Measurements, outcomes, endpoints

Question 1

Define outcome

Answer 1

something measured to determine whether a change has occurred in response to an exposure

Question 2

What does clinical trial endpoint mean

Answer 2

It is a parameter analysed in a clinical trial that reflects the effects or safety of the drug

Question 3

Define primary outcome

Answer 3

Outcome that establish the drug’s effectiveness and/or

safety to support regulatory action

Question 4

Define secondary outcome

Answer 4

Demonstrates additional effects after success of the

primary endpoint. E.g. Benefits, safety, disease mechanisms

Question 5

Define exploratory outcomes/endpoints

Answer 5

explore new hypotheses, rare but important outcomes

Question 6

Define endpoint

Answer 6

analysed parameter which reflects the efficacy or safety of a drug

Question 7

Define clinically-meaningful outcomes

Answer 7

how the patient feels, functions or survives

Question 8

Define biomarker

Answer 8

a biochemical, physiological, structural, genetic measurement which indicates normal biological or pathological processes and/or pharmacological response

Question 9

Define surrogate markers

Answer 9

biomarkers that predict a clinically-meaningful outcome

Question 10

What is the purpose of a chi-square test and when can chi-square tests be used

Answer 10

they compare the observed value to the expected value (categorical data)

can only be used if:

• data are frequencies or counts
• study groups are independent and each participant contributes data only to one cell
• value of cell expected should be >5 in more than 80% of cells and not zero

Question 11

Define absolute risk

Answer 11

number of people with event/total number of people

Question 12

define relative risk (ratio)

Answer 12

absolute risk treatment group/absolute risk comparator group

Question 13

define absolute risk reduction

Answer 13

absolute risk comparator group - absolute risk treatment group