Exam II Flashcards
(110 cards)
What are design selections based on?
- perspective of question (hypothesis)
- ability/desire to force group allocation (randomization)
- ethics of methodology
- efficiency and practicality (time and resource devotion)
- cost
- validity of acquired information
What are the different hypotheses researchers formulate before the study takes place?
null hypothesis (Ho)- a research perspective which states there will be no (true) difference between the groups being compared
-conservative and commonly utilized
-perspectives
alternative hypothesis (H1): perspective which states there will be a (true) difference between the groups being compared
What are the two blanket designs for human designs?
observational: study design considered “natural”
- researchers observe subject elements occurring naturally or selected by individual (naturally or freely)
- typically not able to prove causation
- NO RESEARCHER FORCED GROUP ALLOCATION
- useful for forced interventions (not ethical)
Interventional: study designs considered experimental
- investigator selects interventions (exposure)
- there is researcher forced group allocation -> randomization
What the studies that can be found under the two blanket designs?
Observational:
- cases (reports/seres)
- ecological
- cross sectional (analytical)
- case control (analytical)
- cohort (analytical)
Interventional:
- pre-clinical
- phase I
- phase II
- phase III
- phase IV
What are the two types of error and what do they mean?
Type I error: false positive
Type II error: false negative
What three perspectives are taken for a hypothesis?
- superiority
- noninferiority
- equivalency
What is the difference between a population and sample?
population: all individuals making up a common group, from which a sample can be obtained, if desired
- not study population which is group of individuals selected for the study
sample: a subset or portion of the full complete population -> representatives
- random processes to draw sample
- when complete population is not feasible
How is a study population selected?
-research hypothesis/question
-inclusion and exclusion selection criteria and case and control group or exposed vs nonexposed
+desired vs logical vs plausible
+impact generalizability -> external validity
-ethics (principles of bioethics must be met)
+some don’t agree with the use of placebo when treatment is available
-equipoise (genuine confidence that an intervention may be worthwhile in order to use on humans)
What are the 4 key principles of bioethics?
- autonomy: self rule/self determination. Participants must…
- have full and complete understanding of the risks and benefits
- decide for oneself without outside influences
- beneficence: benefit or do good for the patient (NOT SOCIETY)
- justice: equal and fair treatment regardless of patient characteristics
- nonmaleficence: DO NO HARM! Researchers must not…
- withhold information
- provide false information
- exhibit professional incompetence
What is the Belmont Report? What are the three guiding principles?
-document that determines ethicality of a study.
- respect for persons: research voluntary, subjects autonomous
- beneficence: research risks are justified by potential benefits
- justice: risks and benefits are equally distributed
What is consent vs assent?
consent: agreement to participate, based on being fully and completely informed (given by mentally capable individuals of legal consenting age)
assent: agreement to participate, based on being fully and completely informed, given by mentally capable individuals NOT able to give legal consent (children and mentally handicapped)
- requires assent of subject and consent of guardian
Who determines the ethicality of a study?
Institutional Review Board (IRB) or the Ethics Committee
- protect human subjects from undue risk BEFORE study
- IRB regulated by Federal statues developed by Department of Health and Human Services -> follow Common Federal Rules
What are the different levels of IRB review?
Full Board: used for ALL interventional trials with more than minimal/no risk to patients -> medication related studies
Expedited: minimum risk and/or no patient identification
Exempt: no patient identifiers, low/no risk, data set analysis, environmental studies, use of existing data/specimen
Who determines safety AFTER a study has begun?
-Data Safety and Monitoring Board (DSMB)
-semi-independent committee not involved with the conduct of the study but charged with reviewing study data as study progresses, to assess for undue risk or benefit
+pre-determined review period
+can end study early for overly positive or negative effects
What occurs during the pre-clinical phase of an interventional study?
- occurs prior to human research
- bench and animal research
Whats occurs during Phase I of an interventional study?
SAFETY
-small N (20-80), usually healthy volunteers to assess safety/toxicity, dosing and pharmacokinetics in population of interest, short duration
*doesn’t always include healthy subjects
What occurs during Phase II of an interventional study?
safety and EFFICACY
-larger N (100-300), commonly utilize patients with condition of interest and nothing else, used to expand on purpose of phase I study(safety) but also begin assessing efficacy in diseased population, short-medium duration
*narrower inclusion criteria
What occurs during phase III of an interventional study?
EFFICACY
-larger N(1000-3000), used patients with condition of interest to continue determination of safety, primary purpose is to assess efficacy, longer duration
+superiority v noninferiority v equality format
+FDA mandates phase 3 before drug approval, must beat placebo and exhibit equipoise
+companies only have to show efficacy in 3 out of 5 studies
What occurs during phase IV of an interventional study?
SAFETY
-assesses long term effects (risks and benefits) in a large population of diseased patients, drug CAN die after phase 4 due to long term effects, always named drug/device
What are the advantages and disadvantages of interventional trials?
advantages:
- cause precedes effect -> shows causation
- only design used by FDA approval process
disadvantages:
-cost
-complexity/time (development/approval/conductance)
-ethical considerations**this is good***
-generalizability (external validity)
+study pop may not be equivalent to general pop
What is the difference between an explanatory and pragmatic study?
explanatory: trying to prove causation or explain why something is happening, set methods and they can’t change to suit patient
pragmatic: clinical setting (adjust dosage, etc to treat patients), allow people with comorbidies and multiple meds
+bad -> no control or regiment (adds extra confounding)
Differentiate between simple and factorial studies.
simple: randomizes(divides) subjects into 2 groups, tests a single hypothesis, randomizes only once
factorial: randomizes multiple times! Tests multiple hypotheses, but must increase sample size
What are some positives and negatives of the factorial study?
- improves efficiency for answering clinical qs
- increases study pop size
- increases complexity
- increase risk of drop out (due to complexity)
- may restrict generalizability of results
What is the difference between a parallel and cross-over study?
parallel: group simultaneously and exclusively managed
-NO SWITCHING of intervention groups after randomization
+simple and factorial are parallel
cross-over (Self control): groups serve as their own control by crossing over from one intervention to another during the study
- allows for smaller N
- control for the same person taking the same drugs (identical people)
- requires wash out period