Drug discovery

Lore & Background

Historically, drugs were discovered by identifying active ingredients from traditional remedies or by serendipity, as with penicillin. Later, classical pharmacology screened substances in intact cells or whole organisms without knowledge of the biological target. After the human genome was sequenced, reverse pharmacology became common, using high-throughput screening of large compound libraries against isolated targets hypothesized to be disease-modifying. Hits are then tested in cells and animals for efficacy.

Reader's Guide

Drug discovery is a capital-intensive process involving large investments from pharmaceutical corporations and national governments. Despite technological advances, it remains lengthy, expensive, and inefficient, with a low rate of new therapeutic discovery. Basic discovery research in the 21st century is funded primarily by governments and philanthropic organizations, while late-stage development is funded by pharmaceutical companies or venture capitalists. Drugs must pass several phases of clinical trials and a new drug approval process, such as the New Drug Application in the United States. The process involves complex interactions between investors, industry, academia, patent laws, regulatory exclusivity, marketing, and the need to balance secrecy with communication. For rare disorders, the orphan drug funding process provides hope for pharmacotherapeutic advances.

Did You Know?

• Historically, drugs were discovered by identifying active ingredients from traditional remedies or by serendipitous discovery, as with penicillin.
• In the 2020s, qubit and quantum computing started to be used to reduce the time needed to drug discovery.