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Fundamentals of Clinical Trials

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Presentation on theme: "Fundamentals of Clinical Trials"— Presentation transcript:

1 Fundamentals of Clinical Trials
Endpoints Betsy Garofalo MD Michigan Technology and Research Institute

2 Clinical Trial Features
Study objective – hypothesis testing Type of study comparisons Primary and secondary endpoints Measures taken to minimizing bias Treatment groups (drug & control), dosage and dose regimen

3 Objectives and Endpoints by Phase
Efficacy/Endpoint Phase I Safety evaluation Patient tolerance None Phase II Identify efficacy endpoints and doses for Phase III “Exploratory” Primary Secondary Phase III Efficacy & Safety Evaluate for general use Primary- Indication Secondary- may be described in label Phase IV Expand efficacy & safety Expand patient reported outcomes

4 Endpoint Definition “Results, condition or events associated with individual study patients that are used to assess study treatments”

5 Characteristics of Endpoint Measures
Easy to diagnose Easy to identify no evaluator judgment needed Free of measurement error Reliable with repeated measure Internal validity Directly linked to property of interest External validity Ability to generalize to a wider population

6 Characteristics of Endpoint Measures
Clinically relevant Influence Rx choices by physicians and patients “How” and “When” to prescribe the drug

7 Surrogate Endpoint Surrogate endpoint – used instead of direct endpoint – ex BP or Cholesterol LDL Reduction as 1˚ endpoint as opposed to a reduction in CV morbidity or mortality

8 “The difference between a surrogate and a true endpoint is like the difference between a check and cash. You often get the check earlier but then, of course, it may bounce.”

9 Primary, Secondary, Tertiary…..
Primary – single (may be co-endpoint) endpoint parameter for rejection of the null hypothesis – regulatory implication in PhIII Secondary- other endpoints pre-specified, may be powered for hypothesis testing Tertiary – exploratory and all other

10 Example Epilepsy “Event” of interest: Seizures
Maintain seizure diary for study Video EEG in infants for accurate counts Global Impression of Change QOL scales 10

11 Example Depression/Anxiety
“Result” of Interest: Functional improvement/ symptom relief Hamilton Scale for Depression (HAM-D) Hamilton Scale for Anxiety (HAM-A) Global Impression of Change Suicide rates (small numbers of events, efficacy and safety issue)

12 Example Multiple Sclerosis – Relapsing Remitting
“Result” of interest: Neurological Disability “Episodes” of focal Neuro signs and symptoms Disability rating scales MRI changes CSF changes

13 Example Diabetic Neuropathy
“Event” of Interest: Foot ulcers, foot injuries Nerve conduction velocity changes Surrogate measurement Sensory changes Rating scales

14 Conclusions - Endpoints
Critical and challenging study design decision Consider Phase of development Disease under study Characteristics of measure Question to be answered by trial

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