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

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Presentation on theme: "Fundamentals of Clinical Trials Endpoints Betsy Garofalo MD Michigan Technology and Research Institute."— 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 PhaseObjectiveEfficacy/Endpoint Phase ISafety evaluation Patient tolerance None Phase IIIdentify efficacy endpoints and doses for Phase III Exploratory Primary Secondary Phase IIIEfficacy & Safety Evaluate for general use Primary- Indication Secondary- may be described in label Phase IVExpand 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

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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