Step 1: Discovery and Development

Discovery

Typically, researchers discover new drugs through:

• New insights into a disease process that allow researchers to design a product to stop or reverse the effects of the disease.

• Many tests of molecular compounds to find possible beneficial effects against any of a large number of diseases.

• Existing treatments that have unanticipated effects.

• New technologies, such as those that provide new ways to target medical products to specific sites within the body or to manipulate genetic material.

At this stage in the process, thousands of compounds may be

potential candidates for development as a medical treatment. After early testing, however, only a small number of compounds look promising and call for further study.

Development

Once researchers identify a promising compound for development, they conduct experiments to gather information on:

• How it is absorbed, distributed, metabolized, and excreted.

• Its potential benefits and mechanisms of action.

• The best dosage.

• The best way to give the drug (such as by mouth or injection).

• Side effects or adverse events that can often be referred to as toxicity.

• How it affects different groups of people (such as by gender, race, or ethnicity) differently.

• How it interacts with other drugs and treatments.

• Its effectiveness as compared with similar drugs.