MCQ Quiz: Outcomes in Pharmacoepidemiology

The value of any pharmacoepidemiology study hinges on the outcomes it measures. As a pharmacist, understanding and critically evaluating these outcomes is an essential skill for evidence-based practice. As taught in the Principles of Evidence-Based Practice course, it is crucial to differentiate between a direct clinical outcome, like preventing a stroke, and a surrogate outcome, like lowering a lab value. This quiz will test your knowledge on the different types of outcomes, the concept of surrogate vs. clinical endpoints, and their importance in interpreting pharmacoepidemiologic research.

1. In pharmacoepidemiology, an “outcome” is best defined as:

• a. The drug or exposure being studied.
• b. The population in which the study is conducted.
• c. The health event or state of interest that is measured in relation to an exposure.
• d. The statistical analysis plan.

Answer: c. The health event or state of interest that is measured in relation to an exposure.

2. Which of the following is considered a “hard” clinical outcome?

• a. Lowering LDL cholesterol
• b. Reducing blood pressure
• c. Improving A1c
• d. Prevention of a myocardial infarction

Answer: d. Prevention of a myocardial infarction

3. A surrogate outcome is:

• a. A direct measure of how a patient feels, functions, or survives.
• b. A laboratory measure or physical sign that is used as a substitute for a clinically meaningful endpoint.
• c. Always a reliable predictor of the clinical outcome.
• d. The primary focus of post-marketing surveillance.

Answer: b. A laboratory measure or physical sign that is used as a substitute for a clinically meaningful endpoint.

4. A new drug for osteoporosis is shown to significantly increase bone mineral density (BMD). In this context, BMD is what type of outcome?

• a. A clinical outcome
• b. A humanistic outcome
• c. A surrogate outcome
• d. An economic outcome

Answer: c. A surrogate outcome

5. The ECHO model is a framework for classifying outcomes in pharmacoeconomics and pharmacoepidemiology. The “H” stands for:

• a. Hospitalization
• b. Humanistic
• c. Health
• d. Hematologic

Answer: b. Humanistic

6. The lecture “Clinical versus surrogate outcomes” is a specific topic in which course?

• a. PHA5244 Principles of Evidence-Based Practice
• b. PHA5104 Sterile Compounding
• c. PHA5703 Pharmacy Law and Ethics
• d. PHA5787C Patient Care 5

Answer: a. PHA5244 Principles of Evidence-Based Practice

7. A patient’s score on a quality-of-life survey is an example of what type of outcome?

• a. Clinical
• b. Economic
• c. Surrogate
• d. Humanistic (Patient-Reported Outcome)

Answer: d. Humanistic (Patient-Reported Outcome)

8. The total cost of hospitalizations and medications for a disease is what type of outcome?

• a. Clinical
• b. Economic
• c. Humanistic
• d. Surrogate

Answer: b. Economic

9. What is the main advantage of using a surrogate outcome in a clinical trial?

• a. They are always more clinically relevant than hard outcomes.
• b. They can be measured sooner and with a smaller sample size than waiting for a clinical endpoint.
• c. They are free from bias.
• d. They do not require statistical analysis.

Answer: b. They can be measured sooner and with a smaller sample size than waiting for a clinical endpoint.

10. A major limitation of relying on surrogate outcomes is that:

• a. A drug’s effect on a surrogate endpoint does not always translate to an improvement in a true clinical outcome.
• b. They are difficult to measure.
• c. They are very expensive to measure.
• d. They are not recognized by the FDA.

Answer: a. A drug’s effect on a surrogate endpoint does not always translate to an improvement in a true clinical outcome.

11. The appraisal of studies, including their outcomes, is a specific “Transcending Concept” in the Patient Care 5 curriculum.

• a. True
• b. False

Answer: a. True

12. The primary outcome of interest in pharmacoepidemiology safety studies is often:

• a. The cost of the drug.
• b. The incidence of adverse drug events (ADEs).
• c. Patient satisfaction.
• d. The drug’s mechanism of action.

Answer: b. The incidence of adverse drug events (ADEs).

13. A study measures the prevalence of hypertension in a population at a single point in time. What is the outcome?

• a. The incidence of hypertension.
• b. The prevalence of hypertension.
• c. The risk of developing hypertension.
• d. The change in blood pressure.

Answer: b. The prevalence of hypertension.

14. In a cohort study, the outcome should ideally be measured:

• a. Only in the exposed group.
• b. Using different methods for the exposed and unexposed groups.
• c. Using the same, valid, and reliable method in both the exposed and unexposed groups.
• d. By asking participants to self-diagnose.

Answer: c. Using the same, valid, and reliable method in both the exposed and unexposed groups.

15. Reporting adverse drug events is a key objective for student pharmacists in their experiential rotations.

• a. True
• b. False

Answer: a. True

16. Which of the following is an example of a patient-reported outcome (PRO)?

• a. A serum creatinine level.
• b. A change in tumor size on a CT scan.
• c. A patient’s rating of their pain on a 1-10 scale.
• d. A blood pressure reading.

Answer: c. A patient’s rating of their pain on a 1-10 scale.

17. The “Pharmacoepidemiology Study Designs” module is part of the EBP course.

• a. True
• b. False

Answer: a. True

18. A study’s outcome definition must be:

• a. Broad and vague.
• b. Decided after the data is analyzed.
• c. Specific, objective, and clearly defined before the study begins.
• d. The same for every study.

Answer: c. Specific, objective, and clearly defined before the study begins.

19. Why is “all-cause mortality” considered a “hard” and objective clinical outcome?

• a. It is easy to measure and not subject to interpretation bias.
• b. It reflects the overall impact of an intervention on survival.
• c. Both a and b.
• d. It is a surrogate endpoint.

Answer: c. Both a and b.

20. A “Journal Club” is an activity where students practice critically appraising all parts of a study, including its outcomes.

• a. True
• b. False

Answer: a. True

21. A study finds a new drug lowers A1c by 1%. The reduction in A1c is the _____, while the potential future reduction in retinopathy is the _____.

• a. clinical outcome, surrogate outcome
• b. surrogate outcome, clinical outcome
• c. economic outcome, humanistic outcome
• d. humanistic outcome, economic outcome

Answer: b. surrogate outcome, clinical outcome

22. “Outcome ascertainment” refers to the process of:

• a. Choosing the outcome to study.
• b. Statistically analyzing the outcome data.
• c. Systematically identifying and verifying the occurrence of the outcome in the study participants.
• d. Defining the study population.

Answer: c. Systematically identifying and verifying the occurrence of the outcome in the study participants.

23. “Experimental Studies” and “Observational Studies” are modules in the EBP course.

• a. True
• b. False

Answer: a. True

24. An active learning session on EBP is part of which course?

• a. PHA5244 Principles of Evidence-Based Practice
• b. PHA5163L Professional Skills Lab 3
• c. PHA5781 Patient Care I
• d. PHA5787C Patient Care 5

Answer: a. PHA5244 Principles of Evidence-Based Practice

25. A pharmacist reading a study must ask if the outcome measured is important to:

• a. The study authors only.
• b. The drug manufacturer only.
• c. The pharmacist only.
• d. The patient.

Answer: d. The patient.

26. A study uses hospital admission for heart failure as its primary outcome. This is an example of a(n):

• a. Clinical outcome
• b. Surrogate outcome
• c. Economic outcome
• d. Humanistic outcome

Answer: a. Clinical outcome

27. Using a very sensitive but non-specific case definition for an outcome can lead to:

• a. A smaller sample size.
• b. Misclassification bias.
• c. A decrease in confounding.
• d. A perfect study.

Answer: b. Misclassification bias.

28. An active learning session on appraising cohort studies is part of which course module?

• a. Module 5: Urological Disorders
• b. Module 1: Diabetes Mellitus
• c. Module 4: Medication Safety
• d. Module 8: Men’s Health

Answer: a. Module 5: Urological Disorders

29. The “C” in the ECHO model stands for:

• a. Confounding
• b. Case-control
• c. Clinical
• d. Cost

Answer: c. Clinical

30. The “O” in the ECHO model stands for:

• a. Observational
• b. Odds Ratio
• c. Outcomes
• d. It does not exist; the model is ECH.

Answer: c. Outcomes

31. In a pharmacoepidemiology study using a claims database, an outcome like “myocardial infarction” is often identified using:

• a. Patient interviews
• b. A physical exam
• c. ICD-10 diagnostic codes
• d. A review of the patient’s will

Answer: c. ICD-10 diagnostic codes

32. A limitation of using diagnostic codes to ascertain outcomes is that:

• a. They are always 100% accurate.
• b. They can be incorrect or used for “rule-out” purposes rather than a confirmed diagnosis.
• c. They are too specific.
• d. They are not standardized.

Answer: b. They can be incorrect or used for “rule-out” purposes rather than a confirmed diagnosis.

33. The principles of pharmacoepidemiology are foundational to understanding outcomes research.

• a. True
• b. False

Answer: a. True

34. A “composite outcome” in a clinical trial is:

• a. A single, specific endpoint.
• b. An outcome that combines several individual clinical endpoints into one group.
• c. Always a surrogate outcome.
• d. An outcome that is not pre-specified.

Answer: b. An outcome that combines several individual clinical endpoints into one group.

35. A pharmacist’s ability to critically appraise a study’s outcomes is essential for:

• a. Applying the results to their patient population.
• b. Understanding the true implications of the study.
• c. Avoiding being misled by marketing claims that focus on surrogate endpoints.
• d. All of the above.

Answer: d. All of the above.

36. A study on a new weight loss drug reports the primary outcome as “pounds lost.” This is a:

• a. Surrogate outcome
• b. Clinical outcome
• c. Economic outcome
• d. Both a and b could be argued.

Answer: a. Surrogate outcome

37. Which of the following is a surrogate outcome for a new diabetes drug?

• a. Reduction in nephropathy
• b. Reduction in cardiovascular death
• c. Reduction in A1c
• d. Reduction in retinopathy

Answer: c. Reduction in A1c

38. The lecture on clinical versus surrogate outcomes is part of the EBP curriculum.

• a. True
• b. False

Answer: a. True

39. A “patient-centered outcome” is one that:

• a. Matters directly to a patient’s quality of life or survival.
• b. Is easy for researchers to measure.
• c. Is always a lab value.
• d. Is chosen by the pharmaceutical company.

Answer: a. Matters directly to a patient’s quality of life or survival.

40. An active learning session covering EBP is part of which course?

• a. PHA5244 Principles of Evidence-Based Practice
• b. PHA5163L Professional Skills Lab 3
• c. PHA5781 Patient Care I
• d. PHA5787C Patient Care 5

Answer: a. PHA5244 Principles of Evidence-Based Practice

41. The number of asthma-related emergency department visits is what type of outcome?

• a. Humanistic
• b. Surrogate
• c. Clinical
• d. Economic

Answer: c. Clinical

42. Why is it important that an outcome assessor in an RCT is blinded to the treatment allocation?

• a. To prevent bias in how the outcome is measured or reported.
• b. It is not important.
• c. To ensure the patient is adherent.
• d. To control for confounding.

Answer: a. To prevent bias in how the outcome is measured or reported.

43. A pharmacist counseling a patient is managing their therapy to achieve specific:

• a. Financial goals
• b. Research objectives
• c. Therapeutic outcomes
• d. Publication targets

Answer: c. Therapeutic outcomes

44. A study’s chosen outcome must be clinically relevant to the disease being studied.

• a. True
• b. False

Answer: a. True

45. Which of the following is an example of a humanistic outcome?

• a. Patient satisfaction with therapy.
• b. The incidence of stroke.
• c. The cost of a hospitalization.
• d. A change in blood pressure.

Answer: a. Patient satisfaction with therapy.

46. A study using a scale to measure a patient’s depression symptoms is using what kind of outcome?

• a. Economic
• b. Patient-Reported Outcome (PRO)
• c. Clinical lab value
• d. A mortality outcome

Answer: b. Patient-Reported Outcome (PRO)

47. A study on a new heart failure drug that only reports changes in BNP (a lab value) without reporting on hospitalizations or mortality is relying on a(n):

• a. Clinical outcome
• b. Surrogate outcome
• c. Economic outcome
• d. Perfect outcome

Answer: b. Surrogate outcome

48. An active learning session on study design is part of which course module?

• a. Module 2: Pharmacoepidemiology Study Designs
• b. Module 1: Formulating a Clinical Question
• c. Module 6: Summarizing the Evidence
• d. Module 3: Applying Biostatistics

Answer: a. Module 2: Pharmacoepidemiology Study Designs

49. The overall goal of pharmacoepidemiologic research is to generate evidence about drug-related outcomes in real-world populations to:

• a. Promote the use of more expensive drugs.
• b. Improve public health and patient safety.
• c. Make clinical trials obsolete.
• d. Increase the regulatory burden.

Answer: b. Improve public health and patient safety.

50. The ultimate reason for a pharmacist to understand outcomes in pharmacoepidemiology is to:

• a. Critically evaluate whether a study’s results are meaningful for their patients.
• b. Pass the EBP final exam.
• c. Be able to design their own study.
• d. Memorize the ECHO model.

Answer: a. Critically evaluate whether a study’s results are meaningful for their patients.