This document provides an overview of comparative effectiveness research (CER), patient-centered outcomes research (PCOR), and the Patient-Centered Outcomes Research Institute (PCORI). It discusses the history and establishment of PCORI in 2010, its focus on patient-centered outcomes directly impacting patients' lives, and its funding priorities and first round of funded projects examining topics like treatment options, healthcare systems, communication/dissemination, and disparities. The agenda outlines a symposium focused on stakeholder engagement in CER and PCOR.

1. CER, PCOR, & the History of PCORI:
Making Sense of Alphabet Soup
Michael Steinman, MD
Director of Comparative Effectiveness Research, CTSI
Associate Professor of Medicine, Division of Geriatrics

2. Thanks and Disclosure
• Thank you to participants and organizers
– Tracy Lieu – Howard Pinderhughes
– Kathryn Phillips – Claire Brindis
– David Thom – Mark Pletcher
– Diane Allen
– Kevin Grumbach – Aria Yow
– Laura Schmidt – Deborah Grady
– Michael Potter – Clay Johnston
• No conflicts of interest

3. History of PCORI
• 2010 health reform created Patient Centered
Outcomes Research Institute
– CER politically unpalatable (fears that → rationing)
– So…PCOR
– Define mandate, create systems to review and
administer grants distinct from NIH or AHRQ
– Clear mandate what is it not – cannot consider cost in
coverage decisions; cannot use QALYs; will not fund
cost-effectiveness analyses

4. Patient-Centered Outcomes
• Patient-centered outcomes – outcomes that
directly impact patient’s lives and well-being
– Functional status; ability to concentrate; care for
loved ones; pain
– NOT biomarkers
– Effect size should be meaningful
– Evaluate heterogeneity of treatment effects
– Real-world settings

5. What is PCORI Funding?
• Five priority areas:
– Prevention, Diagnosis, and Treatment Options
– Improving Healthcare Systems
– Communication and Dissemination
– Disparities
– Methodological Research

6. What is PCORI Funding?
• 2011-2012
– Small grants; pilot grants - methods and research priorities
• 2012
– Recurrent (main) PFAs
• Up to $500,000 /yr for 3 years
– Innovation Challenge
• Match patients and researchers; $50,000
• 2013
– Subject-specific grants
• Treatment options for uterine fibroids; treatment options for
severe asthma; fall prevention in the older adults

7. First Round of PFAs
• 25 proposals funded (out of ~500)
– $41 million over 3 years
– (anticipate $355 million in 2013)

8. First Round of PFAs
• Prevention, Diagnosis, Treatment
– Cognitive outcomes of antiepileptic drugs in pediatric epilepsy
– Helping patients choose peritoneal dialysis vs. hemodialysis
• Healthcare Systems
– Hospital discharge planning – impact on medication problems,
functional status, re-hospitalization

9. First Round of PFAs
• Prevention, Diagnosis, Treatment
– Cognitive outcomes of antiepileptic drugs in pediatric epilepsy
– Helping patients choose peritoneal dialysis vs. hemodialysis
• Healthcare Systems
– Hospital discharge planning – impact on medication problems,
functional status, re-hospitalization
Patient-centered outcomes
(Outcomes, heterogeneous effects, real-world)

10. Stakeholder Engagement
• Stakeholder engagement critical
– Identifying relevant outcomes
– Identifying pertinent research questions
– Study design and implementation
• Stakeholders = patients, families, caregivers,
policymakers, clinicians, etc.
• Key criterion - stakeholders an active role in
partnering with investigators and developing
proposals
• Stakeholders involved in review process

11. Stakeholder Engagement
• How is this actually done?
• Main focus of this symposium

12. Agenda
8:30-9:15 Population-Based Networks for CER: Potholes and Potential
- Tracy Lieu, Division of Research, Kaiser Permanente NorCal
9:15-10:00 What PCORI Wants – Priorities and Preferred Methods
- Kathryn Phillips, UCSF TRANSPERS Center
10:00-10:20 Refreshments and Networking
10:20-11:30 Stakeholder Engagement – What is it? How do I do it?
- Kevin Grumbach, Dept. of Family and Community Med., UCSF
11:30-12:10 Resources at UCSF to Support CER and PCOR
- Multiple speakers
12:30-1:30 Cancer Interest Group Breakout – Room 384

14. First Round of PFAs
• Prevention, Diagnosis, Treatment
– Cognitive outcomes of antiepileptic drugs in pediatric epilepsy
– Helping patients choose peritoneal dialysis vs. hemodialysis
• Healthcare Systems
– Hospital discharge planning – impact on medication problems,
functional status, re-hospitalization
• Communication and Dissemination
– Dementia care for rural and Hispanic populations
• Disparities
– Literacy-adapted psychosocial treatments for chronic pain