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Case Study 4–1

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  • 1. Yes, any study enrolling human subjects requires IRB approval. Even though the EPE product is a nonprescription agent, the subjects may still be at risk while partaking in this study. The IRB approval is needed to protect the patient.
  • 2. Placebo being selected as the control for this controlled clinical trial is appropriate. The purpose of this study is to measure and quantify the LDL-C lowering effects produced by EPE. This is the first clinical trial published evaluating EPE. The differences in the LDL-C change between the two groups can document whether EPE has a pharmacological effect. This study was designed with a small sample size to measure changes in LDL-C levels but not place too many subjects at risk. After LDL-C lowering by EPE is documented by this study, using an active control (e.g., statin) would be appropriate as the control for future studies. Historical controls would not be appropriate to measure changes in LDL-C level since this outcome is not a significant risk to the subjects; many therapies are available that are considered safe to reduce LDL-C levels.
  • 3. This indicates that 68% (one standard deviation from the mean) of the LDL-C values collected from the patients taking EPE were measured to be between 128 and 184 mg/dL.
  • 4. Since all patients were to follow the same diet, this would be classified as adjunctive therapy. The diet should not interfere with measuring a difference in LDL-C lowering between EPE and placebo since both groups are following the same diet.
  • 5. The probability of rejecting a true Ho is < 4.5%. Since this p-value is less than the stated alpha value, the Ho would be rejected and H1 accepted. The probability of a Type I error is less than 4.5%, which could be due to chance. In addition, the probability of chance being the reason a difference was calculated between EPE and placebo would be less than 4.5%.
  • 6. The use of the EPE product would not be recommended to the patient at this time. Primary reasons include:
    • Only one clinical trial published evaluating the efficacy and safety of EPE;
    • The clinical trial included a small sample size (< 75 total patients) and had a duration of only 12 weeks;
    • The p-value is less than the established alpha value (5%), but this does not automatically indicate a clinical difference between EPE and placebo;
    • Although the mean LDL-C level increased by 6 mg/dL (~4%), the mean LDL-C level decreased only 8 mg/dL (< 5%) with the EPE product;
    • A body of reliable evidence that documents significant mean LDL-C level reductions is lacking.
    • The EPE product does not have evidence of reduction in clinical outcomes, such as MIs or stroke, whereas, other FDA-approved cholesterol medications do have this data.

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Case Study 4–2

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  1. Double-blinding is the most appropriate blinding type. The primary and secondary endpoints are subjective in nature. Thus, neither the patients nor investigators should know who is taking which therapy. Reduction in pain is not going to be reported by patients knowing they are taking placebo. In addition, the probability is highly likely that changes in ...

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