Lecture 6: Pragmatic Trials for comparative effectiveness and safety Flashcards Preview

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Flashcards in Lecture 6: Pragmatic Trials for comparative effectiveness and safety Deck (4):
1

Premise for Pragmatic Trials

In the real world, there is always confounding by indication which can be addressed in design and/or analysis, but if there is a chance for strong confounding or lots of unmeasured confounders, a pragmatic trial is a real-world study wth baseline randomization

2

When should LST be considered?

-When objective is to demonstrate comparative effectiveness/safety in the real world
-when randomization is required to avoid confounding
-when endpoints can be evaluated through FU
-If these considerations are not met, the choice you are left with is: Phase IV RCT, observational (non-randomized) study, or non-randomized prospective study with follow-up and endpoint collection

3

To be considered "real world", LST designs must:

-inclusion/exclusion must be minimal and focused on investigators interpretation of local label
-enroll investigators and subjects to reflect actual settings for the former and key patient demographics for the latter
-comparator should be active
-open label (patient/physician can discontinue randomized tX
-Study may need to be larger/longer than pooled clinical data

4

To be considered successful, LST designs must:

-Success depends on sufficient enrollment and continuation over a long term
-requires informed consent and case-report forms to be short and easy to understand
-data collected based on routine practice