Drug Discovery and Development: The Road from an Idea to Promoting Human Health

We are almost where we need to be to grasp the tales of drug discovery that make up the final seven chapters of this book. The three previous chapters have laid the necessary scientific foundation. Here is my take on what you still need to know to understand drug discovery and development: the process of getting from an idea to a product that meets a medical need—from the laboratory to the bedside. We begin with a look at the process from 35,000 feet. Realize one thing at the outset: there is more than one way of getting from an idea to approval of a new drug for use in human medicine. The process described in this chapter captures the essential features of getting this done. However, each drug discovery effort poses specific problems, and getting around them may have an effect on the actual process followed. Nonetheless, what is described here is well worth understanding. Imagine running a maze that may have more than two dimensions, a huge number of entry points, and a very small number of exits or perhaps no exit at all, depending on your entry point. Have a look at Figure 5.1 to get the idea. You can see the external features but have no clue what awaits you inside. You can wander in this maze for a long time without getting very far. This pretty well symbolizes what happens during a lot of drug discovery projects. In target-based drug discovery, described in the next section, each entry point in the maze corresponds to a molecule chosen as a starting place to begin work. Most of these entry points are dead ends. No matter what you do or how hard you try, the only exit from the maze is the place you entered—nothing gained. The journey may be long, there may be encouraging signs along the way, but at the end of the day, you are back where you started.