PCORI’s Approach to Providing Support for
Comparative Effectiveness Research
PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE
...
Key Questions for this Presentation

PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE

• What is patient centered outcomes res...
Preaching to the Choir?

PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE

PCORI Awards to UCSF Investigators
David Thom Healt...
Preaching to the Choir?

PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE

PCORI Awards to UCSF Investigators (cont.)
Tung Ngu...
Preaching to the Choir?

PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE

PCORI Awards to UCSF Investigators (cont.)
Rebecca ...
PCORI Aims to Improve Healthcare
Decision Making
Making
PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE

• An independent, no...
What is Patient Centered Outcomes
Research?
PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE

Patient-Centered Outcomes Resear...
PCORI Funding – Approximate

PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE

2012

$150,000,000*

2013

$300,000,000

2014

...
PCORI’s Rationale for Engagement
PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE

Advise Us on What PCORI
Advise Us on What P...
Our National Priorities for Research
PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE

10
Characteristics of Projects that will Receive
PCORI Funding
PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE

• The studies ad...
Two Pathways to PCORI Funding
Investigator-Initiated or “Broad” Pathway”

PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE

Di...
Two Pathways to PCORI Funding
PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE

Patient/Stakeholder-Initiated Pathway – “TARGE...
PCORI Targeted Funding Programs
PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE

Programs launched in 2013:
•Treatment option...
Some Projects are Outside of PCORI’s
Priorities
PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE

•Cost-effectiveness analyses...
Patients are Engaged in Reviewing Research
Applications
PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE

• PCORI uses multi-s...
Criteria Used by PCORI Merit
Review Panels
PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE

• Impact of the condition on the ...
Which Strategies are Likely to be Successful
when Seeking Funding in 2014?

PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE

...
Program on Assessment of Prevention,
Diagnosis, and Treatment Options

PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE

Devel...
Targeted Funding Initiatives in the
Assessment of Options Program

PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE

New clini...
Addressing Disparities Program
PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE

PCORI’s
Vision, Mission, Strategic Plan

Prog...
Improving Healthcare Systems Program
PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE

PCORI’s IHS program will incorporate th...
Focus of Research in the IHS Program
PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE

Innovations in:
Use of health informati...
Accelerating PCOR and
Methodologic Research
PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE

• Methods
– Broad announcement, ...
PCORI Requires Adherence to its
Methodology Standards
PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE

47 Individual Methodol...
Characteristics of the Methodology
Standards

PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE

• Are minimal standards for pe...
The Methodology Standards are Reasonable

PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE

• Engage people representing the p...
The Methodology Standards are Reasonable
but also Rigorous

PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE

Report the assum...
Conclusions

• Patient-centered outcomes research (PCOR) provides
relevant information to help patients and providers choo...
Join Us at PCORI.org

PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE

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  • Why was PCORI created?
    For all of the research conducted; for all of the therapies created; for all of the advances in care that have been made – Patients do not have in many cases the information they need to make choices that reflect their unique situation, priorities or personal preferences.
    Many patients find themselves drowning in a sea of information. From the internet to direct-to-consumer marketing, patients have access to more health information than ever, but in many ways we are “information rich” and “wisdom poor.” We do not have sufficient tools to analyze and appropriately apply that information to our care.
    In some cases, where good evidence exists, we have failed to give patients information they can easily access, understand and utilize.
    For many areas, research has yet to ask some of the most important questions patients have. Take chronic back pain, for example, where surgery, physical therapy or medication are among the treatment options. Patients do not have reliable information that will tell them what the comparative differences are in terms of their short- and long-term functionality and quality of life.
    PCORI is an opportunity to do a better job of disseminating existing quality research information, while answering some of patients’ persistent questions.
  • Why was PCORI created?
    For all of the research conducted; for all of the therapies created; for all of the advances in care that have been made – Patients do not have in many cases the information they need to make choices that reflect their unique situation, priorities or personal preferences.
    Many patients find themselves drowning in a sea of information. From the internet to direct-to-consumer marketing, patients have access to more health information than ever, but in many ways we are “information rich” and “wisdom poor.” We do not have sufficient tools to analyze and appropriately apply that information to our care.
    In some cases, where good evidence exists, we have failed to give patients information they can easily access, understand and utilize.
    For many areas, research has yet to ask some of the most important questions patients have. Take chronic back pain, for example, where surgery, physical therapy or medication are among the treatment options. Patients do not have reliable information that will tell them what the comparative differences are in terms of their short- and long-term functionality and quality of life.
    PCORI is an opportunity to do a better job of disseminating existing quality research information, while answering some of patients’ persistent questions.
  • I want to say a few words about what we mean and why we say that we will engage, intensely and continuously. with patients and other key stakeholders in the research process. First, we want patients to help us decide what to study. This means raising the research questions and then joining with other stakeholders to prioritize those questions. We’ll fund the high priority questions. Then, after we decide what we want to fund, we invite researchers to apply. When we review their applications, there are patients and other stakeholders ON THE REVIEW PANELS.
    They help identify the highest quality applications, looking especially at whether the research got the questions right. But beyond the review, we will have patients participating on the research teams. In fact, you don’t get funded if you don’t have relevant patients and other relevant stakeholders involved in the entire project. Why?? Because we think that will increase the chances that the research is actually relevant and sticks to its purpose of answering patient-driven questions. And why do we do all of that?? Because we b
    Finally, our Advisory Panels will have….
  • The second approach is unique to PCORI and involves reaching out to the wider community to ask patients and stakeholders about the questions that matter to them, and what information would help them make better informed health decisions. [Read the slide]
  • Why was PCORI created?
    For all of the research conducted; for all of the therapies created; for all of the advances in care that have been made – Patients do not have in many cases the information they need to make choices that reflect their unique situation, priorities or personal preferences.
    Many patients find themselves drowning in a sea of information. From the internet to direct-to-consumer marketing, patients have access to more health information than ever, but in many ways we are “information rich” and “wisdom poor.” We do not have sufficient tools to analyze and appropriately apply that information to our care.
    In some cases, where good evidence exists, we have failed to give patients information they can easily access, understand and utilize.
    For many areas, research has yet to ask some of the most important questions patients have. Take chronic back pain, for example, where surgery, physical therapy or medication are among the treatment options. Patients do not have reliable information that will tell them what the comparative differences are in terms of their short- and long-term functionality and quality of life.
    PCORI is an opportunity to do a better job of disseminating existing quality research information, while answering some of patients’ persistent questions.
  • Pcori hickam ucsf 1 16 2014 ver 2

    1. 1. PCORI’s Approach to Providing Support for Comparative Effectiveness Research PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE How to Improve your Likelihood of Success David H. Hickam, MD MPH Director, Clinical Effectiveness Program Patient-Centered Outcomes Research Institute (PCORI) UCSF Symposium San Francisco, CA January 16, 2014 1
    2. 2. Key Questions for this Presentation PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE • 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? 2
    3. 3. Preaching to the Choir? PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE 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 3
    4. 4. Preaching to the Choir? PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE PCORI Awards to UCSF Investigators (cont.) Tung Nguyen A Patient-Centered Intervention to Increase Screening of Hepatitis B and C Among AsianAmericans 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 4
    5. 5. Preaching to the Choir? PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE 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 5
    6. 6. PCORI Aims to Improve Healthcare Decision Making Making PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE • 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 betterinformed health care decisions. 6
    7. 7. What is Patient Centered Outcomes Research? PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE 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 7 care environments.
    8. 8. PCORI Funding – Approximate PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE 2012 $150,000,000* 2013 $300,000,000 2014 $500,000,000 2015 $500,000,000 .... 2019 8 $500,000,000 * 20% of this amount goes to AHRQ/HHS each year for
    9. 9. PCORI’s Rationale for Engagement PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE Advise Us on What PCORI Advise Us on What PCORI Should Study: Should Study: Topic generation Research prioritization Tell Us How We Are Doing Tell Us How We Are Doing How can we improve on what we are doing? 9 Review Proposals and Review Proposals and Partner in Research Partner in Research Patients Patients and and Stakeholders Stakeholders Merit Review Participate in research Help Us Share the Findings Help Us Share the Findings Dissemination Implementation
    10. 10. Our National Priorities for Research PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE 10
    11. 11. Characteristics of Projects that will Receive PCORI Funding PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE • 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. 11 11
    12. 12. Two Pathways to PCORI Funding Investigator-Initiated or “Broad” Pathway” PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE Diverse portfolio addressing Diverse portfolio addressing high-priority questions high-priority questions 12 12
    13. 13. Two Pathways to PCORI Funding PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE Patient/Stakeholder-Initiated Pathway – “TARGETED” 13 Focused portfolio addressing Focused portfolio addressing high-priority questions high-priority questions
    14. 14. PCORI Targeted Funding Programs PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE 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. 14
    15. 15. Some Projects are Outside of PCORI’s Priorities PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE •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. 15 15
    16. 16. Patients are Engaged in Reviewing Research Applications PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE • 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. 16
    17. 17. Criteria Used by PCORI Merit Review Panels PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE • 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 17
    18. 18. Which Strategies are Likely to be Successful when Seeking Funding in 2014? PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE • 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. 18
    19. 19. Program on Assessment of Prevention, Diagnosis, and Treatment Options PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE 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. 19
    20. 20. Targeted Funding Initiatives in the Assessment of Options Program PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE 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 20
    21. 21. Addressing Disparities Program PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE PCORI’s Vision, Mission, Strategic Plan Program’s Mission Statement To reduce disparities in healthcare outcomes and advance equity in health and health care Program’s Guiding Principle PCORI is not interested in studies that describe disparities; instead, we want studies that will identify best options for eliminating disparities. 21
    22. 22. Improving Healthcare Systems Program PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE 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. 22
    23. 23. Focus of Research in the IHS Program PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE Innovations in: Use of health information technology Deployment of human resources Incentives to drive system improvement Special clinical programs 23
    24. 24. Accelerating PCOR and Methodologic Research PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE • 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 24
    25. 25. PCORI Requires Adherence to its Methodology Standards PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE 47 Individual Methodology Standards • Formulating Research Questions • Patient-Centeredness • Data Integrity and Rigorous Analyses • Preventing/Handling Missing Data • Heterogeneity of Treatment Effects 25 • Data Networks • Data Registries • Adaptive and Bayesian Trial Designs • Causal Inference • Studies of Diagnostic Tests • Systematic Reviews
    26. 26. Characteristics of the Methodology Standards PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE • 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. 26
    27. 27. The Methodology Standards are Reasonable PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE • 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. 27
    28. 28. The Methodology Standards are Reasonable but also Rigorous PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE 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. 28
    29. 29. 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. 29
    30. 30. Join Us at PCORI.org PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE 30

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