According to a study published in the journal Nature, for the ten best-selling medicines in the United States, the proportion of patients responding positively to treatment is frighteningly low. Despite their great popularity, some drugs help only four percent of people taking them. Nicholas Schork, Director of the Department of Human Biology at the J. P. Morgan Institute. Craig Venter (USA), notes that statins, commonly used to lower cholesterol, benefit only one in 50 patients. Or, for example, Abilifay, which is popular in the treatment of schizophrenia, causes a clinical effect in only one out of five patients. This means that the remaining 75% of people remain without proper treatment. However, the sales volume of such drugs per year exceeds 10 billion US dollars.

A diagram showing the number of patients responding positively to treatment with the ten most popular modern drugs, according to a 2014 study. Patients responding positively to therapy are indicated in blue; patients who do not respond to treatment are indicated in red.

Translation (from left to right): The Medicine of inaccuracies For every person (blue) who is helped by one of the ten most profitable medicines in the United States, 3-24 people (red) do not improve.

1. Abilifay (aripiprazole), schizophrenia. 2. Nexium (esomeprazole), heartburn. 3. Humira (adalimumab), arthritis. 4. Crestor (rosuvastatin), high cholesterol. 5. Simbalta (duloxetine), depression. 6. Advair discus (fluticasone propionate), bronchial asthma. 7. “Enbrel” (etanercept), psoriasis. 8. “Remicade” (infliximab), Crohn’s disease. 9. Copaxone (glatiramer acetate), multiple sclerosis. 10. Neulasta (pegfilgrastim), neutropenia. Source: N. Schork, Personalized Medicine: Time for One-Person Trials. Nature (2015): 520 (7549), 609–611.

The current approach to conducting clinical trials and drug development is ineffective at best. Insufficient data is being collected on factors such as genetics, lifestyle, diet, ecology, etc. The test results often indicate the need for additional research to establish the relationship between the individual characteristics of patients and the degree of response to therapy. For example, in the course of clarifying studies, it was found that the drug Glivek (imatinib) is effective mainly for leukemia patients with a chromosomal abnormality called Philadelphia translocation in tumors. Similarly, it turned out that Erbitux (cetuximab) prolongs the life of people with colorectal cancer, whose tumor cells carry an EGFR gene mutation, but not a KRAS gene mutation. However, according to Nicholas Schork, trials tailored to the individual characteristics of patients are not yet popular due to the high cost, although they can save considerable material resources and prolong the lives of modern people on a national scale. According to the scientist, classical trials can only benefit future generations, while contemporaries spend a lot of money on buying “blockbuster drugs”, bringing huge benefits to pharmaceutical companies.