New Era Of Comparative Effectiveness Research

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

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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
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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
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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

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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)
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Recovery Act CER studies funded through NIH span
the disease areas prioritized by IOM
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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

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Creating A High Performance System For
Comparative Effectiveness Research

• Lynn Etheredge
• Rapid Learning Project
• George Washington University

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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

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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

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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

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Comparative Effectiveness And Personalized
Medicine Evolving Together or Apart?

• Robert S. Epstein
• J Russell Teagarden

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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)

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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.
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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.
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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.
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Comparative effectiveness can also provide
evidence of clinical utility
Source Mallal S et al HLA-B5701 screening
for hypersensitivity to abacavir. NEJM
2008358568-79.
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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

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A Patient Advocates Perspective On
Patient-Centered Comparative Effectiveness
Research

• Tony Coelho
• Former Chair, Board of Directors, Epilepsy
  Foundation of America

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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
•  

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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?
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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

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Comparative Effectiveness Research in the
Decision Maker-Evidence-Policy Loop
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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
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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.
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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.

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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

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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
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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
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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
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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
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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

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Why Observational Studies Should Be Among The
Tools Used In Comparative Effectiveness Research

• Nancy Dreyer
• Chief of Scientific Affairs, Outcome

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Evidence Is Needed
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• 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
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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

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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?

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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)

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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
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Adding The Patient Perspective To Comparative
Effectiveness Research

• Albert Wu
• Professor, Health Policy and Management,
  Bloomberg School of Public Research, Johns
  Hopkins University

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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)

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Types of Information Captured In Data Used For CER
Research Data Clinical Care Data Administrative Data
Utilization -
Clinical detail
Patient perspective - - -
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Linking Data Sources for CER
Administrative Data
Patient Reported Outcome Data
Clinical Research and EHR Data
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Electronic Health Records (EHR)

• How can EHR be structured to include PRO
• As clinical indicator that can be tracked,
  profiled
• Useable for CER?

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Johns Hopkins Medical Institutions EPR
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Clinician Website

• Clinician can schedule a patient a survey

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Patient Website

• Patient is presented with survey(s) to complete

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Patient Website

• Patient survey sample question

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Clinician Website

• Clinician can view patients results

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

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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

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Thanks To Co-Authors
Claire Snyder Carolyn Clancy
Don Steinwachs
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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

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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

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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.

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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

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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.

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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.

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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

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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.

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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.

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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

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Paying Well For Meaningful Innovation
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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

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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

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• 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
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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

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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

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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

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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

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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

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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.

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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.

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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

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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

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Moving Comparative Effectiveness Research Into
Practice

• Implementation Science And The Role Of Academic
  Medicine

Ann Bonham Chief Scientific Officer, Association
of American Medical Colleges
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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.

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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.

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Role Of Academic Medicine

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

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The Way Forward

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

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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

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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.

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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

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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