Basic introduction to clinical trials and the placebo effect. Definitions, examples and cartoons illustrating the subject. Ends with short info on informed consent.

1. CLINICAL TRIALS & THE PLACEBO EFFECT
Plus Calculating Safety
& Effectiveness
In Clinical Trials
jschmied©2013

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

5. CLINICAL TRIALS TEST EFFECTIVENESS & SAFETY
Some people
volunteer for
lots of
Clinical trials!
jschmied©2013

6. PLACEBOS & THE PLACEBO EFFECT
What is a placebo anyways?
jschmied©2013

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

8. DO DOCTORS GIVE PLACEBOS?
jschmied©2013

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

11. PLACEBO ANYONE?
jschmied©2013

12. “If I don’t think it’s going to work, will it still work?”
jschmied©2013

13. The Opposite of the Placebo Effect is the Nocebo Effect
jschmied©2013

14. CLINICAL TRIALS IN A NUTSHELL
jschmied©2013

15. HOW TO CALCULATE SAFETY & EFFECTIVENESS
USING CLINICAL TRIAL DATA
jschmied©2013

16. INFORMED CONSENT - 2 PARTS
1. KNOW THE RISKS & 2. AGREE TO PARTICIPATE
jschmied©2014

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

23. THATS ALL FOLKS!!!!
jschmied©2014