Understanding how new drugs are developed is crucial for any Pharm.D. student, as it bridges the gap between pharmaceutical science and patient care. This MCQ quiz will challenge your knowledge of drug discovery, clinical trials, regulatory approval, and more, based on official Pharm.D. coursework such as PHA5176 (Drug Delivery Systems) and PHA5439 (Principles of Medicinal Chemistry and Pharmacology I).
1. What is the initial step in drug discovery?
- A. Filing a New Drug Application
- B. Identifying disease symptoms
- C. Target identification and validation
- D. Drug formulation
Correct answer: C. Target identification and validation
2. Which phase of clinical trials focuses on safety in healthy volunteers?
- A. Phase I
- B. Phase II
- C. Phase III
- D. Phase IV
Correct answer: A. Phase I
3. What is the main goal of preclinical testing?
- A. Establishing marketing strategy
- B. Testing drug on humans
- C. Evaluating safety and biological activity in animals
- D. Determining manufacturing costs
Correct answer: C. Evaluating safety and biological activity in animals
4. Which agency oversees the drug approval process in the United States?
- A. CDC
- B. DEA
- C. NIH
- D. FDA
Correct answer: D. FDA
5. Which application must be submitted before clinical trials can begin?
- A. ANDA
- B. NDA
- C. IND
- D. BLA
Correct answer: C. IND
6. Which of the following best defines ‘orphan drugs’?
- A. Drugs sold over the counter
- B. Drugs developed for rare diseases
- C. Herbal remedies
- D. Experimental nutrition supplements
Correct answer: B. Drugs developed for rare diseases
7. What is evaluated in Phase III clinical trials?
- A. Optimal dosage in animals
- B. Drug metabolism
- C. Efficacy and side effects in large populations
- D. Basic chemical structure
Correct answer: C. Efficacy and side effects in large populations
8. Which document is filed after successful clinical trials for marketing approval?
- A. Investigational New Drug
- B. Common Technical Document
- C. New Drug Application
- D. Drug Master File
Correct answer: C. New Drug Application
9. What does the Biopharmaceutics Classification System (BCS) categorize drugs by?
- A. Color and smell
- B. Solubility and permeability
- C. Route of administration
- D. Duration of action
Correct answer: B. Solubility and permeability
10. What act introduced the requirement for drug effectiveness in addition to safety?
- A. Pure Food and Drug Act
- B. FDA Modernization Act
- C. Durham-Humphrey Amendment
- D. Kefauver-Harris Amendment
Correct answer: D. Kefauver-Harris Amendment
11. Which dosage form provides the fastest drug absorption?
- A. Oral tablet
- B. IV injection
- C. Rectal suppository
- D. Transdermal patch
Correct answer: B. IV injection
12. What is the role of pharmacokinetics in drug development?
- A. Describes the drug’s marketing strategy
- B. Explains patient feedback
- C. Studies drug absorption, distribution, metabolism, and excretion
- D. Focuses only on drug safety
Correct answer: C. Studies drug absorption, distribution, metabolism, and excretion
13. What term describes a drug’s ability to produce a desired therapeutic effect?
- A. Efficacy
- B. Affinity
- C. Selectivity
- D. Potency
Correct answer: A. Efficacy
14. What phase comes immediately after FDA approval?
- A. Phase I trials
- B. Formulation development
- C. Post-marketing surveillance (Phase IV)
- D. IND filing
Correct answer: C. Post-marketing surveillance (Phase IV)
15. What is the purpose of pharmacovigilance?
- A. Drug pricing control
- B. Marketing drug brands
- C. Monitoring drug safety post-approval
- D. Optimizing supply chain
Correct answer: C. Monitoring drug safety post-approval
16. What term describes the minimum amount of drug required to produce an effect?
- A. Bioavailability
- B. Potency
- C. Efficacy
- D. Tolerance
Correct answer: B. Potency
17. What is the primary purpose of an ANDA (Abbreviated New Drug Application)?
- A. To approve a drug for rare disease
- B. To market a new biologic
- C. To approve a generic drug
- D. To approve a controlled substance
Correct answer: C. To approve a generic drug
18. Which of the following is not a key requirement of Good Manufacturing Practices (GMP)?
- A. Proper hygiene
- B. Sterile marketing techniques
- C. Documented procedures
- D. Quality control testing
Correct answer: B. Sterile marketing techniques
19. What does bioequivalence mean?
- A. Drug has identical cost
- B. Drug is a prodrug
- C. Drug has similar bioavailability and effect as a reference product
- D. Drug is a natural product
Correct answer: C. Drug has similar bioavailability and effect as a reference product
20. Which organization publishes guidelines on clinical trials internationally?
- A. WHO
- B. CDC
- C. ICH
- D. USP
Correct answer: C. ICH
21. In drug development, what does ‘hit-to-lead’ refer to?
- A. Transition from failed to approved compound
- B. Optimization of promising compounds for efficacy
- C. Target deactivation
- D. Pricing strategy development
Correct answer: B. Optimization of promising compounds for efficacy
22. What parameter determines the drug’s half-life?
- A. Volume of distribution and clearance
- B. Solubility and taste
- C. Route of administration
- D. Dosage form
Correct answer: A. Volume of distribution and clearance
23. What does ‘first-pass metabolism’ refer to?
- A. Drug reaching renal system first
- B. Metabolism in skin
- C. Liver metabolism before systemic circulation
- D. Drug bypassing liver
Correct answer: C. Liver metabolism before systemic circulation
24. What is the purpose of a placebo group in clinical trials?
- A. To administer more drug
- B. To test marketing responses
- C. To compare against active treatment
- D. To confuse patients
Correct answer: C. To compare against active treatment
25. What is a ‘prodrug’?
- A. Drug used in sports
- B. Inactive compound that becomes active in the body
- C. Drug that blocks metabolism
- D. Experimental nutritional aid
Correct answer: B. Inactive compound that becomes active in the body
26. What phase of trials typically involves thousands of patients?
- A. Phase I
- B. Phase II
- C. Phase III
- D. Phase IV
Correct answer: C. Phase III
27. What term refers to the ability of a compound to bind to its biological target?
- A. Potency
- B. Efficacy
- C. Affinity
- D. Bioavailability
Correct answer: C. Affinity
28. Which route of administration avoids first-pass metabolism?
- A. Oral
- B. Intravenous
- C. Sublingual
- D. Both B and C
Correct answer: D. Both B and C
29. What is the main goal of Phase II clinical trials?
- A. Assess drug’s long-term impact
- B. Test drug efficacy and side effects in diseased patients
- C. Compare drugs head-to-head
- D. Prepare dosage forms
Correct answer: B. Test drug efficacy and side effects in diseased patients
30. What does the term ‘lead optimization’ refer to?
- A. Changing lead researcher
- B. Improving pharmacological profile of a compound
- C. Packaging design
- D. Lowering cost of raw materials
Correct answer: B. Improving pharmacological profile of a compound
31. Which regulatory document outlines preclinical safety and toxicology?
- A. IND
- B. NDA
- C. ANDA
- D. BLA
Correct answer: A. IND
32. What is the significance of the orphan drug status granted by the FDA?
- A. Limits drug usage
- B. Enables advertising to children
- C. Provides incentives for rare disease drug development
- D. Requires global approval
Correct answer: C. Provides incentives for rare disease drug development
33. The purpose of the Institutional Review Board (IRB) is to:
- A. Approve marketing materials
- B. Develop chemical synthesis plans
- C. Ensure ethical treatment of human subjects
- D. Issue patents
Correct answer: C. Ensure ethical treatment of human subjects
34. In biopharmaceutics, what does ‘bioavailability’ refer to?
- A. Rate of biodegradation
- B. Drug cost efficiency
- C. Proportion of drug reaching systemic circulation
- D. Amount of drug in urine
Correct answer: C. Proportion of drug reaching systemic circulation
35. What is ‘QSAR’ used for in drug development?
- A. Quality synthesis analysis
- B. Quantitative structure-activity relationship modeling
- C. Quantifying standard assay results
- D. Quality strategy and action review
Correct answer: B. Quantitative structure-activity relationship modeling
36. What law mandated proof of drug efficacy before marketing?
- A. Durham-Humphrey Act
- B. Orphan Drug Act
- C. Kefauver-Harris Amendment
- D. Hatch-Waxman Act
Correct answer: C. Kefauver-Harris Amendment
37. What is the ‘therapeutic index’ a measure of?
- A. Dose required to treat infection
- B. Safety margin of a drug
- C. Bioequivalence
- D. Rate of drug approval
Correct answer: B. Safety margin of a drug
38. What is ‘in silico’ screening in drug development?
- A. Using laboratory animals
- B. Using plant extracts
- C. Computer-aided compound screening
- D. Clinical trial simulations
Correct answer: C. Computer-aided compound screening
39. Which technique is used to assess protein-ligand binding?
- A. PCR
- B. Western blot
- C. Surface plasmon resonance
- D. ELISA
Correct answer: C. Surface plasmon resonance
40. Why are pharmacogenomic considerations important in drug development?
- A. To lower costs
- B. To standardize treatments
- C. To individualize therapy based on genetic makeup
- D. To avoid natural products
Correct answer: C. To individualize therapy based on genetic makeup
41. What is the endpoint of most Phase III clinical trials?
- A. Animal safety profile
- B. Market exclusivity
- C. Statistical demonstration of efficacy and safety
- D. Generic substitution
Correct answer: C. Statistical demonstration of efficacy and safety
42. What does ‘compassionate use’ refer to in drug development?
- A. Using placebos
- B. Pricing for low-income patients
- C. Access to investigational drugs for seriously ill patients
- D. Discounted pharmacy programs
Correct answer: C. Access to investigational drugs for seriously ill patients
43. Which chemical property most affects a drug’s ability to cross cell membranes?
- A. Color
- B. Molecular weight
- C. Lipophilicity
- D. Smell
Correct answer: C. Lipophilicity
44. A clinical hold by the FDA means:
- A. Trial is paused for safety or protocol issues
- B. Drug is fast-tracked
- C. Marketing is allowed
- D. The drug is recalled
Correct answer: A. Trial is paused for safety or protocol issues
45. What is the final step in drug development before commercial launch?
- A. Formulation testing
- B. NDA approval
- C. Phase II trials
- D. Label design
Correct answer: B. NDA approval
46. What analytical method is used to assess drug purity?
- A. Microscopy
- B. High-performance liquid chromatography (HPLC)
- C. Immunohistochemistry
- D. SDS-PAGE
Correct answer: B. High-performance liquid chromatography (HPLC)
47. What term refers to the unwanted effects caused by a drug?
- A. Potency
- B. Bioavailability
- C. Adverse effects
- D. Efficacy
Correct answer: C. Adverse effects
48. How are biologics different from small molecule drugs?
- A. They are synthetic
- B. They are produced in living systems
- C. They are faster acting
- D. They are always oral
Correct answer: B. They are produced in living systems
49. The Hatch-Waxman Act primarily addresses:
- A. Herbal remedies
- B. Orphan drug policy
- C. Generic drug approval and patent extension
- D. International drug pricing
Correct answer: C. Generic drug approval and patent extension
50. What is the role of the USP in drug development?
- A. Conducts animal testing
- B. Regulates drug advertisements
- C. Sets quality standards for drugs and ingredients
- D. Approves drugs for market
Correct answer: C. Sets quality standards for drugs and ingredients