USMLE

Epidemiology and Biostatistics

Meta-Analysis: pools data from several studies (greater power), limited by quality/bias of individual studies
Clinical Trial: compares two groups in which one variable is manipulated and its effects measured
Cohort (relative risk): compares group with risk factor to a group without – asks “what will happen?” (prospective). Proves
cause-effect
Case Control (odds ratio): compares group with disease to group without disease – asks “what happened?” (retrospective).
Issues with confounding and inability to prove causation
Case Series: good for rare diseases, describe clinical presentation of certain disease
Cross-Sectional: data from a group to assess disease prevalence at a particular point in time – asks “what is happening?”

Sensitivity (rule out – screening): proportion of people with
disease who test positive: TP / (TP + FN) = 1 - FN. If 100%, DISEASE
then all negative tests are TN. + -
Specificity (rule in – confirmatory): proportion of people TEST + TP FP
without disease who test negative: TN / (TN + FP) = 1 - FP. - FN TN
If 100%, then all positive tests are TP.
PPV: proportion of positive tests that are true positives: TP / (TP + FP). If disease
prevalence is low, then PPV will be low.
NPV: proportion of negative tests that are true negatives. TN / (TN + FN)
Higher specificity -> higher PPV Higher sensitivity -> higher NPV

Odds ratio (case control): odds of having disease in exposed group divided by odds in
unexposed group. (a/b) / (c/d) = (ad) / (bc) DISEASE
Relative risk (cohort): relative probability of getting disease in exposed group versus + -
unexposed. [a/(a+b)] / [c/(c+d)] TEST + a b
Attributable risk: proportion of cases attributable to one risk factor.
[a/(a+b)] - [c/(c+d)] - c d
Absolute risk reduction (ARR): [c/(c+d)] - [a/(a+b)]
NNT = 1 / ARR
Standardized mortality ratio (SMR) = observed # deaths / expected # deaths
Incidence: # of new cases in a unit of time / pop. at risk
Prevalence: total # of cases at a given time / pop. at risk
Prevalence = incidence * dz duration. Prevalence > incidence in chronic dz. Prevalence = incidence in acute dz

Normal distribution: mean = median = mode
Standard deviation: 1 (68%) – 2 (95%) – 3 (99.7%)
SEM = σ / √n
Positive skew (mean > median > mode), negative skew (mean < median < mode)

Reliability (“precision”) – reproducibility of test. Affected by random error
Validity (“accuracy”) – measures trueness of data. Affected by systematic error
Correlation coefficient measures how related two values are: χ2 % or fractions
+1 = perfect positive correlation, -1 = perfect negative correlation, 0 = no correlation
T-test 2 means

H0 (null hypothesis): no relationship between two measurements ANOVA > 2 means
Type I (α) error: reject null when it’s true
Type II (β) error: accept null when it’s false
Power (1-β): probability of rejecting null when it is indeed false (increase sample size to increase power)

Selection bias: nonrandom assignment of subjects
Sampling bias: subjects not representative of population
Recall bias: risk for retrospective studies (pts cannot remember things); knowledge of disorder presence alters recall
Late-look bias: data gathered at inappropriate time
Lead-time bias: early detection confused with increased survival
Confounding bias: a factor is related to both exposure and outcome, but not on the causal pathway
Procedure bias: subjects in different groups not treated the same


Rishi Kumar, MD https://rk.md
HIGH-YIELD PRINCIPLES IN

Public Health Sciences

“Medicine is a science of uncertainty and an art of probability.” ` Epidemiology and

—William Osler Biostatistics 256

“There are two kinds of statistics: the kind you look up and the kind you ` Ethics 265
make up.”
—Rex Stout ` The Well Patient 270

“On a long enough timeline, the survival rate for everyone drops to zero.” ` Healthcare Delivery 270
—Chuck Palahniuk
` Quality and Safety 273
“There are three kinds of lies: lies, damned lies, and statistics.”
—Mark Twain

A heterogenous mix of epidemiology, biostatistics, ethics, law, healthcare

delivery, patient safety, quality improvement, and more falls under the
heading of public health sciences. Biostatistics and epidemiology are the
foundations of evidence-based medicine and are very high yield. Make
sure you can quickly apply biostatistical equations such as sensitivity,
specificity, and predictive values in a problem-solving format. Also, know
how to set up your own 2 × 2 tables. Quality improvement and patient
safety topics were introduced a few years ago on the exam and represent
trends in health system science. Medical ethics questions often require
application of principles. Typically, you are presented with a patient
scenario and then asked how you would respond.

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Observational studies
STUDY TYPE DESIGN MEASURES/EXAMPLE
Cross-sectional study Frequency of disease and frequency of risk- Disease prevalence.
related factors are assessed in the present. Can show risk factor association with disease, but
Asks, “What is happening?” does not establish causality.
Case-control study Compares a group of people with disease to a Odds ratio (OR).
group without disease. Patients with COPD had higher odds of a
Looks to see if odds of prior exposure or risk smoking history than those without COPD.
factor differ by disease state.
Asks, “What happened?”
Cohort study Compares a group with a given exposure or risk Relative risk (RR).
factor to a group without such exposure. Smokers had a higher risk of developing COPD
Looks to see if exposure or risk factor is than nonsmokers.
associated with later development of disease. Cohort = relative risk.
Can be prospective or retrospective.
Crossover study Compares the effect of a series of ≥2 treatments Allows participants to serve as their own
on a participant. controls.
Order in which participants receive treatments
is randomized. Washout period occurs
between each treatment.
Twin concordance Compares the frequency with which both Measures heritability and influence of
study monozygotic twins vs both dizygotic twins environmental factors (“nature vs nurture”).
develop the same disease.
Adoption study Compares siblings raised by biological vs Measures heritability and influence of
adoptive parents. environmental factors.

Clinical trial Experimental study involving humans. Compares therapeutic benefits of ≥2 treatments, or of
treatment and placebo. Study quality improves when study is randomized, controlled, and double-
blinded (ie, neither patient nor doctor knows whether the patient is in the treatment or control
group). Triple-blind refers to the additional blinding of the researchers analyzing the data.
Four phases (“Does the drug SWIM?”).
DRUG TRIALS TYPICAL STUDY SAMPLE PURPOSE
Phase I Small number of either healthy volunteers or “Is it Safe?” Assesses safety, toxicity,
patients with disease of interest. pharmacokinetics, and pharmacodynamics.
Phase II Moderate number of patients with disease of “Does it Work?” Assesses treatment efficacy,
interest. optimal dosing, and adverse effects.
Phase III Large number of patients randomly assigned “Is it as good or better?” Compares the new
either to the treatment under investigation or treatment to the current standard of care (any
to the standard of care (or placebo). Improvement?).
Phase IV Postmarketing surveillance of patients after “Can it stay?” Detects rare or long-term adverse
treatment is approved. effects (eg, black box warnings). Can result in
treatment being withdrawn from Market.
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Evaluation of Sensitivity and specificity are fixed properties Disease

–
diagnostic tests of a test. PPV and NPV vary depending on
disease prevalence in population being tested. PPV
TP FP = TP/(TP + FP)

Test
NPV
– FN TN = TN/(TN + FN)

Sensitivity Specificity Prevalence

TP + FN
= TP/(TP + FN) = TN/(TN + FP) (TP + FN + FP + TN)

Sensitivity (true- Proportion of all people with disease who test = TP / (TP + FN)
positive rate) positive, or the probability that when the = 1 – FN rate
disease is present, the test is positive. SN-N-OUT = highly SeNsitive test, when
Value approaching 100% is desirable for ruling Negative, rules OUT disease
out disease and indicates a low false-negative High sensitivity test used for screening
rate.
Specificity (true- Proportion of all people without disease who = TN / (TN + FP)
negative rate) test negative, or the probability that when the = 1 – FP rate
disease is absent, the test is negative. SP-P-IN = highly SPecific test, when Positive,
Value approaching 100% is desirable for ruling rules IN disease
in disease and indicates a low false-positive High specificity test used for confirmation after a
rate. positive screening test
Positive predictive Probability that a person who has a positive test PPV = TP / (TP + FP)
value result actually has the disease. PPV varies directly with pretest probability
(baseline risk, such as prevalence of disease):
high pretest probability  high PPV
Negative predictive Probability that a person with a negative test NPV = TN / (TN + FN)
value result actually does not have the disease. NPV varies inversely with prevalence or pretest
probability

Possible cutoff values for (+) vs (-) test result

Disease Disease A = 100% sensitivity cutoff value
Number of people

absent present B = practical compromise between specificity and sensitivity

C = 100% specificity cutoff value

TN TP Lowering the cutoff value: ↑ Sensitivity ↑ NPV

↑ ↑ ↑
B A (↑ FP FN) Specificity PPV
↑

FN FP
Raising the cutoff value: ↑ Specificity ↑ PPV
A B C B C (↑ FN FP)
↑ ↑ ↑
Sensitivity NPV
↑

Test results

Likelihood ratio Likelihood that a given test result would be sensitivity TP rate
LR+ = =
expected in a patient with the target disorder 1 – specificity FP rate
compared to the likelihood that the same result
would be expected in a patient without the 1 – sensitivity FN rate
LR– = =
target disorder. specificity TN rate
LR+ > 10 indicates a highly specific test, while
LR– < 0.1 indicates a highly sensitive test.
LRs can be multiplied with pretest odds of
disease to estimate posttest odds.
 a/c ad
Odds ratio = =
b/d bc
a/(a + b)
258
Relative risk =
SEC TION II PUBLIC HEALTH SCIENCES PUBLIC HEALTH
`  SCIENCES—EPIDEMIOLOGY c/(c + d)AND BIOSTATISTICS
a c
Attributable risk =
a+b c+d

Disease or outcome
Quantifying risk Definitions and formulas are based on the classic
2 × 2 or contingency table.

or intervention
Exposure
a b

c d

TERM DEFINITION EXAMPLE FORMULA

Odds ratio Typically used in case-control If in a case-control study, 20/30 lung a/c ad
OR = =
studies. Represents the odds of cancer patients and 5/25 healthy b/d bc
exposure among cases (a/c) vs individuals report smoking, the OR a b
odds of exposure among controls is 8; so the lung cancer patients are 8 20 5
(b/d). times more likely to have a history of c d
10 20
smoking.
Relative risk Typically used in cohort studies. If 5/10 people exposed to radiation are a/(a + b)
RR =
Risk of developing disease in the diagnosed with cancer, and 1/10 people c/(c + d)
exposed group divided by risk in not exposed to radiation are diagnosed a b
the unexposed group. with cancer, the RR is 5; so people 5 5
RR = 1  no association between exposed to radiation have a 5 times c d
1 9
exposure and disease. greater risk of developing cancer.
RR > 1  exposure associated with For rare diseases (low prevalence), OR
 disease occurrence. approximates RR.
RR < 1  exposure associated with
 disease occurrence.
Relative risk The proportion of risk reduction If 2% of patients who receive a flu RRR = 1 − RR
reduction attributable to the intervention as shot develop the flu, while 8% of
compared to a control. unvaccinated patients develop the flu,
then RR = 2/8 = 0.25, and RRR = 0.75.
Attributable The difference in risk between If risk of lung cancer in smokers is 21% a c
AR = −
risk exposed and unexposed groups. and risk in nonsmokers is 1%, then the a+b c+d
attributable risk is 20%. RR − 1
AR% = × 100
RR
Absolute The difference in risk (not the If 8% of people who receive a placebo c a
ARR = −
risk proportion) attributable to the vaccine develop the flu vs 2% of people c+d a+b
reduction intervention as compared to a who receive a flu vaccine, then ARR =
control. 8%–2% = 6% = 0.06.
Number Number of patients who need to NNT = 1/ARR
needed to be treated for 1 patient to benefit.
treat Lower number = better treatment.
Number Number of patients who need to NNH = 1/AR
needed to be exposed to a risk factor for 1
harm patient to be harmed. Higher
number = safer exposure.
Case fatality Percentage of deaths occurring If 4 patients die among 10 cases of deaths
CFR% = × 100
rate among those with disease. meningitis, case fatality rate is 40%. cases
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Incidence vs # of new cases Incidence looks at new cases (incidents).

Incidence = (per unit of time)
prevalence # of people at risk
# of existing cases (at a point in Prevalence looks at all current cases.
Recurrence Prevalence =
Total # of people time)
Incidence
in a population
Prevalence Prevalence = average duration
Incidence rate ×
1 – prevalence of disease
Mortality Cure
Prevalence ≈ incidence for short duration disease Prevalence ∼ pretest probability.
(eg, common cold).  prevalence   PPV and  NPV.
Prevalence > incidence for chronic diseases, due to
large # of existing cases (eg, diabetes).
SITUATION INCIDENCE PREVALENCE

 survival time — 
 mortality — 
Faster recovery time — 
Extensive vaccine administration  
 risk factors  

Precision vs accuracy
Precision (reliability) The consistency and reproducibility of a test. Random error  precision in a test.
The absence of random variation in a test.  precision  standard deviation.
 precision  statistical power (1 − β).
Accuracy (validity) The closeness of test results to the true values. Systematic error  accuracy in a test.
The absence of systematic error or bias in a test.

Accuracy Accuracy
High Low High Low

High High Low Low

Precision Precision
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Receiving operating ROC curve demonstrates how well a diagnostic Ideal test (AUC = 1)
1
characteristic curve test can distinguish between 2 groups (eg, 1)
<
disease vs healthy). Plots the true-positive rate UC
<A
(sensitivity) against the false-positive rate .5
t (0

TP rate (sensitivity)
)
(1 – specificity). 0.5

s
l te
C=

ua
The better performing test will have a higher (AU

Act
e
alu
area under the curve (AUC), with the curve i vev
ict
closer to the upper left corner. pr
ed
No

FP rate (1 – specificity) 1

Bias and study errors

TYPE DEFINITION EXAMPLES STRATEGIES TO REDUCE BIAS
Recruiting participants
Selection bias Nonrandom sampling Berkson bias—cases and/ Randomization
or treatment allocation or controls selected from Ensure the choice of the right
of subjects such that hospitals are less healthy and comparison/reference group
study population is not have different exposures than
representative of target general population
population. Attrition bias—participants lost
Most commonly a sampling to follow up have a different
bias. prognosis than those who
complete the study
Performing study
Recall bias Awareness of disorder alters Patients with disease recall Decrease time from exposure
recall by subjects; common in exposure after learning of to follow-up
retrospective studies similar cases
Measurement bias Information is gathered in a Using a faulty automatic Use objective, standardized,
systemically distorted manner sphygmomanometer to and previously tested methods
measure BP of data collection that are
Hawthorne effect—participants planned ahead of time
change behavior upon Use placebo group
awareness of being observed
Procedure bias Subjects in different groups are Patients in treatment group Blinding (masking) and
not treated the same spend more time in highly use of placebo reduce
specialized hospital units influence of participants and
Observer-expectancy Researcher’s belief in the An observer expecting researchers on procedures and
bias efficacy of a treatment changes treatment group to show signs interpretation of outcomes
the outcome of that treatment of recovery is more likely to as neither are aware of group
(aka, Pygmalion effect) document positive outcomes assignments
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Bias and study errors (continued)

TYPE DEFINITION EXAMPLES STRATEGY TO REDUCE BIAS
Interpreting results
Confounding bias Factor related to both exposure An uncontrolled study shows Multiple/repeated studies
and outcome (but not on an association between Crossover studies (subjects act
causal path) distorts effect drinking coffee and lung as their own controls)
of exposure on outcome (vs cancer. However, coffee Matching (patients with
effect modification, in which drinkers also smoke more, similar characteristics in both
the exposure leads to different which can account for the treatment and control groups)
outcomes in subgroups association
stratified by the factor)
Lead-time bias Early detection is confused Early detection makes it seem Measure “back-end” survival
with  survival like survival has increased, (adjust survival according to
but the disease’s natural the severity of disease at the
history has not changed time of diagnosis)
Length-time bias Screening test detects diseases A slowly progressive cancer A randomized controlled trial
with long latency period, is more likely detected by a assigning subjects to the
while those with shorter screening test than a rapidly screening program or to no
latency period become progressive cancer screening
symptomatic earlier
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Statistical distribution
Measures of central Mean = (sum of values)/(total number of values). Most affected by outliers (extreme values).
tendency Median = middle value of a list of data sorted If there is an even number of values, the median
from least to greatest. will be the average of the middle two values.
Mode = most common value. Least affected by outliers.
Measures of Standard deviation = how much variability σ = SD; n = sample size.
dispersion exists in a set of values, around the mean of Variance = (SD)2.
these values. SE = σ/√n.
Standard error = an estimate of how much SE  as n .
variability exists in a (theoretical) set of sample
means around the true population mean.
Normal distribution Gaussian, also called bell-shaped.
–1σ +1σ
Mean = median = mode.
–2σ +2σ
–3σ +3σ

68%
95%
99.7%
–1σ +1σ
Nonnormal distributions –2σ
–1σ +1σ
+2σ
Bimodal Suggests two different populations (eg, –3σ–2σ +2σ +3σ
–3σ +3σ
metabolic polymorphism such as fast vs 68%
slow acetylators; age at onset of Hodgkin 68%
95%
lymphoma; suicide rate by age). 95%
99.7%
Positive skew Typically, mean > median > mode. Mode 99.7%
Median
Mode
Asymmetry with longer tail on right. Median
Mean
Mean

Mode
Median
Negative skew Typically, mean < median < mode. Median
Mode
Mean
Asymmetry with longer tail on left. Mean

Statistical hypotheses
Null (H0) Hypothesis of no difference or relationship (eg, there is no association between the disease and the
risk factor in the population).
Alternative (H1) Hypothesis of some difference or relationship (eg, there is some association between the disease
and the risk factor in the population).
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Outcomes of statistical hypothesis testing

Correct result Stating that there is an effect or difference when Reality
one exists (null hypothesis rejected in favor of H1 H0
alternative hypothesis).
Stating that there is no effect or difference when Power α
none exists (null hypothesis not rejected). Study rejects H0
(1 – β) Type I error

Study does not reject H0 β

Type II error

Blue shading = correct result.

Incorrect result
Type I error (α) Stating that there is an effect or difference Also called false-positive error.
when none exists (null hypothesis incorrectly
rejected in favor of alternative hypothesis).
α is the probability of making a type I error. p is α = you accused an innocent man.
judged against a preset α level of significance You can never “prove” the alternate hypothesis,
(usually 0.05). If p < 0.05 for a study outcome, but you can reject the null hypothesis as being
the probability of obtaining that result purely very unlikely.
by chance is < 5%.
Statistical significance ≠ clinical significance.
Type II error (β) Stating that there is not an effect or difference Also called false-negative error.
when one exists (null hypothesis is not rejected
when it is in fact false).
β is the probability of making a type II error. β β = you blindly let the guilty man go free.
is related to statistical power (1 – β), which is If you  sample size, you  power. There is power
the probability of rejecting the null hypothesis in numbers.
when it is false.
 power and  β by:
ƒ  sample size
ƒ  expected effect size
ƒ  precision of measurement

Confidence interval Range of values within which the true mean If the 95% CI for a mean difference between 2
of the population is expected to fall, with a variables includes 0, then there is no significant
specified probability. difference and H0 is not rejected.
CI for sample mean = x̄ ± Z(SE) If the 95% CI for odds ratio or relative risk
The 95% CI (corresponding to α = .05) is often includes 1, H0 is not rejected.
used. As sample size increases, CI narrows. If the CIs between 2 groups do not overlap
For the 95% CI, Z = 1.96.  statistically significant difference exists.
For the 99% CI, Z = 2.58. If the CIs between 2 groups overlap usually
no significant difference exists.
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Meta-analysis A method of statistical analysis that pools summary data (eg, means, RRs) from multiple studies
for a more precise estimate of the size of an effect. Also estimates heterogeneity of effect sizes
between studies.
Improves power, strength of evidence, and generalizability of study findings. Limited by quality of
individual studies and bias in study selection.

Common statistical tests

t-test Checks differences between means of 2 groups. Tea is meant for 2.
Example: comparing the mean blood pressure
between men and women.
ANOVA Checks differences between means of 3 or more 3 words: ANalysis Of VAriance.
groups. Example: comparing the mean blood pressure
between members of 3 different ethnic groups.
Chi-square (χ²) Checks differences between 2 or more Pronounce Chi-tegorical.
percentages or proportions of categorical Example: comparing the percentage of
outcomes (not mean values). members of 3 different ethnic groups who
have essential hypertension.
Fisher’s exact test Checks differences between 2 percentages or Example: comparing the percentage of 20 men
proportions of categorical, nominal outcomes. and 20 women with hypertension.
Use instead of chi-square test with small
populations.

Variables to be compared

Numerical (means) Categorical (proportions)

2 groups ≥ 3 groups Small sample size Large sample size

t-test ANOVA Fisher exact test Chi-square test

Pearson correlation r is always between −1 and +1. The closer the absolute value of r is to 1, the stronger the linear
coefficient correlation between the 2 variables. Variance is how much the measured values differ from the
average value in a data set.
Positive r value positive correlation (as one variable , the other variable ).
Negative r value negative correlation (as one variable , the other variable ).
Coefficient of determination = r 2 (amount of variance in one variable that can be explained by
variance in another variable).
r = –0.8 r = –0.4 r=0 r = +0.4 r = +0.8

Strong negative Weak negative No correlation Weak positive Strong positive

correlation correlation correlation correlation
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PUBLIC HEALTH SCIENCES—ETHICS

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Core ethical principles

Autonomy Obligation to respect patients as individuals (truth-telling, confidentiality), to create conditions
necessary for autonomous choice (informed consent), and to honor their preference in accepting
or not accepting medical care.
Beneficence Physicians have a special ethical (fiduciary) duty to act in the patient’s best interest. May conflict
with autonomy (an informed patient has the right to decide) or what is best for society (eg,
mandatory TB treatment). Traditionally, patient interest supersedes.
Nonmaleficence “Do no harm.” Must be balanced against beneficence; if the benefits outweigh the risks, a patient
may make an informed decision to proceed (most surgeries and medications fall into this
category).
Justice To treat persons fairly and equitably. This does not always imply equally (eg, triage).

Informed consent A process (not just a document/signature) that Exceptions to informed consent (WIPE it away):
requires: ƒ Waiver—patient explicitly waives the right of
ƒ Disclosure: discussion of pertinent informed consent
information (using medical interpreter, if ƒ Legally Incompetent—patient lacks decision-
needed) making capacity (obtain consent from legal
ƒ Understanding: ability to comprehend surrogate)
ƒ Capacity: ability to reason and make one’s ƒ Therapeutic Privilege—withholding
own decisions (distinct from competence, a information when disclosure would severely
legal determination) harm the patient or undermine informed
ƒ Voluntariness: freedom from coercion and decision-making capacity
manipulation ƒ Emergency situation—implied consent may
Patients must have an intelligent understanding apply
of their diagnosis and the risks/benefits of
proposed treatment and alternative options,
including no treatment.
Patient must be informed that he or she can
revoke written consent at any time, even orally.

Consent for minors A minor is generally any person < 18 years old. Situations in which parental consent is usually
Parental consent laws in relation to healthcare not required:
vary by state. In general, parental consent ƒ Sex (contraception, STIs, pregnancy)
should be obtained, but exceptions exist for ƒ Drugs (substance abuse)
emergency treatment (eg, blood transfusions) ƒ Rock and roll (emergency/trauma)
or if minor is legally emancipated (eg, married, Physicians should always encourage healthy
self-supporting, or in the military). minor-guardian communication.
Physician should seek a minor’s assent even if
their consent is not required.
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Decision-making Physician must determine whether the patient is psychologically and legally capable of making a
capacity particular healthcare decision. Note that decisions made with capacity cannot be revoked simply
if the patient later loses capacity. Intellectual disability alone (eg, Down syndrome, autism) is not
an exclusion criterion for informed decision-making.
Capacity is determined by a physician for a specific healthcare-related decision (eg, to refuse
medical care). Competency is determined by a judge and usually refers to more global categories
of decision making (eg, legally unable to make any healthcare-related decision).
Components (think GIEMSA):
ƒ Decision is consistent with patient’s values and Goals
ƒ Patient is Informed (knows and understands)
ƒ Patient Expresses a choice
ƒ Decision is not a result of altered Mental status (eg, delirium, psychosis, intoxication), Mood
disorder
ƒ Decision remains Stable over time
ƒ Patient is ≥ 18 years of Age or otherwise legally emancipated

Advance directives Instructions given by a patient in anticipation of the need for a medical decision. Details vary per
state law.
Oral advance directive Incapacitated patient’s prior oral statements commonly used as guide. Problems arise from variance
in interpretation. If patient was informed, directive was specific, patient made a choice, and
decision was repeated over time to multiple people, then the oral directive is more valid.
Written advance Specifies specific healthcare interventions that a patient anticipates he or she would accept or reject
directive during treatment for a critical or life-threatening illness. A living will is an example.
Medical power of Patient designates an agent to make medical decisions in the event that he/she loses decision-
attorney making capacity. Patient may also specify decisions in clinical situations. Can be revoked by
patient if decision-making capacity is intact. More flexible than a living will.
Do not resuscitate DNR order prohibits cardiopulmonary resuscitation (CPR). Other resuscitative measures that may
order follow (eg, feeding tube) are also typically avoided.

Surrogate decision- If a patient loses decision-making capacity and has not prepared an advance directive, individuals
maker (surrogates) who know the patient must determine what the patient would have done. Priority of
surrogates: spouse  adult Children  Parents  Siblings  other relatives (the spouse ChiPS
in).
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Confidentiality Confidentiality respects patient privacy and autonomy. If the patient is incapacitated or the
situation is emergent, disclosing information to family and friends should be guided by
professional judgment of patient’s best interest. The patient may voluntarily waive the right to
confidentiality (eg, insurance company request).
General principles for exceptions to confidentiality:
ƒ Potential physical harm to others is serious and imminent
ƒ Alternative means to warn or protect those at risk is not possible
ƒ Self-harm is likely
ƒ Steps can be taken to prevent harm
Examples of exceptions to patient confidentiality (many are state specific) include the following
(“The physician’s good judgment SAVED the day”):
ƒ Suicidal/homicidal patients.
ƒ Abuse (children, elderly, and/or prisoners).
ƒ Duty to protect—state-specific laws that sometimes allow physician to inform or somehow
protect potential Victim from harm.
ƒ Epileptic patients and other impaired automobile drivers.
ƒ Reportable Diseases (eg, STIs, hepatitis, food poisoning); physicians may have a duty to warn
public officials, who will then notify people at risk. Dangerous communicable diseases, such as
TB or Ebola, may require involuntary treatment.
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Ethical situations
SITUATION APPROPRIATE RESPONSE
Patient is not adherent. Attempt to identify the reason for nonadherence and determine his/her willingness to
change; do not coerce the patient into adhering and do not refer him/her to another
physician.
Patient desires an unnecessary Attempt to understand why the patient wants the procedure and address underlying
procedure. concerns. Do not refuse to see the patient and do not refer him/her to another
physician. Avoid performing unnecessary procedures.
Patient has difficulty taking Provide written instructions; attempt to simplify treatment regimens; use teach-back
medications. method (ask patient to repeat regimen back to physician) to ensure comprehension.
Family members ask for information Avoid discussing issues with relatives without the patient’s permission.
about patient’s prognosis.
A patient’s family member asks you Attempt to identify why the family member believes such information would be
not to disclose the results of a test detrimental to the patient’s condition. Explain that as long as the patient has decision-
if the prognosis is poor because making capacity and does not indicate otherwise, communication of information
the patient will be “unable to concerning his/her care will not be withheld. However, if you believe the patient
handle it.” might seriously harm himself/herself or others if informed, then you may invoke
therapeutic privilege and withhold the information.
A 17-year-old girl is pregnant and Many states require parental notification or consent for minors for an abortion. Unless
requests an abortion. there are specific medical risks associated with pregnancy, a physician should not
sway the patient’s decision for, or against, an elective abortion (regardless of maternal
age or fetal condition).
A 15-year-old girl is pregnant and The patient retains the right to make decisions regarding her child, even if her parents
wants to keep the child. Her disagree. Provide information to the teenager about the practical issues of caring for
parents want you to tell her to give a baby. Discuss the options, if requested. Encourage discussion between the teenager
the child up for adoption. and her parents to reach the best decision.
A terminally ill patient requests Overwhelming majority of states refuse involvement in any form of physician-assisted
physician assistance in ending his/ death. Physicians may, however, prescribe medically appropriate analgesics even if
her own life. they shorten the patient’s life.
Patient is suicidal. Assess the seriousness of the threat. If it is serious, suggest that the patient remain in the
hospital voluntarily; patient can be hospitalized involuntarily if he/she refuses.
Patient states that he/she finds you Ask direct, closed-ended questions and use a chaperone if necessary. Romantic
attractive. relationships with patients are never appropriate. It may be necessary to transition care
to another physician.
A woman who had a mastectomy Find out why the patient feels this way. Do not offer falsely reassuring statements (eg,
says she now feels “ugly.” “You still look good”).
Patient is angry about the long time Acknowledge the patient’s anger, but do not take a patient’s anger personally. Apologize
he/she spent in the waiting room. for any inconvenience. Stay away from efforts to explain the delay.
Patient is upset with the way he/she Suggest that the patient speak directly to that physician regarding his/her concerns. If
was treated by another doctor. the problem is with a member of the office staff, tell the patient you will speak to that
person.
An invasive test is performed on the Regardless of the outcome, a physician is ethically obligated to inform a patient that a
wrong patient. mistake has been made.
 PUBLIC HEALTH SCIENCES PUBLIC HEALTH SCIENCES—ETHICS
`  SEC TION II 269

Ethical situations (continued)

SITUATION APPROPRIATE RESPONSE
A patient requires a treatment not Never limit or deny care because of the expense in time or money. Discuss all
covered by his/her insurance. treatment options with patients, even if some are not covered by their insurance
companies.
A 7-year-old boy loses a sister to At ages 5–7, children begin to understand that death is permanent, that all life
cancer and now feels responsible. functions end completely at death, and that everything that is alive eventually
dies. Provide a direct, concrete description of his sister’s death. Avoid clichés and
euphemisms. Reassure the boy that he is not responsible. Identify and normalize fears
and feelings. Encourage play and healthy coping behaviors (eg, remembering her in
his own way).
Patient is victim of intimate partner Ask if patient is safe and has an emergency plan. Do not necessarily pressure patient to
violence. leave his or her partner, or disclose the incident to the authorities (unless required by
state law).
Patient wants to try alternative or Explore any underlying reasons with the patient in a supportive, nonjudgmental
holistic medicine. manner. Advise the patient of known benefits and risks of treatment, including
adverse effects, contraindications, and medication interactions.
Physician colleague presents to If impaired or incompetent, colleague is a threat to patient safety. Report the situation
work impaired. to local supervisory personnel. Should the organization fail to take action, alert the
state licensing board.
Patient is officially determined to Gently explain to family that there is no chance of recovery, and that brain death is
suffer brain death. Patient’s family equivalent to death. Movement is due to spinal arc reflex and is not voluntary. Bring
insists on maintaining life support case to appropriate ethics board regarding futility of care and withdrawal of life
indefinitely because patient is still support.
moving when touched.
A pharmaceutical company offers Reject this offer. Generally, decline gifts and sponsorships to avoid any appearance of
you a sponsorship in exchange for conflict of interest. The AMA Code of Ethics does make exceptions for gifts directly
advertising its new drug. benefitting patients; gifts of minimal value; special funding for medical education
of students, residents, fellows; grants whose recipients are chosen by independent
institutional criteria; and funds that are distributed without attribution to sponsors.
Patient requests a nonemergent Provide accurate and unbiased information so patients can make an informed decision.
procedure that is against your Explain to the patient that you do not perform the procedure but offer to refer him/
personal or religious beliefs. her to another physician.
Mother and 15-year-old daughter Transfuse daughter, but do not transfuse mother. Emergent care can be refused by the
are unresponsive following a healthcare proxy for an adult, particularly when patient preferences are known or
car accident and are bleeding reasonably inferred, but not for a minor based solely on faith.
internally. Father says do not
transfuse because they are
Jehovah’s Witnesses.
A child presents with injuries Contact child protective services and ensure child is in a safe location. Physicians are
inconsistent with parental story. required by law to report any reasonable suspicion of child abuse or endangerment.
270 SEC TION II PUBLIC HEALTH SCIENCES PUBLIC HEALTH SCIENCES—HEALTHCARE DELIVERY
` 

PUBLIC HEALTH SCIENCES—THE WELL PATIENT

` 

Changes in the Sexual changes:

elderly ƒ Men—slower erection/ejaculation, longer refractory period, but unchanged libido.
ƒ Women—vaginal shortening, thinning, and dryness
Sleep patterns:  REM and slow-wave sleep,  sleep latency,  early awakenings
 suicide rate
 vision and hearing
 immune response
 renal, pulmonary, and GI function
 muscle mass,  fat
Intelligence does not decrease

PUBLIC HEALTH SCIENCES—HEALTHCARE DELIVERY

` 

Disease prevention
Primary disease Prevent disease before it occurs (eg, HPV vaccination)
prevention
Secondary disease Screen early for and manage existing but asymptomatic disease (eg, Pap smear for cervical cancer)
prevention
Tertiary disease Treatment to reduce complications from disease that is ongoing or has long-term effects
prevention (eg, chemotherapy)
Quaternary disease Quit (avoid) unnecessary medical interventions to minimize incidental harm (eg, imaging studies,
prevention optimizing medications to reduce polypharmacy).
 PUBLIC HEALTH SCIENCES PUBLIC HEALTH SCIENCES—HEALTHCARE DELIVERY
`  SEC TION II 271

Major medical insurance plans

PLAN PROVIDERS PAYMENTS SPECIALIST CARE
Exclusive provider Restricted to limited panel No referral required
organization (except emergencies)
Health maintenance Restricted to limited panel Denied for any service that Requires referral from
organization (except emergencies) does not meet established, primary care provider
evidence-based guidelines
Point of service Patient can see providers Higher copays and Requires referral from
outside network deductibles for out-of- primary care provider
network services
Preferred provider Patient can see providers Higher copays and No referral required
organization outside network deductibles for all services
Accountable care Providers voluntarily enroll Medicare Specialists voluntarily enroll
organization

Healthcare payment models

Bundled payment Healthcare organization receives a set amount per service, regardless of ultimate cost, to be divided
among all providers and facilities involved.
Capitation Physicians receive a set amount per patient assigned to them per period of time, regardless of how
much the patient uses the healthcare system. Used by some HMOs.
Discounted fee-for- Patient pays for each individual service at a discounted rate predetermined by providers and payers
service (eg, PPOs).
Fee-for-service Patient pays for each individual service.
Global payment Patient pays for all expenses associated with a single incident of care with a single payment. Most
commonly used during elective surgeries, as it covers the cost of surgery as well as the necessary
pre- and postoperative visits.
272 SEC TION II PUBLIC HEALTH SCIENCES PUBLIC HEALTH SCIENCES—HEALTHCARE DELIVERY
` 

Medicare and Medicare and Medicaid—federal social MedicarE is for Elderly.

Medicaid healthcare programs that originated from MedicaiD is for Destitute.
amendments to the Social Security Act.
Medicare is available to patients ≥ 65 years old, The 4 parts of Medicare:
< 65 with certain disabilities, and those with ƒ Part A: HospitAl insurance, home hospice
end-stage renal disease. care
Medicaid is joint federal and state health ƒ Part B: Basic medical bills (eg, doctor’s fees,
assistance for people with limited income and/ diagnostic testing)
or resources. ƒ Part C: (parts A + B = Combo) delivered by
approved private companies
ƒ Part D: Prescription Drugs

Hospice care Medical care focused on providing comfort and palliation instead of definitive cure. Available to
patients on Medicare or Medicaid and in most private insurance plans whose life expectancy is
< 6 months.
During end-of-life care, priority is given to improving the patient’s comfort and relieving pain
(often includes opioid, sedative, or anxiolytic medications). Facilitating comfort is prioritized
over potential side effects (eg, respiratory depression). This prioritization of positive effects over
negative effects is called the principle of double effect.

Common causes of death (US) by age

< 1 YR 1–14 YR 15–34 YR 35–44 YR 45–64 YR 65+ YR
#1 Congenital Unintentional Unintentional Unintentional Cancer Heart disease
malformations injury injury injury
#2 Preterm birth Cancer Suicide Cancer Heart disease Cancer
#3 Maternal Congenital Homicide Heart disease Unintentional Chronic
pregnancy malformations injury respiratory
complications disease

Conditions with Readmissions may be reduced by discharge planning and outpatient follow-up appointments. The
frequent hospital table below is based on readmission for any reason within 30 days of discharge.
readmissions
MEDICARE MEDICAID PRIVATE INSURANCE UNINSURED

#1 Congestive HF Mood disorders Maintenance of Mood disorders

chemotherapy or
radiotherapy
#2 Septicemia Schizophrenia/ Mood disorders Alcohol-related
psychotic disorders disorders
#3 Pneumonia Diabetes mellitus with Complications of Diabetes mellitus with
complications surgical procedures complications
or medical care
 PUBLIC HEALTH SCIENCES PUBLIC HEALTH SCIENCES—QUALITY AND SAFETY
`  SEC TION II 273

PUBLIC HEALTH SCIENCES—QUALITY AND SAFETY

` 

Safety culture Organizational environment in which everyone Event reporting systems collect data on errors for
can freely bring up safety concerns without internal and external monitoring.
fear of censure. Facilitates error identification.

Human factors design Forcing functions (those that prevent Deficient designs hinder workflow and lead to
undesirable actions [eg, connecting feeding staff workarounds that bypass safety features
syringe to IV tubing]) are the most effective. (eg, patient ID barcodes affixed to computers
Standardization improves process reliability (eg, due to unreadable wristbands).
clinical pathways, guidelines, checklists).
Simplification reduces wasteful activities (eg,
consolidating electronic medical records).

PDSA cycle Process improvement model to test changes in

real clinical setting. Impact on patients: Do
ƒ Plan—define problem and solution

n
Pla
ƒ Do—test new process
ƒ Study—measure and analyze data
ƒ Act—integrate new process into workflow

y
ud
Ac

St
t

Quality measurements
MEASURE EXAMPLE

Structural Physical equipment, resources, facilities Number of diabetes educators

Process Performance of system as planned Percentage of diabetic patients whose HbA1c was
measured in the past 6 months
Outcome Impact on patients Average HbA1c of patients with diabetes
Balancing Impact on other systems/outcomes Incidence of hypoglycemia among patients who
tried an intervention to lower HbA1c

Swiss cheese model Focuses on systems and conditions rather than

Potential failures
an individual’s error. The risk of a threat in defense strategy
becoming a reality is mitigated by differing Hazard
layers and types of defenses. Patient harm can
occur despite multiple safeguards when “the
holes in the cheese line up.”

Harm
Defense
strategies
274 SEC TION II PUBLIC HEALTH SCIENCES PUBLIC HEALTH SCIENCES—QUALITY AND SAFETY
` 

Types of medical May involve patient identification, diagnosis, monitoring, nosocomial infection, medications,
errors procedures, devices, documentation, handoffs. Medical errors should be disclosed to patients,
independent of immediate outcome (harmful or not).
Active error Occurs at level of frontline operator (eg, wrong Immediate impact.
IV pump dose programmed).
Latent error Occurs in processes indirect from operator but Accident waiting to happen.
impacts patient care (eg, different types of IV
pumps used within same hospital).
Never event Adverse event that is identifiable, serious, and Major error that should never occur.
usually preventable (eg, scalpel retained in a
surgical patient’s abdomen).

Burnout vs fatigue
Burnout Prolonged, excessive stress  cynicism, detachment,  motivation and interest, sense of failure and
helplessness,  immunity. Medical errors due to lack of concern.
Fatigue Sleep deprivation   energy and motivation, cognitive impairment. Medical errors due to
compromised intellectual function.

Medical error analysis

DESIGN METHODS
Root cause analysis Retrospective approach. Applied after failure Uses records and participant interviews to identify
event to prevent recurrence. all the underlying problems (eg, process,
people, environment, equipment, materials,
management) that led to an error.
Failure mode and Forward-looking approach. Applied before Uses inductive reasoning to identify all the ways
effects analysis process implementation to prevent failure a process might fail and prioritizes them by
occurrence. their probability of occurrence and impact on
patients.
 CHAPTER 6

ETHICS AND STATISTICS

Basic Principles 100 PHYSICIAN-ASSISTED SUICIDE AND EUTHANASIA 103

PALLIATION AND HOSPICE 103
Autonomy 100
WITHDRAWAL OF TREATMENT 103
INFORMED CONSENT 100
RIGHTS OF MINORS 100 Biostatistics 103
SENSITIVITY AND SPECIFICITY 103
Competency 100
PREDICTIVE VALUES 104
COMPETENCY VS CAPACITY 100
INCIDENCE 104
DETENTION AND USE OF RESTRAINTS 101
PREVALENCE 104
DURABLE POWER OF ATTORNEY FOR HEALTH CARE 101
ABSOLUTE RISK 104
SURROGATE/PROXY 101
RELATIVE RISK 104
Confidentiality 101 ODDS RATIO 104
IMPORTANCE OF CONFIDENTIALITY (AND HIPAA) 101 ABSOLUTE RISK REDUCTION OR ATTRIBUTABLE RISK 105
WHEN TO VIOLATE CONFIDENTIALITY 101 RELATIVE RISK REDUCTION 105
REPORTABLE CONDITIONS 101 NUMBER NEEDED TO TREAT 105
ASKING FOLLOW-UP QUESTIONS 101 STATISTICAL SIGNIFICANCE/P VALUE 105
CONFIDENCE INTERVAL 105
End-of-Life Care 102
ADVANCE DIRECTIVES 102 Study Design 105
DO NOT RESUSCITATE ORDERS/CODE STATUS 102 SURVEYS 106
PAIN IN TERMINALLY ILL PATIENTS 102 PROSPECTIVE AND RETROSPECTIVE STUDIES 106
THE PRINCIPLE OF “DOUBLE EFFECT” 102 COHORT STUDIES 106
PERSISTENT VEGETATIVE STATE 102 CASE-CONTROL STUDIES 106
QUALITY OF LIFE 103 RANDOMIZED CONTROLLED TRIALS 107

99
100 CHAPTER 6 ETHICS AND STATISTICS

Basic Principles
Be familiar with the following principles:
■ Autonomy: The right to make decisions for oneself in accordance with
one’s own system of morals and beliefs.
■ Paternalism: Providing for your perception of patients’ needs without their
input.
■ Beneficence: Action intended to bring about a good outcome.
KEY FACT ■ Nonmaleficence: Action not intended to bring about harm.
■ Truth telling: Revealing all pertinent information to patients.
Patients have the right to refuse care ■ Proportionality: Ensuring that a medical treatment or plan is commensu-
as long as they can understand and rate with the illness and with the goals of treatment.
articulate the risks and benefits. ■ Distributive justice: Allocation of resources in a manner that is fair and
just, though not necessarily equal.

Autonomy

INFORMED CONSENT

Involves discussing diagnoses and prognoses with patients as well as any pro-
posed treatment, its risks and benefits, and its alternatives. Only with such
information can a patient reach an informed decision. Do not conceal a diag-
nosis from a patient, as doing so would violate the principle of truth telling.
However, respect your patients’ wishes if they ask you to share only certain
things with them. Under emergent circumstances, if a patient’s wishes are
unknown, consent is implied.

RIGHTS OF MINORS

The treatment of patients < 18 years of age requires parental consent unless:
■ They are emancipated (ie, financially independent, married, pregnant,
raising children, living on their own, or serving in the armed forces).
■ They are requesting contraception or treatment of pregnancy, sexually
transmitted diseases, or psychiatric illness. Note that many states require
parental consent or notice for termination of pregnancy in a minor.

Most Step 3 exam questions on parental consent will deal with situations such
as those cited above. In general, this means that for the Step 3 exam, the gov-
erning principle should be to let minors make their own decisions.

Competency

C O M P E T E N C Y V S C A PA C I T Y

The terms “competency” and “capacity” should not be used interchangeably.

Competency is a legal determination made only by a court, whereas capacity
is a clinical assessment. Both capacity and competency involve the doctor’s
assessment of a patient’s ability to think, reason, and act rationally (though not
necessarily wisely). Incapacity may be temporary and situational; it is applied
to a specific clinical question/scenario (eg, “can the patient refuse a platelet
infusion despite his untreated schizoaffective disorder”) and is not broadly
 ETHICS AND STATISTICS CHAPTER 6 101

assigned (eg, “this patient does not have capacity”). Incompetence is more
permanent (eg, severe dementia), and incompetent patients are generally KEY FACT
assigned a surrogate or guardian by the court.
Capacity can be assessed by any doctor,
but it often becomes a psychiatric
DETENTION AND USE OF RESTRAINTS consult if unclear.
Psychiatric patients may be involuntarily hospitalized only if they are a dan-
ger to themselves or to others (in accordance with the principle of benefi-
cence). The use of restraints can be considered if a patient is at risk of doing
harm to self or others, but such use must be evaluated on at least a daily basis.

D U R A B L E P O W E R O F AT T O R N E Y F O R H E A LT H C A R E

Durable power of attorney (DPoA) has two related meanings. First, it can
refer to a document signed by the patient assigning a surrogate decision
maker if he or she becomes incapacitated. Second, it can refer to the person
to whom that authority has been granted.

S U R R O G AT E / P R O X Y

A surrogate or proxy is defined as an alternate decision maker who is desig-

nated by the patient (or DPoA) and charged with making decisions in accor-
dance with the patient’s preferences.

Confidentiality

I M P O R TA N C E O F C O N F I D E N T I A L I T Y A N D H I PA A

Maintaining the confidentiality of patient information is critical. Violations are

unethical, may have legal implications, and may irreparably harm the patient-
physician relationship. The Health Insurance Portability and Accountability
Act of 1996 (HIPAA) outlines rules and guidelines for preserving patient
privacy.

W H E N T O V I O L AT E C O N F I D E N T I A L I T Y

If a physician learns about a threat to an individual’s life or well-being (ie, a

danger to self or to others), violating confidentiality is mandatory. In a similar
manner, information about child abuse or elder abuse must be reported. Inti-
mate partner violence is not a mandated reportable condition.

R E P O R TA B L E C O N D I T I O N S
A 22-year-old Jehovah’s Witness
The list of reportable conditions varies by state but often includes HIV/AIDS, presents with GI bleeding but states
syphilis, gonorrhea, chlamydia, TB, mumps, measles, rubella, smallpox, and that he does not want a blood
suspected bioterrorist events. Such reporting is mandatory, is anonymous, and transfusion. His hematocrit falls from
does not constitute a violation of patient confidentiality. 40 to 22%, and his BP falls as well. The
patient is urged to accept lifesaving
treatments but refuses. When his
ASKING FOLLOW UP QUESTIONS BP reaches a critical level, one of his
physicians initiates plans to transfuse.
Follow-up questions should be used to clarify unclear issues, such as which The rest of the team vetoes the plan.
family members can be included in discussions of care, who is the primary What ethical principles are involved,
surrogate, and what patients want to know about their own conditions. and which principle trumps the
other?
102 CHAPTER 6 ETHICS AND STATISTICS

End-of-Life Care
Patients in the end stages of a terminal illness have the right to obtain medical
treatment that is intended to preserve human dignity in dying. The best
means of reaching an agreement with the patient and family regarding end-of-
life care is to continue to talk about the patient’s condition and to resolve
decision-making conflicts. Ultimately, this is the same task that an ethics con-
sultant would attempt to perform for the physician and the patient.
KEY FACT There is a growing body of literature addressing the importance of cultural
issues in end-of-life care. In the United States, emphasis is placed on patient
The Elisabeth Kübler-Ross psychological autonomy, full disclosure of medical information, and shared decision mak-
stages at the end of life are denial, ing. However, members of other cultures may lend more credibility to family-
anger, bargaining, depression, and based decisions, particular methods of diagnosis communication, and the
acceptance. importance of subjective aspects of illness. It is important to elicit and respect
these cultural frameworks and dynamics in end-of-life care.

ADVANCE DIREC TIVES

Advance directives are oral or written instructions regarding what a patient

would want in the event that the patient loses capacity to make health-care
decisions. These instructions can be detailed or broad. Oral statements are
ethically binding but are not legally binding in all states. Remember that an
informed, competent adult can refuse treatment even if it means that doing so
would lead to death. Such instructions must be honored.

D O N O T R E S U S C I TAT E O R D E R S / C O D E S TAT U S
KEY FACT The express wishes of a patient (eg, “I do not want to be intubated”) super-
sede the wishes of family members or surrogates. Physicians should inquire
Do not resuscitate ≠ do not treat! about and follow DNR orders during each hospitalization. If code status has
not been addressed and the matter becomes relevant, defer to the surrogate.

PA I N I N T E R M I N A L LY I L L PAT I E N T S

Terminally ill patients are often inadequately treated for pain. Prescribe as
much narcotic and non-narcotic medication as needed to relieve patients’ pain
and suffering. Do not worry about addiction in this setting. Two thirds of termi-
nally ill patients reported moderate to severe pain in the last 3 days of life.

THE PRINCIPLE OF “DOUBLE EFFEC T ”

Actions can have more than one consequence, some intended, others not.
Unintended medical consequences are acceptable if the intended conse-
quences are legitimate and the harm proportionately smaller than the benefit.
For example, a dying patient can be given high doses of analgesics even if it
may unintentionally shorten life.

P E R S I S T E N T V E G E TAT I V E S TAT E
This is a conflict between beneficence
and autonomy. The physician aims to
bring a good outcome for the patient
Defined as a state in which the brainstem is intact, and the patient has sleep-
(beneficence), but the patient is wake cycles, but there is no awareness, voluntary activity, or ability to interact
deciding in accordance with his belief with the environment. Reflexes may be normal or abnormal. Some patients
system (autonomy). The principle of survive this way for 5 years or more, with the aggregate annual cost reaching
autonomy trumps beneficence in this into the billions of dollars.
situation.
 ETHICS AND STATISTICS CHAPTER 6 103

QUALIT Y OF LIFE

Quality of life refers to a subjective evaluation of a patient’s current physical,

emotional, and social well-being. This must be evaluated from the perspective
of the patient.

PHYSICIAN ASSISTED SUICIDE AND EUTHANASIA

Physician-assisted suicide is currently legal only in six states (WA, OR, MT,
VT, CA, CO) and refers to physicians prescribing medication to hasten death.
Each state has slightly different criteria; generally, patients must be informed KEY FACT
and competent, have a prognosis of 6 months or less, and must be able to self-
administer the drug. Euthanasia refers to the physician directly participating Euthanasia is illegal in all states.
in the administration of medication to end life.

PA L L I AT I O N A N D H O S P I C E

Palliative care focuses on reducing symptom burden and improving quality of

life for patients with serious medical conditions, such as cancer, heart failure, KEY FACT
chronic obstructive pulmonary disease, and amyotrophic lateral sclerosis
(ALS). A patient may still undergo treatment with curative intent and receive Palliative care is appropriate for any
palliative care. Hospice is a related specialty that focuses on providing this patient with a high symptom burden,
care in patients who are no longer receiving curative treatment. Both involve even those with a good prognosis.
interdisciplinary collaboration (MD, RN, chaplain, social worker) to manage Hospice is appropriate for patients with
the patient’s psychosocial and physical well-being in a manner that preserves a prognosis of 6 months or less.
dignity and maximizes comfort.

W I T H D R A W A L O F T R E AT M E N T

Withdrawal of treatment is the removal of life-sustaining treatment and is

legally and ethically no different from never starting treatment. The decision
to withdraw treatment may come from the patient, an advance directive, a
DPoA, or—absent any of these—the patient’s closest relative and/or a physi-
cian. It is easiest when all parties are in agreement, although this is not
required. When there is conflict, the patient’s wishes take precedence. In
futile cases or those involving extreme suffering, a physician may withdraw or
withhold treatment; if the family disagrees, the physician should seek input
from an ethics committee or obtain a court’s approval.
KEY FACT

Biostatistics Remember: sense (sensitivity) who does

have a disease; specify (specificity) who
Not everyone with a given disease will test positive for that disease, and not does not.
everyone with a positive test result has the disease. SPIN: SPecificity rules IN
SNOUT: SeNsitivity rules OUT
SENSITIVIT Y AND SPECIFICIT Y

Sensitivity is the probability that a person with a disease will have a positive
result on a test (true positive rate). Specificity is the probability that a person
without the disease will have a negative result on a test (true negative rate).
High specificity is desirable for a confirmatory test.
Ideally, a test will be highly sensitive and specific, but this is rare. A test You have a test that has a sensitivity
that is highly sensitive but not specific will yield many false positives, whereas of 0.95 and a specificity of 0.95. How
one that is highly specific but not sensitive will yield many false negatives. helpful is this test in your diagnostic
reasoning for a disease prevalence
of 50%?
104 CHAPTER 6 ETHICS AND STATISTICS

PREDIC TIVE VALUES

Positive predictive value (PPV) is the probability that a person with a positive
test result has the disease (true positives/all positives; see Table 6-1). If a dis-
ease has a greater prevalence, then the PPV is higher. Negative predictive
value (NPV) is the probability that a person with a negative test result is dis-
ease free (see Table 6-1). A test has a higher NPV value when a disease has a
lower prevalence. It is important to note that PPV and NPV can be deter-
mined only if the incidence in the sample is representative of the population.
For example, if the data for Table 6-1 are derived from a case-control study,
then the PPV and NPV cannot be calculated. Generally, one needs a cohort
study design to get PPV or NPV.

INCIDENCE

Defined as the number of new cases of a given disease per year; for example,
four cases of X per year.

PREVALENCE

Defined as the total number of existing cases of a given disease in the entire
population; for example, 20 people have X (right now).

ABSOLUTE RISK

Defined as the probability of an event in a given time period; for example,

0.1% chance of developing X in 10 years.

R E L AT I V E R I S K
KEY FACT
Used to evaluate the results of cohort (prospective) studies. The relative risk
RR is used in prospective studies to (RR) compares the incidence of a disease in a group exposed to a particular
calculate risk of developing a disease risk factor with the incidence in those not exposed to the risk factor (see
with a particular exposure. OR is used in Table 6-2). An RR < 1 means that the event is less likely in the exposed
retrospective studies to look at who has group; conversely, an RR > 1 signifies that the event is more likely in that
the exposure from among those who group.
are known to have the disease.

O D D S R AT I O

Used in case-control (retrospective) studies. The odds ratio (OR) compares

the rate of exposure among those with and without a disease (see Table 6-2). It
is considered less accurate than RR, but in rare diseases the OR approximates
Very helpful. Both positive and the RR.
negative results make significant
changes in disease probability and
can confirm or disprove a diagnosis. TA B L E 6 - 1. Determination of PPV and NPV
This is the situation in which a
laboratory test is most helpful.
DISEASE PRESENT NO DISEASE
The test would not be helpful for a
disease prevalence of 1%; most of
Positive test a b PPV = a/(a + b)
the positives will be false positives, so
further evaluation will be necessary.
Negative test c d NPV = d/(c + d)
It would not be helpful for a disease
prevalence of 90%; the positive
result adds nothing to your clinical Sensitivity = a/(a + c) Specificity = d/(b + d)
suspicion, and a negative test is likely
to be a false negative.
 ETHICS AND STATISTICS CHAPTER 6 105

TA B L E 6 - 2. Determination of RR and OR

DISEASE DEVELOPS NO DISEASE

Exposure a b RR = [a/(a + b)]/[c/(c + d)]

No exposure c d OR = ad/bc

A B S O L U T E R I S K R E D U C T I O N O R AT T R I B U TA B L E R I S K

Measures the risk accounted for by exposure to a given factor, taking into
account the background of the disease. It is useful in randomized controlled tri-
als. Numerically, absolute risk reduction (ARR) = the absolute risk (rate of KEY FACT
adverse events) in the placebo group minus the absolute risk in treated patients.
ARR and RRR give different values and
R E L AT I V E R I S K R E D U C T I O N should not be confused. ARR is a much
better measure of benefit; because it is
Also used in randomized controlled trials; this is the ratio between two risks. a ratio, RRR can look deceptively large.
Numerically, relative risk reduction (RRR) = [the event rate in control Watch out for drug advertising that
patients minus the event rate in experimental patients] plus the event rate in touts RRR.
control patients.
RRR can be deceptive and is clinically far less important than ARR. Con-
sider a costly intervention that reduces the risk of an adverse event from
0.01% to 0.004%. ARR is 0.01 − 0.004 = 0.006%, but RRR is (0.01 −
0.004)/0.01 = 0.6, or 60%! Would you order this intervention?

N U M B E R N E E D E D T O T R E AT

The number of patients needed to treat (NNT) = who would need to be treated
to prevent one event. NNT = 1/ARR. In the example above, the NNT is 167.

S TAT I S T I C A L S I G N I F I C A N C E / P V A L U E

The P value expresses the likelihood that an observed outcome was due to
random chance. A P value < .05 is generally accepted as indicating that an
outcome is statistically significant. KEY FACT

CONFIDENCE INTERVAL If a 95% CI includes 1.0, the results are

not significant. Therefore, if an RR is 1.9,
Like the P value, the confidence interval (CI) expresses the certainty that the but the 95% CI is 0.8–3.0, the RR is not
observation is real or is a product of random chance. Used with ORs and RR, significant.
the 95% CI indicates that the observed risk or odds have a 95% chance of
being within the interval. Thus, in Figure 6-1, the RR of cancer with smoking
is 2.0 with a 95% CI of 1.3–3.5—meaning that the observed RR of cancer was
2.0, and there is a 95% certainty that the actual RR of cancer from smoking 3
falls somewhere between 1.3 and 3.5.
2

Smoker
1
Study Design Nonsmoker

0
Statistical analyses are used as a means of assessing relationships between
events and outcomes. They do not prove irrefutably that a relationship exists –1
but point to the likelihood of this being the case. The validity of the results
depends on the strength of the design. FIGURE 6-1. Relative risk of cancer.
106 CHAPTER 6 ETHICS AND STATISTICS

KEY FACT SURVEYS

Self-reports of symptoms, exposures, feelings, and other subjective data. Such

Beware! Many of these types of surveys
data may be analyzed with descriptive statistics or qualitative methodologies.
are subject to recall bias, where patients
with a disease may be more likely to
report a previous exposure. PROSPECTIVE AND RETROSPECTIVE STUDIES

■ Prospective studies assess future outcomes relating to present or future

events; this enables the study designer to control for bias and to modify
inputs/exposures.
■ Retrospective studies relate to outcomes from past events. They may be
less reliable than prospective studies.

COHORT STUDIES

In a cohort study (see Figure 6-2), a population is observed over time,

grouped by exposure to a particular factor, and watched for a specific out-
come. Such studies are not good for rare conditions. Studies can be prospec-
tive or retrospective. Use RR to interpret results. Examples include the
Nurses’ Health Study and the Framingham Heart Study.

CASE CONTROL STUDIES

A case-control study (see Figure 6-3) is a retrospective study involving a group

of people with a given disease and an otherwise similar group of people with-
out the disease who are compared for exposure to risk factors. Case-control
studies are good for rare diseases. Use OR to interpret results.

Onset Time
of study
Disease
Exposed
No disease

Eligible
subjects

Disease

Unexposed
No disease

Direction of inquiry
F I G U R E 6 - 2 . Schematic diagram of a cohort study. Shaded areas in the diagram represent
exposed persons; unshaded areas represent unexposed persons. (Reproduced with permission from
Greenberg RS et al. Medical Epidemiology, 4th ed. New York: McGraw-Hill, 2005, Fig. 8-2.)
 ETHICS AND STATISTICS CHAPTER 6 107

Onset Time
of study

Exposed
Cases

Unexposed

Exposed
Controls

Unexposed

Direction of inquiry
F I G U R E 6 - 3 . Schematic diagram of a case-control study. (Reproduced with permission from
Greenberg RS et al. Medical Epidemiology, 4th ed. New York: McGraw-Hill, 2005, Fig. 9-1.)

RANDOMIZED CONTROLLED TRIALS

A prospective study that randomly assigns participants to a treatment group or

to a placebo group (see Figure 6-4). The placebo group and the treatment
group are then compared to determine if the treatment made a difference.
The double-blind randomized controlled trial is the gold standard of experi-
mental design.

Onset Time
of study

Retinopathy
Intensive
insulin
therapy
No retinopathy

Eligible Randomize
subjects

Retinopathy
Standard
insulin
therapy
No retinopathy

Direction of inquiry
F I G U R E 6 - 4 . Schematic diagram. (Reproduced with permission from Greenberg RS et al. Medical Epidemiol-
ogy, 4th ed. New York: McGraw-Hill, 2005, Fig. 7-2.)