One of These Patients is Not Like The Others:
Development and Application of a Tool for Health
Care Decision Makers to Assess and Consider
Heterogeneity in Coverage Decisions
An ISPOR Workshop
Jennifer S. Graff, PharmD, Director, National Pharmaceutical Council
Karen Van Nuys, PhD, Senior Research Economist, Precision Health Economics
Michael X. West, PhD, Senior Payer Medical Science Liaison, GlaxoSmithKline
May 21, 2013
Disclosures
JG is employed by the National Pharmaceutical Council,
a policy research organization supported by the
nation’s major research-based pharmaceutical
companies
KVN serves as a consultant for the life sciences industry.
MW is employed by GSK
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Workshop Description
• Environmental contest: bridging the gap between
heterogeneity research and decision-making
• Demonstrate a framework illustrating how and when
applying average CER results to individual patients is
problematic for decision-makers
• Interactive discussion
– Audience application of the model
– Considerations of heterogeneity in formulary
decision-making
The “Average” Patient
…is based upon the results of many
Kent D. American Scientist 2007;95:60-68.
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“If it were not for the great variability
among individuals, medicine might as well
be a science and not an art….”
—Sir William Osler,
The Principles and Practice of Medicine 1892
Variation in Treatment Response Matters…
A treatment that is safe and effective for some
does not mean that it is safe and effective for all
BUT
A treatment that is not safe and not effective for
some does not mean that it is not safe and
not effective for all
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Our Understanding of Biology
is Growing Exponentially
Genome-wide Associations Reported
through March 2010
Baker. Nature 2012;487:7407.
Manolio TA. N Engl J Med 2010;363:166-176.
There are Many Causes
of Treatment Variability
• Age
• Gender
• Disease severity
• Use of concomitant medications
• Care setting
• Comorbidities
• Environment (e.g., diet)
…and then there are genetics…
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Methods to Analyze Heterogeneity of
Treatment Effect are Advancing
Increasing Recognition of the Importance
Of Individual Treatment Variation
AHRQ
“Comparative Effectiveness of
Medications to Reduce Risk of Primary
Breast Cancer”
“…Remaining Issues: While the efficacy
…has been demonstrated …it is not
clear which women in clinical practice
would optimally benefit from risk
reduction”
AMCP
Dossier 3.1
“More specifically, CER addresses
patient's preferences, their healthrelated quality of life and other
outcomes important to them.
It also emphasizes generating evidence
regarding subgroup differences, also
known as treatment heterogeneity.”
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Less Than 1 in 3 Plans Routinely Consider
Treatment Response Diversity in Medical Policy
N= 47 How often do you consider heterogeneity for setting the following types of medical policy?
Safeguards to Accommodate Individual
Patient Differences at a Policy Level
Medicare Part D
Protected Classes and
Criteria
“All or substantially all drugs”
Private Payer Benefit
Design and
Management
• Coverage of the medication class
– oral contraceptives, branded
antidiabetic medications,
• Coverage of individual medications
– Specific medication, tier status
and structure
• Utilization Management
– Quantity limits
– Step edits
– Prior authorization
Section 176 of the Medicare Improvements for Patients and Providers Act (MIPPA) of 2008 (Pub. L. No. 110275, 122 Stat. 2494, 2581 — 82)
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Individualized Care Exists When
Predictable Markers Exist
13
“According to our records, you had the
same illness 200 years ago in a
previous life. That qualifies as a preexisting condition.”
Copyright permission – Randy Glasbergen
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Consideration of Heterogeneity in
Newly Forming State Exchanges is Unknown
IMPLICATIONS FOR EVIDENCE
DEVELOPMENT AND COMMUNICATION
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Audience Discussion
Populations
Individuals
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