Clinical Trials

Question 1

Why are clinical trials important?

Answer 1

They provide evidence - practice is evidence based

Question 2

What is defined as pre-clinical development?

Answer 2

Investigating animal pharmacology

Investigating animal toxicology

Tissue culture

Question 3

What is phase 1 of clinical development?

Answer 3

Volunteer studies
Clinical pharmacology

100 subjects

Question 4

Which drugs are not usually seen in phase 1 clinical trials?

Answer 4

Certain cytotoxic drugs - that cause harm to the individual

Question 5

What is phase 2 of a clinical trial?

Answer 5

Involves up to 500 patients

Provides some evidence of efficacy and identifies a likely dosage range

Confirms kinetics and dynamics

Question 6

What is phase 3 of a clinical trial?

Answer 6

Efficacy is established and evidence of safety is obtained

It determines if the drug works for the condition we are testing

All data collected and used to request a license to sell the drug

1000-10000 patients

Question 7

What is phase 4 of clinical development of a drug?

Answer 7

Post marketing surveillance to produce evidence of long term safety
10,000 - 100,000

Question 8

What is the purpose of a pilot study?

Answer 8

To test study design

Question 9

What are the different methods of trials?

Answer 9

Double blind- patient doctor blinded
Single blind- patient blinded
Prospective- protocal decided before hand
Retrospective- less gd open to bias

Question 10

What are the stages of a prospective clinical trial?

Answer 10

Design the study, recruit, then follow up

Question 11

What is a retrospective clinical trial?

Answer 11

When data is collected from case records after treatment is given

Question 12

What is a cross over study?

Answer 12

When patients receive both methods of treatment, one after the other with a cross over stage in the middle called a “wash out period” - removes all the old drug

Question 13

What is the difference between a superiority VS a Non-inferiority trial?

Answer 13

Superiority - Shows that a new treatment is better then control (standard or placebo)

Non-inferiority - shows that the new treatment is not worse than the standard by much

Shows that the new treatment would have beaten a placebo arm

Question 14

Who might you exclude from a clinical trial?

Answer 14

Pregnant women
Children
Seriously ill
Elderly
Patients at risk of side effects