Symptom Management in Patients with Advanced Illnesses Applicant - - PowerPoint PPT Presentation

Applicant Town Hall June 19, 2018 Cycle 2 2018

Symptom Management in Patients with Advanced Illnesses

Agenda

• Programmatic Overview
• Administrative Overview
• Engagement Overview
• Merit Review Criteria
• Questions and Answers

Submit questions via the Question box in GoToWebinar control panel

Today’s Presenters

3

Roycelynn Mentor- Marcel, PhD, MPH Merit Review Officer Lauren Massey Contract Administrator Denese Neu, PhD Engagement Officer Sarah Daugherty, PhD, MPH Senior Program Officer

Programmatic Overview

4

Sarah Daugherty, PhD, MPH Senior Program Officer, Clinical Effectiveness and Decision Science

• Limited number of large trials with long-term outcomes

evaluating head-to-head comparisons of pharmacological and non-pharmacological treatments.

• Additional research is needed in symptom management on:

– Illnesses other than advanced cancer – Pediatric and adolescent populations – Full range of clinically meaningful outcomes including caregiver outcomes – Long term follow-up

Symptom Management Evidence Gap Continues

5

Cycle 2 2018: Symptom Management PFA

• PCORI seeks to fund high-quality

clinical studies that compare the effectiveness of evidence-based treatments for relief of common symptoms experienced by patients with life-limiting advanced illness.

• The Cycle 2 2018 Symptom

Management PFA is available at https://www.pcori.org/funding-

• pportunities/announcement/sympto

m-management-patients-advanced- illness-cycle-2-2018

Available Funds & Project Duration:

• Competitive LOI

Process

• Up to $2 Million in

total direct costs per project

• Projects should be

completed within 3 years

6

What has NOT Changed:

• Advanced illness population with high symptom burden and

life-limiting disease.

• Minimum length of follow-up is six months.

Changes Made in Requirements:

• Proposed interventions may include pharmacological

treatments, nonpharmacological approaches, a combination, or complementary therapies. A pharmacological intervention is encouraged but not required.

• Depression was removed from symptom list.

Reposting of Symptom Management PFA

7

• Symptoms of interest include and ARE limited to:

– Pain – Fatigue – Dyspnea – Insomnia – Anorexia/cachexia – Nausea/vomiting – Anxiety

Symptoms of Interest are Limited to PFA List

8

• Population: Patients, of any age, with life-limiting advanced

illness.

• Intervention: Head-to-head comparison of clinical treatment
• ptions with demonstrated efficacy.
• Outcomes: Diverse type and source of relevant outcomes.
• Follow-up: Long-term ascertainment of outcomes.

PCORI Areas of Interest in Symptom Management PFA

9

• If you are resubmitting an LOI, be clear about what scientific and

logistical changes have been made in the revised LOI. DO NOT SUBMIT the same LOI as last cycle.

• If your LOI is invited for full application, please provide a resubmission

letter identifying the changes that have been made in the revised research plan.

General Guidance for Resubmissions

10

• Adhere to the PFA and Application Guidelines
• Provide a sound rationale for why the research question as proposed is

important to patients.

• Clearly demonstrate the feasibility of the study.

– Show that you have the right team. – Describe and support your recruitment and retention plan. – Document sites are already committed to participating. – Include realistic timelines for study start-up.

• Demonstrate adherence to the PCORI Methodology Standards.

Application Guidance

11

• Propose a comparative effectiveness study that matters to patients.
• Justify the choice of the population and setting.
• Provide evidence of efficacy of the intervention and comparator(s) for

the symptom of choice through citations or demonstrate widespread use in the population proposed.

• Justify the hypothesized effect size with citations that support efficacy
• f the interventions within the proposed population.
• Address the LOI feedback you receive from PCORI in your full

application.

Tips for Success - Programmatic

12

• Submit the completed Letters-of-Intent (LOI) by June 28, 2018

by 5:00 pm ET.

• Provide all information requested, as outlined in the LOI and

Application template.

• For programmatic inquiries, please email the Helpdesk at

pfa@pcori.org

Tips for Success - Administrative

13

Administrative Overview

Lauren Massey Contract Administrator, Contract Management

14

• 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

Using the PCORI Online System

15

• Any private sector (non-profit or for-profit) research organization.
• Any public sector research organization (university or college hospital
• r healthcare system, laboratory or manufacturer, unit of local, state,
• r 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.

Letters of Intent to Apply: Eligibility to Submit

16

• An LOI is required and must be submitted prior to the deadline. Letters of

Intent are due Thursday, June 28, 2018, by 5:00 p.m. ET.

• To submit an LOI, download the 2018 Cycle 2 Symptom Management Letter
• f 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.

Letter of Intent (LOI): Requirements

17

From 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

18

19

Applying for a PCORI Award

• 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
• Submit the completed application on/before the due date by

Tuesday, September 25, 2018, by 5:00 p.m. ET.

Invited to Apply: Tips for Success

20

• Using the wrong browser

– Access PCORI Online via Chrome or Safari browsers

• Not entering information into all required fields in the system
• Not clicking the ‘Save’ button
• Having multiple people working on the application at the same

time

• Having the incorrect file extension

– Only PDF files can be uploaded

• Not choosing the correct document type from the drop-down

menu

• AO is unable to view the application

Common Application Errors

• Research Strategy: 12 pages

– Provide all the information requested, as outlined in the template:

*Specific Aims *Significance *Background *Study Design or Approach

• Research Team & Environment: 2 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.

• 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.

Application Components: Research Plan

22

• 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: (NEW) no page limit
• Applicants must complete each column of this checklist, as appropriate, and

include it with the Research Plan PDF upload.

Application Components: Research Plan (cont.)

23

• Consortium Contractual Arrangements: 10 pages

– 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: (NEW) no page limit

– Applicants must complete each column of this checklist, as appropriate, and include it with the Research Plan PDF upload

Application Components: Research Plan (cont.)

24

Additional Application Components

• Milestones/Deliverables: enter into PCORI online
• People & Places Template

➢ 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

• Letters of Support: no page limit
• Resubmission letter: 2 pages (if applicable)

25

• In PCORI Online, for the Budget tab complete the following sections:

– Detailed Research Project Budget for Each Year of the Research Project Period – Detailed Peer-Review Budget for Peer-Review-Related Costs – Budget Summary for Entire Project

• In the Templates and Uploads tab, upload the Budget Justification Template for

the prime applicant and each subcontracted organization for the entire Research Project Budget and Peer-Review Budget for all research and peer-review-related

• costs. Include the federally negotiated or independently audited indirect cost

rate letter (prime contractor) and fringe benefit rate policy verification document (prime contractor).

Application Components: Budget & Justification

26

Patient and Engagement Plan Overview

Denese Neu, PhD Engagement Officer, Public and Patient Engagement

27

Patients and Other Stakeholders

PCORI Community

Patient/ Consumer

Caregiver/ Family Member of Patient

Clinician

Patient/ Caregiver Advocacy Org

Hospital/ Health System

Training Institution

Policy Maker Industry Payer

Purchaser

Patient-Centeredness vs. Patient Engagement

• Patient-Centeredness

– Addresses outcomes (both benefits and harms) that important to patients and caregivers. – The interventions proposed for comparison are currently available.

• Patient and Stakeholder Engagement

– Demonstrates active engagement between scientists, patients, caregivers and other stakeholders. – Involves relevant organizations, patients, and caregivers through existing relationships or presents a logical and reasonable plan to establish these partnerships.

29

Evidence of Appropriate Engagement Patients/Caregivers and Other Stakeholders

• Applicants are expected to consult with patients/caregivers and
• ther stakeholders on their decisional dilemma and evidence

needs OR reference previously documented decisional dilemmas.

• Identify the patient/caregivers and stakeholders who were

consulted.

• Indicate your commitment to continuing to actively engage

them throughout the conduct of the study.

Budgeting for Engagement

• Financial compensation for serving as a study partner
• Expenses of partners (transportation, childcare, caregiver)
• Budgeting for program staff dedicated to engagement tasks
• Costs of engagement meetings and events (travel, food, audio

visual)

• Additional time and resource to incorporate partner feedback

into various project process

The Engagement Rubric

The rubric is intended to provide guidance to applicants, merit reviewers, awardees, and engagement/program

• fficers (for creating milestones and monitoring projects)

regarding engagement in the conduct of research. It is divided into three segments:

Planning the Study Conducting the Study Disseminating the Study Results

Public Posting of Partner Names

• Many members of the patient and stakeholder community have requested

that PCORI make the names of partnering individuals and organizations available to credit the contributions of the full research team adequately

• You should provide PCORI only those names of patient or stakeholder

partners for whom you have obtained appropriate permission to disclose their identity to PCORI and for PCORI to use their names in public communications

• If partners wish to remain anonymous, you may use pseudonyms or

categorical descriptors (e.g., caregiver to husband with COPD, breast cancer survivor of 20 years)

• If you are selected for funding, the individuals and organizations you

provided (including those described by pseudonym or categorical descriptor) will be listed on the project description page along with the

• ther information about your project (such as abstract and PI)

• PCORI’s “The Engagement Rubric”

http://www.pcori.org/sites/default/files/Engagement-Rubric.pdf

• Sample Engagement Plans

http://www.pcori.org/sites/default/files/PCORI-Sample-Engagement- Plans.pdf

• Compensation Framework

http://www.pcori.org/sites/default/files/PCORI-Compensation-Framework- for-Engaged-Research-Partners.pdf

• Engagement Budgeting

http://www.pcori.org/sites/default/files/PCORI-Budgeting-for-Engagement- Activities.pdf

• Engagement in Research website page

http://www.pcori.org/funding-opportunities/what-we-mean- engagement

• PCORI’s Methodology Standards PC-1 to PC-4

https://www.pcori.org/research-results/about-our-research/research- methodology/pcori-methodology-standards

Engagement Resources

34

Merit Review Overview

Roycelynn Mentor-Marcel, PhD, MPH Merit Review Officer, Merit Review

35

• pcori.org/content/merit-review-process

Merit Review Process

36

Merit Review Criteria

Crosswalk of PCORI Merit Review Criteria with NIH Criteria SIGNIFICANCE

• 1. Potential for the study to fill critical gaps in evidence
• 2. Potential for the study findings to be adopted into clinical

practice and improve delivery of care APPROACH

• 3. Scientific merit (research design, analysis, and outcomes)
• 4. Investigator(s) and environment

PCORI-only Merit Review Criteria PATIENT-CENTEREDNESS/ ENGAGEMENT

• 5. Patient-centeredness
• 6. Patient and stakeholder engagement

Building an Inclusive Merit Review

• Panels include 3 reviewer types to bring diverse perspectives to

the merit review process

• Each application is reviewed by 3 scientists, 1 patient and 1
• ther stakeholder
• PCORI guides reviewers to use the bullet points under each

merit review criterion to evaluate their assigned applications (see Merit Review section in PFA)

Patients Other Stakeholders Scientists Including 1 methodologist

Submission and Key Dates

What When LOI Deadline June 28, 2018 by 5:00 pm ET Application Deadline September 25, 2018 by 5:00 pm ET Merit Review Dates December 2018 Awards Announced April 2019 Earliest Start Date June 2019

39

Refer to the funding opportunities page in our Funding Center (http://www.pcori.org/funding/opportunities) for the following resources:

• PFA and Application Guidelines
• PCORI Online User Manuals
• Sample Engagement Plans
• 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

Resources

40

Where can I find help?

Visit pcori.org/apply ▪ Application Guidelines ▪ 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 – administrative and technical inquiries ▪ E-mail help@pcori.org – PCORI Online help

Q&A

Ask a question via the question box in GoToWebinar control panel

If we are unable to address your question during this time, e-mail the Helpdesk at pfa@pcori.org.

Thank You