iths comparative effectiveness research symposium iths comparative effectiveness research (cer)...

9/23/2010

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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

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

$5,791

$12,680 119%

Increase

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

$5,791

117% Increase

Demographics = unsustainable spending growth rates???

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g ($

T ril

lio ns

)

CBO Projections for Social Security, Medicare, and Medicaid

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

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20 03

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20 07

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20 11

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20 27

20 29

20 31

20 33

20 35

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20 39

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Fe de

ra l S

pe nd

in g

$265B $158B $455B

Medicare $13.2T

Medicaid $4.4T

$17.6T

$423B Social Security

$5.2T

42x

11x

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

Percentage of Gross Domestic Product

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40 Actual Projected

1962 1972 1982 1992 2002 2012 2022 2032 2042 2052 2062 2072 2082 0

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20 Medicare and Medicaid

Social Security

Other Spending (Excluding debt service)

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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

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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

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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

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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 y

topics for ARRA funding – 3) Identify necessary requirements to support “a robust national CER

enterprise”

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

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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

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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

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

• Intermediate measure