Pharmaceutical Development Process

The development of a pharmaceutical candidate into an approved product is a highly regulated process. This process typically covers eight to ten years of work from the time that the pharmaceutical candidate enters preclinical phase until approval.

Preclinical Phase

Emphasis is on testing through the use of animal studies, to determine if the Candidate Drug (CD) is sufficiently safe for testing in humans.

Phase I

The first tests on humans are carried out. Normally,
a small group of healthy volunteers (20–100) is selected. The CD is given in increasing doses in order to test its safety and how it is absorbed and broken down in the body.

Phase II

Trials are performed to test the CD's effect on patients and to establish an appropriate dosage level. Study sizes vary greatly depending on the area of disease, from around ten up to several hundred patients.

Phase III

The efficacy of the CD is compared to a placebo and/or existing therapies. Data from these studies form the basis for subsequent applications for market approval of the pharmaceutical. The clinical trials in Phase III are normally multicenter studies on large patient groups, from around 300 to 3,000 or more depending on the target indication.

Market

PROOF-OF-PRINCIPLE
The first indication that the CD can be expected to have the intended effect in the body at a dosage level that will not give rise to unwanted side effects. Typically this is shown using relevant animal models and in Phase I.

PROOF-OF-CONCEPT
Proof-of-concept is achieved when the CD has shown effect in patients at the intended dosage level, typically achieved during Phase II.