Outcome measures

Question 1

Key outcomes for a phase I clinical trial

Answer 1

Safety
Tolerability
Dose - PK

Question 2

Key outcomes for a phase II clinical trial

Answer 2

Efficacy
Safety
Tolerability

Question 3

Key outcomes for a phase III clinical trial

Answer 3

Efficacy
Safety
Tolerability

Question 4

Key outcomes for a phase IV clinical trial

Answer 4

Safety

Effectiveness

Question 5

Define efficacy

Answer 5

Efficacy is the extent to which an intervention does more good than harm under ideal circumstances

Question 6

Define effectiveness

Answer 6

Effectiveness assesses whether an intervention does more good than harm when provided under usual circumstances of healthcare practice

Question 7

Outcome measures for clinical trials; medical history

Answer 7

Presenting complaint 
Past medical history 
Medications 
Social history 
Family history

Question 8

Outcome measures for clinical trials; examination

Answer 8

General observation
Vital signs
Systems examination

Question 9

Outcome measures for clinical trials; investigations

Answer 9

Sampling
Imaging; Xray, CT scan
Physiology; ECG, lung function

Question 10

What are ‘events’

Answer 10

Disease onset or flare; heart attach, stroke, COVID infection

Healthcare contact
Survival

Question 11

What are clinically meaningful outcomes?

Answer 11

How the patient feels, functions.

E.g. symptom score, health care, disability

Question 12

What are biomarkers?

Answer 12

Biochemical, physiological, structural measurements.

Indicate normal biological or pathophysiological processed.

Question 13

What are surrogate markers?

Answer 13

Biomarkers that predict a clinically-meaningful outcome