The document summarizes key themes from a webinar on developing medical policies and coverage guidelines for next generation sequencing in oncology. It discusses the challenges of evaluating genomic tests and gaining insurance coverage. Recommendations include requiring laboratories to obtain accreditation for analytic validity, covering small gene panels when clinical utility is established, and facilitating data collection to support coverage of larger tests and off-label drug use. The webinar included perspectives from various stakeholders on addressing these issues.

1. INITIAL MEDICAL POLICY AND
MODEL COVERAGE GUIDELINES
WEBINAR: POLICIES FOR NEXT GEN
SEQUENCING IN ONCOLOGY
Sean Tunis, MD, MSc, President and CEO
Donna A. Messner, PhD, Vice President, Sr. Research Director
November 24, 2015

2. WHO WE ARE
The Center for Medical Technology Policy (CMTP): independent, non-profit
501(c)(3) organization…aims to make health care more effective and affordable
by improving the quality, relevance, and efficiency of health care research
We are committed to engaging all relevant stakeholders…
• in research design, implementation, and dissemination
• in the development of new policy approaches for evaluating and paying for
promising new medical technologies
Green Park Collaborative-USA (GPC-USA) is a major initiative of CMTP:
• a neutral forum to support dialogue and consensus among stakeholders on
methodological standards for clinical research and
• focused on real-world effectiveness and value, emphasizing evidence
expectations of payers, informed by the views of patients and clinicians.

3. KEY THEMES FOR WEBINAR
What
challenge do
we work to
address?
What
conclusions
have we
reached so
far?
Where do
we go from
here?
Panel
perspectives
Group
questions

4. BACKGROUND
What is the challenge we seek
to address?

5. CONCEPTS FOR GENOMIC ASSAY EVALUATION
Clinical utility
Clinical
benefit to
patient when
test is used
for care
Clinical validity
Variant is
significantly
correlated with a
phenotype of
interest
Analytic
validity
Assay
identifies
presence or
absence of a
variant of
interest

6. COVERAGE BOTTLENECK
In past, large proportion of genetic
tests on market have failed to gain
positive coverage decisions
Coverage reviews often never
done due to lack of clinical utility
studies1
Possible reasons:
For some tests, there is no benefit
Lack of clarity over required
studies
Lack of incentives/infrastructure to
conduct necessary studies 1. Hresko A & Haga SB. Insurance
coverage policies for personalized
medicine. J Pers Med 2012;2:201-16.

7. CHALLENGE: SHIFT FROM “TEST” TO “SEQUENCING”
Past and Continuing
Molecular
Pathology Methods
Single
mutation
Single gene
Few genes
Genomic Analysis:
Clinically Used Now
Gene Panels
Whole exome
Potential Future
Uses of Genomics
Whole
Genome
Transcriptome
(RNA)
Adapted from College of American Pathologists, Genomics Resource Guide v. 5.0
Large-scale sequencing
Many
answers
per
question
Many
questions
per test
One test
per
question
One
answer per
test
11

8. NGS PROJECT PHASE 1
Approach and
Recommendations to Date

9. NGS PROJECT OVERVIEW
Kickoff workshop
July 2014
Teleconferences
Multi-stakeholder
deliberation of
key questions
Fall 2014 – Spring
2015
In-Person Conference
April 2015
Wrap-up
teleconferences
May and June
2015
Demonstrating
the Clinical
Utility of Next
Generation
Sequencing in
Oncology,
Meeting
Summary
Initial Medical Policy and
Model Coverage Guidelines
for Clinical Next Generation
Sequencing in Oncology,
Report and
Recommendations

10. RECOMMENDATION: COLLEGE OF AMERICAN
PATHOLOGISTS ACCREDITATION
Payers assess
utility – not analytic
performance
How can they
have confidence in
testing validity,
consistency?
Require covered
laboratories to
participate in new
CAP NGS
accreditation
• Includes wet-bench,
dry-bench proficiency
testing
• CAP/CMS must allow
transparency for
payers
Challenge
Recommendation

11. RECOMMENDATION: COVERAGE OF SMALL PANELS
How to cover
panels that
analyze many
different genes
and variants at
one time?
Cover NGS panels
conforming to 5-to-
50 CPT codes when:
• >= 5 guideline-
directed genes
sequenced for
patient care
• Panel cost <= cost
of sequencing
individual genes by
other methods
Challenge
Recommendation

12. WHAT ABOUT LARGER PANELS?
• No consensus on covering >50 gene panels when smaller
panels can address guideline-directed patient care
– Some say: do comprehensive tumor profiling on 1st
diagnosis; don’t wait until patient has advanced disease
and is heavily pretreated to get full picture
– Payers respond: benefit of doing so is unproven
• GPC report: consider covering >50 panels when patient
has unknown primary site, no standard treatment, or
exhausted treatment options
– Topic for future discussion

13. NGS PROJECT PHASE 2
Looking ahead

14. MORE RAPID LEARNING IMPERATIVE
• Need to investigate clinical
significance of genomic variants
more rapidly
• “Extra” variant data on covered
panels represents potentially rich
source of info for research
• Topics for future discussion:
• How can payers use policy
levers to facilitate high-quality
data collection?
• How can various data collection
efforts be made interoperable?

15. MORE SEQUENCING AND THE EVIDENCE NEEDED
Additional Discussion Points
• What is place of large panels, whole
exome, whole genome sequencing?
• What evidence is needed to support
coverage?
• Will high-quality registries and
observational studies support coverage?

16. RATIONAL COVERAGE OF OFF-LABEL USE
Issue
How to pay for off-label use of
molecularly-targeted therapies when
clinically justified?
Proposal for discussion:
pharma and payers share risk
Pharma provides off-label agent for first
3 months of treatment
(expanded access or other program)
If patient exhibits benefit after 3 months
(stable disease), health plan begins to
cover treatment on month 4.

17. PANELIST PERSPECTIVES
Jeff Allen, PhD
Executive
Director
Friends of
Cancer Research
Dane Dickson,
MD
Chief Executive
Officer
Molecular
Evidence
Development
Consortium
Robert
Dumanois
Manager,
Reimbursement
Strategy
ThermoFisher
Scientific
Michael
Kolodziej, MD
National Medical
Director,
Oncology
Solutions
Aetna
Vincent Miller,
MD
Chief Medical
Officer
Foundation
Medicine

18. FOR MORE INFORMATION
Get involved!
To learn more, contact:
Marty Johnson
GPC Marketing & Project Coordinator
marty.johnson@cmtpnet.org