Novel approaches

The productivity of the pharmaceutical industry has declined consistently over the last 10-15 years. The traditional approach of targeting membrane-bound receptors and enzymes with relatively small molecules has, to a large extent, been exploited. Modern targets, such as complex second messenger systems, nucleic acids and cellular networks, present very different challenges.

The range of new approaches to drug development is extremely broad and the potential of these novel therapeutic agents is enormous. The greatest challenge to their successful exploitation is a lack of development science to guide the translation of the molecule into a useful medicine with a well-defined benefit-risk profile. A selection of novel approaches, their potential applications, and key challenges to their development are outlined in Table 1.

Table 1 Novel approaches to drug development.