Introduction to pharmacoepidemiology: definition, scope and need MCQs With Answer

Introduction

Introduction to pharmacoepidemiology: definition, scope and need MCQs With Answer

Pharmacoepidemiology applies epidemiologic methods to the study of drug use and effects in large populations. For M.Pharm students, mastering this field is essential for evaluating drug safety, effectiveness, utilization patterns and public-health impact after regulatory approval. This blog provides focused multiple-choice questions that reinforce concepts such as study designs (cohort, case–control, cross-sectional), measures of disease occurrence and association, sources of pharmacoepidemiologic data, bias and confounding, signal detection, and the role of pharmacoepidemiology in regulatory decision-making and rational drug use. These MCQs are designed to deepen understanding and support exam preparation and practical application in clinical and regulatory settings.

Q1. What is the most accurate definition of pharmacoepidemiology?

• The study of molecular mechanisms of drug action
• The application of epidemiologic methods to study the use and effects of drugs in populations
• The economic evaluation of drug pricing and reimbursement
• The clinical phase of drug development involving healthy volunteers

Correct Answer: The application of epidemiologic methods to study the use and effects of drugs in populations

Q2. Which study design is most appropriate to estimate incidence of an adverse drug reaction over time?

• Cross-sectional study
• Case-control study
• Cohort study
• Ecological study

Correct Answer: Cohort study

Q3. In a case–control study of drug exposure and rare adverse event, which measure of association is commonly used?

• Risk ratio (relative risk)
• Odds ratio
• Incidence rate
• Hazard ratio

Correct Answer: Odds ratio

Q4. Which of the following best describes ‘pharmacovigilance’ within pharmacoepidemiology?

• Evaluation of cost-effectiveness of medicines
• Identification, assessment, understanding and prevention of adverse effects or other drug-related problems
• Design and execution of preclinical toxicity studies
• Formulation development for improved bioavailability

Correct Answer: Identification, assessment, understanding and prevention of adverse effects or other drug-related problems

Q5. Which data source is characterized by voluntary reports of suspected adverse drug reactions collected by regulators and manufacturers?

• Claims/administrative databases
• Spontaneous (spontaneous reporting) systems
• Electronic health records from hospitals
• Clinical trial databases

Correct Answer: Spontaneous (spontaneous reporting) systems

Q6. Which term refers to the proportion of individuals in a population who have a disease or condition at a specific time?

• Incidence rate
• Attack rate
• Prevalence
• Hazard

Correct Answer: Prevalence

Q7. In pharmacoepidemiology, which bias occurs when the exposure is misclassified differently between cases and controls?

• Selection bias
• Confounding
• Non-differential misclassification
• Differential misclassification

Correct Answer: Differential misclassification

Q8. Which measure indicates the excess risk of outcome among the exposed that can be attributed to the exposure?

• Relative risk
• Attributable risk (risk difference)
• Odds ratio
• Prevalence ratio

Correct Answer: Attributable risk (risk difference)

Q9. Which approach is most effective to control confounding in the design phase of a pharmacoepidemiologic cohort study?

• Stratification during analysis
• Randomization of exposure
• Restriction or matching at enrollment
• Post-hoc multivariable regression only

Correct Answer: Restriction or matching at enrollment

Q10. Which of the following is a limitation of spontaneous reporting systems?

• They provide precise incidence rates for adverse events
• They are free from reporting bias
• Under-reporting and lack of denominator data
• They always establish causality

Correct Answer: Under-reporting and lack of denominator data

Q11. Which study design is most efficient for investigating a rare adverse drug reaction?

• Randomized controlled trial
• Cross-over trial
• Case–control study
• Cross-sectional study

Correct Answer: Case–control study

Q12. The primary need for pharmacoepidemiology after drug approval is to:

• Reduce time of preclinical testing
• Monitor drug safety and effectiveness in real-world populations
• Replace randomized controlled trials entirely
• Increase drug prices through market surveillance

Correct Answer: Monitor drug safety and effectiveness in real-world populations

Q13. Which measure is most appropriate to compare time-to-event outcomes between exposed and unexposed groups accounting for varying follow-up?

• Prevalence ratio
• Incidence proportion
• Hazard ratio from survival analysis
• Attributable fraction

Correct Answer: Hazard ratio from survival analysis

Q14. In pharmacoepidemiology, ‘signal detection’ primarily refers to:

• Detecting drug–drug interactions in preclinical studies
• Identifying potential new safety concerns from data sources
• Measuring drug bioequivalence
• Assessing medication adherence in trials

Correct Answer: Identifying potential new safety concerns from data sources

Q15. Which analytic method helps adjust for measured confounding in observational pharmacoepidemiologic studies?

• Intention-to-treat analysis
• Propensity score matching or weighting
• Blinding of investigators
• Simple random sampling

Correct Answer: Propensity score matching or weighting

Q16. Which of the following differentiates pharmacoepidemiology from pharmacoeconomics?

• Pharmacoepidemiology focuses on costs and budgets
• Pharmacoeconomics studies drug safety signals
• Pharmacoepidemiology studies drug effects and utilization in populations; pharmacoeconomics evaluates cost-effectiveness and resource allocation
• They are identical fields with different names

Correct Answer: Pharmacoepidemiology studies drug effects and utilization in populations; pharmacoeconomics evaluates cost-effectiveness and resource allocation

Q17. Which indicator is useful to evaluate appropriate prescribing and utilization patterns across populations?

• Number needed to treat (NNT) only
• Drug utilization studies and defined daily dose (DDD) metrics
• Laboratory bioequivalence data
• Preclinical ADME parameters

Correct Answer: Drug utilization studies and defined daily dose (DDD) metrics

Q18. In assessing causality for a suspected adverse drug reaction, which criterion is NOT part of Bradford Hill considerations?

• Temporality
• Biological gradient (dose–response)
• Specificity
• Price of the drug

Correct Answer: Price of the drug

Q19. Which metric expresses how many patients need to be treated for one additional patient to benefit compared with a control in clinical terms often used by clinicians and pharmacoepidemiologists?

• Relative risk
• Number needed to treat (NNT)
• Odds ratio
• Incidence rate ratio

Correct Answer: Number needed to treat (NNT)

Q20. Which factor most strongly threatens internal validity of an observational pharmacoepidemiologic study?

• High external generalizability
• Confounding by indication (patients receive drugs for reasons linked to outcomes)
• Large sample size
• Prospective data collection

Correct Answer: Confounding by indication (patients receive drugs for reasons linked to outcomes)

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