Drug discovery and development: Role of basic biological research

Abstract

This article provides a brief overview of the processes of drug discovery and development. Our aim is to help scientists whose research may be relevant to drug discovery and/or development to frame their research report in a way that appropriately places their findings within the drug discovery and development process and thereby support effective translation of preclinical research to humans. One overall theme of our article is that the process is sufficiently long, complex, and expensive so that many biological targets must be considered for every new medicine eventually approved for clinical use and new research tools may be needed to investigate each new target. Studies that contribute to solving any of the many scientific and operational issues involved in the development process can improve the efficiency of the process. An awareness of these issues allows the early implementation of measures to increase the opportunity for success. As editors of the journal, we encourage submission of research reports that provide data relevant to the issues presented.

Fig. 1

A diagram of the stages of drug discovery and development with estimates of cost and duration.

Fig. 2

A summary of the information to be developed before the selection of a clinical candidate molecule proposed for testing in humans.

Fig. 3

A summary of the critical compound-related information needed from phase 2 to improve the likelihood of moving to phase 3.

Fig. 4

A partial list of the kinds of data and research tools that contribute to a successful drug discovery and development program.

Fig. 5

A schematic of the activities involved in the drug discovery and development process. At the left are shown icons depicting small molecules (NMEs) and biological molecules (NBEs) being considered for development. At the top are the time lines for quality assurance guides governing the process; they are good laboratory practice (GLP), good manufacturing practice (GMP), and good clinical practice (GCP). Specific activities in the stages of development are shown at the bottom; they include studies of absorption, distribution, excretion and metabolism (ADEM), screening for activity at cytochrome P450 (CYP) liver enzymes, and regulatory filings for Investigational New Drug (IND) and New Drug Application (NDA). Abbreviations: NBE, New Biological Entity; NME, New Molecular Entity.