ICH Topic E 8 General Considerations for Clinical Trials

Question 1

Phase 1 type of study

Answer 1

Human pharmacology

Question 2

Phase 2 type of sutdy

Answer 2

Therapeutic Exploratory

Question 3

Phase 3 type of study

Answer 3

Therapeutic Confirmatory

Question 4

Phase 4 type of study

Answer 4

Therapeutic Use

Question 5

Objective of Phase 1

Answer 5

Assess tolerance
Define PK/PD
Explore drug metabolism adn drug interactions
Estimate Activity

Question 6

Objective of Phase 2

Answer 6

Explore use of the targeted indication
Estimate dosage for subsequent studies
Provide basis for confirmatory study design, endpoints, methodologies

Question 7

Objective of PHase 3

Answer 7

Demonstrate/ confirm efficacy
Establish safety profile
Provide and adequate basis for assessing the benefit/risk relationship to support licensing
establish dose-response relationship

Question 8

Objective of Phase 4

Answer 8

Refine understanding of benefit/risk relationship in general or special populations and/or environments
identify less common adverse reactions
refine dosing recommendation

Question 9

Dose Tolerance Study

Answer 9

Phase 1

Question 10

Single and multiple dose PK and or PD studies

Answer 10

Phase 1

Question 11

Drug interaction studies

Answer 11

Phase 1

Question 12

Trials in relatively short duration, well defined narrow patient populations using surrogate o pharmacologic endpoints or clinical measures

Answer 12

Phase 2

Question 13

Dose-response exploration studies

Answer 13

Phase 2

Question 14

Well controlled studies to establish efficacy

Answer 14

Phase 3

Question 15

Randomized parallel dose-response studies

Answer 15

Phase 3

Question 16

Clinical safety studies

Answer 16

Phase 3

Question 17

Studies of mortality/morbidity outcomes

Answer 17

Phase 3 or Phase 4

Question 18

Large simple trials

Answer 18

Phase 3 or Phase 4

Question 19

Comparative studies

Answer 19

Phase 3

Question 20

Comparative effectiveness studies

Answer 20

Phase 4

Question 21

Pharmacoeconomic studies

Answer 21

Phase 4