Policy and Funding for CER: Making Sense of a Confusing Landscape

Policy and Funding for CER: Making
Sense of a Confusing Landscape
Michael Steinman, MD
Director of Comparative Effectiveness Research
UCSF Clinical and Translational Science Institute

Panel
• Sheldon Greenfield, MD
Donald Bren Professor of Medicine
Executive Co-Director, Health Policy Research Institute
University of California, Irvine
Senior Editor, Journal of Comparative Effectiveness Research

• Claire Brindis, DrPH
Professor of Pediatrics and Health Policy
Director, Phillip R. Lee Institute for Health Policy Studies
UCSF

• Len Finocchio, DrPH
Associate Director
California Department of Health Care Services

Disclosure
• No conflicts of interest

Outline
• What is PCORI?
• PCOR vs. CER
• PCORI funding opportunities
• Other funding sources
• So…what should I be doing?

What is PCORI?
• Prior to several years ago, no coordinated effort for
CER
• 2009 stimulus package provided $1.1 billion for
CER
• 2010 health reform legislation created Patient
Centered Outcomes Research Institute
• Funded by tax on government and private insurers
for each covered life (~$500 million per year)

What is PCORI?
• Quasi-governmental organization
– AHRQ almost eliminated in 1990s after published guideline that
surgery for back pain was unnecessary
– Provide political protection
– PCORI funding does not depend on annual Congressional
appropriation

• No infrastructure for determining funding priorities, issuing
grant opportunities, reviewing and administering grants
– Outsourcing (NIH, AHRQ, CMS)
• Peer review, contract management
• May receive $ from PCORI to originate grants
– PCORI will be its own entity

PCOR vs. CER
• Why PCORI, not CERI?
• Political concerns that determining which treatments
better → “rationing”
– Restrict patients’ ability to choose inferior treatment
• Compromise focus on patient-centered outcomes
research
• Legislation barred PCORI from
– Developing or using QALYs
– Considering cost in making coverage recommendations
– In practice, PCORI will not fund cost-effectiveness analyses

Patient-Centered Outcomes Research
• Not just a semantic difference
• Focus on PCOR essential role in shaping funding
priorities and “rules of the game” for getting funding
• Can’t just replace placebo control with active
comparison group, do everything else the same
• Distinct elements
– Outcomes should be patient-centered
– Effectiveness, not efficacy
– Heterogeneity of treatment effects
– Involvement of stakeholders

• Outcomes should be patient-centered
– “…outcomes that people notice and care about
such as survival, function, symptoms, and health-
related quality of life”
– Not biomarkers (BP, serum cholesterol, etc.)

• Effectiveness
– Research done in clinically relevant populations
– Representative of patients
– Representative of settings

• Heterogeneity of treatment effects
– “Given my personal characteristics, conditions and
preferences, what should I expect will happen to me?”
– “What are my options and what are the benefits and
harms of those options?”

• Stakeholders play a key role
– Should be intimately involved in the study design
process to identify important outcomes
• Trials of anti-seizure drugs in children
• Traditional measure – seizure frequency
• Patient-centered measure – parent want to know how
drug will affect school performance
• Drug ↓ seizures but ↑ sedation may not be preferable

• Decision-makers are stakeholders
– Government payors and regulators; health plans; AMCs
– During study design, work with decision-makers to consider how
results will shape policy and practice
– May be important to focus on effect size, not just superiority
– Bayesian approaches – need to account for prior probability
• Value of information analysis
– Cost and cost-effectiveness
• PCORI not allowed to evaluate cost in making coverage
recommendations, won’t fund “pure” CEA
• Cost can be studied and used by decision-makers

• Implications for research design
– RCTs
• Pragmatic - real-world settings; large N’s to assess subgroups
• Bayesian and adaptive approaches to design
– Observational studies
• Causal inference modeling
• Tension for studies using pure administrative data – powerful and
low-cost; lack high-quality patient-centric data
– Which research method is best suited to which question?
• Translation table

PCORI Funding Opportunities
• Limited RFAs / RFPs to date
– Developmental
– RFPs (Sept 2011)
• ~$30-$100K each
• Methods for setting research priorities
– Pilot Projects Grant Program (Dec 2011)
• $13 million in year 1 for ~40 projects
• Develop research priorities and methods
• Develop ways of bringing stakeholders together
• Develop measurement tools for novel patient-centered outcomes

PCORI Funding Opportunities
• $500 million/year
• ?????
– Likely NOT be “traditional” research
– Patient-centered outcomes
• Including developing methods
– Real-world populations
– Heterogeneity of treatment effects
– Involvement of stakeholders

Other Funding Opportunities
• Existence of PCORI does not preclude government agencies or
foundations from funding CER
• AHRQ has Effective Healthcare Program
– Large evidence-synthesis and dissemination projects
– EBPCs, DEcIDE network, CERTS
– Does not support investigator-initiated projects
• Areas of CER explicitly outside PCORI’s mandate
– CER with “traditional” outcomes
– Cost-effectiveness analyses
• Remains to be seen

So…what should I be doing?
• Still many unknowns
– It’s not just you who doesn’t understand

• Maintain a diverse portfolio and be flexible

• Triangulate between funding agencies

• Pay close attention to unique priorities and “rules of
the road” established by PCORI
– Require substantial changes to the way we traditionally do
research

Extra slides on following pages

PCORI Definition of PCOR
PCOR questions

1. “Given my personal characteristics, conditions and
preferences, what should I expect will happen to
me?”
2. “What are my options and what are the benefits and
harms of those options?”
3. “What can I do to improve the outcomes that are
most important to me?”
4. “How can the health care system improve my chances
of achieving the outcomes I prefer?”

PCORI Definition of PCOR
Characteristics of PCOR that will facilitate answering these questions
• Assesses the benefits and harms of preventive, diagnostic,
therapeutic, or health delivery system interventions to inform
decision making, highlighting comparisons and outcomes that
matter to people;
• Is inclusive of an individual’s preferences, autonomy and needs,
focusing on outcomes that people notice and care about such as
survival, function, symptoms, and health-related quality of life;
– Incorporates a wide variety of settings and diversity of participants to
address individual differences and barriers to implementation and
dissemination; and
• Investigates (or may investigate) optimizing outcomes while
addressing burden to individuals, resources, and other stakeholder
perspectives

What is CER?
• Comparative effectiveness research is the conduct and
synthesis of systematic research comparing different
interventions and strategies to prevent, diagnose, treat
and monitor health conditions. The purpose of this
research is to inform patients, providers, and decision-
makers, responding to their expressed needs, about
which interventions are most effective for which
patients under specific circumstances. To provide this
information, comparative effectiveness research must
assess a comprehensive array of health-related
outcomes for diverse patient populations…
– Federal Coordinating Council for Comparative Effectiveness
Research, definition; [cited 28 July 2010].

Policy and Funding for CER: Making Sense of a Confusing Landscape

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Policy and Funding for CER: Making Sense of a Confusing Landscape