1. The Art of Drug Discovery

Question 1

First-in-class drug

Answer 1

targets a protein that has not been targeted before by another drug

Question 2

The FDA approved ____ novel drugs in ____, the fewest to pass regulatory scrutiny since 2016

Answer 2

37

2022

Question 3

Takes about ___ years for molecule to be approved

Answer 3

15

Question 4

_____ prescription drugs marketed in North America

Answer 4

1000

Question 5

Discovery/ Preclinical Testing

Answer 5

1. lab & animal studies
2. Asses safety, biological activity and formulations
3. 5000 compounds evaluated
4. 6.5 years

Question 6

Phase 1

Answer 6

1. 20-100 healthy volunteers
2. Determine safety and dosage
3. 5 compounds
4. 1.5 years

Question 7

Phase 2

Answer 7

1. 100-500 patient volunteers
2. Evaluate effectiveness & look for side effects
3. 5 compounds
4. 2 years

Question 8

Phase 3

Answer 8

1. 1000-5000 patient volunteers
2. Confirm effectiveness, monitor adverse reactions from long term use
3. 5 compounds
4. 3.5 years

Question 9

FDA review and approval

Answer 9

1. 1.5 years
2. one compound approved
3. review and approval process

Question 10

Phase 4

Answer 10

(whole process 15 years total)
1. additional post marketing testing required by FDA

Question 11

World Health Organization takes into account…

Answer 11

1. disease prevalence
   
   2. impact on disability-adjusted life years, DALYs
   3. public health, community health
   4. Evidence of efficacy
   5. evidence that the drug is safe

Question 12

Highest proportion of drugs improved in which therapeutic area?

Answer 12

1. Oncology
2. Infectious diseases
3. Neurology

Question 13

Who decides medical needs?

Answer 13

• FDA (US)
• Health Canada (Canada)

Question 14

Drug target

Answer 14

a molecule (usually a protein) involved in the disease, that the drug binds to, often inactivation the molecule

Question 15

What makes a protein “druggable”?

Answer 15

good protein structure

Question 16

Phase 1 Clinical Trials Purpose

Answer 16

–Determine a safe drug dose

Question 17

Pharmacokinetics

Answer 17

what happens to the drug in the body, how the drug moves in the body

Question 18

Phase 1 Clinical Trials Design

Answer 18

Usually dose-escalation. Start low, increase dose (Cautious)

Question 19

Phase 1 Clinical Trials Subjects

Answer 19

– Normal volunteers usually
– Sometimes patients with the disease (cancer)
– Must obtain informed consent from subjects

Question 20

Placebos

Answer 20

Placebos look like real pills but contain no active drug ingredient (controls for the “placebo effect”) is the expected response to taking a drug, (real brain circuitry involved)

Question 21

Phase 2 Clinical Trials Purpose

Answer 21

To obtain preliminary information on the efficacy of the drug

Question 22

Phase 2 Clinical Trials Design

Answer 22

Compare treated individuals to control individuals

Question 23

Phase 2 Clinical Trials Subjects

Answer 23

Patients with the disease

Question 24

Efficacy

Answer 24

does the drug work to treat the medical condition?

Question 25

Single-blind trials

Answer 25

subjects don’t know who is getting treatment and who is getting placebo

Question 26

Double-blind trials

Answer 26

neither subjects nor investigators know (Computer records – we ultimately do know who received which treatment)- MOST COMMON

Question 27

Phase 3 Clinical trials Purpose

Answer 27

To compare efficacy of the new treatment with the standard regimen

Question 28

Phase 3 Clinical trials Design

Answer 28

Usually randomized control

Question 29

Phase 3 Clinical trials Subjects

Answer 29

Patients with the disease

Question 30

Informed Consent

Answer 30

the process of understanding what you are agreeing (consent) to

Question 31

Generic drugs

Answer 31

same chemical, off-patent ie: Plan-B & contingency one

Question 32

Patent protection

Answer 32

20 years following filing (gives the manufacturer a monopoly for this amount of time- usually files for patent during discovery phase)

Question 33

Cost and time of developing a drug

Answer 33

Costs about >2 billion dollars & takes about 15 years