Professional Documents
Culture Documents
“It is a mathematical fact that fifty percent of all doctors graduate in the ``Epidemiology and
bottom half of their class.” Biostatistics 252
—Unknown
``Ethics 260
“There are two kinds of statistics: the kind you look up and the kind you
make up.” ``The Well Patient 264
—Rex Stout
``Healthcare Delivery 265
“On a long enough timeline, the survival rate for everyone drops to zero.”
—Chuck Palahniuk ``Quality and Safety 267
“There are three kinds of lies: lies, damned lies, and statistics.”
—Mark Twain
251
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 differs 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.
Can be prospective (asks, “Who will
develop disease?”) or retrospective (asks,
“Who developed the disease [exposed vs
nonexposed]?”).
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 or more 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 healthy volunteers or patients “Is it Safe?” Assesses safety, toxicity,
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 best available treatment (or placebo). Improvement?).
Phase IV Postmarketing surveillance of patients after “Can it stay?” Detects rare or long-term
treatment is approved. adverse effects. Can result in treatment being
withdrawn from Market.
Test
NPV
disease prevalence in population being tested. – FN TN = TN/(TN + FN)
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 If sensitivity is 100%, then FN is zero. So, all
rate. High sensitivity test used for screening in negatives must be TNs.
diseases with low prevalence.
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 rules IN disease
ruling in disease and indicates a low false- If specificity is 100%, then FP is zero. So, all
positive rate. High specificity test used for positives must be TPs.
confirmation after a 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
FN FP
Raising the cutoff point: ↑ 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 and/or LR– < 0.1 indicate a very useful
diagnostic test.
LRs can be multiplied with pretest odds of
disease to estimate posttest odds.
Quantifying risk Definitions and formulas are based on the classic Disease
2 × 2 or contingency table.
or intervention
Risk factor
a b
c d
Incidence vs # of new cases (during a specified Incidence looks at new cases (incidents).
Incidence =
prevalence # of people at risk time period)
# 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).
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 trueness of test measurements. Systematic error accuracy in a test.
The absence of systematic error or bias in a test.
Accuracy Accuracy
High Low High Low
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).
Incorrect result
Type I error (α) Stating that there is an effect or difference Also known as 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, then there is less but you can reject the null hypothesis as being
than a 5% chance that the data will show very unlikely.
something that is not really there.
Type II error (β) Stating that there is not an effect or difference Also known as 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:
sample size
expected effect size
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. 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.
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 strength of evidence and generalizability of study findings. Limited by quality of
individual studies and bias in study selection.
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.
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
BEHAVIORAL SCIENCE—ETHICS
``
Informed consent A process (not just a document/signature) that Exceptions to informed consent (WIPE it away):
requires: Waiver—patient explicitly waives the right of
Disclosure: discussion of pertinent informed consent
information Legally Incompetent—patient lacks decision-
Understanding: ability to comprehend making capacity (obtain consent from legal
Capacity: ability to reason and make one’s surrogate)
own decisions (distinct from competence, a Therapeutic Privilege—withholding
legal determination) information when disclosure would severely
Voluntariness: freedom from coercion and harm the patient or undermine informed
manipulation decision-making capacity
Patients must have an intelligent understanding Emergency situation—implied consent may
of their diagnosis and the risks/benefits of apply
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 Sex (contraception, STIs, pregnancy)
should be obtained, but exceptions exist for Drugs (substance abuse)
emergency treatment (eg, blood transfusions) 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.
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.
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):
Decision is consistent with patient’s values and Goals
Patient is Informed (knows and understands)
Patient Expresses a choice
Decision is not a result of altered Mental status (eg, delirium, psychosis, intoxication), Mood
disorder
Decision remains Stable over time
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, intubation) 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).
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 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 In the overwhelming majority of states, refuse involvement in any form of physician-
physician assistance in ending his/ assisted suicide. Physicians may, however, prescribe medically appropriate analgesics
her own life. that coincidentally 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.
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.
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:
Potential physical harm to others is serious and imminent
Likelihood of harm to self is great
No alternative means exist to warn or to protect those at risk
Physicians can take steps to prevent harm
Examples of exceptions to patient confidentiality (many are state-specific) include the following
(“The physician’s good judgment SAVED the day”):
Suicidal/homicidal patients
Abuse (children, elderly, and/or prisoners)
Duty to protect—State-specific laws that sometimes allow physician to inform or somehow
protect potential Victim from harm.
Epileptic patients and other impaired automobile drivers.
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.
Car seats for children Children should ride in rear-facing car seats until they are 2 years old and in car seats with a
harness until they are 4 years. Older children should use a booster seat until they are 8 years old
or until the seat belt fits properly. Children < 12 years old should not ride in a seat with a front-
facing airbag.
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 Identifying patients at risk of unnecessary treatment, protecting from the harm of new interventions
prevention (eg, electronic sharing of patient records to avoid duplicating recent imaging studies)
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 known as the principle of double effect.
Hospitalized Defined as readmission for any reason within 30 days of discharge from original admission.
conditions Readmissions may be reduced by discharge planning and outpatient follow-up appointments.
with frequent
readmissions
MEDICARE MEDICAID PRIVATE INSURANCE UNINSURED
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).
Quality measurements
MEASURE EXAMPLE
Harm
Defense
strategies
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).