1. The promise, current state, andfuture of personalized medicineJeffrey M. Otto, PhD MBA National Director, CHI’s Center for Translational Research April 17, 2013
2. Overview§  Intro to personalized medicine §  Short look at the early days, circa2000-2001 §  Review of current state §  Discussion of the gap between the initialpromise and the current state §  The CTR’s approach §  Summary and conclusion
3. Definitions§  Personalized medicine: the tailoring of medical treatment tothe individual characteristics of each patient in order to classifyindividuals into subpopulations that differ in their susceptibilityto a particular disease or their response to a speciﬁc treatment. Preventative or therapeutic interventions can then beconcentrated on those who will beneﬁt, sparing expense and sideeffects for those who will not. §  Biomarkers: An indicator or pattern in a patient that reﬂectsnormal biologic processes, disease processes, or the effect ofmedical treatment. §  Translational Research: Translational research transformsscientiﬁc discoveries arising from laboratory, clinical, orpopulation studies into clinical applications to reduce diseaseincidence, morbidity, and mortality.
4. Personalized Medicine 101The Promise §  Better diagnoses and earlier interventions §  More efﬁcient drug development §  More effective therapies The Challenges §  Intellectual property §  Regulatory oversight §  Reimbursement Retrieved 03/28/2013 fromhttp://www.personalizedmedicinecoalition.org/about/about-personalized-medicine
5. The ClassicPersonalized Medicine ParadigmToxic   Not  Toxic  Eﬀec%ve  Not  Eﬀec%ve  The standard approach to medicine does not distinguish between individuals…. …although individuals within a population are often very different. Biomarkers can be used to stratify patients… …and to select a safer, more efﬁcacious treatment for the individual.
6. Personalized medicine is akin to shoemanufacturing:Like shoes at a department store, manydifferent drugs are available. Althoughseveral drugs may be available to treata particular disease, all drugs are notsafe or effective for all people.Similar to sizing for a shoe,molecular diagnostic testsinform the selection of theappropriate drug.Although the selected drugwas not createdspecifically for you, it ismore likely to work for you.
7. Biomarkers currently usedin clinical medicine§  Electrocardiogram §  PET brain image §  Bone densitometricmeasurement §  Serum chemistries §  Auto-antigens in blood §  Pulmonary function test §  X-ray §  MRI
8. Examples of -Omic Biomarkers§  DNA variation q  SNPs, rearrangements,CNVs §  DNA methylation §  Chromosomalrearrangements §  microRNA §  RNA expression §  Protein panels
9. The beginning of “irrationalexuberance” in personalized medicine
11. The Challenge of “chasing the tail”§  Statistically signiﬁcantresults are easier toachieve betweenpopulations at the leftand right ends of thediagram, but are notnecessarily meaningfulfrom a health economicsperspective Treatment efficacyFrequencyinpopulation
13. Overview of Targeted Cancer TherapiesManchana, T., Ittiwut, C., Mutirangura, A., & Kavanagh, J. J. (2010). Targeted therapies for rare gynaecological cancers. LancetOncol, 11(7), 685-693. doi: http://dx.doi.org/10.1016/S1470-2045(09)70368-7
14. Why so few success stories?§  Genomic era of medicine isless than 15 yrs old §  Technology is notsufﬁcient on its own §  Biomarkers are notnecessarily “ﬁt forpurpose” §  Test needs to work withinthe existing healthcareworkﬂow §  Stakeholder alignment §  Is the patient the customer? Cartoon: Agres, Ted. (2009) The hunt for personalization. Retrieved 03/08/2013 fromhttp://www.dddmag.com/articles/2009/06/hunt-personalization
15. Catholic Health Initiatives &The Center for Translational Research
16. CHI: 5th Largest Hospital Network in USStrength in Numbers§  5th largest US network§  81 acute care hospitals in 17 states§  40 LTC facilities§  86,000 employees§  2,900 physicians and midlevel providers§  Diverse markets with 90% ranked #1 or #2§  $15B in assets, $9.8B in annual revenue§  FY 2012 – provided $715M+ in charity care16