Good Clinical Practice (GCP)
Full course · Clinical Research Foundations
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Understand the large-scale confirmatory trials that determine whether an investigational therapy will receive regulatory approval.
What you will learn7 objectives
By the end of this lesson, you will be able to:
A pivotal trial is one designed to provide the definitive evidence that regulatory authorities need to make an approval decision. Not all Phase III trials are pivotal, but the most important ones carry this designation because they serve as the pivot point, the fulcrum on which the entire drug development program turns.
Before Phase III, researchers have asked exploratory questions. Is the drug safe enough to give to humans? Does it show any sign of working? What dose should we use? These questions are important, but the answers are preliminary. Phase III asks the confirmatory question: Does this drug work well enough, with an acceptable safety profile, to justify making it available to patients?
The FDA and other regulatory authorities base their approval decisions primarily on evidence from pivotal trials. This evidence must demonstrate that the drug provides a meaningful clinical benefit that outweighs its risks. The trials must be large enough to detect this benefit reliably. They must be designed rigorously enough to rule out alternative explanations for the results. And they must be conducted with sufficient quality to ensure the data are trustworthy.
Progress saves itself — finish reading and this lesson completes
When an investigator enrolls the first patient in a pulmonary fibrosis trial, she is contributing to a body of evidence that will ultimately be reviewed by regulatory scientists around the world. Every data point matters. Every procedure must be performed according to protocol. The quality of work at each research site directly influences whether the drug ever reaches the patients who need it.
