Transcript Click here to view presentation slides

Clinical Trials
What Are They and When Are They
Right For You?
Maura N. Dickler
Assistant Attending Physician
Breast Cancer Medicine Service
Memorial Sloan-Kettering Cancer Center
What is a Clinical Trial?
A study designed to answer a specific
question
• Drug development
• New surgical technique
• Psychosocial intervention
• Long term side-effects of therapy
What goes into designing a clinical trial?
• Generate a hypothesis/ask a question
• Design a trial to test that hypothesis
• Involves a team of researchers including
physicians from multiple disciplines and
biostatisticians
• Review by multiple committees
– Scientific committees
– Patient safety committees (Institutional Review Board
or IRB)
– FDA
– National Cancer Institute (NCI)
Types of Trials
• Phase 1: tests dose, schedule, feasibility
– Primary endpoint: side effects
• Phase 2: tests whether the drug or
combination works
– Primary endpoint: effectiveness
– All patients have the same disease
• Phase 3: randomized trial
– Primary endpoint: is one therapy better than
the other
Different Trial Designs
• Open label
• Blinded
• Placebo-controlled
Who is Eligible to Participate?
• Inclusion criteria
– Type of breast cancer (e.g. ER+, HER2+)
– Measurable or non-measurable disease
• Exclusion criteria
– Pre-existing conditions
– Baseline organ function (e.g. good blood
counts, normal kidney function)
Informed Consent
• What is the informed consent?
• Why is it necessary?
• What information is provided?
– Rationale of the trial
– Who can participate
– Risks/benefits
– Side effects
– What is covered by the trial vs. what will be
billed to insurance
When is a Trial Right for You?
• Talk with your physician
• Utilize resources available on the web
– NCI website
– Academic websites
• Seek other opinions at academic centers
• Talk with fellow patients/advocates
• Be your own advocate, but you should not
feel alone