Drugs

Question 1

The probability of successful transition to the next stage of drug development

Answer 1

P(TS)

Question 2

In drug development, WIP means

Answer 2

Work in Progress

Question 3

In drug development, NME means

Answer 3

New molecular entity

Question 4

For drug discovery, we want to develop an assay to evaluate activity of compounds on the

Answer 4

Target

Question 5

Then we want to identify a

Answer 5

Lead compound

Question 6

Then we use animal models to optimize the lead compound to give a

Answer 6

Proof-of-concept Molecule

Question 7

Then we optimize to give the compound

Answer 7

Drug-like properties

Question 8

When we study this drug in vitro, we want to define the drugs

Answer 8

Pharmacology

Question 9

In vitro studies (i.e. cell lines), drug formulation, and drug chemistry are examples of

Answer 9

Components of non-clinical drug development

Question 10

Used to show efficacy of the drug

Answer 10

In vivo studies on animal models

Question 11

Pharmacodynamics, pharmacokinetics, drug interactions, and receptor sensitivity are examples of variables in

Answer 11

Clinical trials

Question 12

Once the preclinical studies are completed, we submit an application to the FDA called

Answer 12

IND or investigational new drug

Question 13

Allows you to pursue phased clinical trials

Answer 13

IND

Question 14

It is estimated that what percentage of drugs fail in phase I trials because of unsuitable pharmacokinetics?

Answer 14

40%

Question 15

These unsuitable pharmacokinetics are determined in

Answer 15

Phase 0 trials

Question 16

The drug must have a wide therapeutic index and the target must be known, with a validated bio marker in order to be tested in

Answer 16

Phase 0 trials

Question 17

No therapeutic benefit can be conferred by the small doses given in a

Answer 17

Phase 0 study

Question 18

While taking part in a phase 0 study, patients are not allowed to enroll in a trial with

Answer 18

Therapeutic intent

Question 19

Studies ADME: Absorption, Distribution, Metabolism, and Excretion

Answer 19

Phase I trials

Question 20

Performed on normal volunteers, in patients with target disease, in subpopulations (elderly, liver impairment) and in common clinical settings (food effects, drug interactions) (20-100 individuals)

Answer 20

Phase I trials

Question 21

Phase I trials have dose escalation designs to determine

Answer 21

Maximum Tolerated Dose (MTD)

Question 22

Fibonacci dose escalations are often used in

Answer 22

Phase I trials

Question 23

Classified as therapeutic exploratory studies

Answer 23

Phase II studies

Question 24

Classified as human pharmacology studies

Answer 24

Phase 0 and I studies

Question 25

Explore efficacy and safety in patients with target disease (100-1000 individuals)

• Estimate dosage for definitive studies

Answer 25

Phase II studies

Question 26

Explores endpoints and study methodologies for definitive studies

Answer 26

Phase II studies

Question 27

Called therapeutic confirmatory studies

Answer 27

Phase III studies

Question 28

Used to confirm efficacy and safety profile in target population

-Provide adequate basis for assessing benefit- risk relationship to support regulatory approval

Answer 28

Phase III studies

Question 29

Provide adequate basis for assessing benefit- risk relationship to support regulatory approval

Answer 29

Phase III studies

Question 30

Following successful completion of clinical trials the investigators submit a

Answer 30

NDA: New Drug Application

Question 31

Done to determine if a drug or treatment is safe over time, or to see if a treatment or medication can be used in other circumstances

Answer 31

Phase IV clinical trials

Question 32

Done after a treatment has gone through phase I, phase II and phase III trials, and is already approved by the FDA. This is called post market surveillance

Answer 32

Phase IV clinical trials

Question 33

Were created as an alternative to non- selective NSAIDs to decrease stomach, kidney and gastrointestinal problems commonly associated with these drugs

Answer 33

COX2 inhibitors

Question 34

The mechanism of action of Vioxx and other drugs in the same class is believed to be through strictly
inhibiting cyclooxygenase 2, the enzyme that is primarily responsible for

Answer 34

Inflammation and pain

Question 35

Inhibiting strictly COX 2 enzymes creates an imbalance in favor of COX 1 enzymes resulting in a greater likelihood for

Answer 35

Platelet aggregation and endothelial constriction

Question 36

The only remaining COX2 inhibitor available because of lower specificity for COX2 over COX1

Answer 36

Celebrex/Celecoxib