pcori hickam ucsf 1 16 2014 ver 2
TRANSCRIPT
PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE
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UCSF SymposiumSan Francisco, CAJanuary 16, 2014
David H. Hickam, MD MPHDirector, Clinical Effectiveness ProgramPatient-Centered Outcomes Research Institute (PCORI)
PCORI’s Approach to Providing Support for Comparative Effectiveness Research
How to Improve your Likelihood of Success
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Key Questions for this PresentationKey Questions for this Presentation
• What is patient centered outcomes research (PCOR)?
• How is PCORI promoting best practices in research?
– System for review of applications
– Methodology standards
• What types of projects have the best chance of receiving funding from PCORI?
• What are the pitfalls to avoid?
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Preaching to the Choir?
PCORI Awards to UCSF Investigators
David Thom Health Team Support for Patient Informed Decision Making
Stephen Hauser A BioScreen for Multiple Sclerosis
Carol Mathews Comparison of Peer-Facilitated Support Group and Cognitive Behavioral Therapy for Hoarding Disorder
Christine Dehlendorf Patient-Centered Support for Contraceptive Decision Making
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Preaching to the Choir?
PCORI Awards to UCSF Investigators (cont.)
Tung Nguyen A Patient-Centered Intervention to Increase Screening of Hepatitis B and C Among Asian-Americans
Mary Whooley Improving Delivery of Patient-Centered Cardiac Rehabilitation
Anna-Barbara Moscicki Randomized Trial to Increase Adherence to Cervical Cancer Screening Guidelines for Young Women
Rebecca Smith-Bindman UCSF CT Radiation Dose Registry to Ensure a Patient-Centered Approach for Imaging
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Preaching to the Choir?
PCORI Awards to UCSF Investigators (cont.)
Rebecca Sudore Preparing Spanish-speaking Older Adults for Advance Care Planning and Medical Decision Making
David Thom Health Coaching to Reduce Disparities for Patients with Chronic Obstructive Pulmonary Disease
Charles McCulloch Methods for Analysis and Interpretation of Data Subject to Informative Visit Times
Mark Pletcher The Health eHeart Alliance
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PCORI Aims to Improve Healthcare Decision Making Making
• An independent, non-profit organization authorized by Congress.
• Mission is to fund research that will provide patients, their caregivers and clinicians with the evidence-based information needed to make better-informed health care decisions.
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What is Patient Centered Outcomes Research?
Patient-Centered Outcomes Research (PCOR) helps people and their caregivers communicate and make informed health care decisions.
PCOR is a type of comparative effectiveness research.
PCOR has the following characteristics:
•Actively engages patients and key stakeholders throughout the research process.
•Compares important clinical management options.
•Evaluates the outcomes that are most important to patients.
•Addresses implementation of the research findings in clinical care environments.
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PCORI Funding – ApproximatePCORI Funding – Approximate
2012
2013
2014
2015. . . .
2019
$150,000,000*
$300,000,000
$500,000,000
$500,000,000
$500,000,000
* 20% of this amount goes to AHRQ/HHS each year for dissemination and capacity-building
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Advise Us on What PCORIShould Study:
Advise Us on What PCORIShould Study:
Tell Us How We Are DoingTell Us How We Are Doing
Review Proposals andPartner in Research
Review Proposals andPartner in Research
Help Us Share the Findings
Help Us Share the Findings
Patientsand
Stakeholders
Patientsand
Stakeholders
Topic generationResearch prioritization
Merit ReviewParticipate in research
How can we improve on what we are doing?
DisseminationImplementation
PCORI’s Rationale for Engagement
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Our National Priorities for Research
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Characteristics of Projects that will ReceiveCharacteristics of Projects that will Receive PCORI FundingPCORI Funding
• The studies address critical decisions that face patients, their caregivers, and clinicians.
• The decisions must be consequential and be occurring without key evidence about the comparative effectiveness of two or more options.
• The studies address comparative benefits and harms of the options and provide information about outcomes that are important to patients.
• The new knowledge can be used by patients, caregivers, and their clinicians for making decisions.
• The studies Include relevant patients and other stakeholders on the research team.
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Two Pathways to PCORI Funding
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Diverse portfolio addressing high-priority questions
Diverse portfolio addressing high-priority questions
Investigator-Initiated or “Broad” Pathway”
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Patient/Stakeholder-Initiated Pathway – “TARGETED”
Focused portfolio addressing high-priority questions
Focused portfolio addressing high-priority questions
Two Pathways to PCORI Funding
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Programs launched in 2013:
•Treatment options for uterine fibroids in women of childbearing age.
•Prevention of falls in residents of assisted living facilities.
•Improving the care of asthma in Latino and African American populations.
PCORI Targeted Funding Programs
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Some Projects are Outside of PCORI’sSome Projects are Outside of PCORI’s PrioritiesPriorities
•Cost-effectiveness analyses or studies that primarily address costs of care as an outcome.
•Studies that develop new methods of measurement.
•Studies of the efficacy of unproven treatments.
•Natural history studies.
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Patients are Engaged in Reviewing Research Patients are Engaged in Reviewing Research ApplicationsApplications
• PCORI uses multi-stakeholder review panels to review the applications submitted by investigators.
– 50% of the members of review panels are either patients or other key stakeholders.
• The panels evaluate and score the applications and stakeholder engagement.
• Patients and other relevant stakeholders must be included as members of research teams and involved in all aspects of the project.
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Criteria Used by PCORI Merit Review Panels
• Impact of the condition on the health of individuals and populations
• Potential for the study to improve health care and outcomes
• Technical merit• Patient-centeredness• Patient and stakeholder engagement
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Which Strategies are Likely to be Successful when Seeking Funding in 2014?
• PCOR should compare clear clinical options.
• Be cautious with a “usual care” comparator.
• The clinical interventions should be easy to replicate: path to dissemination.
• Do not neglect engagement.
• Make sure that the outcomes are meaningful.
• Address both benefits and harms.
• Cover all of the Methodology Standards.
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Program on Assessment of Prevention, Diagnosis, and Treatment Options
Developing a portfolio of comparative effectiveness research that assesses important clinical management options.
•Broad range of clinical populations.
•Studies of specific clinical interventions.
• Procedures, medications.
• Care strategies and tools.
•Have funded 67 projects in first four cycles.
•Portfolio includes both clinical trials and observational studies.
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Targeted Funding Initiatives in the Assessment of Options Program
New clinical areas being developed for targeted initiatives
– Management strategies for ductal carcinoma in situ
– Treatment of bipolar disorder in adolescents and adults
– Treatments for osteoarthritis
– Treatments for migraine headaches
– Announcements due in 2014
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Addressing Disparities Program
Program’s Mission StatementTo reduce disparities in healthcare outcomes and advance equity in
health and health care
Program’s Guiding PrinciplePCORI is not interested in studies that describe disparities; instead,
we want studies that will identify best options for eliminating disparities.
PCORI’s Vision, Mission, Strategic Plan
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Improving Healthcare Systems Program
PCORI’s IHS program will incorporate the perspectives of patients, stakeholders and scientists to produce new
scientific evidence that will:
•Improve the quality and efficiency of critical components of the US healthcare system
•Improve the health-related outcomes that matter most to patients and their caregivers
•Inspire others to emulate PCORI’s success.
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Focus of Research in the IHS Program
Innovations in:
Use of health information technology
Deployment of human resources
Incentives to drive system improvement
Special clinical programs
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Accelerating PCOR and Methodologic Research
• Methods– Broad announcement, every 4 months, ~ $12 mil– Up to $250,000 direct costs per year, up to 3 years– Analytic methods, outcomes measurement, engagement
methods– Increasingly will align with Methodology Committee
priorities
• Data Infrastructure Announcements– Clinical Data Networks– Patient-Powered Networks– Coordinating Center
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PCORI Requires Adherence to its PCORI Requires Adherence to its
Methodology StandardsMethodology Standards
• 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
47 Individual Methodology Standards
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Characteristics of the Methodology StandardsCharacteristics of the Methodology Standards
• Are minimal standards for performing comparative effectiveness research.
• Are intended to provide helpful guidance to researchers and those who use research results.
• Reflect generally accepted best practices.
• Provide guidance for both project protocols and reporting of results.
• Are used to assess the scientific rigor of funding applications.
• Context of research should drive use of the standards.
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The Methodology Standards are Reasonable
• Engage people representing the population of interest and other relevant stakeholders in ways that are appropriate and necessary in a given research context.
• Use Patient-Reported Outcomes When Patients or People at Risk of a Condition Are the Best Source of Information.
• Document Validated Scales and Tests.
• Specify Plans for Data Analysis that Correspond to Major Aims.
• Use Validated Methods to Deal with Missing Data that Properly Account for Statistical Uncertainty Due to Missingness.
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The Methodology Standards are Reasonable but also Rigorous
Report the assumptions underlying the construction of propensity scores and the comparability of the resulting groups in terms of the balance of covariates and overlap.
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Conclusions
• Patient-centered outcomes research (PCOR) provides relevant information to help patients and providers choose among alternative clinical strategies.
• Applicants for funding should engage patients and stakeholders in identifying questions and defining important outcomes.
• Projects should directly address important clinical comparisons and examine meaningful outcomes.
• The PCORI Methodology Standards guide best practices for planning, conducting and reporting research.
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Join Us at PCORI.orgJoin Us at PCORI.org