Lecture 7 - Is This Intervention Effective?

Question 1

What is the sequence in modern clinical trials?

Answer 1

1. In vitro studies
2. Animal in vivo studies
3. Human Phase 1 - toxicology and pharmacokinetics
4. Human Phase 2 - evidence of efficacy
5. Human Phase 3 - RCTs
6. Human Phase 4 - post-marketing surveillance

Question 2

What are the critical appraisal guides?

Answer 2

• Is it valid?
• How large and precise is the effect?
• Can I apply these results to my patient

Question 3

What are some key characteristics for critical appraisal?

Answer 3

• Randomization
• Ascertainment (comparable groups, treated equally, follow-up complete)
• Measurement (blinded, objective)

RAMbo

Question 4

What is the best and most valid technique in ensuring allocation concealment?

Answer 4

Central computer randomization

Question 5

What is the principle in maintaing randomization?

Answer 5

Once a patient is randomized, s/he should be analyzed in the group randomized to (even if they discontinue, never receive treatment, or crossover

Question 6

What is the exception to the principle in maintaining randomization?

Answer 6

If patient is found on BLIND reassessment to be ineligible based on pre-randomization criteria

Question 7

What’s the difference between Intention to Treat vs Per Protocol?

Answer 7

• ITT provides a measure of effectiveness of an intervention
• PP analysis proves efficacy measure (ideal world because it only measure pt who are able to completely adhere to full treatment regime)