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Title: New Era Of Comparative Effectiveness Research


1
New Era Of Comparative Effectiveness Research
Hyatt Regency Capitol Hill Washington,
D.C. October 5, 2010
2
  • With thanks to

3
An Evaluation Of Recent Federal Spending On
Comparative Effectiveness Research
  • Priorities, Gaps, Next Steps

Joshua S. Benner Marisa R. Morrison Erin K.
Karnes S. Lawrence Kocot Mark McClellan
4
The Recovery Act provided unprecedented federal
spending on CERin a 2 year period
FY09/10 Based upon ARRA actual/projected
obligation amounts FY09/10 Based upon actual
and projected spending amounts
Sources AHRQ http//www.hhs.gov/recovery/reports
/plans/pdf20100610/AHRQ20CER20June202010.pdf
http//www.ahrq.gov/about/cj2011/cj2011.pdf NIH
http//report.nih.gov/rcdc/categories/ Office of
Secretary http//www.hhs.gov/recovery/reports/pla
ns/pdf20100610/OS20CER20June202010.pdf
5
As of Aug 4, nearly all of the 1.1B was spent,
but tracking it is challenging
  • Sources
  • Recovery.gov
  • Grants.gov
  • FBO.gov
  • NIH Research Portfolio Online Reporting Tools
    Expenditures and Results (RePORTER)
  • NIH Research, Condition, and Disease
    Categorization (RCDC) system
  • AHRQ Grants On-Line Database (GOLD)
  • AHRQ website

6
More than half of funds were spent on improving
capacity to do CER
Recovery Act CER spending, by project type(As of
Aug 4, 2010)
7
Recovery Act CER studies funded through NIH span
the disease areas prioritized by IOM
8
Implications for future CER spending
  • Areas where more funding is needed
  • Experimental research
  • Evaluation of system-level reforms
  • Identification of effects in subgroups
  • Inclusion of understudied populations
  • Dissemination of results
  • Need timely, comprehensive, dashboard
  • Opportunities and ongoing projects
  • Specific aims, including priorities areas
    addressed
  • Methods and data sources
  • Budget amount, duration, and granting agency
  • Linked to CER Inventory

9
Creating A High Performance System For
Comparative Effectiveness Research
  • Lynn Etheredge
  • Rapid Learning Project
  • George Washington University

10
A Rapid Learning Health System
  • Designed to learn as rapidly as possible about
    the best treatment for each patient
  • Key concept in silico research (on large
    computerized databases)
  • Complements in vitro and in vivo methods
  • Studies many more patients, richer (EHR) data,
    more researchers more, different, faster
    studies
  • Researchers multi-year data collection ? log-on
    to worlds evidence base. Databases designed for
    CER

11
A High-Performance CER System
  • Learning about new technologies
  • Registries, research plans, reporting
  • E.g. new cancer therapies, off-label Rx use,
    surgery
  • Comparability standards for studies
  • To accelerate comparative research, data
    networking
  • A National Database For Effectiveness Research
    Studies
  • Clinical studies submit standardized data-sets, a
    growing world evidence base for CER
  • Established by Presidential order

12
A High-Performance CER System
  • A national network of clinical research
    registries and databases
  • Fill gaps in the evidence base for elderly,
    children, pregnant women, persons with multiple
    chronic conditions, minorities, rare diseases,
    surgery
  • Getting results
  • HHS public work plans, w/ due dates,
    accountability
  • Create a national rapid-learning culture for CER,
    w/ websites, networks, rapid-learning communities

13
Comparative Effectiveness And Personalized
Medicine Evolving Together or Apart?
  • Robert S. Epstein
  • J Russell Teagarden

14
The Worry
  • Comparative Effectiveness can help determine the
    average effects between alternative treatments
  • But this may mask results from important
    sub-populations (e.g. Personalized Medicine)

15
There is some evidence to support this concern
  • A recent random sample of 5 major journals and
    319 trials revealed
  • Only 29 of the studies tested comparative
    effects within sub-populations

Source Gabler NB et al Dealing with
heterogeneity of treatment effects is the
literature up to the challenge? Trials
20091043.
16
Comparative trial showed 16 reduction in CV
events if on high dose statin vs. std. dose at 2
years
Source Cannon CP et al Intensive versus
moderate lipid lowering with statins after acute
coronary syndromes. NEJM 2004350(15)1495-1504.
17
Personalized medicine finding40 CV drop in
genetic sub-population!
Source Iakoubova OA et al Polymorphism in
KIF6 gene and benefit from statins after acute
coronary syndromes results from the PROVE-IT
TIMI 22 study. JACC 200829456-8.
18
Comparative effectiveness can also provide
evidence of clinical utility
Source Mallal S et al HLA-B5701 screening
for hypersensitivity to abacavir. NEJM
2008358568-79.
19
Conclusions
  • Comparative effectiveness can
  • Uncover findings not available elsewhere due to
    studying diverse patient populations
  • Provide evidence of utility in the real world
  • Must remember to structure CE studies with broad
    entry criteria and include genetics to help
    each discipline grow together

20
A Patient Advocates Perspective On
Patient-Centered Comparative Effectiveness
Research
  • Tony Coelho
  • Former Chair, Board of Directors, Epilepsy
    Foundation of America

21
A Flexible Approach To Evidentiary Standards For
Comparative Effectiveness Research
  • Louis P. Garrison, Jr.
  • Department of Pharmacy, University of Washington,
    Seattle WA
  • Peter J. Neumann
  • Center for the Evaluation of Value and Risk in
    Health, Tufts Medical Center, Boston, MA
  • Paul Radensky
  • McDermott Will Emery LLP, Washington, D.C.
  • Sheila D. Walcoff
  • McDermott Will Emery LLP, Washington, D.C.
  • Research support National Pharmaceutical
    Council
  •  

22
Patient-Centered Outcomes Research Institute
(PCORI)--Responsibilities
  • Setting priorities
  • Developing methodological standards
  • Communicating research results to decision makers

Question How can the institute support the
different standards for acceptable evidence used
by various government agencies, providers,
patients, and other decision makers?
23
Evidentiary Standards
  • Rules that decision makers apply in using
    different types of evidence to carry out their
    mission
  • Evidentiary standards vary among decision makers
  • FDA for new drugs substantial evidence
  • FDA for devices reasonable assurance
  • FDA for promotion substantial clinical
    experience
  • CMS reasonable and necessary
  • FTC Competent and reliable scientific evidence

24
Comparative Effectiveness Research in the
Decision Maker-Evidence-Policy Loop
25
Key Issues And Implications
  • The information produced through CER will be a
    public good, benefiting all decision makers.
  • Models are a valuable tool for synthesizing data.
  • Value-of-information analysis can help PCORI with
    research prioritization.

Experiment, observation, and mathematics,
individually and collectively, have a crucial
role in providing the evidential basis for modern
therapeutics. Arguments about the relative
importance of each are an unnecessary
distraction. Hierarchies of evidence should be
replaced by acceptingindeed embracinga
diversity of approaches. Sir Michael
Rawlins, NICE
26
Methodological Standards
  • Best practice for a given type of methodology
  • Calls for a translation table to link methods
    to research questions
  • ACA provide specific criteria for internal
    validity, generalizability, feasibility, and
    timeliness of research

There is a need for balanceand tradeoffsamong
these objectives.
27
Conclusion
  • PCORI should take a balanced and flexible
    approach to the types of research it sponsors,
    being careful not to let rigorous scientific
    methods become a rigid evidentiary standard.

28
Designing Comparative Effectiveness Research On
Prescription DrugsLessons From The Clinical
Trial Literature
  • Aaron S. Kesselheim, M.D., J.D., M.P.H.
  • Co-authors Dave A. Chokshi, M.D., M.Sc., Jerry
    Avorn, M.D.
  • Division of Pharmacoepidemiology and
    Pharmacoeconomics
  • Brigham and Womens Hospital, Harvard Medical
    School, Boston MA

29
CER Not A New Phenomenon
  • CER existed before recent developments
  • Government and commercial funding
  • Review of some examples of drug CER
  • Goal Identify methodological issues
  • 3 areas of special concern
  • Choice of comparison treatments
  • Time frame
  • External validity

Sources Hochman McCormick, JAMA, 2010 Clement
et al., JAMA, 2009
30
Designing Better CER Trials
  • Choice of comparator
  • The straw man problem
  • Ex. 1 Comparator doesnt represent standard of
    care in efficacy trial (ASCOT)
  • Ex. 2 Comparator with high side effect risk in
    safety trial (MEDAL)
  • Study time frame
  • The surrogate endpoint problem
  • Ex. 1 Surrogate endpoint not valid (DCCT)
  • Ex. 2 Surrogate endpoints may mask safety
    problems (RECORD, torcetrapib)

Sources Dahlof et al., Lancet, 2005 Cannon et
al., JAMA, 2006 DCCT Research Group, NEJM, 1993
Home et al., Lancet, 2009 Psaty Lumley, JAMA,
2008
31
Designing Better CER Trials (contd)
  • External validity
  • How are CER findings applied? (If at all!)
  • Ex. 1 Trial design doesnt anticipate
    real-world use (RALES)
  • Ex. 2 Non-representative enrollment
  • Ex. 3 Inadequate effort to change prescribing
    practices (ALLHAT)
  • Ex. 4 Failure to account for critical pushback
    (CATIE)

Sources Juurlink et al., NEJM, 2004 Jagsi et
al., Cancer, 2009 ALLHAT Research Group, JAMA,
2002 Lieberman et al., NEJM, 2005
32
Moving Forward
  • Options for the new era of CER in US
  • Innovative approaches to study methods
  • Ex. 1 Adaptive trial design (STAR-D)
  • Alternatives to randomized controlled trials
  • Observational studies
  • Meta-analyses and systematic reviews

Sources Rush et al., NEJM, 2006 Nelson, JNCI,
2010 Lumley, Stat Med, 2002 Hlatky, Lancet, 2009
33
Policy Recommendations
  • CER must compare the best available alternatives
  • Use validated surrogate endpoints, but follow
    with hard endpoints and safety studies
  • Emphasize pragmatic clinical trials with
    ongoing reassessment
  • Implement observational studies with
    methodological rigor
  • Link CER to effective strategies for
    dissemination (academic detailing)
  • Market findings to health care institutions

34
Why Observational Studies Should Be Among The
Tools Used In Comparative Effectiveness Research
  • Nancy Dreyer
  • Chief of Scientific Affairs, Outcome

35
Evidence Is Needed
19
  • Goal Informed decision making about real-world
    practices and outcomes that result from that
    behavior or treatment
  • Seeking Evidence
  • Relevant to Patients and Providers
  • Valid
  • Feasible

19
36
The Framework
  • Decision-makers need to rely on a full range of
    high-quality comparative effectiveness research
  • Methods and challenges
  • Guidance for determining which type of study to
    employ
  • Whatever methods are used, research should be
    designed and implemented using best practices
  • Bodies of evidence
  • Systematic reviews, meta-analysis, grading
    systems
  • Individual study types
  • RCT, observational studies, other applications

37
Choosing The Right Fit
  • How much certainty is required?
  • Time and budget?
  • What are the comparators of interest?
  • Is there enough variability in treatment without
    randomization?
  • Is there a reasonable overlap of patient
    characteristics between treatments?
  • Treatment complexity? Single, multiple, etc.?
  • Would it be ethical and/or feasible to randomize?

38
Use Observational CE Studies When RCTs Are
Impractical Or Infeasible (Examples)
  • Patients and conditions not typically included in
    RCT (frail elderly, children, pregnancies)
  • Multiple treatment paradigms (hearing loss)
  • Treatment adherence differs (asthma)
  • Off-label uses result in different popns being
    treated (stents)
  • Operator training (ICDs)
  • Operator or institutional experience (surgical
    procedures)
  • Large study sizes needed (small differences)

39
The Most Informed Decisions Will Benefit From
Considering The Full Range Of High Quality
Research

Optimal study design depends on purpose, validity
and feasibility within constraints of time and
budget
40
Adding The Patient Perspective To Comparative
Effectiveness Research
  • Albert Wu
  • Professor, Health Policy and Management,
    Bloomberg School of Public Research, Johns
    Hopkins University

41
Some Questions Cannot Be Answered Without Asking
The Patient
  • The main objective of much of health care is
    improving how patient feels and functions
  • Reduction in pain (hip replacement)
  • Improved functioning (cataract extraction)
  • Patient is best judge
  • Patient best observer of some events and health
    outcomes (complications)

42
Types of Information Captured In Data Used For CER
Research Data Clinical Care Data Administrative Data
Utilization -
Clinical detail
Patient perspective - - -
43
Linking Data Sources for CER
Administrative Data
Patient Reported Outcome Data
Clinical Research and EHR Data
44
Electronic Health Records (EHR)
  • How can EHR be structured to include PRO
  • As clinical indicator that can be tracked,
    profiled
  • Useable for CER?

45
Johns Hopkins Medical Institutions EPR
46
Clinician Website
  • Clinician can schedule a patient a survey

47
Patient Website
  • Patient is presented with survey(s) to complete

48
Patient Website
  • Patient survey sample question

49
Clinician Website
  • Clinician can view patients results

50
  • Listen to the patient He is telling you the
    diagnosis
  • - William Osler

51
Patient-Centered Outcomes Research Institute
(PCORI)
  • Capturing patient perspective vital to complete
    picture of treatment impact
  • Strategies to accelerate development of useful
    evidence
  • Apply research-grade standardized questionnaires
  • Include more uniformly in clinical trials,
    registries
  • Inject into EHRs
  • Incentivize addition to administrative data,
    e.g., pay for collection require for
    reimbursement

52
Thanks To Co-Authors
Claire Snyder Carolyn Clancy
Don Steinwachs
53
Contact
  • Albert W. Wu, MD, MPH
  • Health Services Research Center
  • Johns Hopkins Bloomberg School of Public Health
  • 624 N. Broadway, Room 653
  • Baltimore, MD, 21230
  • awu_at_jhsph.edu

54
The Role Of Costs In CER
  • Alan M. Garber, M.D., Ph.D
  • Department of Veterans Affairs and Stanford
    University
  • Harold C. Sox, M.D.
  • Dartmouth Medical School

55
Four key elements of CER
  • Compares all relevant alternatives for real-world
    decision making.
  • Patients are representative of typical clinical
    settings.
  • Accounts for differences among individual
    patients in responding to interventions.
  • Measures all outcomes that are important to
    patients.

56
Cost-effectiveness analysis
  • Inherently comparative the incremental C-E ratio
    (?C/?E).
  • ?E measured in QALYs (quality-adjusted life
    years)
  • Inputs to CEA
  • Costs
  • Outcome measure typically life expectancy (LE)
  • Preference measure for each outcome

57
ACA limitations on CEA
  • The PCORI shall not develop or employ a
    dollars-per-quality adjusted life year . as a
    threshold to establish what type of health care
    is cost effective or recommended.

58
Should costs be a part of CER?
  • People will need data on utilization and costs
  • It will be important to many patients
  • Future changes in organization and financing of
    care may make cost information more relevant
  • Private insurers will pay a tax to support PCORI
    CER should benefit them.

59
Should cost-effectiveness analysis be a part of
CER?
  • No and Yes
  • No
  • PCORI should not routinely provide CE analyses or
    rankings based on them.
  • Rationale
  • ACA says PCORI should not draw a bright line
    defining good value.
  • The public is concerned about government
    standardization of care

60
Should CEA be a part of CER?
  • Yes
  • CER should measure comprehensive health effects,
    including utilization.
  • Others can do the C-E analyses.
  • Rationale
  • The law doesnt proscribe CEA by private parties
    (insurers, health plans)
  • The public is paying for CER, and these
    measurements are a public good.
  • We cannot afford to ration information.

61
How Medicare Could Use Comparative Effectiveness
Research To Set Payment Levels At The Time of
Coverage
  • Steven D. Pearson, M.D., M.Sc.
  • Peter B. Bach, M.D.

62
Current Coverage And Payment
  • Historically separate silos
  • Payment is generally set to reimburse providers
    or manufacturers for costs of care plus some
    profit
  • Higher pricing w/o comparative evidence
  • Proton beam therapy vs. IMRT for prostate cancer
  • Drug-eluting stents vs. bare metal stents
  • ESAs in cancer
  • Lucentis vs. Avastin

63
Paying Well For Meaningful Innovation
64
Challenges
  • Methods
  • A single time limit for all interventions?
  • Reference pricing drugs vs. surgery?
  • Grandfather in older interventions?
  • Politics
  • Implementation of new pricing policies at
    Medicare in the setting of PPACA
  • Medicare payment changes require new legislation

65
Conclusion
  • The current Medicare cost-plus pricing system is
    obsolete, even harmful.
  • Managing the disconnect between price and results
    downstream is messy at best.
  • Using evidence to avoid perverse incentives
  • Not no but yes, and.
  • Use market forces to incentivize better evidence
    development
  • Reward innovators who demonstrate that their
    interventions improve patient outcomes compared
    to other alternatives
  • Help Medicare shift to a more sustainable path

66
  • Applying Results Of Comparative Effectiveness To
    Patients
  • Obstacles and Solutions to Implementing CER in a
    Large Health System the VA Experience

Joel Kupersmith Chief Research and Development
Officer Director, Quality Enhancement Research
Initiative U.S. Department of Veterans Affairs
67
VA Healthcare System
  • 8.1 M enrollees, 5.7 Million patients/yr
  • gt1200 Sites of Care
  • 153 Medical Centers
  • 773 Community-based Outpatient Clinics
  • 260 Readjustment Counseling Centers
  • gt90,000 Providers
  • Electronic Health Record
  • Data driven practice network
  • Complex environment
  • Older, sicker Pts with substantial co-morbidity
  • Dual use of VA and other healthcare
  • Intramural research program embedded in health
    care system with 35 years of CER accomplishment

68
VA Approaches to Implementation
  • New Medical Media
  • EHR tools
  • Reminders
  • Computerized decision support
  • Tools for shared decision-making with patients
  • Old Medical Media
  • Guidelines
  • Used by individual providers, also create
    consensus
  • CME
  • Systems
  • Pharmacy Benefits Management
  • Performance Measures
  • Clinical leadership and Program Office policies
  • Evidence Synthesis Program
  • Quality Enhancement Research Initiative
  • Brings approaches together

69
Example - VA Diabetes Care
  • Translation of individualized diabetes care
  • E.g. Tighter control for younger patients,
    influence of co-morbidity
  • Formulary policy emphasizes those drugs shown to
    improve long-term clinical outcomes
  • Others available under specific conditions
  • Guidelines emphasize risk-based strategies that
    vary and individualize treatment based on age,
    co-morbidity, underlying disease
  • Performance measures focus on those where
    benefits of tighter control greatest (HbA1c lt7-9)
  • Research testing performance measures linked to
    clinical actions and patient conditions
  • Diabetes database allows facility to look at care
    for different strata of patients
  • E.g. Elderly vs. younger, newly diagnosed

70
Challenges/Lessons Learned
  • Evidence base is modest overall
  • Must keep up with fast-changing evidence
  • Individualization
  • How do we implement results in different
    settings, different patients etc.?
  • How much variation is allowable while improving
    care for populations?
  • Patient centered approaches
  • Need to align approaches to implementation and
    diminish complexity
  • Make the right thing the easy thing for busy
    practices
  • Need to reward performance that reflects CER data
    and patient circumstances
  • Performance measures are constantly evolving
  • Organizational and local characteristics are
    important
  • As is leadership involvement
  • Implementation should be an element of early
    research and not an afterthought

71
Bench To Behavior Translating Comparative
Effectiveness Research Into Improved Clinical
Practice
  • Jerry Avorn, M.D.
  • Professor of Medicine, Harvard Medical School
    Chief, Division of Pharmacoepidemiology and
    Pharmacoeconomics, Brigham and Women's Hospital

72
The final translational hurdle
  • We are not effectively using most of the
    treatments that already exist.
  • We already have some excellent drugs to manage
    many chronic conditions, but often use them
    poorly or not at all.
  • Inadequate control of these conditions is
    widespread
  • high blood pressure
  • cholesterol
  • diabetes
  • osteoporosis
  • etc.
  • The result massive amounts of preventable
    illness, cost, and death
  • Great CER findings are likely to suffer the same
    fate.

73
Why is this so?
  • Its nobodys job to disseminate purely
    evidence-based CE information in an effective way
    to practitioners.
  • The delivery system deals poorly (if at all) with
    QA for clinical decisionmaking.
  • The drug industry (and other vendors) are very
    effective in achieving their goals.
  • Marketing, continuing medical education are
    sometimes counter-productive, CE-wise.

74
But much can be done
  • Social marketing of evidence-based CE
    recommendations
  • e.g., academic detailing
  • Better separation of commercial agendas and
    content from CME courses
  • Use of prompts in electronic health records
  • Who will control this real estate?
  • Incentivize evidence-based practice

75
For more information
  • The BWH Division of Pharmaco-epidemiology and
    Pharmaco-economics (DoPE)
  • www. DrugEpi.org
  • Academic detailing programs
  • www. RxFacts.org
  • Powerful Medicines the Benefits, Risks, and
    Costs of Prescription Drugs
  • (Knopf, 2005)
  • www.PowerfulMedicines.org

76
Moving Comparative Effectiveness Research Into
Practice
  • Implementation Science And The Role Of Academic
    Medicine

Ann Bonham Chief Scientific Officer, Association
of American Medical Colleges
77
The Measures Of Success
  • Real gains in patient and population health
    outcomes through adoption of timely and relevant
    evidence.
  • Gains that reach diverse groups.
  • Metrics that matter to patients, providers, and
    policymakers in 2020, as well as 2012.

78
Implementation Science Helping Insure Success
  • Using the power of science to promote the uptake,
    dissemination and endurance of discoveries from
    comparative effectiveness research.
  • Real changes in routine practice and behaviors of
    health care systems, care providers, and
    patients.

79
Role Of Academic Medicine
  • Tripartite mission.
  • Learning laboratory for testing and implementing
    change.
  • Access to diverse and underserved populations.
  • Collaborative models across broad disciplines.

80
The Way Forward
  • From silos to solutions integrating missions
    around patient outcomes.
  • Integrated leadership.
  • Strategic allocation of resources and human
    potential.
  • New transdisciplinary partnerships.

81
The Case For A National Patient LibraryAuthors
Jeffrey C. Lerner, Daniel M. Fox, Sheryl B.
Ruzek, Gail E. Shearer
  • Jeffrey C. Lerner, Ph.D.
  • President and Chief Executive Officer
  • ECRI Institute

82
Perspective
  • Health services researchers put relatively little
    time, intellectual effort, or money into
    sophisticated communication of evidence-based
    information to patients and their families, and
    towards influencing the clinician/patient
    encounter.
  • Manufacturers of medical products spend almost as
    much on advertising as on R D, including
    billions on direct-to-consumer promotions.
  • Hospitals and clinicians advertise to patients.
  • Advocacy groups focus on communicating with
    patients.
  • A National Patient Library of evidence-based
    information can help create a better balance in
    the information available to the public.

83
Background
  • The Patient Protection and Affordable Care Act
    calls for shared decision making by patients
    and clinicians. See sections 931 and 936.
  • The Act establishes the Patient Centered Outcomes
    Research Institute (PCORI). The National Patient
    Library could promote public awareness and the
    use of findings of PCORIs independent research,
    and underscore the central role of public finance
    in conducting that research.

84
What A Library Would Deliver And To Whom
  • Primary purposea repository and clearinghouse of
    evidence-based information designed for patients
    and their lay and professional caregivers.
  • Other central purposes
  • assess patients information needs and the degree
    to which they are met effectively.
  • serve as a center of innovation for health
    communication across the spectrum of media.
  • assess and disseminate evidence about the effects
    of patients use of information on the use and
    cost of healthcare.

85
Challenges And Opportunities
  • Meet patients needs for trustworthy, accessible,
    and timely information.
  • Achieving sufficient initial and sustainable
    funding.
  • Ensuring that the public believes that the
    purpose of the National Patient Library is to
    serve them.

86
Politics of Comparative Effectiveness Research
  • Gail Wilensky
  • Senior Fellow, Project HOPE

87
Politics of Comparative Effectiveness Research
Gail R. Wilensky Project HOPE October 5, 2010
88
Comparative Effectiveness Research
  • Convergence of Interests -- Major focus on
    ing uninsured Need to value, slow
    spending
  • To change from where we are, need
  • Better Information Better Incentives
  • (These used to be good Republican concepts)

89
Explosive Politics Around CER
CER has become a lightening rod for criticism
by some conservatives some in industry
govt run health care socialized
medicine govt monitoring your
doctor code words for denying care by birth
date
More to come? Pre-election? Post-election?
90
CER Now Part of Reform
But --
? Concept remains controversial ? And with a
variety of limitations ? And an uncertain role
in decision-making
CER Still Very Fragile
91
Early ARRA Results Will Be Important
? Interesting mix of studies
? Important mix of methodologies -- IOM
top 100 ½ RCT ½ not
? Some results could be controversial --
Need input from affected parties
? Dissemination/explanations will be important
92
Ultimate Challenge Making Use of CER
? Primary stated purpose improved health
outcomes
? Secondary purpose building block to
spending smarter
-- CER more suited to reimbursement decisions
-- Value-based reimbursement/value-based insuranc
e principles
93
Using CER to Moderate Spending
A major challenge!
? PCORI cant mandate coverage or
reimbursement
? Other limitations on PCORI
? CMS cant use cost in coverage decision
no authority to use CER in reimbursement
94
First Steps to Moderating Spending
? Encourage private payers to use CER in
making reimbursement decisions
? Encourage use of value-based insurance
by private payers
? Promote pilots/demos of VBI for
Medicare reimbursement
Need to focus on the long run
95
Will CER Be Politically Sustainable?
  • Insights From National Public Opinion Surveys
  • Alan S. Gerber, Eric M. Patashnik, David Doherty
    and Conor Dowling

96
Overall, the Public Supports Government Funding
of CER
Survey conducted 5/21-24/2010 (N2017)
97
A Sharp Partisan Divide Over CER Among Voters
Survey conducted 5/21-24/2010 (N2017)
98
The Public is Enthusiastic About Some Uses of
CER, Opposes Others
Survey conducted 5/21-24/2010 (N2017)
99
Americans Fear Guidelines will Lead to Rationing
and "One-Size-Fits-All" Medicine
Mean support for Board issuing mandatory
guidelines 34 on 0-100 scale (May 21-24, 2010
survey)
Survey conducted 11/5-12/31/2009 (N1026)
100
Experiment 1 Effects of Exposure to Arguments
For/Against CER
  • Baseline support of CER assessed (100-point
    scale)
  • Respondents randomly assigned to receive one of
    two pro arguments
  • The information will improve health care outcomes
  • Will help reduce the budget deficit by cutting
    wasteful spending on ineffective treatments
    without lowering quality of care
  • Respondents randomly assigned to receive one of
    several con arguments
  • CER will lead to one-size-fits-all medicine
  • CER will lead to rationing

Survey conducted 7/30-31/2010 (N2010)
101
Experiment 1 Results
Pro-CER Argument Anti-CER Argument Anti-CER Argument
One Size Fits All Ration Care
The information from CER will improve health care outcomes -10 -6.9
CER can help reduce the budget deficit by cutting wasteful medical spending without lowering the quality of care -9.9 -8.0
102
Experiment 2 Best Responses To Anti-CER Arguments
  • Respondents randomly assigned to receive one of
    two anti-CER arguments one-size-fits-all and
    rationing
  • Respondents randomly assigned to receive one
    pro-CER rebuttal argument
  • For one-size-fits-all argument, the four
    rebuttals were
  • CER is supported by doctors
  • One-size-fits-all is a scare tactic
  • It is important to learn what works best for most
    patients
  • Research is not limited to studying average
    treatment effects and can incorporate group
    differences
  • Respondents rated persuasiveness of pro and con
    arguments (0con more persuasive 100pro more
    persuasive)

Survey conducted 7/30-31/2010 (N2000)
103
Experiment 2 Results
  • One size fits all
  • Doctors Support Scare tactic Knowledge of
    Can design
  • 65.8 51.3 typical patient research
    to
  • is valuable study
    subgroups 52.4 59.9

104
Conclusions
  • While many Americans favor CER, public support is
    conditional on how research findings are
    translated into practice
  • Uses of CER that are perceived to interfere with
    the doctor-patient relationship or that smack of
    rationing will spark opposition among politically
    engaged citizens
  • It will be critical for the Institute to generate
    information that consumers find personally useful
    and to demonstrate that CER can produce tailored,
    nuanced recommendations that wont lead to
    cookie-cutter medicine
  • The public trusts doctors and looks to them for
    guidance
  • The political sustainability of CER will depend
    in part on whether individual doctors and medical
    societies actively defend CER both in general and
    with respect to the usefulness of studies
    conducted within their practice areas

105
Many Americans Oppose A CER Board Establishing
Mandatory Guidelines
Survey conducted 5/21-24/2010 (N2017)
106
To Tame Costs, Link CER To Consumer Rewards
Rather Than Penalties
Survey conducted 5/21-24/2010 (N2017)
107
Health Reforms Tortuous Route To The
Patient-Centered Outcomes Research Institute
  • Kavita Patel, M.D., M.S.
  • Director, Health Policy Program
  • patel_at_newamerica.net

108
Process and Obstacles
  • Evolution from previous proposals, similar ideas
    and language
  • Starting in 2007
  • Dem control of Congress
  • 2009 Health reform debate
  • Senate side
  • HELP and Finance Committees
  • House side
  • Leadership Tri-Committee bill
  • USPSTF recommendations, Nov. 2009

109
Creating PCORI
  • Structural concerns
  • Inside government (AHRQ) vs. independent outside
    entity
  • Scope of power
  • Cost and coverage provisions in bill language
  • Fears of rationing
  • Must fill need for unbiased scientific evidence

110
PCORI Membership
  • Chairman Eugene Washington MD, MSc
  • Vice-Chair Steve Lipstein MHA
  • Clinicians, Researchers and Delivery System
    Experts
  • Sharon Levine MD Robert Zwolak MD, PhD Arnold
    Epstein MD Harlan Krumholz MD Debra Barksdale
    PhD, RN Christine Goertz DC, PhD Lawrence
    Becker Grayson Norquist MD, MSPH Kerry Barnett
    JD
  • Consumer groups
  • Ellen Sigal PhD Allen Douma MD Andrew Imparato
    JD
  • Industry
  • Freda Lewis-Hall MD Harlan Weisman MD Richard
    E. Kuntz MD
  • State or Federal Government
  • Robert Jesse MD, PhD Leah Hole-Curry JD AHRQ
    NIH Directors

Source GAO Announces Appointments to New
Patient-Centered Outcomes Research Institute
(PCORI) Board of Governors. Sept 23, 2010.
http//www.gao.gov/press/pcori2010sep23.html
111
Outlook
  • What if Scott Brown had not won?
  • Potential loss of momentum if Democrats lose
    majority in upcoming elections?
  • High value of CER to clinicians and patients?
  • What will happen to the NIH, AHRQ, FDA operations
    around CER and their methods?
  • Could there be an independent review process
    established by PCORI for NIH?AHRQ CER ?

112
  • With thanks to
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