MCQ Quiz: Comparative Effectiveness Research (CER)

Comparative Effectiveness Research (CER) is designed to answer a crucial clinical question: “Which treatment works best, for whom, and under what circumstances?” Unlike traditional placebo-controlled trials that focus on efficacy, CER directly compares active treatment options to inform real-world medical decisions for patients, clinicians, and policymakers. Understanding the principles of CER, a key topic in the Principles of Evidence-Based Practice course, is essential for modern pharmacists. This quiz will test your knowledge on the study designs, purpose, and application of Comparative Effectiveness Research.

1. What is the primary goal of Comparative Effectiveness Research (CER)?

• a. To get a new drug approved by the FDA as quickly as possible.
• a. To generate evidence on the benefits and harms of alternative treatments to assist in decision-making.
• c. To determine the maximum tolerated dose of a new medication.
• d. To study the effects of a drug versus a placebo in a highly controlled setting.

Answer: b. To generate evidence on the benefits and harms of alternative treatments to assist in decision-making.

2. A key feature of CER is that it often involves what type of comparison?

• a. A new drug versus a placebo.
• b. A new drug versus an active, standard-of-care treatment (“head-to-head”).
• c. An active drug versus no treatment.
• d. A high dose versus a low dose of the same drug.

Answer: b. A new drug versus an active, standard-of-care treatment (“head-to-head”).

3. A “Pragmatic Clinical Trial” (PCT) is a type of RCT often used in CER because it aims to:

• a. Enroll a very specific, homogenous patient population.
• b. Be conducted under highly controlled, artificial conditions.
• c. Reflect real-world clinical practice by having broad inclusion criteria and comparing clinically relevant alternatives.
• d. Have a very short follow-up period.

Answer: c. Reflect real-world clinical practice by having broad inclusion criteria and comparing clinically relevant alternatives.

4. Which type of observational study is frequently used to conduct CER using large healthcare databases?

• a. Case report
• b. Case series
• c. Cohort study
• d. All of the above

Answer: c. Cohort study

5. The main difference between “efficacy” and “effectiveness” is that effectiveness refers to how a drug works:

• a. In a laboratory setting.
• b. In healthy volunteers.
• c. In a real-world clinical practice setting.
• d. In an animal model.

Answer: c. In a real-world clinical practice setting.

6. The “Introduction to Comparative Effectiveness Research (CER)” is a specific learning module 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. Which U.S. organization was established to fund patient-centered CER?

• a. The Food and Drug Administration (FDA)
• b. The National Institutes of Health (NIH)
• c. The Patient-Centered Outcomes Research Institute (PCORI)
• d. The Drug Enforcement Administration (DEA)

Answer: c. The Patient-Centered Outcomes Research Institute (PCORI)

8. A major challenge when conducting observational CER is:

• a. A lack of available data.
• b. The small sample sizes.
• c. The high potential for confounding by indication.
• d. The inability to measure costs.

Answer: c. The high potential for confounding by indication.

9. A study comparing two different statins using an insurance claims database would be an example of:

• a. A Phase I clinical trial
• b. A CER study using Real-World Data (RWD).
• c. A pre-clinical study
• d. A case report

Answer: b. A CER study using Real-World Data (RWD).

10. What is a key question that CER aims to answer?

• a. Does this drug work better than a placebo?
• b. What is the maximum tolerated dose of this drug?
• c. Is Drug A better than Drug B for a specific patient population in a real-world setting?
• d. What is the chemical structure of this drug?

Answer: c. Is Drug A better than Drug B for a specific patient population in a real-world setting?

11. The appraisal of cohort studies, a common CER design, is a “Transcending Concept” in the Patient Care 5 curriculum.

• a. True
• b. False

Answer: a. True

12. Which of the following is NOT a primary goal of CER?

• a. To assess the comparative effectiveness of different treatments.
• b. To inform decision-making for patients and clinicians.
• c. To focus exclusively on the benefits of a new drug without considering its harms.
• d. To compare the effectiveness of treatments in different subgroups of patients.

Answer: c. To focus exclusively on the benefits of a new drug without considering its harms.

13. A systematic review or meta-analysis that combines the results of multiple “head-to-head” trials is a powerful form of:

• a. Primary literature
• b. Anecdotal evidence
• c. Comparative Effectiveness Research
• d. A case-control study

Answer: c. Comparative Effectiveness Research

14. A pharmacist on a P&T committee would use CER to:

• a. Determine the most cost-effective and clinically effective agent among alternatives for the hospital formulary.
• b. Decide on the pharmacy’s staffing schedule.
• c. Compound a sterile preparation.
• d. Report an adverse drug reaction.

Answer: a. Determine the most cost-effective and clinically effective agent among alternatives for the hospital formulary.

15. “Journal Club” is an activity where students would practice appraising CER studies.

• a. True
• b. False

Answer: a. True

16. An “active comparator” is typically used in which type of research?

• a. A placebo-controlled trial
• b. Comparative Effectiveness Research
• c. A Phase I safety trial
• d. A bioequivalence study

Answer: b. Comparative Effectiveness Research

17. The “Observational Studies” module is part of the EBP course.

• a. True
• b. False

Answer: a. True

18. Why might a pragmatic clinical trial have broader inclusion criteria than a traditional RCT?

• a. To make the study results more generalizable to a real-world patient population.
• b. To make the study more difficult to enroll.
• c. To increase the internal validity of the study.
• d. To test the drug in the healthiest possible population.

Answer: a. To make the study results more generalizable to a real-world patient population.

19. A key role of a pharmacist is to be a critical consumer of CER literature to guide therapeutic recommendations.

• a. True
• b. False

Answer: a. True

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 study that compares the use of an ACE inhibitor versus an ARB for preventing kidney disease in patients with diabetes is an example of:

• a. CER
• b. A drug discovery study
• c. A toxicology study
• d. A marketing study

Answer: a. CER

22. Which of the following outcomes is LEAST likely to be the primary focus of a CER study?

• a. Reduction in mortality
• b. Improvement in quality of life
• c. The effect on a novel biomarker
• d. Reduction in hospitalizations

Answer: c. The effect on a novel biomarker

23. The “Experimental Studies” module, which describes RCTs, is part of 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 major reason for the growth of CER is:

• a. The need to make informed decisions about how to allocate healthcare resources effectively.
• b. The desire to only use brand-name drugs.
• c. The fact that RCTs answer all possible clinical questions.
• d. The lack of new drugs being developed.

Answer: a. The need to make informed decisions about how to allocate healthcare resources effectively.

26. Patient-Centered Outcomes Research (PCOR) emphasizes:

• a. Outcomes that are important to researchers only.
• b. Outcomes that matter most to patients, such as quality of life and functional status.
• c. Only laboratory-based outcomes.
• d. Only economic outcomes.

Answer: b. Outcomes that matter most to patients, such as quality of life and functional status.

27. A limitation of using observational RWD for CER is the potential for unmeasured:

• a. Confounders
• b. Bias
• c. Data errors
• d. All of the above

Answer: d. All of the above

28. 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 3: Applying Biostatistics

Answer: a. Module 2: Pharmacoepidemiology Study Designs

29. The term “head-to-head” trial implies a comparison between:

• a. A drug and a placebo.
• b. A drug and no treatment.
• c. Two or more active treatments.
• d. A high dose and a low dose.

Answer: c. Two or more active treatments.

30. The “Introduction to Comparative Effectiveness Research” is a required reading in the EBP course.

• a. True
• b. False

Answer: a. True

31. A pharmacist explaining the pros and cons of two different types of inhalers for a patient’s asthma is applying the principles of:

• a. Compounding
• b. Individualized comparative effectiveness.
• c. Drug distribution.
• d. Pharmacogenomics.

Answer: b. Individualized comparative effectiveness.

32. What is the role of systematic reviews in CER?

• a. They are not used in CER.
• b. To combine and synthesize the evidence from multiple studies comparing different interventions.
• c. To conduct a new clinical trial.
• d. To report a single patient case.

Answer: b. To combine and synthesize the evidence from multiple studies comparing different interventions.

33. The principles of EBP are foundational to understanding CER.

• a. True
• b. False

Answer: a. True

34. A key difference between a pragmatic trial and an explanatory trial (a typical RCT) is that a pragmatic trial prioritizes:

• a. Internal validity over external validity.
• b. External validity (generalizability) over internal validity.
• c. A placebo comparison.
• d. A very select patient population.

Answer: b. External validity (generalizability) over external validity.

35. A pharmacist is a key stakeholder in the process of:

• a. Generating CER through practice-based research.
• b. Disseminating CER findings to other providers.
• c. Applying CER findings to individual patient care.
• d. All of the above.

Answer: d. All of the above.

36. A CER study finds that a new, expensive biologic is only slightly more effective than an older, cheaper generic drug. This information is most useful for:

• a. Making a value-based formulary decision.
• b. The FDA approval process for the new drug.
• c. A Phase 1 clinical trial.
• d. Determining the mechanism of action.

Answer: a. Making a value-based formulary decision.

37. Statistical techniques like propensity score matching are used in observational CER to:

• a. Replicate the effects of randomization and reduce confounding.
• b. Increase the sample size.
• c. Simplify the data analysis.
• d. Blind the investigators.

Answer: a. Replicate the effects of randomization and reduce confounding.

38. The lecture on appraising cohort studies is part of the EBP curriculum.

• a. True
• b. False

Answer: a. True

39. A pharmacist reading a CER study must first:

• a. Accept the conclusion as fact.
• b. Critically appraise the study’s methods for bias and confounding.
• c. Implement the findings for all patients immediately.
• d. Ignore the results if they contradict personal experience.

Answer: b. Critically appraise the study’s methods for bias and confounding.

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 results from a CER study are intended to inform, not dictate, clinical decisions.

• a. True
• b. False

Answer: a. True

42. Which of the following is LEAST likely to be a source of data for a CER study?

• a. A national patient registry.
• b. An insurance claims database.
• c. A pre-clinical animal study.
• d. A pragmatic clinical trial.

Answer: c. A pre-clinical animal study.

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. A key question in a CER study comparing a new oral anticoagulant to warfarin would be:

• a. Is the new drug superior to placebo?
• b. What is the maximum tolerated dose of the new drug?
• c. How does the new drug’s balance of stroke prevention and bleeding risk compare to warfarin in a real-world population?
• d. What is the chemical structure of the new drug?

Answer: c. How does the new drug’s balance of stroke prevention and bleeding risk compare to warfarin in a real-world population?

45. Why is “effectiveness” a key word in CER?

• a. It implies a focus on how a treatment works under ideal, controlled conditions.
• b. It implies a focus on how a treatment works in typical, real-world conditions with average patient adherence.
• c. It is a synonym for cost.
• d. It is a synonym for safety.

Answer: b. It implies a focus on how a treatment works in typical, real-world conditions with average patient adherence.

46. A study comparing two different pharmacist-led MTM services to see which leads to better patient outcomes is a form of CER.

• a. True
• b. False

Answer: a. True

47. “Summarizing the Evidence” is a module in the EBP course that would cover systematic reviews used for CER.

• a. True
• b. False

Answer: a. True

48. An active learning session on CER 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 CER is to:

• a. Prove that new drugs are always better than old drugs.
• b. Provide patients, clinicians, and payers with the evidence needed to make informed and value-based healthcare decisions.
• c. Limit the number of available treatment options.
• d. Increase the cost of healthcare.

Answer: b. Provide patients, clinicians, and payers with the evidence needed to make informed and value-based healthcare decisions.

50. The ultimate reason for a pharmacist to learn about CER is to:

• a. Be able to recommend the most effective and appropriate therapy for their individual patients based on the best available evidence.
• b. Pass the EBP final exam.
• c. Design and conduct their own CER studies.
• d. Only recommend the cheapest medications.

Answer: a. Be able to recommend the most effective and appropriate therapy for their individual patients based on the best available evidence.