Phase I - Human Pharmacology

The maximum tolerated dose (MTD) is the dose at which limited adverse events occur in more participants. The adverse events are defined by objective criteria. The MTD should not by systematically searched for in healthy participants.

If the MTD is set too high, patients could be exposed to unsafe doses, and the resulting intolerance of adverse events may even lead to the discontinuation of the medicine’s development.

If the MTD is set too low, placebo or sub-therapeutic doses could be administered to patients in Phase II efficacy studies, wasting years of development and conducting no-effect studies. This may also lead to the discontinuation of the medicine’s development.