An update on the first decade of the European centralized procedure: how many innovative drugs?

Abstract

What is already known about this subject We recently proposed an algorithm to assess the degree of therapeutic innovation of new therapeutic agents. It was based on the disease seriousness, the availability of previous treatments and the extent of the therapeutic effect, and was applied to all therapeutic agents approved by the EMEA in the period 1995–2003. A low percentage (32%) of important therapeutic innovation was found. This figure may be an underestimate of the actual level of innovation, because common biotechnological products, such as recombinant human insulins, must follow the centralized procedure. What this study adds Details for each agent, focusing on the comparison of the degree of therapeutic innovation between biotechnological and nonbiotechnological therapeutic agents approved by EMEA during the its first decade of activity (1995–2004). The underlying hypothesis was that the latter have a higher degree of innovation because they followed the centralized procedure on the assumption that they are innovative. The percentage of important therapeutic innovation was low not only for biotechnological products (25%), as expected because they include many already known products such as insulins, but also for nonbiotechnological therapeutic agents (29%). Aims In a previous paper, we proposed an algorithm to assess the degree of therapeutic innovation of the agents approved by the European centralized procedure, which must be followed by biotechnological products and is optional for drugs claimed as innovative. A low overall degree of therapeutic innovation (about 30%) was found. This figure may be an underestimate of the actual level of innovation, because common biotechnological products, such as recombinant human insulins, must follow this procedure. To test the hypothesis that therapeutic innovation prevails among nonbiotechnological products, we evaluated separately the degree of therapeutic innovation of biotechnological vs. nonbiotechnological agents in the first decade of European Medicines Agency activity, also studying a possible time trend. Methods We assessed, for each drug: (i) the seriousness of the target disease, (ii) the availability of previous treatments, and (iii) the extent of therapeutic effect according to the previously proposed algorithm. Results Our analysis considered 251 medicinal products corresponding to 198 active substances, classified according to four main areas as therapeutic agents (88.9%), diagnostics (5.5%), vaccines (5.1%) and life‐style drugs (0.5%). Among all therapeutic agents, 49 out of 176 agents (28%) were classified as having an important degree of therapeutic innovation. Fifteen out of 60 biotechnological therapeutic agents were considered important therapeutic innovations (25%), whereas this figure was 29% for nonbiotechnological agents. Conclusions Among active substances claimed as innovative by the manufacturers, only a minority deserve this definition according to our algorithm.

EMA’s reflection on placebo does not reflect patients’ interests

“…There should be no uncertainty, however, if the current use of the active comparator is based on solid evidence (i.e., a systematic review of randomized clinical trials with adequate design, sample size, comparators, outcome measures, length of follow-up, etc.). Unfortunately, this is not always the case with newly developed medicines, which often reach the market without satisfactory demonstration of their efficacy and safety [5][6][7][8]. Then two possible scenarios follow.…”

The impact of innovation for biotech drugs: an Italian analysis of products licensed in Europe between 2004 and 2011

“…An analysis of biotech drugs approved by the European Medicines Agency between 2004–2011 was performed by using the algorithm proposed by Motola et al 19 20…”

“…In order to perform this analysis, we applied the algorithm developed by Motola et al 20 and approved by the Technical-Scientific Committee of the Italian Agency for Drugs (AIFA) in 2007. This algorithm is intended to assess the degree of therapeutic innovation of drugs approved through the European centralised procedure.…”

The burden of rheumatoid arthritis and access to treatment: determinants of access

“…A substantial number of NCEs are introduced each year, although not all of these represent important innovation. Motola and colleagues [6], for example, estimated that only 32% of all new substances provided an important therapeutic innovation. Many of the new biological treatments in RA introduced during the past decade, however, belong to this minority of truly innovative drugs [6].…”