Flashcards in Classes #16-#19: Interventional Study Designs Deck (19)
What type of study is required by the FDA in order to get consideration for approval of a new drug?
What are some other common names for interventional study designs?
*NOT the only design for these terms for studies
When comparing observational studies and interventional study design, what are the key differences of interventional studies?
>Investigator selects "interventions" and allocates study subjects to forced-intervention groups
>More "rigorous" in ability to show cause-and-effect
- can demonstrate CAUSATION
What are the phases of interventional studies?
Pre-Clinical -> Phase 1 -> Phase 2 -> Phase 3 -> Phase 4
Bench and Animal research would be in which phase of interventional studies?
Pre-Clinical (prior-to human investigation
What is the primary focus of the researchers in phases 1 and 4 in an interventional study?
*remember that safety is always of concern
Which phase of an interventional study would most likely be expected if the study was of short duration and had an N-value of 79?
Phase 1 (new drug/device/procedure)
What is the primary focus of phases 2 and 3 in an interventional study design?
Primary focus is on effectiveness (efficacy).
In a study that reaches phase 4 that is looking at a new drug, what change would you expect about the drug between phase 3 and 4?
The drug would not reach phase 4 without having a name. Thus, a named drug.
What is the average N-value in a phase 2 study and what is the expected duration of the study? Also, is restriction present in participants allowed to enter the study?
N-value = 100-300 people
Short-to-Medium Duration (a few to several months)
Yes, likely to have a narrower inclusion criteria for participants in study
What kind of patients are usually chosen for phase 2 studies?
Commonly utilize patients with condition of interest, used to expand on purpose of Phase 1 study (safety) but also to begin assessing efficacy in diseased population.
For a phase 3 study, what is the expected N-value, study duration, and typical patient selection criteria?
Long duration (many months to a year (or few years))
Used in patients with condition of interest to continue determination of safety, with primary purpose to assess efficacy.
In which phase of interventional study design does the FDA approve or not prove a drug of interest?
Can a drug that's in phase 3 of an interventional study be named?
*Dr. Segars said, "don't get confused on the exam if I give you a study that has a named drug and think that it can't be phase 3 and that it is only in phase 4".
Would an interventional study enter phase 4 after or before approval from the FDA?
After approval from the FDA.
*phase 4 (post-marketing)
Discus phase 4 of a study and why it is performed and sometimes mandated by the FDA?
Long-term effects (risks and benefits) in a large population of diseases patients (expanded use population (age, ethnic)).
- registries, survey's (e.g., FDA's MedWatch/FAERS/VAERS programs)
What are 2 advantages of an interventional trial, versus other designs?
1) Cause precedes effect (shows Causation)
2) Only design used by FDA for "approval" process (on-label)
What are a few of the disadvantages of interventional trials, versus other designs?
2. complexity/time (development/approval/conductance)
3. ethical considerations (risk vs. benefit evaluation)
4. generalizability (a.k.a.; External Validity) - is study population similar to general population and will methodology and findings be applicable to them?