THE SCOPE AND RELEVANCE OF CLINICAL RESEARCH

For all of us who work in science, it is a basic tenet that scientific and technological advances generate progress in our society. We have learned in our day-to-day lives that bringing this sense of science to the general population is our duty. We have clearly realized, over the last few decades, more through errors than by lack of development, that this explanation and popularization of science, which support clinical research, is an obligation. People do not generally read scientific publications for pleasure. Today only those who are working on a given subject have the capacity to understand concretely what is being expressed in scientific articles. They are not easy reading, often filled with scientific nomenclature or even complex formulas, incomprehensible to those who are not in the field.

One of the best definitions of science I know defines it as the generation of knowledge, but the knowledge obtained does not necessarily provide a benefit to society. Especially in health issues, there are numerous examples of studies that replicate previous findings, repeat things already known, or lack methodological support. Their results are simply not valid.

I believe that in this way clinical research can be methodically formulated in order to generate knowledge that can be applied as a benefit to our society. Clearly, it will not happen overnight, but it is a basic principle that the subjects participating in the research have a potential benefit and that society can benefit from that knowledge generated.

Throughout our history, and especially over the last two hundred years (to set a rather arbitrary date), society has seen the greatest scientific findings in the field of health care. Van Leevwenhoeck and Spallanzani began the era of microbiology, putting a number of “living beings” in the dock as responsible for numerous diseases. Koch, with his postulates, classified matter as living or non-living (within the scope of his time). In the late 1800s and early 1900s, Paul Ehrlich formulated perhaps the best definition of the target molecules in pharmacological therapeutics, identifying “the magic bullet” as the one that would exclusively destroy microbes and thus developing, after having evaluated hundreds of active principles, the first arsenicbased compound to treat syphilis, the now famous compound 606, the p,p’-Dihydroxydiaminoarsenobenzene.

Without detailing a long list of biomedical advances in recent years, it might be noted that, thanks to clean drinking water and proper sewage and waste treatment, along with the development of drugs and vaccines, life expectancy has increased nowadays to between sixty and seventy-five years in Latin America. (In global terms, there is an enormous disparity in this value, depending on the country under consideration, and ranging from thirty years in sub-Saharan Africa to over ninety years in the Principality of Monaco). What is certain, however, is that life expectancy at the end of the 1800s was no more than thirty-five years and had been stable since the Middle Ages. Although scientific health research has improved both life expectancy and quality of life, not everything has been necessarily positive. Research means evaluating something of which we do not know the result, and although this may seem obvious, the results can be good (ratifying the hypothesis raised) or bad (rejecting it). In fact, if we knew what the outcome of pharmacological research would be, it would simply be unethical to carry it out.

In other words, clinical research in particular generates risks. I am not referring specifically to therapeutic failures for a particular subject, since this can also occur in normal medical practice, but rather, for example, studies that generate negative results, those that fail to detect adverse effects with a very low incidence, and methodological or recording errors.

LIFE EXPECTANCY

Basically, there are many more potential therapeutic molecules that do not continue to be developed than those that reach the commercial market. This is due either to lack of clinical efficacy (and the consequent risk of treating a patient population with a treatment that is not effective, or less effective than its comparator) or to safety problems (the participating subjects received a treatment that caused them an adverse effect). In both cases, these are the risks associated with performing science.

Over the last few decades there have been some examples of safety problems, one of the best known being the increased cardiovascular risk (myocardial infarction and cardiovascular accident) due to the use of celecoxib (in view of the imbalance of endothelium-platelet functions). Scientific research should be carried out in accord with a strict risk/benefit assessment for each protocol and the precept of doing no harm should always prevail. But I would like to close this small article by mentioning just two of the countless positive results of clinical research. According to the World Health Organization, it is estimated that vaccines currently save the lives of some three million people a year and that tens of millions were saved by the discovery of antimalarial drugs. It is up to all of us to continue this legacy and to generate more scientific knowledge, in order to improve the quality of life for generations to come.

Federico Lerner

Operations Director at LatinaBA, a regional CRO; and President of CAIC, Argentine Congress of Clinical Research. Dr. Lerner was responsible for leading many different clinical research teams for the first time to conduct large global registry trials, he was a former Latin American chief of four major global CROs.