MCQ Quiz: New Drug Approval

The journey of a new drug from laboratory discovery to the pharmacy shelf is a long, complex, and highly regulated process overseen by the U.S. Food and Drug Administration (FDA). Understanding this new drug approval process, a key topic in the Principles of Medicinal Chemistry and Pharmacology I course, is essential for pharmacists to critically evaluate new therapies as they enter the market. This quiz will test your knowledge on the different phases of clinical trials, the types of regulatory applications, and the foundational laws that ensure the safety and efficacy of our medications.

1. Before a new drug can be tested in humans, a manufacturer must submit what type of application to the FDA?

• a. New Drug Application (NDA)
• b. Investigational New Drug (IND) application
• c. Abbreviated New Drug Application (ANDA)
• d. Biologics License Application (BLA)

Answer: b. Investigational New Drug (IND) application

2. What is the primary goal of a Phase 1 clinical trial?

• a. To confirm efficacy in a large patient population.
• b. To assess the safety, tolerability, and pharmacokinetics of a drug in a small group of healthy volunteers.
• c. To compare the new drug to the current standard of care.
• d. To conduct post-marketing surveillance.

Answer: b. To assess the safety, tolerability, and pharmacokinetics of a drug in a small group of healthy volunteers.

3. The efficacy of a new drug (“does it work?”) is first established in which phase of clinical trials?

• a. Phase 1
• b. Phase 2
• c. Phase 3
• d. Phase 4

Answer: b. Phase 2

4. The large, pivotal, multicenter studies that are used to confirm a drug’s safety and efficacy against a comparator (like placebo) are conducted in which phase?

• a. Phase 1
• b. Phase 2
• c. Phase 3
• d. Phase 4

Answer: c. Phase 3

5. A pharmaceutical company submits a New Drug Application (NDA) to the FDA:

• a. Before starting any human trials.
• b. After completing Phase 1 trials.
• c. After completing Phase 2 trials.
• d. After completing Phase 3 trials and analyzing the data.

Answer: d. After completing Phase 3 trials and analyzing the data.

6. The “Drug Approval Process” is a specific lecture in which course?

• a. PHA5439 Principles of Medicinal Chemistry and Pharmacology I
• b. PHA5104 Sterile Compounding
• c. PHA5703 Pharmacy Law and Ethics
• d. PHA5787C Patient Care 5

Answer: a. PHA5439 Principles of Medicinal Chemistry and Pharmacology I

7. A generic drug manufacturer wishing to market their product must submit a(n):

• a. NDA
• b. IND
• c. BLA
• d. ANDA (Abbreviated New Drug Application)

Answer: d. ANDA (Abbreviated New Drug Application)

8. To gain approval, a generic drug must be proven to be what to the brand-name product?

• a. Bioequivalent
• b. Therapeutically superior
• c. Chemically different
• d. Less expensive

Answer: a. Bioequivalent

9. The Kefauver-Harris Amendment of 1962 was a landmark law that required manufacturers to prove what before marketing?

• a. Safety only
• b. Purity only
• c. Both safety and efficacy
• d. That the drug was affordable

Answer: c. Both safety and efficacy

10. Post-marketing surveillance studies, which collect data on a drug’s use in the general population after it is approved, are known as:

• a. Phase 1 studies
• b. Phase 2 studies
• c. Phase 3 studies
• d. Phase 4 studies

Answer: d. Phase 4 studies

11. The design of randomized controlled trials is a key topic in the Principles of Evidence-Based Practice course.

• a. True
• b. False

Answer: a. True

12. The FDA’s “Orange Book” is a resource used to identify:

• a. Investigational new drugs.
• b. Drugs with black box warnings.
• c. Approved drug products and their therapeutic equivalence ratings.
• d. Compounding formulas.

Answer: c. Approved drug products and their therapeutic equivalence ratings.

13. A drug that receives “Breakthrough Therapy” designation from the FDA is one that:

• a. Is intended to treat a serious condition and preliminary evidence indicates it may be a substantial improvement over available therapy.
• b. Has no side effects.
• c. Has completed all clinical trials.
• d. Is a new generic drug.

Answer: a. Is intended to treat a serious condition and preliminary evidence indicates it may be a substantial improvement over available therapy.

14. A pharmacist’s role in the new drug approval process can include:

• a. Serving as a clinical trial investigator.
• b. Working for the FDA as a reviewer.
• c. Working for a pharmaceutical company in regulatory affairs.
• d. All of the above.

Answer: d. All of the above.

15. Evaluating relevant primary literature, such as a clinical trial, is a key objective for student pharmacists.

• a. True
• b. False

Answer: a. True

16. A “Biologics License Application” (BLA) is submitted for which type of product?

• a. A generic small molecule drug.
• b. A dietary supplement.
• c. A monoclonal antibody or vaccine.
• d. A new medical device.

Answer: c. A monoclonal antibody or vaccine.

17. The “Principles of Pharmacy Law and Ethics” course provides the legal foundation for the drug approval process.

• a. True
• b. False

Answer: a. True

18. “Accelerated Approval” can be granted by the FDA based on a drug’s effect on:

• a. A surrogate endpoint that is reasonably likely to predict clinical benefit.
• b. A hard clinical outcome like mortality.
• c. The cost of the medication.
• d. The manufacturer’s marketing plan.

Answer: a. A surrogate endpoint that is reasonably likely to predict clinical benefit.

19. What is the primary purpose of an Institutional Review Board (IRB)?

• a. To approve the final marketing of a drug.
• b. To protect the rights and welfare of human subjects participating in a research study.
• c. To analyze the statistical data from a trial.
• d. To secure funding for a clinical trial.

Answer: b. To protect the rights and welfare of human subjects participating in a research study.

20. The “How New Drugs are Developed” module is part of the Patient Care 5 course.

• a. True
• b. False

Answer: b. False

21. A major reason for a drug to fail in clinical trials is:

• a. Lack of efficacy
• b. Unacceptable safety profile
• c. Both a and b
• d. Neither a nor b

Answer: c. Both a and b

22. “Pre-clinical” studies involve testing a new drug candidate in:

• a. Healthy human volunteers.
• b. Patients with the target disease.
• c. Computer models and animal models.
• d. The general population after approval.

Answer: c. Computer models and animal models.

23. The “Experimental Studies” module, covering RCTs, is part of the EBP course.

• a. True
• b. False

Answer: a. True

24. A “biosimilar” is a biologic product that is highly similar to an already-approved reference biologic. It is approved via a(n):

• a. NDA
• b. ANDA
• c. BLA
• d. Abbreviated BLA or similar pathway.

Answer: d. Abbreviated BLA or similar pathway.

25. A pharmacist is responsible for staying up-to-date on new drug approvals.

• a. True
• b. False

Answer: a. True

26. The Food, Drug, and Cosmetic Act of 1938 was passed after a tragedy involving:

• a. An elixir of sulfanilamide made with a toxic solvent.
• b. The thalidomide disaster.
• c. A contaminated vaccine.
• d. An adulterated patent medicine.

Answer: a. An elixir of sulfanilamide made with a toxic solvent.

27. The concept of “informed consent” is a critical ethical requirement for:

• a. Dispensing any prescription.
• b. Enrolling a patient in a clinical trial.
• c. Selling an OTC product.
• d. Compounding a medication.

Answer: b. Enrolling a patient in a clinical trial.

28. An active learning session on drug development is part of which course?

• a. PHA5439 Principles of Medicinal Chemistry and Pharmacology I
• b. PHA5163L Professional Skills Lab 3
• c. PHA5781 Patient Care I
• d. PHA5787C Patient Care 5

Answer: a. PHA5439 Principles of Medicinal Chemistry and Pharmacology I

29. The Prescription Drug User Fee Act (PDUFA) allows the FDA to:

• a. Set the prices of drugs.
• b. Collect fees from manufacturers to fund the drug approval process and speed up reviews.
• c. Regulate dietary supplements.
• d. Control the import of drugs.

Answer: b. Collect fees from manufacturers to fund the drug approval process and speed up reviews.

30. The “Medication Safety” module in Patient Care 5 covers post-marketing surveillance.

• a. True
• b. False

Answer: a. True

31. In a double-blind, randomized controlled trial:

• a. Only the investigator knows the treatment assignment.
• b. Only the patient knows the treatment assignment.
• c. Neither the patient nor the investigator knows the treatment assignment.
• d. Everyone knows the treatment assignment.

Answer: c. Neither the patient nor the investigator knows the treatment assignment.

32. A drug’s “package insert” is:

• a. A marketing brochure.
• b. An FDA-approved legal document that summarizes the safe and effective use of a drug.
• c. A guide for patients only.
• d. A list of all possible off-label uses.

Answer: b. An FDA-approved legal document that summarizes the safe and effective use of a drug.

33. The primary role of the FDA ends once a drug is approved and marketed.

• a. True
• b. False

Answer: b. False

34. A “therapeutic equivalence” code of “AB” in the Orange Book means that:

• a. The generic product has a potential bioequivalence issue.
• b. The generic product is considered therapeutically equivalent to the brand-name product.
• c. The product is a biologic.
• d. The product is not FDA approved.

Answer: b. The generic product is considered therapeutically equivalent to the brand-name product.

35. A pharmacist can contribute to post-marketing surveillance by:

• a. Reporting adverse drug reactions to the FDA’s MedWatch program.
• b. Ignoring patient complaints about side effects.
• c. Only dispensing brand-name drugs.
• d. Prescribing off-label uses.

Answer: a. Reporting adverse drug reactions to the FDA’s MedWatch program.

36. A drug that treats a rare disease affecting fewer than 200,000 people in the U.S. may receive what designation?

• a. Fast Track
• b. Breakthrough Therapy
• c. Orphan Drug
• d. Generic Drug

Answer: c. Orphan Drug

37. The Purple Book is a resource for identifying:

• a. Approved generic drugs.
• b. Licensed biological products and their biosimilars.
• c. Drugs with REMS programs.
• d. Compounding ingredients.

Answer: b. Licensed biological products and their biosimilars.

38. The lecture “The Drug Approval Process” is part of which course module?

• a. Module 3: How New Drugs are Developed
• b. Module 1: Relationships of Functional Groups to Pharmacological Activity
• c. Module 4: Drug Biotransformation
• d. Module 5: Prodrugs and Soft Drugs

Answer: a. Module 3: How New Drugs are Developed

39. A “black box warning” is the FDA’s most stringent warning for drugs and appears on the package insert.

• a. True
• b. False

Answer: a. True

40. An active learning session covering drug development is part of which course?

• a. PHA5439 Principles of Medicinal Chemistry and Pharmacology I
• b. PHA5163L Professional Skills Lab 3
• c. PHA5781 Patient Care I
• d. PHA5787C Patient Care 5

Answer: a. PHA5439 Principles of Medicinal Chemistry and Pharmacology I

41. The primary goal of a Phase 2 trial is:

• a. To assess safety in healthy volunteers.
• b. To determine the drug’s efficacy and dose range in a small group of patients with the disease.
• c. To confirm safety and efficacy in a large-scale trial.
• d. To monitor for long-term side effects.

Answer: b. To determine the drug’s efficacy and dose range in a small group of patients with the disease.

42. Which of the following is NOT required in an IND application?

• a. Animal pharmacology and toxicology data.
• b. Manufacturing information.
• c. Clinical protocols for human studies.
• d. Data from a completed Phase 3 trial.

Answer: d. Data from a completed Phase 3 trial.

43. A pharmacist’s knowledge of the drug approval process helps them to:

• a. Understand the strength of evidence supporting a drug’s use.
• b. Critically evaluate new therapies as they become available.
• c. Answer patient questions about new drugs.
• d. All of the above.

Answer: d. All of the above.

44. A major focus of the “Principles of Evidence-Based Practice” course is the appraisal of clinical trials.

• a. True
• b. False

Answer: a. True

45. The term “off-label” use means a drug is being prescribed for an indication not approved by the FDA.

• a. True
• b. False

Answer: a. True

46. A drug company cannot legally market its drug for an off-label use.

• a. True
• b. False

Answer: a. True

47. The FDA drug approval process is designed to balance:

• a. The manufacturer’s profit with the pharmacy’s profit.
• b. The need to make new, effective treatments available to the public with the need to ensure they are safe.
• c. The speed of approval with the cost of the drug.
• d. The ease of administration with the drug’s taste.

Answer: b. The need to make new, effective treatments available to the public with the need to ensure they are safe.

48. An active learning session on drug development is part of which course module?

• a. Module 3: How New Drugs are Developed
• b. Module 1: Relationships of Functional Groups to Pharmacological Activity
• c. Module 4: Drug Biotransformation
• d. Module 5: Prodrugs and Soft Drugs

Answer: a. Module 3: How New Drugs are Developed

49. The overall management of the drug pipeline from discovery to post-marketing is overseen by the:

• a. The pharmaceutical company
• b. The FDA
• c. The DEA
• d. The Board of Pharmacy

Answer: b. The FDA

50. The ultimate goal of a rigorous new drug approval process is to:

• a. Make drugs more expensive.
• b. Protect the public health.
• c. Limit the number of available medications.
• d. Make it difficult for companies to get drugs approved.

Answer: b. Protect the public health.