PCORI Research Priorities and Relevant Examples Anne Trontell, MD, MPH Associate Director, Clinical Effectiveness and Decision Sciences Patient-Centered Outcomes Research Institute Women’s Health Technologies CRN Annual Meeting FDA White Oak Campus in Silver Spring September 7, 2018

Conflicts of Interest/Disclaimer No conflicts to disclose Statements and opinions are mine alone and should not be attributed as the opinions, practices, or policies of the Patient Centered Outcomes Research Institute

PCORI’s Approach to Research An independent, non-profit research institute supporting comparative effectiveness research to address multi-stakeholder questions about health care choices Focus on patient-centered outcomes in comparing the clinical effectiveness of 2 or more options to care for a clinical condition or to improve health care delivery Research done in real-world populations and settings with attention to subgroup differences Engagement of patients and other stakeholders throughout the research process National Research Priorities established with input from PCORI Board of Governors & Methodology committee, as well as patients, other stakeholders, and open public comments Bolded text shows how PCORI’s trial fit the NIH definition of pragmatic trials

PCORI National Priorities for Research Assessment of Prevention, Diagnosis, and Treatment Options Research that: Compares the effectiveness and safety of alternative prevention, diagnosis, and treatment options Determines which options work best overall and for different subgroups of patients Examples

PCORI National Priorities for Research Assessment of Prevention, Diagnosis, and Treatment Options Research that: Compares a variety of health system-level approaches to optimize the quality, outcomes, and efficiency of patient care System levels include patients, families, providers, care teams, organizations, practice settings, delivery options, or policies Examples: Innovative use of health information technology, care coordination for complex conditions, and effective workforce deployment Improving Healthcare Systems

PCORI National Priorities for Research Assessment of Prevention, Diagnosis, and Treatment Options Improving Healthcare Systems Research on: Effective ways to provider information produced by CER Empowering people to ask for and use the information Supporting shared decision making between patients and their providers Communication and Dissemination Research

PCORI National Priorities for Research Assessment of Prevention, Diagnosis, and Treatment Options Improving Healthcare Systems Research on: Reduction or elimination of disparities in health or health care Prevention, diagnosis, or treatment effectiveness AND health care system interventions to reduce barriers or identify best practices Healthcare required to achieve best outcomes in each population Communication and Dissemination Research Addressing Disparities

PCORI National Priorities for Research Assessment of Prevention, Diagnosis, and Treatment Options Improving Healthcare Systems Research on: Building data infrastructure (PCORnet) Determining best methods of current approaches such as registries Improving analytic methods Training researchers, patients, and other stakeholders in how to participate in comparative effectiveness research Communication and Dissemination Research Addressing Disparities Accelerating Patient-Centered Outcomes Research and Methodological Research

PCORI Research Investments

Questions Anne Trontell, MD, MPH www.pcori.org info@pcori.org