PCORI in Practice: Highlighting Opportunities for Medical Specialty - - PowerPoint PPT Presentation

PCORI in Practice: Highlighting Opportunities for Medical Specialty Societies September 11, 2014

Welcome and Introductions

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We welcome your questions and comments via the chat function

• n the right side of your screen

We welcome your comments via Twitter to @PCORI and #PCORI

Agenda for today’s webinar

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Time Agenda Item Speakers 1:00 - 1:10 p.m. Welcome and Introductions Norman B. Kahn, Jr., MD, Executive Vice President and CEO, Council of Medical Specialty Societies 1:10 - 1:30 p.m. Background on PCORI and Exploring our Portfolio Joe V. Selby, MD, MPH, Executive Director, PCORI 1:30 – 1:45 p.m. Funding Opportunities: Large Pragmatic Studies to Evaluate Patient-Centered Outcomes Joe V. Selby, MD, MPH, Executive Director, PCORI 1:45 – 2:00 p.m. Question and Answer Session Moderated by PCORI and CMSS Staff 2:00 p.m. Adjourn

Goals for Today

Familiarize the community of medical specialty societies with PCORI and PCORnet Engage the community of medical specialty societies more actively in PCORI’s work and funding, especially the Pragmatic Clinical Studies Encourage the community of medical specialty societies to activate their research capacity for conducting patient- centered comparative effectiveness research

4 #PCORI

Background on PCORI and Exploring our Portfolio

Joe V. Selby, MD, MPH, Executive Director, PCORI

Patient-centeredness is at the heart of PCORI’s name, and of its mission and vision

• An independent, non-profit health research organization authorized by the Patient

Protection and Affordable Care Act of 2010.

• PCORI funds patient-centered research to assist patients, caregivers, and other

stakeholders in making informed health decisions. Mission PCORI helps people make informed healthcare decisions and improves healthcare delivery and outcomes by producing and promoting high integrity, evidence-based information that comes from research guided by patients, caregivers, and the broader healthcare community. Vision Patients and the public have the information they need to make decisions that reflect their desired health

• utcomes.

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Why PCORI?

Research tends not to address many of the practical questions faced by patients, caregivers and clinicians People often need to know which treatment is better for them, given their characteristics and their personal preferences This kind of research is best done in “real world” settings

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Engagement: A Critical Path to Useful, High-Quality Research

Evaluation Proposal Review; Design and Conduct

• f Research

Topic Selection and Research Prioritization Dissemination and Implementation of Results

Research We Support

pcori.org/research-we-support

Key Features of Our Funded Research

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

We Pay Particular Attention to…

Conditions that heavily burden individual patients, families, the health care system and/or society Chronic or multiple chronic conditions Rare and understudied conditions Conditions for which outcomes vary across subpopulations

Our Growing Research Portfolio

pfaawards.pcori.org

Funded Projects to Date

Total number of research projects awarded : 313 Total funds awarded: $549 million Number of states where we are funding research: 38 states (plus the District of

Columbia and Quebec, Canada)

Snapshot of Funded Projects

Improve the nation’s capacity to conduct clinical research more efficiently, by creating a large, representative, national patient- centered clinical research network with a focus on conducting comparative effectiveness studies – both randomized and

• bservational.

Support a learning US healthcare system, which would allow for large-scale research to be conducted with enhanced accuracy and efficiency within real-world care delivery systems.

The National Patient-Centered Clinical Research Network (PCORnet)

PCORnet

System-based networks, such as hospital systems $76.8 million awarded

18 Patient-Powered Research Networks (PPRNs)

Patients with a single condition form a research network $16.8 million awarded

11 Clinical Data Research Networks (CDRNs) Coordinating Center

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

Geographic Coverage of PPRNs and CDRNs

This map indicates how many of the 18 PPRNs and 11 CDRNs have a presence in each state and territory.

PCORnet’s 18 Month Aim – End of Phase I

PCORnet will bring together the expertise, populations, resources, and data of its participating organizations to create a national infrastructure that enables more efficient, patient-centered clinical

• research. Hallmarks of PCORnet include:
• Highly engaged patients, clinicians, health systems, researchers and
• ther partners
• A collaborative community across networks
• Analysis-ready standardized data with strong privacy and data

security protections

• Research oversight that protects patients, supports the timely

conduct of research, and builds trust in the research enterprise

• Research that is integrated into care settings and with communities
• f patients

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PCORnet’s 4 Year Aim – End of Phase II

PCORnet will be recognized as a national research infrastructure for conducting rapid, efficient, patient-centered observational and interventional research that improves healthcare delivery and health

• utcomes. Hallmarks include:
• Highly engaged patients, researchers, clinicians, health systems, and

the public participate in network governance and topic generation

• Greatly expanded analysis-ready standardized data, preserving strong

privacy and data security protections

• An efficient oversight framework that fosters public trust in research
• A collaborative community that attracts a diverse set of researchers,

funders and other networks

• Research that is integrated into care settings and with communities of

patients, with research findings that transform care

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Potential Roles of Medical Specialty Societies with PCORnet

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Joining with PCORnet researchers to create a new registry within PCORnet or to expand an existing registry by adding additional registry members and data from PCORnet Linking an existing registry with PCORnet data to obtain outcomes and other longitudinal data not captured routinely by the registry Collaborating with PCORnet to conduct a CER study within PCORnet Collaborating with PCORnet to develop data standards for identifying cases from electronic data or disease-specific patient-reported

• utcomes measures

New Funding Opportunity: Large Pragmatic Studies to Evaluate Patient-Centered Outcomes

Pragmatic Clinical Studies

Available Funds and Duration:

• A total of $90 million

(direct + indirect) each cycle

• 2 cycles per year
• Up to $10 million in

direct costs per project

• Projects should be

completed within 5 years

Objectives of this Initiative:

Address critical clinical and health-related comparative effectiveness questions faced by patients, their caregivers, and their clinicians Strong involvement of medical specialty and patient organizations required.

PCORI seeks to fund:

Pragmatic clinical trials Large simple trials Large scale observational studies

Essential characteristics of studies

Directly compare two or more clinical options (head to head comparison) Aim to:

• Examine prevention, diagnosis, treatment, options
• Improve performance of healthcare systems
• Eliminate health or healthcare disparities

Take place within typical clinical care and community settings Sample sizes large enough for precise effect estimates and for detecting subgroup differences in treatment effectiveness Measure health outcomes meaningful to patients

Comparators of interest

Specific drugs, devices, and procedures Medical and assistive devices and technologies Techniques for behavioral modification Complementary and alternative medicine Delivery-system re-structuring or innovations Usual care or no specific intervention, if these are realistic choices for patients (e.g., choosing not to have a cancer screening, a diagnostic test, or a surgical procedure)

Sources for high-priority topics

PCORI priority topics (see following slides) IOM 100 priority topics for CER AHRQ Future Research Needs Projects Investigator initiated topics will also be considered. Researchers must make a strong case for the importance of the topic.

PCORI Priority Topics

Medical versus invasive procedures for asymptomatic carotid artery disease Surgical options for hip fracture in the elderly Comparative effectiveness and safety of pelvic floor mesh implants Diagnosis and management of bipolar disorder in children and adolescents Management of ductal carcinoma in situ (DCIS) of the breast Strategies for preventing the progression of episodic acute back pain into chronic back pain

PCORI Priority Topics

Strategies for follow-up of pulmonary nodules identified by imaging studies (e.g., chest CT) Treatment options for people with opioid substance abuse Treatment options for patients with multiple sclerosis Proton beam therapy for patients with lung, breast, and prostate cancer Biologic agents in the management of patients with Crohn’s disease

PCORI Priority Topics

Treatment strategies for adults with migraine headaches Treatment strategies for symptomatic osteoarthritis, including timing of joint replacement Treatment strategies for patients with autism spectrum disorder Integration of mental and behavioral health services into the primary care of the general population Integration of mental and behavioral health services into the primary care of persons at risk for disparities in health care and outcomes

PCORI Priority Topics

Reduction of cardiovascular disease (CVD) risk in underserved populations such as racial and ethnic minorities and those living in rural communities Effectiveness of innovative strategies for enhancing patients’ adherence to medication regimens Effectiveness of specific features of health insurance

• n access to care, use of care, and other outcomes

that are especially important to patients

Roles of Medical Specialists in These Studies

Identifying and refining relevant, useful research questions Designing pragmatic studies that allow conduct within routine clinic/office operations and minimize disruption to participants’ daily routines Helping to capitalize on existing electronic health records and other computerized information

• to identify and recruit eligible patients
• to monitor study conduct and patient safety
• to collect study outcomes information

Disseminating appropriate study findings

How to Apply for Funding

Submit letter of intent (LOI)

• Next deadline is 10/01/2014

If LOI is approved, full proposal can be submitted. Applications will be reviewed by multi-stakeholder merit review panels.

Visit the PCORI Funding Center for more information: http://www.pcori.org/funding-

• pportunities/funding-center/

Question and Answer Session

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Submitting Questions: Submit questions via the chat function in on the right of your screen If we are unable to address your question during this time, please e-mail your question to us at getinvolved@pcori.org An archive of this webinar will be posted to http://www.pcori.org/get-involved/pcori-in- practice/ following this event.

#PCORI

Have a Question?

General Inquiries info@pcori.org | (202) 827-7200 Research/Programmatic Questions sciencequestions@pcori.org | (202) 627-1884 Administrative/Financial/Technical Questions pfa@pcori.org Find us Online at: www.pcori.org