Thanks to Ardis Hanson, CBCS Research Coordinator (prepared a
number of slides) Grayson Norquist, PCORI (shared slides)
Slide 3
My background with PCORI Reviewer for 5 cycles (back to early
2013) Ad hoc for Improving Healthcare Systems Standing Member,
Addressing Disparities Investigator for successfully funded project
(Susan McMillan, PI) Patient outcomes of a self-care management
approach to cancer symptoms: A clinical trial. 2013-2016.
($1,984,020) Investigator for several other proposals submitted or
in process
Slide 4
Goals for today What is PCORI looking for? Funding streams and
priorities. What is different about PCORI reviews than NIH process?
Steps to take before you submit a letter of intent Steps to have
completed before you submit a proposal How to make your proposal
shine Resources Questions and discussion
Slide 5
Authorized by the Patient Protection and Affordable Care Act of
2010 as a non-profit, nongovernmental organization to help
patients, clinicians, purchasers, and policy makers make better
informed health decisions by advancing the quality and relevance of
evidence about how to prevent, diagnose, treat, monitor, and manage
diseases, disorders, and other health conditions.
Slide 6
Funding Funded through the Patient-Centered Outcomes Research
Trust Fund (PCORTF), which was authorized by Congress as part of
the Patient Protection and Affordable Care Act of 2010 Two funding
streams: the general fund of the Treasury and a small fee assessed
on Medicare, private health insurance and self-insured plans. PCORI
is expected to receive an estimated $3.5 billion from the PCOR
Trust Fund to fund patient-centered outcomes research through
September 30, 2019.
Slide 7
Funding per year For FY 2010, $10 million For FY 2011, $50
million For FY 2012, $150 million For FY 2013, $150 million from
the general fund in appropriation plus an annual $1 fee per
individual assessed on Medicare and private health insurance and
self-insured plans. Estimated total is $320 million. For FYs
2014-2019, the PCORTF will receive $150 million from the general
fund in appropriation plus an annual $2 fee per individual assessed
on Medicare and private health insurance and self-insured plans and
an adjustment for increase in healthcare spending. The combined
estimated total averages $650 million per year.
Slide 8
Definition of PCOR Assesses the benefits and harms of
preventive, diagnostic, therapeutic, palliative, or health delivery
system features to inform decision-making, highlighting comparisons
of outcomes that matter to people. Is inclusive of an individuals
preferences, autonomy, and needs, focusing on outcomes that people
notice and care about, such as survival, function, symptoms, and
health- related quality of life. Incorporates a wide variety of
settings and diversity of participants to address individual
differences and barriers to implementation and dissemination.
Investigates (or may investigate) optimizing outcomes while
addressing burdens to individuals, availability of services,
technology, personnel, and other stakeholder perspectives.
Slide 9
How does the research PCORI funds differ from Community Based
Participatory Research (CBPR)? The main difference is the focus of
CBPR on social action, which is not a requirement for
patient-centered CER funded by PCORI. Both are similar in the
inclusion of end users in the production of evidence.
Slide 10
Research Areas of Interest Compares interventions to reduce or
eliminate disparities in patient- centered outcomes, including
health, health care, and patient-reported outcomes. For example, by
accounting for possible differences at the patient, provider, or
systems level, we are interested in research to determine what
interventions can be most effective for eliminating disparities in
outcomes. Identifies and compares promising practices that address
contextual factors (e.g., socioeconomic, demographic, or community)
and their impact on patient- centered health outcomes. Compares
benefits and risks of treatment, diagnostic, prevention, or service
options across different patient populations, with attention to
eliminating disparities. Compares and identifies best practices
within various patient populations for information sharing about
treatment outcomes and patient-centered research.
Slide 11
Priority Patient Populations Racial and ethnic minority groups
Low-income groups Women Children (age 017) Older adults (age 65 and
older) Residents of rural areas Individuals with special healthcare
needs, including individuals with disabilities Individuals with
multiple chronic diseases Individuals with rare diseases
Individuals whose genetic make-up affects their medical outcomes
Patients with low health literacy/numeracy and/or limited English
proficiency Lesbian, gay, bisexual and transsexual (LGBT)
persons.
Slide 12
Sample Research Questions NOTE: All questions must have a
comparative component Compare which characteristics of the
patient-centered medical care are most critical to implement to
improve patient-centered outcomes, reduce disparities, and promote
health equity. How does the availability of a patient navigator for
patients and/or caregivers improve patients health outcomes
compared to usual strategies? Under what circumstances, or for what
conditions, are patient navigators most effective? Compare best
options, materials, and venues for patient education materials that
take into consideration patient and caregiver culture, beliefs,
literacy, and numeracy to reduce disparities and improve outcomes.
How do the practices of the top-performing facilities that
primarily serve racial or ethnic minority groups, low-income
populations, or other groups at risk for experiencing disparities
compare with lower performing facilities? Which of the promising
practices are replicable? Given that effective interventions to
improve care in vulnerable populations often require a multipronged
approach, compare what options work best under different
circumstances?
Slide 13
Research Studies Should Be based in comparative effectiveness
research. Study the benefits and harms of different interventions
and strategies that can be delivered in actual settings. Compare at
least two alternative approaches. Compares health outcomes that are
meaningful to the patient population under study. Address questions
about conditions that lead to high costs to the individual or to
society. Examine differentials in healthcare resources or costs as
a determinant of, or barrier to, good outcomes. Evaluate
interventions to reduce health system waste or increase health
system efficiency.
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The Review Process Competitive Letter of Intent PI chooses
panel (e.g. Addressing Disparities) Panel members likely NOT
experts in condition you are studying Online reviews by two
scientific reviewers, two stakeholder reviewers Triage Discussion
at face to face meeting2 minutes per reviewer Vote
Slide 31
The review process is unconventional Stakeholder reviewers
Scientific reviewers Neither will know the science in your area
Relatively little emphasis on preliminary data Relatively little
attention to scientific track record of Investigators Heavy
attention to stakeholder engagement and community partners
Slide 32
PCORI Rating Scale
Slide 33
Key points in writing a proposal Your proposal should be
dripping with patient centeredness Provide evidence that you have
engaged stakeholders Provide clear plans that you will engage
stakeholders Stakeholders not just advisorsparticipants Write for
your audiencenot specialists in your disease condition
Slide 34
From the PCORI Funding Guide
Slide 35
Criterion 1. Impact of the condition on the health of
individuals and populations Is the condition or disease associated
with a significant burden in the US population, in terms of
prevalence, mortality, morbidity, individual suffering, or loss of
productivity? Alternatively, does the condition or disease impose a
significant burden on a smaller number of people who have a rare
disease? Does the proposal include a particular emphasis on
patients with one or more chronic condition?
Slide 36
Addressing Criterion 1 Provide prevalence figures Provide
figures on health disparities Include information on patient
centered outcomes such as quality of life, daily functioning,
emphasize these are important to patients
Slide 37
Criterion 2. Potential for the study to improve health care and
outcomes Does the research question address a critical gap in
current knowledge as noted in systematic reviews, guideline
development efforts, or previous research prioritizations? Has it
been identified as important by patient, caregiver, or clinician
groups? Do wide variations in practice patterns suggest current
clinical uncertainty? Is the research novel or innovative in its
methods or approach, in the population being studied, or in the
intervention being evaluated, in ways that make it likely to
improve care? Do preliminary studies indicate potential for a
sizeable benefit of the intervention relative to current practice?
How likely is it that positive findings could be disseminated
quickly and affect changes in current practice?
Slide 38
Addressing Criterion 2 Make the case that you have an
intervention that is likely to have a significant effect size Make
the case that it could actually be implemented in usual care
settings Show plans for dissemination that involve engagement with
organizations representative of stakeholders
Slide 39
Criterion 3. Technical merit A clear research plan with
rigorous methods and key milestones clearly articulated A research
team with necessary expertise, and an appropriate project
organizational structure A research environment sufficient to
support the conduct of the work with appropriate resources A
diverse study population with respect to age, gender, race,
ethnicity, and clinical status as appropriate for research A focus
on a defined population for whom effectiveness information is
particularly needed
Slide 40
Addressing Criterion 3 Justify your choice of comparator
intervention well Reviewers vary on their like of Usual Care versus
Attention Control or Rival InterventionSee FAQ for information on
this Be sure that your outcome measures are patient centered and
were endorsed by patients/stakeholders
Slide 41
Criterion 4. Patient-centeredness Is the research focused on
questions that affect outcomes of specific interest to patients and
their caregivers? Does the research address one or more of the key
questions mentioned in PCORIs definition of patient-centered
outcomes research? How credible are claims that engaged patients
and stakeholders will exert meaningful influence on the design and
conduct of the research, to ensure patient-centeredness of the
questions and outcomes addressed?
Slide 42
Addressing Criterion 4 Look at PCORI list of questions defining
patient centered outcomes research Be sure that you have ALREADY
engaged patients and other stakeholders in identifying research
questions, methods Document that stakeholders are enthusiastic
about your measures Dont focus on outcomes such as cost, cost
effectiveness, biomedical outcomes such as blood pressure Show how
you changed your intervention and/or measures based on interaction
with stakeholders
Slide 43
Criterion 5. Patient and stakeholder engagement Does the
proposal describe how patients and stakeholders were or will be
identified and engaged in the research? Are the roles of patients
and key stakeholders significant in formulating the studys research
questions, hypotheses and design and in the studys conduct and
dissemination of results? Are the roles proposed for patients and
stakeholders in any planned dissemination or implementation plans
meaningful and likely to be effective? If engagement is not
applicable to the proposed research, does the application justify
why it is not?
Slide 44
Addressing Criterion 5 Propose an active Advisory Boardmeet
often Have a large number of diverse patients and family
stakeholders-PAY THEM Include some front line practice stakeholders
Include concrete plans for continuous engagement of Advisory
Board
Slide 45
Other Considerations Methodological Studies must adhere to
PCORI Methodological StandardsPCORI Methodological Standards Issues
of possible heterogeneity of treatment effects must be considered
and discussed. Observational comparisons must employ study designs
and analytic methods that convincingly protect against selection
bias and other threats to validity. Dissemination and
Implementation Potential Also address any foreseeable barriers to
dissemination and implementation PCORI has separate funding
announcements for D&I.
Slide 46
Proposal development PCORI constantly revises its procedures
Join the PCORI listserve Participate in their webcasts Include an
Investigator or have a reviewer who knows the PCORI process Engage
the PCORI expert before submitting LOI Plan to engage stakeholders
before submitting proposal LOI should note process UNDERWAY to
engage stakeholders
Slide 47
Before you submit Engage diverse stakeholders in review of your
Aims, Methods, Measures, Dissemination Plan Treat stakeholders with
respect as collaborators in the process Gain letters of support
from stakeholders
Slide 48
Final comments and looking to the future PCORIs approach to LOI
unknown Percent screened out unknown Should increase funding rate
if invited for full proposal Screening based on existing portfolio
Funding beyond 2019 Potential for patient centered research
movement to spread more broadly
Slide 49
Applicant Resources PCORI
http://www.pcori.org/http://www.pcori.org/ Subscribe to PCORI
http://www.pcori.org/content/subscribehttp://www.pcori.org/content/subscribe
PCORI Funding Opportunities
http://www.pcori.org/funding/opportunitieshttp://www.pcori.org/funding/opportunities
PCORI FAQ http://www.pcori.org/http://www.pcori.org/ PCORI Funding
Awards Past Awards Search:
http://pcori.org/pfaawards/?viewby=priorityhttp://pcori.org/pfaawards/?viewby=priority
Past Awards Map
http://www.pcori.org/research-results/maphttp://www.pcori.org/research-results/map
PCORI Methodology Standards
http://pcori.org/assets/PCORI-Methodology-Standards.pdf PCORI
Patient and Family Engagement Rubric
http://www.pcori.org/assets/2014/02/PCORI-
Patient-and-Family-Engagement-Rubric.pdfhttp://www.pcori.org/assets/2014/02/PCORI-
Patient-and-Family-Engagement-Rubric.pdf