Management of Nonspecific Chronic Low Back Pain LOI Applicant Town - - PowerPoint PPT Presentation

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

LOI Applicant Town Hall April 20, 2016

Welcome Overview PFA Requirements Administrative Requirements Resources

Submitting Questions:

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

Agenda

Introduction

Yen-Pin Chiang, PhD Interim Program Director Clinical Effectiveness Research Holly Ramsawh, PhD Program Officer Clinical Effectiveness Research Jana-Lynn Louis, MPH Program Associate Clinical Effectiveness Research Suzanne Schrandt, JD Deputy Director Patient Engagement Maricon Gardner, CRA Associate, Contracts Operations

Overview

Our Focus at PCORI

• Patient-centered
• Answers questions that matter to patients

and other clinical decision makers

• Compares outcomes that matter to patients
• Attention to possible heterogeneity of

treatment effects

Comparative Clinical Effectiveness Research

PFA Overview: Clinical Management of Chronic Low Back Pain

Available Funds and Duration:

• A total of $22 million

(direct and indirect)

• Up to $15 million in total

direct costs

• Projects should be

completed within 5 years

Objective of this PCORI Funding Announcement (PFA):

Address critical clinical and healthcare delivery choices faced by patients, their caregivers, clinicians,

• r delivery systems.

In this PFA we seek to fund:

Randomized clinical trials Healthcare delivery choices faced by patients, their caregivers, clinicians,

• r delivery systems

• Answers a practical, real-world comparative effectiveness

research question

• Assesses whether two or more options differ in effectiveness

when administered as they would be in real life, and the project is conducted in a clinical setting that is as close as possible to a real-world setting

• The methodological approach (including study design, outcome

measures, and follow-up) is as simple as possible without sacrificing scientific rigor

What is a CER Trial?

Programmatic Requirements

• Include patients with chronic nonspecific low back pain who are

representative of the population faced with choosing between the proposed study interventions

• Compare the effectiveness of lumbar fusion surgery versus non-

surgical interventions for improving patient-centered outcomes for chronic low back pain

• Have a sufficiently large study sample to enable precise

estimates of effect sizes and to support evaluation of potential differences in intervention effectiveness in patient subgroups

• Take place within typical clinical care and community settings
• Have strong endorsement and participation by stakeholders

Essential Characteristics of Studies for this PFA

What is the comparative effectiveness of lumbar fusion surgery versus an optimized nonsurgical comprehensive multidisciplinary program for chronic nonspecific low back pain?

PCORI Priority Research Question

• The PICOTs that would apply to this research question include:
• Population: Adults with chronic nonspecific low back pain
• n at least 50% of days during the past 6 months despite

usual care

• Intervention: Lumbar fusion surgery
• Comparator: Comprehensive multidisciplinary nonsurgical

approach to manage chronic low back pain

• Outcome: NIH Low Back Pain Task Force outcome

measures; healthcare utilization; safety; quality of life; validated general and low back pain-specific disability measures

• Time: 5-year study period, including 2-year follow-up
• Setting: Community practice

PCORI Priority Research Question

• Investigators must propose projects that address the priority

research question

• Other investigator-initiated projects will not be considered

responsive to this PFA

• Applicants should provide a convincing rationale for the

comparison of lumbar fusion versus the chosen comparator, citing evidence gaps that are justified on the basis of up-to-date literature reviews.

• Applicants should carefully address the risk of selection bias

that often occurs in such surgical trials and the proposed approach to mitigate such concerns.

• Applicants should carefully consider and provide details

supporting how the target sample size will be met across all study sites

Responsive Applications

• Pilot studies
• Efficacy trials
• Cost-effectiveness analyses
• Direct comparisons of the costs of care between two or more

alternative approaches

• Evaluation of new or existing decision-support tools
• Studies of the natural history of disease, instrument

development, pharmacodynamics, and fundamental science of biological mechanisms

• Evaluation of new or existing decision-support tools
• Development of clinical prediction or prognostication tools

Research Activities Not Supported in the Management

• f Nonspecific Chronic Low Back Pain PFA

• 47 standards in 11 groups
• The Methodology Standards do not address all issues related to

study designs and methods

• Note that PCORI is not using a specific subset of

methodological standards for this PFA.

• Refer to other respected sources for additional guidance.
• View report and standards here:

http://www.pcori.org/research-we-support/research- methodology-standards/

PCORI Methodology Standards

“Gap analysis and systematic reviews should be used to support the need for a proposed study. If a systematic review is not available, a systematic review should be performed using accepted standards in the field (see standard SR-1), or a strong rationale should be presented for proceeding without a systematic review. In the case where a systematic review is not possible, the methods used to review the literature should be explained and justified.”

from: http://www.pcori.org/assets/2013/11/PCORI-Methodology-Report-Appendix-A.pdf

PCORI Methodology Standard* RQ1 – Identifying Gaps in Evidence

• Suggest reviewing pragmatic–explanatory continuum indicator

summary (PRECIS) tool

• Consider tradeoffs
• Eligibility criteria
• Flexibility of intervention
• Range and types of outcomes
• Follow-up intensity
• Adherence
• Etc.
• Source: A pragmatic–explanatory continuum indicator summary

(PRECIS): a tool to help trial designers. Thorpe, et al. CMAJ 2009; 180:E47-E57.

Justification for the Design Elements of Proposed Studies

Patient and Stakeholder Engagement

Patient-Centeredness

• Does the LOI mention outcomes (both benefits and harms)

important to patients?

• Are the interventions being proposed for comparison

available to patients now? Patient engagement

• Does the LOI mention intent to build an interdisciplinary

study team that includes appropriate patient and stakeholder representation in consultation with PCORI?

Patient-Centeredness vs. Patient and Stakeholder Engagement

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Evidence of appropriate engagement of relevant stakeholders and researchers

• Funding applicants are expected to consult with patients

and other stakeholders on their decisional dilemma and evidence needs or to reference previously documented decisional dilemmas in preparation for the submission of LOIs

• Identify the patients and stakeholders you consulted in

determining that the proposed study addresses their evidentiary needs for decision-making and indicate your commitment to continuing to engage them actively in the conduct of the study.

What PCORI looks for when reviewing LOIs

Several approaches to engagement can succeed. PCORI provides many engagement resources for applicants:

• PCORI’s “The Engagement Rubric”

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

• Compensation Framework

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

• Sample Engagement Plans

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

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

Engagement Resources

20

Administrative Requirements

• 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

• f 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.

Eligibility to Submit a Letter of Intent

Letter of Intent (LOI)

• Download the Letter of Intent Template specifically for the

Comparison of Surgical and Nonsurgical Options for Management of Nonspecific Chronic Low Back Pain from the Funding Center to begin your LOI.

• Do not upload additional documents as part of your LOI.
• Letters of endorsements or support are not accepted at this

stage.

• You must upload your LOI as a PDF in PCORI Online.
• You must follow the naming conventions stated on the

guidelines.

• Four page limit – See requirements for font size and type,

margins, and line spacing. – LOIs that exceed four pages will not be reviewed.

• All references should be included as in – text citations.

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

Budget Information

• In the LOI, provide a realistic estimate of the study’s budget.
• Do not just make statements such as “The budget will be

within the $15 million limit.”

• Keep budget estimates reasonable. Do not just propose the

maximum amount.

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 of the study by the health system or payer and of the likelihood that they will implement or use the study‘s findings if definitive.

• In exceptional cases, PCORI may consider coverage of the co-

payment or 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 cost questions.

• An applicant can save information by clicking the

‘Save and Review’ button on the save and review page.

• A PI can add an Administrative Official. The PI and

the AO cannot be the same individual

• Begin the LOI as soon as possible.
• Log into the PCORI system early to address

additional LOI questions.

• Please only use Chrome or Safari browsers to

access the system.

Using the PCORI Online System

Resources

Refer to the Comparison of Surgical and Nonsurgical Options for Management of Nonspecific Chronic Low Back Pain in our Funding Center (http://www.pcori.org/funding-opportunities/announcement/comparison-

surgical-and-nonsurgical-options-management) for the following resources:

• PFA and Application Guidelines
• PCORI Online User Manuals
• Sample Engagement Plans
• General Applicant FAQs: http://bit.ly/applicant_faqs
• PCORI Online: https://pcori.fluxx.io/
• Methodology Standards: http://www.pcori.org/research-we-

support/research-methodology-standards/

Resources

Submission and Key Dates

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What When LOI-Applicant Town Hall April 19, 2016 at 3:00pm ET LOI due in PCORI Online May 4, 2016 by 5:00pm ET Applicants notified as to whether they have been selected to submit a full application June 13, 2016 by 5:00pm ET Applicant Town Hall (if invited) June 2016 Application Deadline (by invitation only) August 8, 2016 by 5:00pm ET Merit Review Dates November 2016 Awards Announced January 2017 Earliest Start Date March 2017

Questions and Answers

Submit questions via the chat function in Meeting Bridge Ask a question via phone (press 7)

Contact Us:

• Schedule a call at http://bit.ly/programmatic_inquiry
• Call 202-627-1884 ( programmatic inquires)
• E-mail us at sciencequestions@pcori.org
• Call 202-627-1885 ( administrative and technical inquires)
• E-mail us at pfa@pcori.org