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

Agenda Welcome Submitting Questions: Submit questions via Overview the chat function in Meeting Bridge. PFA Requirements Ask a question via Administrative Requirements phone (an operator will standby to take your questions). Resources

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

Overview

Our Focus at PCORI Comparative Clinical Effectiveness Research  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

PFA Overview: Clinical Management of Chronic Low Back Pain Objective of this PCORI Funding Available Funds and Announcement (PFA): Duration: Address critical clinical and • A total of $22 million healthcare delivery choices faced by (direct and indirect) patients, their caregivers, clinicians, • Up to $15 million in total or delivery systems. direct costs In this PFA we seek to fund: • Projects should be completed within 5 years Randomized clinical trials Healthcare delivery choices faced by patients, their caregivers, clinicians, or delivery systems

What is a CER Trial? • 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

Programmatic Requirements

Essential Characteristics of Studies for this PFA  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

PCORI Priority Research Question 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 on 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

Responsive Applications  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

Research Activities Not Supported in the Management of Nonspecific Chronic Low Back Pain PFA  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

PCORI Methodology Standards  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 Standard* RQ1 – Identifying Gaps in Evidence “ 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

Justification for the Design Elements of Proposed Studies  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.

Patient and Stakeholder Engagement

Patient-Centeredness vs. 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? 18

What PCORI looks for when reviewing LOIs 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.

Engagement Resources 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 20

Administrative Requirements

Eligibility to Submit a Letter of Intent  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.

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.

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