This document discusses the development of PCORI's research agenda on personalized and precision medicine. It outlines Kathryn Phillips' role as an advisor to PCORI on this topic. Specifically, it describes efforts to develop targeted funding opportunities through PCORI that address key issues within personalized medicine while adhering to PCORI's mandate of comparative effectiveness research. This includes conducting expert interviews and stakeholder meetings to inform the development of research funding announcements focused on direct comparisons of personalized medicine approaches in real-world settings.

1. Development of PCORI
Agenda on
Personalized/Precision
Medicine
Kathryn Phillips
Professor of Health Economics
Dept of Clinical Pharmacy, HDFCCC, & PRL- IHPS

2. 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

3. 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
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.
Lots of Interest in CER and PM

4. 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

6. 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)

7. 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

8. Resources & Collaborations
• Outcomes/CER Facilitator for Cancer
Center
– Interest group in personalized medicine
• Clinical pharmacy – Interest in CER of
PGx

9. Stay Tuned & Keep Your Eyes Open