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Spring 2015 Cycle: Large PragmaticStudies to Evaluate Patient-Centered Outcomes
Applicant Town Hall
June 10, 2015
Your letter of intent (LOI) was reviewed and you have been invited to submit a full application……..
CONGRATULATIONS!
AgendaWelcome
Overview
Merit Review Criteria
Other topics
What to think about before you apply
Questions?Submit questions via the chat function in Meeting Bridge.
Ask a question via phone (an operator will standby to take your questions).
Purpose of the Pragmatic Studies PFATo assist applicants to prepare strong proposals in response to this funding announcement:
Applicants proposing “clinical comparative effectiveness research (CER)”Applicants proposing “improving healthcare systems (IHS) CER”Applicants proposing “CER to reduce or eliminate health and health care disparities”
What is a Pragmatic CER Study?
Answers a practical, real world comparative effectiveness research question. Assesses whether two or more options differ in effectiveness when administered as they are in real lifeProject 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 Pragmatic CER Study? (Cont.)
Pragmatic CER trials addressing populations at risk for disparities may: Require higher levels of outcome data collection. Require tailoring of intervention (e.g., language, culture,
access). Consist of multi-component, multi-level interventions
(e.g., targeting the patient, provider, and systems), as evidenced by disparities literature.
Justification for the Design Elements of a Large Pragmatic Study
Suggest reviewing pragmatic–explanatory continuum indicator summary (PRECIS) toolConsider 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.
Research Activities Not Supported in this PFA
Studies of decision aidsEfficacy trialsEvidence synthesesCost-effectiveness analysisResearch that aims to compare the overall costs of care between two or more alternatives and use the results to determine the preferred alternative
Merit Review CriteriaImpact and Potential to Improve Care
Steven Clauser, PhD, MPAProgram DirectorImproving Healthcare Systems
Merit Review Criteria
Impact of the condition on health of individuals and populationsPotential for the study’s results to improve care and outcomesTechnical meritPatient-centerednessEngagement with key patient (including caregiver), clinician, and/or other stakeholder organizations
Impact of the Condition
In the US population, or in underserved sub-populations:
Is the condition or disease associated with a significant burden - in terms of prevalence, mortality, morbidity, costs to society, individual suffering, or loss of productivity?
When the Condition is a Healthcare System Problem
In the US population, or in underserved sub-populations:
Is the healthcare system problem associated with poor access to care, suboptimal care quality, high burden to society or to healthcare systems, individual suffering, mortality, morbidity, and/or loss of productivity?
Addressing Impact of the Condition or System Problem
Clinical CER State the prevalence of the condition Discuss the impact of the condition on the individual
Improving Healthcare Systems CER Describe the magnitude of the healthcare system
problem Discuss the impact of the system problem on healthcare
access and quality – and on individual and population suffering, morbidity, mortality, productivity and costs
Potential for the Study’s Findings to Improve Care and Outcomes
What knowledge gap underlies the problem?How does that gap manifest in practice?Would positive study findings close the gap and improve practice and outcomes?Would such findings be adopted widely?
Addressing Potential for Study Findings to Improve Care and OutcomesProvide evidence of an important knowledge gap: A recent systematic review or clinical guidelines pointing to a
research need. An update of previous systematic review is also acceptable.
Provide evidence of substantial variation in practice, resulting from the knowledge gap, if known.Provide indications that the proposed CER has a substantial potential to improve practice and patient outcomes? Should refer to existing efficacy or effectiveness studies IHS CER should refer to prior evidence of efficacy or
effectiveness of components of current intervention, or of effectiveness of the interventions in smaller studies or other settings
Addressing Potential (cont.)
Summarize ongoing studies of the interventionDiscuss the likelihood that positive study findings related to improvements in practice and patient outcomes would be implemented widely and quickly Breadth of current use in existing practice Extent of patient, practice or organizational barriers and facilitators to
rapid adoption by patients, clinicians and relevant health organizations
Applicability to diverse delivery systems Endorsement of the current study by key patient, physician and other
stakeholder groups.Describe how the partners in your proposal (e.g., national and/or regional stakeholder organizations) would help disseminate the study’s findings to potential adopters
Merit Review CriteriaTechnical Merit and Methodology Standards
David Hickam, MD, MPHProgram Director, Clinical Effectiveness Research
Technical Merit
A clear research plan with rigorous methods that adhere to PCORI’s Methodology Standards and accepted best practicesA clear justification for the study designA carefully constructed and realistic timeline that includes specific scientific and engagement milestones A research team with documented appropriate expertise and experience - and a rational organizational structureAn excellent research environment, including the appropriate delivery system(s) to host the study, adequate resources, and strong support of the proposed research
Addressing Technical Merit
Describe how all relevant PCORI Methodology Standards are addressedJustify the proposed sample size estimates, including the anticipated effect size and standard deviation, and all other relevant parametersDescribe anticipated rates of and feasibility of recruitment Justify the proposed study designDescribe the validity of the proposed measures of patient outcomesDiscuss proposed analytic methods, including subgroup analysesDiscuss proposed approach to consent and protection of human participants
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 set of methodological standards for “pragmatic studies.” Consider design tradeoffs (e.g., blinding vs not blinding) Refer to other respected sources for additional guidance. View here: http://www.pcori.org/assets/2013/11/PCORI-
Methodology-Report.pdf
Sample Size Estimate and Subgroups
Document previous relevant studies to justify use of the proposed effect size.Assure appropriate adjustments based on the particular study circumstances, including the study population, expected dropout and crossover, and other parameters.Demonstrate sufficient power to analyze important pre-specified subgroups; these subgroups should be based on patient attributes with strong a priori pathophysiological or empirical justification.
Cluster Randomized Trial Design
Need justification for the use of this design State the benefits and disadvantages of a cluster design
vs. individual randomization, based on the clinical issues and the interventions being studied.
Sample size estimates need to account for reasonable estimates of intraclass correlation.Maximize the number of clusters that are randomized.
Merit Review Criteria Patient-Centeredness and Patient Engagement
Sue Sheridan, MIM, MBA, DHL
Program Director, Patient Engagement
Patient-Centeredness
Is the research focused on questions that affect outcomes 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?
Does the research address one or more of the key questions mentioned in PCORI’s definition of patient-centered outcomes research?
Addressing Patient-Centeredness
Provide evidence that the research question(s) and outcomes are important to patients (and/or their caregivers) Have you obtained the endorsement and participation of
major patient/caregiver organizations that would use the research?
Describe your strategy for measuring outcomes that are important to patients.
Selecting Important Patient-Centered Outcomes
Do proposed outcomes include outcomes based on input from patients and other stakeholders?What is the validity of this outcome measure?What is a minimal important difference in this outcome measure?Have you considered the use of a previously validated patient reported outcome (PRO)?
Patient and Stakeholder Engagement
Evidence that patients, caregivers, patient and caregiver organizations, clinician organizations, and other stakeholders have been and will be engaged in: Formulating the research questions Defining the characteristics of study participants, comparators and
outcomes 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 Demonstration of the principles of reciprocal relationships, co-learning, partnership, trust, transparency, and honesty
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 opposed 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 officers (for creating milestones and monitoring projects) regarding engagement in the conduct of research. It is divided into four segments:
Planning the Study
Conducting the Study
Disseminating the Study Results
PCOR Engagement Principles
Addressing Engagement
Several approaches to engagement can succeedPCORI provides many engagement resources: PCORI’s “The 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/announcement-
resources/PCORI-Sample-Engagement-Plans.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
Other Topics (EHRs, Recruitment, Research Protections, & Dissemination)
Anne Trontell, MD, MPHSenior Program Officer, Clinical Effectiveness Research
Use of Electronic Health Records and Other Computerized Data Sources
Pragmatic studies should take advantage of existing electronic clinical and demographic data whenever possible.Discuss any proposed uses of existing electronic data in the approach section: Cohort identification and recruitment Collection of covariate and outcome data
Provide evidence of the validity and completeness of available data (e.g., that all follow-up and outcomes of interest are captured) PCORI Standards on Data Networks and Registries
Recruitment
Discuss past experiences with recruitmentProvide best available evidence to support your estimated recruitment successConsider barriers to recruitment – and how you plan to overcome themSome strategies for successful recruitment Engaged clinical sites Clinician advocates for the study Proactive, experienced research coordinator Minimize disruptions in clinical care processes Protocol flexibility, within reason
Study Participant Protection
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 If you anticipate seeking waiver of individual informed
consent, provide the rationale Refer to NIH standards for research involving human
subjects
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
Disseminating and Implementing Research Findings
PCORI does not expect applicants to disseminate and implement findings at this time. Applicants should describe the potential for dissemination and implementation.
Describe the potential for disseminating and implementing the study results in other settings.Describe possible barriers to dissemination and implementation.
What To Think About While Completing Your Application
Iris Giggetts, MSW, CRAContracts Associate, Pre-Award
Letter of Intent and Application
You were invited to submit a full application based on the information provided in the LOI, changes after the LOI require PCORI approval.
Show stoppers include:
Changes to the PI
Changes to the
Institution
Changes to the Study
Design
Changes in Engagement
PCORI Online: Application
► PI and Contact Information► Project Information► Key Personnel► Milestones► Templates and Uploads► Save and Review
Public Abstract
Project Information
Technical Abstract
Project Narratives
Milestones
► PI and Contact Information► Project Information► Key Personnel► Milestones► Templates and Uploads► Save and Review
PCORI Monitors Projects
Deliverables:Measurable and verifiable
outcomes 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
Templates and Uploads
► PI and Contact Information► Project Information► Key Personnel► Milestones► Templates and Uploads► Save and Review
People and Places Template: Biosketch
You may use the NIH biosketch or PCORI’s formatBiosketches are required for all key personnelList all partners within the Key Personnel sectionPatient/Stakeholder Biosketch
PageLimit
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.
PageLimit15
Professional Profile/Biosketch
Budget Templates
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 project. Complete each budget section for the prime applicant and any/each subcontractor.
Budget and Period Limitations
Funds & BudgetDirect costs up to $10 million over the life of the projectIndirect costs: up to 40% capped for the prime and sub contractsPrime organization can only charge up to 40% IDC on first $25,000 for all sub contracts combinedInstitutional base salary up to $200,000The limit for Scientific Travel is $10,000 over the duration of the project. There is no cap on Programmatic Travel.
Period of Performance Maximum of 5 yearsRequests to extend are not permitted during any stageDo not anticipate receiving a cost OR no-cost time extension.Propose realistic timelines accounting for the burdens associated with obtaining IRB approval
Budget JustificationProvide a narrative that fully supports and explains the basis for the information found in the Detailed Budget
Provide sufficient detail to understand the basis for costs, the reason that the costs are necessary, and an explanation for major cost variances
– Provide justification as to why the costs are reasonable for the work to be performed
Provide a 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, and 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 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.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 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 questions.
Letters of SupportAll letters of support should be addressed to the PI and institution to demonstrate the commitment of key personnel and supporting organizations 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.
Please be sure to use the Letters of Support Table provided in the funding center.
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
General Guidance
Refer to the Pragmatic Studies page in our Funding Center: http://www.pcori.org/funding-opportunities/announcement/large-pragmatic-studies-evaluate-patient-centered-outcomes-spring
Use the applicant checklist.Adhere to the formatting, page limit, and template requirements.Convert all documents to PDF and upload to PCORI Online using the appropriate filing convention.Use Biosketches for all key personnel (scientists and patient/stakeholder team members).Address Letters of Support to the PI’s institution.
Common Application Errors
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 and Review’ 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
Resources
Refer to the Pragmatic Studies page in our Funding Center for the following resources
PFA and Application GuidelinesPCORI Online User ManualsSample Engagement PlansGeneral Applicant FAQs: Large Pragmatic Studies Applicant FAQs: PCORI Online: https://pcori.fluxx.io/Research Methodology: http://www.pcori.org/node/4020
Submission and Key Dates
What When
Application Deadline (by invitation only)
July 31, 2015 by 5:00pmET
Merit Review Dates October 29-30, 2015
Awards Announced January 2016
Earliest Start Date March 2016
Questions and Answers
Submit questions via the chat function in Meeting Bridge
Ask a question via phone (press 7)
Contact Us:• Programmatic Questions: E-mail [email protected]• Schedule a call at http://bit.ly/programmatic_inquiry• Call 202-627-1884 • Administrative Questions: E-mail [email protected] or call 202-627-1885