Lecture 2

Question 1

Pharmacy

Answer 1

The science of preparing, compounding, and dispensing substances for
medicinal use.

Question 2

Symptom

Answer 2

Effect reported by the patient

Question 3

Sign

Answer 3

Effect observed by the clinician

Question 4

Palliative

Answer 4

Drug used to relieve symptoms

Question 5

Therapeutic

Answer 5

Drug used to cure disease

Question 6

Prophylactic

Answer 6

Drug used to prevent disease

Question 7

Toxicity

Answer 7

Any adverse effect of a xenobiotic on a biological system

Question 8

Xenobiotic

Answer 8

A molecule that is foreign to the organism. From the Greek xeno (ξένο)
for “foreign” and bios (βίος) for “life”.

Question 9

Hazard

Answer 9

The likelihood that a xenobiotic will cause toxicity at a specific dose or
exposure level.

Question 9

Risk

Answer 9

A quantitative measure of hazard

Question 9

Safety

Answer 9

The reciprocal of hazard; the likelihood that toxicity will not occur.

Question 10

Dose Formula

Answer 10

μg/kg

Question 11

Dose Rate Formula

Answer 11

μg/kg-day

Question 12

IND Application Process Definition

Answer 12

IND (Investigational New Drug) is a new drug that is proposed to
be used in a clinical trial.

Question 13

What does the IND contain?

Answer 13

– Data on chemistry, manufacturing, and control.
– Pre-clinical safety information.
– Description of proposed first human trial. (Mainly used to decide what dose would be used).

Question 14

Blind Trial

Answer 14

– Patient doesn’t know if getting drug or “placebo”.

Question 15

Double Blind Trial

Answer 15

– Patient, doctor, and investigator don’t know if patient is getting
drug or placebo.

Question 16

Phase 1 Trials

Answer 16

• “First-in-human” study.
• Usually 20-50 healthy volunteers.
• Several months in length.

Question 17

Phase 1 Trials Objectives

Answer 17

– Focus on toxicity (tolerated dose range).
– Determine metabolic and excretory pathways.
– Assess variability between individuals; effect of route of
exposure; bioavailability.

Question 18

Phase 2 Trials

Answer 18

“Proof-of-concept” phase.
* Usually 150-350 people with disease.
* Several months to two years in length.
* Often includes patients for whom other treatments have
failed.

Question 19

Phase 2 Trials Objectives

Answer 19

– Focus on efficacy. (will it work?)
– Perform pharmacokinetic studies in diseased people.
– Identify nature and severity of side effects.
– Dose Range: not everyone gets the same dose.

Question 20

Phase 3 Trials

Answer 20

• Several hundred to several thousand patients with disease.
• Often multicenter, replicated; length up to 1 – 4 years.
• Most expensive component of drug development.

Question 21

Phase 3 Trials Objectives

Answer 21

– Focus on safety, dosage, efficacy.
– Identify interactions between drugs in the larger population.
– Supports NDA.

Question 22

NDA

Answer 22

New Drug Application (NDA)
NDA is an application for marketing the drug.
* NDAs are submitted for:
– New molecular entities.
– New formulation of previously approved drug.
– New combination of two or more drugs.
– New indication (claim) for already marketed drug.
* 50-400 volumes (30,000-150,000 pages)

Question 23

Toxicologists deal with two types of responses

Answer 23

Continuous (graded; observed in an individual). (Can measure it on a continuous scale such as BP change.)
– Quantal (all-or-none; observed in a population). (Dead or alive, cancer or no cancer)

Question 24

SR

Answer 24

Substance receptor complex (What drives the response)