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Caprice C. Greenberg, MD, MPH Associate Professor of Surgery
WARF Professor of Surgical Research Director, Wisconsin Surgical Outcomes Research
University of Wisconsin-Madison
Patient-Centeredness
Objectives
• Introduce the concept of patient-centeredness
• Discuss how it informed development of patient-centered outcomes research
• Describe patient and stakeholder engagement
• Compare patient-centered outcomes to traditional outcome measurements
IOM Quality of Care
• Safe • Effective • Patient Centered • Timely • Efficient • Equitable
* Institute of Medicine. Crossing the Quality Chasm
IOM Quality of Care
• Safe • Effective • Patient Centered • Timely • Efficient • Equitable
* Institute of Medicine. Crossing the Quality Chasm
Traditional Approach
21st Century Healthcare System
Stage 1—The physician determines what is in the best interest of the patient and controls care. The patient’s role tends to be passive, with care being organized for the benefit of the professional and/or institution. Stage 2—Members of the professional team informally share control among themselves, but physician autonomy predominates. Care is organized for the benefit of the professional and/or institution. Patients have informal mechanisms for input on their care. Stage 3—Formal mechanisms for patient input exist. Care is organized for the benefit of the professional and/or institution, but there is some movement toward a patient-centered system. Stage 4—Care processes and transactions are based on the new rules. Care is patient-centered, with patient and family being part of the health care team. Patients have access to as much information as they wish to have and opportunities to exercise as much control over their care as they desire.
Traditional Approach
The Evidence Paradox
• 18,000+ RCTs are published each year • Tens of thousands of non-experimental studies • Many systematic reviews, health technology
assessments, clinical guidelines conclude that the “available evidence is limited or studies are poor quality”
• 17 years from publication to impact
21st Century Research
21st Century Research
Stakeholders
Individuals, organizations or communities that have a direct interest in the process and outcomes of a project, research or policy endeavor
Patients
& Caregivers
Clinicians Payers &
Purchasers Researchers Policy-makers
Federal Coordinating Council
• 2009 Comparative Effectiveness Research (CER) report to President and Congress
• Outlined stakeholder engagement as core component of CER
• Resources would be allocated to research questions with the greatest potential to impact clinical practice and health policy
The Politics of CER
• “None of us agree on what CER actually is, but we all agree it will cost about $5B to do it” Jack Rowe, former CEO, Aetna
• “Whenever you observe unanimous support for a new idea in Washington, it means that the concept has not been adequately defined”
Anonymous policy insider
Proponents say: • Provides information to help patients and
clinicians make better decisions
• Promotes evidence-based medicine
• Improves quality of care
• Decreases variation in care
• One strategy to help control cost of care
Opponents Say: • Results in “cookbook medicine”
• Leads to denied coverage for treatments – deemed less clinically effective – deemed less cost effective
• Leads to “rationed” care
• Disrupts the doctor-patient relationship
• Threatens “personalized medicine”
CER and PCOR • Provide information to inform care
– Patients that do not fit clinical trials criteria – Answer questions that cannot be addressed by
traditional efficacy clinical trials • Stakeholder driven • Increase knowledge transfer • Greater participation in research
– Contribution to registries – Practical clinical trials – Regional collaboratives
CER Definition - 1 “ Comparative Effectiveness Research (CER) is the conduct and synthesis of research comparing the benefits and harms of different interventions and strategies to prevent, diagnose, treat and monitor health conditions in ‘real world’ settings.” (Federal Coordinating Council for Comparative Effectiveness Research: Report to the President and the Congress, June 30, 2009)
CER Definition - 2
“The purpose of this research is to improve health outcomes by developing and disseminating evidence-based information to patients, clinicians, and other decision-makers, responding to their expressed needs, about which interventions are most effective for which patients under specific circumstances.”
What’s the difference? • Where is the patient? • Not in the name • Not very patient-centered approach • Doesn’t emphasize the patient point of view
• May have contributed to demonizing of the term
by certain political parties
• Patient-Centered Outcomes Research v. Comparative Effectiveness 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. This research answers patient-centered questions such as: • ‘Given my personal characteristics, conditions and
preferences, what should I expect will happen to me?’ • ‘What are my options and what are the potential benefits and
harms of those options?’ • ‘What can I do to improve the outcomes that are most
important to me?’ • ‘How can clinicians and the care delivery systems they work in
help me make the best decisions about my health and healthcare?’”
PCOR Definition
PCOR • Engage patients and other stakeholders in the
research process and in particular in determining appropriate outcomes.
• Often in research, we set intermediate outcomes that are easy to measure and compare, but do not impact patients directly.
• The goal in PCOR is to ensure that the outcomes of all studies are meaningful and important to patients.
CER PCOR
CER PCOR
Incorporates patient-reported
outcome
Active patient engagement
Compare different prevention, treatment,
diagnostic strategies
Generates evidence to inform healthcare
decisions
CER PCOR
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.
Deverka PA, Lavallee DC, Desai PJ, Esmail LC, Ramsey SD, Veenstra DL, Tunis SR. Stakeholder participation in comparative effectiveness research: defining a framework for effective engagement. Journal of Comparative Effectiveness Research, 2012; 1(2): 181-194.
Definition
• Stakeholder: individual or group who is responsible for or affected by health- and health-related decisions that can be informed by research evidence
• Engagement: bi-directional relationship between stakeholder and researcher resulting in informed decision-making about selection, conduct, and use of research
Concannon. A new taxonomy for stakeholder engagement in patient-centered outcomes research. JGIM (2012); 27(8): 895-91.
Types of Stakeholders • Patients and Public • Providers • Payers • Policymakers • Purchasers • Product Makers • Principal Investigators
Concannon. A new taxonomy for stakeholder engagement in patient-centered outcomes research. JGIM (2012); 27(8): 895-91.
Research Process
1: Topic Identification
8: Implementation of Practice/
Policy Change
2: Topic Prioritization
3: Protocol Development/ Study
4: Data Collection
5: Analysis
6: Interpretation
7: Dissemination of Results
Mullins CD et al. Continuous Patient Engagement in Comparative Effectiveness Research. JAMA 2012;307(15):1587-1588.
Literature Review
• Lack of consistent terminology, definitions, or structured processes
• Variations in methodologies used – Qualitative techniques for topic identification – Mixed methods or quantitative approach to topic
prioritization
• Design considerations and logistics O’Haire C, Methods for engaging stakeholders to identify and prioritize future research
needs. Methods Future Needs Report No. 4. AHRQ Publication 11-EHC044-EF.
Design Considerations
• Intended purpose for engagement • Explicit identification of stakeholder
groups • Distribution of constituency • Complexity of the topic • Nature of the feedback
Logistics
• Sampling and Recruitment – Leverage existing contacts – Snowball sampling – Postal or electronic mailings – Media / Internet
• Engagement – Email – Conference calls / webinars – Workshops / conferences – Delphi
Principles of Engagement
• Balanced representation among all groups
• Stakeholders’ understanding and acceptance of role
• Neutral, expert facilitations of discussions
• Connection among stakeholders
• Sustained stakeholder engagement Hoffman A. How best to engage patients, doctors, and other stakeholders in designing CER studies. Health Affairs (2010); 29(10): 1834.
Framework for Engaging Stakeholders
AHRQ DEcIDE Cancer Consortium • Conduct patient-centered outcomes research,
including comparative clinical effectiveness
• 4 major disease areas: cancer, diabetes, cardiovascular, mental health
• One of the first federally-funded initiatives to operationalize stakeholder engagement as a core tenement of the research program
• Lead stakeholder activities
Topic Identification Process Brainstorming – Generate Many Topics• Provider Stakeholders• Policy/Payer Stakeholders• Patient Informants
Discuss, Prioritize, Operationalize• All Stakeholders
Develop Draft Concepts• DEcIDE Investigators
Discuss, Prioritize,Operationalize
• All Stakeholders
Identify Highest Priority Topic
>15 topics
6-12topics
6topics
1-6topics
1topic
g p
TOPIC IDENTIFICATION AND PRIORITIZATION
Year 1 and 2
Initial Approach
• 2010: First annual DEcIDE Cancer Consortium Stakeholder Meeting, AHRQ headquarters
• Stakeholder groups represented: – Federal and state government agencies – Professional oncologic societies – Patient advocacy organizations
• Orientation and Context • Topic Generation and Prioritization • Deliverables
40
Framework for Identification
• What data is most needed to improve the everyday care of cancer patients?
• Where are the gaps in what has already been generated?
• Can the proposed IOM high-priority topics be refined and operationalized?
Iglehart J. N Engl J Med 2009;361:325-328
Distribution of the IOM Priorities
IOM Priorities and Cancer (6) • Management strategies for DCIS
• Imaging technologies in diagnosing, staging, and monitoring patients with cancer including PET, MRI, and CT
• Genetic and biomarker testing v. usual care in preventing and treating…cancer…
• Mammo +/- MRI in community practice-based screening in high-risk women of different ages, risk factors, and race
Many others include cancer….
• Minimally invasive v. open surgery
• Patient decision support tools on informing diagnostic and treatment decisions for elective surgery
• Compare approaches to reduce disparities
44
Topic Identification
• Population
• Intervention
• Comparator
• Outcome
• Study design
Topic 1 “Randomized Trial of Breast MRI for Newly Diagnosed Invasive Breast Cancer”
Population Women diagnosed with invasive breast cancer between the age of 21-75, clinical stage I, II or III
Intervention Breast MRI
Comparator No Breast MRI
Outcomes 1. Survival 2. Surgical procedure recommended and performed based on
the breast MRI findings 3. Number of surgeries required to achieve clear margins 4. Mastectomy rate 5. Number of additional biopsies recommended and
performed 6. Time from diagnosis to definitive local therapy 7. Time from diagnosis to local recurrence
Study Design Prospective randomized trial
Topic 2 “Neoadjuvant chemotherapy or primary debulking surgery for stage IIIB, IIIC and IV
ovarian cancer”
Population Stage IIIB, IIIC and IV ovarian cancer patients
Intervention Platinum-based neoadjuvant chemotherapy followed by interval cytoreductive surgery and platinum-based post-operative chemotherapy
Comparator Primary cytoreductive surgery with platinum-based post-operative chemotherapy
Outcomes 1. Overall survival 2. Surgical complications 3. Stoma rates 4. Short-term surgical mortality
Study Design Retrospective observational study using SEER-Medicare and the NCCN ovarian cancer database
Framework for Prioritization
• Potential impact on mortality, morbidity and/or suffering
• Potential impact on quality of care • Potential impact on specific stakeholders • Degree of uncertainty • Feasibility
Limitations
• Spectrum of cancer not well-represented
• Task not matched to appropriate stakeholder
• Scope of meeting too broad
• Identified topics difficult to operationalize
• Poor stakeholder engagement via email
• Value and utility od deliverable uncertain
Revised Approach
• 2011: three-part DEcIDE Cancer Consortium stakeholder meeting series
• Two separate forums: – Clinicians & researchers – Federal partners & payers
• Patient advocates included in both
Revised Approach
• Each meeting had a distinct purpose and was matched to the appropriate stakeholder group – Meeting 1: Topic Identification by clinicians,
researchers and patients – Meeting 2: Topic Prioritization by policymakers,
federal partners, payors, and patients – Meeting 3: Operationalization by clinicians,
researchers and patients
The Alliance
• Merger of CALGB, NCCTG, ASOSOG • Comprehensive clinical trials infrastructure
built for efficacy trials • Leverage to facilitate prospective and
retrospective CER and PCOR • Access to stakeholders across disciplines • 20 years of data for retrospective studies • CCOP for pragmatic clinical trials
Comparison of Approach 2010 2011 Goals • Set cancer-related CER agenda
• Identify topics • Prioritize topics
• Set cancer-related CER agenda • Identify topics • Prioritize topics • Operationalize topics to ensure feasibility
Engagement • In-person meeting at AHRQ • Follow up via email • No 3rd party facilitator
• Meeting 1 and 3 - Utilize existing infrastructure of cancer cooperative groups
• Meeting 2 - Used 3rd party moderator at a hotel
Attendees • NCI • CMS • NYState Dept of Health • DC Dept of Healthcare Finance • ASCO • ACS • ACoS • ONS • ASTRO • National Coalition for Cancer
Survivorship
Meeting 1: • Clinicians (Add pathologists, radiologists) • Patient advocates
Meeting 2: • Payers • Federal partners (Add FDA) • Patient advocates Meeting 3: • Clinicians • Patient advocates
Methods • Small group discussions • Priority ranking activity
• Large group discussions • Worksheets
Conclusion • 2 specific research protocols
• Post-treatment Surveillance
OPERATIONALIZATION Year 3
Topic Identification Process Brainstorming – Generate Many Topics• Provider Stakeholders• Policy/Payer Stakeholders• Patient Informants
Discuss, Prioritize, Operationalize• All Stakeholders
Develop Draft Concepts• DEcIDE Investigators
Discuss, Prioritize,Operationalize
• All Stakeholders
Identify Highest Priority Topic
>15 topics
6-12topics
6topics
1-6topics
1topic
g p
Operationalize
• Highest Priority Topic: “Surveillance Approaches following Active Treatment for Cancer: A Critical Target for Comparative Effectiveness Research”
• Expand into a proposal submitted to AHRQ with DEcIDE Cancer Consortium’s recommendation for future research and funding activities
Audience Response Units
• Interactive clickers to facilitate engagement by providing anonymity – Minimize social desirability bias – Admit uncertainty – Contradict thought leaders
• Definitive quantitative capture of input • Improved response rates • Results shared with the group for in-depth
discussion of salient issues
Sample Questions
• Impact – “Would routine surveillance be more effective than
our current symptom-driven approach to detecting recurrence?”
– “Should I have MRI to detect local recurrence or is annual mammogram sufficient?”
• Uncertainty and Feasibility – “Would you enroll Her2neu + patients in a clinical trial
randomizing to routine head CT v symptom-driven?”
Salient Points
• Uncertainty around optimal f/u is faced by survivors and providers daily
• Issue reaches across all cancer disease sites and disciplines = parallel studies
• Major impact given need for data to improve clinical guidelines
• Little attention in CER to date on survivorship and long-term outcomes
• Impact of unnecessary tests and false positives
Guidelines for Patient-Reported Outcomes
“Engage patient informants,
persons representative of the population of
interest, in all phases of PCOR”
Public comment draft report of the Patient-Centered Outcomes Research Institute (PCORI) Methodology Committee July 23, 2012
Outcomes Measures • In order to improve care
– We must know and agree upon what is “right” or what are the meaningful outcomes
– We must be able to measure it
• Difficult to capture data required for clinically meaningful measures
• Measures often focus on what can be measured rather than what can improve quality of care
Patient-Centered Outcomes • Quality goes beyond whether the patient
lives or dies • Patients and caregivers in general support the
current state of cancer research but feel that it is driven by the interests of clinicians and academics, not patients
• Little attention to the patient point of view Jenks S. The public applauds cancer research but not
how research priorities are set. Journal of the National Cancer Institute, 1997; 89: 350–351.
Patient Centered Outcomes
Patient Centered Outcomes
• PROs are a type of patient centered outcome but PCOs can be much broader
• PCORI definition - “were it to be the only thing that changed, patients would be willing to undergo a treatment with associated risk, cost, or inconvenience”
Patient Stakeholder Quote
“Survival and recurrence are not just patient-centered outcomes,
they are the ultimate patient-centered outcomes.”
Examples
Outcome
Mortality
Complication Rate
Ejection Fraction
Ability to walk up 1 flight of stairs
Hb A1c
Patient Centered Outcome
Yes
Yes
No
Yes
???
Patient satisfaction
• Emotional or cognitive evaluation of a health encounter by a patient as defined by his experience • technical skill • Communicaiton • Accessibility • Convenience • physical layout
Decision Regret
• The negative, cognitively based emotion that is experienced when one realizes or imagines that his present situation would have been better had he acted differently
• Patients with increasingly active role in determining treatment course
• Increased shared decision making and preference sensitive care
Health Related Quality of Life • Describes the experience of different domains of health
as modified by both disease and treatment processes • Main constructs
• Physical function • Social function • Emotional function • Cognitive function • Pain • Vitality • Overall well being
Generic Measures
• Comprehensive and assess the overall impact of treatments
• Independent of specific disease type, treatment or patient population
• Capture physical, psychological, and social aspects of health • Short Form Health Survey (SF36, SF12 ) • Sickness Impact Profile
Condition-Specific Measures
• More sensitive • Capture symptoms that are specific to a given
medical condition • Measure direct effect of a condition on QOL • e.g. FACT-C (functional assessment of cancer
therapy-colorectal) • Condition-specific supplement to generic
instrument (e.g. Quality of life questionnaire) • EORTC QLQ-C30 + QLQ-PAN26