PAT IE N T- C E N T E R ED O U TC O ME S R E SE A RC H I N ST IT U T E

JAMA Media BriefingNational Press Club, Washington, DCApril 17, 2012

Anne C. Beal, MD, MPHCOO, Patient-Centered Outcomes Research Institute (PCORI)

Setting PCORI’s Priorities – How the Patient-Centered Outcomes Research Institute Plans to Make a Difference

PAT IE N T- C E N T E R ED O U TC O ME S R E SE A RC H I N ST IT U T E

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 Purpose: Defining Legislation

PAT IE N T- C E N T E R ED O U TC O ME S R E SE A RC H I N ST IT U T E

PCORI MISSION STATEMENT• 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.

PCORI Mission

PAT IE N T- C E N T E R ED O U TC O ME S R E SE A RC H I N ST IT U T E

Why PCORI?

• Patients’ individual needs, values and concerns have not been an “outcome measure” in traditional research.

• Patients who face a range of treatment options, especially for serious conditions, want clinicians to help them figure out what different choices mean for them or people like them.

• Clinicians often lack the high-quality evidence they could use to advise patients making complex clinical decisions.

• Even the best traditional clinical effectiveness research has not had this “patient-centric” focus.

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PAT IE N T- C E N T E R ED O U TC O ME S R E SE A RC H I N ST IT U T E

A Board Broadly Representing the Health Care Community

• Patients• Caregivers • Physicians, nurses and clinicians• Hospitals and health systems• Health plans• Health services researchers • State and federal health officials • Pharmaceutical, device, and

diagnostic manufacturers • Private payers • Employers 5

PAT IE N T- C E N T E R ED O U TC O ME S R E SE A RC H I N ST IT U T E

What is patient-centered outcomes research?*

Complements traditional CER; answers patient-centered questions like:

• “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?”

*From PCOR definition adopted by PCORI Board of Governors, March 5, 2012

PAT IE N T- C E N T E R ED O U TC O ME S R E SE A RC H I N ST IT U T E

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

Making Outcomes Research Patient-Centered

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PAT IE N T- C E N T E R ED O U TC O ME S R E SE A RC H I N ST IT U T E

PCORI seeks to do its work by:

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• Establishing national research priorities

• Establishing and carrying out a research agenda

• Developing and updating methodological standards

• Disseminating research findings• Engaging patients, caregivers, clinicians and other

stakeholders as its work evolves

PAT IE N T- C E N T E R ED O U TC O ME S R E SE A RC H I N ST IT U T E

PCORI’s Path from Priorities to Research Patients Can Use

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Future priorities and agendas will be informed by the development process, ongoing stakeholder engagement, and PCORI’s research results.

PAT IE N T- C E N T E R ED O U TC O ME S R E SE A RC H I N ST IT U T E

Criteria for Research Outlined by Law

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Impact on Health of Individuals and

Populations

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

PAT IE N T- C E N T E R ED O U TC O ME S R E SE A RC H I N ST IT U T E

Establishing PCORI’s First National Priorities for Research and Initial Research Agenda

9criteria

outlined by law

5draft priorities

proposed

Corresponding agenda drafted

Public input received and

evaluated

Priorities and agenda revised and approved

First primary funding

announcements issued

Aug-Dec 2011 Jan-Apr 2012 May 2012

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PAT IE N T- C E N T E R ED O U TC O ME S R E SE A RC H I N ST IT U T E

1. Comparing Preventive,

Diagnostic and Treatment Options

  Better Decisions

  Better Outcomes

Better Information Better Decisions

??

National Priorities for Research

PAT IE N T- C E N T E R ED O U TC O ME S R E SE A RC H I N ST IT U T E

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

National Priorities for Research

PAT IE N T- C E N T E R ED O U TC O ME S R E SE A RC H I N ST IT U T E

A rigorous methodological basis for PCOR

• To do CER and PCOR properly, methods matter.• PCORI’s Methodology Committee is established by statute.• Committee is charged with developing and periodically updating

methodological standards for CER/PCOR. • Committee is required to deliver first report to PCORI’s Board in

early May. – prioritizing research questions– using appropriate study designs– incorporating patient perspectives throughout the research

continuum– fostering efficient dissemination and implementation of

results.

PAT IE N T- C E N T E R ED O U TC O ME S R E SE A RC H I N ST IT U T E

A commitment to public engagement

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Formal 53-day Public Comment Period

• Nearly 500 comments received through website, e-mail or postal mail

• All comments will be posted at pcori.org

Additional Forums• National Patient and Stakeholder Dialogue • Patient, caregiver and clinician focus groups • Individual meetings with diverse mix of

stakeholders

PAT IE N T- C E N T E R ED O U TC O ME S R E SE A RC H I N ST IT U T E

Patient and Caregiver Focus Groups

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Columbus, OH (Dec. 7)• Parents of children with ADD/ADHD

(caregivers)• Patients with Mental Health

conditions• Patients who survived Cancer

• Insured and uninsured• Age: 30-70+

Phoenix, AZ (Dec. 8)• Patients with Respiratory Disease

(chronic bronchitis, emphysema)• Hispanic patients with mix of

chronic conditions• Patients with Heart Disease

• Insured and uninsured• Age: 21-69

Atlanta, GA (Nov. 21)• Patients with Diabetes• Caregivers to Alzheimer’s patients• Patients with Chronic Pain

• Insured and uninsured• Age: 21-75+

Baltimore, MD (Nov. 9)• Patients with Arthritis• Parents of children with Pediatric

Asthma (caregivers)• Patients with mix of chronic

conditions• Insured and uninsured• Age: 21-69

PAT IE N T- C E N T E R ED O U TC O ME S R E SE A RC H I N ST IT U T E

Receiving Public Feedback on the National Priorities and Research Agenda

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• Clinician Focus Groups

o Clinician focus groups took place in cities across the USo Philadelphiao Birminghamo Californiao Chicago

o Four groups of physicians and four groups of nurseso Behavioral Health Professionalso Physician Assistants

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Next Steps• Analysis of public comment received on Priorities and AgendaMarch-April 2012• Adoption of first National Priorities for Research and Research AgendaApril 2012• First Methodology Report posted for public commentMay 2012

• Broad Funding Announcements IssuedMay 2012• Patient and Stakeholder Recruitment and Training for Research ReviewJune 2012• Broad Funding Applications DueJuly 2012• Advisory Groups Formed to Advance Agenda SpecificitySummer 2012• Targeted Funding Announcements IssuedAugust 2012• Broad Funding Awards AnnouncedDecember 2012

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