Exposures

Question 1

What is the purpose of an outcome measure

Answer 1

it is an instrument that objectively measures the difference between a particular function at baseline and then following intervention - it answers the research question, it is simple and clinically meaningful e.g. improves survival

Question 2

What are the 3 main importances of outcome measures

Answer 2

1. impacts the study design
2. impacts sample size due to the primary outcome that needs to be seen to be considered as clinically effective
3. impacts cost and time

Question 3

What is the difference between an outcome and endpoint

Answer 3

outcome = measured variable e.g. blood glucose before and fater new drug or placebo

endpoint = analysed parameter e.g. change in blood glucose from baseline

Question 4

What is the purpose of a secondary endpoint

Answer 4

used to demonstrate additional success on primary outcome like demonstrating the underlying mechanism of the beneficial success (side effects and tolerability)

Question 5

What is the purpose of exploratory endpoint

Answer 5

outcomes that are used to frame future research (other areas of interest) - explore new hypotheses

Question 6

Name 3 criteria outcome measures should meet in terms of specificity

Answer 6

1. time specific e.g. number of events over study period
2. what is being collected e.g. length of hospital stay
3. how it is collected e.g. health questionnaire for depression

Question 7

What does COMET stand for and what is the purpose

Answer 7

core outcome measures in effectiveness trials

it represents the minimum that should be measured in all clinical trials for a specific condition

this allows outcomes of studies to be compared

Question 8

What is a surrogate endpoint

Answer 8

substitute for a clinically meaningful endpoint e.g blood pressure for cardiovascular death - it is easier to obtain and clinical outcome can take a long time e.g. death

Question 9

what is a patient reported outcome

Answer 9

any report on the status of a patient’s health condition that comes directly from the patient without interpretation of the patient’s response by a clinician or anyone else

basically the patient reports on their own condition e.g. symptoms, quality of life

Question 10

why is patient reported outcome important

Answer 10

it shows what really matters to the patient

funding in the healthcare system

its reliable

Question 11

how is patient reported outcomes compromised

Answer 11

the methods used to validate a PRO is difficult as a ‘score’ may be difficult to understand

Question 12

What is a composite endpoint

Answer 12

a single endpoint that combines a combination of individual endpoints that are related

Question 13

How is a composite endpoint beneficial

Answer 13

• useful when intervention can benefit patient in a number of ways