Unit 1: New Drug Development

Question 1

First testing tests for?

Answer 1

Toxicity

Question 2

3 Dose Levels in Toxicity Testing

Answer 2

— no-effect dose
— minimum lethal dose
— median lethal dose (LD50)

Question 3

Gold standard in evaluating drugs

Answer 3

Randomized Control Trial

Question 4

Phase 1 Purpose

Answer 4

Safety testing: do no harm

Question 5

Phase 1 trial size

Answer 5

20-100 healthy volunteers

Question 6

Phase 2 trial size

Answer 6

100-300 participants who have the disease

Question 7

Phase 2 purpose

Answer 7

Efficacy: will the drug work for the disease

Question 8

Phase 3 trial size

Answer 8

1000-3000 participants

Question 9

Phase 3 trial purpose

Answer 9

Minimize errors from placebo effects

Question 10

Phase 4 trial

Answer 10

Drug goes to market but continues to be monitored closely by the FDA

In first couple years, we will begin to see rare side effects that didn’t come up in phase 3

Question 11

Orphan Drug Amendment of ______

Answer 11

1983

Question 12

Orphan Drug Amendment of 1983

Answer 12

Incentives for development of drugs for treatment of rare disease which

1) affects < 200K in USA, OR
2) affects > 200K in USA but there is no reasonable expectation that the cost of development could be recovered from sales

Question 13

no-effect dose

Answer 13

maximum dose at which toxic effect not seen

Question 14

minimum lethal dose

Answer 14

smallest dose that will kill any experimental animal

Question 15

median lethal dose (LD50)

Answer 15

dose that kills approx 50% of animals in test group