Clinical Studies

Question 1

What are the two main types of clinical studies?

Answer 1

observational studies

clinical trials

Question 2

What are principles involved in observational studies?

Answer 2

• it does have a plan/protocol
• it could be for a procedure or an intervention, like a drug or a medical device
• all participants involved receive the same procedure or intervention

Question 3

How are principles involved in clinical trials?

Answer 3

• participants receive an intervention assigned by an investigator using some method
• trials may be used to compare known treatments/procedures or study new ones
• phases are designed by the FDA

Question 4

What is involved in each phase designated by the FDA?

Answer 4

Phase 1: drug or treatment is tested for the first time on a small group of people
Phase 2: drug or treatment is given to a larger number of people to study effectiveness
Phase 3: drug or treatment given to a larger number of people to compare with other treatments, monitor side effects, and collect safety information
Phase 4: safety tracking after FDA approval

Question 5

What does the FDA’s Expanded Access policy entail?

Answer 5

For patients with life-threatening conditions/serious diseases to have access to investigational medical products when no alternative therapy is available

Question 6

What does PICO refer to?

Answer 6

All of the categories a research question should address
Population
Intervention 
Comparator
Outcome

Question 7

What are single-blinded and double-blinded trials?

Answer 7

Single-blinded: research team knows which intervention is given but participants do not
Double-blinded: neither the research team nor participants know which intervention is being given until results are analyzed or unless medically necessary

Question 8

What does the term parallel refer to?

Answer 8

Treatment is compared against no treatment, placebo, or standard of care

Question 9

What does the term crossover refer to?

Answer 9

Participants are assigned to one arm, given a “wash-out period” and then assigned to the other arm

Question 10

What is a way to avoid having to use a control arm (especially if the affected or intended population is small)?

Answer 10

Perform retrospective chart review or look through retrospective disease registry data
perform natural history trials