CER, PCOR, & the History of PCORI:Making Sense of Alphabet Soup

Michael Steinman, MDDirector of Comparative Effectiveness Research, CTSIAssociate Professor of Medicine, Division of Geriatrics

Thanks and Disclosure

– Tracy Lieu– Kathryn Phillips– David Thom– Diane Allen– Kevin Grumbach– Laura Schmidt– Michael Potter

– Howard Pinderhughes– Claire Brindis– Mark Pletcher

– Aria Yow– Deborah Grady– Clay Johnston

• No conflicts of interest

• Thank you to participants and organizers

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

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

What is PCORI Funding?

• Five priority areas:– Prevention, Diagnosis, and Treatment Options– Improving Healthcare Systems– Communication and Dissemination– Disparities– Methodological Research

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

First Round of PFAs

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

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

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)

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

Stakeholder Engagement

• How is this actually done?

• Main focus of this symposium

Agenda

8:30-9:15

9:15-10:00

10:00-10:20

10:20-11:30

11:30-12:10

12:30-1:30

Population-Based Networks for CER: Potholes and Potential

- Tracy Lieu, Division of Research, Kaiser Permanente NorCal

What PCORI Wants – Priorities and Preferred Methods

- Kathryn Phillips, UCSF TRANSPERS Center

Refreshments and Networking

Stakeholder Engagement – What is it? How do I do it?

- Kevin Grumbach, Dept. of Family and Community Med., UCSF

Resources at UCSF to Support CER and PCOR

- Multiple speakers

Cancer Interest Group Breakout – Room 384

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