Cycle 2 2021 Pragmatic Clinical Studies (PCS) - Applicant Town Hall May 21st, 2021 - PCORI
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Cycle 2 2021 Pragmatic Clinical Studies (PCS) Applicant Town Hall May 21st, 2021
Agenda
• About PCORI
• Programmatic Overview
• Patient and Stakeholder Engagement
• Administrative Overview
• Merit Review Process
• Resources
• Questions and Answers
Submit questions via the Question box in GoToWebinar
2
Today’s Presenters
Brigette Scott, Anne Trontell, Carly Khan, PhD,
MA MD, MPH RN, MPH
Senior Project Associate Program Officer
Manager Director, Healthcare Delivery
Program Support Clinical and Disparities
& Information Effectiveness and Research
Management Decision Science
Iris Giggetts, Laura Sheahan,
MSW Julie Kennedy PhD
Senior Contract Lesch, MPA Merit Review Officer
Administrator Senior Program Support
Program Support Engagement & Information
& Information Officer Management
Management Public and Patient
Engagement
3
Patient Centered Outcomes
Research Institute (PCORI)
• An independent, non-profit research institute to support informed health
decisions of diverse stakeholders via research & dissemination activities
• Funds comparative clinical effectiveness research (CER) of >2 head-to-
head options to care for a clinical condition or to improve health care delivery
‒ Nearly 1800 research and research-related awards
‒ Funding to-date in excess of $2.9 billion
‒ Special interests in subgroup differences, disparities, vulnerable
populations, and research methodologies
• Funds research contracts using milestones and deliverables to assess
progress
4
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 throughout the entire
research process.
5
PCORI Funds Comparative Clinical
Effectiveness Research (CER)
What is CER?
• Generates and synthesizes evidence comparing benefits and harms of at least
two different methods to prevent, diagnose, treat, and monitor a clinical
condition or improve care delivery
• Measures patient-centered outcomes in real-world populations
• Helps consumers, clinicians, purchasers, and policy makers make informed
decisions that will improve care for individuals and populations
• Informs a specific clinical or policy decision
• Describes results in subgroups of people
6
Research PCORI Does Not Fund
PCORI does not fund research whose findings will include
• Development of clinical practice guidelines
• Coverage recommendations
• Payment or policy recommendations
7
Research PCORI Does Not Fund
PCORI does not fund studies of cost effectiveness analysis (CEA) of
two or more alternatives such as:
PCORI may fund studies that capture data on economic impacts
such as out-of-pocket costs to patients
For more info, please see FAQ here
8
PCORI Principles for
Full Consideration of Outcomes
• PCORI’s reauthorizing legislation directs PCORI to capture, as appropriate,
the full range of outcomes data in the course of our research studies.
• PCORI is still prohibited from developing QALY measures and conducting
cost-effectiveness analysis via our authorizing legislation.
• Information and guidance are available at
– Principles for the Consideration of the Full Range of Outcomes Data
– FAQs on Cost and Cost-Effectiveness Analysis
9
Updated Cost Principles: From the PFA
• Allowed:
– Data collection on cost burdens or economic impacts associated with interventions that
are relevant to patients and caregivers.
– Data collection on cost burdens and economic impacts relevant to other stakeholders,
when these outcomes have a near-term or longer-term impact on patients
• Non-Responsive:
– Conducts a formal cost-effectiveness analysis of alternative approaches to providing care
– Directly compares the costs of care between two or more alternative approaches to
providing care, or relies on modeling to develop estimates of “total costs of care” designed
to enable such comparisons
– Proposed research may not measure economic impacts as the primary outcome of a
proposed study. Proposals that have economic measures as the primary outcome will be
considered nonresponsive.PCORI Methodology Standards
Research funded by PCORI must adhere to the PCORI Methodology Standards, which
represent minimal requirements for the design, conduct, analysis, and reporting of
patient-centered outcomes research.
The 65 standards can be grouped into 2 broad categories and 16 topic areas.
Cross-Cutting Standards Design-Specific Standards
• Formulating Research Questions • Data Registries
• Data Networks
• Patient Centeredness
• Causal Inference Methods*
• Data Integrity & Rigorous Analyses • Adaptive & Bayesian Trial Designs
• Preventing/Handling Missing Data • Studies of Medical Tests
• Heterogeneity of Treatment Effects • Systematic Reviews
• Research Designs Using Clusters
• Studies of Complex Interventions
• Qualitative Methods
• Mixed Methods Research
• Individual Participant-Level Data Meta-
*The first standard for Causal Inference Methods (CI-1) is
considered cross-cutting and applicable to all PCOR/CER
Analysis (IPD-MA)
studies.
11PCORI’s National Patient-Centered
Clinical Research Network – PCORnet
• A collaborative network of health systems, researchers, patients, and clinicians
with EHRs from more than 100 health systems and 70 million patients
• Use of existing PCORnet data resources or health systems may facilitate
observational studies and randomized trials proposed under this PFA.
• Additional information on PCORnet can be found at PCORnet.org
• To leverage this PCORI resource, contact the PCORnet Front Door to
schedule a consultation and ensure the proposed use of PCORnet is
appropriate and feasible, and that budget materials accurately reflect the cost
of using PCORnet resources.
‒ Initiate consultations by completing the intake form located here
(https://pcornet.org/contact-us/).
12PCS PFA: Overview
Goals of PCS PFA
• To address critically important evidence gaps where insufficient comparative
effectiveness information is a major barrier to clinical or healthcare system
decision making about 2 or more healthcare options
• To gain insight into the heterogeneity of treatment effects (HTE) for subgroups
and individuals through the study of large, diverse, and heterogeneous
populations
• To support research conducted under conditions that can be generalized to real-
world populations and settings as reflected by
‒ The study of actual choices currently faced by patients, clinicians, or policy-
makers
‒ Protocols which reflect usual clinical care delivery settings and practices
‒ Study designs, outcome measures, and follow up that are as simple as
possible without sacrificing scientific rigor
14PCS PFA Overview
• Seeks to fund clinical trials, large simple trials, or large-
scale observational studies comparing two or more
healthcare alternatives that address PCORI national
Available Funds:
priority areas
‒ Assessment, prevention, diagnosis, treatment, or Up to $90 million
management of a disease or symptom Total Direct Cost:
‒ Improving healthcare system-level approaches to
managing care Up to $10 million
‒ Health or healthcare disparities Maximum Project
‒ Maternal morbidity and mortality Period:
‒ Intellectual and developmental disabilities
5 years
• Randomized study designs are strongly encouraged
but not required.
15Pragmatic Design Features
• Seek simplified yet rigorous designs, outcome measures, and follow up
• Avoid design features considered to be more explanatory or
mechanistically-focused in addressing their research question
‒ Highly selected and homogeneous populations relatively free of
comorbidities
‒ Specialized research settings and conditions
‒ Rigidly controlled research protocols unlike typical care delivery
conditions
‒ Comparisons made to a placebo
16PCORI Expectations for
Pragmatic Study Designs
• Research design features should be “fit for purpose” in answering the
research questions of stakeholders
• Pragmatic elements should serve the research question not maximal
pragmatism
• Tabular PRECIS summary in submitted applications should emphasize how
design choices best inform future implementation of study findings
• Important supplementary references to PRECIS citations in the PFA
‒ PCORI Methodology Standards, especially for Studies of Complex
Interventions to help define allowable adaptations or “guardrails” in how
flexibly the protocol can be interpreted and carried out
‒ PCORI Guidance on the Design and Conduct of Trials in Real-World
Settings
17PCS PFA Requirements
• Must answer a critical choice or decisional dilemma among healthcare
options, each with established efficacy
• Deploy interventions that are sufficiently well defined to be replicable in their
dissemination and implementation in US health care
• Propose a sample size sufficiently large to estimate effect sizes with precision
and to support evaluation of potential subgroup differences (H.T.E)
• Adhere to PCORI Methodology Standards establishing minimum
requirements for patient-centered outcomes research
18PCS PFA Requirements
• Focus on clinical comparative effectiveness
‒ Explain how the research is comparative
‒ Name the comparators
‒ State the importance of the comparison(s)
• Describe the potential for findings to be adopted and improve clinical practice and
delivery of care
‒ Identify end-users and how study findings will support their information needs
‒ Address patient and practice/organizational barriers and facilitators to rapid
adoption
‒ Demonstrate applicability to diverse practices and delivery systems
‒ Show study endorsement by key patient, physician, and other stakeholder
groups
19Comparisons to Usual Care
• “Usual care” is typically a suboptimal comparator for CER studies
‒ It is ill-defined, difficult to quantify, and subject to geographic and
temporal variations that limit its interpretability, applicability, and
reproducibility
• If proposing “usual care” as a comparator
‒ Describe it in detail
‒ Justify its use rather than an active comparator and how it is a
legitimate and coherent comparator (e.g. it is guidelines-based)
‒ Explain how the care given in the usual care group will be measured for
each individual and how appropriate inferences will be drawn from its
inclusion
20Considerations for Complex Interventions
If proposing a multi-component intervention
• Components of the intervention should each have evidence of effectiveness
• If the bundled components have not been tested for effectiveness as a
package
‒ Justify why the package of all components is likely to be more effective
than a single component or subset of the components
‒ Discuss issues that might arise in the dissemination and sustainability
of the package as a whole
• Refer to PCORI Methodology Standards for Complex Interventions
21Considerations for
Interventions Targeting Disparities
Evidence-based interventions shown effective in the general population may
lack evidence of effectiveness in populations at risk for disparities
• PCORI is interested in studies that tailor and test effectiveness in populations
at risk for disparities
• If necessary, interventions with documented efficacy or effectiveness in
similar situations may be used with some adaptation
‒ Efficacy should be well-documented by prior research or systematic review
‒ A strong rationale should be provided for expecting the intervention to be
efficacious in its new setting(s) and/or population(s)
‒ Efforts to adapt and validate an intervention should be minor with most of the
proposed effort, time, and budget aimed at assessing comparative
effectiveness
22PCS Pilot Funding Initiative
Patient Care/Intervention Costs
• Applicants may request PCORI funding support for critical and unmet needs to provide
coverage of patient care costs that fall upon study participants
• Patient care costs may include study interventions that constitute the procedures, treatments,
interventions, or the clinical personnel costs of providing standard clinical care (patient care)
• Interested applicants must provide realistic, well-supported, and justified requests addressing
3 criteria:
‒ The strategic importance of the research question as shown by a systematic review, national
PCORI priority area, and/or its potential for significant impact upon clinical decision-making or
policy formation
‒ Evidence of significant barriers to recruitment of participants or to inclusion of key subgroups
‒ The likelihood of sustained uptake of study results without future cost subsidies, as shown by
commitments of > 1 key stakeholders to support the future uptake of study results as provided via
letters of support or contributions of funding/other resources
• If an application is chosen for award, PCORI will consider its potential funding of patient care
costs during contract and budget negotiations
23What does PCORI look for when reviewing
Letters of Intent (LOIs)?
• Importance and relevance of the CER question as demonstrated by
‒ a health decision of high relevance and critical importance to stakeholders
‒ a research need or gap documented in a systematic review or other high
quality evidence syntheses
‒ Readiness of findings to be implemented into clinical practice or care delivery
• Clarity and credibility of applicants’ responses to the LOI questions about the
study population, comparators, outcomes, and proposed research methods
(e.g., study design, sample size, effect size)
• Programmatic fit and balance
24Impact of COVID-19
• Due to the multiple risks and uncertainties of COVID-19, applications should
include
‒ an explicit assessment of potential risks and risk management plans or
contingencies for the proposed research as it may be affected by COVID-19
‒ provisions in leadership and staffing plan to assure study continuity in the
event of personnel absences due to the pandemic
25Patient About
and Stakeholder
PCORI
Engagement
pcori.orgPatients and Other Stakeholders
Patient/
Consumer
Caregiver/
Family
Purchaser
Member
of Patient
Payer Clinician
PCORI
Community
Patient/
Caregiver
Industry
Advocacy
Org
Hospital/
Policy
Health
Maker
System
Training
Institution
Definitions can be found here: https://www.pcori.org/about-us/our-programs/engagement/public-and-patient-engagement/pcoris-stakeholders 27Patient-Centeredness vs. Patient Engagement
• Patient-Centeredness
‒ Addresses outcomes (both benefits and harms) that are important to patients
‒ The interventions proposed for comparison are currently available to patients
‒ Study design is low-burden to participants
• Patient and Stakeholder Engagement
‒ Patients and stakeholders are partners in research, not only “subjects”
‒ Incorporates the expertise of patients, clinicians and other stakeholders for the purpose of
improving study design, conduct and analysis
‒ Range of engagement approaches: Input, Consultation, Collaboration, Shared leadership
‒ Can occur by building upon existing relationships, or developing new ones
28“Research Done Differently”
PCORI’s research has shown that engagement can impact studies:
Acceptability:
studies in which patients and clinicians will be willing to participate based on burden,
usability, and alignment with preferences, values, and needs
Feasibility:
mitigating potential or actual roadblocks—particularly to making interventions,
enrollment, retention, and data collection more doable in real-world settings
Rigor:
choices that minimize bias and enhance data quality
Relevance:
applicability and importance of the research for patients and clinicians making decisions,
including the importance of research questions and outcomes, generalizable study
samples, interpretations of findings in a real-world context, and more effective
dissemination
Forsythe LP, Carman KL, et al. Patient Engagement In Research: Early Findings From The Patient-Centered Outcomes Research Institute. Health Aff (Millwood). 2019 Mar;38(3):359-367;
29Engagement Requirements for LOI
For the LOI, there are two main objectives related to engagement:
1. Explain why the research question is important to patients and
other stakeholders and how it was determined
2. Briefly describe how patients and stakeholders will be actively
engaged throughout the phases of the study
30Engagement Requirements for Application 1. List of proposed patient and other healthcare research partners (include names and affiliations, if available), and justification for their inclusion 2. Goals for working with stakeholders, which may include affecting the acceptability, feasibility, rigor, and/or relevance of the study 3. Description of the approaches the team will use to engage stakeholders at key decision points over the course of the study 4. Proposed ideas for how stakeholder advisory committees and/or governance committees that include stakeholders will be structured and their responsibilities Funded awardees are required to submit a more detailed engagement plan six months after contract execution. 31
Administrative Requirements
Using the PCORI Online System
• Register as a New User and create your LOI as soon as possible
• Please note that the PI and AO cannot be the same person
• Please only use Chrome, Safari, and Firefox browsers to access the system
https://pcori.force.com/engagement
PCORI Online Training Slides
PCORI Online Application Cheat Sheet
PCORI Online Training Resources
33Letters of Intent to Apply: Eligibility to
Submit
• Any private sector (non-profit or for-profit) research organization.
• Any public sector research organization (university or college hospital or healthcare
system, laboratory or manufacturer, unit of local, state, or federal government).
• Non-domestic components of organizations based in the US and foreign
organizations may apply, as long as there is demonstrable benefit to the US
healthcare system and US efforts in the area of patient-centered research can be
clearly shown.
• Individuals are not permitted to apply.
34Letter of Intent (LOI): Requirements
• An LOI is required and must be submitted prior to the deadline.
• To submit an LOI, download the PFA-specific Letter of Intent Template from the
Funding Center to begin your LOI.
• You must answer all questions.
• Do not upload additional documents as part of your LOI. Letters of endorsements or
support are not accepted at this stage.
• Only those LOIs deemed most responsive (programmatically and administratively) to
this PFA will be invited to submit a full application.
35From LOI to Application
• Full applications are invited based on the information provided in the LOI.
• Changes to the following require PCORI’s approval:
‒ Principal investigator
‒ Institution
‒ Research question(s)
‒ Specific aims
‒ Study design
‒ Comparators
‒ Budget/period of performance
36Invited to Apply: Tips for Success
• Adhere to the Submission Instructions for the appropriate PFA and funding
cycle
• Start and submit application early
• Have a copy of your approved LOI readily accessible
• Ensure that all team members can see the application in the system (check
during the LOI stage)
• Inform your AO of your intent to submit
• Clearly describe comparators for the study
• Submit the completed application on/before the due date by 5:00 PM ET
37Application Components
• Milestones/Deliverables: no page limit (upload as excel file)
• PI Template (Previously People & Places Template) (upload as a pdf file)
‒ Professional Profile/Biosketch 5 pages per individual
‒ Leadership Plan Template (Dual PI Applications only): 5 pages
‒ Patient/Stakeholder Partner Profile/Biosketch: 5 pages per individual
• Key Personnel Template (upload as a pdf file)
‒ Professional Profile/Biosketch 5 pages per individual
‒ Patient/Stakeholder Partner Profile/Biosketch: 5 pages per individual
• Project/Performance Site(s) and Resources: 15 pages
38Application Components: Research Plan
• Research Strategy: 15 pages (upload all components of Research Plan as a single pdf file)
‒ Provide all the information requested, as outlined in the template:
*Specific Aims *Significance
*Background *Study Design or Approach
• Research Team & Environment: 3 pages
‒ Describe the research team’s capabilities to accomplish the goals of the proposed research project and the
appropriateness of the research environment to conduct the study.
• Dissemination & Implementation: 1 page
‒ Describe how you will make study results available to study participants after you complete the analyses,
and possible barriers to disseminating and implementing the results of this research in other settings.
• Return of Aggregate Study Results: 1 page
‒ Describe the approach that will be used to communicate the research findings to study participants once the
study is complete.
• Protection of Human Subjects: 5 pages
‒ PCORI follows the Federal Policy for the Protection of Human Subjects (45 CFR part 46), including the
Common Rule.
39Application Components: Research Plan
(cont.)
• Consortium Contractual Arrangements: 10 pages
‒ Describe the proposed components of the research project that will be performed by
subcontracted organizations.
‒ Explain the strengths that these partners bring to the overall project to ensure successful
submission of contract deliverables in accordance with the milestone schedule.
• References Cited: 10 pages
‒ List the full bibliographical citation for each reference. Each reference must include the names of
all authors the article title and journal or book title, the volume number, the page numbers, and
the year of publication
40Application Components: Research Plan
(cont.)
• Appendix: 2 pages (To be completed only if requesting PCORI funding of patient care costs).
‒ Description and justification of request for Patient Care Costs coverage
• Appendix: 10 pages (optional)
‒ Applicants can include additional materials that they believe are useful, but reviewers are not
required to review the appendix materials in evaluating the application.
• Methodology Standards Checklist: no page limit (upload as excel file)
‒ Applicants must complete each column of this checklist, as appropriate, and include it with the
Research Plan PDF upload
• Subcontractor Detailed Budget Template: (upload as a excel file)
• Budget Justification Template: no page limit (upload as a pdf file)
• Letters of Support: no page limit (upload as a pdf file)
41How to Request Coverage of
Patient Care Costs
In the LOI and full application (if invited)
• Describe the procedures, treatment, interventions, or clinical personnel
services for which funding is requested and provide their estimated costs.
In submitting an application
• Limit the description and justification to 2 pages
• Provide stakeholder Letters of support
42Merit Review
Merit Review Process
• pcori.org/content/merit-review-process
44Application Review
Applications are reviewed against six criteria:
1. Potential for the study to fill critical gaps in evidence
Each application
2. Potential for study findings to be adopted into clinical is reviewed by
practice and improve delivery of care scientists, patients, and
3. Scientific merit (research design, analysis, outcomes) other stakeholders who
together provide content,
4. Investigator(s) and environment
statistical, and clinical
5. Patient-centeredness trials expertise
6. Patient and stakeholder engagement
45Submission and Key Dates
What When
LOI Deadline June 1, 2021 by 5:00 pm ET
Application Deadline August 31, 2021 by 5:00 pm ET
Merit Review Dates December 2021
Awards Announced March 2022
Earliest Start Date August 2022
46Resources
Refer to the funding opportunities page in our Funding Center
(http://www.pcori.org/funding-opportunities) for the following resources:
• PFA and Submission Instructions
• PCORI Online User Manuals
• General Applicant FAQs: https://help.pcori.org/hc/en-
us/categories/200010230-Applicant-Resources
• PCORI Online: https://pcori.force.com/engagement/
• Research Methodology: http://www.pcori.org/node/4020
47Specific Engagement Resources
Please visit PCORI's website for updated content and resources on the Public
and Patient Engagement pages:
https://www.pcori.org/engagement/engagement-resources
• The value of engagement and planning for effective engagement in research
• Definitions and examples of different stakeholders
• Engagement Resources
‒ Engagement Rubric
‒ Compensation Framework
‒ PCORI in Practice webinars
‒ Research Fundamentals training for lay person partners
• Engagement in Health Research Literature Explorer
48Where can I find help?
Visit pcori.org/apply
• Submission Instructions
• FAQs
• PCORI Online User Manuals
• Sample Engagement Plans
Schedule a Call with a Program Officer
• Submit a request at
pcori.org/content/research-inquiry
• E-mail sciencequestions@pcori.org
Contact our Helpdesk
• E-mail pfa@pcori.org
49Q&A
Ask a question via the question box in GoToWebinar
If we are unable to address your question during this time, e-mail the
Helpdesk at pfa@pcori.org.
50Thank you
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