Investigational use of drugs – description and principles MCQs With Answer

Investigational use of drugs covers the scientific and regulatory framework for evaluating new or repurposed medicines in humans. This introduction summarises core principles: clinical trial phases (I–IV), IND/CTA submissions, protocol design, preclinical toxicology, Good Clinical Practice (GCP), ethics committee/IRB oversight, informed consent, data integrity, pharmacovigilance and reporting of adverse events including SUSARs. It outlines sponsor and investigator responsibilities, monitoring, DSMB roles, compassionate use/expanded access and accelerated approvals. Mastery of these keywords and principles enables B. Pharm students to participate effectively in clinical research, regulatory documentation and safe drug development. Now let’s test your knowledge with 30 MCQs on this topic.

Q1. What best defines an investigational drug?

• A medicine approved for marketing and used in routine care
• A compound under study that has been withdrawn due to safety
• A substance not yet approved for general marketing being tested in clinical trials
• A generic product already proven bioequivalent

Correct Answer: A substance not yet approved for general marketing being tested in clinical trials

Q2. IND in clinical research commonly stands for which regulatory submission?

• Investigational New Drug application
• International New Drug dossier
• Investigational Nonclinical Data
• Initial Notification Document

Correct Answer: Investigational New Drug application

Q3. Primary objectives of Phase I clinical trials are:

• Confirm long-term efficacy and market access strategies
• Assess safety, tolerability and pharmacokinetics in humans
• Evaluate cost-effectiveness compared with standard care
• Conduct large-scale comparative effectiveness

Correct Answer: Assess safety, tolerability and pharmacokinetics in humans

Q4. Phase II clinical trials mainly focus on:

• Post-marketing surveillance for rare adverse events
• Preclinical toxicology assessment in animals
• Preliminary efficacy, dose finding and short-term safety
• Manufacturing scale-up and stability testing

Correct Answer: Preliminary efficacy, dose finding and short-term safety

Q5. The main purpose of Phase III trials is to:

• Characterize pharmacokinetics in healthy volunteers only
• Confirm efficacy and safety in a large patient population
• Develop in vitro assays for potency
• Obtain animal efficacy data for IND submission

Correct Answer: Confirm efficacy and safety in a large patient population

Q6. Phase IV studies are performed to:

• Evaluate first-in-human safety
• Monitor drug safety and effectiveness after marketing approval
• Determine maximum tolerated dose in healthy volunteers
• Replace preclinical studies during development

Correct Answer: Monitor drug safety and effectiveness after marketing approval

Q7. Which party is primarily responsible for initiating and funding a clinical trial?

• Institutional Review Board (IRB)
• Sponsor
• Clinical trial participant
• State regulatory authority

Correct Answer: Sponsor

Q8. An Investigator’s Brochure should include:

• Only the final marketing label and pricing
• Complete preclinical and available clinical safety and efficacy data
• Individual subject medical records from unrelated studies
• Proprietary manufacturing processes in detail

Correct Answer: Complete preclinical and available clinical safety and efficacy data

Q9. The primary role of an Ethics Committee/IRB is to:

• Maximize trial enrollment regardless of risk
• Protect the rights, safety and well-being of research participants
• Approve marketing claims for promotional use
• Set pricing for investigational products

Correct Answer: Protect the rights, safety and well-being of research participants

Q10. Essential elements of informed consent include:

• Only the study sponsor’s contact information
• Purpose, procedures, risks, benefits and alternatives
• Guaranteed therapeutic benefit to the participant
• Confidential financial information of the sponsor

Correct Answer: Purpose, procedures, risks, benefits and alternatives

Q11. Which event qualifies as a Serious Adverse Event (SAE)?

• A mild transient headache that resolves without treatment
• An adverse event leading to hospitalization or death
• A planned follow-up visit per protocol
• A laboratory value outside reference range with no clinical significance

Correct Answer: An adverse event leading to hospitalization or death

Q12. SUSAR stands for:

• Serious Uncommon Safety Assessment Report
• Suspected Unexpected Serious Adverse Reaction
• Standardized Unblinded Safety and Research
• Safety Update Summary and Annual Report

Correct Answer: Suspected Unexpected Serious Adverse Reaction

Q13. Good Clinical Practice (GCP) guidelines are harmonized by which body?

• World Trade Organization (WTO)
• International Conference on Harmonisation (ICH)
• International Criminal Court (ICC)
• United Nations Development Programme (UNDP)

Correct Answer: International Conference on Harmonisation (ICH)

Q14. Who must approve the clinical trial protocol before subject enrolment?

• The study sponsor only
• The Institutional Ethics Committee/IRB
• The drug manufacturer’s marketing team
• The study monitor alone

Correct Answer: The Institutional Ethics Committee/IRB

Q15. The Data Safety Monitoring Board (DSMB) is responsible for:

• Manufacturing the investigational drug
• Monitoring accumulating safety data and recommending trial continuation or stoppage
• Marketing approved drug indications
• Recruiting study participants at sites

Correct Answer: Monitoring accumulating safety data and recommending trial continuation or stoppage

Q16. Randomization in clinical trials primarily reduces which bias?

• Reporting bias by journals
• Selection bias
• Manufacturing bias
• Regulatory approval bias

Correct Answer: Selection bias

Q17. Blinding a clinical trial is intended to minimize:

• Supply chain delays
• Observer and participant expectancy bias
• Animal welfare concerns
• Analytical chemistry variation

Correct Answer: Observer and participant expectancy bias

Q18. A placebo-controlled trial is useful because it:

• Removes any need for informed consent
• Controls for placebo effects and natural history of disease
• Ensures every participant receives active drug
• Eliminates the requirement for safety monitoring

Correct Answer: Controls for placebo effects and natural history of disease

Q19. Equivalence trials differ from non-inferiority trials by:

• Testing whether a new therapy is significantly superior only
• Demonstrating similar efficacy within predefined margins in both directions
• Being used only for vaccines and not drugs
• Assessing cost versus benefit exclusively

Correct Answer: Demonstrating similar efficacy within predefined margins in both directions

Q20. Bioavailability refers to:

• The proportion of administered drug reaching systemic circulation and the rate at which it does so
• The environmental impact of manufacturing
• The price at which a drug is sold in different markets
• The stability of a drug under refrigeration

Correct Answer: The proportion of administered drug reaching systemic circulation and the rate at which it does so

Q21. In India, Schedule Y of the Drugs and Cosmetics Rules relates to:

• Clinical trial requirements and approval processes
• Import tariffs for pharmaceuticals
• Pharmacopoeial monographs for excipients
• Marketing codes for pharmacy retailers

Correct Answer: Clinical trial requirements and approval processes

Q22. CDSCO stands for:

• Central Drugs Standard Control Organization
• Civil Drug Safety and Compliance Office
• Clinical Data Standards and Compliance Organization
• Comprehensive Drug Submission Coordination Office

Correct Answer: Central Drugs Standard Control Organization

Q23. The DCGI in India is responsible for:

• Approving cosmetic advertising only
• Regulating and approving clinical trials and marketing authorizations for drugs
• Manufacturing investigational products
• Funding academic research exclusively

Correct Answer: Regulating and approving clinical trials and marketing authorizations for drugs

Q24. Compassionate use or expanded access programs allow:

• Use of approved generics for research purposes
• Access to investigational drugs for patients with no satisfactory alternatives outside a clinical trial
• Marketing an investigational drug without any monitoring
• Replacement of informed consent with physician approval only

Correct Answer: Access to investigational drugs for patients with no satisfactory alternatives outside a clinical trial

Q25. Key investigator responsibilities include:

• Ensuring informed consent, protocol compliance and SAE reporting
• Setting global regulatory policy
• Determining national pricing for the drug
• Manufacturing the investigational product

Correct Answer: Ensuring informed consent, protocol compliance and SAE reporting

Q26. A Contract Research Organization (CRO) typically provides:

• Regulatory authority adjudication
• Operational services such as monitoring, data management and biostatistics
• Permanent approval for marketing applications
• Clinical care for unrelated conditions

Correct Answer: Operational services such as monitoring, data management and biostatistics

Q27. A protocol deviation differs from a protocol violation by:

• Deviation is minor and unlikely to affect participant safety or data integrity; violation is major and may impact safety or integrity
• Deviation always requires termination of the trial
• Violation is a manufacturer-only issue
• Deviation and violation are interchangeable terms with no regulatory distinction

Correct Answer: Deviation is minor and unlikely to affect participant safety or data integrity; violation is major and may impact safety or integrity

Q28. Pharmacovigilance encompasses:

• Only preclinical toxicity testing in animals
• Detection, assessment, understanding and prevention of adverse effects in humans
• Marketing strategies for post-approval promotion
• Clinical site selection and recruitment methods

Correct Answer: Detection, assessment, understanding and prevention of adverse effects in humans

Q29. A surrogate endpoint is best described as:

• A direct measure of how a patient feels or survives
• An indirect marker expected to predict clinical benefit
• A financial metric used in health economics
• A regulatory submission checklist item

Correct Answer: An indirect marker expected to predict clinical benefit

Q30. An essential component of an IND/CTA application is:

• Only the proposed marketing strategy
• Comprehensive preclinical safety and toxicology data
• Patient names from previous studies
• Retail pricing projections for the drug

Correct Answer: Comprehensive preclinical safety and toxicology data

G S Sachin

I am a Registered Pharmacist under the Pharmacy Act, 1948, and the founder of PharmacyFreak.com. I hold a Bachelor of Pharmacy degree from Rungta College of Pharmaceutical Science and Research. With a strong academic foundation and practical knowledge, I am committed to providing accurate, easy-to-understand content to support pharmacy students and professionals. My aim is to make complex pharmaceutical concepts accessible and useful for real-world application.

Mail- Sachin@pharmacyfreak.com