Transcript Heterogeneity of Treatment Effects

Engagement in Clinical
Research: The PCORI
Engagement Rubric
Kim Bailey, Engagement Officer
UVA Webinar
October 10, 2014
1
Kim Bailey
Engagement
Officer
Session Objectives
1) Provide an overview of PCORI and
engagement at PCORI
2) Discuss PCORI’s programmatic areas
and merit review process
3) Walk through PCORI’s Engagement
Rubric
4) Introduce additional funding
opportunities at PCORI
About PCORI
Our Mission
PCORI helps people make informed health care decisions,
and improves health care delivery and outcomes, by
producing and promoting high integrity, evidence-based
information
that comes from research
guided by patients,
caregivers and the
broader health care
community.
Our Strategic Goals
Increase Quantity, Quality and
Timeliness of Research Information
Speed the Implementation and
Use of Evidence
Influence Research Funded by Others
We Engage Patients and
Other Stakeholders at Every Step
Topic
Selection and
Research
Prioritization
Merit Review
Evaluation
Study Design/
Implementation
Patient/
Consumer
Caregiver/
Family
Member of
Patient
Purchaser
Payer
Clinician
PCORI
Patient/
Stakeholder
Community
Patient/
Caregiver
Advocacy
Org
Industry
Hospital/
Health
System
Policy
Maker
Training
Institution
Research We Support
PCORI National Priorities for Research
Assessment of
Prevention, Diagnosis,
and Treatment Options
Improving
Healthcare Systems
Addressing
Disparities
Communication &
Dissemination
Research
Accelerating PCOR
and Methodological
Research
We Fund Research That…
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 Focus Attention On…
Conditions
 Conditions that affect large numbers of people across a
range of populations
 Conditions that place a heavy burden on individuals,
families, specific populations, and society
 Rare diseases, which are difficult to study
Populations




Racial and ethnic minorities
Older adults
Low-income and rural populations
Children
PCORI Merit Review
Applications are reviewed against five criteria:
Impact of the condition on the
health of individuals/populations
Potential for the study to improve
healthcare and outcomes
Technical merit
Patient-centeredness
Patient and stakeholder
engagement
• Applications are reviewed by
a panel of two scientists, one
patient, and one other
stakeholder.
• PCORI’s Board of Governors
makes funding decisions
based on merit review and
staff recommendations.
Patient-Centeredness and Patient and
Stakeholder Engagement
Patient-Centeredness
Does the project aim to answer questions or examine
outcomes that matter to patients?
Do research questions and outcomes reflect what is
important to patients and caregivers?
Patient and Stakeholder Engagement
Patients are partners in research, not just “subjects”
Active and meaningful engagement between scientists,
patients, and other stakeholders
Community, patient, and caregiver involvement already in
existence or a well-thought out plan
The Engagement Rubric
The Engagement Rubric
The rubric is intended to provide guidance to applicants, merit reviewers, awardees, and
engagement/program officers (for creating milestones and monitoring projects) regarding
patient and family engagement in the conduct of research. It is divided into four segments:
Planning the Study
Conducting the Study
Disseminating the Study Results
PCOR Engagement Principles
Planning the Study
Planning the Study
Potential Activities Include;
• Identifying the topic and developing the
research question to be studied
• Creating the intervention
• Identifying the comparators
• Defining the characteristics of study
participants
Planning the Study
Real World Examples;
• Epilepsy study: The patients and parents of patients with
epilepsy pose the question: Which anti-epileptic drugs
best preserve sufficient cognition to go to work or school
and to function normally, while still preventing seizures
adequately?
• Diabetes study: Clinicians who reviewed the initial study
design indicated that clinical practice is quite variable
and suggested that a three-arm approach would be more
appropriate for the study. The study design was revised
accordingly.
• Cancer study: Patient partners determine that all women
with breast cancer would be eligible versus only women
who had completed active treatment.
Conducting the Study
Conducting the Study
Potential Activities Include;
• Participating in and monitoring the conduct of
the project
• Assisting with the recruitment of study
participants
• Assisting with data collection and data
analysis
• Participating in the evaluation of patient and
stakeholder engagement
Conducting the Study
Real World Examples;
•
•
•
Chronic pain study: The informed consent document
is developed with patient partners to make it
understandable to study participants.
Depression study: Patient advocacy groups assist with
recruitment through their patient networks—the “book
club” model.
Preeclampsia study: Study team will recruit via a
national network of local health departments and
community health centers, as well as a preeclampsia
advocacy group’s website, and Facebook page.
Disseminating the Study Results
Disseminating the Study Results
Potential Activities Include;
• Identifying partner organizations for
dissemination
• Planning dissemination efforts
• Participating in dissemination efforts, such as
the authoring of manuscripts and the
presentation of study findings
Disseminating the Study Results
Real World Examples;
• Chronic Trauma study: The research team will convene
a policy summit with relevant professional societies
during the third year of the study to focus on identifying
ways to speed the implementation of findings into
practice.
• Cardiac study: A Patient Dissemination Board is
helping to craft the dissemination plan and advise the
research team on how to best share study findings.
• Chronic pain study: Patient partners co-author
manuscripts, present at scientific and lay conferences,
and share study findings through their networks.
Engagement Principles
Engagement Principles
•
•
•
•
•
•
Reciprocal Relationships
Co-learning
Partnership
Trust
Transparency
Honesty
Additional Funding
Opportunities
Pipeline to Proposal Awards
Researchers who unsuccessfully
submitted a PFA and need to
improve proposal
Tier 1
Up to $15,000
Up to 9 month
term
Tier 2
Up to $25,000
Up to 12 month
term
Tier 3
Up to $50,000
Up to 12 month
term
PCORI Funding
Announcement
Or submissions to other
PCOR/CER Funders
Tier I Pipeline Awards
(Up to $15,000 for up to 9 months)
Available to individuals,
consumer/patient organizations,
clinician(s), researcher(s) or a
combination of the above to
support:
 Community building around an
area of research interest to
improve outcomes for patients
 Creation of structure and
communication strategies
 Develop an understanding of
PCORI, and “research done
differently”
Tier II Pipeline Awards
(Up to $25,000 for up to one year)
Available to emerging
research/non-research
partnerships to support:
 Data network and registry
development
 Development of infrastructure
 Generation and refinement of
research question through
community events, town hall
meetings, etc.
Tier III Pipeline Awards
(Up to $50,000 for up to one year)
Available to advanced
research/non-research
partnerships, including those who
submitted PCORI proposals and
were not funded, to support:
 PCORI research proposal
(re)submission focusing on
development of engagement
plan
 Research partnership skill
development
Additional Funding Opportunities
Engagement Awards
 Intended to strengthen the capacity of patients
and other stakeholders to engage in the research
process
 Up to $250,000 and up to two years
Research Meeting and Conference Support
 Up to $50,000 for one-time awards
 Up to $250,000 for multi-year, multi-event awards
Engagement Resources
PCORI’s Engagement Rubric
http://www.pcori.org/assets/2014/08/PCORIEngagement-Rubric-with-Table.pdf
Sample Engagement Plans
http://www.pcori.org/assets/2013/11/PCORI-SampleEngagement-Plans.pdf
Engagement in Research website page
http://www.pcori.org/get-involved/what-is-engagement-inresearch/
PCORI’s Methodology Standards PC-1 to PC-4
http://www.pcori.org/assets/PCORI-MethodologyStandards1.pdf
Find Us Online
www.pcori.org
Kim Bailey, Engagement Officer
[email protected]