Taking Research and Development To The Clinic… Are We There Yet? Paula Kim , TRAC- Translating Research Across Communities AAAS-FDLI Personalized Medicine: Planning for the Future Colloquium 1: Diagnostics and Diagnoses: Paths to Personalized Medicine Washington DC • June 1, 2009

The Art of Translation… “ From the standpoint of medicine as an art for the preparation and cure of disease, the man who translates the hieroglyphics of science into the plain language of healing is certainly more useful.” -Sir William Osler, Physician

Across The Scientific Spectrum Physical Behavioral Social Cultural Economic Political Spiritual Technological

H PE

For Our Road Trip… How Do We Describe Personalized Medicine? Journey Destination Vehicle Highway Road Map Compass

Patient Dilemma with Words and Decisions… Personalized medicine? Donate tissue? Biospecimen? Biopsy? 2nd pass? Targeted Therapies? Genomics? Proteomics? Invasive ? Non-invasive? Academic Center ? Local ? Informed Consent? How to pick treatment? Do my kids need genetic testing? Where is my tissue? Eligible ? Insurance ? Am I Going to Live?

The Promise Personal Targeted Therapies Seek and Control instead of Search and Destroy Genetic Testing Reduced Toxicities Predictive/Preventive Capability Reduced drug failure rates A CURE Hype or Not?

POSITIVE MESSAGE ? OR MIXED MESSAGE ? NEGATIVE MESSAGE ?

Hide and Seek? Currently… No coordinated system to assist patients Many patients with difficult to treat disease have poor prognosis, limited options, and a very short timeframe to make decisions; lack access to internet Patients and caregivers often receive very little direction & guidance for treatment, system navigation and financial options

Treatment Decision Making: Outside Sources of Info for Patients Answer Options Greatly influences Somewhat Barely Does not influence Not applicable Information received from a patient advocacy organization 15 63 19 7 11 Information read in the newspaper or mainstream magazine 5 37 35 27 10 Information heard on the TV or radio 1 20 41 Information retrieved on the internet 60 25 9 8 Source: TRAC Perspectives on Costs of Cancer Care Survey-2006

Understanding Choices In Order To Make Decisions: What Patients Want Source: TRAC Perspectives on Costs of Cancer Care Survey-2006

The Law and Spice of Life “Variability is the law of life, and as no two faces are the same, so no two bodies are alike, and no two individuals react alike and behave alike under the abnormal conditions which we know as disease.” -Sir William Osler, Physician 1849-1919

Hide and Seek… …Olly Olly Oxen Free Ready or not, personalized medicine is moving… towards becoming the norm and not the exception…How fast we get there…will depend on many factors with perhaps two of the most important ones being the access and use of high quality well annotated biospecimens… and how well or not everyone… behaves along the way.

Who is in the Biospecimen Sandbox? Personalized Medicine Who is in the Biospecimen Sandbox? Patients Caregivers Public Organizations Advocacy Research and Medical NCI FDA CMS HRSA AHRQ NIH 3rd Party Payers Industry P&P Agencies and Industry Researchers Clinical Trial PI Community MDs Academic Center MDs and PhDs Pathologist Frame: Possibly use in the discussion – Check anyone you give info to, need info from PKI © 2008 All Rights Reserved

Challenges and Questions That Will Arise Do We Have a National System Will It Level the Playing Field or Not Will Access Be More Inclusive or Exclusive How Does This Affect Me and My Family Just Because You Build It, Does Not Mean They Will Come

Why We Must Get This Right… “ We must harness the scientific, industrial, and social will to accelerate discoveries into accessible and meaningful clinical applications. Patients, families, friends, and loved one are counting on us…Because in the end, it’s not about you, or me, or the science, it’s about the patient.” Paula Kim - Research & Patient Advocate

Patient & Advocate Perspectives Don’t Leave Home Without Them www.tracnetwork.net