| Phase and Purpose |
|
Sample Size |
|
Length of Study |
|
Questions Researchers Aim to Answer |
| I. Assess the safety of a drug |
|
20-100 health volunteers or people with the disease/condition |
|
Several months |
|
- How it works in the body?
- What are the side effects associated with increased dosage?
- Might the IMP work in patients?
|
| II. Test the efficacy of a drug |
|
Up to several hundred people with the disease/condition |
|
Several months to 2 years |
|
- Is the IMP safe in patients?
- Does the IMP seem to work in patients?
|
| III. Efficacy and monitoring of adverse reactions |
|
300 to 3000 volunteers with the disease/condition |
|
1 to 4 years |
|
- Is the IMP really safe in patients?
- Does the IMP work in patients?
|
| IV. Safety and efficacy |
|
Several thousand volunteers with the disease/condition |
|
After the FDA approval during the Post-Market Safety Monitoring |
|
- How safe is the new medicine?
- Does the medicine work in the real world?
|