11TH ANNUAL MEETING OF ISMPP 1

1 1 T H A N N U A L M E E T I N G O F I S M P P

OPTIMIZING SCIENTIFIC VALUE: SMART AND SYSTEMATIC APPROACHES

TO MEDICAL PUBLICATIONS April 27–29, 2015

Hyatt Regency Crystal City Arlington, VA, USA

Jean Slutsky Chief Engagement and Dissemination Officer and Program Director, Communication and Dissemination Research ISMPP Crystal City, VA 27 April 2015

Optimizing Patient-Centered Outcomes Research

About PCORI

About PCORI

An independent research institute authorized by Congress in 2010. Governed by a 21-member Board representing the entire healthcare community.

Funds comparative clinical effectiveness research (CER) that engages patients and other stakeholders throughout the research process.

Seeks answers to real-world questions about what works best for patients based on their circumstances and concerns.

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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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.

Our Mission

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PCORI’s Obligation Under its Authorizing Legislation Conduct Peer Review of Primary Research

• Assess scientific integrity • Assess adherence to PCORI’s Methodology Standards

Release of Research Findings • No later than 90 days after “conduct or receipt” • Make available to clinicians, patients, and general public • Make comprehensible and useful to patients and providers for healthcare

decisions • Include considerations specific to certain sub-populations, risk factors, and

comorbidities • Describe process and methods, including conflicts of interest • Include limitations and further research needed

Research We Support

What Types of Research Does PCORI Support From the Authorizing Legislation:

“The terms ‘comparative clinical effectiveness research’ and ‘research’ mean research evaluating and comparing health outcomes and the clinical effectiveness, risks, and benefits of two or more medical treatments, services, and items…”

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Compares two or more options for prevention, diagnosis, or treatment (can include “usual care”)

Considers the range of clinical outcomes relevant to patients

Conducted in real-world populations and real-world settings

Attends to differences in effectiveness and preferences across patient subgroups

Often requires randomized trial design

What is Comparative Effectiveness Research?

Helps people and their caregivers communicate and make better-informed healthcare decisions.

Actively engages patients and key stakeholders throughout the research process.

Compares effectiveness of important clinical management options.

Evaluates the outcomes that are the most important to patients.

Addresses implementation of findings in clinical care environments.

What is Patient-Centered Outcomes Research?

Pivotal $100M Infrastructure Investment

Provides technical and logistical assistance under the direction of a steering committee and PCORI program staff

System-based networks, such as integrated delivery systems, academic medical centers, federally qualified health centers, 18 Patient-Powered Research Networks (PPRNs) Patients with a condition in common form a research network, often in collaboration with academic researchers

11 Clinical Data Research Networks (CDRNs)

Coordinating Center

PCORnet Goals

Hallmarks of PCORnet’s success will include the following goals: 1. Highly engaged patients, caregivers, and researchers participate in network

governance and research topic generation

2. Analysis-ready standardized data, use of the PCORnet Common Data Model, and preserving strong privacy and data-security protections

3. An infrastructure for supporting clinical trials embedded within network delivery systems and patient communities

4. An oversight framework that fosters public trust in research

5. A collaborative community that attracts a diverse set of researchers, funders, and other networks

6. Research networks that are sustainable

Engaging Patients in Research

Why Engage Patients in Research?

Patient engagement in the design and conduct of research offers a greater likelihood of:

• influencing research to be patient centered, useful, and relevant

• establishing trust and a sense of legitimacy in its findings • successful use and uptake of research results by the

patient community.

Why Engage Patients in Research?

Patients contribute:

“Lived experience” of condition or disease

Research questions that are important to patients

Outcomes important to patients such as quality of life

Real world input on risk tolerance of interventions

Power of data (new currency)

Focus on patient-centeredness, ethics, safety, and urgency Connections to other patients like them, which facilitates recruitment and dissemination of research findings

We Fund Research That…

Studies the benefits and harms of interventions and strategies delivered in real-world settings

Compares at least two alternative approaches

Adheres to PCORI’s Methodology Standards

Is based on health outcomes that are meaningful to the patient population Engages patients and other stakeholders at every stage Is likely to improve current clinical practices

Supporting Engagement as a Tool to Advance Research

and Dissemination of Findings

Engagement Goals

Promote dissemination and implementation of PCOR research findings

Engage the PCOR community in research

Build a patient-centered outcomes research, or PCOR, community

Evaluation

ENGAGEMENT

Engagement as a Path To Useful, High-Quality Research

Proposal Review; Design and Conduct of Research

Topic Selection and Research Prioritization

Dissemination and Implementation of Results

We Require Patient-Centeredness and Patient and Stakeholder Engagement

Patient and Stakeholder Engagement Patients are partners in research, not just “subjects”

Active and meaningful engagement between scientists, patients, and other stakeholders

Community, patient, and caregiver involvement already in existence or a well-thought out plan

Patient-Centeredness Does the project aim to answer questions or examine outcomes that matter to patients within the context of patient preferences?

Research questions and outcomes should reflect what is important to patients and caregivers

In Summary:

Comparative Effectiveness Research (CER) should be a public good that: o Gives health care decision-makers – patients,

clinicians, purchasers and policy- makers – access to the latest open and unbiased evidence-based information about treatment options

o Informs choices and is closely aligned with the sequence of decisions patients and clinicians face

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In Summary

Patient-Centered Outcomes Research involves patients but also other participants in health care decision making Involving patients and stakeholders in the beginning will increase the chances of rapid dissemination and uptake Meaningful engagement is challenging but very rewarding Patient engagement in research is necessary to ensure the trust and relevance of PCOR

Find Us Online

www.pcori.org

11TH ANNUAL MEETING OF ISMPP 25

1 1 T H A N N U A L M E E T I N G O F I S M P P

OPTIMIZING SCIENTIFIC VALUE: SMART AND SYSTEMATIC APPROACHES

TO MEDICAL PUBLICATIONS April 27–29, 2015

Hyatt Regency Crystal City Arlington, VA, USA