Introduction to drug discovery and development MCQs With Answer

Introduction to drug discovery and development MCQs With Answer

Drug discovery and development is a multidisciplinary process that transforms biological insights into safe, effective medicines. This concise MCQ set for B.Pharm students covers key concepts: target identification, high-throughput screening, lead optimization, medicinal chemistry, ADME/Tox, pharmacokinetics, pharmacodynamics, preclinical studies, clinical trial phases, regulatory affairs, formulation, and intellectual property. Emphasis on techniques such as in silico modeling, SAR/QSAR, DMPK studies, safety pharmacology, and bioavailability prepares students for real-world drug R&D and pharmaceutical practice. These questions reinforce critical thinking about experimental design, data interpretation, and regulatory requirements central to modern drug development. Now let’s test your knowledge with 30 MCQs on this topic.

Q1. Which stage of drug discovery focuses on validating a biological target linked to disease pathology?

• Lead optimization
• Target identification and validation
• Clinical trials
• Formulation development

Correct Answer: Target identification and validation

Q2. High-throughput screening (HTS) is primarily used to:

• Determine pharmacokinetic parameters in volunteers
• Rapidly test large compound libraries against a target
• Optimize drug formulation for bioavailability
• Conduct long-term toxicity studies in animals

Correct Answer: Rapidly test large compound libraries against a target

Q3. Which technique is used to predict relationships between chemical structure and biological activity?

• Mass spectrometry
• SAR/QSAR modeling
• Chromatography
• Blinding in clinical trials

Correct Answer: SAR/QSAR modeling

Q4. Lipinski’s Rule of Five helps to assess:

• In vivo toxicity in rodents
• Drug-likeness and oral bioavailability
• Stability of formulations
• Regulatory submission format

Correct Answer: Drug-likeness and oral bioavailability

Q5. Which parameter describes the rate at which a drug is removed from the body?

• Volume of distribution (Vd)
• Clearance (Cl)
• Bioavailability (F)
• Partition coefficient (Log P)

Correct Answer: Clearance (Cl)

Q6. First-pass metabolism primarily affects a drug after which route of administration?

• Intravenous
• Oral
• Inhalation
• Topical

Correct Answer: Oral

Q7. Which cytochrome P450 enzyme is most commonly involved in drug metabolism and drug–drug interactions?

• CYP1A2
• CYP2D6
• CYP3A4
• CYP2C19

Correct Answer: CYP3A4

Q8. The primary objective of Phase I clinical trials is to:

• Demonstrate long-term efficacy in large populations
• Assess safety, tolerability, and pharmacokinetics in healthy volunteers
• Compare to standard therapy in randomized trials
• Obtain marketing approval

Correct Answer: Assess safety, tolerability, and pharmacokinetics in healthy volunteers

Q9. NOAEL in preclinical toxicology stands for:

• Number Of Adverse Events Listed
• No Observed Adverse Effect Level
• New Observational Animal Experiment Limit
• Normal Organ Activity Evaluation Limit

Correct Answer: No Observed Adverse Effect Level

Q10. Which regulatory document must a sponsor submit to begin human clinical trials in many countries?

• New Drug Application (NDA)
• Investigational New Drug (IND) application
• Biologics License Application (BLA)
• Clinical Study Report (CSR)

Correct Answer: Investigational New Drug (IND) application

Q11. A prodrug is designed to:

• Be active without metabolic conversion
• Enhance patient compliance by pleasant taste only
• Undergo metabolic conversion to an active form
• Reduce the manufacturing cost

Correct Answer: Undergo metabolic conversion to an active form

Q12. The therapeutic index (TI) is best described as:

• Ratio of maintenance dose to loading dose
• Difference between Cmax and Tmax
• Ratio of toxic dose to effective dose
• Measure of drug solubility

Correct Answer: Ratio of toxic dose to effective dose

Q13. Which study determines how formulation affects rate and extent of absorption for orally administered drugs?

• Bioequivalence study
• Preclinical safety study
• Phase I efficacy trial
• Stability study under ICH guidelines

Correct Answer: Bioequivalence study

Q14. hERG channel inhibition is primarily associated with which safety concern?

• Hepatotoxicity
• QT interval prolongation and cardiotoxicity
• Nephrotoxicity
• Teratogenicity

Correct Answer: QT interval prolongation and cardiotoxicity

Q15. Which in vitro model is commonly used to predict intestinal absorption?

• HepG2 hepatocyte monolayer
• Caco-2 cell monolayer
• Renal proximal tubule slice
• Cardiomyocyte contraction assay

Correct Answer: Caco-2 cell monolayer

Q16. Structure-based drug design typically requires which information?

• Target gene expression data only
• Crystal structure or 3D model of the target
• Patient-reported outcomes
• Regulatory guidelines for marketing

Correct Answer: Crystal structure or 3D model of the target

Q17. Which classification divides drugs based on solubility and intestinal permeability?

• BCS — Biopharmaceutics Classification System
• ATC — Anatomical Therapeutic Chemical
• ICH — International Council for Harmonisation
• EMA — European Medicines Agency

Correct Answer: BCS — Biopharmaceutics Classification System

Q18. In lead optimization, which property is NOT typically optimized?

• Potency at the target
• ADME profile
• Synthetic accessibility and selectivity
• Clinical trial sample size

Correct Answer: Clinical trial sample size

Q19. Which guideline set focuses on harmonizing safety, quality, and efficacy requirements internationally?

• GMP only
• ICH guidelines
• USP monographs
• Pharmacopoeial standards only

Correct Answer: ICH guidelines

Q20. Bioavailability (F) is defined as:

• Fraction of unchanged drug eliminated by kidneys
• Fraction of administered dose reaching systemic circulation
• Fraction of drug bound to plasma proteins
• Fraction of drug excreted in feces

Correct Answer: Fraction of administered dose reaching systemic circulation

Q21. Which is the correct order of clinical phases primarily concerned with safety then efficacy?

• Phase III → Phase II → Phase I
• Phase I → Phase II → Phase III
• Phase II → Phase I → Phase III
• Phase I → Phase III → Phase II

Correct Answer: Phase I → Phase II → Phase III

Q22. A randomized, double-blind, placebo-controlled trial is important because it:

• Ensures all participants receive the active drug
• Minimizes bias and placebo effects to assess true efficacy
• Eliminates the need for statistical analysis
• Is required only for herbal products

Correct Answer: Minimizes bias and placebo effects to assess true efficacy

Q23. DMPK studies primarily evaluate:

• Drug manufacture scalability
• Drug metabolism, pharmacokinetics, and distribution
• Patient adherence patterns
• Legal patentability

Correct Answer: Drug metabolism, pharmacokinetics, and distribution

Q24. Which approach uses computer simulations to design or screen drug candidates?

• In vivo toxicology
• In silico modeling
• Clinical pharmacology
• Good Manufacturing Practice (GMP)

Correct Answer: In silico modeling

Q25. Orphan drug designation is granted to therapies that:

• Treat very common conditions
• Address rare diseases with small patient populations
• Are natural supplements without clinical data
• Are generic copies of existing drugs

Correct Answer: Address rare diseases with small patient populations

Q26. Which is the primary reason for conducting GLP-compliant studies in preclinical research?

• To speed up manufacturing processes
• To ensure data quality, integrity, and regulatory acceptance
• To optimize product packaging
• To market products directly to consumers

Correct Answer: To ensure data quality, integrity, and regulatory acceptance

Q27. Which outcome is most appropriate to demonstrate proof-of-concept in early clinical development?

• Regulatory approval
• Signal of clinical activity and mechanistic biomarker change
• Large-scale manufacturing validation
• Patent grant

Correct Answer: Signal of clinical activity and mechanistic biomarker change

Q28. Which type of assay directly measures target enzyme inhibition in a purified system?

• Cell-based functional assay
• Biochemical (enzyme) assay
• Clinical endpoint study
• Stability-indicating assay

Correct Answer: Biochemical (enzyme) assay

Q29. Patent protection in pharmaceuticals is important because it:

• Ensures the drug is free from side effects
• Provides exclusive marketing rights to recoup R&D investment
• Removes the need for clinical trials
• Guarantees reimbursement by insurers

Correct Answer: Provides exclusive marketing rights to recoup R&D investment

Q30. Which metric best indicates how quickly a drug reaches peak plasma concentration?

• Half-life (t1/2)
• Tmax
• Volume of distribution (Vd)
• Clearance (Cl)

Correct Answer: Tmax

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