Therapeutic innovation in the European Union: analysis of the drugs approved by the EMEA between 1995 and 2003

Abstract

Since January 1995, all European Union applications for marketing approval for medicinal products derived from biotechnology and other drugs considered potentially innovative follow the EMEA centralized procedure. In order to assess the overall degree of therapeutic innovation of these drugs, we considered, for each approved agent, its target, the availability of previous treatments and the extent of its therapeutic effect. The following scores for therapeutic innovation were assigned through a consensus process: 'A' (important), 'B' (moderate) and 'C' (modest). The overall degree of important/moderate therapeutic innovation was 47% of all therapeutic agents (32% important; 15% moderate). Most (80%) of the EMEA-approved therapeutic agents were for serious diseases. The remaining ones were for risk factors (7%) or nonserious diseases (13%).

Figure 1

Algorithm used to assign the overall score for innovation. Available treatments: A = drugs for diseases without recognized standard treatment; B = drugs for diseases where subsets of patients are less responsive to marketed drugs and/or other medical interventions, C = drugs for diseases responsive to marketed drugs or other medical interventions (C₁ = more effective or safer than existing drugs; C₂ = mere pharmacological innovation, i.e. drugs with better kinetics or new mechanism of action; C₃ = mere technological innovation, i.e. a new chemical or biotechnological product with therapeutic role similar to already existing ones). Therapeutic effect: A = major benefit on clinical end-points (e.g. increased survival rate and/or quality of life) or validated surrogate end-points; B = partial benefit on the disease (on clinical or validated surrogate end-points) or limited evidence of a major benefit (inconsistent results); C = minor or temporary benefit on some aspects of the disease (e.g. only partial symptomatic relief of a serious disease).