Methodology for the Evaluation and Measurement of Therapeutic Progress

Abstract

When a candidate drug is likely to become available to prescribers and healthcare policy makers, evaluation of therapeutic progress moves forward in two stages. First, the level of expected therapeutic progress must be established. This first stage requires the determination of therapeutic needs and the comparison of these against the results of the clinical studies that will form the basis of the marketing authorisation of the drug. This determination helps anticipate the therapeutic progress that is attributable to the approved use of the new drug. The second stage of the process, the evaluation of the actual therapeutic progress, involves therapeutic drug monitoring and bases itself on observation. Since such observational data are intended to challenge the initial hypotheses and uncertainties (in terms of benefits and risks), goals and methods must be laid out before the drug becomes available to the general public. Keywords: therapeutic progress, epidemiology, evaluation When penicillin G was introduced as a therapeutic drug for human use, the therapeutic progress was so clear that no one envisaged measuring it objectively. Moreover, this question apparently sparked no interest in either physicians or scientists at that time, because of the major innovation of the antibiotic and the tangible medical progress it represented. Clinical studies were sufficient for observing that its administration cured certain infections that, until that time, systematically killed the afflicted. Although the question of therapeutic progress went unaddressed, as important as it seemed, it was, nonetheless, important to demonstrate the efficacy of the drug. In addition, the implementation of an experimental protocol defining a random controlled study was not required for this molecule to receive marketing authorisation. PHARMACOLOGIE CLINIQUESince then, the context has changed. Today, no one would consider introducing a new therapeutic molecule onto the market without data on its efficacy, based on the results of clinical trials. In the same way, anticipating the therapeutic gain of a candidate drug and measuring real therapeutic progress, obtained through its use, have become indispensable.