Clinical Trials

Intervention (unlike observation)

Has control group

3 Phases for any drug to be approved

by FDA
 Phase I Healthy (volunteers)

Phase II Few Patients

Phase III Many Patients

 Phase I Healthy (volunteers)
Assesses Intervention Safety

Phase II Few Patients

Assesses Intervention Protocol

Phase III Many Patients

Assesses Intervention Efficacy
and SE
 Types of Clinical Trials
In RCT in general, subjects are RANDOMLY assigned into Intervention group
or Control group

Researchers know whos getting intervention: I know

Patient knows if hes getting Intervention: He know

Randomized Controlled Trial

I know, He DONT know

Double Blind Randomized Controlled Trial

I DONT know, He DONT know
The Best, Least Bias, Most expensive
Types of Clinical Trials
Community Trial
Test on entire community
Tells u if Intervention works in THE REAL WORLD

Crossover Studies Ethical

No group involved can remain untreated
Control & Intervention Group switch
But at each point, one group is control, other is
intervention

Cont Interventi
rol on
 Confounding: universally present in ALL
studies
relationship between exposure and outcome is
distorted by the presence of another variable

It is a risk factor for the disease, independent

of the assumed risk factor.
It is associated with assumed risk factor.
It is not in the causal pathway between
exposure and disease.

Best way to solve

Confounding:
 Bias: deviation from truth

Reliability: precision/consistency

Validity: accuracy (bulls eye)

If something is valid, then it must also

be reliable (not the other way)
 Selection Bias: sample not representative of population
Berkson Bias: using Hospital records as a representative
sample
Non-Respondent Bias: people refuse to participate in
research study
Mostly seen in Cohort study (cuz u need large sample)
Solution: Get a good random sample

Measurement (Information) Bias: info gathered in a

way that distorts the info
Asking leading questions
Hawthorne Effect: subjects behavior changes because
they realize they are being studied
Solution: Get a control group
 Experimenter Expectancy Bias:
expectations are unintentionally
communicated to subjects so they
produce the desired effect
U tell teacher these 5 students are the
brightest. So the teacher ends up spending
most time with them and their grades are
best in the end
Aka Pygmalion Effect
Solution: Double Blind Design
I dont know, He dont know
Lead Time Bias: False estimate of
Survival Rate
1st: Usual process

2nd: Screened early (detected earlier) but death still at

same time

3rd: Detected earlier but Death is later

Disease Diagnosis Deat

Onset h
Lead Time

Improved
Survival
Lead Time Bias

Solution: STOP measuring/saying

how long patient lives after
Diagnosis. And instead, use the
measure/term Life Expectancy
If you already have the solution,
then you cant have that BIAS!!

If in the question the study is a Double

Blind, then you CANT have Experimenter
Expectancy Bias

If in the question the study has a

Control Group, then you CANT have
Measurement Bias
 Recall Bias: failure to recall correctly
Different than not recalling at all (cuz that
would be missing data, not bias)
Retrospective study: Case-Control
Solution: Confirmation or
Triangulation
Most Common Bias for

Cohort: Selection(Sample)
Bias

Case-Control: Recall Bias

 Late Look Bias: severe disease less
likely to be covered
Cuz the severe cases are dead or too
demented; so these arent well
represented
Solution: Stratify by Disease
Severity
In order to get equal representation across
different disease severities
 Sample not
Get Good Random
Selection representative (esp.
Sample
seen in Cohort)
Using Hospital
Berkson Records
Non- People refuse to
Respondent Participate

Data gathered in a
Measurement method that distorts Control Group
the data

Hawthorne People act differently

cuz they know theyre
Effect being studied
Expectations
Experimenter unintentionally Double Blind
Expectancy communicated to
subjects
Study
Earlier diagnosis
Measure Life
Lead Time confused for
Expectancy
improved survival
Subjects incorrectly
Confirmation/Trian
 Design Bias: Non-comparable Control
Group
Not a selection/sample bias, this is a
subject assignment bias

Solution: Random assignment into

Intervention and Control Group