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Biostatistics for Health Care Researchers: A Short Course
Hypothesis Testing and
Confidence Interval Estimation
Presented
ese ed by
by:
Susan M. Perkins, Ph.D.
Division of Biostatistics
Indiana University School of Medicine
1
Obj ti
Objectives



Introduce concepts of hypothesis
testing
g and confidence interval
estimation
Understand how decisions are
made based on the sampled data
Distinguish statistical and clinical
significance
2
Examples
• Do children with new onset seizures have
lower levels of neuropsychological
functioning than a normative sample?
• Are women of childbearing age drinking
more caffeine than recommended?
3
St ti ti l Inference
Statistical
I f
Target
Population
Generalization
Population
Sampled
Sample
about larger
population
Inference about
population
Example 1
• Target Population: All children
ages 6-14 with new onset
seizures
• Population Sampled: Children in
Indiana and in Cincinnati area
with new onset seizures
• Sample: Children that participate
in study
Example 2
• Target Population: All women
age 16-45
16 45 in UK
• Population Sampled: women in
Manchester area
• Sample: women recruited from
b i
business
offices
ffi
Hypothesis Testing: Basic Concepts






Null and Alternative (H0 and H1)
One-sided
One
sided and two-sided
two sided tests
Test statistic and its sampling
distribution
Rejection region
Type I and Type II errors
Sample size and power
7
Example
p 1
Question: Is processing speed (measured
by WISC-III Coding) for children with new
onsett seizures
i
different
diff
t th
the normative
ti
score of 10?
Population:
 Normally distributed
 Standard deviation =3
Sample summary:
y
 n  282 ,
X  9.8
Fastenau et al. under review
8
Example 1 (Continued)
(C
)



Null Hypothesis H0 := 10
Alternative Hypotheses:
yp
One-sided: H1 : < 10, or H1 : > 10
Two-sided: H1 :  10
In this case, we are interested in
H0 :
 = 10 versus H1 :
  10
9
T t Statistic
Test
St ti ti
Two characteristics:
 The value of a test statistic
depends on a sample
 The sampling distribution of a test
statistic under the null hypothesis
is known
10
O Sample
One
S
l Test
T t off th
the M
Mean


When the sample is from a normal
population (OR when the sample
size
i iis llarge),
) one can use Z
Z-Test
T t
to test the mean of the population
Test statistic
X  0
Z
/ n
11
Central Limit Theorem and the
Sampling Distribution of Z Under H0


CLT: Let X1,…Xn be a random sample
from a population with mean  and
standard deviation .
If H0: = is true, the distribution of Z
can be approximated by a N(0,1)
distribution.
0
12
0.15
0.10
0.05
0
0.0
f(z)
0.20
0.25
0.30
Sampling Distribution
of Z
-4
-2
0
2
Z
4
6
•Centered
at 0
•Most
values
b t
between
±5
•Area
under
curve = 1
13
Steps in Hypothesis Testing



Assume H0 is true
Under H0, Z will have a known
sampling distribution (normal
distribution)
If the Z from your sample takes an
unlikely (large) value, H0 is not
likely to be true (proof by
contraction).
14
Significance Level and
Rejection Region




We reject the null hypothesis when
the Z value is “large”.
g But how large
g is
“large”?
Significance level (): The maximum
probability of incorrectly rejecting H0
you are willing to accept.
Rejection region: We reject H0 if the
value of Z falls into the region.
R j ti R
Rejection
Region
i d
depends
d on H1.
15
Rejection
j
Region
g for H : 
<

(=0.05)
1
0
0.4
z = -1.645
0.0
0.1
Denssity of Normal
Curve
0.2
0.3
3
Area below z = 5 %
-3
-2
-1
0
1
2
3
Z
16
Rejection Region for H : >
(=0.05)
1
0
0.4
z = 1.645
1 645
0.0
0.1
Density of Normal
Cu
urve
0.2
0
0.3
Area above z = 5 %
-3
-2
-1
0
1
2
3
Z
17
Rejection
j
Region
g for H : 


(=0.05)
1
0
0.4
z = -1.96
Area above z = 2.5 %
0.0
0.1
Density o
of Normal
Currve
0.2
0.3
Area below z = 2.5 %
-3
-2
-1
0
1
2
3
Z
18
Significance Level and p-value
p value




Significance level is also called α (alpha) –
decided up-front
If the p
probability
y of the observed results is
below this number, you reject the null
hypothesis
P-value: probability of obtaining a result as
or more extreme than the observed data if
H0 is
i true – calculated
l l d ffrom the
h d
data
A small p-value leads to the rejection of the
nullll h
hypothesis
th i
19
E
Example
l 1 (Continued)


H0: = 10 versus H1:   10
=0.05
X  0 9.8  10
Z

 1.1
 / n 3 / 282


Rejection
j
Region:
g
Z<-1.96,, Z>1.96
P-value = .26
20
One Sample t Test



When the population standard
deviation  is unknown, use
sample
l standard
t d dd
deviation
i ti s to
t
estimate .
X  0
Test statistics T  s / n
T has t-distribution with n-1
degrees of freedom
21
0.3
0
T Distribution with
Various Degrees of Freedom
0.0
0.1
f((x)
0.2
DF=2
DF=10
-10
-5
0
5
10
x
22
Example 2
Question: Is the mean intake of caffeine in
Women of childbearing age different that
the FDA guidelines of 300 mg/day?
Population:
 Normally distributed
 Standard deviation s=128 mg/day
g y
Sample summary:
 n  70 ,
g y
X 174 mg/day
Derbyshire
y
& Abdula. Habitual Caffeine Intake in Women of
Childbearing Age. J Hum Nutr Diet, 21 (159-164)
23
E
Example
l 2 (Continued)


H0: = 300 versus H1:   300
=0.05
X  0
174  300
T

 8.2
82
s / n 128 / 70


Rejection Region: Z<-1.96, Z>1.96
P-value
P
value < .0001
24
Type I and Type II Errors
Decision
Reject H0
Not Reject H0
Null Hypothesis
True
False
Correct
Type I ()
Correct Type II ()
25
Type I and Type II Errors




Type I : Pr{rejecting H0| H0 true} = 
T
Type
II:
II Pr{not
P{
rejecting
j i H0| H1 true}} = 
Pr{rejecting H0| H1 is true} = 1- = Power
Typically use:  = 0.05; Power = 0.80 for
hypothesis testing and calculating
sample sizes
26
Sample Size and Power




How does sample size affect the
power?
X  0
E
Example
l Z statistic
t ti ti Z   / n
One can always increase the
power of a test by increasing the
sample size
Sample size calculation in the
planning stage
27
Clinical and Statistical
Significance




The two do not always agree
Clinicallyy significant
g
but not
statistically significant: n too small
Statistically significant but not
clinically significant: n too large
Think about what clinical difference
is important when designing a
study or critiquing someone else's
study.
study
28
C fid
Confidence
Intervals
I t
l




100(1-)%
100(1
)% confidence inter
interval
al for the
mean
X  Z / 2 / n
 known:
 unknown:
X  t / 2 ( n1) s / n
I t
Interpretation:
t ti
100(1 )% confident
100(1-)%
fid t
that this interval will contain the true
mean 
29
I t
Interpreting
ti C
Confidence
fid
Intervals
I t
l
30
Example: Confidence Interval

95% CI for mean processing speed
9 8  11.96
9.8
96  3 / 282

(9.4, 10.2): We are 95% confident
that the interval between 9.4 and
10.2 captures the true mean
processing speed for this group
group.
31
Summary



Hypothesis testing: concepts and
procedure
Errors, power and sample size
Confidence intervals
32
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