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

Fundamentals of Clinical Trials Endpoints Betsy Garofalo MD Michigan Technology and Research Institute. Clinical Trial Features. Study objective – hypothesis testing Type of study comparisons Primary and secondary endpoints Measures taken to minimizing bias

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

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  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

  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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