Patient-Centeredness

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1 Patient-Centeredness Caprice C. Greenberg, MD, MPH Associate Professor of Surgery WARF Professor of Surgical Research Director, Wisconsin Surgical Outcomes Research University of Wisconsin-Madison

2 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

3 IOM Quality of Care Safe Effective Patient Centered Timely Efficient Equitable * Institute of Medicine. Crossing the Quality Chasm

4 IOM Quality of Care Safe Effective Patient Centered Timely Efficient Equitable * Institute of Medicine. Crossing the Quality Chasm

5 Traditional Approach

6 21 st Century Healthcare System

7 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.

8 Traditional Approach

9 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

10 21 st Century Research

11 21 st Century Research

12 Stakeholders Individuals, organizations or communities that have a direct interest in the process and outcomes of a project, research or policy endeavor Patients & Caregivers Researchers Clinicians Payers & Purchasers Policymakers

13 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

14 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

15 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

16 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

17 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

18 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)

19 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.

20 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

21 PCOR Definition 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?

22 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.

23 CER PCOR

24 CER PCOR Generates evidence to inform healthcare decisions Active patient engagement Compare different prevention, treatment, diagnostic strategies Incorporates patient-reported outcome

25 CER PCOR

26 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):

27 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):

28 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):

29 Research Process 2: Topic Prioritization 4: Data Collection 6: Interpretation 1: Topic Identification 8: Implementation of Practice/ Policy Change 3: Protocol Development/ Study 5: Analysis 7: Dissemination of Results

30 Mullins CD et al. Continuous Patient Engagement in Comparative Effectiveness Research. JAMA 2012;307(15):

31 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.

32 Design Considerations Intended purpose for engagement Explicit identification of stakeholder groups Distribution of constituency Complexity of the topic Nature of the feedback

33 Logistics Sampling and Recruitment Leverage existing contacts Snowball sampling Postal or electronic mailings Media / Internet Engagement Conference calls / webinars Workshops / conferences Delphi

34 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.

35 Framework for Engaging Stakeholders

36 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

37 Topic Identification Process g p >15 topics 6-12 topics Brainstorming Generate Many Topics Provider Stakeholders Policy/Payer Stakeholders Patient Informants Discuss, Prioritize, Operationalize All Stakeholders 6 topics 1-6 topics 1 topic Develop Draft Concepts DEcIDE Investigators Discuss, Prioritize, Operationalize All Stakeholders Identify Highest Priority Topic

38 Year 1 and 2 TOPIC IDENTIFICATION AND PRIORITIZATION

39 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? 40

41 Distribution of the IOM Priorities Iglehart J. N Engl J Med 2009;361:

42 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

43 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 44

45 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 Comparator Breast MRI 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

46 Topic 2 Neoadjuvant chemotherapy or primary debulking surgery for stage IIIB, IIIC and IV ovarian cancer Population Intervention Comparator Stage IIIB, IIIC and IV ovarian cancer patients Platinum-based neoadjuvant chemotherapy followed by interval cytoreductive surgery and platinum-based post-operative chemotherapy 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

47 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

48 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 Value and utility od deliverable uncertain

49 Revised Approach 2011: three-part DEcIDE Cancer Consortium stakeholder meeting series Two separate forums: Clinicians & researchers Federal partners & payers Patient advocates included in both

50 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

51 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

52 Comparison of Approach Goals Set cancer-related CER agenda Identify topics Prioritize topics Engagement In-person meeting at AHRQ Follow up via No 3 rd party facilitator Attendees NCI CMS NYState Dept of Health DC Dept of Healthcare Finance ASCO ACS ACoS ONS ASTRO National Coalition for Cancer Survivorship Methods Small group discussions Priority ranking activity Set cancer-related CER agenda Identify topics Prioritize topics Operationalize topics to ensure feasibility Meeting 1 and 3 - Utilize existing infrastructure of cancer cooperative groups Meeting 2 - Used 3 rd party moderator at a hotel Meeting 1: Clinicians (Add pathologists, radiologists) Patient advocates Meeting 2: Payers Federal partners (Add FDA) Patient advocates Meeting 3: Clinicians Patient advocates Large group discussions Worksheets Conclusion 2 specific research protocols Post-treatment Surveillance

53 Year 3 OPERATIONALIZATION

54 Topic Identification Process g p >15 topics 6-12 topics Brainstorming Generate Many Topics Provider Stakeholders Policy/Payer Stakeholders Patient Informants Discuss, Prioritize, Operationalize All Stakeholders 6 topics 1-6 topics 1 topic Develop Draft Concepts DEcIDE Investigators Discuss, Prioritize, Operationalize All Stakeholders Identify Highest Priority Topic

55 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

56 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

57 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?

58 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

59 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

60 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

61 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:

62 Patient Centered Outcomes

63 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

64 Patient Stakeholder Quote Survival and recurrence are not just patient-centered outcomes, they are the ultimate patientcentered outcomes.

65 Examples Outcome Mortality Complication Rate Ejection Fraction Ability to walk up 1 flight of stairs Hb A1c Patient Centered Outcome Yes Yes No Yes???

66 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

67 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

68 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

69 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

70 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