Comparison of Surgical and Nonsurgical Options for Management of - - PowerPoint PPT Presentation

Comparison of Surgical and Nonsurgical Options for Management of Nonspecific Chronic Low Back Pain

Applicant Town Hall June 20, 2016

Agenda

Application Requirements Patient Engagement Budget The Merit Review Process Questions

Submitting Questions:

Submit questions via the chat function in Meeting Bridge. Ask a question via phone (an operator will standby to take your questions).

Introduction

Yen-Pin Chiang, PhD Interim Program Director Clinical Effectiveness Research Holly Ramsawh, PhD Program Officer Clinical Effectiveness Research Suzanne Schrandt, JD Deputy Director Patient Engagement Enas Areiqat Assistant Director Contracts Operations Jana-Lynn Louis, MPH Program Associate Clinical Effectiveness Research David Hickam, MD, MPH Director, CER Methods Carolyn Mohan, DrPH, MPH, MIA Merit Review Officer

Your letter of intent (LOI) was reviewed and you have been invited to submit a full application.

CONGRATULATIONS!

LOI Changes

• You were invited to submit a full application based on the

information you provided in the LOI

• Please contact PCORI (pfa@pcori.org) if you wish to make any

significant changes to the proposed study including the following: – PI – Institution – Study design – Budget or period of performance – Research question – Aims – Comparators

• PCORI must approve any changes before the full application is

submitted

Application Requirements

Preparing Your Application

• To begin, all applicants should:
• Read the funding announcement and review the PCORI

Application Guidelines Document

• Review the PCORI Research Plan Template
• Have a copy of your approved LOI readily accessible
• Carefully consider the feedback you received on your LOI

7

Milestones/Deliverables Template

Deliverables: Measurable and verifiable

• utcomes or objects that a

project team must create and deliver according to the contract terms Milestones: Significant events or accomplishments within the project; may have deliverables associated with them

Research Strategy: Overview

Maximum 20 pages in length Use the Research Plan Template as your guide

• Background
• Significance
• Patient Population
• Recruitment Plan
• Estimated Racial/Ethnic and Gender Enrollment Table
• Study Design or Approach
• Engagement Plan
• Research Team and Environment

PLEASE provide all the information requested, as

• utlined in the template.

Page Limit 20

9

Research Strategy: Recruitment Plan

Discuss past experiences with recruitment of the target population Provide preliminary evidence of the potential for successful recruitment Provide numbers for the pool of potential participants, those estimated to be eligible, and the expected participation rate Discuss barriers to recruitment and how you plan to overcome them Strategies for successful recruitment – Engaged clinical sites – Clinical advocates – Proactive, experienced research coordinator – Alignment and integration of recruitment activities with clinical workflow

Research Strategy: Study Design/Approach Tips

Document how proposed study will fill the evidence gap Document efficacy/effectiveness and/or frequency in clinical practice Provide a clear research plan with rigorous methods that adhere to PCORI’s Methodology Standards and accepted best practices A clear justification for the study design and outcome measures Clearly described and justified comparators Sample sizes and power estimates based on careful evaluations of the anticipated effect size Feasibility Realistic strategies for participant recruitment and retention

Methodology Standards

In any study, methods are critical. PCORI’s Methodology Committee developed Methodology Standards to which patient- centered CER must adhere

Methodology Standards: 11 Specific Categories

• Formulating Research

Questions

• Patient-Centeredness
• Data Integrity and Rigorous

Analyses

• Preventing/Handling Missing

Data

• Heterogeneity of Treatment

Effects

• Data Networks
• Data Registries
• Adaptive and Bayesian

Trial Designs

• Causal Inference
• Studies of Diagnostic

Tests

• Systematic Reviews

Research Plan Template: Dissemination & Implementation Potential

Describe the potential for disseminating and implementing the results of this research in other settings. Describe possible barriers to disseminating and implementing the results of this research in other settings. Describe how you will make study results available to study participants after you complete your analyses. PCORI does not expect applicants to disseminate and implement findings at this time.

Page Limit 2

Research Plan Template: Replication & Reproducibility of Research and Data Sharing

Describe the ability to reproduce potentially important findings from this research in other data sets and populations. Describe how you will make a complete, cleaned, de- identified copy of the final data set used in conducting the final analyses available within 90 days of the end of the final year of funding, or your data-sharing plan, including the method by which you will make this data set available, if requested. Propose a budget to cover costs of your data-sharing plan, if requested.

Page Limit 2

Research Plan Template: Protection of Human Subjects

• Provide a Data and Safety Monitoring Plan that operates

under the auspices of your institution

• Assure that key personnel are educated on human subjects

protections

– Assure appropriate informed consent – Establish procedures to minimize risks to participants – Establish procedures to protect privacy and maintain confidentiality

• Refer to NIH standards for research involving human

subjects

Page Limit 5

Research Plan Template: References Cited

List the source material cited in the Research Plan using American Medical Association (AMA) citation style.

Page Limit 10

Research Plan Template: Appendix

• PCORI applications may include an appendix for

additional materials the investigators think may be useful – Reviewers will not be required to include the appendices in the review and assessment of the project

• Examples of additional materials are:

– Survey instruments – Papers and publications

Page Limit 10

People and Places Template: Biosketch

You may use the NIH biosketch or PCORI’s format Biosketches are required for all key personnel List all partners within the Key Personnel section Patient/Stakeholder Biosketch

Page Limit 5 Per person

People and Places Template: Project / Performance Site(s)

Demonstrate that the proposed facilities have the appropriate resources required to conduct the project to plan, within budget, and on time. Provide a description of the facilities that will be used during the project, including capacity, capability, characteristics, proximity, and availability to the project

Page Limit 15

Professional Profile/Biosketch

Letters of Support

• Save all letters of support as a single PDF file prior to

uploading to the PCORI Online System.

• All letters of support should be addressed to the PI and

demonstrate the commitment of key personnel and supporting

• rganizations to your proposed project.
• Letters of support should clearly reflect the substantive

involvement and material contribution to be provided by the signatory parties, and are meant to substantiate the commitment of collaboration of all forms.

Leadership Plan Template ( Dual PI application)

Describe the governance and organizational structure of the leadership team and the research project; Delineate the administrative, technical, scientific, and engagement responsibilities for each PI and the rationale for submitting a dual-PI application; Discuss communication plans and the process for making decisions on scientific and engagement direction; Describe the procedure for resolving conflicts.

Page Limit 5

Use of FDA-Regulated Medical Products

All drugs, biologics, devices, and diagnostics used in your study must be FDA-approved

• Indicate within your application whether investigational

new drug (IND) or other regulatory approval will be needed to conduct your study For example, if your study administers an approved medical product in an off-label manner (e.g., a dose, route, frequency, indication, or patient population that is unapproved by FDA) you may need an IND

• If uncertain, seek guidance from FDA or your legal

counsel

Engagement

Patient-Centeredness

Is the research focused on questions that affect

• utcomes of interest to patient and their

caregivers?

• Does the research question address choices that are

important to - and faced frequently by - patients, their caregivers, or clinicians?

• Is the study powered on outcomes that are important to

patients?

Are the interventions being compared in the study available to patients now?

Addressing Patient-Centeredness

Provide evidence that the research question(s) and

• utcomes are important to patients (and/or their

caregivers) Describe your strategy for measuring outcomes that are important to patients. Remember that a study can be patient-centered even if the end-user is not the patient, as long as patients will benefit from its information.

If the end-user is not the patient, be sure to carefully describe how your study is still patient-centered.

Patient and Stakeholder Engagement

Evidence that patients, caregivers, clinicians, and other stakeholders have been and will be engaged in:

• Formulating the research questions
• Defining the characteristics of study participants, comparators and
• utcomes
• Selecting the important outcomes to be assessed
• Monitoring study conduct and progress
• Designing plans for dissemination of study results

Clear statement of the roles and the decision-making authority of all patient and stakeholder research partners An organizational structure, including a Study Advisory Committee or similar entity, which will bring together national patient and stakeholder groups to further the goals of the study

Patient-Centeredness vs. Patient Engagement

Patient-centeredness is about whether the project aims to answer questions or examine outcomes that matter to patients/caregivers. Patient engagement is about having patients/caregivers as partners in research, as

• pposed to merely being recruited as study

participants.

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

As of April 2016, patient and stakeholder partners will be listed on the abstract landing page Partners may be individuals or organizations By providing these names, the PI is asserting that appropriate consents have been provided If partners wish to remain anonymous, pseudonyms or categorical descriptions can be used

Engagement Resources

PCORI’s “Engagement Rubric”

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

Sample Engagement Plans

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

PCORI Compensation Framework

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

Engagement in Research website page

http://www.pcori.org/content/engagement-research

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

http://www.pcori.org/assets/PCORI-Methodology-Standards1.pdf

Budget Information

Budget Templates: Overview Three budget sections must be submitted as part of the online application process:

Detailed Budget Budget Summary Budget Justification NOTE:

A detailed budget is needed for each year of the

• program. Complete each budget section for the

prime applicant and any/each subcontractor.

Budget Justification

Narrative that fully supports and explains the basis for the information in the Budget Detail – Provide sufficient detail to understand the basis for costs, the reason that the costs are necessary, and an explanation for major cost variances – Use the budget template to tell PCORI why the costs are reasonable for the work to be performed Breakdown of costs proposed for each consortia or contractor Must specify any other sources of funding that are anticipated to support the proposed research project Provide quotes, indirect cost rate letter, fringe benefit policy

Costs of Interventions

PCORI will not cover costs for clinical care alternatives that are being compared in the project. PCORI will consider covering costs for ancillary tasks necessary in the implementation or monitoring of a clinical intervention or strategy as part of the research program.

• Examples include costs for obtaining consent,

collecting data, or monitoring that would not normally be performed in routine care Support for the study by the involved healthcare delivery systems must be documented.

Guidelines for Intervention Cost/Coverage

Costs for study interventions must be covered by delivery system, payer, manufacturer or developer of the intervention. The willingness of one or more of the stakeholder groups to cover treatment costs, even when one of the proposed intervention arms is not currently covered by insurance, will be taken as strong endorsement

• f the study by the health system or payer and of the likelihood that they

will implement or use the study‘s findings if definitive. This material support for the study by host delivery system, payer or developer should therefore be discussed in the application. In exceptional cases, PCORI may consider coverage of the co-payment

• r coinsurance costs of participating patients when that is necessary to

preserve blinding in a study or to assure access to the study for vulnerable populations. Contact PCORI with questions.

Common Application Errors

Using the wrong browser, access PCORI Online via Chrome

• r 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

What happens to your application after you submit it?

Administrative Screening

Applicants must follow administrative requirements set in PCORI’s Application Guidelines.

► Exceeding page limits, budget, or time

limitations

► Not using PCORI’s required templates ► Submitting incomplete sections or applications

Missing the Mark

Programmatic Screening

► Study deviates from approved LOI ► Study includes cost-effectiveness analysis ► Study is not responsive to PFA and/or does NOT

address the PFA’s “Research Areas of Interest”

► Study is not comparative

Missing the Mark

PFA Responsiveness PCORI Mission Advance Through to Merit Review

Merit Review

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 other stakeholder.

• The panel chair facilitates discussion and promotes a

culture of mutual respect and understanding among reviewer types. Patients Other Stakeholders Scientists

Merit Review Criteria

Criterion #1: Potential for the study to fill critical gaps and generate actionable evidence Criterion #2: Potential for the study to be adopted into clinical practice and improve delivery of care Criterion #3: Scientific Merit (Research Design, Analysis, and Outcomes) Criterion #4: Patient-centeredness Criterion #5: Patient and stakeholder engagement Patient and Stakeholder Reviewers Scientist Reviewers

   



    

Multi-phase process for review Preliminary (online) review In-Person review Post-Panel review (PCORI program staff)

Submission and Key Dates

What When Application Deadline August 8, 2016 by 5:00pm (ET) Merit Review Dates November 2016 Awards Announced January 2017 Earliest Start Date March 2017

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
• 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)

Q&A

Ask a question via the chat function in Meeting Bridge. Ask a question via phone (an operator will standby to take your questions).

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