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Development of PCORI Agenda on Personalized/Precision Medicine Kathryn Phillips Professor of Health Economics Dept of Clinical Pharmacy, HDFCCC, & PRL- IHPS Today’s Discussion • Why is PCORI moving towards a research agenda on precision medicine • What opportunities might emerge • What my role has been as advisor to PCORI Lots of Interest in CER and PM Compare the effectiveness of genetic and biomarker testing and usual care in preventing and treating breast, colorectal, prostate, lung, and ovarian cancer, and possibly other clinical conditions for which promising biomarkers exist. Comparative effectiveness research and genomic medicine: An evolving partnership for 21st century Medicine Building the evidence base for decision making in cancer genomic medicine using comparative effectiveness research Comparative Effectiveness Research, Genomics-Enabled Personalized Medicine, and Rapid Learning Health Care: A Common Bond But No Specific RFAs • A few topic briefs • Future Research Prioritization: Genetic Testing in Children in Whom a Rare Genetic Disease is Suspected • Future Research Prioritization: Genetic Testing in Children in Whom a Rare Genetic Disease is Suspected • A few funded studies • Enabling a Paradigm Shift: A Preference-Tolerant RCT of Personalized vs. Annual Screening for Breast Cancer – Principal Investigator Laura J. Esserman, MBA, MD Goal: RFAs that Address Key Issues – but Within PCORI Mandate • • • • • Connected with Joe Selby Developed overview of field Conducted interviews with experts Attended PMI Working Group Writing (with experts) systematic landscape review of PM & CER – where have we been, where we want to go • Stakeholder meetings? • Issuance of RFA(s) Lessons Learned • PM is a very broad topic with rapidly evolving technologies – “Let 1000 Flowers Bloom” • Need for direct comparisons of PM in real world settings & multi-disciplinary teams – NIH PMI more focused on discovery • Relevance of patient outcomes for PM • PCORI has to determine funding priorities in a complex environment – but they are open to ideas Resources & Collaborations • Outcomes/CER Facilitator for Cancer Center – Interest group in personalized medicine • Clinical pharmacy – Interest in CER of PGx Stay Tuned & Keep Your Eyes Open