Cycle 3 2016: Pragmatic Studies to Evaluate Patient-Centered - PowerPoint PPT Presentation

Cycle 3 2016: Pragmatic Studies to Evaluate Patient-Centered Outcomes Applicant Town Hall Date November 9, 2016

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

Agenda Welcome Overview Merit Review Criteria Other topics What to think about before you apply Submit questions via the Questions? chat function in Meeting Bridge. Ask a question via phone (an operator will standby to take your questions).

Overview Overview Anne Trontell , MD. MPH Associate Director Clinical Effectiveness Research

Purpose of Town Hall To 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” Applicants proposing “CER to communicate or disseminate research results to patients, caregivers, or clinicians”

What is a Pragmatic CER Study? Answers a practical, real world comparative effectiveness research question – a decisional dilemma Assesses whether two or more options differ in effectiveness when administered as they are in real life (vs. tightly controlled research situations) 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.

Justify the Design Elements in Your Large Pragmatic Study Suggest reviewing pragmatic – explanatory continuum indicator summary (PRECIS) tool Consider tradeoffs in relation to your research question  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 to develop, validate, or test the efficacy of new or existing decision support tools Efficacy trials Evidence syntheses Cost-effectiveness analysis Research that aims to compare the overall costs of care between two or more alternatives and use the results to determine the preferred alternative

Addressing Disparities Ayodola Anise, MHS Program Officer

Studies Specifically Addressing Disparities We seek pragmatic clinical trials to test the comparative effectiveness of evidence-based interventions set in real world settings for patients who are at risk for experiencing disparities in outcomes Study should test the ability of interventions to improve outcomes and reduce disparities for at risk populations Target populations include:  Racial and ethnic minorities  Low-income groups  Residents of rural areas  Individuals with disabilities  Patients with low health literacy/numeracy and limited English proficiency  Lesbian, gay, bisexual, transsexual

Studies Specifically Addressing Disparities (cont.) Studies should measure a broad range of patient-centered, clinical, and structural outcomes that contribute to reducing disparities in care We encourage applications from organizations that serve majority underserved populations (e.g., safety net settings) Studies addressing populations at risk for disparities may:  Require a more frequent, comprehensive and diverse set of outcomes data to adequately capture the impact of the intervention (e.g., follow-up that takes place outside of clinical setting, additional validated measures to assess outcomes).  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.

Merit Review Criteria Potential to Fill Evidence Gaps and Improve Care Penny Mohr, MA Senior Program Officer Improving Healthcare Systems

Merit Review Criteria Crosswalk of PCORI Merit Review Criteria with NIH Criteria 1. Potential for the study to fill critical gaps in evidence SIGNIFICANCE 2. Potential for the study findings to be adopted into clinical practice and improve delivery of care 3. Scientific merit (research design, analysis, and APPROACH outcomes) NEW 4. Investigator(s) and environment PCORI-only Merit Review Criteria PATIENT-CENTEREDNESS/ 5. Patient-centeredness ENGAGEMENT 6. Patient and stakeholder engagement

Potential to fill critical gaps The application should clearly define and describe: A clear clinical burden A critical gap in current knowledge based on systematic reviews, clinical practice guidelines, or previous research prioritizations Variations in practice patterns that suggest clinical uncertainty Decisional dilemmas experienced by patients and other stakeholders Does the study have the potential to fill these gaps and inform decision making for key stakeholders

Addressing Potential to Fill Critical Gaps Clinical CER  Describe the decisional dilemma from information gathered directly from stakeholders and patients, or based on evidence synthesis efforts and prioritization Improving Healthcare Systems CER  Discuss the impact of the system problem on healthcare access and quality – and on individual and population suffering, morbidity, mortality, productivity and costs CER to Address Disparities  Describe the critical gaps in evidence related to health, health care, and health outcomes for sub-populations with known health disparities

Potential for the Study’s Findings to be Adopted into Clinical Practice and Improve Delivery of Care Describe how evidence generated from this study could be adopted into clinical practice and care delivery Who are the end-users and how would the information from this study support a demand for information from end-users? How likely are the findings to be reproduced by others? What are the barriers? What is the dissemination plan for study results, beyond traditional publication and presentation?

Addressing Potential for Study Findings to be Adopted into Clinical Practice 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  CER to Address Disparities should refer to existing efficacy or effectiveness studies in the target population where possible, or highlight efficacy or effectiveness studies in the general population that may be promising for the target population

Addressing Potential (cont.) Discuss 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 Criteria Scientific Merit and Methodology Standards; Investigators and Environment Nora McGhee, PhD Senior Merit Review Officer

Scientific Merit 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 A carefully constructed and realistic timeline that includes specific scientific and engagement milestones Realistic strategies for participant recruitment and retention

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.