Biostatistics and Pharmacoeconomics

1. In biostatistics, what is meant by the mean of a group of values? A. The calculated value by adding up all the numbers and subtracting by 100 B. The product of all the values divided by the number of values C. The value that occurs most frequently in the series D. The average E. The value in the middle of the list 2. In biostatistics, what is meant by the median in a group of values? A. The relative risk reduction B. The value in the middle of the list C. The average D. The variance squared. E. The value that occurs most frequently 3. In biostatistics, what is meant by the mode in a group of values? A. The value that corresponds to the 25th percentile B. The value used to calculate the standard deviation of the mean C. The value that occurs most frequently D. When the values are stated numerically in order, it is the value half way down the list. E. The value that corresponds to the 95th percentile 4. What is the term used for the inactive or inert substance administered to a patient that serves as a reference to determine the effects of a real drug or other intervention? A. The relative risk B. The standard deviation C. The null hypothesis D. The placebo E. The desiccant 5. Choose the name of the assertion that a clinical trial is designed to disprove which states there is no difference between two groups (Drug A = Drug B). A. The assertive hypothesis B. The null hypothesis C. The alternative hypothesis D. Type II error

E. The disclaimer

6. Choose the name of the term used to determine statistical significance in a trial: A. The Z-value B. The T score C. The p-value D. The hazard ratio E. The odds ratio 7. Two drug trials are conducted. Drug Trial I compared Drug A to a placebo and found that Drug A lowered blood pressure more than the placebo. This trial had a p-value of 0.04. Drug Trial II found that Drug B lowered blood pressure more than the placebo. This trial had a p-value of <0.01. Which trial is more likely to have a greater chance of providing benefit of the drug versus placebo? A. Drug Trial I, since it had a lower p-value B. Drug Trial I, since it had a higher p-value C. Drug Trial II, since it had a lower p-value D. Drug Trial II, since it had a higher p-value E. A trial comparing Drug A to Drug B would need to be conducted. 8. A trial compared Drug A to a placebo and found that Drug A lowered systolic blood pressure 3 mmHg and lowered diastolic blood pressure 1.5 mmHg, on average. The p-value was reported to be 0.03. This trial displayed the following characteristic/s: A. Statistical significance of Drug A versus placebo. B. A probable lack of clinical benefit C. A 50% decrease in blood pressure compared to placebo D. A and B E. All of the above 9. Marital status (married, single, divorced) can be described as this type of data: A. Ordinal B. Nominal C. Continuous D. Random E. None of the above

10. Blood pressure, hemoglobin A1C and LDL cholesterol can each be described as this type of data: A. Ordinal B. Nominal C. Continuous D. Random E. None of the above 11. What is the definition of a Type I error? A. The degree to which a study accurately reflects or assesses the specific concept that the researcher is attempting to measure but misses the mark. B. The null hypothesis is true, but is rejected in error. C. The study was not large enough to detect a meaningful difference between treatment groups. D. The null hypothesis is false, but is accepted in error. E. A clinical trial with too many confounding variables. 12. A clinical trial is conducted on a new drug, LoSod. LoSod is found to reduce serum sodium to 138 mEq/L, with a 95% confidence interval of 136.7 mEq/L to 139.3 mEq/L. What is the correct interpretation of this 95% confidence interval? A. There is a 95% chance that the interval contains the true population value. B. When using this drug in a larger population, one can expect that 95% of the patients will have a serum sodium between 136.7 to 139.3 mEq/L. C. When using this drug in a larger population, one can say with 95% confidence that the serum sodium will be 138 mEq/L. D. There is a 5% chance that the true population mean is within the stated range. E. There is a 5% chance that the results of this study are rejected in error. 13. A clinical trial is published about a new drug, Nopainz, for pain reduction. There are 200 patients randomized; 100 patients to Nopainz and 100 patients to placebo. The study found that pain was alleviated in 90 patients taking Nopainz and in 50 patients taking placebo. Calculate the relative risk in this trial? A. 0.18

B. 0.20 C. 0.40 D. 0.50 E. 1.8 14. What is the definition of a Type II error? A. When a Type I error happens twice, in two different studies. B. The null hypothesis is false, but is accepted in error. C. Accepting the null hypothesis when it is true. D. The clinical trial was not large enough to detect a meaningful difference between treatment groups. E. The null hypothesis is true, but is rejected in error. 15. A very large study has measured the weight of everyone living in Texas. If the values of all the weights were plotted, the graph would resemble this shape: A. A bell-shaped curve B. A parabola C. A quadrilateral D. A bell curve skewed to the left E. A bell curve skewed to the right 16. A hospital pharmacist presents a Journal Club article. She reports that the average weight of the subjects in the study is 142 pounds, with a standard deviation of 10. Choose the correct statement: A. Most of the subjects had a weight within 5 pounds of the mean. B. Most of the subjects had a weight within 10 pounds of the mean. C. Most of the subjects had a weight within 15 pounds of the mean. D. Most of the subjects had a weight within 20 pounds of the mean. E. None of the above statements are correct. 17. A pharmacy intern has been asked by his preceptor to gather 200 discharged patient charts from the chart room. He has been told that half the charts should be patients who received proton pump inhibitor (PPI) therapy while hospitalized. The other half should be patients with similar conditions and length of stay but who did not receive PPI therapy. The pharmacist wishes to conduct a study to see if there is any difference in the incidence of nosocomial infection in the PPI group versus the non-PPI group. Describe this type of study: A. Meta analysis B. Cohort study C. Controlled clinical trial

D. Case control study E. Observational study 18. Over 35,000 nurses were studied in a longitudinal study based in Framingham, Massachusetts. Each year, the nurses were followed up and asked to report on any incidence of heart disease. The researchers wanted to study incidence of heart disease in the subjects using hormone therapy versus those who did not use hormone therapy. Describe this type of study: A. Controlled clinical trial B. Meta analysis C. Case control trial D. Cohort study E. Crossover analysis 19. A researcher gathered all vitamin E studies from the past ten years. Vitamin E was used for a variety of conditions. The populations studied as well as the vitamin E formulations and doses were all different. However, the researcher did the best he could and compared the incidences of cardiovascular-related mortality in those taking vitamin E supplements versus those that did not. Describe this type of study: A. Case control study B. Cohort study C. Controlled clinical trial D. Meta analysis E. Observational study 20. A pharmacist at a very large mail-order facility in Louisiana noticed that the pharmacists at her job were feeling happy lately. She took a survey to see if the pharmacists were eating or drinking anything that might contribute to the elevated mood. She found out that the majority of pharmacists who were feeling happy were using fish oils, and more of the ones that were not as happy were not taking fish oils. This led the pharmacist to conclude that the use of fish oils makes pharmacists happy. Describe this type of study: A. Case control study B. Cohort study C. Controlled clinical trial D. Meta analysis E. Observational study 21. A pharmaceutical company wished to show that their antiplatelet agent worked better than placebo. They enrolled 12,000 patients at

many different research sites. They worked with the physicians to ensure that the patients were randomly assigned to the antiplatelet agent or to a placebo. The physicians did not know which of their patients received the active drug. Describe this type of trial: A. Open label clinical trial B. Controlled clinical trial C. Cross-over trial D. Cross-sectional clinical trial E. Propensity matching study 22. A pharmaceutical company wished to show that their antiplatelet agent worked better than placebo. They enrolled 12,000 patients at many different research sites. They worked with the physicians to ensure that the patients were randomly assigned to the antiplatelet agent or to a placebo. The physicians did not know which of their patients received the active drug. Choose the correct statement: A. The trial was open-label. B. The trial was single-blinded. C. The trial was double-blinded. D. The trial was observational in nature. E. This was a cohort study. 23. Correlation in a clinical trial describes: A. The ability of 1 or more variables to predict another B. The relationship between 2 variables C. The nominal data D. Confounding variables E. A cause and effect relationship 24. Choose the best description of the purpose of a pharmacoeconomic analysis: A. To measure and compare all costs, risks and benefits of drug therapy and determine the most useful treatments, in terms of efficacy, cost and effect on quality of life. B. To cut health care costs by limiting expensive drugs. C. To get the best treatments available to as many Americans as possible. D. To assess the acquisition costs of drugs and their impact on the pharmacy budget to determine what medications to add to the formulary.

E. To assess which drugs will prevent the most patient harm and encourage using them. 25. A clinical trial reports that patients taking a new drug, No-go, experience less incontinence episodes. The number of incontinent episodes (the primary endpoint) was 16.4% in the No-go group and 28.7% in the placebo group. The trial randomized 600 patients (300 patients in each arm). What is the absolute risk reduction? A. 57% B. 45% C. 32.8% D. 9.1% E. 12.3% 26. Choose the categories that represent direct medical costs: A. Lost productivity B. Quality of life C. Cost of drugs D. Length of hospital stay E. C and D 27. A pharmacist is conducting an analysis to determine the benefits of a treatment in terms of both cost and outcomes. Choose the type of analysis the pharmacist will perform: A. A cost utility analysis B. A cost minimization analysis C. A cost effectiveness analysis D. A cost control analysis E. Any of the above 28. A pharmacist is considering which beta blocker should be preferred at his institution. He has narrowed his search down to two agents. Each drug provides similar health benefits, has similar tolerability and is dosed once daily. The pharmacist will base his decision on the drug that can be purchased at the lower cost. He will use the following analysis to choose the beta blocker for his institutions formulary: A. A cost utility analysis B. A cost minimization analysis C. A cost effectiveness analysis D. A cost control analysis E. Any of the above

29. A clinical trial is published about a new drug, Nopainz, for pain reduction. There are 200 patients randomized; 100 patients to Nopainz and 100 patients to placebo. The study found that pain was alleviated in 90 patients taking Nopainz and in 50 patientstaking placebo. Calculate the relative risk reduction (RRR) in this trial? A. 0.10 B. 0.20 C. 0.40 D. 0.60 E. 0.80 30. A clinical trial looked at the effects of a chemotherapeutic given to patients with osteosarcoma. The trial duration was three months. During this time, there were two deaths among patients who received the placebo and one death among the patients that received the active drug. It could be stated that the drug decreased the risk of death by 50%. The relative risk of death in the placebo group was 2, or twice the amount in the drug group. The benefit sounds great, but in reality the benefit was very small. Choose the correct statement: A. An important feature of relative risk is that it tells you little about the actual risk outside the trial. B. The relative risk can be useful, or it can be used to attempt to make a small benefit appear larger than is warranted. C. Relative risk is also called the risk ratio. D. A relative risk < 1 means that the event is less likely to occur in the group with the intervention compared to the control group. E. All of the above. 31. A report of a clinical trial of a new antipyretic, Feverstop, versus a placebo, noted that the new drug gave a higher proportion of success than did the placebo. The report ended with the statement the trial was statistically significant (p < 0.05). In light of this information we may conclude: A. Fewer than 1 patient in 20 will fail to benefit from the drug. B. The chance that an individual patient will fail to benefit is less than 0.05. C. If the drug were effective, the probability of the reported finding or one more extreme is less than 1 in 20. D. If the drug were ineffective, the probablility of the reported finding or one more extreme is less than 0.05. E. The power of the test exceeds 0.95.

32. In a small, randomized, double-blind trial of a new treatment in patients with acute myocardial infarction, the mortality in the treated group was half that in the control group, but the difference was not significant. We can conclude that: A. The treatment is useless. B. There is no point in continuing to develop the treatment. C. The reduction in mortality is so great that we should introduce the treatment immediately. D. We should keep adding cases to the trial until the Normal test for comparison of two poplulations is significant. E. We should carry out a new trial of much greater size. 33. A new drug for stroke prevention, Clotbust, was studied. The trial enrolled 2,546 patients. In the Clotbust arm, 39 out of 1,281 patients had a stroke compared with 64 patients out of 1,265 patients in the aspirin arm, p-value = 0.025. What was the relative risk of stroke in this trial? A. 1 B. 0.9 C. 0.7 D. 0.6 E. 0.33 34. A new drug for stroke prevention, Clotbust, was studied. The trial enrolled 2,546 patients. In the Clotbust arm, 39 out of 1,281 patients had a stroke compared with 64 patients out of 1,265 patients in the aspirin arm, p-value=0.025. What was the relative risk reduction of stroke in this trial? A. 0.9 B. 0.7 C. 0.4 D. 0.3 E. 0.2 35. A new drug for stroke prevention, Clotbust, was studied. The trial enrolled 2,546 patients. In the Clotbust arm, 39 out of 1,281 patients had a stroke compared with 64 patients out of 1,265 patients in the aspirin arm, p-value=0.025. What was the absolute risk reduction of stroke in this trial? A. 10% B. 7.5% C. 5%

D. 3% E. 2% 36. A new drug for stroke prevention, Clotbust, was studied. The trial enrolled 2,546 patients. In the Clotbust arm, 39 out of 1,281 patients had a stroke compared with 64 patients out of 1,265 patients in the aspirin arm, p-value=0.025. What is the number needed to treat to prevent one stroke in this trial? A. 50 B. 25 C. 10 D. 5 E. 0.5 37. You are presenting a clinical trial to the medical team. The medical team wants to learn more about the confidence intervals. They would like greater confidence than the standard 95% confidence interval reported in the clinical trial. Greater confidence will have what effect on the interval? A. It will widen the interval. B. It will narrow the interval. C. It will have no effect on the interval. D. It will make the data invalid. E. It will cause the p-value to be invalid. 38. A pharmacist is presenting the Helsinki trial which investigated gemfibrozil 1,200 mg daily versus control. The results showed that coronary heart disease (CHD) events were 2.7% in the gemfibrozil arm versus 4.1% in the control group, with a p-value of < 0.02. What is the relative risk of CHD events in this trial? A. 88% B. 75% C. 66% D. 57% E. 53% 39. A pharmacist is presenting the Helsinki trial which investigated gemfibrozil 1,200 mg daily versus control. The results showed that CHD events were 2.7% in the gemfibrozil arm versus 4.1% in the control group, with a p-value of < 0.02. What is the relative risk reduction of CHD events in this trial? A. 44%

B. 34% C. 24% D. 22% E. 18% 40. A pharmacist is presenting the Helsinki trial which investigated gemfibrozil 1,200 mg daily versus control. The results showed that CHD events were 2.7% in the gemfibrozil arm versus 4.1% in the control group, with a p-value of < 0.02. What is the absolute risk reduction of CHD events in this trial? A. 1.4% B. 15% C. 1.7% D. 10% E. 17% 41. A pharmacist is presenting the Helsinki trial which investigated gemfibrozil 1,200 mg daily versus control. The results showed that CHD events were 2.7% in the gemfibrozil arm versus 4.1% in the control group, with a p-value of < 0.02. What is the number needed to treat to prevent one CHD event in this trial? A. 50 B. 72 C. 77 D. 134 E. None of the above 42. You are analyzing the JUPITER trial regarding the benefits of rosuvastatin 20 mg PO daily versus placebo. The primary outcome of the trial was the occurrence of a first major cardiovascular event, defined as nonfatal myocardial infarction, nonfatal stroke, hospitalization for unstable angina, an arterial revascularization procedure, or confirmed death from cardiovascular causes. At the time of study termination, 142 first major cardiovascular events had occurred in the rosuvastatin group, as compared with 251 in the placebo group, p-value 0.00001. Each arm had 8,901 patients. What was the relative risk reduction if randomized to rosuvastation in this trial? A. 44% B. 40% C. 30% D. 27% E. 19%

43. Background: Angiotensinconvertingenzyme inhibitors improve the outcome among patients with left ventricular dysfunction, whether or not they have heart failure. We assessed the role of an angiotensin convertingenzyme inhibitor, ramipril, in patients who were at high risk for cardiovascular events but who did not have left ventricular dysfunction or heart failure. Methods: A total of 9,297 highrisk patients who had evidence of vascular disease or diabetes plus one other cardiovascular risk factor and who were not known to have a low ejection fraction or heart failure were randomly assigned to receive ramipril or matching placebo for a mean of five years. The primary outcome was a composite of myocardial infarction, stroke, or death from cardiovascular causes. Results: A total of 4,645 patients were assigned to receive ramipril 10 mg daily and 4,652 were assigned to receive placebo. Of the patients taking ramipril, 651 patients reached the primary endpoint as compared with 826 patients who were assigned to receive placebo (p < 0.001). Treatment with ramipril reduced the rates of death from cardiovascular causes (6.1% vs. 8.1% in the placebo group, p < 0.001), myocardial infarction (9.9% vs. 12.3%, p < 0.001), stroke (3.4% vs. 4.9%, p < 0.001) and death from any cause (10.4% vs. 12.2%, p = 0.005). Conclusion: Ramipril significantly reduces the rates of death, myocardial infarction, and stroke in a broad range of high-risk patients who are not known to have a low ejection fraction or heart failure. From the abstract above, calculate the relative risk of the primary endpoint in the ramipril group compared to the placebo group. A. 0.1 B. 0.22 C. 0.44 D. 0.78 E. 0.85 44. From the abstract of the previous question, what is the relative risk reduction of the primary endpoint of myocardial infarction, stroke, or death from cardiovascular causes? A. 0.1 B. 0.15 C. 0.22 D. 0.33 E. 0.56 45. From the abstract above, calculate the absolute risk reduction of the composite primary endpoint of myocardial infarction, stroke, or death from cardiovascular causes.

A. 1.9% B. 2.8% C. 3.1% D. 3.8% E. 12.3% 46. From the abstract above, what is the number needed to treat to prevent the composite endpoint of myocardial infarction, stroke, or death from cardiovascular causes? A. 19 B. 25 C. 27 D. 48 E. 91 47. A clinical trial was conducted in patients with the seasonal flu. The null hypothesis states Drug A was equivalent to placebo in reducing the duration of the flu. Drug A was found to significantly reduce the duration of the flu compared to placebo (120.1 hours vs. 121.7 hours, p<0.04).Select all the TRUE statements: 1. This trial is statistically significant due to a p-value <0.05. 2. This trial is clinically significant and Drug A should be added to all hospital formularies. 3. The null hypothesis of the trial should be rejected. 4. The null hypothesis of the trial should fail to be rejected. 5. There is no statistical difference in Drug A compared to placebo. A. 1 and 2 B. 1 and 3 C. 2 and 3 D. 1. 3 and 4 E. 1, 2 and 5 48. Background: Strongheart is a new drug being investigated for chronic heart failure patients. Methods: We enrolled 3,991 patients with chronic heart failure in New York Heart Association (NYHA) functional class II-IV and with an ejection fraction of 0.40 or less, stabilized on optimal standard therapy, in a double-blind, randomized controlled study. Randomization was preceded

by a 2-week single-blind placebo run-in period. Of the 3,991 patients, 1,990 patients were randomly assigned to Strongheart 12.5 mg daily and 2,001 patients were assigned to placebo. The target dose was 200 mg once daily and doses were uptitrated over 8 weeks. Our primary endpoint was all-cause mortality, analyzed by intention to treat. Results: The study was stopped early on the recommendation of the independent safety committee. Mean follow-up time was 1 year. Allcause mortality was lower in the Strongheart group than in the placebo group (145 patient-deaths versus 219 patient-deaths; p=0.00009, [95% CI 0.53-0.81]). There were fewer sudden deaths in the Strongheart group than in the placebo group (79 vs. 132; p=0.0002) and deaths from worsening heart failure (30 vs 58; p= 0.0023). Conclusions: Strongheart once daily in addition to optimal standard therapy improved survival. From the abstract above, what is the relative risk of the primary endpoint in the Strongheart group versus the placebo group? A. 0.9 B. 0.88 C. 0.12 D. 0.27 E. 0.66 49. Based on the information provided on question 48, what is the relative risk reduction of the primary endpoint of all-cause mortality in patients randomized to the Strongheart group? A. 10% B. 12% C. 34% D. 26% E. 58% 50. Based on the information provided on question 48, what is the absolute risk reduction of the primary endpoint of all-cause mortality in patients randomized to the Strongheart group? A. 3.6% B. 2.8% C. 2.4% D. 1.8% E. 1.5%

51. Based on the information provided on question 48, what is the number needed to treat with Strongheart to prevent 1 death? A. 11 B. 28 C. 45 D. 71 E. 116 ANSWERS 1. D. The mean is found by adding up the values in a list, and dividing by the number of items 2. B. The median is found by arranging all the observations in numerical order (lowest to highest or highest to lowest) and picking the middle value. 3. C. The mode is the value that occurs most frequently. 4. D. Placebos are used in clinical trials to blind people to their treatment allocation and minimize bias. Placebos should be indistinguishable from the active intervention to ensure adequate blinding. 5. B. A clinical trial is testing the null hypothesis which states there is no difference between the two treatment groups. 6. C. In most clinical trials, the statistical significance is defined as a pvalue less than 0.05. 7. C. The lower the p-value, the less chance that the null hypothesis will be rejected in error. A p-value of 0.04 means that there is a 4% chance that the null hypothesis was rejected in error. A p-value less than 0.01 means that there is less than a 1% chance that the null hypothesis was rejected in error. 8. D. A measure of statistical significance is not the same as clinical significance. For example, if a blood pressure drug lowers systolic blood pressure by 3 mmHg, it may be statistically significant versus the placebo, but clinically it will not be used since other drugs lower blood pressure to a much greater degree. 9. B. Discrete data can be divided into groups or categories (e.g., marital status, gender, ethnicity). These are called nominal discrete values. Another type of discrete data is called ordinal data. These are ranked by

severity, such as Apgar scores whichare used to assess the health status of newborn babies. The Apgar scale ranks the babys health on a scale of zero to 10. A baby with an Apgar score of 2 is not twice as healthy as a baby with an Apgar score of 1; there is not a direct linear correlation with the scoring system. 10. C. Continuous variables can take an infinite number of possible values (within a range), such as height, weight, A1C, blood pressure. For example, a patients A1C can be 7.2 or 7.3 or 7.4 and so on. 11. B. A Type I error means that the null hypothesis is true but is rejected in error. For example, if the p-value is 0.05, it means that 5% of the time the null hypothesis will be rejected in error, or a type I error will have occurred. 12. A. A confidence interval gives an estimated range of values which is likely to include an unknown population parameter; the estimated range being calculated from a given set of sample data. ~ The proportion of times that an estimate will be correct. 13. B. Relative risk is the likelihood of an event (in this case, pain) in the exposed group occurring the the risk of the event in the non-exposed group occurring. Therefore, 10 of the 100 patients had pain in the Nopainz group, and 50 of the 100 patients had pain in the placebo group. So, 10/50 is 0.20. 14. B. A type II error means that the null hypothesis is false, yet it is accepted in error. The study authors conclude that there is no difference, when there actually is a difference 15. A. If many data points are plotted from a large population group, the distribution would look like a Normal or Gaussian bell-shaped curve. 16. B. The standard deviation (SD) shows how much variation there is from the average. For example, if the average weight in a study population is 142 pounds, with a SD of zero, then all subjects weigh the same (142 pounds). If the SD is 10, then most of the subjects have a weight within 10 pounds of the mean (from 132 to 152 pounds). 17. D. Case-control trials examine cases (those with the intervention) and compare them to subjects without the intervention (the controls). The cases are compared to the controls to see if the intervention (the drug, for our purposes), caused a problem. Case -control studies are retrospective as they look back in time.

18. D. A study group (the cohort) is followed over time and outcomes are compared to a subset of the group who were not exposed to an intervention, such as a drug (e.g., the Framingham studies). These are generally prospective in design. 19. D. A meta analysis is a systematic review of many different but related studies in order to integrate the results. These are often done and can be useful to pool smaller trials into a larger group for analysis (e.g., Cochrane reviews). However, there are many flaws to this type of pooled data. For example, the populations studied can differ, there can be different lengths of treatment and the inclusion and exclusion criteria may differ. 20. E. In an observational study, the results may or may not be valid. It is possible that the happy pharmacists cared about living, and having read the NCEP guidelines, decided to consume healthier types of oils. 21. B. A study group is compared to one or more control (comparison) groups in a controlled setting. The gold standard for doing clinical drug trials is a randomized, placebo-controlled, double-blinded, multicenter trial with adequate statistically power. Statistical significance is usually defined as a p-value <0.05 in most studies 22. C. Single blinding means that the subjects do not know if they received the active drug or placebo. Double-blinding, the best type of controlled clinical study, means that neither the subjects nor the researchers know which subjects received the active drug and which received the placebo. Blinding reduces bias on the part of the subjects and researchers. 23. B. If the data is correlated and the data points are plotted, a straight line can be drawn that will run through, or close to, most of the data points. Data can be positively or negatively correlated depending on the direction of the slope of the line. If the data points are not correlated, the data points will be scattered all over the plot. 24. A. Pharmacoeconomics takes into account broader costs beyond just the drugs acquisition costs and is an important policy tool to ensure money is not wasted. Quality of life, costs (direct and indirect) and efficacy are included in the analysis. 25. E. The absolute risk reduction is calculated by subtracting the event rates of the 2 groups. Therefore, 28.7% - 16.4% = 12.3% 26. E. The cost of drugs, the cost of surgery, hospital length of stay are all examples of direct costs.

27. C. The most common tool used in pharmacoeconomics is the costeffectiveness analysis, which compares the clinical effects of various therapies (such as mortality, morbidity) to their net costs. 28. B. Once the benefit of various drugs is considered equivalent (such as choosing which ACE Is or ARBs to have on the formulary), the next step is to find which one is less expensiveif the company is large, this will include a bidding process to suppliers. Or, the pharmacy benefit manager (PBM) chooses which drugs should be included in the formulary. 29. E. Relative risk reduction (RRR) = 1-RR (relative risk). The relative risk was calculated in an earlier question and was found to be .20. Therefore, 1-.20 = 0.8. 30. E. Relative risk (RR) only gives you a measure (ratio) of what the risk of an event is in one group compared to the risk of that event in a comparison group. It does not give you an idea of how important (or large) the treatment effect really is in the population-at-large 31. D. The p-value refers to the null hypothesis which is Group 1 = Group 2 (meaning the drug is ineffective). P-value is the probability of obtaining a test statistic as extreme as the one actually observed. 32. E. The trial is small and some benefit was seen with the new treatment. Many times smaller studies are done to evaluate initial effects (many times referred to as pilot studies). Performing a larger trial will increase statistical power and then may show a statistically significant benefit. 33. D. The relative risk of stroke in this trial is calculated by taking the risk of stroke in each arm: 39/1,281 = 0.03 and 64/1,265 = 0.05. So, 0.03/0.05 = 0.6 34. C. Relative risk reduction = 1-RR. Since the RR is calculated to be 0.6, the relative risk reduction is 1 0.6 = 0.4. 35. E. The absolute risk reduction is the difference in risk of the outcome in question (in this case, stroke). The rate of stroke in the aspirin arm was 0.05 (or 5%). The rate of stroke in the Clotbust arm was 0.03 (or 3%). 0.05 0.03 = 0.02 (or 2%). 36. A. Number needed to treat (NNT) is 1/ARR. Therefore, 1/0.02 = 50 patients. Fifty patients will need to be treated to prevent one stroke.

37. A. Having a higher confidence level will widen the interval. For example, 99% CIs are wider than 95% CIs. 95% CIs are wider than 90% CIs. 38. C. The relative risk of CHD events in this trial is calculated by taking the risk of each arm: 2.7%/4.1% = 0.66, or 66%. 39. B. Relative risk reduction = 1-RR. Since RR = 0.66, then relative risk reduction is 1 0.66 = 0.34. 40. A. The absolute risk reduction is the difference in risk of the outcome in question (in this case, CHD events). The rate of CHD events in the control arm was 4.1%. The rate of CHD events in the gemifibrozil arm was 2.7%. 4.1% - 2.7% = 1.4%. 41. B. Number needed to treat (NNT) is 1/ARR. Therefore, 1/0.014 = 72 patients. 72 patients will need to be treated with gemfibrozil to prevent one CHD event. 42. A. Relative risk reduction = 1-RR. Event rates per arm: 142/8,901 = 1.59% in rosuvastatin arm and 251/8,901 = 2.82% in placebo arm. 1.59/2.82 = 0.56. Therefore, 1-0.56 = 0.44, or 44%. 43. D. The relative risk is the probability of the event occurring in the exposed group versus the non-exposed group. The risk of the primary outcome is 14% in the ramipril group versus 17.8% in the placebo group. 14%/17.8% gives a relative risk of 0.78 44. C. Relative risk reduction is defined as 1 minus relative risk. The relative risk is calculated as 0.78, so the relative risk reduction is 0.22 (1-0.78). 45. D. The absolute risk reduction is the absolute difference in outcome rates between two groups. 46. C. The number needed to treat is defined as 1/ARR, or 26.3 rounded up to 27. Therefore, 27 patients need to be treated for 5 years to prevent 1 composite endpoint. 47. B 48. E. The relative risk is the probability of the event occurring in the exposed group versus the non-exposed group.

49. C. Relative risk reduction is defined as 1 minus relative risk. The relative risk is calculated as 0.66, so the relative risk reduction is 0.34 (1-0.66), or 34%. 50. A. The absolute risk reduction is the absolute difference in outcome rates between two groups. 145/1,990 = 0.073, or 7.3%. 219/2,001 = 0.109, or 10.9%. 10.9% - 7.3% = 3.6% 51. B. The number needed to treat is defined as 1/ARR, or 27.7 rounded up to 28. Therefore, 28 patients need to be treated for 1 year to prevent 1 death.