2.01 – The Intersection of Strategy and Drug Research

This chapter provides an introduction to strategy and drug research on selected topics, directed to medicinal chemists in particular, but also of interest to others who follow the broad biotechnology and pharmaceutical sector, both technical and lay. Different perspectives and more detail from a strategic and technical viewpoint on the topics discussed herein can be found elsewhere in Volumes 1 and 2. More detail from a scientific perspective can be found in Volume 3 (Technologies), Volume 4 (Computer-Aided Drug Design), Volume 5 (ADMET Approaches), Volumes 6 and 7 (Therapeutic Areas), and in the case histories in Volume 8. It is the author's hope that the present chapter will provide the reader interested in medicinal chemistry and drug discovery with a general background to the field as well as a suitable entry point to other chapters in this reference work and to leading references in the general literature of this critically important field. Many of the references given in this chapter are review articles, which will provide the next layer of overview. Others are primary articles, old and new, that convey an important point. A few are so recent that they have been taken from newspapers or other periodicals. Scattered throughout are examples, anecdotes, curiosities, and brief but representative case histories. What is strategy in drug research? A strategy is a plan of action designed to achieve a specific goal – in very simple terms, a means to an end. The ultimate goal of drug research is to advance a product candidate through progressively later stages of research and development (R&D) to commercialization. Once that goal has been achieved, with patients being treated successfully with the new drug, research does not stop. Rather, additional safety data are collected, new therapeutic indications are pursued, alternative formulations and dosing regimens are tested, new approaches to disease are advanced, and much more, in a seemingly never ending cycle. At times, this process may seem to be little more than trial and error, albeit sophisticated, on a large and expensive scale. Of course it is much more than that, as you will see. The present chapter opens with a survey of the many organizational aspects of pharmaceutical and biotechnology R&D. This survey covers major issues like attrition, project management and teams, recruiting and managing human resources, outsourcing, innovation at large and small companies, business development, portfolio management, and a brief primer on what happens after drug discovery, namely, drug development. We then review in more detail selected technologies that have revolutionized drug discovery in recent years, with a bias toward chemical strategies. This section includes combinatorial chemistry, chemical biology, genomics, small molecule drugs versus macromolecules, high-throughput screening, computer-aided drug design, and preclinical development. The changes in the workforce and the training of students who begin careers in these industries are considered, along with tips from drug hunters. Finally, we conclude with a discussion of drug targets, notably transporters.