Drug Discovery and Development (Raymond V. Oliva, MD)

Question 1

What was menoxidil originally designed for?

Answer 1

Treatment of colds (vasodilator)

However, it was eventually marketed as an anti-hypertensive.

Then, it was marketed to prevent baldness.

Question 2

T/F: Drugs can target more than one part of the complicated process.

Answer 2

False

Question 3

How long does it take to develop a new drug?

Answer 3

10 - 15 years

Question 4

What is the average cost of drug development?

Answer 4

$800 M - $1 B

Question 5

What do you call herbal products that were dropped from development before being approved?

Answer 5

Nutraceuticals

Question 6

Describe: Pre-discovery phase

Answer 6

Understanding the disease and its underlying cause

Question 7

What is avastin?

Answer 7

It’s a chemotherapeutic drug.

Question 8

Enumerate the ways of finding a new molecule

Answer 8

1. Nature
2. De Novo
3. High throughput screening
4. Biotechnology

Question 9

How are safety tests conducted?

Answer 9

1. Cells
2. Animals
3. Computation Models

These include PCR and HPLC

Question 10

What are CERA and epoietin beta for?

Answer 10

Treating chronic anemia

Question 11

How would you describe CERA’s binding affinity to its receptor?

Answer 11

Lower association

Question 12

What is lead optimisation?

Answer 12

The process of altering lead compounds to make them safer.

Question 13

What factors are considered in lead optimisation?

Answer 13

1. Formulation
2. Route of Administration
3. Large Scale Manufacturing

Question 14

What are the types of pre-clinical testing?

Answer 14

1. In vitro tests

2. In vivo tests

Question 15

Why is pre-clinical testing performed?

Answer 15

To ensure the drug is safe for humans

Question 16

How long do pre-clinical tests take on average?

Answer 16

1 - 5 years

Question 17

What is the first step in the drug developmental process?

Answer 17

Filing an Investigational New Drug (IND) before testing

Question 18

All clinical trials must be reviewed by which entity?

Answer 18

Investigational Review Board (IRB)

Question 19

What is the ideal clinical trial for drug testing?

Answer 19

Placebo-controlled trial
Randomized
Double-blinded

Question 20

What question is asked in Phase 1?

Answer 20

Is the treatment safe?

Question 21

What is the sample size for Phase 1?

Answer 21

N = 20 - 100

Question 22

Who are the subjects in a Phase 1 trial?

Answer 22

Healthy individuals

Question 23

What is the purpose of a Phase 1 trial?

Answer 23

Determine the safe dosing range

Question 24

How long does a Phase 1 trial usually take?

Answer 24

6 - 9 months

Question 25

What is the question asked in Phase 2?

Answer 25

Does the treatment work?

Question 26

Who are the subjects in a Phase 2 trial?

Answer 26

Diseased patients

Question 27

What is the sample size for a Phase 2 trial?

Answer 27

N = 100 - 500

Question 28

How long does a Phase 2 trial usually take?

Answer 28

6 months - 3 years

Question 29

What can you say about the doses included in a Phase 2 trial?

Answer 29

Presence of low and high dose and negative control It is concerned with the maximum therapeutic threshold.

Question 30

Phase IIA studies are also known as?

Answer 30

Proof of concept studies

Question 31

T/F: Phase IIA studies are well controlled clinical trials conducted to evaluate safety and efficacy in selected populations of patients.

Answer 31

False They are pilot clinical trials.

Question 32

What do objectives of Phase IIA studies focus on?

Answer 32

Dose-response Type of patient Dosing frequency

Question 33

What phase of clinical trials represents the most rigorous demonstration of a medicine's efficacy?

Answer 33

Phase IIB

Question 34

T/F: Phase IIB studies are sometimes referred to as pivotal studies.

Answer 34

True

Question 35

What are the questions asked in Phase III?

Answer 35

Will the treatment prolong life? | Does the new treatment work better than the standard treatment?

Question 36

What experimental design does Phase III utilise?

Answer 36

Randomized controlled trial (RCT)

Question 37

What is the sample size for Phase III studies?

Answer 37

N = 1000 - 5000 (except for cancer studies)

Question 38

What is the objective of a Phase III study?

Answer 38

To generate significant data on efficacy, safety and overall benefit-risk of the drug

Question 39

How long does a Phase III study usually take?

Answer 39

1 - 4 years of testing

Question 40

The Philippines is involved in several Phase III studies for what types of drugs?

Answer 40

Anti-hypertensive | Anti-diabetic

Question 41

Phase IIIA trials are conducted prior to submission of what to the FDA?

Answer 41

New Drug Application (NDA)

Question 42

Phase IIIA clinical trials are conducted in what kind of population?

Answer 42

The population for which the medicine is intended

Question 43

Trials of which phase provide much of the information contained in the package insert?

Answer 43

Phase IIIA

Question 44

T/F: Clinical trials for special groups or under special conditions are also conducted in Phase IIIA

Answer 44

True

Question 45

Can placebos be used on anti-asthmatic drugs?

Answer 45

No, but they can be used for drugs for diabetes mellitus

Question 46

When does Phase IIIb occur?

Answer 46

After submission of the NDA but prior to medicine's approval and launch

Question 47

Describe the nature of Phase IIIb trials

Answer 47

Supplement earlier trials Complete earlier trials Directed towards new types of trials or Phase IV evaluation

Question 48

With regards to manufacturing, a company must meet the FDA guidelines for what?

Answer 48

Good Manufacturing Practices (GMP)

Question 49

What is the question asked in Phase IV studies?

Answer 49

Is the treatment safe over time?

Question 50

What is the only approved COX-2 inhibitor in the US?

Answer 50

Celebrex

Question 51

What COX-2 inhibitor marketed by Merck & Co. was withdrawn from the market for allegedly causing CVDs?

Answer 51

Viox (Rofecoxib)

Question 52

What anti-diabetic drug marketed by GlaxoSmithKline was withdrawn from the market for allegedly causing heart failure and stroke?

Answer 52

Avandia (Rosiglitazone)

Question 53

T/F: A drug in Phase III trial cannot be given to pregnant women, children, insulin-dependent patients and patients with New York Heart Failure Classification 3 and 4

Answer 53

True

Question 54

How long do the drug discovery and pre-clinical stages take on average?

Answer 54

3 - 6 years collectively

Question 55

How long do Phases I - III of clinical trials take?

Answer 55

6 - 7 years collectively

Question 56

How long does an FDA review of a drug take?

Answer 56

0.5 - 2 years