Not exactly personal medicine

Personalized medicine based on molecular technologies,
takes the first steps and acquires myths

Boris Mazo, "Business Petersburg", 17.05.2012

The ability to use molecular information, which appeared due to the analysis of the human genome, is not yet too widespread in practical medicine.

Most diseases are still treated with blockbuster drugs, the sale of each of which brings billions of dollars in revenue to pharmaceutical companies. The transition to personalized drugs designed to treat much smaller populations is impossible without changing the fundamental principles of the work of pharmaceutical companies. Are the owners of companies ready for this?

Vitaly Prutsky, Head of Information Support for R&D at AstraZeneca Russia, head of the Center for Bioinformatics and Prognostic Medicine in St. Petersburg, believes that in the near future changes in the work of pharmaceutical companies, including in the field of R&D, will allow combining both approaches.

Both the production and development of mass-produced drugs and drugs designed for smaller groups of patients will continue. "The economics of bringing the drug to market remains. This is a business, no one can afford to work at a loss if they want to be able to reinvest in the development of new drugs. But pharmaceutical companies began to pay attention not only to blockbuster drugs, but also to targeted ones. A personal medicine intended for a specific person is from the realm of myths. Personalized medicine focuses not on one patient or even hundreds of patients, but on subpopulations. They must have some kind of commonality. Personalized medicine will never be 100% effective. There is no method that predicts a specific medicine to a specific patient, – Vitaly Prutsky is convinced. – Many definitions of personalized medicine can easily be brought to the point of absurdity.

Let's imagine that we have a mechanism capable of individualizing medicine for each individual. There is a contradiction in this. We want this medicine to be safe in treatment, so that we have objective data based on the conducted clinical studies. We can't do an experiment on every patient. The idea is absurd not only economically, but also practically. We should be treated with what has been tested as safe and effective. Accordingly, it has been tested on someone else."

Personal medicine plays an increasingly important role in the development strategy of pharmaceutical companies focused on the production of innovative drugs and modern diagnostic tools. According to estimates by the Tufts University Medical Research Center, from 12 to 50% of current research by pharmaceutical companies falls on personal medicine. At the same time, oncology is still a priority. The main leaders are Abbott, AstraZeneca, Novartis, Merck, GlaxoSmithKline, Pfizer and Roche. […]
Alina Lavrentieva
Partner, Head of PwC Russia Practice for working with pharmaceutical companies 

Personalized medicine is currently being integrated into the existing healthcare system. The officially adopted procedure for the approval of medicines involves testing on a large heterogeneous group of patients, whereas the essence of the personalization of the drug is its use for the treatment of a specific, including from a genetic point of view, part of the population that will benefit the most from the treatment or will be at minimal risk of side effects.

"The problem of pharmaceutical companies is to find people who need the tested drug for research. Some medicines do not enter the market because they are not effective. They do not work for the entire population, but act on a specific subpopulation with high efficiency. I will give an example of the emergence of the drug Herceptin. It began to be developed in the mid-1980s, when it turned out that 25% of women with breast cancer have duplicated the HER2 gene in tumors, which determines a poor prognosis for the development of the disease. This is an example of a targeted drug that came out with the test. If the drug had been tested on the general population, it would never have appeared on the market. In order to demonstrate its statistical effectiveness, it would take 7000 patients and 182 months. Financially, no company can withstand such a test. The drug entered the market in 1998, it was tested for 2.5 years on 800 patients," says Vitaly Prutsky.

Approximately 1% of all medicines in the Western world can truly be attributed to personalized medicine. A prerequisite for existence is a proven testing mechanism for the need to use this particular drug by this particular patient. Most of these drugs are oncological.

"Diseases may have one etiology, but at the molecular level, individual groups of patients may have their own characteristics. Changes in the gene chain lead to a breakdown in a healthy body. There is a very large amount of information here, and we know some of it. And it turns out to find a target, the defeat of which normalizes the pathological process. This is what targeting therapy is all about. It begins with understanding what is broken in this particular cell. There are mechanisms of breakdown, there are mechanisms of repair.

Personalized medicine is a kind of triangle: to understand the disease (the molecular mechanism of the breakdown), to come up with a medicine that effectively corrects this particular breakdown, to understand the need for this medicine for this patient (the testing mechanism). Moreover, we will never achieve a 100% hit in the breaking mechanism. Even with one disease, there are several dozen mechanisms for breaking. It is desirable at the molecular level to understand the mechanism of breaking most cells and defeat them, the body will cope with the rest by itself," says Vitaly Prutsky.

The cost of the drug in the total cost of healthcare is not the main one. Significantly more is spent on hospitals, personnel, equipment. Often, even expensive medications can be cost-effective, since they allow you to reduce other expenses.

"Evidence-based medicine began to develop when methods of population statistics appeared. Biostatistics works well when we have a lot of patients and few parameters. Now imagine that we have 100 patients and for each 1.3 million parameters. Standard statistical approaches are not applicable here. But the problem can be solved with the development of biomarkers, if you make the right selection and apply the right analytical approaches. For real use, a test is needed to know which medicine is suitable. But the devil is in the details. There are few simple biomarkers, sometimes it is not clear why the marker works. Today it is necessary to prove that not only the medicine is effective, but also the test is effective. This means that the cost and duration of the clinical trial may increase by 2 times. When the cost of testing the drug is already $ 150 million, it is not a fact that the company will agree. And the researchers do not guarantee that a working medicine and test will turn out," explains Vitaly Prutsky.

The pharmacogenetic model has not yet been worked out, but now there is not a single pharmaceutical company that, at least in words, does not declare that any biomarker component of personalized medicine is not included in the development.

Portal "Eternal youth" http://vechnayamolodost.ru17.05.2012