EBM Exam 2

• some type of occurrence of an event or outcome
• can be time of an event
• reflects functioning or survival

overlook other benefits

• relevant
• accurate
• precise
• reliable
• assessable

define a single primary endpoint

many endpoints

• used when benefit of just one endpoint is not sufficient
• ex. immediate cardiac failure (w/in 2 hours) and mid-term mortality (14 days)

• "in our study we defined HTN as DBP >95 mmHG."
• simple to understand
• more clinically relevant

• "In our study we measured DBP in mmHG over a six month period"
• increased efficiency (fewer pt needed)
• Avoid arbitrary cut-off values

• too costly (2 phase two trials = cost of 1 RTC (phase 3) trial)
• time consuming
• require too many patients

biomarker that is intended to substitute for a clinical endpoint

• faster trial completion and drug approval
• more frequent measurements
• provides insight to MOA and pharmacology
• guides dose selection
• more efficient screening of drug candidates
• less cost to mfg or sponsor

• should be based on clinical insight and empirical evidence
• understand pathways and MOA
• requires meta-analysis of many trials

easier to show effect of an intervention on clinical endpoints than to validate a surrogate marker

• two or three endpoints grouped together
• often in large CV trials
• can blunt meaningful endpoints

• are the components of the endpoints of similar importance to patients?
• did the more and less important end points occur with similar frequency?
• are the component endpoints likely to have similar relative risk reductions?

• laboratory, radiologic or physically assessed measure believed to correlate with clinically important outcome
• ex. BP, BG...

surrogate outcome may not correlate with clinically important outcomes

increased since surrogate endpoints like BP are more responsive to intervensions than hard clinical endpoints such as stroke

endpoint consisting of two or more individual endpoints grouped together

may amalgamate different endpoints of unequal clinical importance making it difficult to interpret effects of an intervention.

increased since multiple endpoints are amalgamated together, increaseing event rates