The role of engagement in PCORI’s topic selection and funded clinical research Jason Gerson, PhD Clinical Effectiveness and Decision Science SCT/ICTMC Invited Presentation Liverpool, UK May 9, 2017

Roadmap Overview of PCORI Role of engagement in topic selection Role of engagement in research PCORI Methodology Standards – recruitment and retention

Engagement is central to PCORI’s mission PCORI is a funder of patient-centered clinical comparative effectiveness research (CER). PCORI was created to support CER that provides high-integrity, evidence-based information to patients, clinicians, and the broader healthcare community. PCORI’s mission Meaningful involvement of patients, caregivers, clinicians, and other healthcare stakeholders throughout the research process—from topic selection through design and conduct of research to dissemination of results. What we mean by engagement Engagement Rubric PCORI Methodology Standards on Patient-Centeredness Several funding mechanisms to support partnerships between patient organizations and researchers How we operationalize engagement

Engagement in topic selection: Hepatitis C PCORI hosted a multi-stakeholder workshop in 10/2014 on whether CER can help answer questions about hepatitis C diagnosis and treatment.  40 representatives of a wide range of stakeholder groups—including researchers, patients, caregivers, clinicians, industry, payers, and the public health and policy communities -- participated. Based on discussion at that workshop, PCORI issued a $50 million targeted Funding Announcement (FA) addressing key questions in clinical management of HCV infection. Through this FA, PCORI made two awards: THE PRIORITIZE STUDY: A Pragmatic, Randomized Study of Oral Regimens for Hepatitis C: Transforming Decision-Making for Patients, Providers, and Stakeholders Patient-Centered Models of HCV Care for People Who Inject Drugs PCORI reconvened participants of this workshop in 2016. The participants offered further advice and input on additional investments PCORI may wish to make on this topic. A similar approach has been used for our work in palliative care, sickle cell disease, and multiple sclerosis, among others.

Engagement in research PCORI promotes a model that encourages investigators to operationalize engagement at every phase of the study. Research proposals have a dedicated section to describe planned engagement. Fairly steep learning curve for most applicants of conventional clinical research. PCORI has provided many tools and resources to help applicants think about meaningful (thoughtful, respectful, parsimonious(?)) engagement, as well as funding mechanisms to directly support engagement activities. Exemplar studies: Advance Planning for Home Services for Seniors (PI: Lindquist) Long-Term Outcomes of Community Engagement to Address Depression Outcomes Disparities (PI: Wells) Preventing Venous Thromboembolism: Enabling Patient-Centered Care via Health Information Technology (PI: Haut)

Engagement in recruitment and retention PCORI Methodology Standards on Patient-Centeredness: Identify, select, recruit, and retain study participants representative of the spectrum of the population of interest and ensure that data are collected thoroughly and systematically from all study participants Engage people representing the population of interest and other relevant stakeholders in ways that are appropriate and necessary in a given research context Some thought from various stakeholders that the above Standard could more directly address (in revisions) RAR-1: Ensure the trial has a systematic process in which a broad diversity of potential participants are informed and approached about trial participation RAR-3: Ensure the provision of adequate support to encourage retention throughout the trial RAR-4: Form partnerships to increase referrals for and inquiry about the trial.

Jason Gerson, PhD jgerson@pcori.org