Treatment of family members MCQs With Answer

Introduction: This collection of MCQs on the treatment of family members is designed for M.Pharm students studying Research Methodology & Biostatistics. The questions focus on ethical, methodological and statistical issues that arise when family members of investigators, trial staff, or enrolled participants receive treatment in clinical research or routine care. Topics include consent and undue influence, allocation and blinding challenges, handling correlated outcomes from family clusters, sample size and intraclass correlation, analytic approaches (paired tests, mixed models, GEE), reporting requirements, and regulatory guidance. These questions will help students think critically about potential bias, conflicts of interest, and appropriate statistical methods when family relationships affect treatment and data analysis.

Q1. When a clinical trial enrolls multiple related family members (e.g., parent and child), which statistical issue is most important to account for during analysis?

• Missing data mechanisms
• Intracluster correlation due to non-independence of observations
• Selection bias from non-random sampling
• Multiple testing corrections

Correct Answer: Intracluster correlation due to non-independence of observations

Q2. Which trial design explicitly randomizes groups or families rather than individuals to prevent contamination between family members?

• Cross-over trial
• Cluster randomized trial
• Factorial design
• Adaptive trial

Correct Answer: Cluster randomized trial

Q3. In a study where family members receive the same intervention, which analytic method appropriately models correlated binary outcomes?

• Student’s t-test for independent samples
• Generalized estimating equations (GEE) with exchangeable correlation
• Kaplan–Meier survival analysis
• Chi-square test for independence

Correct Answer: Generalized estimating equations (GEE) with exchangeable correlation

Q4. Which ethical concern is most prominent when treating family members of the principal investigator in a clinical trial?

• Insufficient study duration
• Potential conflict of interest and coercion
• Lack of statistical power
• Choice of placebo

Correct Answer: Potential conflict of interest and coercion

Q5. For a continuous outcome measured in related family members, which statistical model can incorporate both fixed treatment effects and random family effects?

• Simple linear regression without random effects
• Mixed-effects (multilevel) linear model
• Wilcoxon signed-rank test
• Fisher’s exact test

Correct Answer: Mixed-effects (multilevel) linear model

Q6. When family members are preferentially selected into a treatment arm due to clinician choice, which bias is most likely introduced?

• Performance bias
• Allocation bias (selection bias)
• Detection bias
• Observer bias

Correct Answer: Allocation bias (selection bias)

Q7. If a protocol allows treating a patient’s relative as an open-label convenience participant, which regulatory document should explicitly address this practice?

• Investigator brochure only
• Informed consent form and ethics committee submission
• Clinical trial registry summary only
• Adverse event reporting form only

Correct Answer: Informed consent form and ethics committee submission

Q8. Which method is appropriate for sample size calculation when the unit of analysis is family clusters rather than individuals?

• Treat families as individuals using standard sample size formulae
• Adjust individual-based sample size using the design effect (1 + (m-1)ICC)
• Use only pilot study estimates without ICC
• Ignore clustering and increase alpha to compensate

Correct Answer: Adjust individual-based sample size using the design effect (1 + (m-1)ICC)

Q9. In reporting results where family members may share exposures and genetics, which statement should be included in the discussion?

• That family membership has no effect on results and need not be discussed
• A limitation that shared exposures/genetics may confound treatment effects and may limit generalizability
• That sample size was sufficient to ignore any family effects
• That familial clustering reduces measurement error

Correct Answer: A limitation that shared exposures/genetics may confound treatment effects and may limit generalizability

Q10. When family members are enrolled in both treatment and control arms, what is the best approach to minimize contamination of behaviors between them?

• Ensure complete blinding is impossible
• Use cluster randomization at family level or stratify randomization by family
• Allow families to choose arms based on preference
• Do not collect information on inter-family communication

Correct Answer: Use cluster randomization at family level or stratify randomization by family

Q11. Which consent-related risk increases when treating family members in research conducted by a clinician-investigator?

• Therapeutic misconception and undue influence from the clinician-investigator
• Lower procedural costs
• Automatic waiver of informed consent
• Improved blinding

Correct Answer: Therapeutic misconception and undue influence from the clinician-investigator

Q12. For paired continuous measurements within a family pair (e.g., siblings), which statistical test compares before-after or between-pair differences?

• Independent samples t-test
• Paired t-test
• Mann–Whitney U test
• Log-rank test

Correct Answer: Paired t-test

Q13. In adverse event reporting, why must safety data be stratified by family clusters when multiple relatives participate?

• To inflate type I error
• Because shared genetics or environment may produce correlated adverse events and influence causality assessment
• To anonymize data without additional analyses
• To ensure adverse events are ignored for related participants

Correct Answer: Because shared genetics or environment may produce correlated adverse events and influence causality assessment

Q14. Which analytic approach is preferable when the number of family clusters is small but within-family observations are many?

• Use ordinary least squares without adjustments
• Use cluster-robust standard errors cautiously and consider mixed models or permutation tests
• Ignore clustering and report unadjusted p-values
• Use repeated measures ANOVA without random effects

Correct Answer: Use cluster-robust standard errors cautiously and consider mixed models or permutation tests

Q15. Which guideline explicitly addresses inclusion of vulnerable populations and undue influence in research ethics, applicable when family members are treated by investigators?

• Good Clinical Practice (GCP) and Declaration of Helsinki
• ICD-10 coding manual
• Pharmacopoeia monograph
• Manufacturing SOPs

Correct Answer: Good Clinical Practice (GCP) and Declaration of Helsinki

Q16. If investigators treat their own family members outside the trial protocol but collect data, what primary issue arises?

• Enhanced external validity
• Violation of protocol and potential introduction of observer and reporting bias
• Guaranteed data quality
• No need for ethical review

Correct Answer: Violation of protocol and potential introduction of observer and reporting bias

Q17. Which measure quantifies the degree of similarity among outcomes within family clusters?

• Effect size (Cohen’s d)
• Intraclass correlation coefficient (ICC)
• Hazard ratio
• Confidence interval width

Correct Answer: Intraclass correlation coefficient (ICC)

Q18. When designing a trial that may include related participants, which operational step reduces selection of many family members from a single household?

• Allow unlimited enrollment per household
• Implement a household cap or sampling rule per family
• Avoid collecting household identifiers
• Exclude all families to increase heterogeneity

Correct Answer: Implement a household cap or sampling rule per family

Q19. For binary outcomes in small family clusters with rare events, which analytic approach can improve inference reliability?

• Standard logistic regression ignoring clustering
• Exact methods or conditional logistic regression for matched or clustered data
• Use of chi-square test without adjustments
• Linear probability model without cluster terms

Correct Answer: Exact methods or conditional logistic regression for matched or clustered data

Q20. In trial monitoring, what specific documentation should the data safety monitoring board (DSMB) review when family-member enrollment is substantial?

• Only the overall recruitment numbers without subgroup checks
• Patterns of enrollment by family cluster, correlated adverse events, and potential protocol deviations related to familial treatment
• Manufacturing batch records only
• Marketing materials for the intervention

Correct Answer: Patterns of enrollment by family cluster, correlated adverse events, and potential protocol deviations related to familial treatment

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