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 We welcome your questions and comments via the chat function on the right side of your screen We welcome your comments via Twitter to @PCORI and #PCORI 2

Agenda for today’s webinar Time Agenda Item Speakers Norman B. Kahn, Jr., MD, Executive Vice President 1:00 - 1:10 p.m. Welcome and Introductions and CEO, Council of Medical Specialty Societies Background on PCORI and Joe V. Selby, MD, MPH, 1:10 - 1:30 p.m. Exploring our Portfolio Executive Director, PCORI Funding Opportunities: Large Pragmatic Studies to Evaluate Joe V. Selby, MD, MPH, 1:30 – 1:45 p.m. Patient-Centered Outcomes Executive Director, PCORI Moderated by PCORI and 1:45 – 2:00 p.m. Question and Answer Session CMSS Staff 2:00 p.m. Adjourn 3

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 #PCORI 4

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 Vision Patients and the public PCORI helps people make have the information informed healthcare decisions they need to make and improves healthcare decisions that reflect delivery and outcomes by their desired health producing and promoting high outcomes . integrity, evidence-based information that comes from research guided by patients, caregivers, and the broader healthcare community. 6

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 7

Engagement: A Critical Path to Useful, High-Quality Research Proposal Review; Topic Selection Design and Conduct and Research of Research Prioritization Dissemination and Evaluation 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

The National Patient-Centered Clinical Research Network (PCORnet) 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 observational. 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.

PCORnet 11 Clinical Data Research Networks (CDRNs) 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 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 other 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 of patients 18

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 outcomes. 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 19

Potential Roles of Medical Specialty Societies with PCORnet 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 outcomes measures 20

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

Pragmatic Clinical Studies Available Funds and Duration: Objectives of this Initiative: • A total of $90 million Address critical clinical and health-related (direct + indirect) each comparative effectiveness questions faced cycle by patients, their caregivers, and their clinicians • 2 cycles per year Strong involvement of medical specialty and patient organizations required. • Up to $10 million in direct costs per project PCORI seeks to fund: • Projects should be Pragmatic clinical trials completed within 5 years 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