Drug Development II: Clinical

Question 1

What THREE key factors are involved in drug trial design?

Answer 1

1. Variability of disease
2. Other diseases or risk factors
3. Subject & observer bias

Question 2

What THREE factors mitigate Subject & Observer Bias?

Answer 2

1. Placebo response
2. Single Blind design (removes subject bias)
3. Double Blind design (removes observer bias)

Question 3

What is the aim of Phase 1 in clinical trials?

Answer 3

• Is it safe?
• Dose vs. effect in ~10 healthy volunteers
• Pharmacokinetics in humans
• 1 year

Question 4

What is the aim of Phase 2 in clinical trials?

Answer 4

• Is it effective?
• Safety & efficacy in 100-500 patients with disease
• Single blind
• 2-5 years

Question 5

What is the aim of Phase 3 in clinical trials?

Answer 5

• Does it work double blind?
• 1000-3000 patients
• 2-4 years
• Verify effectiveness & safety

Question 6

What is the aim of Phase 4 in clinical trials?

Answer 6

• Post marketing surveillance
• Observe rare adverse side-effects