Fundamentals

Question 1

Interventional vs Observational Study

Answer 1

Intervention - give drug, Observation - watch

Question 2

4 types of observational

Answer 2

Cross section, cohort, case control, ecological

Question 3

Cross sectional Study example

Answer 3

Survey

Question 4

Cohort study

Answer 4

Follow a population over time

Question 5

Case control Study

Answer 5

1 group with outcome, 1 without

Question 6

Ecological

Answer 6

Measure disease rates in population

Question 7

Gold Standard of Clinical Trials

Answer 7

Randomised Controlled Trials

Question 8

Randomisation

Answer 8

Allocate treatment groups at random

Question 9

What type of bias does randomisation avoid?

Answer 9

selection bias

Question 10

What is bias?

Answer 10

Tendency of estimate to deviate one way or other

Question 11

What does bias impact on? (2)

Answer 11

Validity and Reliability

Question 12

Why do clinical trials follow rules? 4

Answer 12

to make sure they are well designed, safe, right measurements,meaningful results

Question 13

Nuremberg code

Answer 13

Research ethics for trials on humans - end of WWII

Question 14

What was first sig effort to regulate trials? year

Answer 14

Declaration of Helsinki 1964

Question 15

CTIMP vs non CTIMP

Answer 15

CTIMP - fall within law, non CTIMP - NHS governance

Question 16

Phase I trials - describe

Answer 16

First in man and healthy people or patients

Question 17

How many in Phase I?

Question 18

What is Phase1 used for?5

Answer 18

Tolerability, safety, bioavailability, pharmacodyn and kin, max tolerated dose

Question 19

Phase II who?

Answer 19

Larger group - with condition - narrow inclusion criteria

Question 20

How many people in phase II?

Answer 20

30 - 50 people

Question 21

What does phase II test?

Answer 21

Safety and effect

Question 22

Phase III how many?

Answer 22

100s to 1000s

Question 23

Phase III describe

Answer 23

New vs Placebo - looking for approval

Question 24

Phase IV

Answer 24

After it has been approved

Question 25

Four features of good clinical trials

Answer 25

Randomisation, controlled, blinding, large sample size

Question 26

Control group - why

Answer 26

existing standard of care or placebo - compare to cancel external factors

Question 27

Randomisation

Answer 27

Neither doctor nor patients choose, sequence concealed, so treatment and control are sim in respect to prog factors

Question 28

Blinding why?

Answer 28

Minimise observer bias

Question 29

Why large sample size?

Answer 29

Stat power

Question 30

7 trial designs

Answer 30

Parallel, cross over, factorial, group allocation, superiority, equivalence, non inferiority, adaptive

Question 31

Parallel

Answer 31

Most trials, intervention and control simultaneously

Question 32

Cross over

Answer 32

A then B or B then A

Question 33

Cross over design assumptions 2

Answer 33

One effect doest carry over into other, effects are independent of which one first

Question 34

When is cross over used? 2

Answer 34

2 versions of same drug - bioequivalence , not if conditions change

Question 35

Factorial design

Answer 35

2x2 - 2 interventions, one control

Question 36

Group allocation

Answer 36

Group randomised - when risk of contamination

Question 37

Superiority

Answer 37

New better than old

Question 38

Equivalence

Answer 38

New more or less equal to old

Question 39

Non inferiority

Answer 39

new no worse than old

Question 40

Adaptive

Answer 40

multi -arm, multi stage , add or subtract arms