The Patient-Outcomes Research Institute: National Priorities for Research and Initial Research Agenda Bryan Luce, PhD, MBA Chief Science Officer

Why PCORI?

!   Research has not answered many questions patients face

!   People want to know which treatment is right for them

!   Patients need information they can understand and use

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“The purpose of the Institute is to assist patients, clinicians, purchasers, and policy-makers in making informed health decisions by advancing the quality and relevance of evidence through research and evidence synthesis that considers variations in patient subpopulations and the dissemination of research findings with respect to the relative health outcomes, clinical effectiveness, and appropriateness of medical treatments, services.”

Source: Affordable Care Act. Subtitle D—Patient-Centered Outcomes Research Institute PUBLIC LAW 111–148—MAR. 23, 2010 3

“The Institute shall establish a standing methodology committee to…develop and improve the science and methods of comparative clinical effectiveness research”

Our Mission

PCORI helps people make informed health care decisions, and improves health care delivery and outcomes, by producing and promoting high integrity, evidence-based information that comes from research guided by patients, caregivers and the broader health care community.

4 Pictured: PCORI Board of Governors

Our Focus

Comparative Clinical Effectiveness Research

  Patient-centered   Answering questions that matter to patients and

other clinical decision makers   Comparisons of outcomes that matter to patients

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Our Work Answers Patients’ Questions

Given my personal characteristics, conditions and preferences…

“What should I expect will

happen to me?”

“What are my options and what are the

potential benefits and

harms of those options?”

“What can I do to improve the outcomes that

are most important to

me?”

“How can clinicians and the

care delivery systems they work

in help me make the best decisions about my health and healthcare?”

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We Use Engagement as a Path to

Rigorous Research

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Our National Priorities for Research

Assessment of Prevention, Diagnosis, and Treatment

Options Improving Healthcare Systems Communication &

Dissemination Research

Addressing Disparities Accelerating PCOR and Methodological Research

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How We Pick Research Questions to Study

Our Advisory Panels

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!   Leverage knowledge and experience of clinicians, researchers, patients, and other experts to help PCORI achieve its goals

!   Assure meaningful patient engagement in:   Identifying research priorities and topics   Monitoring research activities   Conducting randomized clinical trials

Addressing Disparities

Assessment of Prevention,

Diagnosis, and Treatment Options

Improving Healthcare Systems

Patient Engagement

Two Mandated Advisory Panels

!  Clinical Trial Methods

!  Rare Diseases

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How We Review Research Proposals

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Who: Each proposal is reviewed by two scientists, a patient, and another stakeholder. What: Reviewers critique and score applications, making recommendations that our staff and Board of Governors consider carefully in deciding which applications to approve. How: We provide training so all participants are prepared and understand PCORI’s review criteria. When: You can apply at any time to serve as reviewer.

Learn more pcori.org/reviewers Contact us reviewers@pcori.org

Our Review Criteria

Priorities 1-4: (CER)

•  Impact of the condition on health of individuals and populations

•  Potential for improving care and outcomes •  Technical merit •  Patient-centeredness •  Patient and stakeholder engagement

Priority 5: (Methods)

•  Impact on field of patient-centered outcomes research

•  Potential for the study to improve PCOR methods •  Technical merit •  Patient-centeredness •  Patient and stakeholder engagement

Our Methodology Standards

!   Formulating Research Questions !   Patient-Centeredness !   Systematic Reviews !   General and Crosscutting Methods For All

PCOR !   Causal Inference Standards !   Heterogeneity of Treatment Effect (HTE) !   Preventing and Handling Missing Data !   Data Networks !   Adaptive and Bayesian Trial Designs !   Data Registries !   Studies of Diagnostic Tests

Research we fund must adhere to PCORI’s methodological standards.

How We Promote Participation in Research

Engagement Awards

Matching Challenge

Pilot Projects

!   Pilot Projects: address a broad range of questions about methods for engaging patients in various aspects of the research and dissemination process.

!   Matching Challenge: two challenge winners created innovative ways to connect patients and researchers as partners in research.

!   Engagement Awards: Pipeline to

Proposal Awards will build a national community of patients and stakeholders who have the expertise and passion to participate in PCORI research, and create partnerships within that community that lead to high-quality research proposals.

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Our Growing Research Portfolio

Total number of research projects approved to date: 126 Total funds committed to date: $159.3 million Number of states where we are funding research: 33 states Expected research commitment in 2013: ~ $417 million

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PCORI Pilot Projects

Advance methods for engaging patients in the research process and identify gaps in comparative clinical effectiveness research that need further development 50 Awards

$31,000,000

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Chronic Conditions

25%

Cancer 11%

Acute 8%

Behavioral Health 13% Pediatrics

10%

Geriatrics 10%

Rare Diseases

4%

Other 6%

Heart Disease

13%

By Health Topic

Assessment of Prevention, Diagnosis and Treatment Options Program Director: David Hickam

Projects that address critical decisions that patients, their caregivers, and clinicians face with too little information.

29 Awards

$48,931,421

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Back/Spine 10%

Cancer 25%

Cardiovascular 17%

Infectious Disease

7%

Mental Health 10%

Neurological

10%

Pulmonary 7%

Other conditions

14%

By Health Topic

Improving Healthcare Systems (Program Director: Chad Boult)

Projects that address critical decisions that face healthcare systems, the patients and caregivers who rely on them, and the clinicians who work within them.

19 Awards

$34,455,538

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Care Transitions

16% Access to Care 10%

End-of-Life and Home Services

16%

Chronic Illness 26%

Mental Health 21%

Cancer 11%

By Health Topic

Communication & Dissemination Research Program Director: Still Searching!

Projects that address critical elements in the communication and dissemination process among patients, their caregivers, and clinicians.

14 Awards

$22,564,060

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Children 19%

All Patients 12%

Older Adults 12% Chronic

Condition Patients

13%

Racial/ Ethnic

Minorities 13%

Other 31%

By Population

Some studies address multiple populations.

Addressing Disparities Program Director: Romana Hasnain-Wynia

Projects that inform the choice of strategies to eliminate disparities.

14 Awards

$23,631,498

Chronic conditions

36%

Sexual & Reproductive Health

14%

Mental Health 29%

Infectious Disease

7% Cancer

7%

Non-Condition Specific

7%

By Health Topic

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Accelerating PCOR and Methodological Research Program Director: Rachael Fleurence

Projects that aim to improve the nation’s underlying capacity to conduct patient-centered outcomes research rapidly, efficiently, and consistently over time.

Improving Methods for Conducting Patient-Centered Outcomes

Research

Award Date: September 2013

National Patient-Centered Clinical Research Networks

Anticipated Award Date:

December 2013

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Recently Approved Methods Slate

Methods Area Number funded projects Causal  Inference   3  

Causal  Inference/Evidence  Synthesis   1  

Evidence  Synthesis   1  

HTE   2  

Missing  Data   3  

Pa?ent  Engagement   2  

PRO/PCO   4  

Registries   1  

Research  Priori?za?on   1  

Validity  of  Data  Sources   1  

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Data Infrastructure for Patient-Centered Outcomes Research

Ideal Data Infrastructure

for PCOR

Covers large, diverse, defined

populations from usual care

settings

Allows for complete capture

of longitudinal data

Capacity for collecting patient

reported outcomes

Active patient and clinician

engagement in governance of

data use Is efficient in

terms of costs for data acquisition, storage, analysis

Linkages to health systems for rapid

dissemination of findings

Capable of randomization—at individual and

cluster levels

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National Patient-Centered Clinical Research Network

!  Clinical Data Research Networks (CDRNs), which are system-based networks (such as hospital systems) that have the potential to become an ideal electronic network, without structural impediments.

!  Patient-Powered Research Networks (PPRNs), which are groups of patients interested in forming a research network and in participating in research.

!  A Coordinating Center which will provide technical and logistical assistance under the direction of the Steering Committee and PCORI Staff.

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National Patient-Centered Clinical Research Network: Our Vision

Steering Committee

•  Awardees •  PCORI •  AHRQ, NIH, FDA,

ONC, CMS, VA

Scientific Advisory Board

Special Expert Group

Potential Participants

  Patient Organizations   Practice Based Research Networks   Medical Groups   Medical Centers   Health Plans   Integrated Delivery Systems   Disease Registries   Centers for Medicaid and Medicare

Services   State and Local Health Agencies

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Targeted Topics

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Obesity Back Pain

Advisory Panel Topics

Initial Targeted PFA Topics Next Round of Targeted Topics

And more new prioritized topics…

Falls

Uterine Fibroids

Asthma

Possible Future Targeted Topics

Addressing Disparities

Hypertension in minorities

Heart attacks among racial & ethnic minorities

Interventions for improving perinatal

outcomes

Reduce lower extremity amputations in minorities

Clinical Effectiveness

Management strategies for ductal carcinoma in situ

Medication treatment options for Bipolar disorder

Treatment strategies for symptoms of osteoarthritis

Treatment strategies for adults with migraines

Improving Healthcare Systems

Hospital to Home Transition al Care

Patient empowering care management for patients

with chronic conditions

Interventions for improving perinatal outcomes

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Future Directions Under Consideration

!   Increased focus on “targeted” areas of interest

!   Increased proportion of larger, longer projects within “broad” PFAs   Presently: $500K/year; max 3 years ($1.5M total)   Considering including some: $1M/yr; max 5 years

!   Increased emphasis on “areas of interest” within broad PFAs

Connect with PCORI

Bryan Luce, PhD, MBA Chief Science Officer bluce@pcori.org

Romana Hasnain-Wynia, MA Program Director- Addressing Disparities rhasnainwynia@pcori.org

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Kara O. Walker, MD, MPH, MSHS Deputy Chief Science Officer kwalker@pcori.org

Joe Selby, MD, MPH Executive Director jvselby@pcori.org

Rachael Fleurence, PhD Program Director- CER Methods and Infrastructure rfleurence@pcori.org

David Hickam, MD, PhD Program Director- Clinical Effectiveness Research dhickam@pcori.org

Actively Recruiting, Program Director- Communication and Dissemination @pcori.org

Chad Boult, MD, MPH, MBA Program Director- Improving Healthcare Systems cboult@pcori.org

Lori Frank, PhD Program Director- Research Integration and Evaluation lfrank@pcori.org

Find Us Online

www.pcori.org

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