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

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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