Pharmacoepidemiological methods: case reports and case series MCQs With Answer

Pharmacoepidemiological methods: case reports and case series MCQs With Answer

This set of MCQs is designed for M.Pharm students to deepen understanding of case reports and case series as pharmacoepidemiological tools. The questions cover definitions, purpose, strengths and limitations, causality assessment, reporting standards, ethical issues, and their role in pharmacovigilance and signal detection. Emphasis is placed on critical appraisal: when and how these designs can suggest adverse drug reactions, what they cannot determine (such as incidence), and how they inform further analytical studies. These items will help students prepare for examinations and practical interpretation of clinical safety evidence in drug monitoring and regulatory contexts.

Q1. Which statement best defines a case report in pharmacoepidemiology?

• A detailed description of a single patient’s clinical course and suspected drug-related event
• A controlled trial comparing two drugs in a small population
• A population-based study estimating drug incidence
• A randomized crossover study of drug efficacy

Correct Answer: A detailed description of a single patient’s clinical course and suspected drug-related event

Q2. What is a case series?

• A meta-analysis of randomized controlled trials
• A collection of multiple case reports describing patients with similar exposures or outcomes
• An observational cohort with matched controls
• A database-derived disproportionality analysis

Correct Answer: A collection of multiple case reports describing patients with similar exposures or outcomes

Q3. What is the primary scientific purpose of publishing case reports and case series in pharmacovigilance?

• To provide definitive estimates of drug safety incidence
• To generate hypotheses and detect early safety signals about potential adverse drug reactions
• To replace randomized trials for regulatory approval
• To perform cost-effectiveness analyses

Correct Answer: To generate hypotheses and detect early safety signals about potential adverse drug reactions

Q4. Which is a major limitation of case reports and case series when assessing drug-outcome relationships?

• They always include a randomized control group
• They can estimate relative risk precisely
• They lack a control group and cannot reliably establish causality or incidence
• They provide population-based prevalence data

Correct Answer: They lack a control group and cannot reliably establish causality or incidence

Q5. How do case reports contribute to regulatory action regarding medicines?

• They provide final evidence for causation sufficient for labeling changes without further study
• They cannot be used at all in regulatory decisions
• They can prompt safety reviews, signal prioritization, and further investigations
• They replace phase IV surveillance studies

Correct Answer: They can prompt safety reviews, signal prioritization, and further investigations

Q6. Which tools are commonly used to assess causality in individual case reports?

• Framingham risk score and Kaplan–Meier estimator
• Naranjo algorithm and WHO–UMC causality categories
• ANOVA and logistic regression
• Number needed to treat and number needed to harm

Correct Answer: Naranjo algorithm and WHO–UMC causality categories

Q7. What reporting guideline is recommended for improving transparency and completeness of case reports?

• CONSORT
• STROBE
• CARE (CAse REport) guideline
• PRISMA

Correct Answer: CARE (CAse REport) guideline

Q8. In which situation is a case series most useful?

• Estimating drug incidence in a population
• Identifying and describing rare or novel adverse drug reactions and patterns
• Testing efficacy hypotheses in blinded fashion
• Conducting cost-effectiveness comparisons

Correct Answer: Identifying and describing rare or novel adverse drug reactions and patterns

Q9. Which statistical approach is most appropriate for analyzing data from case series?

• Descriptive statistics summarizing clinical features, timelines, and outcomes
• Multivariable survival analysis to estimate hazard ratios
• Random-effects meta-analysis
• Complex propensity score matching

Correct Answer: Descriptive statistics summarizing clinical features, timelines, and outcomes

Q10. Why are case reports and case series considered low on the evidence hierarchy?

• Because they require large sample sizes and long follow-up
• Because they are randomized and thus biased
• Because of high susceptibility to bias, confounding, and lack of generalizability
• Because they always use administrative databases

Correct Answer: Because of high susceptibility to bias, confounding, and lack of generalizability

Q11. What is an important ethical requirement before publishing a case report?

• No permissions are needed for de-identified cases
• Obtaining informed consent from the patient or their legal representative when identifiable information is present
• Only institutional approval is required; patient consent is never necessary
• Publishing requires only authors’ signatures

Correct Answer: Obtaining informed consent from the patient or their legal representative when identifiable information is present

Q12. How does a case series differ from a case-control study?

• Case series include matched controls, case-control studies do not
• Case series describe exposed cases without controls; case-control studies compare cases with matched controls to estimate associations
• Case series estimate incidence rates; case-control studies only describe individual patients
• There is no methodological difference between them

Correct Answer: Case series describe exposed cases without controls; case-control studies compare cases with matched controls to estimate associations

Q13. Which level of evidence are single case reports and small case series typically considered?

• High-level evidence equivalent to randomized trials
• Moderate-level evidence like cohort studies
• Low-level evidence often at the bottom of the hierarchy
• They are not part of evidence hierarchies

Correct Answer: Low-level evidence often at the bottom of the hierarchy

Q14. What is the best use of aggregate case series data when multiple similar reports are available?

• To calculate precise population incidence rates directly
• To characterize clinical features, time course, dose–response patterns, and outcomes that inform hypotheses
• To perform randomized comparisons between treatments
• To establish definitive regulatory causation on their own

Correct Answer: To characterize clinical features, time course, dose–response patterns, and outcomes that inform hypotheses

Q15. What does a positive drug rechallenge documented in a case report indicate?

• It proves causality beyond doubt
• It is irrelevant for causality assessment
• It strengthens evidence for a causal relationship but may be ethically risky
• It demonstrates the drug is safe to continue

Correct Answer: It strengthens evidence for a causal relationship but may be ethically risky

Q16. What is meant by the “denominator problem” in case reports/series?

• Lack of detailed clinical descriptions
• Inability to determine how many patients were at risk, so incidence cannot be estimated reliably
• Numerator data are always incorrect
• Denominator refers to the statistical significance threshold

Correct Answer: Inability to determine how many patients were at risk, so incidence cannot be estimated reliably

Q17. How should pharmacoepidemiologists use aggregated case reports from spontaneous reporting systems?

• As definitive proof of harm without further work
• To perform signal detection, prioritize hypotheses, and plan analytic epidemiology studies for confirmation
• To estimate treatment efficacy in meta-analyses
• To determine exact risk ratios for regulatory labeling

Correct Answer: To perform signal detection, prioritize hypotheses, and plan analytic epidemiology studies for confirmation

Q18. Which elements should be included in a high-quality case report to aid interpretation?

• Only the final diagnosis without timeline or drug details
• Clear patient demographics, timeline of exposure and events, dose, dechallenge/rechallenge, alternative causes, and outcome
• Only laboratory data without clinical context
• Generic statements without specific dates or drug names

Correct Answer: Clear patient demographics, timeline of exposure and events, dose, dechallenge/rechallenge, alternative causes, and outcome

Q19. When is publication of a case report especially justified?

• When the event is common and well described in textbooks
• When the adverse event is serious, unexpected, newly identified, or suggests a novel mechanism
• When authors want to market a product
• Only when randomized trial data are also available

Correct Answer: When the adverse event is serious, unexpected, newly identified, or suggests a novel mechanism

Q20. Which analytic approach is appropriate when synthesizing information from multiple case series or spontaneous reports?

• Simple descriptive synthesis and pattern analysis; formal disproportionality methods require large spontaneous reporting databases
• Direct calculation of incidence rates identical to cohort studies
• Randomized effect estimation using case series alone
• Systematic randomized pooling without assessing heterogeneity

Correct Answer: Simple descriptive synthesis and pattern analysis; formal disproportionality methods require large spontaneous reporting databases

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