What is Drug Discovery?

Drug discovery is a process used by scientists and pharmaceutical researchers to identify new pharmaceutical products and develop them. This is a very lengthy process and only a small minority of compounds of interest make it all the way from initial exploration to approval from regulatory agencies for use in patients. The discovery process is one reason why drugs are so costly when they are new, because pharmaceutical companies invest substantial amounts in the development of new drugs and they must recoup these funds from the medications they manage to bring to market.

Historically, much of drug discovery was trial and error. Today, researchers focus on identifying targets, such as specific types of cells, that play a role in disease and infection. Genetics plays a major role in this process as it allows researchers to understand targets on an intimate level. The target is researched and the resulting information is applied to the development of a compound that can be introduced to the target to achieve a desired effect. For example, a cancer drug might target the line in the genetic code which tells cancer cells to replicate.

Plants, animals, and other organisms, along with minerals, can all yield compounds that may be pharmacologically useful. In addition, researchers can synthesize chemical compounds. This includes synthetic versions of natural compounds along with entirely new drugs. In the lab, analysis of potential pharmaceuticals includes meticulous work to break down their components and understand how they function.

Researchers may have multiple compounds in development at any given time. They use a variety of biotechnology techniques to explore potential chemical compounds of interest during drug discovery. Part of this work also includes following progress at other labs to get an idea of what kinds of competing products may be introduced in the near future by rival companies. Being ahead of the competition can be a benefit for a pharmaceutical company that wants to get a jump on the market.

Simply identifying a target and finding a compound that can work with it can take years. At this pre-clinical development stage of drug discovery, the compound has not been tested in any living organisms. If the drug discovery process appears promising, steps can be taken to develop prototypes for testing. A series of clinical trials will be used to test the drug to see if it is effective, to identify the side effects, and to weigh the risks and benefits of the drug against each other to see if it is marketable. These trials are used in an application to a regulatory agency to get permission to sell the finished product on the open market.