MCQ Quiz: Endpoints in Pharmacoepidemiology

The choice of an endpoint is one of the most critical decisions in designing a clinical or pharmacoepidemiologic study, as it determines what is being measured and how the results will be interpreted. Pharmacists must be adept at scrutinizing study endpoints to differentiate between a change in a lab value (a surrogate endpoint) and a true clinical benefit for the patient. This quiz focuses on the application and interpretation of different study endpoints

1. In a clinical study, an “endpoint” refers to:

• a. The final day of the study.
• b. The conclusion written by the authors.
• c. A pre-defined event or outcome that is measured to determine the effect of an intervention.
• d. The statistical analysis method.

Answer: c. A pre-defined event or outcome that is measured to determine the effect of an intervention.

2. A study of a new antihypertensive drug uses “change in systolic blood pressure from baseline” as its primary endpoint. This is an example of a(n):

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

Answer: b. Surrogate endpoint

3. Which of the following is the best example of a “hard” clinical endpoint?

• a. Change in LDL cholesterol
• b. All-cause mortality
• c. Change in bone mineral density
• d. Change in A1c

Answer: b. All-cause mortality

4. A “composite endpoint” is used in a clinical trial to:

• a. Make the study more difficult to interpret.
• b. Increase the number of events, thereby increasing the statistical power of the study.
• c. Measure only the most important outcome.
• d. Blind the investigators to the treatment.

Answer: b. Increase the number of events, thereby increasing the statistical power of the study.

5. A cardiovascular trial uses a composite endpoint of “MACE” (Major Adverse Cardiovascular Events), which includes CV death, non-fatal MI, and non-fatal stroke. When appraising this study, it is most important for the pharmacist to:

• a. Only look at the overall composite result.
• b. Examine the results for each individual component of the composite endpoint.
• c. Assume the benefit was equal across all components.
• d. Ignore the composite endpoint entirely.

Answer: b. Examine the results for each individual component of the composite endpoint.

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. The “surrogate endpoint fallacy” refers to instances where:

• a. A drug’s positive effect on a surrogate endpoint fails to translate into a real clinical benefit, or even causes harm.
• b. The surrogate endpoint is too difficult to measure.
• c. The surrogate endpoint is the same as the clinical endpoint.
• d. The clinical endpoint is not important to patients.

Answer: a. A drug’s positive effect on a surrogate endpoint fails to translate into a real clinical benefit, or even causes harm.

8. A Patient-Reported Outcome (PRO) is an endpoint that measures:

• a. A laboratory value.
• b. A physical sign.
• c. The patient’s perspective on their health status (e.g., quality of life, symptoms) without interpretation from a clinician.
• d. The cost of the intervention.

Answer: c. The patient’s perspective on their health status (e.g., quality of life, symptoms) without interpretation from a clinician.

9. A study uses a validated depression scale (e.g., PHQ-9) to measure the effect of a new antidepressant. The change in the PHQ-9 score is what type of endpoint?

• a. A surrogate endpoint
• b. A Patient-Reported Outcome
• c. An economic endpoint
• d. A biomarker

Answer: b. A Patient-Reported Outcome

10. A key strength of using a hard clinical endpoint like “stroke” is that it:

• a. Is directly meaningful to the patient.
• b. Can be measured very quickly.
• c. Requires a very small sample size.
• d. Is easy to influence with bias.

Answer: a. Is directly meaningful to the patient.

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

• a. True
• b. False

Answer: a. True

12. Why might a pharmaceutical company choose to use a surrogate endpoint for a pivotal Phase 3 trial?

• a. It allows for a smaller, shorter, and less expensive trial.
• b. It is more impressive to clinicians.
• c. It guarantees the drug is truly beneficial.
• d. The FDA requires the use of surrogate endpoints for all approvals.

Answer: a. It allows for a smaller, shorter, and less expensive trial.

13. A study’s primary endpoint must be:

• a. Chosen after the study is complete.
• b. A composite endpoint.
• c. A surrogate endpoint.
• d. Clearly defined and pre-specified in the study protocol.

Answer: d. Clearly defined and pre-specified in the study protocol.

14. A study investigating a new drug for heart failure reports a significant reduction in the composite endpoint of “cardiovascular death or hospitalization for heart failure.” The benefit was driven almost entirely by a reduction in hospitalizations. This finding is:

• a. Just as clinically important as a reduction in mortality.
• b. Less clinically impactful than if the benefit had been driven by a reduction in death.
• c. Invalid because the composite endpoint was used.
• d. A reason to stop using the drug.

Answer: b. Less clinically impactful than if the benefit had been driven by a reduction in death.

15. A “Journal Club” is an activity where students practice appraising studies, including their choice and measurement of endpoints.

• a. True
• b. False

Answer: a. True

16. The process of identifying and confirming cases of the endpoint in a study is called:

• a. Randomization
• b. Blinding
• c. Ascertainment
• d. Confounding

Answer: c. Ascertainment

17. “Observational Studies,” which often use RWD to measure endpoints, is a module in the EBP course.

• a. True
• b. False

Answer: a. True

18. If the outcome/endpoint assessors in a trial are not blinded to the treatment allocation, what type of bias can be introduced?

• a. Selection bias
• b. Detection bias
• c. Recall bias
• d. No bias will be introduced.

Answer: b. Detection bias

19. A study on a new chemotherapy agent uses “tumor shrinkage on an MRI” as its primary endpoint. This is a:

• a. Patient-reported outcome
• b. Hard clinical outcome
• c. Surrogate endpoint
• d. Humanistic outcome

Answer: c. Surrogate endpoint

20. 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

21. A drug that lowers LDL cholesterol (surrogate endpoint) will always reduce the risk of myocardial infarction (clinical endpoint).

• a. True
• b. False

Answer: b. False

22. A key role of the pharmacist when evaluating a clinical trial is to determine if the study’s primary endpoint is:

• a. Clinically relevant and important to patients.
• b. The easiest one to measure.
• c. The same as all other trials in that field.
• d. A secret until the study is published.

Answer: a. Clinically relevant and important to patients.

23. The “Experimental Studies” module is part of the EBP course.

• a. True
• b. False

Answer: a. True

24. An active learning session on appraising cohort studies is part of the Patient Care 5 curriculum.

• a. True
• b. False

Answer: a. True

25. A “secondary endpoint” in a clinical trial is:

• a. The most important outcome of the study.
• b. An additional outcome of interest that is pre-specified but is not the primary focus of the study.
• c. An outcome that is discovered after the study is over.
• d. Always a surrogate endpoint.

Answer: b. An additional outcome of interest that is pre-specified but is not the primary focus of the study.

26. The results for secondary endpoints should be interpreted with more caution than the primary endpoint, especially if the primary endpoint was not met.

• a. True
• b. False

Answer: a. True

27. Using all-cause mortality as an endpoint is beneficial because it avoids bias associated with:

• a. Determining the specific cause of death.
• b. The patient’s quality of life.
• c. The cost of the medication.
• d. The patient’s lab values.

Answer: a. Determining the specific cause of death.

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

• a. Module 4: Experimental Studies
• b. Module 1: Formulating a Clinical Question
• c. Module 6: Summarizing the Evidence
• d. Module 3: Applying Biostatistics

Answer: a. Module 4: Experimental Studies

29. The ECHO model classifies “lost work days” as what type of endpoint/outcome?

• a. Clinical
• b. Economic (indirect cost)
• c. Humanistic
• d. Surrogate

Answer: b. Economic (indirect cost)

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

• a. True
• b. False

Answer: a. True

31. A study on a new Alzheimer’s drug uses the score on a cognitive function test as its primary endpoint. This is a:

• a. Biomarker
• b. Hard clinical endpoint
• c. Clinical outcome scale (can be considered a type of PRO)
• d. Economic outcome

Answer: c. Clinical outcome scale (can be considered a type of PRO)

32. The choice of endpoints in a study can significantly influence:

• a. The study’s conclusions and clinical interpretation.
• b. The study’s duration and cost.
• c. The required sample size.
• d. All of the above.

Answer: d. All of the above.

33. An ideal surrogate endpoint should be:

• a. On the causal pathway of the disease.
• b. Highly correlated with the clinical endpoint.
• c. Responsive to the effects of the intervention.
• d. All of the above.

Answer: d. All of the above.

34. A pharmacist must be able to explain the difference between a surrogate and clinical endpoint to:

• a. Other healthcare professionals.
• b. Patients.
• c. P&T committee members.
• d. All of the above.

Answer: d. All of the above.

35. A “time-to-event” analysis is used when the primary endpoint is:

• a. A change in a continuous lab value.
• b. The occurrence of an event over time (e.g., time to first hospitalization).
• c. A patient’s quality of life score.
• d. The cost of care.

Answer: b. The occurrence of an event over time (e.g., time to first hospitalization).

36. A study’s primary endpoint was “change in blood pressure,” but the drug’s marketing focuses heavily on a secondary endpoint of “reduced risk of stroke,” which was not statistically significant. This is:

• a. An example of excellent scientific reporting.
• b. Potentially misleading.
• c. Standard practice.
• d. A reason to trust the marketing over the study.

Answer: b. Potentially misleading.

37. Reporting of adverse drug events is a key objective for pharmacy students and provides data for safety endpoints.

• a. True
• b. False

Answer: a. True

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

• a. True
• b. False

Answer: a. True

39. A well-defined endpoint is crucial for the _______ of a study.

• a. validity and reproducibility
• b. funding
• c. marketing
• d. length

Answer: a. validity and reproducibility

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. Which of the following is the most patient-centered endpoint?

• a. A change in a biomarker.
• b. A reduction in mortality.
• c. The cost of the drug.
• d. The mechanism of action.

Answer: b. A reduction in mortality.

42. The problem with using a composite endpoint is that the treatment may have a large effect on a ____ component but little to no effect on a ____ component.

• a. less serious, more serious
• b. more serious, less serious
• c. surrogate, clinical
• d. clinical, surrogate

Answer: a. less serious, more serious

43. A pharmacist’s ability to critically appraise all forms of evidence is a core professional competency.

• a. True
• b. False

Answer: a. True

44. If a study is “stopped early for benefit,” a pharmacist should be cautious because:

• a. The effect size may be overestimated.
• b. Long-term safety data will be limited.
• c. The decision to stop may have been premature.
• d. All of the above.

Answer: d. All of the above.

45. “Number of pills taken per month” could be used as what type of endpoint in a study?

• a. A clinical endpoint
• b. A surrogate endpoint for medication adherence.
• c. A humanistic endpoint
• d. A safety endpoint

Answer: b. A surrogate endpoint for medication adherence.

46. A study’s endpoints should be directly related to its:

• a. Funding source.
• b. Primary research question.
• c. The lead author’s previous work.
• d. The journal it will be published in.

Answer: b. The primary research question.

47. Understanding study design is a module in the EBP course.

• a. True
• b. False

Answer: a. True

48. An active learning session on EBP 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 4: Experimental Studies

Answer: d. Module 4: Experimental Studies

49. The overall goal of selecting endpoints in a clinical study is to:

• a. Ensure the study is as short as possible.
• b. Measure the most clinically relevant and important effects of the intervention in a valid and reliable way.
• c. Guarantee a statistically significant result.
• d. Make the study easy to analyze.

Answer: b. Measure the most clinically relevant and important effects of the intervention in a valid and reliable way.

50. The ultimate reason for a pharmacist to scrutinize study endpoints is to:

• a. Accurately assess the true clinical value of a therapy for their patients.
• b. Find fault with all research.
• c. Pass the EBP final exam.
• d. Lengthen their journal club presentation.

Answer: a. Accurately assess the true clinical value of a therapy for their patients.