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Biomedical Literature Evaluation & Statistics

Exam Study Guide
Michael A. Mancano, Pharm.D.

1. In a randomized controlled clinical trial of a new antihypertensive agent, patients with diastolic blood
pressure readings greater than 90 mmHg on their most recent clinic visit are selected as subjects. A patient
assigned to the experimental treatment group is normotensive at his first clinic visit after initiating drug
therapy. All of the following factors represent plausible explanations for this observation EXCEPT:

a) Simpson’s Paradox
b) Random variation in blood pressure measurements.
c) Regression to the mean.
d) Efficacy of the experimental drug.
e) Measurement error.

2. A study is carried out to compare the effect of two low fat diets on vascular plaque accumulation. The
investigator conducting the study obtained a list of individuals who had been continuously enrolled
between 1985 and 1990 in one of two commercial weight loss programs based on the two diets.
Participants in both diet programs were invited to participate in the study. The protocol described above is
an example of which of the following study designs?

a) Randomized, double-blind
b) Randomized block design
c) Meta-analysis
d) Double-blind, crossover study.
e) Observational study: historical cohort study.

3. A group of patients with coronary heart disease volunteered to participate in a study of the relationship
between diet and vascular plaque accumulation. The subjects were randomized to one of three diets: very
low fat (< 10% total calories from fat), low fat (< 30% of the total calories from fat), or a reduced calorie
diet with no restrictions on fat intake. At the end of a five-year follow-up period, vascular plaque
accumulation in the three diet groups was compared to determine which diet achieved the greatest
reduction in plaque accumulation. The protocol described above is an example of which of the following
study designs?

a) Randomized clinical trial.
b) Case report
c) Meta-analysis
d) Double-blind, crossover study.
e) Observational study: historical cohort study.

4. A pharmaceutical company conducted a double-blind randomized controlled clinical trial of a new
antipruritic drug. One group of subjects received a lotion containing the experimental drug, a second group
received an identical lotion lacking the active ingredient, and a third group received no treatment at all.
Two weeks later, the “itching intensity” scores (the lower the score, the less severe the itching) for the three
groups were 12 (experimental group), 15 (placebo group) and 65 (no treatment group). Which of the
following factors BEST explains the result obtained for the subjects who received the lotion lacking the
active ingredient?

a) The Hawthorn effect
b) Regression to the mean.
c) Selection bias.
d) The placebo effect.
e) Failure to adequately blind subjects and investigators.

The result was that patients who received the new antihypertensive agent had a reduction in blood pressure of 5 % and the patients who received the placebo had a reduction in blood pressure of 3%. Which of the following statements are TRUE with regard to the study results? a) The new antihypertensive agent is very effective in women. c) Simpson’s paradox d) The Greenhouse effect. 6. b) A clinically important difference in average weight gain between the two groups of lower-weight infants may exist. with a p-value for the comparison between the two groups of p=0. the pediatrician reports at a research conference that the results are “not statistically significant (p>0. with a p-value for the comparison between the groups of p= 0. In a clinical trial of a novel antihypertensive agent. “Is the average weight gain higher among infants receiving the experimental formula than among those receiving the standard formula?” After conducting the study. Weight gains for both groups will be recorded at the end of this time period in an attempt to answer the research question. e) Regression to the mean. low power. d) The observed difference in average weight gain between the experimental and control groups is likely to be due to random chance.” All of the following statements about this conclusion are true except: a) The average weight gain in the infants fed the experimental formula is EXACTLY THE SAME as the average weight gain among infants fed the standard formula.06. c) Initial patient selection bias probably accounts for the observed results.8. c) The chance of type II error may be high.Please refer the following scenario to answer questions 5 and 6. 7. but the power of the test may have been too low to detect it. . b) Further study is warranted in a study designed to look specifically at the female population. 60 patients were randomly assigned to receive the new drug for 14 days and 60 patients were randomly assigned to receive a placebo for 14 days.05). The results observed in the female sub-group in the above described clinical trial is an example of: a) The Hawthorne effect. b) Bonferroni error. Fifty such infants will be randomly assigned to receive either the experimental formula or a standard commercially available formula for three months. 5. i. The primary research objective was to determine if the mean change from baseline in diastolic blood pressure was greater for the new drug than for the placebo.e. The 10 women who received the new antihypertensive agent experienced a reduction in blood pressure of 25% and the 10 women who received placebo had a reduction in blood pressure of 8%. d) The decision to analyze the female data is an example of an “a priori” hypothesis. A pediatrician wishes to evaluate the efficacy of an experimental formula in infants who fall below the tenth percentile in weight for their age group. Upon completion of the study the investigator decided to look at the 10 women who were randomly assigned to each of the two groups and the results were shocking. e) None of the above. Changes in blood pressure from baseline during the treatment period were recorded for all patients.

The pediatrician conducting this study reports the existence of a statistically significant association between chronic otitis media in children between the ages of one and three and a parental history of such infections (p<0. c) Extraneous. Please refer the following scenario to answer questions 9 and 10.8. b) An completely unbiased comparison was made between study groups c) There is a cause and effect relationship between chronic otitis media in young children and a family history of this disorder. treated by the same practice for other illnesses. b) The observed difference occurred by random chance. a physician measures serum cholesterol levels in a sample of men receiving drug A and in a sample of men receiving drug B and learns that the average serum cholesterol is lower in those receiving drug B. Fifty different children in the same age group. he identified 50 children between one and three years of age who had experienced at least three middle ear infections during the preceding year. Of the children with recurrent ear infections. To compare the efficacy of two drugs in lowering serum cholesterol levels. A pediatrician wished to determine the relationship between chronic otitis media in young children and parental history of such infections. From the records of a large pediatric practice. d) There is a less than 5% chance that the observed association occurred by random chance . compared to 20 of the children treated for other illnesses. were also identified as control subjects. uncontrolled differences (other than drug therapy) between the sample of the men receiving drug A and the sample of men receiving drug B account for the observed difference in efficacy. 30 had a family history of chronic otitis media.05). d) The observed difference may be attributed to a small p-value for the statistical test. Plausible explanations for the observed difference in the response to the two drugs include all of the following EXCEPT: a) Drug B is superior to drug A in lowering cholesterol levels among the population of potential recipients. 9. Which of the following represents the MOST appropriate interpretation of this finding? a) A strong clinically significant association exists between the study variables in the populations from which the samples were drawn. The pediatrician interviewed the parents of the subjects in both groups to determine their history of chronic otitis media as young children. This study is an example of a: a) Meta-analysis b) Prospective cohort study c) Case-control study d) Randomized controlled clinical trial 10.

to be considered a clinically important difference.Please refer the following scenario to answer questions 11 and 12. . 11. The probability that this statistical test will detect the specified clinically important difference in average hair growth. In the study hospitalization was decreased as evidenced by the following data: 13.4 d) 12. a) Increases as the specified effect size decreases.2 c) 7. and carry out this test at the 5% level of significance (p = 0.4 b) 18. given that such a difference actually exists between the populations from which the study samples were drawn. b) Increases as the sample size increases c) Increases as the power of the test increases. The researchers decide the average hair count must increase by 20% in the drug treated group. This represented an absolute reduction of 5. In the PREVENT trial.5 e) Cannot be calculated from the data provided.05). d) None of the above 12. It was a randomized. placebo controlled trial. A pharmaceutical company conducts a randomized controlled clinical trial to determine the efficacy of a newly developed topical solution for inducing hair growth in men with male pattern baldness. given that such a difference does indeed exist between the populations represented by the study samples. double-blind. The probability that this statistical test WILL FAIL TO DETECT a clinically important difference in average hair growth between the comparison groups. They select a sample size that will ensure 80% power for the statistical test. compared to the placebo group.5% of patients receiving placebo were hospitalized versus 8% of patients in the intravenous RSV immune globulin group. What is the NNT with intravenous RSV immune globulin to prevent one hospitalization? a) 2. intravenous immune globulin for RSV was administered to premature infants with BPD (Bronchopulmonary Dysplasia). is: a) 95% b) 80% c) 20% d) 5% 13.5% and a relative reduction of 41%.

b) Lovastatin and newstatin appear to be equally efficacious in lowering LDL levels. is an example of which type of data? a) Interval Scale b) Nominal Scale c) Ordinal Scale d) Ratio Scale 15. as an outcome. Which of the following statements is true? a) Both drugs are clinically equivalent in providing analgesia. The p value was reported as 0. newstatin. The Atenolol Silent Ischemia Study investigated the use of atenolol in mildly symptomatic patients with coronary artery disease. the mean baseline LDL level was 192 mg/dL in the lovastatin group and 193 mg/dL in the newstatin group. At the beginning of the trial. Death. d) A priori assignment of p<0. At the end of the 4-month trial. Which of the following is true about the interpretation of these results? a) A further discussion of power would be necessary to draw meaningful conclusions. c) Lovastatin and newstatin could be used interchangeably in patients.Please refer the following scenario to answer questions 14 and 15.09. Results showed that one agent (Drug X) decreased pain scale scores an average of 2 points (95% CI –5 points to –1. double-blind. Which of the following would be the appropriate statistic to test the null hypothesis in question 37? a) Student-t test b) Chi squared test c) ANOVA d) Wilcoxon signed-rank 16. 17. b) Drug X would be preferred clinically c) Drug Y would be preferred clinically. d) Neither drug appears clinically effective. mean LDL levels for the lovastatin group were 152 mg/dL and for the newstatin group they were 165 mg/dL. 14. . (n=60) compared its ability to decrease LDL levels to that of lovastatin. ventricular tachycardia/ventricular fibrillation. Adverse outcomes were defined as death. A trial of a new lipid-lowering agent. A study compared the mean decrease in visual analog pain scales for two analgesics was reported. while the other agent (Drug Y) decreased scores an average of 4 points (95% CI –6 to +1). placebo controlled multicenter study assessed whether treatment with atenolol decreased adverse outcomes after 1 year. This randomized.5 points).1 would have made the study more clinically useful. aggravation of angina or revascularization.

21. The variable documenting the evidence of bleeding (yes/no) is considered what type of data? a) Ratio b) Ordinal c) Nominal d) Interval e) Definitive 19. 18. 19 & 20 A study was performed that evaluated the difference in platelet count and evidence of bleeding after patients were treated with unfractionated heparin. What is the best statistical test to evaluate the difference in evidence of bleeding between the three groups? a) Student – t b) Chi square c) Wilcoxon signed rank d) Paired Student – t e) ANOVA 20.Use the following information to complete questions 18. Three hundred patients were enrolled and randomly assigned to one of the three different groups. Blood pressure measurements are classified as what type of statistical data? a) Ratio b) Ordinal c) Nominal d) Interval e) Descriptive . What is the best statistical test to evaluate the difference between the mean platelet counts of the three groups? a) Student – t b) Chi square c) Wilcoxon signed rank d) Paired Student – t e) ANOVA Use the following information to complete questions 21 & 22 A researcher is evaluating sixty patients using a cross-over design to determine whether propranolol or hydrochlorothiazide is more effective in managing isolated systolic hypertension. low molecular weight heparin or warfarin.

22. which is 5% c) The Type II error rate () which is unknown d) None of the above 24. patients were asked to rate their degree of pain relief on a scale from 0 to 100. The results were NOT STATISTICALLY SIGNIFICANT at the 5% level of significance. Which of the following statements BEST defines the power of the statistical test? a) Power is the probability that the test will detect a difference between the three drugs when. Choose the appropriate statistical test to analyze whether there is a difference in mean blood pressure when using propranolol and hydrochlorothiazide. and the average score for each treatment was calculated. 25. d) Power is the probability that the test will correctly detect a difference among the three treatments which does. Which of the following statistical tests would be utilized for the analysis of continuous data from a four sample. in fact. codeine 60 mg and a placebo in providing pain relief after minor dental surgery. b) Power is the probability that the test will fail to detect a difference in the level of pain relief provided by the three treatments when they actually do differ. Two hours after receiving the drug or placebo. no such difference exists. in fact. a) Student – t b) Chi square c) Wilcoxon signed rank d) Paired Student – t e) ANOVA Please refer to the following scenario to answer questions 23 and 24. cross-over study that does NOT fit parametric assumptions? a) Mann Whitney U test b) Paired t test c) Friedman’s ANOVA test d) Cochran’s Q test e) McNemar’s test . Fifteen participants were randomly assigned in groups of five to their treatment groups. exist. c) Power is the probability that there is no difference in the level of pain relief provided by the three treatments. The chance of error associated with this conclusion is: a) The type II error rate (). A double-blind clinical study was conducted to compare the efficacy of ibuprofen 400 mg. 23. which is 95% b) The type II error rate ().

It was a randomized. As the sample size is increased in a clinical trial then the power is: a) Increased b) Decreased 31.26.7% of patients receiving placebo were hospitalized versus 11% of patients in the intravenous RSV immune globulin group. placebo controlled trial. This represented an absolute reduction of 17. A nonparametric alternative to the paired-t test which can be utilized for continuous data that does not fit the parametric assumptions. therefore you lose the individuality of the data. In the study hospitalization was decreased as evidenced by the following data: 28. . c) A normal distribution is required prior to testing. What is the NNT with intravenous RSV immune globulin to prevent one hospitalization? a) 3. is: a) Mann Whitney U test b) Wilcoxon signed rank test c) Kruskal-Wallis ANOVA d) Cochran’s Q test e) Friedman ANOVA 29. intravenous immune globulin for RSV was administered to premature infants with BPD (Bronchopulmonary Dysplasia).6 d) 1.5 b) 9. Which of the following is a non-parametric statistical test? a) T-test b) Chi-square c) ANOVA d) Paired-t test e) ANCOVA 28. d) All of the above are true e) None of the above are true 27. Which of the following are true concerning non-parametric statistical tests? a) Non-parametric tests are more powerful then parametric tests b) Many non-parametric tests deal with ranks. The NNT (Number Needed to Treat) is calculated as the inverse of the: a) Relative Risk b) Absolute Relative Risk c) Relative Risk Reduction d) Number Needed to Harm 30. In the PREVENT trial.7% and a relative reduction of 68%. double-blind.5 e) Cannot be calculated from the data provided.1 c) 5.

The probability that the statistical test will fail to detect this difference could then be described by all of the following statements EXCEPT: a) This probability is equal to 0.05. 35 and 36. is less than or equal to 0. Please refer to the following scenario to answer questions 34. A clinical researcher conducts a study to compare the effect of a new histamine receptor antagonist and placebo on peptic ulcer healing times. In a report summarizing the results of a study comparing serum sodium levels in normotensive patients and newly diagnosed hypertensive patients prior to dietary sodium restriction. no such difference exists. . In addition.95. is 0.95. 34. Baseline cholesterol levels were determined for all study participants prior to initiating the program and again in three months. The research question is “Are average serum cholesterol levels lower after the three-month program of diet and exercise?” a) Chi square test b) t test c) Paired t Test d) Mann Whitney U test e) One-way ANOVA 33.05.32. b) The probability of accepting the null hypothesis. The correct interpretation of the level of uncertainty associated with this conclusion is? a) The probability of obtaining a difference greater then or equal to that observed. d) The probability that the statistical test will detect a difference in healing times when.05. given that there is no difference between the two treatments. p < 0. Assume that serum sodium levels in the two populations do. the researchers report that. b) This probability increases as the level of significance is decreased.05). given that the two treatments are actually similarly efficacious.8 or 80%. e) The probability that a particular peptic ulcer patient will benefit from receiving the new drug is 0. c) The probability that the healing time observed after treatment with the new drug truly does differ from the healing time observed after placebo is 0. the power of the chosen statistical test to detect a difference in mean serum sodium levels of at least 5 mEq/L between the two patient populations is 20%.05. in fact. in fact. is greater then 0. Choose the appropriate statistical test for analyzing the data from the following clinical trial: Thirty men between the ages of 30 and 55 participated in an experiment to study the effectiveness of a particular diet and exercise program in reducing serum cholesterol levels. c) This probability represents the type II error rate. for the sample sizes employed in this study. She reports that the difference in healing times between the two treatments is statistically significant. d) This probability is smaller than the probability that the test will fail to detect a difference in serum sodium levels of at least 10 mEq/L. differ by at least 5 mEq/L. a team of clinical researchers states that the difference in mean serum sodium levels was not statistically significant at the 5% level of significance (p>0.

36. given that no such difference actually exists.05). e) The observed correlation is unlikely to have occurred as a result of random chance.01. given that such a difference actually exists. alpha. there is not enough evidence to substantiate the claim of an association between blood pressure and serum cholesterol.01? a) The probability of accepting the null hypothesis when it is false is 0. given that such a difference does exist. b) The probability that they will fail to detect a difference in mean serum sodium levels between the two populations.35. 37. b) The correlation between blood pressure and serum cholesterol level is statistically significant (p < 0. c) The power of the statistical test will decrease. b) The null hypothesis is more likely to be rejected that if the investigator had chosen a level of significance of alpha = 0.01. c) The results are not statistically significant. A statistical test fails to reject the hypothesis that the correlation between blood pressure and serum cholesterol level is equal to zero at the 5% level of significance. increases. Which of the following is a result of carrying out a given statistical test of a hypothesis at a level of significance alpha error = 0. d) Higher blood pressure values tend to be associated with higher serum cholesterol levels.” b) The power of the test will decrease if the probability of a type II error increases. . d) This value represents the probability of detecting a difference of at least 5 mEq/L in mean serum sodium levels when the null hypothesis is true. that is. decreases.01. c) The probability of observing a statistically significant result is 0. d) The probability of rejecting the null hypothesis when it is true is 0. All of the following statements about the power of the chosen statistical test are true EXCEPT: a) The reported value for power suggests that the research team should not conclude that “no difference exists between the mean serum sodium levels of the two populations. 38. e) Given the reported value of power. they would expect which of the following changes to occur? a) The probability that they will detect a difference in mean serum sodium levels between normotensive and hypertensive patients. e) The probability that the statistical test will have a false negative outcome will increase. a statistically significant difference in the mean serum sodium levels of the two patient populations may have been observed if the investigators had used a larger sample size. e) The null hypothesis is true at least 1% of the time. will increase.05. d) The level of significance. What is the correct interpretation of this result? a) The correlation between blood pressure and serum cholesterol levels in the population from which the sample was drawn is equal to zero. c) This value represents a 20% chance that the statistical test will detect a difference in serum sodium levels between the two populations of at least 5 mEq/L. If the investigators repeat the study with a larger sample size.

A survey reports that 65 of 100 patients with multiple sclerosis have cats as household pets.e. The conclusion that exposure to cats causes multiple sclerosis is a) Valid for the study population. c) An increase in the overall type I error rate for the entire set of comparisons. because the sample size is too small to permit generalization to the entire population of multiple sclerosis patients. The use of multiple t-tests (with a 5% level of significance for each test). c) The probability of a type II error may be high. increased alpha error). An experiment was conducted to compare the effect of four drugs on serum cholesterol levels in patients with type III familial hypercholesterolemia. because of the lack of an appropriate control or comparison group. The investigators report that the p values for their comparison of the study groups are all greater than 0. 43. b) It is used to control the overall alpha error rate per experiment for the entire set of comparisons. drug C or drug D.1). because the comparison is not based on exposure rates. Please refer to the following scenario to answer questions 40. to analyze the data obtained in this study would accomplish which of the following? a) A decrease in the chance of type I error. b) Average serum cholesterol levels are exactly the same among the four treatment groups. What statistical test should be utilized to analyze the data from this study? a) Wilcoxon signed rank test b) 1-way ANOVA c) Kruskal-Wallis ANOVA d) Chi square test e) Repeated measures ANOVA 41. One hundred and twenty patients were randomly assigned to receive drug A. but the power of the statistical test may have been to low to detect it. Serum cholesterol levels were recorded after six months of drug therapy. . Which of the following statements about the Bonferroni multiple comparison procedure are true? a) It is used to determine which of a set of means are different while simultaneously controlling for the multiple comparison problem (i. 42.1 (p >0. because of the failure to carry out an appropriate statistical analysis of the data. rather than an analysis of variance. d) All of the above are true. d) A clinically important difference in average serum cholesterol levels after the four drug treatments may exist. d) A decrease in the probability of a false positive result.39. c) Incorrect. d) Incorrect. 40. drug B. b) Incorrect. 41. 42 and 43. e) Incorrect. b) A decrease in the likelihood of erroneously declaring a difference in means statistically significant. All of the following statements about this conclusion are correct EXCEPT a) It is likely that any observed differences in average serum cholesterol levels in response to the four drug treatments occurred by random chance. c) It represents a modification of the t test.

45. A total of 8.84 e) Cannot be calculated from the data provided.16 c) 0. The calculated relative risk reduction (RRR) for this study is? a) 0.152 patients were randomized to receive either Enoxaparin 30 mg subcutaneously every 12 hours (n = 4. 45.09 b) 0. 46 & 47 A study was conducted to assess the prevention of deep vein thrombosis in patients undergoing knee replacement surgery.63 e) Cannot be calculated from the data provided 47. The calculated absolute relative risk (ARR) for this study is? a) 10% b) 25% c) 35% d) 58% e) Cannot be calculated from the data provided 46. All patients did not have a history of deep vein thrombosis.056). The calculated relative risk (RR) for this study is? a) 0.096) or heparin 5000 units subcutaneously every 12 hours (n = 4. The primary endpoint of the study was the occurrence of a deep vein thrombosis during the two month postoperative period. .Use the following information to complete questions 44.8 c) 4 d) 10 e) Cannot be calculated from the data provided. All patients were followed for a total of 2 months after surgery.48 d) 0. The number of patients with the primary endpoint are as follows: Outcome Yes No Enoxaparin 614 3482 Heparin 1622 2434 44.27 c) 0. The calculated number needed to treat (NNT) for this study is? a) 1.37 d) 0.7 b) 2.18 b) 0.