This article provides an overview of the various phases of the drug development process, and describes how statisticians participate in that process. In the United States, the 1962 amendments of the Food, Drug, and Cosmetics Act require substantial evidence of the safety and efficacy of drugs before they can be sold. Most countries around the world have an agency similar to the FDA which evaluates drug research and approves drugs for marketing. Consequently, the FDA and regulatory agencies of other countries have drafted numerous guidelines, including statistical guidelines, for studying and documenting the safety and efficacy of drugs. Statisticians play a key role in the drug experimentation process leading to drug approval, particularly in determining the extent and complexity of the experiments, and interpreting the results of those experiments.
Drug discovery begins in the laboratories of basic research scientists.
Each screening experiment is designed to reveal active chemicals for a specific targeted disease. Once a natural substance is found to possess activity towards a disease target, chemists work to isolate the particular molecule which is responsible for the activity. Another way drugs are discovered is by combinatorial synthesis. In combinatorial synthesis, molecules of several different types, each known to have a certain desirable chemical property, are chemically combined to form all possible combinations of resultant combination molecules. In this way, thousands of potential drug chemical candidates are produced and tested in screening experiments like those mentioned above for natural product screening.