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Descriptive Methods for
  Categorical Data
  Mahmoud Alhussmi, D.Sc., PhD
Topics
• Proportions
• Rates
      – Change rates
      – Measures of morbidity and mortality
      – Standardization of rates
• Ratios
      – Relative risk
      – Odds Ratio

March 28, 2013                                2
Categorical Data
Data that can be classified as belonging to a
distinct number of categories.
      – Binary – data can be classified into one of 2
        possible categories (yes/no, positive/negative)
      – Ordinal – data that can be classified into
        categories that have a natural ordering (i.e..
        Levels of pain: none, moderate, intense)
      – Nominal- data can be classified into >2
        categories (i.e.. Race: Arab, African, and
        other)

March 28, 2013                                        3
Data examples
• What type of data would result from these
  questions?
      – How old are you? ____________
      – How old are you?
            •    A. under 18
            •    B. 19-35
            •    C. 36-49
            •    D. over 50



March 28, 2013                                4
Proportions
• Numbers by themselves may be misleading: they are on
  different scales and need to be reduced to a standard
  basis in order to compare them.
• We most frequently use proportions: that is, the fraction
  of items that satisfy some property, such as having a
  disease or being exposed to a dangerous chemical.
• "Proportions" are the same thing as fractions or
  percentages. In every case you need to know what you
  are taking a proportion of: that is, what is the
  DENOMINATOR in the proportion.
           x                    x
        p=
           n     percent (100) = (100)
                                n
March 28, 2013                                                5
Proportions and Probabilities
• We often interpret proportions as
  probabilities. If the proportion with a
  disease is 1/10 then we also say that the
  probability of getting the disease is 1/10, or
  1 in 10.
• Proportions are usually quoted for samples
  - probabilities are almost always quoted for
  populations.


 March 28, 2013                                6
Workers Example
    Smoking           Workers   Cases Controls
        No            Yes        11      35
                      No         50     203
              Yes     Yes        84      45
                      No        313     270
• For the cases:
    – Proportion of exposure=84/397=0.212 or 21.2%
• For the controls:
    – Proportion of exposure=45/315=0.143 or 14.3%

 March 28, 2013                                  7
Prevalence
Disease Prevalence = the proportion of people with a given
disease at a given time.

disease prevalence =
      Number of diseased persons at a given time
     Total number of persons examined at that time

Prevalence is usually quoted as per 100,000
people so the above proportion should be
multiplied by 100,000.

March 28, 2013                                           8
Interpretation

                                  Cases (old +new)
At time t         Pr evalence =
                                       Total




     Problem of exposure, consequently
     Not comparable measurement
     Old = duration of the disease
     New = speed of the disease

 March 28, 2013                                      9
Screening Tests
• Through screening tests people are
  classified as healthy or as falling into one
  or more disease categories.
• These tests are not 100% accurate and
  therefore misclassification is unavoidable.
• There are 2 proportions that are used to
  evaluate these types of diagnostic
  procedures.

March 28, 2013                                   10
Screening Tests
                 General Population

                                       Diseased
                                       Positive
                                       Test Results




March 28, 2013                                    11
Sensitivity and Specificity
• Sensitivity and specificity are terms used to
  describe the effectiveness of screening tests. They
  describe how good a test is in two ways - finding
  false positives and finding false negatives

• Sensitivity is the Proportion of diseased who
  screen positive for the disease

• Specificity is the Proportion of healthy who screen
  healthy

March 28, 2013                                    12
Sensitivity and Specificity
                    Condition Present              Condition Absent
……………………………………………………………………………………………
Test Positive     True Positive (TP)             False Positive (FP)
Test Negative     False Negative (FN)            True Negative (TN)
……………………………………………………………………………………………
 Test Sensitivity (Sn) is defined as the probability that the test is positive
   when given to a group of patients who have the disease.
     Sn= (TP/(TP+FN))x100.
     It can be viewed as, 1-the false negative rate.
 The Specificity (Sp) of a screening test is defined as the probability that the
  test will be negative among patients who do not have the disease.
     Sp = (TN/(TN+FP))X100.
     It can be understood as 1-the false positive rate.
Positive & Negative Predictive Values

• The positive predictive value (PPV) of a
  test is the probability that a patient who
  tested positive for the disease actually has
  the disease. PPV = (TP/(TP+FP))X 100.
• The negative predictive value (NPV) of a
  test is the probability that a patent who
  tested negative for a disease will not have
  the disease. NPV = (TN/(TN+FN))X100.
The Efficiency
• The efficiency (EFF) of a test is the
  probability that the test result and the
  diagnosis agree.
• It is calculated as:
  EFF = ((TP+TN)/(TP+TN+FP+FN)) X 100
Example
• A cytological test was undertaken to screen
  women for cervical cancer.
                         Test Positive   Test Negative   Total

     Actually Positive    )TP (154        )FP (225       379
   Actually Negative       )FN (362    )TN (23,362 23,724
                         )TP+FN (516 )FP+TN(23587


• Sensitivity =?
• Specificity = ?
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Displaying Proportions
• Types of charts that can be used:
      – Histogram
      – Pie Chart
      – Line Graph


• BEWARE of the type of display you use –
  some charts are better at displaying
  certain types of data than others.

March 28, 2013                              17
Displaying Proportions
                                   Percent of Children LivingSudan
                                    Distribution of Race in in
           African 70%     80%
                                   Crack/cocaine Households

                           80
             Arab 18%      70%
                           70                      Percent of Children Living in
                           60%                     Crack/cocaine Households

            Mixed 8%       60
                           50%
                           50
                           40%
                           40
                           30%

             Other   4%    30
                           20%
                            20
                           10%
                           10
                           0%
                            0    Black     White          American           Other
                                                           Indian
                                 African      Arab               foreigners          others




March 28, 2013                                                                            18
Displaying Proportions
                 Distribution of Race in Sudan        Distribution of Race in Sudan


   others


foreigners
                                                                                      African
                                                                                      Arab
     Arab
                                                                                      foreigners
                                                                                      others
  African


             0          20        40        60   80




  March 28, 2013                                                                               19
Displaying Proportions
              Cause of Death of Deaths# Proportion of Deaths
                  Heart Disease    12,278                                 0.38
                        Cancer      6,448                                 0.20
Cerebrovascular Disease             3,958                                 0.12
              Accidents             1,814                                 0.06
                         Other      8,088                                 0.25
                              Causes of Death


                                                Heart Disease
                                                Cancer
                                                Cerebrovascular disease
                                                Accidents
                                                Other




 March 28, 2013                                                                  20
Rates
• The term rate is often used interchangeably
  with the term proportion although
  sometimes it refers to a quantity of a very
  different nature.
• Types of rates we will cover:
    – Incidence rate
    – Change rates
    – Death rate
    – Follow-up death rate

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Calculation of Incidence
          Rate



March 28, 2013         22
Definition
The incidence rate is the production of new cases in a
  population. It measure the number of cases per unit
  of time, i.e. It measure the average of the speed of
  the apparition of new cases in given population.
There are three measures of incidence:
1. Incidence rate (=news cases/time of participation)
2. Instantaneous incidence
3. Accumulate incidence


 March 28, 2013                                         23
In incidence study we are interested on the
occurrence of event (disease) over the time,.
Such study deals with follow up of
each subject and the moment (ti) that each event
(disease) occurs.
The problem of incidence data is that the existence
of
observation incomplete, subjects are still not
affected at the moment of analysis.


March 28, 2013                                    24
To study incidence rate we should know for
each subject the following information:


Date of origin
          Is the date that an individual enter in the study.
Date of last information
        Is the recent date that we receive information about
the status of the subject. If the subject is affected, so the date
of last information is date of getting the disease.



  March 28, 2013                                                 25
Duration of follow up
          Is the delay between the date of origin and the date
  of last information


  Date of point
        Is the date that we decide to stop collecting
  information about subjects.


Loss of view
       A subject that we do not know his status at the date
of point is called loss of view.

 March 28, 2013                                               26
Time of participation t i
    Time of participation for loss of view




           Time of participation for loss of view

                                                                Time not consider

 t1, t2,
t3, ….,
time 0                                                                              ?

                                   Date of last     Date of point
                                   information

   Time of participation for affected
                subject

                          Time of participation

 March 28, 2013                                                                     27
Example
The following example follow 30 individuals between 1982 to 1988 for the disease D.


                  of individual#   Date of origin   Date of disease   Date of last        Time of
                                                                      information    participation

                               1          11-82               1-84         12-88               14

                               2          11-82              10-87         12-88               59

                               3            6-82              2-86           6-87              44

                               4          11-82              11-86         12-88               36

                               5          11-82               3-85         12-88               28

                               6          11-82               1-88         12-88               62

                               7            6-83              3-84           6-85               9

                               8            1-83             12-86         12-87               47

                               9            1-84              1-87         12-88               36

                             10           11-82              10-85         12-88               35

                             11           12-82               8-86         12-88               44

                             12             1-83              6-83           7-85               5

                             13             6-83             11-87           7-88              53

                             14           11-82               8-84           2-87              21




 March 28, 2013                                                                                      28
15            1-83   3-86   12-88   38

                      16            6-83   5-87   12-88   47

                      17            5-83   1-84   12-88   8

                      18           11-83   6-88   12-88   55

                      19            6-83   5-86   12-87   35

                      20            3-83   2-88   12-88   59

                      21            4-83          12-88   68

                      22           11-82          11-85   36

                      23            1-83          12-88   71

                      24            6-83          12-88   66

                      25           11-82          11-86   48

                      26            6-82          12-88   78

                      27           12-83          12-88   60

                      28           11-82          12-88   73

                      29            6-84          12-88   54

                      30            1-83           1-84   12



Time of participation = 3001
Number of cases = 20
Rate of incidence = 20/3001 = 0.015
Or 1.5 case per 100 individuals.

March 28, 2013                                                 29
Sometimes it is difficult to know the exact date of origin of the case or even the duration of
follow up, and this is always take place when the population under study is open . In this
case what we know very well :
1.Number of new cases, but not the time exact of participation = m
2.Total number of population from the begin to end of the study = N
The calculation using the same method as before will be impossible, because many
information was missed.
The assumption over which we are going to build our hypothesis is that all the cases enter
or quiet the study are distributed uniformly during the period of follow up, i.e. the date of
follow up for each of these subject will be the half average of the period of the follow up of
the study.
The calculation will be as follow:
 Item                           Number                          time of participation
Not diseases                    N                               N*Δt
Enter along the study           Ne                              Ne*Δt/2
Quiet along the study           Ns                              Ns*Δt/2
Disease                         m                               m*Δt/2

                                                    m
                              IR =
                                                                  ∆t
                                     ( 2 N + N e + N s + m)
                                                                  2
March 28, 2013                                                                              30
Change Rates
   • These types of rates are used to describe
     changes after a certain period of time.
                                     new value - old value
                 change rate (%) =                         X 100
                                          old value
   • Example: A total of 35,238 new AIDS cases
     were reported in 1989 compared to 32,196
     reported during 1988.
         – The change rate for new AIDS cases:
                     35,238 − 32 ,196 
                                      100 = 9.4%
                         32 ,196      
March 28, 2013                                                     31
Measures of Morbidity and Mortality
                         # deaths in a calendar year
     crude death rate =
                        the population on that year

                      # of people that developed the disease
                      over a defined period of time (ie. a year)
     incidence rate =
                      # of people at risk who were followed
                      for the defined period of time (ie. a year)

                               number of deaths
     follow - up death rate =
                              total person - years
March 28, 2013                                                      32
Crude Death Rate
• Example: The 1980 population in
  California was 23,000,000 (as estimated
  on 1 July) and there were 190,237 deaths
  during that year.

      – Crude death rate
        =(190,237/23,000,000)*1,000
        = 8.3 deaths per 1,000 per year


March 28, 2013                               33
Displaying Proportions over Time
                 Death Rate per
                    100,000
                                               Female Death Rates (1984-1987)
1984                  793         815

                                  810

                                  805

1985                  807         800

                                  795                     Death Rate per 100,000
                                  790


1986                  809
                                  785

                                  780
                                        1984           1985               1986     1987




1987                  813



March 28, 2013                                                                        34
Standardization of Rates
   • Crude rates are used to describe a population but
     comparisons of crude rates are often invalid because
     the populations may be different w.r.t important
     characteristics (ie. age, gender, race).
   • To account for these differences adjusted rates are
     used in the comparison.

                 # deaths expected 
adjusted rate = 
                 # in standard population  X100,000
                                           
                                          



March 28, 2013                                              35
Group A                    Group B
                  no.             deaths/ no.                    deaths/
Age group        deaths   persons 100000 deaths      persons     100000

    0-4           162     40,000     405.0   2,049   546,000       375.3

   5-19           107     128,000     83.6   1,195   1,982,000      60.3

  20-44           449     172,000    261.0   5,097   2,676,000     190.5

  45-64           451     58,000     777.6 19,904 1,807,000       1101.5

    +65           444      9,000    4933.3 63,505 1,444,000       4397.9

     Totals      1613     407000     396.3   91750   8455000      1085.2



March 28, 2013                                                       36
:Using the X population for 1970 as a standard we get
                                     Group A                     Group B
 Age                         Age spec.         Exp        Age spec.       Exp
group            Standard      rate           deaths        rate         deaths
  0-4              84,416       405.0               342      375.3            317
 5-19             294,353        83.6               246      60.3             177
 20-44            316,744       261.0               827      190.5            603
 45-64            205,745       777.6             1600      1101.5            2266
  +65              98,742       4933.3            4871      4397.9            4343
 Totals          1,000,000                        7886                        7706
Expected deaths for Group A for age group 65+ = (98,742)
(4933.3)/100,000 = 4871
         Age adjusted rate for
                   = Group A             788.6    100,000*(7886/1,000,000)=
         Age adjusted rate for
                   = Group B             770.6    100,000*(7706/1,000,000)=
March 28, 2013                                                                  37
Relative Risk
   • Relative risks are the ratio of risks for two different
     populations (ratio=a/b).
                             disease incidence in group 1
             Relative Risk =
                             disease incidence in group 2

   • If the risk (or proportion) of having the outcome is 1/10
     in one population and 2/10 in a second population, then
     the relative risk is: (2/10) / (1/10) = 2.0

   • A relative risk >1 indicates increased risk for the group
     in the numerator and a relative risk <1 indicates
     decreased risk for the group in the numerator.

March 28, 2013                                                 38
Odd’s Ratio and Relative Risk
• Odds ratios are better to use in case-
  control studies (cases and controls are
  selected and level of exposure is
  determined retrospectively)

• Relative risks are better for cohort
  studies (exposed and unexposed subjects
  are chosen and are followed to determine
  disease status - prospective)

March 28, 2013                              39
Odd’s Ratio and Relative Risk
• When we have a two-way classification of
  exposure and disease we can approximate the
  relative risk by the odds ratio
                         Disease
                         Yes       No
                 Yes       A        B        A+B
    Exposure      No       C        D        C+D
• Relative Risk=A/(A+B) divided by C/(C+D)
• Odd’s Ratio= A/B divided by C/D = AD/BC

March 28, 2013                                     40
Relationship Between the Two
                 Measures
            A       C
   RR =         ÷
          A+B C+D
          A(C + D)
        =
          C(A + B)
   if the number of subjects classified as disease positive
   is small compared to those classified as disease negative, then :
        C+D ≅ D
         A+B≅B
   Therefore the relative risk can be approximated by :
                  A
         A*D
   RR ≅        = B
         B*C C
                    D

March 28, 2013                                                         41
Case Control Study Example
   • Disease: Pancreatic Cancer
   • Exposure: Cigarette Smoking

                        Disease
                             Yes   No
            Exposure   Yes 38      81   119
                        No    2    56    58



March 28, 2013                                42
Example Continued
• Relative risk for exposed vs. non-exposed
      – Numerator- proportion of exposed people that
        have the disease
      – Denominator-proportion of non-exposed that
        have the disease

      – Relative Risk= (38/119)/(2/58)=9.26



March 28, 2013                                     43
Example Continued
• Odd’s Ratio for exposed vs. non-exposed
      – Numerator- ratio of diseased vs. non-
        diseased in the exposed group
      – Denominator- ratio of diseased vs. non-
        diseased in the non-exposed group

      – Odd’s Ratio= (38/81)/(2/56)=(38*56)/(2*81)
                    =13.14


March 28, 2013                                       44
Relative Risk
• Relative risk – the chance that a member of a group
  receiving some exposure will develop a disease relative to
  the chance that a member of an unexposed group will
  develop the same disease.
                       P(disease | exposed)
                 RR =
                      P(disease | unexposed)
• Recall: a RR of 1.0 indicates that the probabilities of
  disease in the exposed and unexposed groups are
  identical – an association between exposure and disease
  does not exist.

March 28, 2013                                           45
Relative Risk
• When we have a two-way classification of
  exposure and disease we can calculate
  the relative risk

                        Disease
                        Yes       No
                  Yes    A         B   A+B
    Exposure       No    C        D    C+D


March 28, 2013                               46
Case Control Study Example
   • Disease: Pancreatic Cancer
   • Exposure: Cigarette Smoking

                        Disease
                             Yes   No
            Exposure   Yes 38      81   119
                        No    2    56    58



March 28, 2013                                47
Data Interpretation
•  Consideration:
1. Accuracy
    1.   critical view of the data
    2.   investigating evidence of the results
    3.   consider other studies’ results
    4.   peripheral data analysis
    5.   conduct power analysis: type I & type II
                            True          False
              True         Correct        Type-II
             False         Type -I        Correct
Types of Errors
If You……          When the Null Then You
                  Hypothesis is… Have…….
Reject the null   True (there really    Made a Type I
hypothesis        are no difference)    Error
Reject the null   False (there really   ☻
hypothesis        are difference)

Accept the null   False (there really   Made Type II
hypothesis        are difference)       Error
Accept the null   True (there really    ☻
hypothesis        are no difference)
• alpha : the level of significance used for
  establishing type-I error
• β : the probability of type-II error
• 1 – β : is the probability of obtaining
  significance results ( power)
• Effect size: how much we can say that the
  intervention made a significance
  difference
2. Meaning of the results
    - translation of the results and make it
     understandable
3. Importance:
     - translation of the significant findings into
     practical findings
4. Generalizability:
      - how can we make the findings useful for all
     the population
5. Implication:
      - what have we learned related to what has
     been used during study
POWER--Uses and Misuses
• Sources
  – Cohen Statistical Power Analysis for the
    Behavioral Sciences (gold standard for power)
  – Kraemer & Thieman How Many Subjects?
  (also a good review)
Needed Parameters
•   Alpha--chance of a Type I error
•   Beta--chance of a Type II error
•   Power = 1 - beta
•   Effect size--difference between groups or
    amount of variance explained or how
    much relationship there is between the DV
    and the IVs
?Remember this in English
• Type I error is when you say there is a
  difference or relationship and there is not
• Type II error is when you say there is no
  difference or relationship and there really
  is
?What Affects Power
• Size of the difference in means or amount
  of variance explained (ES)
• alpha
• Unexplained variance
• N
?Which is more important
• Type I error more important if possibility of
  harm or lethal effect
• Type II error more important in relatively
  unexplored areas of research
• In some studies, Type I and Type II errors
  may be equally important
How to Increase Power
1. Increase the n
2. Decrease the unexplained variance--control by design or statistics
    (e.g. ANCOVA)
3. Increase alpha (controversial)
4. Use a one tailed test (directional hypothesis)--puts the zone of
    rejection all in one tail; same effect as increasing alpha
5. Use parametric statistics as long as you meet the assumptions. If
    not, parametric statistics are LESS powerful
6. Decrease measurement error (decrease unexplained variance)--use
    more reliable instruments, standardize measurement protocol,
    frequent calibration of physiologic instruments, improve inter-rater
    reliability
?What is good power
By tradition, “good” power is 80%

The correct answer is it depends on the nature of
  the phenomenon and which kind of error is most
  important in your study. This is a theoretical
  argument that you have to make.
Using convention (alpha = .05 and power = .80,
  beta = .20) you are saying that Type I error is
  _________ as serious as a Type II error
Effect Size
How large an effect do I expect exists in the
 population if the null is false?
OR
How much of a difference do I want to be
 able to detect?
The larger the effect, the fewer the cases
 needed to see it. (The difference is so big
 you can trip on it.)
The World According to Power
                   Kraemer & Thiemann
• The more stringent the significance level, the greater the
  necessary sample size. More subjects are needed for a
  1% level than a 5% level
• Two tailed tests require larger sample sizes than one
  tailed tests. Assessing two directions at the same time
  requires a greater investment.
• The smaller the effect size, the larger the necessary
  sample size. Subtle effects require greater efforts.
• The larger the power required, the larger the necessary
  sample size. Greater protection from failure requires
  greater effort.
• The smaller the sample size, the smaller the power, ie
  the greater the chance of failure
The World According to Power
              Kraemer & Thiemann

• If one proposed to go with a sample size
  of 20 or fewer, you have to be willing to
  have a high risk of failure or a huge effect
  size
• To achieve 99% power for a effect size of .
  01, you need > 150,000 subjects
Test Yourself
Keeping the other parameters the same:
• As ES decreases, needed n ____
• As alpha decreases, needed n ____
• Higher power requires _____ n
Power for each test
• You do a power analysis for each statistic
  you are going to use.
• Choose the sample size based on the
  highest number of subjects from the power
  analysis.
• Use the most conservative power
  analysis--guarantees you the most
  subjects
?What about multiple time points
• More time points requires fewer subjects
  since more is known about the subjects
  from prior time points as compared to a
  cross sectional study
• In other words, less variance is
  unexplained since you have baseline
  information
• How many fewer? It depends
Power analysis and secondary
           analysis
If you have a set sample size, your power
analysis then works backward. You set the
n, alpha and ES and determine the power
given the first three parameters.
Determining ES
If you want to determine effect size from a
   completed study, you have the n, alpha
   and power and can work backwards to
   determine the ES.
Especially important in relatively unexplored
   areas
Power and MR
• ES is the amount of explained variance
  expected since there may not be group
  differences, based on past research
• Increasing the number of independent
  variables _______ sample size needed to
  achieve adequate power.
Sampling Distribution
• A sample statistic is often unequal to the value of the
  corresponding population parameter because of
  sampling error.
• Sampling error reflects the tendency for statistics to
  fluctuate from one sample to another.
• The amount of sampling error is the difference between
  the obtained sample value and the population
  parameter.
• Inferential statistics allow researchers to estimate how
  close to the population value the calculated statistics is
  likely to be.
• The concept of sampling, which are actually probability
  distributions, is central to estimates of sampling error.
Characteristics of Sampling
             Distribution
• Sampling error= sample mean-population mean.
• Every sample size has a different sampling distribution of
  the mean.
• Sampling distributions are theoretical, because in
  practice, no one draws an infinite number of samples
  from a population.
• Their characteristics can be modeled mathematically and
  have determined by a formulation known as the central
  limit theorem.
• This theorem stipulates that the mean of the sampling
  distribution is identical to the population mean.
• The average sampling error-the mean of the (mean-μ)s-
  would always equal zero.
Standard Error of the Mean
• The standard deviation of a sampling
  distribution of the mean has a special
  name: the standard error of the mean
  (SEM).
• The smaller the SEM, the more accurate
  are the sample means as estimates of the
  population value.
• Estimation
• Hypothesis Testing
Both activities use sample statistics (for
              ̅
  example, X) to make inferences about a
  population parameter (μ).




                                             71
• Why don’t we just use a single number (a point
  estimate) like, say, X̅ to estimate a population
  parameter, μ?
• The problem with using a single point (or
  value) is that it will very probably be wrong. In
  fact, with a continuous random variable, the
  probability that the variable is equal to a
                                      ̅
  particular value is zero. So, P(X=μ) = 0.
• This is why we use an interval estimator.
• We can examine the probability that the
  interval includes the population parameter.


                                                      72
Types of Statistical Inference
• Parameter estimation:
  – It is used to estimate a population value, such as a
    mean, relative risk index or a mean difference
    between two groups.
  – Estimation can take two forms:
     • Point estimation: involves calculating a single statistic to
       estimate the parameter. E.g. mean and median.
         – Disadvantages: they offer no context for interpreting their
           accuracy and a point estimate gives no information regarding
           the probability that it is correct or close to the population value.
     • Interval estimation: is to estimate a range of values that has
       a high probability of containing the population value .
• How wide should the interval be? That depends
  upon how much confidence you want in the
  estimate.
• For instance, say you wanted a confidence interval
  estimator for the mean income of a college
  graduate: You might have That the mean income is
                                         between
                     100%               $∞and $0
                 confidence
                       95%    and $41,000 $35,000
                 confidence
                       90%    and $40,000 $36,000
                 confidence
                       80%    and $38,500 $37,500
                 confidence
• The wider the interval, the greater the confidence
                   …                  …
  you will have in it as containing the true population
             confidence 0%(a point estimate )$38,000
  parameter μ.                                            74
Interval Estimation
• For example, it is more likely the population
  height mean lies between 165-175cm.
• Interval estimation involves constructing a
  confidence interval (CI) around the point
  estimate.
• The upper and lower limits of the CI are called
  confidence limits.
• A CI around a sample mean communicates a
  range of values for the population value, and the
  probability of being right. That is, the estimate is
  made with a certain degree of confidence of
  capturing the parameter.
Confidence Intervals around a
                Mean
• 95% CI = (mean + (1.96 x SEM)
• This statement indicates that we can be 95% confident that the
  population mean lies between the confident limits , and that these
  limits are equal to 1.96 times the true standard error, above and
  below the sample mean.
• E.g. if the mean = 61 inches, and SEM = 1, What is 95% CI.
    – Solution: 95% CI = (61 + (1.96 X 1))
                95% CI = (61 + 1.96)
                95% CI = 59.04 < μ < 62.96
• E.g. if the mean = 61 inches, and SEM = 1, What is 99% CI.
    – Solution: 99% CI = (61 + (2.58 X 1))
                99% CI = (61 + 2.58)
                99% CI = 58.42 < μ < 63.58
Confidence Intervals and the t distribution

• When sample size is small then we cannot use
  confidence intervals around the mean, instead, we
  measure confidence intervals by the t-distribution.
• t-distribution is similar to a normal distribution in a
  standard form.
• The exact shape of the t-distribution is influenced by the
  number cases in the sample.
• Statisticians have developed tables for the area under
  the t-distribution for different sample size and probability
  levels.
• To use this table, we must enter at the appropriate row
  based on the number of degrees of freedom.
Confidence Intervals and the t distribution

• 95% CI = (mean + (t x SEM)
   – Where mean = the sample mean
            T = tables t value at 95% CI for df = N-1
            SEM = the calculated SEM for the sample data
• E.g. SEM = 1, mean = 61, N = 25, df = 25-1, t for the
  95% CI with 24 df is 2.06
   – Solution:
                95% CI = (61 + (2.06 X 1))
                95% CI = (61 + 2.06)
                95% CI = 58.95 < μ < 63.06
   To compute CIs around a mean with SPSS:
   Analyze------descriptive stat----explore then click on the statistics
     pushbutton.
Types of Statistical Inference
• Hypothesis testing:
   – Hypothesis testing is a second approach to inferential statistics.
   – Hypothesis testing involves using sampling distributions and the
     laws of probability to make an objective decision about whether
     to accept or reject the null hypothesis.
   – The sample may deviate from the defined population’s true
     nature by certain amount.
   – This deviation is called sampling error.
   – Drawing the wrong conclusion is called an error of inference.
   – There are two types of errors of inference defined in terms of the
     null hypothesis:
       • Type I error
       • Type II error
• Testing a Claim: Companies often make claims
  about products. For example, a frozen yogurt
  company may claim that its product has no more
  than 90 calories per cup. This claim is about a
  parameter – i.e., the population mean number of
  calories per cup (μ).
• The claim is tested is by taking a sample - say, 100
  cups - and determining the sample mean. If the
  sample mean is 90 calories or less we have no
  evidence that the company has lied. Even if the
  sample mean is greater than 90 calories, it is
  possible the company is still telling the truth
  (sampling error). However, at some point –
  perhaps, say, a sample average of 500 calories per
  cup – it will be clear that the company has not been
  completely truthful about its product.
                                                         80
• A hypothesis is made about the value of a
  parameter, but the only facts available to estimate
  the true parameter are those provided by the
  sample. If the statistic differs (and of course it will)
  from the hypothesis stated about the parameter, a
  decision must be made as to whether or not this
  difference is significant. If it is, the hypothesis is
  rejected. If not, it cannot be rejected.
• H0: The null hypothesis. This contains the
  hypothesized parameter value which will be
  compared with the sample value.
• H1: The alternative hypothesis. This will be
  “accepted” only if H0 is rejected.
   Technically speaking, we never accept H0 What we actually say is that we do
   not have the evidence to reject it.

                                                                                 81
• Two types of errors may occur: α (alpha) and β
  (beta). The α error is often referred to as a Type
  I error and β error as a Type II error.
 – You are guilty of an alpha error if you reject H0 when it
   really is true.
 – You commit a beta error if you “accept” H0 when it is
   false.




                                                               82
• This alpha error is related to the (1- α) we just
  learned about when constructing confidence
  intervals. We will soon see that an α error of .05
  in testing a hypothesis (two-tail test) is
  equivalent to a confidence of 95% in
  constructing a two-sided interval estimator.


                         α/2                   α/2


                               -Zα/2         Zα/2




                                                       83
TRADEOFF!
•There is a tradeoff between the alpha and beta errors.
We cannot simply reduce both types of error. As one
goes down, the other rises.
•As we lower the α error, the β error goes up: reducing
the error of rejecting H0 (the error of rejection) increases
the error of “Accepting” H0 when it is false (the error of
acceptance).
•This is similar (in fact exactly the same) to the problem
we had earlier with confidence intervals. Ideally, we
would love a very narrow interval, with a lot of
confidence. But, practically, we can never have both:
there is a tradeoff.                                           84
• Our legal system understands this tradeoff very well.
  – If we make it extremely difficult to convict criminals
    because we do not want to incarcerate any innocent
    people we will probably have a legal system in which
    no one gets convicted.
  – On the other hand, if we make it very easy to convict,
    then we will have a legal system in which many
    innocent people end up behind bars.
  – This is why our legal system does not require a guilty
    verdict to be “beyond a shadow of a doubt” (i.e.,
    complete certainty) but “beyond reasonable doubt.”

                                                             85
• Quality Control.
 – A company purchases chips for its smart phones, in
   batches of 50,000. The company is willing to live with
   a few defects per 50,000 chips. How many defects?
 – If the firm randomly samples 100 chips from each
   batch of 50,000 and rejects the entire shipment if there
   are ANY defects, it may end up rejecting too many
   shipments (error of rejection). If the firm is too liberal
   in what it accepts and assumes everything is
   “sampling error,” it is likely to make the error of
   acceptance.
 – This is why government and industry generally work
   with an alpha error of .05
                                                                86
1.Formulate H0 and H1. H0 is the null hypothesis, a hypothesis about the value
of a parameter, and H1 is an alternative hypothesis.
  –   e.g., H0: µ=12.7 years;   H1: µ≠12.7 years
2.Specify the level of significance (α) to be used. This level of significance tells
you the probability of rejecting H0 when it is, in fact, true. (Normally,
significance level of 0.05 or 0.01 are used)
3.Select the test statistic: e.g., Z, t, F, etc. So far, we have been using the Z
distribution. We will be learning about the t-distribution (used for small
samples) later on.
4.Establish the critical value or values of the test statistic needed to reject H0.
DRAW A PICTURE!
5.Determine the actual value (computed value) of the test statistic.
6.Make a decision: Reject H0 or Do Not Reject H0.




                                                                                       87
•When we Formulate H0 and H1, we have to decide
whether to use a one-tail or two-tail test.
•With a “two-tail” hypothesis test, α is split into two
and put in both tails. H1 then includes two
possibilities: μ = # OR μ ≠ #. This is why the
region of rejection is divided into two tails. Note
that the region of rejection always corresponds to
H1.
• With a “one-tail” hypothesis test, the α is entirely
in one of the tails.

                                  Hypothesis Testing      88
•For example, if the company claims that a certain
product has exactly 1 mg of aspirin, that would result in
a two-tail test. Note words like “exactly” suggest two tail
tests. There are problems with too much aspirin and too
little aspirin in a drug.
•On the other hand, if a firm claims that a box of its
raisin bran cereal contains at least 100 raisins, a one-tail
test has to be used. If the sample mean is more than
100, everything is ok. The problems arise only if the
sample mean is less than 100. The question will be
whether we are looking at sampling error or perhaps the
company is lying and the true (population) mean is less
than 100 raisins.

                                                               89
•A company claims that its soda vending machines deliver exactly 8 ounces of
soda. Clearly, You do not want the vending machines to deliver too much or
too little soda. How would you formulate this?

Answer:
H0: µ = 8 ounces
H1: µ ≠ 8 ounces
If you are testing at α=.01, The .01 is split into two: .005 in the left tail and .
005 in the right tail The critical values are ±2.575




                                     .005                                .005


                                            -2.575                     2.575




                                                                                      90
•A company claims that its bolts have a circumference
of exactly 12.50 inches. (If the bolts are too wide or
narrow, they will not fit properly):
Answer:
H0: µ = 12.50 inches
H1: µ ≠ 12.50 inches
•A company claims that a slice of its bread has exactly 2
grams of fiber. Formulate this:
Answer:
H0: µ = 2 grams
H1: µ ≠ 2 grams


                                                            91
•A company claims that its batteries have an average life of at least 500
hours. How would you formulate this?

Answer:
H0: µ ≧ 500 hours
H1: µ < 500 hours


If you are testing at an α = .05, The entire .05 is in the left tail (hint: H1 points to
where the rejection region should be.) The critical value is -1.645.




                                                                                           92
A company claims that its overpriced, bottled spring water has no more than 1
mcg of benzene (poison). How would you formulate this:
Answer:
H0: µ ≦ 1 mcg. benzene
H1: µ > 1 mcg. benzene
If you are testing at an α = .05, The entire .05 is in the right tail (hint: H1 points
to where the rejection region should be.) The critical value is +1.645.




                                                        .05


                                                     1.645




                                                                                         93
A pharmaceutical company claims that each of its pills contains exactly 20.00
milligrams of Cumidin (a blood thinner). You sample 64 pills and find that the
sample mean X̅ =20.50 mg and s = .80 mg. Should the company’s claim be
rejected? Test at α = 0.05.
•Formulate the hypotheses
        H0: µ =20.00 mg
        H1: µ ≠ 20.00 mg
•Choose the test statistic and find the critical values; draw region of rejection
Test statistic: Z
At α = 0.05, the critical values are ±1.96.



•Use the data to get the calculated value of the test statistic
Z=            =    =5        [ .80/√.64 = .10 This is the standard error of the mean. ]




•Come to a Conclusion: Reject H0 or Do Not Reject H0
         The computed Z value of 5 is deep in the region of rejection.
         Thus, Reject H0 at p < .05                                                       94
• Suppose we took the above data, ignored the hypothesis, and
  constructed a 95% confidence interval estimator.




20.50 ± 1.96(.10)
95%, CIE: 20.304 mg ←→ 20.696 mg
• We note that 20.00 mg is not in this interval.
• As you can see, hypothesis testing and CIE are virtually the
  same exercise; they are merely two sides of the same coin.
  Both rely on the sample evidence.
• If a claim is made about a parameter, do a hypothesis test. If no
  claim is made and a company wants to use sample evidence to
  estimate a parameter (perhaps to determine what claims may be
  made in the future about a parameter), construct a confidence
  interval estimator.                                                 95
• A company claims that its LED bulbs will last at least
  8,000 hours. You sample 100 bulbs and find that X̅
  =7,800 hours and s=800 hours. Should the
  company’s claim be rejected? Test at α = 0.05.

• H0: µ ≧ 8,000 hours
  H1: µ < 8,000 hours                     5%



                                               -1.645




• Z = 7,800 – 8,000 / (800/√100) = -200/80 = -2.50
    • [800/√100 = 80, the standard error of the mean]
• The computed Z value of -2.50 is in the region of
  rejection. Thus, reject H0 at p < .05
  – Note: When testing a hypothesis, we often have to perform a one-tail test if the
    claim requires it. However, we will always use only two-sided confidence interval
    estimators when using sample statistics to estimate population parameters.
                                                                                        96
• In estimating µ based on sample statistics,
  how large a sample do we need for the
  level of precision we want?
 – To determine the sample size we need, we
   must know the (1) desired precision and (2) σ.
                      e=
                     Pr ecision
                        
                         
                 X ± Zα σ / n
• e, the half-width of the confidence interval
  estimator is the precision with which we
  are estimating. e is also called sampling
  error.                                            97
…continued




We use e to solve for n:
                  Zσ                 Zσ
             e=               n=
                   n                  e
      If               then
                            Z 2σ 2
                         n=
                              e2


              and so
                                          98
1.96 2 20 2
n=
      10 2




                 99
• Similarly, taking e (precision) from formula
  for the half-width of a confidence interval
  estimator for P:
                Z P (1 − P )
                  2


                    e2
• Q: If we are trying to estimate the
  population proportion, P, what do we use
  for P in this formula?

                                                 100
Suppose a pollster wants a maximum error
of
e = .01 with 95% confidence.
We assume that variance is the highest
possible, so we use P=.5. This is the way
we ensure that sampling error will be within
±.01 of the true population Proportion.
Then,1.96 .5(1 − .5)
        2

n=          2
          .01        = 9,604
 That is a VERY large sample.
                                               101
…continued




Let’s try that again with e = .03.

      1.96 2.5(1 − .5)
n=         .032          = 1,067

This is the sample size that most pollsters
work with.

                                              102

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Biostatistics iii

  • 1. Descriptive Methods for Categorical Data Mahmoud Alhussmi, D.Sc., PhD
  • 2. Topics • Proportions • Rates – Change rates – Measures of morbidity and mortality – Standardization of rates • Ratios – Relative risk – Odds Ratio March 28, 2013 2
  • 3. Categorical Data Data that can be classified as belonging to a distinct number of categories. – Binary – data can be classified into one of 2 possible categories (yes/no, positive/negative) – Ordinal – data that can be classified into categories that have a natural ordering (i.e.. Levels of pain: none, moderate, intense) – Nominal- data can be classified into >2 categories (i.e.. Race: Arab, African, and other) March 28, 2013 3
  • 4. Data examples • What type of data would result from these questions? – How old are you? ____________ – How old are you? • A. under 18 • B. 19-35 • C. 36-49 • D. over 50 March 28, 2013 4
  • 5. Proportions • Numbers by themselves may be misleading: they are on different scales and need to be reduced to a standard basis in order to compare them. • We most frequently use proportions: that is, the fraction of items that satisfy some property, such as having a disease or being exposed to a dangerous chemical. • "Proportions" are the same thing as fractions or percentages. In every case you need to know what you are taking a proportion of: that is, what is the DENOMINATOR in the proportion. x x p= n percent (100) = (100) n March 28, 2013 5
  • 6. Proportions and Probabilities • We often interpret proportions as probabilities. If the proportion with a disease is 1/10 then we also say that the probability of getting the disease is 1/10, or 1 in 10. • Proportions are usually quoted for samples - probabilities are almost always quoted for populations. March 28, 2013 6
  • 7. Workers Example Smoking Workers Cases Controls No Yes 11 35 No 50 203 Yes Yes 84 45 No 313 270 • For the cases: – Proportion of exposure=84/397=0.212 or 21.2% • For the controls: – Proportion of exposure=45/315=0.143 or 14.3% March 28, 2013 7
  • 8. Prevalence Disease Prevalence = the proportion of people with a given disease at a given time. disease prevalence = Number of diseased persons at a given time Total number of persons examined at that time Prevalence is usually quoted as per 100,000 people so the above proportion should be multiplied by 100,000. March 28, 2013 8
  • 9. Interpretation Cases (old +new) At time t Pr evalence = Total Problem of exposure, consequently Not comparable measurement Old = duration of the disease New = speed of the disease March 28, 2013 9
  • 10. Screening Tests • Through screening tests people are classified as healthy or as falling into one or more disease categories. • These tests are not 100% accurate and therefore misclassification is unavoidable. • There are 2 proportions that are used to evaluate these types of diagnostic procedures. March 28, 2013 10
  • 11. Screening Tests General Population Diseased Positive Test Results March 28, 2013 11
  • 12. Sensitivity and Specificity • Sensitivity and specificity are terms used to describe the effectiveness of screening tests. They describe how good a test is in two ways - finding false positives and finding false negatives • Sensitivity is the Proportion of diseased who screen positive for the disease • Specificity is the Proportion of healthy who screen healthy March 28, 2013 12
  • 13. Sensitivity and Specificity Condition Present Condition Absent …………………………………………………………………………………………… Test Positive True Positive (TP) False Positive (FP) Test Negative False Negative (FN) True Negative (TN) ……………………………………………………………………………………………  Test Sensitivity (Sn) is defined as the probability that the test is positive when given to a group of patients who have the disease.  Sn= (TP/(TP+FN))x100.  It can be viewed as, 1-the false negative rate.  The Specificity (Sp) of a screening test is defined as the probability that the test will be negative among patients who do not have the disease.  Sp = (TN/(TN+FP))X100.  It can be understood as 1-the false positive rate.
  • 14. Positive & Negative Predictive Values • The positive predictive value (PPV) of a test is the probability that a patient who tested positive for the disease actually has the disease. PPV = (TP/(TP+FP))X 100. • The negative predictive value (NPV) of a test is the probability that a patent who tested negative for a disease will not have the disease. NPV = (TN/(TN+FN))X100.
  • 15. The Efficiency • The efficiency (EFF) of a test is the probability that the test result and the diagnosis agree. • It is calculated as: EFF = ((TP+TN)/(TP+TN+FP+FN)) X 100
  • 16. Example • A cytological test was undertaken to screen women for cervical cancer. Test Positive Test Negative Total Actually Positive )TP (154 )FP (225 379 Actually Negative )FN (362 )TN (23,362 23,724 )TP+FN (516 )FP+TN(23587 • Sensitivity =? • Specificity = ? March 28, 2013 16
  • 17. Displaying Proportions • Types of charts that can be used: – Histogram – Pie Chart – Line Graph • BEWARE of the type of display you use – some charts are better at displaying certain types of data than others. March 28, 2013 17
  • 18. Displaying Proportions Percent of Children LivingSudan Distribution of Race in in African 70% 80% Crack/cocaine Households 80 Arab 18% 70% 70 Percent of Children Living in 60% Crack/cocaine Households Mixed 8% 60 50% 50 40% 40 30% Other 4% 30 20% 20 10% 10 0% 0 Black White American Other Indian African Arab foreigners others March 28, 2013 18
  • 19. Displaying Proportions Distribution of Race in Sudan Distribution of Race in Sudan others foreigners African Arab Arab foreigners others African 0 20 40 60 80 March 28, 2013 19
  • 20. Displaying Proportions Cause of Death of Deaths# Proportion of Deaths Heart Disease 12,278 0.38 Cancer 6,448 0.20 Cerebrovascular Disease 3,958 0.12 Accidents 1,814 0.06 Other 8,088 0.25 Causes of Death Heart Disease Cancer Cerebrovascular disease Accidents Other March 28, 2013 20
  • 21. Rates • The term rate is often used interchangeably with the term proportion although sometimes it refers to a quantity of a very different nature. • Types of rates we will cover: – Incidence rate – Change rates – Death rate – Follow-up death rate March 28, 2013 21
  • 22. Calculation of Incidence Rate March 28, 2013 22
  • 23. Definition The incidence rate is the production of new cases in a population. It measure the number of cases per unit of time, i.e. It measure the average of the speed of the apparition of new cases in given population. There are three measures of incidence: 1. Incidence rate (=news cases/time of participation) 2. Instantaneous incidence 3. Accumulate incidence March 28, 2013 23
  • 24. In incidence study we are interested on the occurrence of event (disease) over the time,. Such study deals with follow up of each subject and the moment (ti) that each event (disease) occurs. The problem of incidence data is that the existence of observation incomplete, subjects are still not affected at the moment of analysis. March 28, 2013 24
  • 25. To study incidence rate we should know for each subject the following information: Date of origin Is the date that an individual enter in the study. Date of last information Is the recent date that we receive information about the status of the subject. If the subject is affected, so the date of last information is date of getting the disease. March 28, 2013 25
  • 26. Duration of follow up Is the delay between the date of origin and the date of last information Date of point Is the date that we decide to stop collecting information about subjects. Loss of view A subject that we do not know his status at the date of point is called loss of view. March 28, 2013 26
  • 27. Time of participation t i Time of participation for loss of view Time of participation for loss of view Time not consider t1, t2, t3, …., time 0 ? Date of last Date of point information Time of participation for affected subject Time of participation March 28, 2013 27
  • 28. Example The following example follow 30 individuals between 1982 to 1988 for the disease D. of individual# Date of origin Date of disease Date of last Time of information participation 1 11-82 1-84 12-88 14 2 11-82 10-87 12-88 59 3 6-82 2-86 6-87 44 4 11-82 11-86 12-88 36 5 11-82 3-85 12-88 28 6 11-82 1-88 12-88 62 7 6-83 3-84 6-85 9 8 1-83 12-86 12-87 47 9 1-84 1-87 12-88 36 10 11-82 10-85 12-88 35 11 12-82 8-86 12-88 44 12 1-83 6-83 7-85 5 13 6-83 11-87 7-88 53 14 11-82 8-84 2-87 21 March 28, 2013 28
  • 29. 15 1-83 3-86 12-88 38 16 6-83 5-87 12-88 47 17 5-83 1-84 12-88 8 18 11-83 6-88 12-88 55 19 6-83 5-86 12-87 35 20 3-83 2-88 12-88 59 21 4-83 12-88 68 22 11-82 11-85 36 23 1-83 12-88 71 24 6-83 12-88 66 25 11-82 11-86 48 26 6-82 12-88 78 27 12-83 12-88 60 28 11-82 12-88 73 29 6-84 12-88 54 30 1-83 1-84 12 Time of participation = 3001 Number of cases = 20 Rate of incidence = 20/3001 = 0.015 Or 1.5 case per 100 individuals. March 28, 2013 29
  • 30. Sometimes it is difficult to know the exact date of origin of the case or even the duration of follow up, and this is always take place when the population under study is open . In this case what we know very well : 1.Number of new cases, but not the time exact of participation = m 2.Total number of population from the begin to end of the study = N The calculation using the same method as before will be impossible, because many information was missed. The assumption over which we are going to build our hypothesis is that all the cases enter or quiet the study are distributed uniformly during the period of follow up, i.e. the date of follow up for each of these subject will be the half average of the period of the follow up of the study. The calculation will be as follow: Item Number time of participation Not diseases N N*Δt Enter along the study Ne Ne*Δt/2 Quiet along the study Ns Ns*Δt/2 Disease m m*Δt/2 m IR = ∆t ( 2 N + N e + N s + m) 2 March 28, 2013 30
  • 31. Change Rates • These types of rates are used to describe changes after a certain period of time. new value - old value change rate (%) = X 100 old value • Example: A total of 35,238 new AIDS cases were reported in 1989 compared to 32,196 reported during 1988. – The change rate for new AIDS cases:  35,238 − 32 ,196   100 = 9.4%  32 ,196  March 28, 2013 31
  • 32. Measures of Morbidity and Mortality # deaths in a calendar year crude death rate = the population on that year # of people that developed the disease over a defined period of time (ie. a year) incidence rate = # of people at risk who were followed for the defined period of time (ie. a year) number of deaths follow - up death rate = total person - years March 28, 2013 32
  • 33. Crude Death Rate • Example: The 1980 population in California was 23,000,000 (as estimated on 1 July) and there were 190,237 deaths during that year. – Crude death rate =(190,237/23,000,000)*1,000 = 8.3 deaths per 1,000 per year March 28, 2013 33
  • 34. Displaying Proportions over Time Death Rate per 100,000 Female Death Rates (1984-1987) 1984 793 815 810 805 1985 807 800 795 Death Rate per 100,000 790 1986 809 785 780 1984 1985 1986 1987 1987 813 March 28, 2013 34
  • 35. Standardization of Rates • Crude rates are used to describe a population but comparisons of crude rates are often invalid because the populations may be different w.r.t important characteristics (ie. age, gender, race). • To account for these differences adjusted rates are used in the comparison.  # deaths expected  adjusted rate =   # in standard population  X100,000    March 28, 2013 35
  • 36. Group A Group B no. deaths/ no. deaths/ Age group deaths persons 100000 deaths persons 100000 0-4 162 40,000 405.0 2,049 546,000 375.3 5-19 107 128,000 83.6 1,195 1,982,000 60.3 20-44 449 172,000 261.0 5,097 2,676,000 190.5 45-64 451 58,000 777.6 19,904 1,807,000 1101.5 +65 444 9,000 4933.3 63,505 1,444,000 4397.9 Totals 1613 407000 396.3 91750 8455000 1085.2 March 28, 2013 36
  • 37. :Using the X population for 1970 as a standard we get Group A Group B Age Age spec. Exp Age spec. Exp group Standard rate deaths rate deaths 0-4 84,416 405.0 342 375.3 317 5-19 294,353 83.6 246 60.3 177 20-44 316,744 261.0 827 190.5 603 45-64 205,745 777.6 1600 1101.5 2266 +65 98,742 4933.3 4871 4397.9 4343 Totals 1,000,000 7886 7706 Expected deaths for Group A for age group 65+ = (98,742) (4933.3)/100,000 = 4871 Age adjusted rate for = Group A 788.6 100,000*(7886/1,000,000)= Age adjusted rate for = Group B 770.6 100,000*(7706/1,000,000)= March 28, 2013 37
  • 38. Relative Risk • Relative risks are the ratio of risks for two different populations (ratio=a/b). disease incidence in group 1 Relative Risk = disease incidence in group 2 • If the risk (or proportion) of having the outcome is 1/10 in one population and 2/10 in a second population, then the relative risk is: (2/10) / (1/10) = 2.0 • A relative risk >1 indicates increased risk for the group in the numerator and a relative risk <1 indicates decreased risk for the group in the numerator. March 28, 2013 38
  • 39. Odd’s Ratio and Relative Risk • Odds ratios are better to use in case- control studies (cases and controls are selected and level of exposure is determined retrospectively) • Relative risks are better for cohort studies (exposed and unexposed subjects are chosen and are followed to determine disease status - prospective) March 28, 2013 39
  • 40. Odd’s Ratio and Relative Risk • When we have a two-way classification of exposure and disease we can approximate the relative risk by the odds ratio Disease Yes No Yes A B A+B Exposure No C D C+D • Relative Risk=A/(A+B) divided by C/(C+D) • Odd’s Ratio= A/B divided by C/D = AD/BC March 28, 2013 40
  • 41. Relationship Between the Two Measures A C RR = ÷ A+B C+D A(C + D) = C(A + B) if the number of subjects classified as disease positive is small compared to those classified as disease negative, then : C+D ≅ D A+B≅B Therefore the relative risk can be approximated by : A A*D RR ≅ = B B*C C D March 28, 2013 41
  • 42. Case Control Study Example • Disease: Pancreatic Cancer • Exposure: Cigarette Smoking Disease Yes No Exposure Yes 38 81 119 No 2 56 58 March 28, 2013 42
  • 43. Example Continued • Relative risk for exposed vs. non-exposed – Numerator- proportion of exposed people that have the disease – Denominator-proportion of non-exposed that have the disease – Relative Risk= (38/119)/(2/58)=9.26 March 28, 2013 43
  • 44. Example Continued • Odd’s Ratio for exposed vs. non-exposed – Numerator- ratio of diseased vs. non- diseased in the exposed group – Denominator- ratio of diseased vs. non- diseased in the non-exposed group – Odd’s Ratio= (38/81)/(2/56)=(38*56)/(2*81) =13.14 March 28, 2013 44
  • 45. Relative Risk • Relative risk – the chance that a member of a group receiving some exposure will develop a disease relative to the chance that a member of an unexposed group will develop the same disease. P(disease | exposed) RR = P(disease | unexposed) • Recall: a RR of 1.0 indicates that the probabilities of disease in the exposed and unexposed groups are identical – an association between exposure and disease does not exist. March 28, 2013 45
  • 46. Relative Risk • When we have a two-way classification of exposure and disease we can calculate the relative risk Disease Yes No Yes A B A+B Exposure No C D C+D March 28, 2013 46
  • 47. Case Control Study Example • Disease: Pancreatic Cancer • Exposure: Cigarette Smoking Disease Yes No Exposure Yes 38 81 119 No 2 56 58 March 28, 2013 47
  • 48. Data Interpretation • Consideration: 1. Accuracy 1. critical view of the data 2. investigating evidence of the results 3. consider other studies’ results 4. peripheral data analysis 5. conduct power analysis: type I & type II True False True Correct Type-II False Type -I Correct
  • 49. Types of Errors If You…… When the Null Then You Hypothesis is… Have……. Reject the null True (there really Made a Type I hypothesis are no difference) Error Reject the null False (there really ☻ hypothesis are difference) Accept the null False (there really Made Type II hypothesis are difference) Error Accept the null True (there really ☻ hypothesis are no difference)
  • 50. • alpha : the level of significance used for establishing type-I error • β : the probability of type-II error • 1 – β : is the probability of obtaining significance results ( power) • Effect size: how much we can say that the intervention made a significance difference
  • 51. 2. Meaning of the results - translation of the results and make it understandable 3. Importance: - translation of the significant findings into practical findings 4. Generalizability: - how can we make the findings useful for all the population 5. Implication: - what have we learned related to what has been used during study
  • 52. POWER--Uses and Misuses • Sources – Cohen Statistical Power Analysis for the Behavioral Sciences (gold standard for power) – Kraemer & Thieman How Many Subjects? (also a good review)
  • 53. Needed Parameters • Alpha--chance of a Type I error • Beta--chance of a Type II error • Power = 1 - beta • Effect size--difference between groups or amount of variance explained or how much relationship there is between the DV and the IVs
  • 54. ?Remember this in English • Type I error is when you say there is a difference or relationship and there is not • Type II error is when you say there is no difference or relationship and there really is
  • 55. ?What Affects Power • Size of the difference in means or amount of variance explained (ES) • alpha • Unexplained variance • N
  • 56. ?Which is more important • Type I error more important if possibility of harm or lethal effect • Type II error more important in relatively unexplored areas of research • In some studies, Type I and Type II errors may be equally important
  • 57. How to Increase Power 1. Increase the n 2. Decrease the unexplained variance--control by design or statistics (e.g. ANCOVA) 3. Increase alpha (controversial) 4. Use a one tailed test (directional hypothesis)--puts the zone of rejection all in one tail; same effect as increasing alpha 5. Use parametric statistics as long as you meet the assumptions. If not, parametric statistics are LESS powerful 6. Decrease measurement error (decrease unexplained variance)--use more reliable instruments, standardize measurement protocol, frequent calibration of physiologic instruments, improve inter-rater reliability
  • 58. ?What is good power By tradition, “good” power is 80% The correct answer is it depends on the nature of the phenomenon and which kind of error is most important in your study. This is a theoretical argument that you have to make. Using convention (alpha = .05 and power = .80, beta = .20) you are saying that Type I error is _________ as serious as a Type II error
  • 59. Effect Size How large an effect do I expect exists in the population if the null is false? OR How much of a difference do I want to be able to detect? The larger the effect, the fewer the cases needed to see it. (The difference is so big you can trip on it.)
  • 60. The World According to Power Kraemer & Thiemann • The more stringent the significance level, the greater the necessary sample size. More subjects are needed for a 1% level than a 5% level • Two tailed tests require larger sample sizes than one tailed tests. Assessing two directions at the same time requires a greater investment. • The smaller the effect size, the larger the necessary sample size. Subtle effects require greater efforts. • The larger the power required, the larger the necessary sample size. Greater protection from failure requires greater effort. • The smaller the sample size, the smaller the power, ie the greater the chance of failure
  • 61. The World According to Power Kraemer & Thiemann • If one proposed to go with a sample size of 20 or fewer, you have to be willing to have a high risk of failure or a huge effect size • To achieve 99% power for a effect size of . 01, you need > 150,000 subjects
  • 62. Test Yourself Keeping the other parameters the same: • As ES decreases, needed n ____ • As alpha decreases, needed n ____ • Higher power requires _____ n
  • 63. Power for each test • You do a power analysis for each statistic you are going to use. • Choose the sample size based on the highest number of subjects from the power analysis. • Use the most conservative power analysis--guarantees you the most subjects
  • 64. ?What about multiple time points • More time points requires fewer subjects since more is known about the subjects from prior time points as compared to a cross sectional study • In other words, less variance is unexplained since you have baseline information • How many fewer? It depends
  • 65. Power analysis and secondary analysis If you have a set sample size, your power analysis then works backward. You set the n, alpha and ES and determine the power given the first three parameters.
  • 66. Determining ES If you want to determine effect size from a completed study, you have the n, alpha and power and can work backwards to determine the ES. Especially important in relatively unexplored areas
  • 67. Power and MR • ES is the amount of explained variance expected since there may not be group differences, based on past research • Increasing the number of independent variables _______ sample size needed to achieve adequate power.
  • 68. Sampling Distribution • A sample statistic is often unequal to the value of the corresponding population parameter because of sampling error. • Sampling error reflects the tendency for statistics to fluctuate from one sample to another. • The amount of sampling error is the difference between the obtained sample value and the population parameter. • Inferential statistics allow researchers to estimate how close to the population value the calculated statistics is likely to be. • The concept of sampling, which are actually probability distributions, is central to estimates of sampling error.
  • 69. Characteristics of Sampling Distribution • Sampling error= sample mean-population mean. • Every sample size has a different sampling distribution of the mean. • Sampling distributions are theoretical, because in practice, no one draws an infinite number of samples from a population. • Their characteristics can be modeled mathematically and have determined by a formulation known as the central limit theorem. • This theorem stipulates that the mean of the sampling distribution is identical to the population mean. • The average sampling error-the mean of the (mean-μ)s- would always equal zero.
  • 70. Standard Error of the Mean • The standard deviation of a sampling distribution of the mean has a special name: the standard error of the mean (SEM). • The smaller the SEM, the more accurate are the sample means as estimates of the population value.
  • 71. • Estimation • Hypothesis Testing Both activities use sample statistics (for ̅ example, X) to make inferences about a population parameter (μ). 71
  • 72. • Why don’t we just use a single number (a point estimate) like, say, X̅ to estimate a population parameter, μ? • The problem with using a single point (or value) is that it will very probably be wrong. In fact, with a continuous random variable, the probability that the variable is equal to a ̅ particular value is zero. So, P(X=μ) = 0. • This is why we use an interval estimator. • We can examine the probability that the interval includes the population parameter. 72
  • 73. Types of Statistical Inference • Parameter estimation: – It is used to estimate a population value, such as a mean, relative risk index or a mean difference between two groups. – Estimation can take two forms: • Point estimation: involves calculating a single statistic to estimate the parameter. E.g. mean and median. – Disadvantages: they offer no context for interpreting their accuracy and a point estimate gives no information regarding the probability that it is correct or close to the population value. • Interval estimation: is to estimate a range of values that has a high probability of containing the population value .
  • 74. • How wide should the interval be? That depends upon how much confidence you want in the estimate. • For instance, say you wanted a confidence interval estimator for the mean income of a college graduate: You might have That the mean income is between 100% $∞and $0 confidence 95% and $41,000 $35,000 confidence 90% and $40,000 $36,000 confidence 80% and $38,500 $37,500 confidence • The wider the interval, the greater the confidence … … you will have in it as containing the true population confidence 0%(a point estimate )$38,000 parameter μ. 74
  • 75. Interval Estimation • For example, it is more likely the population height mean lies between 165-175cm. • Interval estimation involves constructing a confidence interval (CI) around the point estimate. • The upper and lower limits of the CI are called confidence limits. • A CI around a sample mean communicates a range of values for the population value, and the probability of being right. That is, the estimate is made with a certain degree of confidence of capturing the parameter.
  • 76. Confidence Intervals around a Mean • 95% CI = (mean + (1.96 x SEM) • This statement indicates that we can be 95% confident that the population mean lies between the confident limits , and that these limits are equal to 1.96 times the true standard error, above and below the sample mean. • E.g. if the mean = 61 inches, and SEM = 1, What is 95% CI. – Solution: 95% CI = (61 + (1.96 X 1)) 95% CI = (61 + 1.96) 95% CI = 59.04 < μ < 62.96 • E.g. if the mean = 61 inches, and SEM = 1, What is 99% CI. – Solution: 99% CI = (61 + (2.58 X 1)) 99% CI = (61 + 2.58) 99% CI = 58.42 < μ < 63.58
  • 77. Confidence Intervals and the t distribution • When sample size is small then we cannot use confidence intervals around the mean, instead, we measure confidence intervals by the t-distribution. • t-distribution is similar to a normal distribution in a standard form. • The exact shape of the t-distribution is influenced by the number cases in the sample. • Statisticians have developed tables for the area under the t-distribution for different sample size and probability levels. • To use this table, we must enter at the appropriate row based on the number of degrees of freedom.
  • 78. Confidence Intervals and the t distribution • 95% CI = (mean + (t x SEM) – Where mean = the sample mean T = tables t value at 95% CI for df = N-1 SEM = the calculated SEM for the sample data • E.g. SEM = 1, mean = 61, N = 25, df = 25-1, t for the 95% CI with 24 df is 2.06 – Solution: 95% CI = (61 + (2.06 X 1)) 95% CI = (61 + 2.06) 95% CI = 58.95 < μ < 63.06 To compute CIs around a mean with SPSS: Analyze------descriptive stat----explore then click on the statistics pushbutton.
  • 79. Types of Statistical Inference • Hypothesis testing: – Hypothesis testing is a second approach to inferential statistics. – Hypothesis testing involves using sampling distributions and the laws of probability to make an objective decision about whether to accept or reject the null hypothesis. – The sample may deviate from the defined population’s true nature by certain amount. – This deviation is called sampling error. – Drawing the wrong conclusion is called an error of inference. – There are two types of errors of inference defined in terms of the null hypothesis: • Type I error • Type II error
  • 80. • Testing a Claim: Companies often make claims about products. For example, a frozen yogurt company may claim that its product has no more than 90 calories per cup. This claim is about a parameter – i.e., the population mean number of calories per cup (μ). • The claim is tested is by taking a sample - say, 100 cups - and determining the sample mean. If the sample mean is 90 calories or less we have no evidence that the company has lied. Even if the sample mean is greater than 90 calories, it is possible the company is still telling the truth (sampling error). However, at some point – perhaps, say, a sample average of 500 calories per cup – it will be clear that the company has not been completely truthful about its product. 80
  • 81. • A hypothesis is made about the value of a parameter, but the only facts available to estimate the true parameter are those provided by the sample. If the statistic differs (and of course it will) from the hypothesis stated about the parameter, a decision must be made as to whether or not this difference is significant. If it is, the hypothesis is rejected. If not, it cannot be rejected. • H0: The null hypothesis. This contains the hypothesized parameter value which will be compared with the sample value. • H1: The alternative hypothesis. This will be “accepted” only if H0 is rejected. Technically speaking, we never accept H0 What we actually say is that we do not have the evidence to reject it. 81
  • 82. • Two types of errors may occur: α (alpha) and β (beta). The α error is often referred to as a Type I error and β error as a Type II error. – You are guilty of an alpha error if you reject H0 when it really is true. – You commit a beta error if you “accept” H0 when it is false. 82
  • 83. • This alpha error is related to the (1- α) we just learned about when constructing confidence intervals. We will soon see that an α error of .05 in testing a hypothesis (two-tail test) is equivalent to a confidence of 95% in constructing a two-sided interval estimator. α/2 α/2 -Zα/2 Zα/2 83
  • 84. TRADEOFF! •There is a tradeoff between the alpha and beta errors. We cannot simply reduce both types of error. As one goes down, the other rises. •As we lower the α error, the β error goes up: reducing the error of rejecting H0 (the error of rejection) increases the error of “Accepting” H0 when it is false (the error of acceptance). •This is similar (in fact exactly the same) to the problem we had earlier with confidence intervals. Ideally, we would love a very narrow interval, with a lot of confidence. But, practically, we can never have both: there is a tradeoff. 84
  • 85. • Our legal system understands this tradeoff very well. – If we make it extremely difficult to convict criminals because we do not want to incarcerate any innocent people we will probably have a legal system in which no one gets convicted. – On the other hand, if we make it very easy to convict, then we will have a legal system in which many innocent people end up behind bars. – This is why our legal system does not require a guilty verdict to be “beyond a shadow of a doubt” (i.e., complete certainty) but “beyond reasonable doubt.” 85
  • 86. • Quality Control. – A company purchases chips for its smart phones, in batches of 50,000. The company is willing to live with a few defects per 50,000 chips. How many defects? – If the firm randomly samples 100 chips from each batch of 50,000 and rejects the entire shipment if there are ANY defects, it may end up rejecting too many shipments (error of rejection). If the firm is too liberal in what it accepts and assumes everything is “sampling error,” it is likely to make the error of acceptance. – This is why government and industry generally work with an alpha error of .05 86
  • 87. 1.Formulate H0 and H1. H0 is the null hypothesis, a hypothesis about the value of a parameter, and H1 is an alternative hypothesis. – e.g., H0: µ=12.7 years; H1: µ≠12.7 years 2.Specify the level of significance (α) to be used. This level of significance tells you the probability of rejecting H0 when it is, in fact, true. (Normally, significance level of 0.05 or 0.01 are used) 3.Select the test statistic: e.g., Z, t, F, etc. So far, we have been using the Z distribution. We will be learning about the t-distribution (used for small samples) later on. 4.Establish the critical value or values of the test statistic needed to reject H0. DRAW A PICTURE! 5.Determine the actual value (computed value) of the test statistic. 6.Make a decision: Reject H0 or Do Not Reject H0. 87
  • 88. •When we Formulate H0 and H1, we have to decide whether to use a one-tail or two-tail test. •With a “two-tail” hypothesis test, α is split into two and put in both tails. H1 then includes two possibilities: μ = # OR μ ≠ #. This is why the region of rejection is divided into two tails. Note that the region of rejection always corresponds to H1. • With a “one-tail” hypothesis test, the α is entirely in one of the tails. Hypothesis Testing 88
  • 89. •For example, if the company claims that a certain product has exactly 1 mg of aspirin, that would result in a two-tail test. Note words like “exactly” suggest two tail tests. There are problems with too much aspirin and too little aspirin in a drug. •On the other hand, if a firm claims that a box of its raisin bran cereal contains at least 100 raisins, a one-tail test has to be used. If the sample mean is more than 100, everything is ok. The problems arise only if the sample mean is less than 100. The question will be whether we are looking at sampling error or perhaps the company is lying and the true (population) mean is less than 100 raisins. 89
  • 90. •A company claims that its soda vending machines deliver exactly 8 ounces of soda. Clearly, You do not want the vending machines to deliver too much or too little soda. How would you formulate this? Answer: H0: µ = 8 ounces H1: µ ≠ 8 ounces If you are testing at α=.01, The .01 is split into two: .005 in the left tail and . 005 in the right tail The critical values are ±2.575 .005 .005 -2.575 2.575 90
  • 91. •A company claims that its bolts have a circumference of exactly 12.50 inches. (If the bolts are too wide or narrow, they will not fit properly): Answer: H0: µ = 12.50 inches H1: µ ≠ 12.50 inches •A company claims that a slice of its bread has exactly 2 grams of fiber. Formulate this: Answer: H0: µ = 2 grams H1: µ ≠ 2 grams 91
  • 92. •A company claims that its batteries have an average life of at least 500 hours. How would you formulate this? Answer: H0: µ ≧ 500 hours H1: µ < 500 hours If you are testing at an α = .05, The entire .05 is in the left tail (hint: H1 points to where the rejection region should be.) The critical value is -1.645. 92
  • 93. A company claims that its overpriced, bottled spring water has no more than 1 mcg of benzene (poison). How would you formulate this: Answer: H0: µ ≦ 1 mcg. benzene H1: µ > 1 mcg. benzene If you are testing at an α = .05, The entire .05 is in the right tail (hint: H1 points to where the rejection region should be.) The critical value is +1.645. .05 1.645 93
  • 94. A pharmaceutical company claims that each of its pills contains exactly 20.00 milligrams of Cumidin (a blood thinner). You sample 64 pills and find that the sample mean X̅ =20.50 mg and s = .80 mg. Should the company’s claim be rejected? Test at α = 0.05. •Formulate the hypotheses H0: µ =20.00 mg H1: µ ≠ 20.00 mg •Choose the test statistic and find the critical values; draw region of rejection Test statistic: Z At α = 0.05, the critical values are ±1.96. •Use the data to get the calculated value of the test statistic Z= = =5 [ .80/√.64 = .10 This is the standard error of the mean. ] •Come to a Conclusion: Reject H0 or Do Not Reject H0 The computed Z value of 5 is deep in the region of rejection. Thus, Reject H0 at p < .05 94
  • 95. • Suppose we took the above data, ignored the hypothesis, and constructed a 95% confidence interval estimator. 20.50 ± 1.96(.10) 95%, CIE: 20.304 mg ←→ 20.696 mg • We note that 20.00 mg is not in this interval. • As you can see, hypothesis testing and CIE are virtually the same exercise; they are merely two sides of the same coin. Both rely on the sample evidence. • If a claim is made about a parameter, do a hypothesis test. If no claim is made and a company wants to use sample evidence to estimate a parameter (perhaps to determine what claims may be made in the future about a parameter), construct a confidence interval estimator. 95
  • 96. • A company claims that its LED bulbs will last at least 8,000 hours. You sample 100 bulbs and find that X̅ =7,800 hours and s=800 hours. Should the company’s claim be rejected? Test at α = 0.05. • H0: µ ≧ 8,000 hours H1: µ < 8,000 hours 5% -1.645 • Z = 7,800 – 8,000 / (800/√100) = -200/80 = -2.50 • [800/√100 = 80, the standard error of the mean] • The computed Z value of -2.50 is in the region of rejection. Thus, reject H0 at p < .05 – Note: When testing a hypothesis, we often have to perform a one-tail test if the claim requires it. However, we will always use only two-sided confidence interval estimators when using sample statistics to estimate population parameters. 96
  • 97. • In estimating µ based on sample statistics, how large a sample do we need for the level of precision we want? – To determine the sample size we need, we must know the (1) desired precision and (2) σ. e= Pr ecision    X ± Zα σ / n • e, the half-width of the confidence interval estimator is the precision with which we are estimating. e is also called sampling error. 97
  • 98. …continued We use e to solve for n: Zσ Zσ e= n= n e If then Z 2σ 2 n= e2 and so 98
  • 99. 1.96 2 20 2 n= 10 2 99
  • 100. • Similarly, taking e (precision) from formula for the half-width of a confidence interval estimator for P: Z P (1 − P ) 2 e2 • Q: If we are trying to estimate the population proportion, P, what do we use for P in this formula? 100
  • 101. Suppose a pollster wants a maximum error of e = .01 with 95% confidence. We assume that variance is the highest possible, so we use P=.5. This is the way we ensure that sampling error will be within ±.01 of the true population Proportion. Then,1.96 .5(1 − .5) 2 n= 2 .01 = 9,604 That is a VERY large sample. 101
  • 102. …continued Let’s try that again with e = .03. 1.96 2.5(1 − .5) n= .032 = 1,067 This is the sample size that most pollsters work with. 102