Gaining Coverage and Reimbursement in the Age of Comparative Effectiveness

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• Gaining Coverage and Reimbursement in the Age of
  Comparative Effectiveness
• John Hornberger, MD, MS

Bio2Device Presentation June 9, 2009 Presented
at the TIPS Group 1000 Elwell Court, Suite
150 Palo Alto, CA 94303
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Outline

• Healthcare reform
• ARRA Comparative Effectiveness
• Health technology assessment
• Crystal ball
• Implications for product development and
  commercialization

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Outline

• Healthcare reform
• ARRA Comparative Effectiveness
• Health technology assessment
• Crystal ball
• Implications for product development and
  commercialization

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Why healthcare reform?Quality safety
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Why healthcare reform?Quality safety
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Why healthcare reform?Quality safety
Murray C et al. Eight Americas Investigating
Mortality Disparities across Races, Counties, and
Race-Counties in the United States. Plos
Medicine, 2006.
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Why healthcare reform?Quality safety
Murray C et al. Eight Americas Investigating
Mortality Disparities across Races, Counties, and
Race-Counties in the United States. Plos
Medicine, 2006.
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Why healthcare reform?Quality safety
each year more than 40,000 Americans suffer
from a preventable hospital death.
2001 Institute of Medicines report Crossing the
Quality Chasm A New Health System for the 21st
Century
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Why healthcare reform?Insurance the uninsured
Percentage of U.S. workers who were uninsured in
2006-2007, ages 19-64.
19 or more
14 18.9 or more
Less than 14
Estimated gt 48 million in 2009
Covertheinsured.org. A project sponsored by the
Robert Wood Johnson Foundation.
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Why healthcare reform?Insurance the
underinsured
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Why healthcare reform?Affordability
Estimates suggest that as much as 700 billion a
year in health care costs do not improve health
outcomes. It occurs because we pay for more care
rather than better care. Peter Orszag. White
House Budget Director, NRP interview, April 2009
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Why healthcare reform?Affordability
THE COST CONUNDRUM What a Texas town can teach us
about health care. by Atul Gawande. The New
Yorker, June 1, 2009.
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Why healthcare reform?Affordability
McAllen, TX
Population 700,000 Medicare
spending per capita 15,000 Per capita income
12,000
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Why healthcare reform?Affordability
El Paso, TX
Population 736,000 Medicare spending per
capita 7,504 Per capita income 14,388
McAllen, TX
Population 700,000 Medicare
spending per capita 15,000 Per capita income
12,000
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Why healthcare reform?Affordability
El Paso, TX
Population 736,000 Medicare spending per
capita 7,504 Per capita income 14,388
McAllen, TX
Population 700,000 Medicare
spending per capita 15,000 Per capita income
12,000
In situations in which the right thing to do was
well establishedfor example, whether to
recommend a mammogram for a fifty-year-old woman
(the answer is yes)physicians in high- and
low-cost cities made the same decisions.
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Why healthcare reform?Affordability
El Paso, TX
Population 736,000 Medicare spending per
capita 7,504 Per capita income 14,388
McAllen, TX
Population 700,000 Medicare
spending per capita 15,000 Per capita income
12,000
In situations in which the right thing to do was
well establishedfor example, whether to
recommend a mammogram for a fifty-year-old woman
(the answer is yes)physicians in high- and
low-cost cities made the same decisions. But,
in cases in which the science was unclear, some
physicians pursued the maximum possible amount of
testing and procedures.
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Outline

• Healthcare reform
• ARRA Comparative Effectiveness
• Health technology assessment
• Crystal ball
• Implications for product development and
  commercialization

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American Recovery and Reinvestment Act of 2009

• Total 147.7 billion

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Comparative Effectiveness Research

• Funding
• AHRQ - 300 m for Effective Healthcare program
• NIH - 400 m
• Office of HHS Secretary - 400 m
• Setting priorities gathering stakeholder input
• Institute of Medicine (IOM) report due June
  30th
• Guidance Coordination
• Federal Coordinating Council for Comparative
  Effectiveness

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Outline

• Healthcare reform
• ARRA Comparative Effectiveness
• Health technology assessment
• Crystal ball
• Implications for product development and
  commercialization

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Judging Quality is Not Easy
The Salon de Paris
Honoré Daumier 'Free day at the Salon' From the
series "Le Public du Salon," published in Le
Charivari (May 17, 1852) p10
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Perspective of Quality - 1864
Jean-Louis-Ernest Meissonier (1815-1891)
Campagne de France
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Perspectives on Quality Evolved
Édouard Manet (1832-1883) Music at the
Tuileries (1862)
Daumier satirized the bourgeoises scandalized by
the Salon's Venuses, 1864
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Not Consensus
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Its Convergence
Goal Improved health, affordable
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Health technology assessmentWho?

• United States
• Government
• AHRQ Evidence-based practice centers
• CMS
• CDC EGAPP
• ? New separate federal agency
• Private
• BCBS Technology Evaluation Centers
• California Technology Assessment Foundation
• Hayes Group/ECRI
• Outside of United States
• Canada each province plus the Common Drug
  Review (CDR)
• United Kingdom National Institute for Health
  and Clinical Excellence (NICE)
• Australia Pharmacy Benefit xxxx (PBAC)
• France Transparency Commission
• And so forth

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Health Technology AssessmentExample
Molecular classifiers
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Types of tests/evaluation
Known or probable disease
No signs or symptoms, no known disease
Cause unknown
Cause known
Risk-factor assessment (susceptibility)
Determine cause (diagnosis)
Disease extent or severity (staging)
Presence of occult disease (screening)
Refine differential diagnosis
Predict natural history (prognosis)
Predict response to intervention (prediction)

• Information gathering (disease intelligence)
• HPI/ the physical exam
• imaging
• biopsy

Monitor disease course (surveillance)
Assess response to treatment

• Adapted from
• Harrison's Principles of Internal Medicine, 17th
  Edition. Editors Fauci AS et al. The McGraw-Hill
  Companies.
• Whiting P et al. A review identifies and
  classifies reasons for ordering diagnostic tests.
  J Clin Epidemiol 2007 981-9.
• Fischbach T. Manual of Laboratory Diagnostic
  Tests, 7th Edition. Lippincott Williams
  Wilkins Philadelphia. 2004.

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The appraisal processA brief history
2005/6 REMARK
2009 JAMA series
1989 Wald Cuckle
2000 to 2003 ACCE
2009 ACCE
2001 Fryback Thornbury
1996 TMUGS
2006 Ramsey et al.

• 1. Canadian Task Force on Preventative
  Services, 1976
• Frame et al. J Fam Pract 1975
• Blue Cross/Blue Shield Technology Evaluation
  Center, 1985

2001 US Preventive Services Task Force
2006 Simons checklist

• Others
• Hayes Group
• ECRI

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The appraisal processRCT versus Chain of Evidence
Harris RP et al. Current methods of the U.S.
Preventive Services Task Force A review of the
process. 20012021-35.
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iACCEp v3.0 (beta)
ISPOR Workshop, May 2009
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Presenting the evidence

• In a study requiring interpretation of
  mammography outcomes, almost all physicians
  confused the sensitivity of the test with its
  positive predictive value

Jean Slutsky (AHRQ) on June 7, 2007 referring to
Hoffrage et al. Communicating Statistical
Information. Science 20002902261-2.
http//www.google.com/search?hlenqahrqsensitiv
ityslutsky
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Presentation of evidenceDetection Cystic
fibrosis screening
Sensitivity 74 Specificity 99.9994 PPV
99.5 NPV 99.96
http//www.cdc.gov/genomics/gtesting/ACCE/FBR/CF/C
FCliVal.htm
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Presentation of evidence Detection Hereditary
colon cancer risk factor (germ-line mutations by
MSI)
Sensitivity 91 Specificity 82
AUC 87 (se 3)
MSI microsatellite instability
Hampel et al. Screening for the Lynch syndrome
(hereditary nonpolyposis colorectal cancer).
NEJM, 2005 3521851-60.
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Presentation of evidence Prediction
Paik et al. A multigene assay to predict
recurrence of tamoxifen-treated, node-negative
breast cancer. NEJM, 2004 3512817-26.
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Presentation of evidence Prediction
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Communications ResearchAHRQs John M Eisenberg
Center
First established at Oregon Health Sciences
University, led by Dr. David Hickam. In 2008,
moved to Baylor College of Medicine.

• https//www.fbo.gov/index?sopportunitymodeform
  id25f55835a3f1d7004c1e7a709d3e5a50tabcore_cvie
  w1cck1auck

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DetailsResearch design and statistical issues

• Some relevant research design questions
• How many studies required?
• Is randomization required? Why or why not?
• Are surrogates or intermediate endpoints
  appropriate?
• How were cutoffs chosen?
• What is a clinically meaningful minimum
  difference?
• How might homogeneity affect generalizability of
  the findings?
• How to interpret the study findings if the
  standard of care has changed since (or during)
  the study is completed?

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DetailsEconomic implications and validity
Adapted from Weinstein M, OBrien B, Hornberger
J. et al. Principles of good practice for
decision analytic modeling in health-care
evaluation report of the ISPOR Task Force on
Good Research Practices--Modeling Studies. Value
Health 2003 69-17.
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DetailsEconomic implications and validity
Adapted from Weinstein M, OBrien B, Hornberger
J. et al. Principles of good practice for
decision analytic modeling in health-care
evaluation report of the ISPOR Task Force on
Good Research Practices--Modeling Studies. Value
Health 2003 69-17.
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Outline

• Healthcare reform
• ARRA Comparative Effectiveness
• Health technology assessment
• Crystal ball
• Implications for product development and
  commercialization

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Health technology assessment20 years ago
Cant talk about money
Its unethical
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Health technology assessment20 years ago
Irrevelant
Not interested
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Health technology assessment20 years ago
Too many assumptions
Not scientific
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Health technology assessment10 years ago
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Health technology assessmentToday
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CommercializationThe Past
Approval
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CommercializationThe present
Approval
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CommercializationThe present
Approval
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CommercializationThe present
Approval
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Guidelines on GuidelinesInternational Society
for Pharmacoeconomic and Outcomes Research
http//www.ispor.org/workpaper/practices_index.asp
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Guidelines on GuidelinesAcademy of Managed Care
Pharmacy
AMCP Format for Formulary Submissions Version 2.1
Explanation The AMCP Format for Formulary
Submissions is a set of guidelines, a template
that drug companies can use to prepare
submissions of new and existing pharmaceuticals
for a health system's Pharmacy and Therapeutics
(PT) Committee.  Previously, PT Committees
often received drug information passively from
pharmaceutical manufacturers that was biased and
of poor quality.  In this era of dramatically
increasing drug costs, biotechnology, and
information availability, the Format empowers
health systems to pro-actively request specific
information from manufacturers that will allow
them to more accurately determine the total value
that a drug brings to their population as the
basis for accepting or rejecting a drug for its
formulary. 
http//www.amcp.org/amcp.ark?p0F6E1295
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Guidelines on GuidelinesUK National Institute
for Health and Clinical Excellence
Technology appraisal process guides NICE has
produced a series of guides explaining its
technology appraisal process for stakeholder
organisations. The guides explain how to
contribute to an appraisal and submit
evidence. NICE and the Association of British
Pharmaceutical Industry (ABPI) have agreed
guidelines on the release of company data into
the public domain during a technology appraisal.
http//www.nice.org.uk/aboutnice/howwework/devnice
tech/technologyappraisalprocessguides/technology_a
ppraisal_process_guides.jsp
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Health Technology AssessmentThe Future
Various private and public initiatives that may
reduce investment in RD
Greater focus on affordability of health care and
desire to control costs -gt use HTA to address
this issue
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Health Technology AssessmentThe Future
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Health Technology AssessmentThe Future

• What really affects quality?
• Level of educational attainment is most
  important determinant of population
  health(Fuchs, 1988)
• e.g., 1 in 4 high school students fail to
  graduate
• This has gotten relatively little play as part of
  the health policy debate

?
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Health Technology AssessmentThe Future

• What really affects costs?
• After billions spend on War on Cancer, recent
  data suggest very little reduction in overall
  mortality (age-adjusted, etc.)
• Cancer biology is very complex (only understand
  the very tip of the iceberg)
• Solutions are largely empiric
• Same goes with our complex health care system
• Health care reform solutions offered in 2009 are
  largely empiric

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