Post on 01-Apr-2015
EPG Grand Rounds, University of Maryland
February 6, 2014
Patricia Deverka, MD, MS
STAKEHOLDER –DEFINED RESEARCH DESIGNING STUDIES THAT REFLECT THE
PERSPECTIVES OF PATIENTS, CLINICIANS AND PAYERS
Center for MedicalTechnology Policy
The Center for Medical Technology Policy (CMTP) is an independent ,non-profit 501(c) (3) organization that seeks to advance health care innovation and effectiveness by improving the quality, relevance, and efficiency of health care research. CMTP works on methods, infrastructure and policy to support the conduct of comparative effectiveness research that generates information to assist patients, clinicians, and payers in making informed clinical and health policy decisions.
OVERVIEW
• Rationale for engaging stakeholders• Definitions and approach• Case examples• Generative discussion
• How to reconcile:• ~18,000 RCTs are published each year*• A growing number of non-experimental studies• Many systematic reviews, health technology
assessments, clinical guidelines conclude that the available evidence is limited or studies are poor quality• Up to 60% of clinical recommendations made by ACC or AHA
based on expert opinion and/or low quality studies• Systematic review of off-label uses of 19 FDA-approved oncology
drugs (428 pages, several thousand trials) “Because of the paucity of high quality evidence, the data available – though voluminous – may have little meaning or value for informing clinical practice”
THE EVIDENCE PARADOX
* Chalkidou, Tunis, Whicher, et al. The role for pragmatic, randomized controlled trials (pRCTs) in comparative effectiveness research. Clin icalTrials .Published online before print July 2, 2012, doi: 10.1177/1740774512450097
REASONS EVIDENCE PRODUCED BY CURRENT CLINICAL RESEARCH ENTERPRISE NOT TRANSLATED INTO PRACTICE
• Differences between settings where research is conducted and where medicine is practiced– Patient population– Interventions, including usual care– Providers, referral patterns access to care
• Failure to (be able to) report how treatment effects vary in individual patients and subgroups
• Underrepresentation of children, women, elderly, ethnic & racial minorities, patients with comorbidities
• Research priorities, study questions, endpoints, etc. defined by researchers and funders, not end users
THE CER HYPOTHESIS
• Gaps in evidence will be reduced with increased guidance from payers, patients and clinicians in study design
• A functional definition of CER would be research designed in light of meaningful engagement of these decision makers
DEFINITIONS OF CER AND PCOR
Definition of CER
The generation and synthesis of evidence that compares the benefits and harms of alternative methods to prevent, diagnose, treat, and monitor a clinical condition or to improve the delivery of care. The purpose of CER is to assist consumers, clinicians, purchasers, and policy makers to make informed decisions that will improve health care at both the individual and population levels.
Source: Institute of Medicine
Definition of PCOR
Patient-Centered Outcomes Research (PCOR) helps people and their caregivers communicate and make informed health care decisions, allowing their voices to be heard in assessing the value of health care options.
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 finings in clinical care environments.
Source: PCORI
WHAT IS UNIQUE ABOUT CER?
• Many CER studies will require an understanding of the trade-offs between internal validity and increased generalizability, relevance, feasibility and timeliness
• The right balance is not solely a scientific issue, it’s also a social judgment about an acceptable level of uncertainty, involving multiple stakeholders
• Process to achieve this with stakeholder input is evolving
Relevance and ContentRelevance and Content
Knowledge Exchange
Application of Evidence
Dissemination Avenues
Evidence suggests that engaging stakeholders in research increases:
THEORY OF STAKEHOLDER ENGAGEMENT
BARRIERS TO INVOLVING STAKEHOLDERS IN CER
• Confusing terminology, lack of standard definitions
• Timing; restrictions on availability of stakeholders
• Training needs for all stakeholders to maximize participation
• Concerns that process will add time and costs to project plans
• Lack of shared conceptualization of what it means to
“successfully” or “effectively” involve stakeholders in research
• Limited data regarding impact; systematic evaluation rareSources: Guise, O'Haire, McPheeters, et al. A practice-based tool for engaging stakeholders in future research: a synthesis of current practices. J Clin Epidemiol. 2013 Jun;66(6):666-74. doi: 10.1016/j.jclinepi.2012.12.010. Epub 2013 Mar 13. and CMTP experience
ADDRESSING THE BARRIERS
• Literature review– Biomedical– Social science
• Practical experience based on projects involving stakeholders
• Drafted definitions and conceptual model• Review and revision by an expert panel
– Patient and Consumer Advisory Council– NICE Patient and Public Involvement Program and Citizen
Council in the UK
• Applied it to a complex multi-stakeholder project
CBPR*• Reducing health disparities• Social change & action
Health care• Health technology
assessment• FDA• Health research (UK)
Public policy• Environmental
planning• Nuclear power• Biotechnology
Diverse Roots of Public Participation Activities
*Community-based participatory research
TYPOLOGY OF STAKEHOLDER ENGAGEMENT
Source: Nass, Levine, and Yancy. Methods for Involving Patients in Topic Generation for Patient-Centered Comparative Effectiveness Research –An International Perspective
STAKEHOLDER ENGAGEMENT
“LIGHT”
STAKEHOLDER
ENGAGEMENT
STAKEHOLDERS
Individuals, organizations, or communities that have a direct interest in the process and outcomes of a project, organization, or policy.
Deverka, Lavallee, Desai, et al. Stakeholder participation in comparative effectiveness research: defining a framework for
effective engagement. J Compar Effect Res 2012; 2:181-94.
TARGET STAKEHOLDER GROUPS FOR CER
STAKEHOLDER ENGAGEMENT
A process of actively soliciting the knowledge, experience, judgment and values of individuals selected to represent a broad range of direct interests in a particular issue, for the dual purposes of:
1) Creating a shared understanding;2) Making relevant, transparent, and effective
decisions.
Methods of combining evidence
Process Meta-criteria, Trust, Respect, Accountability, Legitimacy,
Fairness, Competence Change in Knowledge/attitudes Change in CER project decisions (e.g. choice of interventions,
study design, funding priorities)
CER More useful evidence for clinical and health policy decision making
More efficient use of healthcare resources Improved health outcomes.
Outputs
CONCEPTUAL MODEL FOR STAKEHOLDER ENGAGEMENT IN CER
A
naly
tic-D
elib
erati
ve M
odel
Methods
Inputs
Outcomes
Types of evidence·Values·Research·Professional Experience·Patient and consumer knowledge and experience
Decisions·Topic generation·Research priorities·Study designs·Evidentiary thresholds for clinical and health policy decision making·Implementation strategies
Quantitative· Questionnaires·Delphi method·Multi-Criteria Mapping·Value of Information modeling
Qualitative·Facilitated workshops/meetings · Stakeholder decision
analysis
DECIDING WHICH STAKEHOLDER GROUPS TO INVOLVE IN A PROJECT
• What topic(s) does the research address?• What health care decision is the research meant to
inform?• Who are the decision makers responsible for these
decisions?• Who are the individuals and groups that are
affected by these decisions?
Concannon TW, Meissner P, Grunbaum JA, et al. A new taxonomy for stakeholder engagement in patient-centered outcomes research. JGIM 2012;27(8):985-91.
WHEN TO INVOLVE PATIENTS AND OTHER STAKEHOLDERS IN RESEARCH?
• Topic identification and refinement• Priority-setting• Writing proposals (including deciding research methods)• Reviewing research conduct• Interpretation of findings• Dissemination of information• Implementation• Evaluation
Curtis, Slaughter-Mason, Thielke, et al. PCORI Expert Interviews Project: Final Report. Portland, OR: Center for Evidence-based Policy. Oregon Health & Sciences University.
Infra-structure*
• Assess needs• Recommend data
elements• Identify applications• Monitor
Research
• Generate & prioritize research questions
• Design studies• Data collection,
processing &analysis• Results interpretation
Education
• Translation • Dissemination• Evaluation• Continuous learning
healthcare system
*Refers to the creation/modification of the Patient Engagement Platform needed to support both clinical management and the conduct of PCOR
• Decision-making
• Data access• Data security• Data privacy• Conflicts of
interest• Consent• Communication• Coordination
with other committees
Adapted from: Rein A, Holve E, Hamilton Lopez M, and Winkler J. A framework for patient and consumer engagement in evidence generation,” EDM Forum,Academy-Health, September 2012.
STAKEHOLDER INVOLVEMENT IN GOVERNANCE
PATient-centered Involvement in Evaluating the effectiveNess of TreatmentS
PATIENTS
Aims of PATIENTS
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1) Foster sustainable partnerships with local, regional, and national communities of diverse patients and healthcare systems
2) Conduct and expand PCOR in partnership with patients and healthcare delivery systems
3) Advance dissemination and implementation strategies for PCOR findings
Vision
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The PATIENTS Program vision is that its projects will:
• Further the process of UM institutional transformation for “MPowering the State” in the area of health
• Eliminate health disparities within Baltimore, throughout Maryland, and across the nation
• Align with the spirit of the NIH roadmap for transformative and interdisciplinary research
University of Maryland has broad participation
• UMB professional schools– Pharmacy– Medicine– Nursing– Social Work– Dentistry– Law
• UM College Park
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PATIENTS Partners
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Innovation in the PATIENTS Program
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• Conducting PCOR with continuous patient and stakeholder engagement
• Translating research into practice
• Continuous development through formative and impact evaluation
• Bidirectional learning
• Sustainability
1. Topic Solicitation
2. Prioritization
3. Framing the Question
Public Announcements
Patient ForumsDelphi Process
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Based on: Mullins CD, Abdulhalim AM, Lavallee DC. Continuous Patient Engagement in Comparative Effectiveness Research. JAMA 2012; 307(15): 1587-8.
The Ten-Step Process for Conducting CER
The Ten-Step Process for Conducting CER
Based on: Mullins CD, Abdulhalim AM, Lavallee DC. Continuous Patient Engagement in Comparative Effectiveness Research. JAMA 2012; 307(15): 1587-8.
4. Selection of Comparators and Outcomes
5. Creation of Conceptual Framework
6. Analysis Plan
7. Data Collection
In-person Meetings
Focus Group Inter-views
Teleconferences
Electronic Social Media
Telephone Calls
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8. Reviewing & Interpreting Results
9. Translation
10. Dissemination
Teach-Back Method
Critique Documents (e.g. Patient Guides)
Media
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Based on: Mullins CD, Abdulhalim AM, Lavallee DC. Continuous Patient Engagement in Comparative Effectiveness Research. JAMA 2012; 307(15): 1587-8.
The Ten-Step Process for Conducting CER
Education and Training: Engaging Partners
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Bidirectional Learning
Research Methods
Manuscript Writing
Grant Writing
UM Faculty and Staff
Stakeholder Engagement
Cultural Competence
Using Online Platforms
Community Partners
Examples:
• PatientsLikeMe: Social Media
• BSBHS/Riverside Heath System: Implementation
EXAMPLES: STUDY DESIGNS REFLECTING VIEWS OF DECISION MAKERS
TWO EXAMPLES
• Genomic Testing in Cancer (CANCERGEN)
• Evidence Guidance Documents– Molecular Dx in Cancer
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THE PROMISE: TRANSFORM CANCER CARE
Molecular diagnostic (MDx) tests have the potential to transform oncology practice by helping physicians classify and manage various cancers
• Diagnose and stage cancers• Help guide therapy selection and
dosing• Assess treatment response• Aid in detection of residual or
recurrent disease
CANCERGEN STRUCTURE
Figure 1: CANCERGEN project milestones including landscape analysis, stakeholder assessment and final selection.
Thariani R, Wong W, Carlson JJ, et al. Prioritization in Comparative Effectiveness Research: The CANCERGEN Experience in Cancer Genomics. Medical Care 2012; 50(5):388-393
No Order 1. ERCC1 Expression Testing for Platinum-Based
Adjuvant Therapy in NSCLC
2. EGFR Mutation Testing for Erlotinib Maintenance Therapy in Advanced NSCLC
3. EGFR gene copy number (FISH) testing and first-line cetuximab therapy in stage IV or recurrent NSCLC
4. BRAF mutation testing in Colorectal Cancer
5. Genetic Expression Profile (GEP) in Multiple Myeloma (MM) to Identify Patients with Poor
Prognosis
6. Breast CA Tumor Markers for Detection of Recurrence After Primary Breast Cancer Therapy
Center for Comparative Effectiveness Research in Cancer Genomics (CANCERGEN)
RANKING OF TESTS
Final Order
1. ERCC1
2. BCTM
3. EGFR mutation
LESSONS LEARNED
• Full participation of all stakeholders on highly technical topics is possible with adequate preparation
• Multi-modal approaches are necessary– Engagement method should be matched to particular
study question– Stakeholders were open to novel methods (e.g., VOI), but
more work needs to be done to ensure full benefits of the approach are fully realized
• Possible to engage most stakeholders for a multi-year project– Federal officials are the most difficult
CASE STUDY: EFFECTIVENESS GUIDANCE DOCUMENTS
A STAKEHOLDER-DRIVEN APPROACH TOIMPROVING THE EVIDENCE BASE
Molecular Diagnostics (MDx) In Oncology
DECISION MAKERS’ KEY QUESTIONS FOR CANCER MDX TESTS
1. Does the MDx test provide correct information? (analytic validity)
2. How well does the test result correlate with clinical outcome? (clinical validity)
3. Does use of the MDx test lead to improved patient outcomes as compared with the alternative? (clinical utility)
4. Does use of the MDx test lead to greater value as compared with the alternative? (cost-effectiveness)
PROBLEM
• Analytic validity and clinical validity now available for an increasing number of MDx tests
• Clinical utility largely unknown for most MDx tests• Uncertain clinical utility has consequences for patients and
health care system– Decreases quality through inconsistent or unnecessary use of tests– Wastes health care resources
WHAT’S NEEDED
• Structured data about MDx test use• Stakeholder-driven process• Clear evidentiary standards for clinical utility• Willingness to consider range of methods,
outcome measures that are relevant to real-world clinical decisions
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THE RESPONSE: EFFECTIVENESS GUIDANCE DOCUMENTS
• Provide specific recommendations on the design of studies intended to inform decisions by patients, clinicians and payers
• Developed for specific clinical conditions and categories of technologies
• Based on a structured, transparent, multi-stakeholder process led by CMTP
• Aim to balance internal validity, relevance, timeliness and feasibility
• provide decision-makers with a reasonable level of confidence that the intervention improves net health outcomes
• Analogous and complementary to FDA guidance
• Targeted to researchers working in industry or academic settings
TECHNICAL WORKING GROUP
• Academic researchers 2• Industry 3• Payers 2• Research funders 1• Policy makers 1• Patient advocate 1
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RECOMMENDATIONS: 10 COVERING CLINICAL VALIDITY AND CLINICAL UTILITY
• MDx test development follows phases similar to the phases of drug development
• Recommendations have been organized around these phases
• Biomarker discovery (Phase 0) and the assessment of population impacts (Phase 5) go beyond the scope of this EGD
HOW EGDS MIGHT BE USED
• By test developers and researchers in designing studies• By payers in evaluating evidence submitted for
coverage and reimbursement• By guidelines developers in judging quality of evidence
and strength of recommendations• By research funding organizations in evaluating grant
proposals• By patient advocacy and other groups generating
guidance for patients
SUMMARY
• Stakeholder engagement is essential for fulfilling the objectives of CER• Research funding requests and support need to account for resources
required to meaningfully implement engagement activities
• The terminology and methods are being developed and tested worked for CER• Methods need to be tailored to the particular phase of research, but
stakeholders should be involved throughout the process• Careful attention to communication at multiple levels is critical to ensuring
true collaboration and a respectful, accountable process
• There is a growing body of examples of rigorous methods of SE being applied to CER
• Evaluation is critical for measuring impact and process improvement
Extra Slides
CMTP: PRINCIPLES FOR INVOLVING PATIENTS IN COMPARATIVE EFFECTIVENESS RESEARCH
• Each CER-related project includes patient representatives.** Examples of such projects include identifying research topics, setting priorities, developing questions to be studied, designing study protocols and establishing methodological standards.
• Project leaders recruit a diverse group of patients for whom the project topic is relevant.
• Project leaders, patients and other stakeholders make their mutual expectations for patient involvement known to each other.
• The project team, other stakeholders and patients disclose potential conflicts of interest.
• Project budget includes appropriate remuneration for patients and support for their participation, including training, stipends, travel and lodging, and other resources critical for their full involvement in the project.
**Includes patients, family caregivers and consumers representatives of all types
CMTP: PRINCIPLES FOR INVOLVING PATIENTS IN COMPARATIVE EFFECTIVENESS RESEARCH……..CONT’D
• The project team and other stakeholders recognize and respect the different skills, knowledge and experience of patients. Patients recognize and respect those of the other participants.
• The project team communicates regularly with patients throughout the life of the project to ensure the quality and sustainability of the involvement process.
• The project team obtains periodic assessments from patients and other stakeholders to evaluate the effectiveness of the engagement process and inform the design of future research involving patients.
• Project reports and publications describe in the methods sections how patients were involved in research.
• The project team and other stakeholders work with patients to present study findings in a way that can be easily understood by patients.