E8: Phase III (Most typical kind of study: Therapeutic Confirmatory)

Question 1

Phase III

Therapeutic
Confirmatory

Objective:

Answer 1

• Demonstrate/confirm efficacy
• Establish safety profile
• Provide an adequate basis for assessing the benefit/risk relationship to support licensing
• Establish dose-response relationship

Question 2

Phase III

Therapeutic
Confirmatory

Study Examples:

Answer 2

• Adequate, and well controlled studies to establish efficacy
• Randomised parallel dose response studies
• Clinical safety studies
• Studies of mortality/ morbidity outcomes
• Large simple trials
• Comparative studies