Investigational New Drug (IND) informational content MCQs With Answer

Investigational New Drug (IND) informational content MCQs With Answer

This set of MCQs is designed for M.Pharm students specializing in Product Development and Technology Transfer. The questions cover essential IND concepts — regulatory framework, required dossier sections (CMC, nonclinical, clinical), sponsor/investigator responsibilities, safety monitoring and reporting, and electronic submission standards. Emphasis is placed on practical aspects such as designing IND-enabling studies, interpreting toxicology data for first-in-human dosing, content and purpose of the Investigator’s Brochure, IND amendments, and types of INDs. These items will help reinforce technical knowledge needed for drafting, reviewing, and managing IND submissions and for successful transition from preclinical to clinical development.

Q1. What is the primary purpose of filing an Investigational New Drug (IND) application with a regulatory authority?

• To obtain marketing approval for a new drug
• To request permission to begin clinical trials in humans
• To register the drug for manufacturing at commercial scale
• To obtain patent protection

Correct Answer: To request permission to begin clinical trials in humans

Q2. Which section of an IND contains detailed information about the drug substance and drug product manufacturing, controls, and stability?

• Investigator’s Brochure
• Clinical Protocol
• Chemistry, Manufacturing and Controls (CMC)
• Nonclinical Pharmacology/Toxicology

Correct Answer: Chemistry, Manufacturing and Controls (CMC)

Q3. What is the Investigator’s Brochure (IB) primarily intended to provide?

• Marketing strategy and pricing information
• Comprehensive nonclinical and clinical safety and efficacy data for investigators
• Details of facility GMP certification
• Sequence of clinical trial recruitment milestones

Correct Answer: Comprehensive nonclinical and clinical safety and efficacy data for investigators

Q4. Which of the following nonclinical studies is most critical for determining an initial safe starting dose for first-in-human trials?

• Repeat dose toxicity studies and determination of NOAEL in relevant species
• In vitro genotoxicity assays only
• Short-term pharmacokinetic study in healthy humans
• Clinical Phase II efficacy studies

Correct Answer: Repeat dose toxicity studies and determination of NOAEL in relevant species

Q5. In the U.S., which regulation primarily governs IND submissions and clinical investigations?

• ICH E6(R2)
• 21 CFR Part 312
• 21 CFR Part 11 only
• Good Laboratory Practice (GLP) guidance

Correct Answer: 21 CFR Part 312

Q6. What type of IND is submitted when a physician initiates and conducts a clinical investigation and intends to be both sponsor and investigator?

• Commercial IND
• Investigator IND
• Emergency Use IND
• Abbreviated IND

Correct Answer: Investigator IND

Q7. Which document within the IND contains the proposed clinical trial design, inclusion/exclusion criteria, dosing regimen, and monitoring plan?

• CMC section
• Clinical Protocol
• Investigator’s Brochure
• Nonclinical study reports

Correct Answer: Clinical Protocol

Q8. What does the term “safe harbor” commonly refer to in relation to the 30-day IND review period?

• The sponsor is guaranteed IND approval after 30 days
• The sponsor may begin clinical trials unless the agency imposes a clinical hold within 30 days
• The sponsor cannot contact the regulatory authority during 30 days
• The IND is automatically converted to a marketing application after 30 days

Correct Answer: The sponsor may begin clinical trials unless the agency imposes a clinical hold within 30 days

Q9. Which of the following is a required component of an IND for demonstrating product quality suitable for clinical trials?

• Full-scale commercial batch records only
• Specifications and validation data for analytical methods
• Sales forecasts for the product
• Patient recruitment advertisements

Correct Answer: Specifications and validation data for analytical methods

Q10. When must a sponsor submit a safety update or IND annual report?

• Only at the end of Phase III trials
• Within 30 days of any unblinding event
• Annually, summarizing safety, progress, and changes
• Only when a serious adverse event occurs

Correct Answer: Annually, summarizing safety, progress, and changes

Q11. Which type of preclinical study is essential to evaluate the potential for a new small-molecule drug to cause genetic damage?

• Pharmacokinetic study in rodents
• In vitro and in vivo genotoxicity assays (e.g., Ames, micronucleus)
• Repeat dose toxicity in non-rodents only
• Clinical pharmacology study

Correct Answer: In vitro and in vivo genotoxicity assays (e.g., Ames, micronucleus)

Q12. What is the main objective of a Phase I clinical trial under an IND?

• Confirm therapeutic efficacy in large patient populations
• Evaluate safety, tolerability, and pharmacokinetics in humans
• Compare the drug to an approved competitor in a head-to-head study
• Obtain final marketing authorization

Correct Answer: Evaluate safety, tolerability, and pharmacokinetics in humans

Q13. Which electronic standard is commonly required for regulatory electronic submission of INDs in many regions?

• eCTD (electronic Common Technical Document)
• HL7 FHIR only
• PDF/A-1b without any folder structure
• CSV spreadsheet file

Correct Answer: eCTD (electronic Common Technical Document)

Q14. What constitutes a clinical hold on an IND?

• A request from the sponsor to delay enrollment for strategic reasons
• An order by the regulatory authority to delay or suspend clinical investigations due to safety or other concerns
• An investigator’s decision to pause recruitment due to low enrollment
• A manufacturing delay for commercial supply

Correct Answer: An order by the regulatory authority to delay or suspend clinical investigations due to safety or other concerns

Q15. Which regulatory action is typically required when there is a major change to a trial protocol after IND submission (e.g., new patient population, dose escalation beyond planned range)?

• No action is required
• Submit an IND amendment describing the change and rationale
• Only inform clinical sites verbally
• Wait until annual report to disclose changes

Correct Answer: Submit an IND amendment describing the change and rationale

Q16. What is an Emergency Use IND (EIND) used for?

• To request marketing approval for emergency products
• To allow use of an investigational drug for a single patient in a life-threatening situation when no alternatives exist
• To streamline commercial manufacturing approvals
• To document routine adverse events

Correct Answer: To allow use of an investigational drug for a single patient in a life-threatening situation when no alternatives exist

Q17. For biologics, which additional documentation is often critical in the IND’s CMC section compared to small molecules?

• Detailed description of chemical synthesis only
• Characterization of critical quality attributes, cell line history, and viral safety data
• Recruitment strategies for clinical sites
• Consumer labeling and advertising drafts

Correct Answer: Characterization of critical quality attributes, cell line history, and viral safety data

Q18. Which adverse event reporting requirement typically triggers expedited reporting to the regulatory authority during an IND study?

• All mild adverse events
• Serious, unexpected suspected adverse reactions (SUSARs)
• Routine lab value fluctuations within expected ranges
• Enrollment delays

Correct Answer: Serious, unexpected suspected adverse reactions (SUSARs)

Q19. When developing an IND dossier, which element helps justify the relevance of animal toxicology species chosen for safety studies?

• Similarity of metabolic pathways and pharmacodynamics between species and humans
• Total number of animals used regardless of species
• Cost of conducting studies in the species
• Ease of dosing and husbandry only

Correct Answer: Similarity of metabolic pathways and pharmacodynamics between species and humans

Q20. Which of the following best describes the role of a sponsor in an IND-regulated clinical trial?

• Only the clinical investigator at each site has sponsor responsibilities
• The sponsor is responsible for initiating, managing, and financing the trial and ensuring compliance with regulations
• The sponsor is exclusively responsible for patient care decisions during the trial
• The sponsor’s only role is to manufacture the drug

Correct Answer: The sponsor is responsible for initiating, managing, and financing the trial and ensuring compliance with regulations

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