Clinical trials placebo effect

2. WHAT ARE CLINICAL TRIALS? THEY ARE Research studies using human volunteers answering specific health questions. The safest and fastest way to find treatments that work in people. Conducted using a plan, called a “protocol”. The protocol describes: types of patients, schedules of tests, procedures, drugs, and dosages. Also length of study, and outcomes to be measured. Each person gives Informed consent stating they know the risks. Participants agree to follow rules in protocol. jschmied©2013

3. PHASES IN A CLINICAL TRIAL 1. Test the new drug on a small group. Find out effectiveness, safety, dosage & side effects 2. Test Larger group. Double check effectiveness & safety. 3. Test Even larger group, confirm effectiveness, safety… side effects, compare effectiveness to other drugs already being used. 4. Studies done after drug released to public to get effect on different populations. Some proposed drugs are Safe, but not Effective jschmied©2013

4. CLINICAL TRIALS Control Trials => Study Subjects are given Placebos! Experimental Trials=> Study Subjects are given the Experimental Medicine

7. THE PLACEBO EFFECT pla·ce·bo ef·fect 1. A positive effect or cure, produced by a placebo “drug” or treatment. The effect is not caused by the placebo. The effect is solely due to the patient's belief in the treatment. jschmied©2014

9. WHAT IS THE PLACEBO? What Is a Placebo? anything that seems to be a "real" medical treatment -- but isn't. It could be a pill, a shot, or some other type of "fake" treatment. all placebos have nothing in them that can treat a medical condition. How Are Placebos Used? Given to study subjects in the Control Trial. The study subjects don’t know if they are taking real medication or not! So researchers can get the effectiveness of the trial drug. Researchers subtract out the Placebo effect to get the effectiveness of the drug. jschmied©2013

10. PLACEBO EFFECT

17. HOW TO CALCULATE EFFECTIVENESS USING CLINICAL TRIAL DATA Calculating Effectiveness Step One – The “Red Zone” • The red zone, is the section of the data table that records all the people that report being cured for each trial in a clinical trial. • In order to calculate the effectiveness of the medication being tested one has to subtract ET = the % people who report being cured with the medicine - CT = the % people who report being cured with a placebo Effectiveness jschmied©2014 Data Table 2 – A Clinical Trial Same as yellow (not cured) Better than yellow (cured) Worse than Yellow (cured w/side effects) Sampl e size Control Trial (Placebo) 5 33% 1 7% 9 60% 15 Experimental Trial (Medicine) 1 7% 12 69% 2 14% 14

18. HOW TO CALCULATE EFFECTIVENESS USING CLINICAL TRIAL DATA Do the Math and check your answer on the next slide jschmied©2014 Data Table 2 – A Clinical Trial Same as yellow (not cured) Better than yellow (cured) Worse than Yellow (cured w/side effects) Sample size Control Trial (Placebo) 5 33 % 1 7% 9 60% 15 Experiment al Trial (Medicine) 1 7% 12 69% 2 14% 14

19. HOW TO CALCULATE EFFECTIVENESS Data Table 2 – A Clinical Trial Same as yellow (not cured) Better than yellow (cured) Worse than Yellow (cured w/side effects) Sample size Control Trial (Placebo) 5 33 % 1 7% 9 60% 15 Experiment al Trial (Medicine) 1 7% 12 69% 2 14% 14 ET = 69% + 14% = 83% - CT = 7% + 60% = 67% Med Effectiveness = 16% Step Two Do the Math! In this scenario the Placebo Effect is 67%! Thus, the drug is Not very Effective = 16%! jschmied©2014

20. HOW TO CALCULATE SAFETY WITH CLINICAL TRIAL DATA Calculating Safety– The “Orange Zone” • The orange zone, is the section of the data table that records all the people that report being cured with Side Effects for the Experimental trial in a clinical trial. • In order to calculate the Safety of the medication being tested one has to subtract the Orange Zone data from 100% 100 - The % people who report being cured w/side effects in the ET jschmied©2014 = % Safety (of the medicine) Data Table 2 – A Clinical Trial Not Cured Cured Cured with Side Effects Sample size Control Trial (Placebo) 5 33% 1 7% 10 60% 15 Experimental Trial (Medicine) 1 7% 12 69% 2 14% 14

21. HOW TO CALCULATE SAFETY & EFFECTIVENESS USING CLINICAL TRIAL DATA Data Table 2 – A Clinical Trial Not Cured Cured Cured with Side Effects Sample size Control Trial (Placebo) 5 33 % 1 7% 9 60% 15 Experime ntal Trial (Medicine) 1 7% 12 69% 2 14% 14 Do the Math and check your answer on the next slide! jschmied©2014

22. HOW TO CALCULATE SAFETY WITH CLINICAL TRIAL DATA Data Table 2 – A Clinical Trial Not Cured Cured Doing the Math Cured with Side Effects Sample size Control Trial (Placebo) 5 33 % 1 7% 9 60% 15 Experimen tal Trial (Medicine) 1 7% 12 69% 2 14% 14 100% -14% (% cured but have side effects) 86% Safe (% with no side effects) jschmied©2014