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Patient-Centered Outcomes Research Institute

Joe V. Selby, PCORI Executive Director May 1, 2012

From Populations to Patients

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Imagine My Surprise!

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

“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 concerning the manner in which diseases, disorders, and other healthconditions can effectively and appropriately be prevented, diagnosed, treated, monitored, and managed 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 appropriatenessof medical treatments, services.

Purpose

PCORI is a Research Institute

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

‘‘(2) COMPARATIVE CLINICAL EFFECTIVENESS RESEARCH; (A) IN GENERAL.—The terms ‘comparative clinical effectiveness research’ and ‘research’ mean research evaluating and comparing health outcomes and the clinical effectiveness, risks, and benefits of 2 or more medical treatments, services, and items described in subparagraph (B).

Purpose

PCORI is a Research Institute

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PCORI MISSION STATEMENT (adopted July 17, 2011)

The Patient-Centered Outcomes Research Institute (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.

Taking Patient-Centeredness Seriously

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Defining Patient-Centered Outcomes Research

Patient Centered Outcomes Research (PCOR) helps people and their caregivers communicate and make informed health care decisions, allowing their voices to be heard in assessing the value of health care options. This research answers patient-centered questions such as:

“Given my personal characteristics, conditions and preferences:

1. What should I expect will happen to me?

2. What are my options and what are the potential benefits and harms of those options?

3. What can I do to improve the outcomes that are most important to me?

4. 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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Understanding the choices

patients face

Aligning research questionsand methods with

patient needs

Providing patients and providers with information

for better decisions

Patient EngagementPatient-Driven

Research Dissemination

Taking Patient-Centeredness Seriously

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Taking Patient-Centeredness Seriously

• Patients and other relevant stakeholders must be included as members of research teams and involved in all aspects of the project - from planning to dissemination

• Applications are evaluated and scored on patient and stakeholder engagement in the review process

• At least 3 trained patient/stakeholder reviewers serve on each review committee

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PCORI National Patient and Stakeholder Dialogue

Feb 27, 2012

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PCORI Hopes to Turn the Research World On Its Head

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Yes!! Including the patient and clinician perspectives in PCOR help to ensure that:

The research questions are genuinely practical

The range of outcomes important to patients are identified and studied

The patients studied are representative of those who require information and come from relevant settings

Possibilities of heterogeneity in effectiveness are carefully examined in various patient subgroups.

Is PCOR still CER?

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The Population and The Patient

The Population

Patients

Patients

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

“Research shall be designed, as appropriate, to take into account the potential for differences in the effectiveness of health care treatments, services, and items as used with various subpopulations, such as racial and ethnic minorities, women, age, and groups of individuals with different comorbidities, genetic and molecular sub-types, or quality of life preferences and include members of suchsubpopulations as subjects in the research as feasible and appropriate.

Purpose

The Legislation re: Treatment Heterogeneity

Treatment Heterogeneity – Type One

HeterogeneousPopulation

• Biomarker• Comorbidity• Literacy

Marker Positive:

Net Benefit

Marker Negative

Net Harm

Therapy withDifferential

Effectiveness

andnon-differential adverse effects

Treatment Heterogeneity – Type Two

Therapy withNon-differential

Effectiveness

And Non-differential Adverse Effects

HeterogeneousPopulation

High Risk

Medium Risk

Low Risk

Outcomes

High Risk:Net Benefit

Medium Risk:No Net Benefit

Low Risk:Net Harm

Heterogeneity in Treatment Effectiveness

Patient Population Intervention Marker

Breast Cancer Trastuzumab HER-2 Expression

Atrial fibrillation Warfarin VKORC1, CYP2C9

Symptomatic Ischemic HD

Coronary Bypass Surgery

Age, major Comorbidity

Type 2 Diabetes eHealth Education Literacy/Numeracy

What Works Best – For Whom?

• Identifying new markers to guide therapy• Biology-driven• Empirical: Re-examining clinical trials data

Evidence synthesesLarge EHR database studies Larger trials

• Focus on developing treatments and approaches for those who are NOT responding well to current therapies

• In the face of imperfect accuracy and ongoing uncertainty, how do we incorporate patient preferences for USING these markers to guide treatment decisions?

PCORI’s

• National Priorities for Research

• Research Agenda

• Funding Announcements

• PCORI Pilot Projects

• Primary Research Funding Announcements

The Funding

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National Priorities and Research Agenda

Incre

asin

g S

pecifi

city

National Priorities and Research Agenda Must be approved before major funding can begin

National Priorities Research Agenda

Individual PFAs

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Building on Other Recent Prioritization Efforts

Source

Preventio

n

Acute

Care

Chronic

Disease

Care

Palliative Care

Care Coordination

Patient

Engagement

Safety

Appropria

te Use

HIT to

improve pt.

experienc

e

Impact of New Technolo

gy

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

√ √ √ √ √ √20

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1. Comparing Preventive,

Diagnostic and Treatment Options

2. Improving Health Care Systems  Better Decisions

 Better Outcomes

5. Accelerating PCOR and Methodological Research- Data- Training- Methods

4. Addressing Disparities

3. Communication/ Dissemination Research

Producing and delivering information to support better health care decisions by individuals

PCORI’s National Priorities for Research

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Comparisons of alternative clinical options Identifying patient differences in response to therapyStudies of patient preferences for various outcomes

Improved support of patient self-managementCoordination of care for complex conditions Improvements in the effectiveness and efficiency of care Improved workforce deployment

Understanding and enhancing shared decision-makingAlternative strategies for dissemination of evidence

Alternative interventions/strategies to eliminate disparities Improvements in alignment of decisions with preferences

Improving study designs and analytic methods of PCORBuilding and improving clinical data networks Methods for training researchers, patients to participate in PCORFacilitating the study of rare diseases

Addressing Disparities

Improving Healthcare Systems

Accelerating PCOR and

Methodological Research

Communication & Dissemination

Research

Assessing Options for Prevention, Diagnosis, and

Treatment

PCORI’s National Priorities for Research

• Public Comment PeriodJanuary - March, 2012

• Analysis of public comment and revisionMarch-April 2012

• Approval of revised National Priorities / Research AgendaApril 25, 2012

May 2012

July 2012

December 2012

July 2012

December 2012

Next Steps:

National Priorities and Research Agenda

Funding

Stakeholder Engagement

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Building Infrastructure for a National Learning Healthcare System

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National Learning Health System

FDA

SentinelOMOP

ONC

Meaningful UseNationwide Health Information Network Federal Health ArchitectureEHR Certification programsStandards & Interoperability Framework

NIH

CTSA’sCollaboratoryCRN, CVRN

AHRQ

DRN’sRegistriesSPANPROSPECTEDM Forum

VA

Industry

SpecialtySocieties

PCORI

IOM

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• Research on the validity and efficiency of data sources that can be used to create a national infrastructure for PCOR;

– improving the volume, completeness, comprehensiveness, accuracy, or efficiency of use of clinical data collected across health care systems, clinical data networks, registries, or payer databases;

– Supporting the routine collection of key patient-reported and patient-centered outcomes

– Engaging patients in PCOR (governance, research prioritization, research participation, dissemination)

– Engaging front-line clinicians in generating research questions, disseminating findings?

Priority #5 Accelerating PCOR and Methodologic Research

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• A clearer long-term national vision and strategy for CER/PCOR

• Re-vamped notions of governance, with greater willingness of organizations to participate and contribute

• More research on data standards, inter-operability, data quality and comparability

• Advances in methods of data storage and analyses

• Greater engagement of patients, clinicians in governance, and as end-users

• Ability to contact patients, and to randomize

What Would it Take?

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Re-examining Ethics of Research in a Patient-Centered World

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Re-examining Ethics

Historical Backdrop:

• Belmont Report (1978): draws bright line between research and clinical care

• different ethical standards and oversight applied to research than to practice.

Current Understanding:• Much of medicine still

practiced without good evidence, exposing patients to avoidable risks;

• with advent of EHRs, other computerized data, health systems and clinicians generate and hold key data for increasing the evidence.

Near Future?• research does

become more patient-centric, addressing questions patients and clinicians raise, with patients participating in the research

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Re-examining Ethics: Questions

Physician and System Perspective: Is it ethical for clinicians and health systems NOT to participate in patient-centered research? Is it ethical to continue practicing with uncertainty and NOT put the data generated in this practice to research use.

Patient Perspective: Is it ethical (or reasonable) for patientsNOT to at least strongly consider participation in research as subjects, lending clinical data with appropriate safeguards and participating in appropriate clinical trials.

IRB Perspective: Given a change to patient-centered research, and the recognized need for representative, real world data, including both HER data and broad participation in clinical trials, do we need to re-think aspects of IRB policy and practice.

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A Research World on Its Head

• Patients and clinicians generating and prioritizing research questions, reviewing research applications, joining teams in conduct of research

• Analytic strategies based on finding differences in treatment response, focusing on patients not yet responding, and aiming to optimize treatments for each patient

• Health care systems participating in a national learning health system based on safety and patient-centered outcomes research

• Ethics re-cast to recognize the ethical imperatives of participation – with appropriate protections – by patients, clinicians and systems

• Growing body of relevant evidence, growing appreciation for research as a national endeavor to improve decision-making and outcomes

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QuestionsComments

….thanks!