1. An investigational new drug(IND) is not required for which of the following?

a) Studies examining a different indication

b) Initiation of clinical studies on a new drug
c) Marketed drugs where the study would use approved doses
d) Studies examining a new route of administration

2.When an investigational new drug(IND) application is “in effect” what does it permit a sponsor to
do?

a) Begin studies in animals

b) Begin studies in humans
c) Set the price of drug
d) Market the drug

3. Which of the following is FALSE statement regarding IND safety reporting?

a) For a fatal or life-threatening AE, 1 calendar day to report

b) For a serious or unexpected AE, 15 calendar days to report
c) For a serious or unexpected AE, written notification must be sent to the FDA and investigators
d) For a fatal or life-threatening AE, must notify FDA via phone or fax

4. Which kind of drugs take comparatively longer time to develop?

a) Oncolytics
b) Biologics
c) Small molecular entities

5. According to the lecture, how many drugs reach the pre-clinical stage if a company starts with -
5000-10,000 lead compounds in pre-discovery stage?

a) 50
b) 250
c) 1000
d) 8000

6. Which countries in the EU have more stringent and scientifically rigorous rules to go through to
get approved by EMA? (Choose 2)

a) France
b) Germany
c) Italy
d) Sweden

7. Which can be potential decisions of an IND review? (Choose 2)

a) IND is “in effect” on day 21 if no negative comments are received from FDA
b) Clinical hold may be put on IND at any point of time
c) IND may not get approved by FDA
8. You have developed inclusion/exclusion(I/E) criteria for a clinical study. Subject enrollment is
slow. Based on regulatory guidelines, are you allowed to revise I/E criteria during the course of the
study?

a) No, you are not allowed to revise I/E criteria

b) Yes, you can revise I/E criteria

9. All of the following are required elements of the informed consent from (IFC) EXCEPT

a) Information about how a drug works

b) Statistical method used in study
c) Study purpose
d) Potential risk and benefits

10. All of the following are required elements of protocol EXCEPT

a) Defining study inclusion/exclusion criteria

b) Requirements to be the study’s primary investigator
c) Identifying study objectives
d) Background of target rationale

11. Which of the following are described as the key objectives of a Phase I trail, regardless of drug?

a) Characterize the pharmachokinetic (PK) profile

b) Assess safety and tolerability
c) Assess appropriateness of stopping rules
d) Determine inclusion/exclusion recommendations

12. What does FIH stand for?

a) Foundational institute of health

b) Fast in host
c) First in human

13. Which of the following are the 3 most important to look at when considering our
inclusion/exclusion criteria?

a) The potential side effect profile

b) The mechanism of the drug
c) The potential for drug interaction
d) The potential for drug abuse

14. True/false: It is required to have an IND before your Phase I trail?

a) False
b) True

15. Drug X is an investigational agent for high blood pressure. Which of the following study designs
would be appropriate for a first in human (FIH) Phase I study?

a) An open label, multiple doses in patients with high blood pressure

 b) An open label, single dose in patients with high blood pressure
c) A placebo- controlled, single dose in healthy adults
d) A placebo- controlled, multiple doses in healthy adults

16. Which pharmacokinetic parameter will allow you to determine drug exposure?

a) AUC (area under the concentration versus time curve)

b) CL (plasma clearance)
c) t1/2 (elimination half-life)
d) Tmax (time to maximum concentration)

17. What is the primary objective of a Phase I study?

a) To study drug efficacy

b) To determine drug safety and tolerability
c) To determine biologic activity of a drug
d) To determine drug manufacturing needs

18. Which 3 people were mentioned as those likely to get together to identify a starting dosage
level?

a) The clinical scientist

b) The toxicologist
c) The pharmacokinetics scientist
d) The FDA expert

19. Normally first in human studies are conducted in heathy volunteers except (choose 2)

a) Mentally challenged patients

b) HIV patients
c) Oncology patients

20. Which of the following was described as the easiest and most important changes that could
have improved the Tegenero Phase I study?

a) Changing the study design

b) Being close to an ICU
c) Changing the starting dosage
d) Staggering timing of dosage

21. What is the primary objective of a Phase 2b study?

a) Determine a drug’s safety and tolerability profile

b) Establish drug dose ranges for subsequent studies
c) To establish bioequivalence of a drug
d) Determine drug pharmacokinetics

22.What would be the most optimal drug to develop based on the therapeutic index?

a) Drug with a narrow therapeutic index

b) Drug with a therapeutic index ratio less than 1
 c) Drug with a therapeutic index ration equal to 0
d) Drug with a therapeutic index ratio of greater than 10

23. Which of the following outcome from a Phase 2 trail would result in continuation to Phase 3?

a) Determining therapeutic dose to be 50 grams

b) Discovery of an irreversible, serious adverse event
c) Placebo providing statistically significant efficacy
d) Establishing the therapeutic dose of a drug

24.Which were two of the things in Phase 2 trails that contribute to greater confidence in your
compound?

a) Therapeutic index
b) Simulation
c) Use in humans
d) Mathematical modelling

25. What is illustrated by the difference between the therapeutic effect and the toxic effect?

a) Therapeutic index
b) Efficacy plateau
c) Dosage rate
d) Hill curve

26. All of the following are design features of a Phase 3 study except?

a) Performance in final dosage form

b) Open- label study
c) Multi- center sites
d) Large sample size

27. All of the following are common causes of study delays EXCEPT:

a) Legal review
b) Marketing authorization
c) Patient recruitment
d) Earlier safety data

28. Which of the follow were stated as benefits of medical research? (Choose 3)

a) Informational changes to pharmaceutical research

b) Development of appropriate costs for future clinical studies
c) Reduction in total burden of chronic disease
d) Impact on disease prevention

29. Historically drug commercialization was more ________ and today it is more ________.

a) US- driven, global

b) Linear, parallel
c) Global, US- driven
 d) Parallel, linear

30. The current estimate of the cost of the full drug development cycle was stated as being in which
range

a) <500 million
b) 500 million to 1 billion
c) 1-1.5 billion
d) 1.5-2 billion

31. Considerations of effect on varying populations usually occurs in which stage trial

a) 2
b) 3
c) 4

32. Why is stage 3 study design and execution so important?

a) Ability to commercialize
b) Evaluation of efficacy
c) Assurance of safety

33. All of the following are required for a U.S FDA Drug Application EXCEPT:

a) Human bioavailability
b) Preclinical data
c) Packaging methods
d) Disclosure of intent to file in other countries

34. All of the following are examples of post-marketing studies EXCEPT:

a) Safety surveillance study

b) Study examining effectiveness in widespread populations
c) Proof of principle study
d) Studies of new users of drug

35. True or false: When a new drug application is submitted by company X, that is public to company
Y

a) True
b) False

36. The two characteristics for fast-track approvals are which

a) An advance on existing treatment

b) Treats a serious illness
c) Fills an unmet need

38. Increased in interest in pharmacoeconomic data was described as coming from where

a) The FDA looking to prioritize future application processing

b) Insurance companies wanting to determine if there would be a cheaper standard of care
c) Pharmaceutical companies wanting to build better models to guide drug development