Evidence-based practice is the cornerstone of modern pharmacy, requiring practitioners to expertly evaluate biomedical literature to make informed patient care decisions. For PharmD students, the ability to dissect a clinical trial, interpret statistics, and identify potential bias is a non-negotiable skill. This quiz will test your understanding of study designs, biostatistics, and the principles of critical appraisal needed to distinguish high-quality evidence from flawed research.
1. In the hierarchy of evidence, which of the following study types generally provides the strongest level of evidence for a therapeutic intervention?
- Case-control study
- Randomized controlled trial
- Systematic review or meta-analysis of RCTs
- Prospective cohort study
Answer: Systematic review or meta-analysis of RCTs
2. A study follows two groups of patients, one that received a new diabetes drug and one that received a placebo, forward in time to measure the incidence of cardiovascular events. What is this study design called?
- Case report
- Randomized Controlled Trial (RCT)
- Cross-sectional study
- Retrospective cohort study
Answer: Randomized Controlled Trial (RCT)
3. A researcher identifies a group of patients with a rare form of cancer and another group without the cancer. They then look back in time to compare past exposures to a specific chemical. This is an example of a:
- Case-control study.
- Randomized controlled trial.
- Prospective cohort study.
- Case series.
Answer: Case-control study.
4. A study reports a p-value of 0.03 for the primary endpoint. Assuming an alpha of 0.05, what is the correct interpretation?
- The result is not statistically significant, and the null hypothesis should be accepted.
- The result is statistically significant, and the null hypothesis is rejected.
- There is a 3% chance that the results are correct.
- The study has low statistical power.
Answer: The result is statistically significant, and the null hypothesis is rejected.
5. A clinical trial for a new blood pressure medication reports that the drug lowers systolic BP by an average of 5 mmHg with a 95% Confidence Interval of [2 mmHg, 8 mmHg]. What does this CI mean?
- There is a 95% probability that the patient’s blood pressure will drop by exactly 5 mmHg.
- There is a 95% confidence that the true mean difference in the population lies between 2 mmHg and 8 mmHg.
- The study results are not statistically significant.
- 95% of patients in the study had a blood pressure reduction between 2 mmHg and 8 mmHg.
Answer: There is a 95% confidence that the true mean difference in the population lies between 2 mmHg and 8 mmHg.
6. A study finds that a new drug reduces the risk of an adverse event from 4% in the placebo group to 2% in the treatment group. What is the Number Needed to Treat (NNT) to prevent one adverse event?
- 2
- 25
- 50
- 100
Answer: 50
7. In a clinical trial, “blinding” or “masking” is used to reduce which type of bias?
- Confounding bias
- Selection bias
- Attrition bias
- Measurement or performance bias
Answer: Measurement or performance bias
8. An “intention-to-treat” (ITT) analysis includes data from:
- Only the patients who completed the study according to the protocol.
- All patients who were randomized to a treatment group, regardless of whether they completed the study or adhered to the therapy.
- Only the patients who experienced the primary outcome.
- Only the patients in the placebo group.
Answer: All patients who were randomized to a treatment group, regardless of whether they completed the study or adhered to the therapy.
9. What is a surrogate endpoint in a clinical trial?
- The primary clinical outcome of greatest importance to the patient (e.g., survival).
- A laboratory measurement or physical sign used as a substitute for a clinically meaningful endpoint (e.g., blood pressure as a surrogate for stroke).
- The secondary outcome of the study.
- An outcome that is measured by the patient themselves.
Answer: A laboratory measurement or physical sign used as a substitute for a clinically meaningful endpoint (e.g., blood pressure as a surrogate for stroke).
10. “External validity” of a study refers to:
- The degree to which the study was conducted without bias.
- The statistical significance of the results.
- The generalizability of the study results to other populations, settings, or times.
- The proper randomization of study participants.
Answer: The generalizability of the study results to other populations, settings, or times.
11. A meta-analysis is a statistical technique used to:
- Combine the results of multiple studies to produce a single, more precise estimate of effect.
- Describe a single, interesting patient case.
- Follow a large group of people over time.
- Compare two different drug formularies.
Answer: Combine the results of multiple studies to produce a single, more precise estimate of effect.
12. In a forest plot from a meta-analysis, if the diamond representing the overall summary estimate does not touch the line of no effect (null value), the result is considered:
- Clinically insignificant.
- Statistically insignificant.
- Statistically significant.
- Invalid due to heterogeneity.
Answer: Statistically significant.
13. Selection bias occurs when:
- The method of choosing subjects for a study leads to a sample that is not representative of the population.
- Measurements are taken incorrectly.
- Patients drop out of the study unevenly between groups.
- The researchers are not blinded to the treatment allocation.
Answer: The method of choosing subjects for a study leads to a sample that is not representative of the population.
14. Which document provides a structured framework and checklist for reporting randomized controlled trials?
- The Belmont Report
- The CONSORT Statement
- The PRISMA Statement
- The STROBE Statement
Answer: The CONSORT Statement
15. A Type I error in hypothesis testing is when a researcher:
- Correctly rejects the null hypothesis.
- Correctly fails to reject the null hypothesis.
- Incorrectly rejects a true null hypothesis (a false positive).
- Incorrectly fails to reject a false null hypothesis (a false negative).
Answer: Incorrectly rejects a true null hypothesis (a false positive).
16. In a study comparing Drug A to Drug B, the Relative Risk (RR) for a negative outcome was 0.75 (95% CI [0.60, 0.95]). How is this interpreted?
- Drug A is associated with a 75% increased risk of the outcome.
- Drug A is associated with a 25% reduction in the risk of the outcome compared to Drug B.
- The result is not statistically significant.
- Drug A is associated with a 75% reduction in the risk of the outcome.
Answer: Drug A is associated with a 25% reduction in the risk of the outcome compared to Drug B.
17. An odds ratio (OR) of 1.0 indicates that:
- The exposure is associated with a 100% increased risk of the outcome.
- The exposure is protective.
- There is no association between the exposure and the outcome.
- The study results are invalid.
Answer: There is no association between the exposure and the outcome.
18. What is a primary limitation of a case report as a form of evidence?
- It provides strong evidence for causation.
- It is not possible to generalize the findings from a single patient.
- It involves a large number of subjects.
- It is always a prospective study.
Answer: It is not possible to generalize the findings from a single patient.
19. A study’s power is the probability of:
- Making a Type I error.
- Making a Type II error.
- Avoiding a Type I error.
- Correctly rejecting a false null hypothesis (i.e., detecting a true effect).
Answer: Correctly rejecting a false null hypothesis (i.e., detecting a true effect).
20. A “non-inferiority” trial is designed to show that a new treatment is:
- Superior to the standard treatment.
- Not unacceptably worse than the standard treatment.
- Equivalent to a placebo.
- The most cost-effective option available.
Answer: Not unacceptably worse than the standard treatment.
21. “Confounding” occurs when:
- A third variable is associated with both the exposure and the outcome, distorting the true relationship between them.
- The results of a study are not statistically significant.
- Patients are lost to follow-up.
- The researchers are aware of the treatment allocation.
Answer: A third variable is associated with both the exposure and the outcome, distorting the true relationship between them.
22. Which is an example of nominal data?
- Patient’s temperature in Celsius.
- Pain score on a scale of 1 to 10.
- Patient’s blood type (A, B, AB, O).
- Patient’s weight in kilograms.
Answer: Patient’s blood type (A, B, AB, O).
23. The statistical test most appropriate for comparing the mean blood pressure between two independent groups is the:
- Chi-square test
- Paired t-test
- Independent samples t-test
- ANOVA
Answer: Independent samples t-test
24. A cross-sectional study measures:
- The incidence of a new disease over time.
- The outcomes of a new intervention.
- Exposures and outcomes at a single point in time.
- Past exposures in patients with a known disease.
Answer: Exposures and outcomes at a single point in time.
25. Recall bias is a major concern in which type of study design?
- Randomized controlled trial
- Prospective cohort study
- Retrospective case-control study
- Meta-analysis
Answer: Retrospective case-control study
26. The PICO format helps a clinician formulate an answerable clinical question. The “C” in PICO stands for:
- Causation
- Correlation
- Comparison or Control
- Conclusion
Answer: Comparison or Control
27. Clinical practice guidelines are designed to:
- Be followed rigidly without any clinical judgment.
- Provide recommendations for optimizing patient care based on a systematic review of evidence.
- Replace the need for primary literature.
- Promote the use of the most expensive medications.
Answer: Provide recommendations for optimizing patient care based on a systematic review of evidence.
28. A key difference between a systematic review and a traditional narrative review is that a systematic review:
- Is based on the author’s opinion.
- Uses explicit, pre-specified methods to identify, select, and appraise relevant research.
- Includes only studies published in the last year.
- Never includes a statistical summary.
Answer: Uses explicit, pre-specified methods to identify, select, and appraise relevant research.
29. Attrition bias is a concern when:
- The study sample is not representative of the population.
- There are systematic differences in the loss of subjects between the study groups.
- The outcome is measured incorrectly.
- The study lacks a control group.
Answer: There are systematic differences in the loss of subjects between the study groups.
30. What does the term “statistical significance” mean?
- The observed effect is large and important to patients.
- The observed effect is unlikely to be due to random chance alone.
- The study was conducted perfectly without any bias.
- The treatment is 100% effective.
Answer: The observed effect is unlikely to be due to random chance alone.
31. The primary purpose of randomization in an RCT is to:
- Make the study groups comparable with respect to known and unknown confounding variables.
- Ensure that the researchers can choose which patients go into which group.
- Make the study less expensive to conduct.
- Guarantee the results will be positive.
Answer: Make the study groups comparable with respect to known and unknown confounding variables.
32. A per-protocol analysis is more likely than an intention-to-treat analysis to:
- Underestimate the effect of a drug.
- Overestimate the efficacy of a drug by excluding non-adherent patients.
- Be the preferred primary analysis for a superiority trial.
- Include all randomized patients regardless of adherence.
Answer: Overestimate the efficacy of a drug by excluding non-adherent patients.
33. The GRADE system is used in clinical guidelines to:
- Grade the quality of the evidence and the strength of the recommendations.
- Rank journals based on their impact factor.
- Score medical students on their exams.
- Determine the cost-effectiveness of a drug.
Answer: Grade the quality of the evidence and the strength of the recommendations.
34. Publication bias in a meta-analysis refers to the phenomenon that:
- Studies with positive or statistically significant results are more likely to be published than those with negative results.
- Journals only publish articles from famous authors.
- Only studies written in English are included.
- All studies on a topic are published with equal probability.
Answer: Studies with positive or statistically significant results are more likely to be published than those with negative results.
35. A “drug evaluation monograph” is primarily used by:
- Patients to learn about side effects.
- Pharmacy and Therapeutics (P&T) committees to make formulary decisions.
- The FDA during the drug approval process.
- Pharmaceutical companies for marketing.
Answer: Pharmacy and Therapeutics (P&T) committees to make formulary decisions.
36. A clinically important difference is:
- Any difference that has a p-value less than 0.05.
- A change in a surrogate marker.
- A difference in outcome that is meaningful to the patient, regardless of its statistical significance.
- Always the same as the statistically significant difference.
Answer: A difference in outcome that is meaningful to the patient, regardless of its statistical significance.
37. When critically appraising a study, the first question to ask should be about the study’s:
- Internal validity.
- Cost.
- Author credentials.
- Publication date.
Answer: Internal validity.
38. Which type of database would you use to find primary literature articles?
- A tertiary resource like a textbook.
- A secondary resource like PubMed or Embase.
- A drug manufacturer’s website.
- A social media platform.
Answer: A secondary resource like PubMed or Embase.
39. A “washout period” is used in which type of study design?
- Parallel RCT
- Cross-sectional study
- Crossover RCT
- Case-control study
Answer: Crossover RCT
40. The measure of central tendency that is most affected by extreme outliers is the:
- Mean
- Median
- Mode
- Range
Answer: Mean
41. The strength of a prospective cohort study is that it can:
- Establish causality definitively.
- Be completed quickly and inexpensively.
- Determine the incidence of a disease and investigate potential risk factors.
- Avoid any loss to follow-up.
Answer: Determine the incidence of a disease and investigate potential risk factors.
42. A conflict of interest in a clinical trial is a concern because it may:
- Improve the validity of the study.
- Introduce bias into the study’s design, conduct, or reporting.
- Reduce the cost of the study.
- It is not a concern.
Answer: Introduce bias into the study’s design, conduct, or reporting.
43. A hazard ratio (HR) of 0.80 for mortality means that at any point in time, the treatment group had:
- An 80% higher rate of death than the control group.
- A 20% higher rate of death than the control group.
- A 20% lower rate of death than the control group.
- An 80% lower rate of death than the control group.
Answer: A 20% lower rate of death than the control group.
44. What is a key limitation of using a surrogate endpoint like cholesterol levels instead of a clinical endpoint like myocardial infarction?
- Surrogate endpoints are more difficult to measure.
- A change in the surrogate may not always translate to a meaningful clinical benefit.
- Surrogate endpoints are more meaningful to patients.
- Clinical endpoints are less reliable.
Answer: A change in the surrogate may not always translate to a meaningful clinical benefit.
45. Which of the following is a tertiary source of drug information?
- A randomized controlled trial published in the NEJM.
- A textbook chapter summarizing the treatment of hypertension.
- A meta-analysis of multiple clinical trials.
- A case report about a single patient.
Answer: A textbook chapter summarizing the treatment of hypertension.
46. When evaluating an article, you find that the baseline characteristics between the treatment and control groups are significantly different. This indicates a potential failure of:
- Blinding
- Randomization
- Statistical analysis
- The primary endpoint measurement
Answer: Randomization
47. A “patient-reported outcome” (PRO) is a health outcome that is:
- Measured directly by a laboratory test.
- Based on a clinician’s physical exam.
- Reported directly from the patient without interpretation by a clinician.
- Always a surrogate marker.
Answer: Reported directly from the patient without interpretation by a clinician.
48. Sensitivity of a diagnostic test refers to its ability to:
- Correctly identify those who do not have the disease.
- Correctly identify those who have the disease.
- Provide a result quickly.
- Work in all patient populations.
Answer: Correctly identify those who have the disease.
49. Specificity of a diagnostic test refers to its ability to:
- Correctly identify those who do not have the disease.
- Correctly identify those who have the disease.
- Be performed by any healthcare professional.
- Use a small amount of blood sample.
Answer: Correctly identify those who do not have the disease.
50. The ultimate goal of evidence evaluation in pharmacy practice is to:
- Find flaws in every published study.
- Memorize p-values and confidence intervals.
- Apply the best available evidence to make informed decisions for individual patient care.
- Rely solely on personal experience.
Answer: Apply the best available evidence to make informed decisions for individual patient care.

I am a Registered Pharmacist under the Pharmacy Act, 1948, and the founder of PharmacyFreak.com. I hold a Bachelor of Pharmacy degree from Rungta College of Pharmaceutical Science and Research. With a strong academic foundation and practical knowledge, I am committed to providing accurate, easy-to-understand content to support pharmacy students and professionals. My aim is to make complex pharmaceutical concepts accessible and useful for real-world application.
Mail- Sachin@pharmacyfreak.com