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Penn Multi–Disciplinary, Community Engaged Research: Opportunities and Challenges J. Sanford (Sandy) Schwartz, MD Leon Hess Professor of Medicine and Health.

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Presentation on theme: "Penn Multi–Disciplinary, Community Engaged Research: Opportunities and Challenges J. Sanford (Sandy) Schwartz, MD Leon Hess Professor of Medicine and Health."— Presentation transcript:

1 Penn Multi–Disciplinary, Community Engaged Research: Opportunities and Challenges J. Sanford (Sandy) Schwartz, MD Leon Hess Professor of Medicine and Health Management and Economics Perelman School of Medicine and The Wharton School University of Pennsylvania

2 Penn “Absence of evidence of effectiveness” increasingly is being interpreted as “evidence of absence of effectiveness”

3 Penn Evaluation of Medical Care SafetySide effects acceptable? EfficacyCan it work? Efficacy: Net benefit optimal conditions EfficiencyIs there sufficient value?

4 Penn

5 Evaluation of Medical Care SafetySide effects acceptable? EfficacyCan it work? Efficacy: Net benefit optimal conditions EfficiencyIs there sufficient value? Comparative– Better than alternatives? Effectiveness– Better in specific subgroups? – Balance of harms and benefits? EffectivenessDoes it work? Effectiveness:Net benefit average conditions

6 Penn Comparative Effectiveness Research: “…comparison of effective interventions among patients in typical patient care settings, with decisions tailored to individual patient needs.” “…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…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.” Institute of Medicine. Initial National Priorities for Comparative Effectiveness Research. National Academies Press. 2009.

7 Penn Redesigning and Restructuring Health Care Patient centered (convenient, responsive, seamless, coordinated, integrated, across transitions) Learn from every patient (“learning system”) Efficient, high value care (evidence based, best practices; quality metrics, “always” events, cost– effective)

8 Penn “Everyone is entitled to his own opinion, but not his own facts.” – Senator Daniel Patrick Moynihan

9 Penn “…to assist patients, clinicians, purchasers, and policy-makers in making informed health decisions by advancing the quality and relevance of evidence concerning the manner in which diseases, disorders, and other health conditions can effectively and appropriately be prevented, diagnosed, treated, monitored, and managed through research and evidence synthesis, …. and the dissemination of research findings with respect to the relative health outcomes, clinical effectiveness, and appropriateness of the medical treatments, services…..” Patient Centered Outcomes Research Institute Mission 9 Patient Protection and Affordable Care Act 2010; Slide adapted from Joseph Selby, Executive Director Patient Centered Outcomes Research

10 Penn. “Research shall… take into account the potential for differences in the effectiveness of health care treatments, services, and items as used with various subpopulations, such as racial and ethnic minorities, women, age, and groups of individuals with different comorbidities, genetic and molecular sub-types, or quality of life preferences and include members of such subpopulations as subjects in the research as feasible and appropriate” Patient Centered Outcomes Research Institute Patient Protection and Affordable Care Act Source: Joseph Selby, Executive Director Patient Centered Outcomes Research

11 Penn “The Patient-Centered Outcomes Research Institute (PCORI) helps people make informed health care decisions – and improves health care delivery and outcomes – by producing and promoting high integrity, evidence-based information – that comes from research guided by patients, caregivers and the broader health care community.” Patient–Centered Outcomes Research Institute: Mission Statement – Patient Protection and Affordable Care Act 2010

12 Penn Comparative Clinical Effectiveness Research Asks questions that matter to patients, caregivers, clinicians, policymakers Compares two or more options for screening, diagnosis, treatment, delivery and management that matter to patients, their families and communities Considers the full range of relevant outcomes Conducted in ‘real world’ populations and ‘real world’ community settings Examines to differences in effectiveness and preferences across patient subgroups

13 Penn Patient-Centered Outcomes Research: Patient Centered Questions 1. “Given my personal characteristics, conditions and preferences, what should I expect will happen to me?” 2. “What are my options and what are the potential benefits and harms of those options?” 3. “What can I do to improve the outcomes that are most important to me?” 4.“How can clinicians and the care delivery systems they work in help me make the best decisions about my health and healthcare? ”

14 Penn Patient-Centered Outcomes Research Assesses benefits and harms of preventive, diagnostic, therapeutic, palliative, or health delivery system interventions to inform decision making, highlighting comparisons and outcomes that matter to people; Is inclusive of an individual’s preferences, autonomy and needs, focusing on outcomes that people notice and care about such as survival, function, symptoms, and health-related quality of life; Incorporates a wide variety of settings and diversity of participants to address individual differences and barriers to implementation and dissemination; and Investigates (or may investigate) optimizing outcomes while addressing burden to individuals, resource availability, and other stakeholder perspectives.

15 Penn Transparent Patient focused Evidence–based Rigorous methodology Stakeholder-driven; Patient engagement Public input/feedback prior to adoption of priorities, agendas, methodological standards, peer review processes, dissemination strategies Patient–Centered Outcomes Research Institute

16 Penn Patient Centered Outcomes Research Priorities Source: Joseph Selby, Executive Director Patient Centered Outcomes Research

17 Penn PCORI Merit Review Committee Composition Reviewer committee comprised of scientists, patients and stakeholders who together bring a broad range of perspectives to the review process Patients Stakeholders Scientists A patient with a rare disease may better understand whether pain, fatigue, or confusion is the most burdensome side effect of a particular therapy. A stakeholder may be in a position to say if a particular intervention is timed appropriately. A scientist may be in a position to evaluate if the study will produce meaningful data that will allow it to advance patient care. Source: Joseph Selby, Executive Director Patient Centered Outcomes Research

18 Penn Patient and Community Engagement: Making Research More Useful Topic/question generation and specification (inform clinical decisions) Study design –Focus on effectiveness; not efficacy –Compare at least two alternative patient management strategies –Measure outcomes that matter to patients, including harms and benefits, at both population and subgroup levels Study conduct Data analysis Dissemination, communication, implementation

19 Penn Patient Centered Outcomes Research Institute Priorities

20 Penn Disease incidence, prevalence and burden (with emphasis on chronic conditions) Gaps in evidence in terms of clinical outcomes, practice variations and health disparities in terms of delivery and outcomes of care Potential for new evidence to improve patient health, well- being and the quality of care Effect on national expenditures associated with a health care treatment, strategy or health conditions Patient needs, outcomes, and preferences Relevance to patients and clinicians in making informed health decisions Priorities in National Strategy for quality care established under section 399H of Public Health Service Act Patient–Centered Outcomes Research Institute: Criteria in Formulating Priorities and Agenda

21 Penn PCORI Initial Priorities PCORI National Priorities for Research and Research Agenda, 2012

22 Penn PCORI National Priorities for Research and Research Agenda, 2012

23 Penn (continued) PCORI Criteria (continued): Criteria, Statutory Language, Questions PCORI National Priorities for Research and Research Agenda, 2012

24 Penn Comparative Effectiveness Research: Methodological Challenges and Development Design All relevant alternatives Relevant patients (targets of intervention) Clearly defined, rigorous, best available experimental, quasi–experimental, observational, qualitative data methods Clinically relevant outcomes and validated surrogate data evidence and outcomes Incremental impact/trade-offs Explicitly characterize uncertainty Address issues of generalizability / transferability Adapted from Drummond MD, Schwartz JS, Jonsson B, Luce B, Neumann PN, Siebert U, Sullivan SD. Int J Tech Assess Health Care. 2008;24:244-58; discussion 362–368.

25 Penn Analysis of Medical Interventions: Required Considerations Clinically relevant patients and subpopulations Clinically relevant, validated outcomes Broad range of empiric data –Experimental (RCT; pragmatic/practical) –Observational (case–control, cohort, registry, administrative claims, EMR) –Synthesis (meta–analysis; systematic review) –Integration (modeling) formal, structured, expanded, multi–factorial outcomes Consistency across methods confirmatory; disagreement requires understanding / explication

26 Penn Novel Aspects of PCORI Substantial financial commitment, independent of annual appropriations process Non-profit, private/public organization Centralization of U.S. clinical research agenda Focus on –Patient outcomes –Patient priorities, preferences and values –Quality of care –Effective communication National effort to build infrastructure

27 Penn Life is full of calculated risks…

28 Penn Occupational Risks

29 Penn Avoidable Risks

30 Penn Unexpected Risks

31 Penn “All policy decisions should be based on absolute measures of risk; relative risk is strictly for researchers only.” – Geoffrey Rose Professor Epidemiology London School of Hygiene and Tropical Medicine Absolute vs. Relative Risk

32 Penn 10–Year Risk of Death from Breast Cancer: Beginning Routine Screening Age 40 vs. Age 50 Ages 40-49 Ages 50-59 Without screening0.33% 0.89% With screening 0.28% 0.69% Absolute RR0.05% 0.20% Source: Steve Woloshin, Veterans Affairs Outcomes Group

33 Penn “The paradox of the clinical trial is that it is the best way to assess whether an intervention works, but arguably the worst way to assess who will benefit from it.” Mant. Lancet. 1999;353:743–746

34 Penn “The benefit or harm of most treatments in clinical trials can be misleading and fail to reveal the potentially complex mixture of substantial benefits for some, little benefit for many, and harm for few.” – R Kravitz, Milbank Quarterly, 2004

35 Penn Shifting the population distribution reduces disease burden more than targeting people at high risk Large benefit to population may provide limited benefit to individuals Large benefit to an individual may have a small population impact in the population Modest benefit to a participating individual may be wiped out by a small harm to many Discounting is counter to behavioral preferences Benefits may not be attractive to the individual, even if beneficial on a social/community basis Population Paradoxes

36 Penn Adapted from Rodger, E. Organization Characteristics Market Forces

37 Penn Diffusion & Adoption of Medical Innovation Adopter characteristics Professional values, standards and culture Organizational characteristics, values, culture Market structure and competition Financial incentives Individual/community characteristics and values Channels and sources of communication Manufacturer strategies Social and environmental factors

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39 “Culture trumps data every time.” “Culture eats strategy for breakfast.”

40 Penn 40 Trans–Theoretical Model Past Behavior Demographics and Culture Attitudes Toward Targets (stereotypes and stigma) Personality, Moods, and Emotions Other Individual Difference Variables (e.g., perceived risk) Intervention or Media Exposure Behavioral Beliefs and Outcome Evaluations Efficacy Beliefs Injunctive and Descriptive Normative Beliefs Attitudes Normative Pressure Self Efficacy IntentionsBehavior External Variables Belief Measures Direct Measures IntentionsBehavior

41 Penn Translating Knowledge Into Decisions and Action Well–accepted, rigorous, transparent methods Understand how new information fits within the context of alternative management options Evidence–based, actionable messages Target decision makers –Understand organization, processes & needs –Key opinion leaders –Interact early & often –Specify expected organizational impact (outcomes, how results will be achieved) Respected, credible messengers Clear communication – “less is more”

42 Penn

43 "What we've got here is a failure to communicate” Paul Newman Cool Hand Luke, 1967

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45 Perceived importance Perceived benefit Perceived barriers Self–efficacy PrecontemplationInformation ContemplationFacilitators ActionCues MaintenanceFeedback/Support Becker M. Health Belief Model Fishbein M. Theory of Reasoned Action Prochaska. Stages of Readiness Changing Behavior: Stages of Readiness

46 Penn Shared objectives and goals Mutual respect/trust Transparent (organization, operations, resources, benefits) Integrated, cooperative, collaborative organization, leadership, responsibilities Communication Aligned incentives (shared benefits) Team Science

47 Penn Quality Assurance: “Bad Apple Approach” Attempt to eliminate the tail, not fix the situation Propagates “Cycle of fear” If tail is quite small, can be productive

48 Penn Quality Improvement Approach Attempts to Reduce variation Improve process Create culture of learning / safety Improve system Measures and seeks to enact change

49 Penn “In the midst of every challenge lies opportunity.” –Albert Einstein

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