1. 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

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

3. 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

4. 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 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.

5. 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)

6. 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.

7. Jason Gerson, PhD jgerson@pcori.org