Outcomes in pharmacoeconomics: clinical, economic, humanistic MCQs With Answer

Introduction: This quiz collection focuses on outcomes in pharmacoeconomics — clinical, economic, and humanistic — tailored for M.Pharm students. It covers definitions, measurement methods, and practical applications used in pharmacoeconomic evaluations. The questions explore clinical endpoints (mortality, morbidity, surrogate markers), economic outcomes (cost categories, resource use, cost-effectiveness and cost-utility metrics) and humanistic outcomes (health-related quality of life, patient-reported outcomes, adherence, satisfaction). Emphasis is placed on measurement instruments (QALY, DALY, EQ-5D, SF-36), study perspective and time horizon selection, and interpretation of results for decision-making. The set aims to deepen understanding and prepare students for applied analyses and exams.

Q1. Which outcome measure is specifically designed to combine both quantity and quality of life into a single metric for economic evaluation?

• Disability-adjusted life year (DALY)
• Quality-adjusted life year (QALY)
• Incremental cost-effectiveness ratio (ICER)
• Health-related quality of life score (HRQoL score)

Correct Answer: Quality-adjusted life year (QALY)

Q2. In pharmacoeconomic studies, which cost category includes lost productivity from absenteeism and presenteeism?

• Direct medical costs
• Direct non-medical costs
• Indirect costs
• Intangible costs

Correct Answer: Indirect costs

Q3. Which instrument is commonly used as a generic preference-based measure to derive utility values for QALY calculations?

• Beck Depression Inventory (BDI)
• EQ-5D
• Visual Analogue Scale for pain only
• Harris Hip Score

Correct Answer: EQ-5D

Q4. What does the Incremental Cost-Effectiveness Ratio (ICER) represent in a pharmacoeconomic analysis?

• The total cost of an intervention divided by the number of patients treated
• The additional cost per additional unit of effect when comparing two alternatives
• The ratio of indirect to direct costs for a treatment
• The percentage reduction in mortality attributable to an intervention

Correct Answer: The additional cost per additional unit of effect when comparing two alternatives

Q5. Which of the following is an example of a humanistic outcome in pharmacoeconomics?

• Hospitalization rate
• Medication acquisition cost
• Health-related quality of life (HRQoL)
• Cost per life saved

Correct Answer: Health-related quality of life (HRQoL)

Q6. In a cost-utility analysis, the primary outcome is typically expressed as:

• Cost per life-year gained
• Cost per QALY gained
• Total program cost
• Incremental net benefit in monetary units without utility adjustment

Correct Answer: Cost per QALY gained

Q7. Which perspective includes costs borne by the health care system only, excluding patient and productivity losses?

• Societal perspective
• Patient perspective
• Payer or health-care system perspective
• Provider perspective

Correct Answer: Payer or health-care system perspective

Q8. Which outcome would be classified as an intangible cost in pharmacoeconomic terms?

• Out-of-pocket medication expenses
• Value of lost work hours
• Pain and suffering experienced by the patient
• Hospital bed-days

Correct Answer: Pain and suffering experienced by the patient

Q9. A surrogate endpoint is best described as:

• The final clinical outcome such as mortality
• An intermediate marker used to predict clinical benefit
• A measure of direct medical costs
• A patient satisfaction score

Correct Answer: An intermediate marker used to predict clinical benefit

Q10. Which method is appropriate to handle uncertainty in key parameters of a pharmacoeconomic model?

• Descriptive statistics only
• Sensitivity analysis (one-way, multi-way, probabilistic)
• Cross-sectional survey
• Intention-to-treat analysis

Correct Answer: Sensitivity analysis (one-way, multi-way, probabilistic)

Q11. The SF-36 questionnaire primarily measures which type of outcome?

• Direct medical costs
• Generic health-related quality of life across multiple domains
• Utility values directly usable for QALY without mapping
• Pharmacokinetic parameters

Correct Answer: Generic health-related quality of life across multiple domains

Q12. In pharmacoeconomic evaluations, the time horizon should be chosen to:

• Match the funding cycle of the payer regardless of disease duration
• Be as short as possible to reduce modelling complexity
• Capture all relevant costs and outcomes for the alternatives being compared
• Only include the period of clinical trial follow-up

Correct Answer: Capture all relevant costs and outcomes for the alternatives being compared

Q13. Which statement distinguishes cost-effectiveness analysis (CEA) from cost-utility analysis (CUA)?

• CEA uses monetary outcomes; CUA uses clinical event counts
• CEA reports cost per natural unit (e.g., life-years); CUA reports cost per utility-based measure (e.g., QALY)
• CEA includes humanistic outcomes; CUA excludes them
• CEA always uses societal perspective; CUA uses payer perspective

Correct Answer: CEA reports cost per natural unit (e.g., life-years); CUA reports cost per utility-based measure (e.g., QALY)

Q14. Which approach is most appropriate to value lost productivity in economic evaluations from a societal perspective?

• Only counting direct medical costs
• Human capital approach or friction cost approach
• Using patient’s willingness to pay for treatment
• Excluding productivity because it is not a health outcome

Correct Answer: Human capital approach or friction cost approach

Q15. When measuring utilities for QALY calculation, which of the following direct elicitation methods involves choices between certain and uncertain outcomes?

• Standard gamble
• Visual analogue scale
• Time-trade off
• SF-36 mapping

Correct Answer: Standard gamble

Q16. A study reporting “cost per responder” is most likely using which type of outcome?

• Humanistic utility-based outcome
• Clinical dichotomous outcome (responder vs non-responder)
• Indirect cost outcome
• Quality-adjusted survival outcome

Correct Answer: Clinical dichotomous outcome (responder vs non-responder)

Q17. Which of the following best represents a humanistic patient-reported outcome commonly used in pharmacoeconomic studies?

• Medication wholesale price
• Patient adherence measured by self-report scales
• Laboratory biomarker level
• Hospital readmission rate

Correct Answer: Patient adherence measured by self-report scales

Q18. In cost-effectiveness planes, a point falling in the southeast quadrant indicates the new intervention is:

• More effective and more costly
• Less effective and less costly
• More effective and less costly (dominant)
• Less effective and more costly (dominated)

Correct Answer: More effective and less costly (dominant)

Q19. Which consideration is critical when translating clinical trial outcomes to real-world pharmacoeconomic models?

• Assume trial adherence equals real-world adherence without adjustment
• Adjust for generalizability, adherence differences, and broader patient populations
• Use only per-protocol trial data to represent real-world effectiveness
• Exclude resource utilization outside trial settings as irrelevant

Correct Answer: Adjust for generalizability, adherence differences, and broader patient populations

Q20. Willingness-to-pay (WTP) thresholds are used in interpreting ICERs. Which statement is correct about WTP?

• WTP is a universal global value applied to all countries
• WTP reflects the maximum amount a decision-maker or society is prepared to pay per unit of health gain
• WTP determines clinical efficacy of an intervention
• WTP replaces the need for sensitivity analysis

Correct Answer: Willingness-to-pay reflects the maximum amount a decision-maker or society is prepared to pay per unit of health gain

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