9/23/2010

1

ITHS Comparative Effectiveness Research (CER) Symposium

Sean D. Sullivan, PhD

Scott D. Ramsey, MD, PhD

Outline

• What is the problem as perceived by the policy makers?

• Health Care Reform in the US

• What is comparative effectiveness: US style?

• Will comparative effectiveness work?

The President, OMB, and HHS

• “Better information about the costs and benefits of different treatment options…could eventually lower health care spending…”– Peter Orszag, CBO, Testimony from Congressional Hearing on 6/12/07

• "I think there's a general recognition that the system we have in America is fundamentally broken. We spend more than any country on Earth. Our health results look like we're a developing nation." – Secretary Kathleen Sebelius, HHS, CNN’s “State of the Union,” 8/16/09

3

9/23/2010

2

Average Health Insurance Premiums and Worker Contributions for Family Coverage, 1999‐2008

$5,791

$12,680119%

Increase

Source: Kaiser/HRET Survey of Employer-Sponsored Health Benefits, 1999-2008.

$5,791

117% Increase

Demographics = unsustainable spending growth rates???

25

20

g (

$ Tr

illio

ns)

CBO Projections for Social Security, Medicare, and Medicaid

Source: Congressional Budget Office, “The Long-Term Budget Outlook,” December 2003Assumptions: Excess cost growth of 2.5% for both Medicare and Medicaid; Social Security benefits paid as scheduled under current law

15

10

5

2003

2005

2007

2009

2011

2013

201

5

201

7

2019

2021

2023

2025

2027

2029

2031

2033

2035

2037

2039

2041

2043

2045

2047

204

9

Fed

eral

Sp

end

ing

$265B$158B$455B

Medicare$13.2T

Medicaid$4.4T

$17.6T

$423B

Social Security$5.2T

42x

11x

5

Federal Spending Under CBO’s AlternativeFiscal Scenario – Health Care Will Bankrupt America

Percentage of Gross Domestic Product

30

40Actual Projected

1962 1972 1982 1992 2002 2012 2022 2032 2042 2052 2062 2072 2082

0

10

20

Medicare and Medicaid

Social Security

Other Spending (Excluding debt service)

9/23/2010

3

Estimated Contributions of Selected Factors to Long‐Term Growth in Real Health Care Spending per Capita, 1940 to 2000

Smith, Heffler, and Freeland (2000)

Cutler (1995)

Newhouse (1992)

Aging of the Population 2 2 2

Changes in Third-Party Payment

10 13 10

Personal Income Growth 11 18 5 <2311-18 5 <23

Prices in the Health Care Sector

11-22 19Not

Estimated

Administrative Costs3-10 13

Not Estimated

Defensive Medicine and Supplier-Induced Demand 0

Not Estimated

0

Technology-Related Changes in Medical Practice 38-62 49 >65

Misdiagnosing the Problem

• Orzag Hypothesis: 

–Rising cost per beneficiary, not the b f b fi i inumber or type of beneficiaries

–What do we get for all the money we spend in the health care sector?

Variation in Medicare Spending

9Source: Dartmouth Atlas of Health Care

9/23/2010

4

10

11Veenstra

Comparative effectiveness research:Definition 

The generation and synthesis of evidence that compares the benefits and harms of alternativecompares the benefits and harms of alternative methods to prevent, diagnose, treat, and monitor a clinical condition, or to improve the delivery of care.

Institute of Medicine

9/23/2010

5

Purpose of CER

• 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

What’s unique about CER?It includes all of the following• Direct, head‐to‐head comparisons.

• Broad range of topics.– tests, treatments, strategies for prevention, care delivery and monitoring

• A broad range of beneficiaries: – patients, clinicians, purchasers, and policy makers.

• Study populations representative of clinical practice

• Focus on patient‐centered decision‐making– tailor the test or treatment to the specific characteristics of the patient.

“Patient‐centered”

• Suppose a RCT shows that A>B, but many patients got better on B.

– Lacking any additional knowledge, you should prefer A.

• Is it possible that some patients would have done better on B than A?

– Can we identify them in advance?

– Role of cross‐over RCTs

9/23/2010

6

Institute of Medicine – Committee on Comparative Effectiveness Research Prioritization

• Formed by ARRA to recommend national priorities for comparative effectiveness research

• IOM operationalized this charge by identifying three objectives*:– 1) Establish a working definition of CER

– 2) Using broad stakeholder input, develop a priority list of research ) g p , p p ytopics for ARRA funding

– 3) Identify necessary requirements to support “a robust national CERenterprise”

16*Committee on Comparative Effectiveness Research Prioritization, Institute of Medicine.  Initial National Priorities for Comparative Effectiveness Research.  June 2009.

The IOM report on initial national priorities for CER

1. A list of 100 conditions

2. 4 recommendations about priority‐setting

3. 8 recommendations about a national CER institute

• Sox and Greenfield.  Ann Int Med. 2009;151:203‐05.

• Ratner R et al. Initial priorities for CER. Washington DC. National Academies Press. 2009 (available free on‐line at iom.edu)

IOM Priority Criteria

• Burden of disease 

– Prevalence (MEPS)

– Mortality (National Vital Statistics)

Morbidity (MEPS)– Morbidity (MEPS)

– Cost (MEPS)

– Variability (Dartmouth Atlas)

• Gaps in evidence

• Potential of CER to change practice

9/23/2010

7

CER Priorities List: Distribution by research area

CER Priorities: Top Quartile List(order does not indicate rank within each quartile)

• Atrial Fibrillation treatment

• Assistive hearing devices

• Preventing falls in elderly

• Use of upper endoscopy in GERD

• Dissemination and translation of CER research results

• Care coordination/medical homes

• Prevention of obesity, diabetes, CHD in at‐risk populations (e.g., urban poor)

• Management of DCIS

• Imaging (PET, MRI, CT) in managing cancer patients

• Genetic & biomarker testing vs usual care in detecting & treating cancerCare coordination/medical homes

• Biologics in inflammatory diseases

• Interventions to eradicate MRSA

• Reducing nosocomial infections

• Management of localized prostate cancer

• Treatment of low back pain

• Detection & mgmt of dementia

• Behavioral disorders in Alzheimer’s

• School‐based interventions to reduce childhood obesity

• Effectiveness of various delivery models in preventing dental caries in children

• Tx modalities for ADHD in children

• Home and community based services in managing serious emotional disorders

• Interventions to reduce health disparities

• Clinical interventions to reduce infant mortality, low birth weight, prematurity, etc.

• Innovative contraception strategies

IOM CER Priority Topics

• Health care delivery systems

• Racial and ethnic disparities

• Cardiovascular and peripheral vascular

• Geriatrics• Geriatrics

• Functional limitations and disabilities

• Neurologic disorders

• Psychiatric disorders

• Pediatrics

9/23/2010

8

The IOM: a national CER program should:

• Do priority‐setting on an ongoing basis.

• Have a broadly representative oversight committee

• Engage public participation at all levels of CER

• Support large‐scale, clinical and administrative data networks

• Do research on dissemination of CER findings

• Support research and innovation in the methods of CER

• Expand and support the CER workforce

CER Approaches• Randomized Controlled Trial

• Pros

• Best quality evidence

• Highest likelihood of changing practice

• Cons

• Artificial nature of trial care and study populations

• Expensive (especially head‐to‐head trials)

• Time consuming (technology marches on)

CER Approaches• Systematic review

– Review of existing studies

• Retrospective study 

– e.g., insurance claims analysis, analysis of patient records

• Pros:• Pros:

– Inexpensive

– Relatively quick

– Claims and chart data more representative of practice

• Cons:

– Systematic reviews merely summarizes existing evidence (with all it’s flaws)

– Retrospective studies subject to biases (especially confounding by indication)

– Limitations may prevent acceptance by clinicians

9/23/2010

9

“Valid” CER Endpoints:“Valid” CER Endpoints:An Ongoing ControversyAn Ongoing Controversy

• Intermediate measures vs. definitive outcomes

• e.g., tumor response vs. survival

• Patient Oriented OutcomesPatient Oriented Outcomes

• Symptoms vs. functional status vs. QOL vs. QALYs

• Caregiver burden

• Workplace productivity

• Cost‐effectiveness

• Decision Quality (HS)

Amendment by Rep Gingrey (R‐Ga) in E&C bill, July 2009

• “CMS may not use federally funded clinical CER data… for medical treatments, services, items… on the basis of costs”

• Approved in voice vote!

Where is CER going? 

• Expanding resources (ARRA funds as a “down payment”)

• PCORI•Synthesizing existing evidence vs generating new•Synthesizing existing evidence vs generating new evidence

•New regional efforts

9/23/2010

10

American Recovery and Reinvestment Act of 2009

• $1.1 billion for CER through 9/30/2010

– AHRQ: $300 million

– NIH : $400 million (appropriated to AHRQ and transferred)

– Office of the Secretary: $400 million

• $20 billion to CMS for Health IT Beginning in 2011 to Provide Incentives for EMR Adoption

– $2 billion in discretionary funds

– $18 billion in Medicare/Medicaid Incentives

28

Acknowledgement to Craig Hunter, UBC for permission to use these slides

• Current CER-type Programs

– Effective Health Care Program (MMA 1013)

• Evidence-based Practice Centers (EPCs)

Agency for Healthcare Research and Quality

• DEcIDE Research Network

• Eisenberg Center

– Centers for Education and Research on Therapeutics (CERTs)

– Technology Assessment Program (TAP)

29

• $700 million of $1.1 billion from ARRA for CER– $400 passed through to National Institutes of Health– $148 million for Evidence Generation, to include:

• $100 million for Clinical and Health Outcomes Initiative in Comparative Effectiveness (CHOICE)

• $48 million for patient registries$

Agency for Healthcare Research and Quality

– $29.5 million to support “innovative translation and dissemination”– $20 million to support CER training and development– $9.5 million for CER infrastructure– $10 million to “Citizen’s Forum” for stakeholder involvement– $1 million in other grants– $50 million toward existing evidence synthesis contracts– $24 million for additional (unspecified) evidence generation– $5 million for translation and dissemination– $3 million for three full-time ARRA-specific positions

30

9/23/2010

11

America’s Healthy Future Act of 2009:Comparative Effectiveness

• Patient‐Centered Outcomes Research Institute– “The purpose of the [private, non‐profit] Institute would be to assist 

patients, clinicians, purchasers, and policy makers in making informed health decisions by advancing the quality and relevance of clinical y g q yevidence through research and evidence synthesis.”β

– Can conduct range of Comparative Effectiveness Research (meta‐analysis, RCTs, novel approaches) within guidance from methods and prioritization advisory panels

β Chairman’s Mark, America’s Healthy Future Act of 2009

31

Patient‐Centered Outcomes Research Institute (PCORI)

• Key appointments by Comptroller General in September– Director

– Governing Board (stakeholder representatives)• AHRQ Director or delegate• NIH Director or delegateP i / i (3)• Patient/consumer representatives (3)

• Provider representatives (7, including 4 physicians, 1 nurse, 1 alternative med practitioner, 1 hospital representative)

• Payers (3, at least one insurer and one self‐insured employer)• Manufacturers (3)• Independent health services researcher • Federal and State Government program representatives (2)

PCORI: Committees• Methodology committee (15 members)

– Experts in health services research, clinical research, CER, biostats, genomics, and research methods

– AHRQ and NIH Directors or representatives

– Very important from our perspective, since this committee will greatly impact the validity and usefulness to our P&T of the results of PCORIimpact the validity and usefulness  to our P&T of the results of PCORI‐sponsored research

• Standing expert advisory panels

– Practicing and research clinicians

– EBM, health services research experts, healthcare delivery experts, patient and manufacturer representatives

• Ad hoc advisory panels

9/23/2010

12

PCORI: Statutory responsibilities • Methodological standards for CER

• Unbiased peer review

• Integrity and adherence to standards

• Process transparency, including public comment

• Primary research (prospective & retrospective)• Primary research (prospective & retrospective)

• Systematic reviews and evidence synthesis

– Publish draft findings of systematic reviews

– Accept public comment before finalizing

• Publicize research findings

PCORI: Contracting and funding

• PCORI can contract for research with:

– AHRQ and NIH (preferred) 

– Other federal agencies

– Academic and other entities 

• Contractor requirements: Transparency COI management MethodologyContractor requirements: Transparency, COI management, Methodology

• Pt‐Centered Outcomes Research Trust Fund  funded by:

– Congressional appropriations:

• FY 2010 ‐ $10 million

• FY 2011 ‐ $50 million

• FY 2012 and thereafter ‐ $150 million/year

– Transfers from federal medical insurance trust funds

– Health plan taxes (Begins in 2013)

America’s Healthy Future Act of 2009:Comparative Effectiveness (cont’d)

• Patient‐Centered Outcomes Research Trust Fund

– Establishes new trust to pay for the Institute

– Funding comes from multiple sources including:

• $1.26 billion from General Treasury (total for FY2010‐2019)

• $10 million from ARRA funds appropriated to the Secretary (2009/10)

• Fees on Medicare insured and self insured (FY2013 2019)• Fees on Medicare, insured, and self‐insured (FY2013‐2019)

– Medicare

» $1 per average number of Part A and B enrollees for FY2013 and $2 per average number of Part A and B enrollees for FY2014‐19 (updated by the rate of medical inflation)

– Insured and self‐insuredµ

» $1 in FY2013 and $2 in FY2014‐19 on each health insurance policy in the United States multiplied by the number of lives covered under that policy (updated by the rate of medical inflation) 

µApplicable self‐insured health plans in the United States would be defined as plans providing accident or health coverage provided other than through an insurance policy and maintained by a plan sponsor for the benefit of members, employees or former employees, etc.

36

9/23/2010

13

America’s Healthy Future Act of 2009:Medicare Commission

• Similar to S. 1110: Medicare Payment Advisory Commission (MedPAC) Reform Act of 2009, but with fewer teeth– Scope of new commission limited to making proposals to Congress

• 15 person commission appointed by the President and confirmed by the Senate

• Creates new commission directed to “…develop and submit proposals to Congress aimed at extending the solvency of Medicare, slowing Medicare cost-growth, and improving the quality of care delivered to Medicare beneficiaries.”

37

Dissemination of research results• PCORI must publicize research results within 90 days in a manner useful 

to clinicians, patients and the public 

• AHRQ Office of Communication and Knowledge Transfer will receive substantial funding to:

– Disseminate results of federally‐funded CER

– Create informational tools for providers patients payers and policy makersCreate informational tools for providers, patients, payers, and policy makers

– Maintain a database of CER results (public & private)

– Supply data to clinical decision support system vendors, professional associations, and health plans

– Obtain user feedback as to the usefulness of the information

PCORI: Statutory Limitations

• Must evaluate potential differences in subpopulations and include as research subjects whenever appropriate:– Racial and ethnic minorities

– Women, elderly and children

– Individuals with different 

• Comorbidities• Genetic and molecular sub‐types• Quality of life preferences 

• Prohibited from making clinical practice guideline recommendations

9/23/2010

14

Limitations on use of CER in coverage decisions for federal programs

• PCORI cannot mandate coverage policy, public or private

• HHS may use results of PCORI‐funded CER to make coverage determinations only if the coverage process is

– Iterative, transparent, and includes public comment

– Considers the effect on subpopulatonsConsiders the effect on subpopulatons

– Does not deny coverage solely on the basis of CER

• May not discount the value of extending life of elderly, disabled or terminally ill may

• QALY thresholds for coverage are explicitly prohibited, but AHRQ can and already does use QALYs!

• This does not prevent the use of Life Years gained at PCORI

Publicly expressed concerns about PCORI• Ability to address needs of small subpopulations

• Office of the Assistant Secretary for Planning and Evaluation (ASPE) is establishing a CER inventory and may set standards that shortcut the PCORI process

• Legislative restrictions on the use of CER

• Ability to deliver required cost savingsAbility to deliver required cost savings

• “Death panels” and denial of care to the elderly

• Cost‐effectiveness and rationing

• Use of lower quality evidence may lead to wrong conclusions and marginalize useful treatments

• Will 2010 elections change the direction?

Veenstra 42

9/23/2010

15

Community CER CollaborationsCommunity CER Collaborations

• Providers

• Health insurers

• Patient representatives

• Purchasers (business)

• Researchers

Veenstra 44

The Promise of CER

Information to help providers and patients make better decisions

9/23/2010

16

CER Symposium Agenda

TODAY

• Involving stakeholders in CER – Sean Tunis and Larry Kessler

• Hazards of CER – Rick DeyoHazards of CER  Rick Deyo

• Community registries and research networks for CER – Jerry Jarvik and Dave Flum

• Qualitative and quantitative evidence synthesis for CER – Fred Wolf and Rahber Thariani

• Using ITC and MTC methods to estimate CER in the absence of head‐to‐head trials – Beth Devine and Rafael Alfonso

CER Symposium Agenda

TOMORROW

• Pragmatic Trials for CER – Scott Ramsey and Scott Emerson

• Observational research in CER – Diana BuistObservational research in C R iana uist

• Health economics and VOI for CER prioritization – Dave Veenstra and Josh Carlson

• Innovative approaches to coverage with CER – Lou Garrison and Josh Carlson

References1. Emanuel EJ, Fuchs VR, Garber AM. Essential Elements of a Technology and 

Outcomes Assessment Initiative. JAMA 2007;298;1323‐25.

2. Federal agency Web sites: www.fda.gov, www.ahrq.gov, www.nih.gov, www.iom.edu, www.cms.gov.

3. IOM Report. Initial National Priorities for Comparative Effectiveness Research.  http://www.iom.edu/Reports/2009/ComparativeEffectivenessResearchPriorities.aspx.

4 Patient Protection and Affordable Care Act Public Law 111‐148 Title VI Sections4. Patient Protection and Affordable Care Act. Public Law 111 148, Title VI, Sections 6301‐6302 (2010).

5. PCORI Should Take Lead On Public CER Inventory, Pharma Groups Tell HHS. The Pink Sheet 8/23/2010. 

6. PHRMA. PhRMA Statement on PCORI Nominees. http://www.phrma.org/news.

7. Reichard J. PCORI Backers Eye PR Strategy to Cool 'Death Panel' Rhetoric. http://www.commonwealthfund.org/Content/ Newsletters/ Washington‐Health‐Policy‐in‐Review/2010/Jun/June‐28‐2010/PCORI‐Backers‐Eye‐PR‐Strategy.aspx.

8. Sox HA. Comparative Effectiveness Research: A Progress Report. Ann Intern Med2010;153(4), E‐pub 8/2/2010. http://www.annals.org.