«You say you want a revolution? Well, you know, we all want to change the world, »sang The Beatles. So-called personalized medicine – also called precision medicine – is one of the medical fields that is associated with many promises of a health revolution. It is about treatment that should be tailored to individuals or small groups and the biological characteristics of their diseases, especially their DNA. A recent example of such promises comes from Ingrid Stenstadvold Ross and Sigrid Bratlie of the Norwegian Cancer Society – a powerful actor who has his interests. In an article in news Ytring, they write in particular about a revolution in gene therapy where genes are replaced or have their expression changed in either cancer cells or other cells in the body. New techniques such as the “DNA scissors” CRISPR reinforce the promises. Like most revolutionaries, Ross and Bratlie believe that the current regime is outdated and unfair, and they demand “new and groundbreaking policies.” According to Ross and Bratlie, patients are not allowed to take a proper part in the revolutionary (and extremely expensive) gene therapies. The problem they point to in precision medicine is about knowledge and what we consider to be “documented” useful, safe and cost-effective. Since there are often so few in the groups who will try each treatment, perhaps only one – it is difficult to carry out the type of large, randomized, controlled studies on which the current knowledge system is based. In particular, it is uncertain how long the effect of the medication lasts, and what side effects there are in the long run. This takes a long time to study. Gene therapies are often given as a single, hopefully curative, but expensive dose. The solution Ross and Bratlie propose is a system in which expensive medicines are used and approved despite greater uncertainty. We think they are addressing something important, but at the same time believe they are taking for granted what is actually to be proven, namely a forthcoming revolution in health. There will be something self-affirming about it all. If it has already been read and decided that it is a revolution, what is to be done with documentation? Important questions are: Is there a revolution? In what way? For whom? We agree that gene therapies are impressive. There is no doubt that individuals get their lives saved and markedly improved. For those who work in the fields where they are used, it must be very gratifying. This is especially true of cancer medicine and the “rare field” which deals with rare, genetic diseases. But is there a revolution now for common, complex public diseases? No. Does it mean a revolution for public health? Hardly. It is undocumented. We agree that we must see if we can develop new, professionally sound and verifiable methods to determine what is considered “documented”. The problem is that this is not easy, and in some cases may not be possible. We recently published an article in the Journal of Evaluation of Clinical Practice which deals with a much-discussed Norwegian case in which the Norwegian Board of Health cut through and considered it to be documented that a cancer patient had had the effect of a treatment. They thought so despite the fact that there were no heavy, disease-specific studies on this drug, and despite the fact that the hospital felt there was a lack of research. The Norwegian Board of Health instead emphasized what happened to the individual after the treatment. This illustrates a shift in knowledge views in the health care system where getting better after trying something undocumented can be considered documentation. But what the rules of the game to prove something should now be, is currently a bit in the blue. Our view is that we are not facing a health revolution in precision medicine that will cause us to lose our heads and throw all principles overboard. Personalized medicine is more about an evolution than a revolution. Firstly, because the health problems we face are, after all, complex and what may seem like a “quick fix” are not necessarily so. Secondly, because it will take time to document the effect and develop new methods for assessing new treatments. At the same time, the possibilities of precision medicine challenge all actors to exercise good judgment and doctors to good medical art. Ross and Bratlie believe that gene therapies should be offered to seriously ill patients without other good treatment options without method evaluation when the results from the first trials are interpreted as good. But then we must also bear in mind that we open up for a form of experimentation on vulnerable, critically ill patients who could spend their last time on something else and also have serious side effects. Ross and Bratlie want to change priorities in health care, and prioritize therapies despite higher uncertainty. They compare with the Petroleum Fund. For Norwegians who like the Oil Fund, this is captivating rhetoric, but the problem is again that we do not know how big the “return” of gene therapy will be. And there is a difference between gambling with money and gambling with life. In terms of priorities, there are also other groups where individuals can benefit greatly from various measures, even if it is difficult to document. Why should cancer patients get medicines for millions while others do not? Is it because gene therapy is spectacular high-prestige medicine with a heavy industry behind it, which can point to visible patient tragedies and has an influential association? The Beatles again: “You say you’ve got a real solution? Well, you know, we’d all like to see the plan ».
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The undocumented health revolution – Expression
