PCORI Funding Announcement (PFA)

Phased Large Awards for Comparative Effectiveness Research

Applicant Town HallJuly 14, 202012:00-1:30 PM EDT

2

Agenda

• About PCORI

• Funding Announcement Overview

• Patient and Stakeholder Engagement

• Administrative Overview

• Merit Review Process

• Key Dates and Resources

• Questions and Answers

Submit questions via the Question box in GoToWebinar

3

Today’s Presenters

Julie Kennedy Lesch

Senior Engagement

Officer,

Public and Patient

Engagement

Carly Parry

Senior Advisor, Care

Coordination &

Transitions,

Healthcare Delivery

and Disparities

Research

Anne Trontell

Associate Director,

Clinical Effectiveness

and Decision Science

Donna Gentry

Assistant Director,

Contract

Management

Carolyn Mohan

Assistant Director,

Program Support,

Merit Review

About PCORI

5

Why PCORI?

• For all the advances it produces, research still has not answered many

questions patients face.

• People want to know which treatment is best for them.

• Patients and their clinicians need information they can understand and use.

6

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.

7

Our Work Answers Patients’ Questions

Given my personal characteristics, conditions and preferences…

8

Focus on Clinical Comparative Effectiveness Research (CER)

Comparative Effectiveness Research (CER) includes:

• Studies that compare health outcomes and the clinical effectiveness, risks, and benefits of two or more approaches to healthcare

• Clinical effectiveness research

• Improving healthcare delivery CER

• Communications and dissemination CER

• CER to reduce/eliminate health and healthcare disparities

• All applicants should:

• Explain how the research is comparative

• Name the comparators

• State why the comparisons are important

9

Research We Do Not Fund

PCORI does not fund research that will produce…

• clinical practice guidelines

• coverage recommendations

• payment or policy recommendations

In general, PCORI will not cover costs for study interventions that constitute the procedures, treatments, interventions, or other standard clinical care (“patient care”) that are being proposed for comparison in the research project (“patient care costs”).

10

Research We Do Not Fund

Examples of Cost-Effectiveness Analysis:

• Research that conducts a formal CEA in the form of dollar-cost per quality-adjusted life-year to compare two or more alternatives

• Research that conducts a formal CEA in the form of dollar-cost per non-adjusted life-year to compare two or more alternatives

PCORI does not fund studies of cost-effectiveness analysis (CEA).

PCORI may fund studies that capture data that reflect economic

impacts — for example, out-of-pocket costs to patients.

11

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

• Formulating Research Questions

• Patient Centeredness

• Data Integrity & Rigorous Analyses

• Preventing/Handling Missing Data

• Heterogeneity of Treatment Effects

Design-Specific Standards

• Data Registries

• Data Networks

• Causal Inference Methods*

• Adaptive & Bayesian Trial Designs

• Studies of Medical Tests

• Systematic Reviews

• Research Designs Using Clusters

• Studies of Complex Interventions

• Qualitative Methods

• Mixed Methods Research

• Individual Participant-Level Data Meta-Analysis

(IPD-MA)

*The first standard for Causal Inference Methods (CI-1)

is considered cross-cutting and applicable to all

PCOR/CER studies.

Funding Announcement Overview

12

13

PLACER PFAOverview

Objective of this PFA: Support large-scale, high-impact randomized trials in CER with attendant risks of achieving their research aims and thus merit an initial phase of testing and refinement to

• determine their feasibility and viability

• maximize the likelihood of full-scale trial success.

Available funds: $150 million

Project Duration: Up to 6.5 years per award

• Up to 1.5 years (feasibility phase)

• Up to 5 years (full-scale study phase)

Total Direct Costs: Up to $22 million per award

• Up to $2 million (feasibility phase)

• Up to $20 million (full-scale study phase)

14

PLACER PFAHighlights

Requirements

• Scope: Proposed studies of significant scale and scope, requiring funding in excess of $10 million in direct costs

• Design: Individual-level or cluster randomized controlled trial

• Data Coordinating Center: Required to be used and to operate with scientific independence

Opportunities

• Development of strong stakeholder engagement: enabled during the initial phase of award

• Use of PCORnet clinical research infrastructure & data resources: PCORnet is the National Patient Centered Clinical Research Network developed by PCORI

15

PLACER PFA : Distinctive Requirements Relative to Prior PCORI PFAs

• Two well-integrated trial phases: an initial feasibility phase, followed by full-scale study and conduct of clinical trial

• Data coordinating center: Applicants must include a data coordinating center (DCC) with an independent scientific leadership role in study decision-making about the analytical, statistical, and data management aspects of both study phases

• Stakeholder engagement:

• Applicants should include experienced leadership in stakeholder engagement as part of overall trial leadership in both study phases

• PLACER applicants are not required to demonstrate that patients and other stakeholders are already engaged as research team members at the time of application submission

16

PLACER PFAFeasibility Phase

Activities in the first phase may include, but are not limited to:

• Development and initiation of stakeholder engagement plan

• Protocol development

• Finalizing clinically relevant and patient-centered outcome measures

• Piloting of data collection methods

• Refinement of recruitment methods

• Establishment of agreements with study sites and the DCC

• Submission of plans for all aspects of ethical and regulatory oversight and protection of human subjects

17

PLACER PFAFull-Scale Study Phase

• Prior to the planned initiation of the full-scale study, PCORI and an external advisory panel (EAP) will evaluate feasibility phase progress to assess

• Accomplishments and findings relative to pre-specified plans

• Likelihood that the planned full-scale study remains feasible with high probability of success

• If continued conduct of the full-scale trial is approved, PCORI will continue funding via modification of the original contract

• Full-scale trial milestones may be revised based on feasibility phase findings

18

PLACER PFA: Data Coordinating Center Review and Expected Functions

PCORI Review of DCC

• Letters of Intent (LOIs) may be submitted without a named DCC, but a DCC must be included if you are invited to submit a full application

• Applications submitted without a DCC will be removed from consideration for review

• Applications must provide sufficient assurances for review to determine if the proposed DCC meets all necessary standards for data integrity, security, and protection

Expected DCC Functions

• Feasibility phase: Contribute substantively to refining the final trial design and protocol and planning of data collection, management, and the statistical analysis plan

• Full-scale study phase: Management/monitoring of data reporting, completeness, and quality, study enrollment and retention, reporting & analyses to the Data Safety and Monitoring Board (DSMB), and conduct of statistical analyses of trial data

19

PLACER PFA: Data Coordinating Center Relationship to Clinical Leadership of the Study

• PCORI advocates separation and independence of clinical and data/statistical leadership of the study and strongly discourages the use of a combined DCC and clinical coordinating center (CCC).

• If such an arrangement is proposed, it must provide evidence of distinct lines of institutional reporting, accountability, and financing between the DCC and CCC.

• Applicants must certify or otherwise document the existence of adequate policies, procedures, and standards to ensure independence, autonomy, absence of conflicts, and firewalls between clinical care and study data

• PCORI recommends that the PI of the DCC be named as a dual-PI to promote DCC parity, scientific independence, and autonomy in study decisions.

• If the DCC is subcontracted to the prime applicant, the prime institution bears responsibility for ensuring that all relevant regulatory and certification requirements of the data coordinating centers are met to guarantee data integrity, safety, confidentiality, etc.

20

PLACER PFAInvited Research Areas

• Investigator-initiated research in one of five PCORI priority areas:

• Addressing disparities

• Assessment of prevention, diagnosis, and treatment options

• Improving healthcare systems

• Intellectual and developmental disabilities

• Prevention of maternal morbidity and mortality

21

PLACER PFAExemplar Topics

Examples of topics meeting the size and scope requirements for PLACER but NOTexplicit PCORI priorities

• Osteoporotic fracture prevention

• Migraine prophylaxis in adults

• Improving outcomes in maternal morbidity and mortality

• Prevention of suicide among youth

• Optimal systolic blood pressure target in older adults

See PCORI Funding Announcement pages 6-10 for more details.

Patient and Stakeholder

Engagement

23

Patients and Other Stakeholders

PCORI

Community

Patient/

ConsumerCaregiver/

Family

Member of

Patient

Clinician

Patient/

Caregiver

Advocacy

Org

Hospital/

Health

System

Training

Institution

Policy

Maker

Industry

Payer

Purchaser

24

Patient-Centeredness vs. Patient Engagement

• Patient-Centeredness

• Addresses comparisons that are of interest to patients, clinicians, and other stakeholders

• Addresses outcomes (both benefits and harms) that are important to patients

• Patient and Stakeholder Engagement

• Patients and stakeholders are partners in research, not only “subjects”

• Forms of engagement include: input, consultation, collaboration, shared leadership

• Can occur by building upon existing relationships, or developing clear plans for active and meaningful partnership between scientists, patients, and other stakeholders

25

“Research Done Differently”

PCORI’s research has shown that engagement makes meaningful differences in studies:

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; PCORI Board of

Governors Meeting, December 9, 2019, https://www.pcori.org/sites/default/files/PCORI-Board-Meeting-Presentation-Slides-120919.pdf, p. 32-69

26

Demonstration of Appropriate Engagement of Relevant Patients and Other Stakeholders

Applicants are expected to:

• include experienced leadership in stakeholder engagement as part of overall trial leadership in both study phases

• describe initial efforts to consult and collaborate with patient and stakeholder partners to determine the relevance of the research question, and plans for continued collaboration

• establish appropriate organizational infrastructure and resources to engage patients and stakeholders throughout the phases of the study

Merit Review

28

Merit Review Process

29

Application Review

Applications are reviewed against six criteria:

1. Potential for the study to fill critical gaps in

evidence

2. Potential for study findings to be adopted into

clinical practice and improve delivery of care

3. Scientific merit (research design, analysis,

outcomes, including DCC and feasibility phase

plans)

4. Investigator(s) and environment

5. Patient-centeredness

6. Patient and stakeholder engagement

Each application is

reviewed by scientists,

patients, and other

stakeholders who

together provide

content, statistical, and

clinical trial expertise.

Administrative Overview

31

• 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

32

Letters 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.

33

Letter 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 within the 4 page limit.

• 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.

34

From LOI to Application

• Full applications will be invited based on satisfactory 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

35

If Invited 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 of January 12, 2021 by 5:00 PM ET

36

Application Components: Research Plan

• Research Strategy: 20 pages (upload as a single PDF file)

• Provide all the information requested, as outlined in the template: Specific Aims, Significance, Background, Study Design/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.

• Leadership Plan Template: 5 pages (REQUIRED FOR THIS PFA)

• Describe the governance and organizational structure of the leadership team and research project.

• 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.

37

Application 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

• Appendices: 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.

38

Additional Application Components

• Milestones/Deliverables: no page limit (upload as Excel file)

• People & Places Template (upload as a PDF file)

➢ Professional/Patient/Stakeholder Biosketch: 5 pages per individual

➢ Project/Performance Site(s) and Resources: 15 pages

• Leadership Plan Template (Dual PI Applications only): 5 pages

• Subcontractor Detailed Budget Template: (upload as PDF file)

• Budget Justification Template: no page limit (upload as PDF file)

• Letters of Support: no page limit (upload as PDF file)

Key Dates and Resources

40

Submission and Key Dates

What When

LOI Deadline September 29, 2020 by 5:00 PM EDT

Application Deadline January 12, 2021 by 5:00 PM EST

Merit Review Dates April 2021

Awards Announced July 2021

Earliest Start Date October 2021

41

Resources

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

42

Engagement-Specific Resources

Please visit PCORI's website for updated content and resources on the Public and Patient Engagement pages: www.pcori.org/engagement, including:

• The value of engagement and planning for effective engagement in research

• Definitions and examples of different stakeholders

• Budgeting for Engagement Activities

• Compensation Framework for Engaged Research Partners

• Engagement Rubric

• PCORI in Practice Webinar Series

• Updated Engagement Plan template

• Engagement in Health Research Literature Explorer

• PCOR Engagement Tool and Resource Repository

43

Where 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

▪ Call 202-627-1884 (programmatic inquiries)

▪ E-mail sciencequestions@pcori.org

Contact our Helpdesk

▪ E-mail pfa@pcori.org

▪ Call 202-627-1885 (administrative and technical

inquiries)

44

Q&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.

www.pcori.org

@pcori

/PCORInstitute

PCORI

/pcori

Contact Information

45

202.827.7700

pfa@pcori.org

Thank you