Science

Dare more sensible medicine

What about medicine? Not good. I say that as a medical doctor. Doctors, nurses and therapy successes in Germany are better than their reputation. Pharmaceutical companies are also more interested in medical advances and less in corruption of doctors than is often assumed. But still modern medicine hits a wall.

Because if it considers fulfilling its rules more important than serving the patient, then it has lost sight of its goal.

Then it lost its meaning.

Modern medicine, this is above all evidence-based medicine (EbM). Its principle is that it is not the “demigod in white” – the Eminence – who can prescribe a therapy at will, but that facts, studies, hard data – the evidence – must be the basis for therapy.

Interests, but not those of the patient

What should be wrong with that? Nothing, as long as the facts are really hard and universally valid, at least. But that is anything but self-evident. And in many places, the EbM has become an IbM, an interest-based medicine. And that's a big problem. Because it is usually not about the interests of the patient.

An example should clarify this.

2017 a therapy study was published in the renowned journal “New England Journal of Medicine”. The question was whether a cholesterol-lowering drug would also reduce cardiovascular diseases. About 13 000 Patients were examined in each group. One got the drug, the other – called the control group – didn't. 11, 3 percent of the patients in the control group got sick. It was only 9.8 percent of those who had received the preparation. The experts were thrilled. You can reduce the “relative” risk by about 15 percent, it said.

But 11, 3 minus 9.8 percent results in only 1.5 percent. In fact, and not that 15 percent, the real difference is the “absolute risk reduction”. So the result is actually: The likelihood that the remedy, which can also have side effects, will help a patient is 1.5 percent. So it's very, very small.

Give and take

I don't care what happens when 13 000 people take a medication. I am interested in the person sitting in front of me. My patient. In the study everything was okay with regard to the EbM rules. No progress was made for my patient. That would be possible. For example, if the company were to be told the following: It was an interesting step forward, but not enough for the drug to be approved. Because it statistically helps only 1.3 of 100 patients. So you should still find out how to identify the patients who benefit from the preparation.

Peter Paul Nawroth ist Ärztlicher Direktor der Klinik für Endokrinologie, Stoffwechsel und Klinische Chemie des Uniklinikums Heidelberg. Sein Buch „Gebt der Medizin ihren Sinn zurück“ ist im Springer Verlag erschienen und kostet 19,99 Euro.

Peter Paul Nawroth is Medical Director of the Clinic for Endocrinology, Metabolism and Clinical Chemistry at the University Hospital … Photo: Heidelberg University Hospital

You also need to clarify whether the disease will only be avoided for a short time or for a really relevant period, because your study says nothing about it. And if the effects were greater for Asians than for Europeans, you have to publish that too. And you have to disclose that you have lumped together many illnesses because only one of them would not have resulted in a significant result.

But one must not only demand, but also say: with it you can explore all of this in peace, your patent protection will be extended. After all, if you really want to go from interest-based to meaning-oriented medicine, you have to set incentives that promote good clinical studies. Current patent law is damaging. A reform would take scientific tasks such as those just listed into account and offer opportunities to extend patent terms. That would be for the benefit of the sick and the manufacturers, who without such grace periods are of little interest to conduct lengthy, expensive studies, which may then also reduce the potential patient group.

Values, laboratory values, ratings

All of this is of course a bit more complex. But one thing is clear: Up to now, there has often been a lack of a decisive perspective when evaluating such studies: that of the individual patient.

According to this study, a benefit was unlikely for him or her. And even overall, averaged over thousands, it was only minor. And there is also a fine line between such little benefit and harmful therapy. You can see that in my subject, diabetology.

When you hear “diabetes”, you first think of blood sugar and insulin. When I treat a diabetic, I first think of: blindness, kidney failure, leg amputations, heart attack, increased risk of cancer, stroke and death. It is the so-called late diabetic damage.

But did you know that with the exception of the risk of a diabetic coma, doctors can only reduce the absolute risk of all these consequential damages by controlling sugar by a single-digit percentage? That is why doctors who have read and understood the studies themselves only adjust the blood sugar in a medium range. And from experience. Experienced, well-informed doctors do not try to lower blood sugar to the level of non-diabetics.

However, the belief in the laboratory value of “sugar” is so great that, although there are indications that Diabetics who are older than 75 years get even fewer heart attacks and strokes than non-diabetics, yet studies have been done to deal with it of all powers and many medications to “normalize” the blood sugar value as far as possible.

Successfully reduced blood sugar, patient dead

The result: No protection, but increased deaths.

Who takes responsibility here? Unfortunately, there are many other examples that speak of evidence-based medicine, but which do not focus on patient well-being. These include: New, ever lower limits for high blood pressure without actual evidence that the drug treatment required is more useful than harmful. Or the orientation towards “metastasis-free survival”. The latter sounds good, but in recent years we have had to learn that it has little to do with quality of life and life expectancy. The same applies to weight loss and “healthy” eating.

The EbM currently practiced offers too many loopholes for interests that are not oriented towards patient welfare.

What to do? It would help to sort out some of the most common scientific errors in medicine. This includes the confusion of correlation – i.e. simple connection – and causality – i.e. real causality, but also excessive belief in limit and threshold values, underestimating the risk of overtreatment and the inability to read study data through the patient's glasses and not the system , And the pressure to report “positive” harms the critical evaluation of skills and benefits. Because knowing and saying what we don't know and can't do is just as important.

What is the use?

What we ask ourselves must: do the structures of evidence-based medicine favor errors like those mentioned above? I think so. Because they put the proof of the effectiveness of a treatment in the center of the scientific work, but not the purpose. If you choose a study group large enough, a marginal effect can be sold statistically as “significant”, ie as an effectiveness, even if it is irrelevant for the vast majority of patients.

Or: If a new cancer drug extends the metastasis-free survival a little or the overall survival by five months, but this is accompanied by pain in the hands and feet, it becomes clear that “meaning” continues to be more significant than in the Statistics. Only the benefit of therapy for the patient who comes to me with his hopes and fears, his concerned family, his plans and his experiences can ultimately be my criterion for action.

The most important question is: what is the patient's point of view?

Studies could also help here. They would have to be interdisciplinary and examine the effects of individual, specific medical interventions over long periods of time. A big problem here is that government institutions have almost completely withdrawn from the funding of large therapy studies with many test subjects and leave them entirely to the pharmaceutical industry.

From the point of view of my patient, it would make sense if all the scientific publications already made a summary of the facts that were important to him – the absolute risk and the expected amount of time for the onset of the disease to begin – should be mentioned as well as the characteristics of the patients who use the preparation and those who do not.

The slightly different courage to fill the gap

If this were to be made compulsory, the biggest step would have been taken. If the professional societies were then forced not to use studies with proven low informative value and a high error rate for decision making for their therapy guidelines, this would be the second big step. Instead, one would have to focus more and more on currently developed tools to use the experience and intuition of nursing staff and doctors.

Since it does not make sense that guidelines contain reliable statements about the effectiveness of therapies in them but there is hardly any talk of the equally secured knowledge gaps and open questions, the disclosure of knowledge gaps would not only be important for research, but as a third step necessary for a sense-oriented medicine.

The system is disturbed. For example, a study was recently published in high-pressure patients in Africa, the data of which only allow conclusions to be drawn as to whether a medication lowers blood pressure, but not whether it prevents infarction or stroke. It had the recognizable goal of marketing more expensive high-pressure preparations there. It would be different if the companies had invested the money to investigate which high-pressure therapy can be used to prevent heart attacks and strokes – specifically and for which patients.

This is how steps from an evidence-based to a sense-oriented medicine could look like.

Medical science urgently needs to rethink. From the planning of a study to its publication, medical researchers must consistently take the patient's perspective. You have to select the examined parameters accordingly. We, the scientists, have the task of providing the doctor and patient with concrete information that can be used to make smart, meaningful decisions individually. The way we now operate evidence-based medicine has led us deep into a dead end. However, we can use your good, sensible sides to develop them into meaning-oriented medicine.

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