Clinical Trials

Question 1

What is required for clinical trials?

Answer 1

Evidence

Question 2

What are the two reasons drugs need to be tested?

Answer 2

Efficiency

Safety

Question 3

What is the point of pilot studies?

Answer 3

To test study design.

Question 4

What are the 4 types of trials?

Answer 4

Double blind
Single blind
Prospective
Retrospective

Question 5

What does a placebo controlled study allow the comparison of?

Answer 5

Outcome in the groups of active drug and placebo drug.

Question 6

What does a comparison with other therapy study allow the comparison of?

Answer 6

End points (is A better than B)

Question 7

What is the problem with observational studies?

Answer 7

Correlation vs. Causation

Question 8

How long can it take to get a chemical structure to a licensed drug?

Answer 8

10 years

Question 9

What are the 3 types of pre clinical development tests?

Answer 9

Animal pharmacology (dose, adverse effects)
Animal toxicology (fertility issues, tumours etc)
Tissue culture

Question 10

What is phase 1 of clinical development?

Answer 10

Volunteer studies

Question 11

What type of drugs bypass phase 1?

Answer 11

Cytotoxics

Question 12

What is phase 2 of clinical development?

Answer 12

Investigation with 500 patients to confirm kinetics and dynamics.

Question 13

What does phase 2 provide?

Answer 13

Evidence of efficacy and identifies a dosage range.

Question 14

What is phase 3 of clinical development?

Answer 14

Formal trials where efficacy will be established and safety confirmed. 1000-3000 patients.

Question 15

What is phase 4 of clinical development?

Answer 15

Post-marketing surveillance to produce evidence of long term safety.

Question 16

What is a double blind study?

Answer 16

When neither the patient nor the doctor knows what drug the patient is getting.

Question 17

What is a single blind study?

Answer 17

When the patient doesn’t know what is happening but the doctor does. Not as good as double blind.

Question 18

What is a prospective study?

Answer 18

Design the study, recruit the patients and give the drugs.

Question 19

What is a retrospective study?

Answer 19

Looking at people on a certain drug and another drug, but more biased as you aren’t doing the work yourself.

Question 20

What can cross over designs decrease?

Answer 20

Number of people in the study

Question 21

What is a cross over design?

Answer 21

When they try both drugs but with a break in between.

Question 22

What are the disadvantages of randomised control clinical trial?

Answer 22

May not represent general patient population (would take drugs correctly which doesn’t always happen)
Twice as many patients needed for the study
Some physicians refuse closure and patients want treatment, not placebo.
Complexity

Question 23

What are 2 commonly used phase 3 designs?

Answer 23

Parallel

Cross voer

Question 24

What does a superiority design show?

Answer 24

New treatment is better than control.

Question 25

What does a non-inferiority study show?

Answer 25

New treatment is not worse than standard by a margin, would have beaten placebo.

Question 26

When designing a study, what should endpoints be?

Answer 26

As simple as possible

Question 27

What 4 things are required when designing a study?

Answer 27

Hypothesis
Endpoints
Number of subjects
Safety endpoints

Question 28

What is usually taken as the statistical test?

Answer 28

p<0.05

Question 29

What does a statistical test prove?

Answer 29

If there is a significance in results.

Question 30

What are the 2 interpretations of insignificant findings?

Answer 30

No difference (may be bad study)
Two treatments are clinically equivalent.