. Trust has been built up and contact made between volunteers and refugees, both of which are valuable foundations for the integration process. Individual solutions such as a system for recording the personal details and resources of the refugees have been developed. Although these spontaneous activities appear uncoordinated and pointless from the authorities’ perspective because they do not appear to be of any use to the official integration process, this response sets the wrong example.

Positive signals to volunteers, refugees and citizens prevent increasing levels of demotivation and dissatisfaction. The interest in the services provided by volunteers and the progress made by refugees should be apparent and the benefits of the commitment identifiable. The volunteers must be registered, instructed, motivated and supported. The establishment of a ‘We will cope’ foundation to support useful projects will help to sustain motivation. Our existing legal framework is possibly unsuited to resolve problems that are the result of a ‘social emergency’. These new challenges confronting our society justify discussing the ‘definition of a legal sphere for refugees’, which can provide refugees with prompt assistance while at the same time protecting them from abuse and exploitation.

Reducing our legally regulated requirements for social safety would enable us to increase the level of freedom that we urgently need to rapidly integrate refugees. This facilitation of the integration process is associated with a consciously accepted increase in risks. An increase in risks does not necessarily mean that harm occurs more often or that greater harm occurs than previously. 

A reduction in the legally regulated requirements during certain phases of the integration is worth discussing because there was no opportunity when the refugee crisis first started to weigh up the possibilities and risks associated with the legal framework of the start of the integration process. Any solution we find could be of international significance because other countries will have to resolve similar problems related to integrating refugees to those we are facing. The citizens of Germany must first feel that we are moving towards the goal of ‘we will cope’.

Suggestions for Health Care Provision

Decisions made with regard to health care provision are just as dependent on value judgements as those decisions made about managing the refugee crisis. Value judgements are not reflected in any objective parameters. They represent the subjective, individually varying perception of qualities such as safety and freedom. Accordingly, our decisions are not guided by objectively measurable conditions, e.g. risks and any limitations/restrictions, but are rather dependent on each individual’s perception of these objective conditions, e.g. ‘perceived safety’ and ‘perceived freedom’.

This article demonstrates that the two factors of safety and freedom compete with one another and that a trade-off between the two is necessary. A decision must be made as to how much of a factor can be sacrificed to enable another factor to be preserved instead. 

This issue of competing factors has not been considered in the social arena or in the health care sector to date. Discussing the concept of competing factors could lead to novel suggestions and paths for finding solutions for problems which have thus far proven difficult to resolve.

Shots in the Olympia Shopping Centre Munich: The risk cycle.

Franz Porzsolt

Shortly before 6 pm on 22 July 2016, shots were heard in the Olympia shopping centre in Munich. Nine bystanders were killed and 23 were injured. These were the violent actions of an 18-year-old. He was found dead two hours later one kilometre from the scene of the crime as the tenth victim. Social networks immediately started spreading what was initially unreliable and unclear information, followed by ongoing reports in the media in which the few facts and a great deal of speculation were very difficult to tell apart.

In a very short time, an impressive contingent of police cars, fire trucks and ambulances arrived, making itself clearly seen and heard, streets were blocked off, traffic ground to a halt, public transport was stopped, the main railway station in Munich was evacuated, special units made their way towards Munich and individuals and hotels offered free accommodation to those affected by the lockdown. In Munich and Berlin, crisis meetings were called over the weekend. Offers of help came from around the world. Threats were to be eliminated, people were to be protected and lives saved. Everyone was frightened.

Fear was the main feeling. It was like a blitz attack – triggered not by shots but rather by information. A massive quantity of information was thrown at a sensitised society. It had been sensitised by the recent killings in Paris, Brussels and Nice and the pseudo-terrorist attack in Würzburg. The only calm influence on 22 July in Munich was the police spokesman who instilled confidence and radiated calm. He certainly gave out less information than most reporters.

A year ago, the Hanns Seidel Foundation (HSS) issued a call for more discussion about domestic security as a result of the increase in burglaries. The shooting rampage in the Olympia shopping centre is considerably more violent than burglary. Nevertheless, no-one can predict what will happen in the weeks ahead. Less terror, overreactions, a society inured to such events, panic? Following the HSS debate, there were discussions about which ideas regarding risks and safety should be taken up and further developed.

We should ask ourselves whether more bad than good came out of the incredible density of information produced by the massive contingent of police and emergency personnel, the collapse of road and rail traffic systems and the reports on the radio and television. Social media played a critical role, appearing almost uncontrollable. Is it really though? Most activities were done in the conviction of doing something essential in a tense situation. What damage would have resulted if the shots in the Olympia shopping centre had only been heard by a few people and the city of a million people had only learnt of the rampage several hours later? What would there have been to discuss if the incident at the Olympia shopping centre had actually been a terrorist attack and the perpetrator(s) had been able to reach other parts of the city?

The initial uncertainty was clarified a few hours after the violent actions and the existing risks properly assessed. It is easy to talk after the event but the details can also be analysed in hindsight. The relationship between the current existing and objective risk, its perception, the resultant reaction and feedback on the change to existing risks can be illustrated with a risk cycle.

This cycle is based on the assumption that in reality no-one makes decision based on objective risks. We all make decisions based on our subjective perception of objective risks. This statement in the risk cycle, that our decisions depend not on objective criteria but rather on subjective perceptions, may raise eyebrows among scientists because it is scientists in particular who believe that they make decisions based on objective criteria. This subjective perception of objective risks – we call it ‘perceived safety’ – is influenced by two different types of factors, on the one hand the largely constant factors such as personality traits and on the other the highly variable factors such as the available information.

However, the risk cycle also includes another significant element, which is the influence of subjectively driven behaviour on the modulation of the objective risk. In other words, our actions, which are driven by perceived safety, affect objective risks, which ultimately means no less than our decisions and thus the risk we are exposed to being extremely easily influenced by, for example, personality traits and information.

It is not the function of this brief history to clarify the details of the risk cycle. But this much must be emphasised: every single event that we encounter requires a careful and comprehensive analysis. We have already learned that decisions in situations of maximum uncertainty are associated with a considerable risk of errors. In a period marked by maximum uncertainty, acting on a principle of ‘perceived safety’ is the top priority until sufficient information is available for decisions that are largely rational. ‘Perceived safety’ cannot, however, be equated with a police presence, flashing lights and wailing sirens. The behaviour of the police spokesman came very close to this goal using the simplest means.

In conclusion, it should also be noted that the principle of perceived safety and the central importance of information were derived from medicine and now return to medicine via domestic politics: As patients, we all want to feel safe in the doctor’s surgery and in the hospital. Perhaps management in some surgeries and clinics can be improved with this concept. Our blog aims to encourage critical reflection.

The Pragmatism Triad in Clinical Research

I have noticed a recent growth in the frequency of the term “pragmatic” in medical literature. A review dedicated to this kind of study was recently published in the New England Journal of Medicine. Still, there is an evident heterogeneity in literature in what concerns the definition and the purpose of this study design. As a result, we recommend to use identical words to describe identical contents and meanings of these words to avoid confusion.  

In a recent review executed by our research team on the subjects “evidences about evidences”, we have realized that publications discuss pragmatic studies but disagree on definitions and fail to point out important details about the distinction of pragmatic from other studies.

This post is meant to discuss in a didactic way the meaning of the word “pragmatic” in scientific studies, classifying them in three different contexts: the pragmatic message of efficacy, the pragmatic clinical trial and the pragmatic study of effectiveness. You will realize along this text that I have emphasized the importance of the preference in the choice of the treatment as a component of effectiveness.

PRAGMATIC MESSAGE OF EFFICACY

This situation refers to a traditional clinical trial, in which the allocation to the type of medical conduct is randomized. In this case, we suggest to use the term “pragmatic message” when the study is limited to guiding what the best conduct is, but its scientific meaning does not prove a mechanical concept. We use this rationality to analyze the SPRINT trial, which brings the pragmatic message that we should chase a more intense control of blood pressure. However, the standard deviation of the blood pressure values reached in this group was wide, meaning that a big portion of the patients randomized to the intensive treatment (BP < 120/80 mmHg) had higher BP levels. That said, the study could not prove the idea that today’s normal BP levels can actually cause vascular injury and predispose to adverse events. In this case, we call the message “pragmatic”, because this is not a study that can proof the concept.

On the other hand, when we analyze the IMPROVE-IT study, we understand that it was meant to prove that cholesterol is a risk factor for coronary artery disease. Some were still in doubt if the benefit of the statin treatment came from the reduction of cholesterol itself or from direct (pleiotropic) effect of this drug class. By demonstrating events reduction with another type of drug (ezetimibe), this study reaffirms the concept that cholesterol is a risk factor. By applying a causality criterion called “reversibility”, this study proves the concept that cholesterol increases cardiovascular risk, because reducing it caused the risk to be reversed. Even though, this study does not have a pragmatic message of telling us to regularly use ezetimibe associated to statins, since risk reduction was small, with a high number needed to treat (NNT).

There is a good hypothetical example about the role of exercising in weight loss. In clinical trials, the diet pattern is identical between the exercise and the no exercise groups, so it evaluates the direct effect of the exercise independently from diet. This direct effect means a mechanical proof of concept. It is explanatory. However, if patients were randomized to exercise and no exercise, while allowing a free diet, maybe those exercising would be motivated to eat better and would lose more weight. Then there would be a pragmatic message that exercise leads to weight loss, but not a mechanical message. The pragmatic message does not explain the reasons for weight reduction but confirms the reduction of the weight (which is indeed an important information).

In these cases, the effect is demonstrated in the controlled world of the randomized clinical trials, where there is no preference in the choice of conduct (randomly defined). For this reason, the message refers to efficacy (ideal study conditions) but not effectiveness (real world conditions).

EFFICACY PRAGMATIC CLINICAL TRIAL

This one refers to the implementation of the conduct. Imagine we want to demonstrate the efficacy of physical therapy on a musculoskeletal condition. The patients are randomized to have physical therapy or not. However, the exact way the therapy will be done (type of exercise, number of sessions) is up to the therapist. The question is whether we should or not indicate physical therapy, but in this case, the therapist’s freedom makes it close to the way it is done in real world. Even though, this is still an efficacy study (not effectiveness), because the professional’s and the patient’s choice are not considered in the decision of having the therapy. It is randomized.

Some other studies randomize patients to be or not subjected to a screening for a specific disease, and then, leave it up to the doctor to decide what to do with the result. The study question refers to the real effect of the screening, so that is why the doctor is free to adopt a conduct. This is where the pragmatism is. Still, this is also not effectiveness. This is efficacy, because the decision of having a screening is artificially made (randomized).

Notice that, in these studies, conduct is randomized but it’s implementation is not necessarily defined, granting the professional a certain freedom to define the way he will do it.

A few other criteria bring some pragmatism to randomized clinical trials, like a wide selection of patients, the non-use of placebo when analyzing the total effect of a treatment and other questions. Although, I believe the most important thing to define pragmatism is the implementation of treatment.

PRAGMATIC EFFECTIVENESS STUDY

As we know, effectiveness describes effects in real world. Efficacy answers the question “can this treatment work?” This is tested in the ideal world, in the clinical trials lab, and it shows that the conduct has an effect. Effectiveness refers to a question that must be made after the valid and controlled demonstration of efficacy: “does this treatment work in real world?”

Effectiveness has two components that make it different from efficacy. The first one refers to a wider variability in types of patients and quality of medical conduct, such as smaller adhesion, less experienced surgeons and other real world characteristics. This first component is constantly remembered, but there is a second component, just as important, frequently forgotten in literature: the doctor-patient preference in the choice of treatment.

In this context, preference means a mental choice, treatment individualization. A choice that is oriented by concepts of efficacy, but that still requires clinical judgement to decide if that patient should indeed receive this treatment. What is the desired outcome? What are the risks for adverse events?

Class I conduct recommendations should be done almost universally, like prescribing antibiotics for a patient with pneumonia. This kind of treatment is not based on one doctor’s or patient’s preference but rather on high plausibility and general acceptance. It should be more like a rule. In this context, it is possible that efficacy (ideal world) is superior to effectiveness (real world).

However, there are class II recommendations, when we should measure the risk/benefit ratio. This is the situation related to the use of anticoagulants in atrial fibrillation, for instance. We should consider the benefit in CVA prevention versus the harm of the bleeding risk. This is also the situation related to the indication of effectual surgeries, in which the risk of complications in a certain group of patients might overcome the benefit. In these cases, an effectiveness study brings additional information because it evaluates if the doctor’s choice, case by case, improves the result of the treatment in real world. Therefore, preference should be an important aspect of effectiveness studies.

In these cases that we have weigh, a good doctor individually deciding might cause a better outcome than the random choice of a treatment.

The patient’s choice might also influence in effectiveness. For example, imagine that praying improves the quality of life of cancer patients. If praying is a patient’s choice, this “treatment” will work better than in a randomized clinical trial in which praying was defined by a raffle instead of preference. 

Imagine we are evaluating the benefit of physical exercise in improving the functional capacity of cancer patients. If exercising is a desire of a patient who likes to do it, it might work better than when exercising is randomly decided. A patient who prefers exercise might execute it better and commit more than a patient who does not, but was randomized to do so. Again, effectiveness (real world) tends to be superior to efficacy (ideal world).

It is clear, then, that a real effectiveness study cannot be randomized, because by doing so, we set aside the choice of treatment by a mental process. I say this because some authors, mistakenly (in my opinion), use the term “randomized effectiveness clinical trial”, which is a contradiction. 

Once we have defined what effectiveness really is, let us describe how a pragmatic study of effectiveness is done. As I said, this should not be a randomized study, but an observational one. It is supposed to compare patients who have the treatment, versus patients who do not. 

There will be confounders, obviously, because there was not randomization and both groups are different, so there should be an adjustment. However, in the actual effectiveness study, the adjustment should focus on the patient’s risk of having the outcome, and not on his tendency of getting the treatment.

This is different from what is normally done in cohort studies that do not evaluate the impact of treatments, like hypothesis generators. After an experimental clinical trial has demonstrated efficacy, a pragmatic study should be made to demonstrate effectiveness. Also, since effectiveness depends on preference, the adjustment should be made to the variables associated to the risk of the outcome, and not to a propensity score to the treatment.

Finally, the chronological sequence of the hypothesis tests should be:

1 – Observational study that generates the hypothesis of efficacy (adjusted to the propensity of getting the treatment). This first study is an observational study. The calculation of a propensity score is necessary to reduce bias.

2 – Randomized clinical trial that proves efficacy. This is an experimental study.

3 – Pragmatic study that proves effectiveness.  The baseline risks of any included patient have to be adjusted to the endpoint assessed. When different endpoints e.g. survival and allergic reactions will be assessed a particular patient may be considered high risk for the endpoint “survival” but low risk for the endpoint “allergic reaction”   

There are two types of observational studies that evaluate treatment: a first one that generates the hypothesis that the treatment is efficacious (demonstrates efficacy), and a second one that confirms it is effective (demonstrates effectiveness). We may use this or another terminology but we should avoid to mix up the concepts. 

These methods of pragmatic effectiveness studies is formally described by Franz Porzsolt, in the journal Pragmatic and Observational Research (2015; 6: 47-54).

In fact, any kind of study might have a pragmatic value, but pragmatic is not a synonym for effectiveness. There are some pragmatisms related to efficacy, and there are some related to effectiveness. We should know how to differentiate the use of the word “pragmatic” in these situations.

* This post was written by Franz Porzsolt and Luis Correia.