Therapeutic repositioning: importance of new therapeutic indications approved for old medicines

Abstract

In the field of health and, particularly in biomedicine, continuous reevaluation has allowed us to see with a different attitude what was apparently known; A large part of the medicines from synthetic chemistry, biotechnology and advanced therapies (genetics, somatics, tissue engineering, etc.) are a clear example of this. The search and investigation of new applications for drugs already approved and in clinical use – and even for compounds that did not reach the clinical research phases at the time – is usually called “repositioning”, which makes it possible to have drugs with a profile of safety and efficacy already known, which represents a significant saving in time and development costs and, sometimes, is the only way to develop therapies for rare diseases, as orphan indications. In fact, it is estimated that a substantial part of the known drugs may have new therapeutic uses and that drugs currently in clinical use could be used for numerous applications other than those for which they were originally approved. The high number of authorizations of new indications or their extensions or modifications by the EMA and the FDA, in relation to that of medicines with new active ingredients, highlights that pharmacological research – basic and clinical – does not end with the marketing authorization of a medicine but, on the contrary, there are many that continue to be the subject of extensive and intensive research by their titular laboratories in order to extract the maximum knowledge and health results from products whose development has a very high economic cost and requires large and multidisciplinary human research teams. In order to examine the current innovative activity in the field of new indications for medicines previously authorized in the European Union, we have proceeded to systematically collect and study the contents of all the summaries of the 36 plenary meetings of the Committee for Medicinal Products for Human Use (CHMP) published by the EMA between January 2021 and March 2024, in which 258 positive recommendations for extension of indications were made, for a total of 181 medicines from 74 laboratories. Keywords: Pharmacology; therapeutic repositioning; clinical research; drug authorization; drug evaluation