DDD Clinical Trials

Question 1

To determine the maximum tolerated dose (MTD) of the new
treatment

Answer 1

Phase I

Question 2

The MTD is found by escalating the treatment dose until the
dose-limiting toxicity (DLT) is reached

Answer 2

Phase I

Question 3

Tolerability and Safety

Answer 3

Phase I

Question 4

Pharmacokinetics

Answer 4

Phase I

Question 5

Pharmacodynamics

Answer 5

Phase I

Question 6

Therapeutic exploratory trial

Answer 6

Phase II

Question 7

Participants: 20-300 subjects

Answer 7

Phase II

Question 8

To confirm effectiveness, monitor side effects, and further
evaluate safety

Answer 8

Phase II

Question 9

First in patients (who have the disease that the drug is expected
to treat)

Answer 9

Phase II

Question 10

Duration: 6 months to several years

Answer 10

Phase II

Question 11

Efficacy in patients (primary objective)

Answer 11

Phase II

Question 12

Safety issues (secondary objective)

Answer 12

Phase II

Question 13

Optimum dose finding

Answer 13

Phase II

Question 14

Dose efficacy relationship

Answer 14

Phase II

Question 15

Therapeutic dose regimen

Answer 15

Phase II

Question 16

Duration of therapy

Answer 16

Phase II

Question 17

Frequency of administration

Answer 17

Phase II

Question 18

Therapeutic window

Answer 18

Phase II

Question 19

First stage of testing in human subjects

Answer 19

Phase I

Question 20

Designed to assess the safety, tolerability, PK and PD of drug

Answer 20

Phase I

Question 21

Participants: 20-25 healthy volunteers

Answer 21

Phase I

Question 22

Duration: 6-12 months

Answer 22

Phase I

Question 23

No blinding/Open labelled

Answer 23

Phase I

Question 24

Therapeutic confirmatory trials

Answer 24

Phase III

Question 25

Large scale, multicenter, randomized, controlled trials

Answer 25

Phase III

Question 26

Participants: several 100’s to 3000 patients

Answer 26

Phase III

Question 27

Duration: up to 5 years (takes a long time)

Answer 27

Phase III

Question 28

To establish efficacy of the drug against existing therapy in larger number of patients, method of usage, and to collect safety data etc.

Answer 28

Phase III

Question 29

To assess overall and relative therapeutic value of the new drug

Answer 29

Phase III

Question 30

Efficacy, Safety and Special Properties

Answer 30

Phase III

Question 31

To determine optimal dosage schedule for use in general

Answer 31

Phase III

Question 32

The dosage schedule in clinical trials should be as close as possible to its anticipated clinical use

Answer 32

Phase III

Question 33

Pre-requisite: Efficacy and dose schedule defined in Phase II studies

Answer 33

Phase III

Question 34

Pre-requisite: No gross/serious ADRs (risks higher than benefits)

Answer 34

Phase III

Question 35

Pre-requisite: Long-term preclinical safety studies completed (Chronic Toxicity, Reproductive Toxicity, Carcinogenicity)

Answer 35

Phase III

Question 36

Pre-requisite: Favorable marketing inputs

Answer 36

Phase III

Question 37

Pre-requisite: IRB and FDA approval obtained

Answer 37

Phase III

Question 38

How many years for the FDA to review the NDA?

Answer 38

2-3 years

Question 39

Post-Marketing Surveillance (PMS)

Answer 39

Phase IV

Question 40

Done after drug has been marketed

Answer 40

Phase IV

Question 41

No fixed duration/patient population

Answer 41

Phase IV

Question 42

Studies continue to collect data about effects in various populations and side effects from long-term use

Answer 42

Phase IV

Question 43

Primarily observational or non-experimental in nature

Answer 43

Phase IV

Question 44

Evaluation in different age groups/types of patients

Answer 44

Phase IV

Question 45

May involve: * Comparative Benefit-Risk assessment * Benefit-Cost assessment (Pharmacoeconomics) * Drug usage in the community * Quality of Life assessment

Answer 45

Phase IV

Question 46

Helps to detect rare ADRs, drug interactions

Answer 46

Phase IV

Question 47

To explore new uses for drugs

Answer 47

Phase IV

Question 48

Confirm the efficacy and safety profile in large populations

Answer 48

Phase IV

Question 49

Detect the unknown/rare adverse drug reaction/s

Answer 49

Phase IV

Question 50

Evaluation of over-dosage

Answer 50

Phase IV

Question 51

Identifications of new indication

Answer 51

Phase IV

Question 52

Dose refinement: Evaluation of new formulations, dosages, durations of treatment

Answer 52

Phase IV