Lecture 1

Question 1

Pharmacology

Answer 1

the study of the effects of drugs

Question 2

Clinical Pharmacology

Answer 2

The study of the effects of drugs in humans

central principle: therapy should be individualized to the needs of the specific patient

Question 3

Epidemiology

Answer 3

the study of the distribution and determinants of diseases in populations

Question 4

Pharmacoepidemiology

Answer 4

The study of the uses and effects of drugs in large numbers

applies the methods of epidemiology to the content area of clinical pharmacology

Question 5

Drug approval: Phase 1

Answer 5

Years: 1-1.5
Test population: 20-80 healthy volunteers
Purpose: determine safety and dosage

Question 6

Drug approval: Phase 2

Answer 6

Years: 2
Test population: 100-300 patients
Purpose: evaluate efficacy, look for side effects

Question 7

Drug approval: Phase 3

Answer 7

Years: 3-3.5
Test population: 1000-3000 patients
Purpose: confirm efficacy, monitor ADE for long term use

Question 8

Drug approval: NDA Review

Answer 8

Years: 0.6-2.5
Purpose: new drug application is reviewed

Question 9

Drug approval: Phase 4

Answer 9

Years: 11-14
Test population: “real world evidence” - testing carried out outside of clinical environment
Purpose: additional post-market testing

Question 10

Study designs listed most to least causal

Answer 10

RCT
Cohort
case- control
analyses of secular trends
case series
case reports

Question 11

Case reports

Answer 11

A report of an event in a single patient
- useful for generating hypotheses
- simple & inexpensive

Question 12

Case Series

Answer 12

collections of patients all of whom have either a single exposure or single outcome
- no control group = no hypothesis testing
- useful to : quantify an ADE, ensure particular ADEs are not happening in population larger than studied prior to drug marketing

Question 13

Analyses of Secular Trends

Answer 13

AKA: ecological studies
Examine trends in an exposure that is a presumed cause and trends in a disease that is a presumed effect and test whether the trends coincide
- trends examined either over time or across boundaries
- lack individual data
- no control for confounding variables

Question 14

Case-control studies

Answer 14

compare cases (with outcome) to controls (without outcome) to look for differences in antecedent exposures

• Useful to study: multiple exposures & uncommon diseases
• quick & inexpensive
• potential for bias in control selection
• potential for bias in exposure determination
• measure of association: odds ratio

Question 15

Cohort Studies

Answer 15

A study that identifies a cohort of subjects and follows them over time to determine outcome
- ascertains exposures at the beginning; cases are exposed, controls are unexposed
- good for: rare exposures, multiple outcomes from one exposure
- selection bias not as likely; loss to follow up is a greater concern
- expensive, time consuming
- potential for outcome ascertainment bias
- measure of association: relative risk and attributable risk

Question 16

Randomized control trials

Answer 16

a study where the participants are randomly assigned between exposure and control groups
- only design that controls for unknown confounders
- EXPENSIVE, most difficult logistically, ethical constraints
- artificial

Question 17

Pragmatic Clinical trials

Answer 17

a study in which the investigator tests the effectiveness of an intervention under real world conditions
- hope to overcome challenges of RCTs
- RTCs are slow
not generalizable
- evidence paradox

Question 18

Study results

Answer 18

• No association
• Artificial association (chance or bias)
• indirect association (confounded)
• causal association (direct or true)

Question 19

Types of error

Answer 19

1.) Random- tested for and fixed statistically
2.) Bias - avoided with good study design
3.) Confounding- adjusted for when recognized

Question 20

Information Bias

Answer 20

1.) Interviewer bias- probing some subjects more thoroughly than others
2.) Recall bias- a type of bias that occurs when participants in a research study or clinical trial do not accurately remember a past event or experience or leave out details when reporting about them

Question 21

Selection bias

Answer 21

• when controls don’t represent the population that produced the cases
• differential loss to follow-up

Question 22

Confounder

Answer 22

A variable related to both the exposure and the outcome

Question 23

Confounding

Answer 23

Occurs when a confounder is distributed unequally between groups