Transcript Today’s Conversation

School of Pharmacy
”Show Me the Money”
What is the “Real” Value of
Personalized Medicine for Cancer
Care?
Kathryn A. Phillips PhD
Professor of Health Economics and Health Services Research
Director and Founder, UCSF Center for Translational and Policy Research
on Personalized Medicine
University of California, San Francisco
Today’s Conversation
• What is the value of personalized medicine in
cancer care?
• How are payers considering coverage and
reimbursement policies?
• How may new sequencing technologies change
the playing field?
2008: TRANSPERS Born
• Objective: Develop evidence of how
personalized medicine can be beneficial
and efficient
– Who has access to the newest technologies?
– How do patients and providers make decisions about using
personalized tests or drugs?
– What information do insurers need to make the most appropriate
decisions about coverage policies?
– How can we design better policies to encourage the most
effective use of technologies?
TRANSPERS Talks with Payers to
Understand Reimbursement
• Evidence & Reimbursement Council
• Founded 2007
• Senior executives:
– All 7 largest US plans & leading regional plans
– Thought leaders with industry (bio, pharma, lab,
PBMs), government, & Medicare perspectives
• Semi-annual meetings
• Funded by multiple NIH grants & foundations
Why is a Health
Economist/Health Services
Researcher Working on
Personalized Medicine?
Using Toolbox of Social
Science Approaches &
Methods…
…To Understand
Health Policy
Issues…
…In the Brave New World of
New Technologies &
Personalized Medicine
Wearing Four Hats
Academia
Patients &
Providers
Industry
Government
What is the value of personalized
medicine in cancer care?
Challenges/Opportunities for
Personalized Medicine
Shifting Industry
Paradigms
Determining Value &
Reimbursement
Balancing
Innovation &
Regulation
Building Evidence
Base
Research on HER2/neu Testing
for Herceptin – Paradigm for the
Future
Clinical Practice Patterns and Cost-Effectiveness of
HER2 Testing Strategies in Breast Cancer Patients.
Phillips KA, Marshall DA, Haas JS, Elkin EB, Liang SY, Hassett MJ, Ferrusi I, Brock JE, Van Bebber
SL
Translating HER2 Testing to
Practice & Policy
No data on
uninsured,
Medicaid
recipients, or
minorities
~20% of IHC tests at
community labs may
be inaccurate
Up to 20% of
negative
women still
get Herceptin
Costeffectiveness
analyses
assume
perfect testing
Some women get
IHC, some FISH,
some both
Claims & medical
records for testing
do not match 25%
of time
60% of positive
women – esp.
lower income – do
not get Herceptin
The economic value of personalized medicine
tests: what we know and what we need to
know
Summary of policy implications
• Information on clinical utility, economic value,
affordability, and public health implications is essential for
appropriately assessing new technologies.
• Methods are needed to prioritize and conduct early and
rapid assessments of clinical utility and economic value,
before widespread adoption of new technologies.
• It is critical to consider the true value of diagnostics and
not impede the need for innovation because of the need
to consider economic value.
• Balancing innovation and affordability is a shared
responsibility.
How are payers considering
coverage and reimbursement
policies?
Long Adoption Curve for Plan
Coverage for OncotypeDx
• OncotypeDX took four years to be adopted
by all payers
– Payers considered same evidence but
weighted factors differently
– Tipping points:
• How clinical evidence interpreted
• Health care system factors (patient & provider
demand, Medicare coverage, guidelines)
• Lack of FDA approval not deal-breaker
Variation in Health Technology
Assessments Used by Payers
P1 P2
P3
P4
P5
P6
P7
P8
P9
BCBS
TEC
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USPSTF
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ICER
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Hayes
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EGAPP
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ECRI
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UP-TODATE
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Total per
payer
7
6
P10
P11
# of
payers

10
9
7

5

5


3
2
6
5
3
3
3
3
2
2
1
How may new sequencing
technologies change the playing
field?
Many Different Types of Sequencing
Gene Panels
Whole
Whole
Exome
Genome
Sequencing Sequencing
Single Gene Testing
- Inherited/germline vs. acquired/somatic
- Disease specific vs. general genomic medicine
Tumor vs. Germline Sequencing
“Person” sequencing – germline
Tumor sequencing
Individual
tumor
targets
Tumor
Sequencing
panels
Whole
tumor –ome
sequencing
Incidental
findings
Whole
genome
sequencing
Multi-gene
susceptibility
panels
Individual
gene tests
Sequencing = Sequencing
Sequencing of tumors for immediate treatment
decisions using established gene panel to look for
variants with known clinical utility
VS
Sequencing in general population to look for any
variants that may be predictive of future risk of
disease regardless of lack of any known clinical
utility for those variants
2008, $1.5M, 4 months
Mutation inhibiting betablockers – but fatal
illness?
2011, $100K
Money well-spent?
8 babies in extended family
died before sequencing
identified cause
Sequencing found genetic
risk for diabetes – but then
couldn’t get life insurance
Cost of Whole Genome
Sequencing is Falling
Emerging Debates:
Should Sequencing Be Widely Used?
“We will only achieve the promises of
sequencing if the information gathered is
“useful, cost-effective, and welcomed”
• Jim Evans, Science, 2011
Emerging Debates:
Who Will Pay?
“It would be very, very challenging to collect
and analyze enough information to convince
CMS that whole-genome sequencing should
be covered by Medicare”
• Jeffrey Roche, CMS medical officer, 2011
Emerging Debates:
Who Will Decide What Results are
Returned?
• Whole genome sequencing will find
“incidental” or secondary findings that are
not the primary reason for testing
- Patients should decide prior to testing?
OR
- Experts should determine a standard set
of results to be returned based on
evidence of clinical utility?
TRADE-OFFS: No Free Lunch
• Would you get sequenced if no cost?
– 81%
• Would you want to know everything?
– 74%
Emerging Debate:
Will We Open Pandora’s Box?
Types of Information
Treatment
Decisions
Outcomes
Treat
Health
Benefits
Clinically actionable
Do Not Treat
WGS
Test
WGS
testing
Decision
Not directly clinically
actionable
Unknown or no clinical
significance
No
WGS
Test
Costs
Tests
Anxiety
Regret
Side
Effects
No additional information
Initial Findings about Sequencing
Reimbursement
• Payers are very interested in sequencing
– Game-changing technology
• Concerns:
–
–
–
–
Complexity
Clinical utility & potential costs
Is sequencing just a bundle of tests
How will be delivered – infrastructure, stored
results, pan-cancer/off-label uses
– Distinguishing research vs. clinical use
Our Ongoing NIH Study:
Benefit-Risk Tradeoffs for Genomic
Sequencing
• What matters to patients and physicians when
they make decisions about sequencing?
• How can payers, guideline organizations, &
society facilitate appropriate & efficient use of
sequencing?
– With Harvard Medical School MedSeq study
• Conducting 1st RCT of whole genome sequencing in
general population