As a rule, a pivotal study can be equated with a phase III study. It is a clinical trial that pursues the goal of obtaining marketing authorization for a new pharmaceutical preparation or medical device. Pivotal studies are also required if drugs that have already received approval are to be considered for another indication in the future. Approval studies must be reviewed by the relevant regulatory authorities with regard to the safety and efficacy of the medical preparations.
The study is usually randomized, double-blind, and controlled. Usually, the number of subjects who receive the drug for testing is significantly higher than in Phase I and II studies. They include up to several thousand patients. The focus is on determining safety, proof of efficacy, and side effects. Furthermore, interactions between the drug with other medications is also administered and examined. After review and positive completion of a pivotal study, the drug receives approval for the market from the drug regulatory agency.