1. 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

2. 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

3. 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)

4. 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

5. Different Trial Designs Open label Blinded Placebo-controlled

6. 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)

7. 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

8. 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