Lecture 11 - Outcome Measures

Question 1

What are objectives?

Answer 1

Objectives are precise statements about the overall goals of the research, describing the intervention, the duration of exposure (if applicable) and the patient population

Objectives are often stated as hypothesis

Question 2

What is an outcome?

Answer 2

An outcome is the measured result or consequence of an event, a disease, a drug, a treatment, etc.

Also commonly referred to as “end point”

Question 3

List and describe some types of outcomes

Answer 3

Clinical outcomes (health outcomes):
-Generally outcomes that patients would notice if they occurred

Surrogate outcomes:
-An event that is regarded as being highly correlated, indicative or predictive of a clinically meaningful outcome in a trial

Safety outcomes (adverse events):
-Negative consequences of an intervention

Question 4

Primary outcome should ideally be ____ outcome

Answer 4

one

Question 5

What does the primary outcome dictate?

Answer 5

how big a sample we need in our study

Question 6

Describe a primary outcome

Answer 6

Parameter that is measured that will provide the answer to the most important question of the clinical trial.

Question 7

Primary outcome used for ____ ____ calculations

Answer 7

sample size

Question 8

Describe secondary outcomes

Answer 8

Parameters that are measured that provide useful additional information but are not the primary outcome (ex. Adverse event rates)

Question 9

What are the outcomes of diabetes?

Answer 9

1) Glycemic outcomes
- Glycosylated Hemoglobin
- Fasting blood glucose
- Post prandial glucose

2) Micro-vascular complications
- Nephropathy
- Retinopathy

3) Macro-vascular complications
- Heart attack
- Stroke
- Peripheral vascular disease

Question 10

What are some endpoints for diabetes?

Answer 10

Hypoglycemic endpoints

Question 11

What are some CV disease outcomes?

Answer 11

Surrogate outcomes:

• cholesterol measures
• interluminal thickness
• C-reactive protein

Clinical outcomes:

• Heart attack
• Heart failure
• Angina
• Revascularization
• Common use of composite endpoints

Question 12

What are some Alzheimer’s Disease Outcomes?

Answer 12

• Measurement of rate of patient decline rather than improvement or prevention of events
• Greater focus on patient testing and functional assessment

Question 13

What are some pain management outcomes?

Answer 13

Highly patient specific symptom with multidimensional nature

• Intensity
• Sensory characteristics
• Affective response
• Ability to cope
• Difficult to quantify pain symptoms
• Total reliance on patient self-reporting