Principles of drug discovery and development MCQs With Answer

Principles of Drug Discovery and Development MCQs With Answer is a focused resource designed for M.Pharm students preparing for exams and professional practice in product development and technology transfer. This set of multiple-choice questions covers core topics such as target identification, lead discovery and optimization, ADME/Tox, preclinical and clinical development stages, regulatory filings (IND/NDA), formulation and CMC considerations, and translational strategies to de-risk candidates. Questions emphasize mechanistic understanding, decision points in development, and practical aspects of technology transfer. Each MCQ includes concise options and a clear answer to help reinforce learning, guide revision, and build competence for real-world pharmaceutical development tasks.

Q1. Which early-stage activity primarily focuses on demonstrating that a biological molecule or pathway contributes to disease and is amenable to therapeutic modulation?

• Lead optimization
• Target identification and validation
• Clinical trial design
• Formulation development

Correct Answer: Target identification and validation

Q2. High-throughput screening (HTS) in lead discovery is mainly used to:

• Assess human pharmacokinetics
• Generate large numbers of chemical hits against a target
• Perform toxicology in two species
• Develop GMP manufacturing processes

Correct Answer: Generate large numbers of chemical hits against a target

Q3. During lead optimization, which property is least likely to be directly optimized through medicinal chemistry modifications?

• Potency at the biological target
• Solubility and permeability
• Intellectual property landscape
• Metabolic stability (intrinsic clearance)

Correct Answer: Intellectual property landscape

Q4. Which in vitro/in silico approach is most commonly used to predict potential drug–drug interactions mediated by cytochrome P450 enzymes?

• hERG channel binding assay
• Microsomal stability assay and CYP inhibition modeling
• In vivo rodent toxicology
• Solubility-pH profiling

Correct Answer: Microsomal stability assay and CYP inhibition modeling

Q5. The IND (Investigational New Drug) application primarily serves to:

• Obtain marketing approval to sell the drug
• Request permission to begin human clinical trials
• Register a manufacturing site for GMP inspection
• Secure patent protection for the molecule

Correct Answer: Request permission to begin human clinical trials

Q6. Which phase of clinical trials is chiefly designed to assess initial safety, tolerability, and pharmacokinetics in humans?

• Phase I
• Phase II
• Phase III
• Phase IV

Correct Answer: Phase I

Q7. In ADME characterization, which parameter best describes the fraction of an oral dose that reaches systemic circulation unchanged?

• Clearance (CL)
• Half-life (t1/2)
• Absolute bioavailability (F)
• Volume of distribution (Vd)

Correct Answer: Absolute bioavailability (F)

Q8. Which regulatory submission is typically required to obtain marketing approval in the United States after successful clinical trials?

• IND (Investigational New Drug)
• ANDA (Abbreviated New Drug Application)
• NDA (New Drug Application)
• Module V dossier

Correct Answer: NDA (New Drug Application)

Q9. Which GLP/GMP-related activity most directly supports technology transfer of an API process to a production site?

• Conducting investigator-initiated clinical trials
• Preparing detailed process qualification (PQ) and validation protocols
• Performing initial target identification assays
• Designing nonclinical disease models

Correct Answer: Preparing detailed process qualification (PQ) and validation protocols

Q10. Structure-based drug design primarily relies on:

• Large-scale animal studies to screen compounds
• Three-dimensional structural information of the target protein
• High-dose toxicity endpoints in rodents
• Empirical formulation screening

Correct Answer: Three-dimensional structural information of the target protein

Q11. Which strategy is most commonly used to reduce the risk of late-stage clinical failure due to unforeseen toxicity?

• Rely solely on in vitro assays
• Implement translational biomarkers and predictive preclinical models
• Skip Phase II and go directly to Phase III
• Increase clinical dose without additional preclinical data

Correct Answer: Implement translational biomarkers and predictive preclinical models

Q12. In formulation development for oral small molecules, a common tactic to improve bioavailability of a poorly soluble compound is to:

• Switch immediately to intravenous administration
• Develop a salt form or solid dispersion to enhance solubility
• Increase tablet size to include more excipient
• Use only aqueous solutions for dosing

Correct Answer: Develop a salt form or solid dispersion to enhance solubility

Q13. Which preclinical toxicology study is generally required by regulators before first-in-human dosing?

• Long-term carcinogenicity studies in rodents
• Acute and repeat-dose toxicity studies in two species including one non-rodent
• Phase IV post-marketing surveillance
• Bioequivalence studies

Correct Answer: Acute and repeat-dose toxicity studies in two species including one non-rodent

Q14. Repurposing an approved drug for a new indication is attractive because it typically:

• Requires completely new CMC development
• Has unknown human safety profile
• Can shorten development time and reduce safety risk
• Always avoids regulatory submission

Correct Answer: Can shorten development time and reduce safety risk

Q15. Which analytical consideration is crucial in CMC documentation to ensure consistent product quality during scale-up?

• Absence of any stability data
• Robust, validated assays for identity, purity, potency, and impurities
• Only relying on theoretical yield estimates
• Using non-qualified raw materials without supplier documentation

Correct Answer: Robust, validated assays for identity, purity, potency, and impurities

Q16. Pharmacokinetic/pharmacodynamic (PK/PD) modeling during development is most useful for:

• Replacing all clinical trials
• Selecting optimal dose and dosing regimen for clinical studies
• Determining nonclinical toxicology endpoints
• Designing manufacturing plant layout

Correct Answer: Selecting optimal dose and dosing regimen for clinical studies

Q17. Which approach is commonly used to protect a new chemical entity’s intellectual property during early development?

• Publishing all experimental details immediately
• Filing a priority patent application covering composition and use
• Sharing the molecule openly with competitors
• Omitting any patenting efforts until after Phase III

Correct Answer: Filing a priority patent application covering composition and use

Q18. In the context of biologics development, a critical early challenge distinct from small molecules is:

• Defining a simple small-molecule synthetic route
• Cell line development and control of product heterogeneity
• Using fixed-dose oral tablets
• Avoiding any immunogenicity testing

Correct Answer: Cell line development and control of product heterogeneity

Q19. Which of the following best describes a “first-in-class” drug candidate?

• An improved formulation of an existing marketed drug
• A compound that targets a mechanism not previously exploited by approved therapies
• A generic version of a small molecule
• A biosimilar to an existing biologic

Correct Answer: A compound that targets a mechanism not previously exploited by approved therapies

Q20. During technology transfer, the receiving manufacturing site should primarily be evaluated for:

• Ability to publish scientific papers
• Compatibility of equipment, trained personnel, and quality systems to reproduce the process
• Proximity to competitor facilities
• Availability of animal housing for GLP studies

Correct Answer: Compatibility of equipment, trained personnel, and quality systems to reproduce the process

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