Introduction to Research Methods

In the Internet Era

Introduction to Biostatistics
Inferential Statistics
Hypothesis Testing
Thomas Songer, PhD
with acknowledgment to several slides provided by
M Rahbar and Moataza Mahmoud Abdel Wahab

Key Lecture Concepts

Assess role of random error (chance) as an
influence on the validity of the statistical
association
Identify role of the p-value in statistical
assessments
Identify role of the confidence interval in
statistical assessments
Briefly introduce tests to undertake
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Research Process
Research question
Hypothesis
Identify research design
Data collection
Presentation of data
Data analysis
Interpretation of data
Polgar, Thomas

Interpreting Results
When evaluating an association between
disease and exposure, we need guidelines
to help determine whether there is a
true difference in the frequency of disease
between the two exposure groups, or perhaps
just random variation from the study sample.
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Random Error (Chance)

1.

Rarely can we study an entire population, so

inference is attempted from a sample of
the population

2.

There will always be random variation

from sample to sample

3.

In general, smaller samples have less

precision, reliability, and statistical power
(more sampling variability)
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Hypothesis Testing
The process of deciding statistically
whether the findings of an
investigation reflect chance or real
effects at a given level of probability.

Elements of Testing hypothesis

Null Hypothesis
Alternative hypothesis
Identify level of significance
Test statistic
Identify p-value / confidence interval
Conclusion
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Hypothesis Testing
H0: There is no association between the
exposure and disease of interest
H1: There is an association between the
exposure and disease of interest
Note: With prudent skepticism, the null hypothesis
is given the benefit of the doubt until the data
convince us otherwise.
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Hypothesis Testing
Because of statistical uncertainty regarding
inferences about population parameters based
upon sample data, we cannot prove or disprove
either the null or alternate hypotheses as
directly representing the population effect.
Thus, we make a decision based on probability
and accept a probability of making an incorrect
decision.
Chernick

Associations
Two types of pitfalls can occur that affect
the association between exposure and
disease
Type 1 error: observing a difference when in
truth there is none
Type 2 error: failing to observe a difference
where there is one.
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Interpreting Epidemiologic Results

Four possible outcomes of any epidemiologic study:

YOUR
DECISION
Do not reject H0
(not stat. sig.)
Reject H0
(stat. sig.)

REALITY
H0 True
H1 True
(No assoc.)

(Yes assoc.)

Correct
decision

Type II
(beta error)

Type I
(alpha error)

Correct
decision
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Four possible outcomes of any epidemiologic study:

YOUR
DECISION
Do not reject H0
(not stat. sig.)
Reject H0
(stat. sig.)

REALITY
H0 True
H1 True
(No assoc.)

(Yes assoc.)

Correct
decision

Failing to find a
difference when
one exists

Finding a
Correct decision
difference when
there is none
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Type I and Type II errors

is the probability of committing type I
error.
is the probability of committing type II
error.

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Conventional Guidelines:
Set the fixed alpha level (Type I error) to 0.05
This means, if the null hypothesis is true, the
probability of incorrectly rejecting it is 5% or less.

Study Result

DECISION

H0 True

H1 True

Do not reject H0
(not stat. sig.)
Reject H0
(stat. sig.)

Type I
(alpha error)
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Empirical Rule
For a Normal distribution approximately,
a) 68% of the measurements fall within one
standard deviation around the mean
b) 95% of the measurements fall within two
standard deviations around the mean
c) 99.7% of the measurements fall within three
standard deviations around the mean
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Normal Distribution
34.13%
13.59%

34.13%
13.59%

2.28%

2.28%

50%

50 %

usually set at 5%)

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Random Error (Chance)

4. A test statistic to assess statistical significance
is performed to assess the degree to which the
data are compatible with the null hypothesis of no
association
5. Given a test statistic and an observed value, you
can compute the probability of observing a value
as extreme or more extreme than the observed
value under the null hypothesis of no association.
This probability is called the p-value

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Random Error (Chance)

6.
By convention, if p < 0.05, then the
association between the exposure and disease is
considered
to be statistically
significant.
(e.g. we reject the null hypothesis (H 0) and
accept the alternative hypothesis (H 1))
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Random Error (Chance)

p-value
the probability that an effect at least as extreme as
that observed could have occurred by chance alone,
given there is truly no relationship between exposure
and disease (Ho)
the probability the observed results occurred by
chance
that the sample estimates of association differ only
because of sampling variability.

Sever

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Random Error (Chance)

What does p < 0.05 mean?
Indirectly, it means that we suspect that the
magnitude of effect observed (e.g. odds ratio) is
not due to chance alone (in the absence of
biased data collection or analysis)
Directly, p=0.05 means that one test result out of
twenty results would be expected to occur due
to chance (random error) alone
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Example:

E+

D+
15

D85

E-

10

90

IE+ = 15 / (15 + 85) = 0.15

IE- = 10 / (10 + 90) = 0.10
RR = IE+/IE- = 1.5, p = 0.30

Although it appears that the incidence of disease may be

higher in the exposed than in the non-exposed (RR=1.5),
the p-value of 0.30 exceeds the fixed alpha level of 0.05.
This means that the observed data are relatively
compatible with the null hypothesis. Thus, we do not
reject H0 in favor of H1 (alternative hypothesis).

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Random Error (Chance)

Take Note:
The p-value reflects both the magnitude of the
difference between the study groups AND the
sample size
The size of the p-value does not
indicate the importance of the results
Results may be statistically significant
but be clinically unimportant
Results that are not statistically
significant may still be important

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Sometimes we are more concerned with

estimating the true difference than the
probability that we are making the decision
that the difference between samples is
significant

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Random Error (Chance)

A related, but more informative, measure known
as the confidence interval (CI) can also be
calculated.
CI = a range of values within which the true
population value falls, with a certain degree of
assurance (probability).
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Confidence Interval - Definition

A range of values for a variable constructed so
that this range has a specified probability of
including the true value of the variable
A measure of the studys precision

Point estimate
Lower limit
Sever

Upper limit
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Statistical Measures of Chance

Confidence interval
95% C.I. means that true estimate of
effect (mean, risk, rate) lies within 2
standard errors of the population
mean 95 times out of 100

Sever

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Interpreting Results
Confidence Interval: Range of values for a point
estimate that has a specified probability of
including the true value of the parameter.
Confidence Level: (1.0 ), usually expressed
as a percentage (e.g. 95%).
Confidence Limits: The upper and lower end
points of the confidence interval.
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Hypothetical Example of 95% Confidence Interval

Exposure:
Outcome:
Risk Ratio:
p-value:
95% C.I.:

Caffeine intake (high versus low)

Incidence of breast cancer
1.32 (point estimate)
0.14 (not statistically significant)
0.87 - 1.98
95% confidence interval

_____________________________________________________
0.0
0.5
1.0
1.5
2.0
(null value)
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Random Error (Chance)

INTERPRETATION:
Our best estimate is that women with high caffeine
intake are 1.32 times (or 32%) more likely to develop
breast cancer compared to women with low caffeine
intake. However, we are 95% confident that the
true value (risk) of the population lies between
0.87 and 1.98 (assuming an unbiased study).
95% confidence interval

_____________________________________________
0.0
0.5
1.0
1.5
2.0
(null value)
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Random Error (Chance)

Interpretation:
If the 95% confidence interval does NOT include
the null value of 1.0 (p < 0.05), then we declare a
statistically significant association.
If the 95% confidence interval includes the null
value of 1.0, then the test result is not statistically
significant.
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Interpreting Results
Interpretation of C.I. For OR and RR:
The C.I. provides an idea of the likely magnitude of
the effect and the random variability of the point
estimate.
On the other hand, the p-value reveals nothing about
the magnitude of the effect or the random variability
of the point estimate.
In general, smaller sample sizes have larger C.I.s due
to uncertainty (lack of precision) in the point estimate.
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Selection of Tests of
Significance

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Scale of Data
1. Nominal: Data do not represent an amount or
quantity (e.g., Marital Status, Sex)
2. Ordinal: Data represent an ordered series of
relationship (e.g., level of education)
3. Interval: Data are measured on an interval scale
having equal units but an arbitrary zero point. (e.g.:
Temperature in Fahrenheit)
4. Interval Ratio: Variable such as weight for which we
can compare meaningfully one weight versus another
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(say, 100 Kg is twice 50 Kg)

Which Test to Use?

Scale of Data
Nominal

Chi-square test

Ordinal

Mann-Whitney U test

Interval (continuous)
- 2 groups

T-test

Interval (continuous)
- 3 or more groups

ANOVA
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Protection against Random Error

Test statistics provide protection from
type 1 error due to random chance
Test statistics do not guarantee
protection against type 1 errors due to
bias or confounding.
Statistics demonstrate association, but
not causation.

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