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Three Steps to Interpret Clinical Trials

Mark C. Granberry , Pharm.D . Professor and Vice Chair of Pharmacy Practice UAMS College of Pharmacy. Three Steps to Interpret Clinical Trials. Learning objectives. At the end of this presentation, you should be able to: 1. Evaluate the validity of a clinical drug trial

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Three Steps to Interpret Clinical Trials

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  1. Mark C. Granberry, Pharm.D. Professor and Vice Chair of Pharmacy Practice UAMS College of Pharmacy Three Steps to Interpret Clinical Trials

  2. Learning objectives At the end of this presentation, you should be able to: 1. Evaluate the validity of a clinical drug trial 2. Assess efficacy, safety, and effectiveness outcomes of a study 3. Objectively interpret clinical trial results

  3. Three Questions must be answered: • 1. What is the probability that the results of this trial are true? • 2. To whom do the results apply? • 3. Assuming the results are likely to be true, will you now have to change the way you do things?

  4. First Question: Do you think it’s true • Good study design • Clearly defined criteria • Focus on primary endpoint! • How well the study is conducted?

  5. The Perfectly Designed Study • Two or more groups, identical in every respect at the beginning • One, and only one thing is changed • If the groups are different at the end, there could only be two causes • The intervention • Play of chance

  6. Getting Groups the Same at the Beginning • Randomization • Avoiding pitfalls of randomization • Intention-to-treat analysis Check Baseline Characteristics

  7. BaselineCharacteristics Are groups similar? N Engl J Med 2014; 371:993-1004

  8. Change Only One Thing:The Intervention Being Studied • Blinding • Unblinded • Single blind • Double Blind • Why So Important? • How do you know if blinding was effective?

  9. Question Two: To Whom Do the Results Apply? • Inclusion Criteria • Exclusion Criteria

  10. Question Two: To Whom Do the Results Apply? Underrepresented Groups

  11. The Third Question: • Based on this new information – Will you now have to change the way you do things? • Are the results CLINICALLY significant? • Do the benefits outweigh the risks?

  12. It’s a Little Like Gambling in Las Vegas Probability of Truth Benefit versus Risk

  13. Assessing Benefit and Risk • Number Needed to Treat (Benefit) • Number Needed to Harm (Risk) • The number of individuals who must be treated for a period of time so that one additional individual will avoid/have event.

  14. Example Exercise • NNT of 5, treatment for 1 month, avoid 1 additional death • NNT of 100, treatment for 5 years, avoid 1 additional hospitalization

  15. Compare Benefit to Risks • NNT = 5 for 1 month to prevent 1 additional death • NNH =20 for 1 month to cause 1 additional major bleeding event • Do the benefits outweigh the risk?

  16. Endpoints • Survival • Quality of Life • Validated QoLquestionnaires • Hospitalizations • Disability Assessments (Modified Rankins Scale for Stroke) • Surrogate Endpoints • Blood Pressure • A1C

  17. Composite Endpoints Fourrier N Engl J Med 2017;376:1713-22

  18. Trials with No Benefit Same size calculation EUCLID N Engl J Med 2017;376:32-40

  19. Summary

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