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Developing PCORI’s National Priorities for Research and First Research Agenda

PCORI Board of Governors January 2012

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

“Assist patients, clinicians, purchasers,

and policy-makers in making informed

health decisions by advancing the quality

and relevance of evidence…[relevant to]

the manner in which diseases, disorders

and other health conditions can

effectively and appropriately be

prevented, diagnosed, treated,

monitored, and managed through

research and evidence synthesis.”

Purpose

PCORI Purpose: Defining Legislation

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

National Priorities for Research

“The Institute shall identify national priorities for research, taking into account factors of disease incidence, prevalence, and burden in the United States (with emphasis on chronic conditions), gaps in evidence in terms of clinical outcomes, practice variations and health disparities in terms of delivery and outcomes of care, the potential for new evidence to improve patient health, well-being, and the quality of care, the effect on national expenditures associated with a health care treatment, strategy, or health conditions, as well as patient needs, outcomes, and preferences, the relevance to patients and clinicians in making informed health decisions, and priorities in the National Strategy for quality care established under section 399H of the Public Health Service Act that are consistent with this section.”

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Research Agenda

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

“The Institute shall establish and update a research project agenda for research to address the priorities identified under subparagraph (A), taking into consideration the types of research that might address each priority and the relative value (determined based on the cost of conducting research compared to the potential usefulness of the information produced by research) associated with the different types of research, and such other factors as the Institute determines appropriate.”

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Stakeholder Input (surveys, focus groups, public

comment, stakeholder meetings, etc.)

Environmental Scans

Pilot Project Applications

Pilot Project Results

PCORI Board

Methodology Committee

PCORI National Priorities and Research Agenda Versions 1

Solicitation

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Generating Greater Specificity: Inputs

Legislative Statute

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In

creasin

g S

pecific

ity

National Priorities and Research Agenda: An Iterative Feedback Process

Inputs • Legislative Statute • Board of Governors • Program Development Committee • Methodology Committee • Environmental Scans • PCORI Pilot Projects • Stakeholder Input and Feedback

Research Agenda

Version 1

National Priorities Version 1

Individual PFAs

Version 1

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We’re on a Journey!

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Task Aug Sept Oct Nov Dec Jan Feb Mar Apr May

NATIONAL PRIORITIES

Develop Framework

Develop Stakeholder Engagement Process

Dialogue w/ Stakeholders

Design and implement web-based survey

Public Comment

Incorporate Public Comment

RESEARCH AGENDA

Environmental Scan for RA Development

Draft Research Agenda

Dialogue w/ Stakeholders

Public Comment

Incorporate Public Comment

2011 2012

Nat’l Priorities v1.0

Research Agenda v1.0

Timeline for Development of National

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National Priorities

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Environmental scan of existing priorities and

criteria

Candidate priorities and

criteria identified

Framework to inter-relate

Priorities and Criteria

Reviewed initial stakeholder input advising us to not “reinvent the wheel”

Reviewed prior CER frameworks (e.g., IOM, FCCCER, National Priorities Partnership, NQF)

Identified broad priorities from prior frameworks and the statutory criteria for PCORI

Developed Framework to be used for refining priorities and for determining Research Agenda and funding announcements

Initial Stakeholder

feedback

Development of a National Priorities Framework

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Environmental Scan

Source P

reve

nti

on

Acu

te C

are

Ch

ron

ic

Dis

eas

e C

are

Pal

liati

ve C

are

Car

e

Co

ord

inat

ion

Pat

ien

t En

gage

me

nt

Safe

ty

Ap

pro

pri

ate

U

se

HIT

to

imp

rove

p

t. e

xpe

rie

nce

Imp

act

of

Ne

w

Tech

no

logy

IOM 2009: Priorities for CER √ √ √ √ √ √ √ √ √

Federal Coordinating Committee for CER

√ √ √ √ √

AHRQ National Quality Strategy

√ √ √ √

AHRQ Effective Health Care Program

√ √ √ √ √ √

National Quality Forum √ √ √ √ √ √ √ √

National Prevention Council √ √

National Priorities Partnership

√ √ √ √ √ √

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12

Health Information

Technology

Prevention &

Screening

Acute Care

Impact of New

Technology

PCORI Draft National Priorities

Appropriate Use

Frequently Cited Priority Areas

Chronic Disease Care

Palliative Care & Pain

Management

Care Coordination

Patient Engagement

Safety

Addressing

Disparities

Accelerating PCOR

and Methodological

Research

Communication and

Dissemination

Research

Improving Health

Care Systems

Comparative Assessment

of Options for Prevention,

Diagnosis, and Treatment

Consideration of Criteria in

Statute

Fits the Definition of PCOR

Making PCORI Priorities Patient-Centered

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Patient-Centered Priorities

Improving

Healthcare

Systems

Comparing healthcare system-level approaches to improving access, supporting patient self-care, innovative use of health information technology, coordinating care for complex conditions, and deploying workforce effectively.

Comparative

Assessment of Options

for Prevention,

Diagnosis, and

Treatment

Comparing the effectiveness and safety of alternative preventive, diagnostic and treatment options.

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Addressing

Disparities

Identifying potential differences in treatment effectiveness or preferred clinical outcomes across patient populations and the health care required to achieve best outcomes in each population.

Communication &

Dissemination

Research

Comparing approaches to providing CER information and supporting shared decision-making between patients and their providers.

Accelerating

PCOR and

Methodological

Research

Improving the nation’s capacity to conduct patient-centered outcomes research, by building data infrastructure, improving analytic methods, training researchers, patients and other stakeholders.

Patient-Centered Priorities (cont’d)

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Developed over 5-month period

Built from prior prioritization efforts

Stakeholder input incorporated along the way

First Draft now ready for public comment

Can be modified based on public comment, or thereafter through a transparent process of stakeholder engagement

PCORI’s Draft National Priorities

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Research Agenda

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Impact on Health of Individuals and

Populations

PCORI Criteria – From Legislation

Improvability through Research

Inclusiveness of Different Populations

Addresses Current Gaps in

Knowledge/Variation in Care

Patient-Centeredness

Impact on Health Care System Performance

Potential to Influence Decision-Making

Rigorous Research Methods

Efficient Use of Research Resources

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Accelerating PCOR

and

Methodological

Research

• Impact on Health of Individuals

and Populations

• Improvability via Research

• Inclusiveness of Different Populations

• Addresses Current Gaps in Knowledge/Variation

in Care

• Impact on Health Care System Performance

• Potential to Influence Decision Making

• Patient-Centeredness

• Rigorous Research Methods

• Efficient Use of Research

Resources

Addressing

Disparities

Improving

Healthcare

Systems

Communication

& Dissemination

Research

Priorities Comparative Assessment

of Options for

Prevention, Diagnosis,

and Treatment PCORI Criteria

PCORI

Research

Agenda

Framework for Developing PCORI Research Agenda from National Priorities

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...compare situations in which the effectiveness of strategies for prevention, treatment, screening, diagnosis, or surveillance have not been adequately studied against alternative options where better evidence is needed to support decision-making by patients, caregivers, and health care professionals.

Research Agenda Item From a National Priority

Comparative

Assessment of

Options for

Prevention,

Diagnosis, and

Treatment

PCORI Criteria

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Improving

Healthcare

Systems

Research Agenda Item From a National Priority

…compares alternative system-level approaches to supporting and improving patient access to care, receipt of appropriate care, coordination of care across health care services or settings for patients with complex chronic conditions, and personal decision-making and self-care.

PCORI Criteria

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Alternative clinical options

Patient differences in response to therapy

Patient preferences for various outcomes

Alternative system interventions

Improvements in the effectiveness and efficiency of care

Patient-centeredness

Improvements in shared decision-making

Alternative strategies for dissemination of evidence

Alternative interventions/strategies to eliminate disparities

Improvements in alignment of decisions with preferences

Improvements study designs and analytic methods of PCOR

Building and improving clinical data networks

Better methods for training researchers, patients to participate in PCOR

Facilitating the study of rare diseases

Addressing

Disparities

Improving

Healthcare

Systems

Accelerating

PCOR and

Methodological

Research

Communication

& Dissemination

Research

Comparative

Assessment of Options

for Prevention,

Diagnosis, and

Treatment

Content of PCORI Research Agenda

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Promote patients and their caregivers –and key stakeholders in implementation settings—as partners, with explicit roles in the design, governance, review, and dissemination of research.

Seek to understand core questions from the expressed perspective of the patient and their caregivers.

Emphasize open and transparent science that involves participants in decisions about making data available for further study, seeking to ensure that the research produces as much new investigative activity as possible and that sharing of information and knowledge among diverse investigators is required.

Select Features of PCORI Research Agenda

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Commit to a diverse research portfolio with respect to patients, geography, health care professionals, investigators, and organizations, seeking to catalyze activity across a broad range of patients, sites, conditions, and questions.

Emphasize knowledge that is likely to make a positive difference in the lives of patients and their caregivers and is suitable for dissemination and application; and emphasize outcomes that are important to patients and their caregivers and likely to be useful in their decision-making.

Emphasize ideas that emerge from the community of patients, caregivers, clinicians and researchers, seeking to listen and learn from the wisdom of those whose lives are most affected by these conditions and those who are committed to generating new knowledge that will promote better decisions and outcomes.

Features of PCORI Research Agenda

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Developed over 4-month period

Stakeholder input incorporated

First draft ready for public comment

Will be modified and expanded thereafter through a transparent process of stakeholder engagement

PCORI’s Draft Research Agenda

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Proposed initial agenda is specific with respect to study questions that will be supported, but not to the conditions (or treatments) that can be studied

This non-specificity allows for a flexibility in the search for the best opportunities

Continuing input from stakeholders will contribute to refinement of research agenda over time

PCORI’s Draft Research Agenda