1.
RETHINK:
Payer Strategies for
Commercializing
Cell & Gene Therapies
Insights from managed care executives on the
future of coverage and outcomes evaluation.

2.
The Potential
What will these new
therapies cost?
How will patients
access them?
Who will pay
for them?
Targeted, personalized and potentially curative … cell and gene therapies present a new frontier for
medical advances. But with that frontier comes a host of new questions, not the least of which are:

3.
For cell and gene therapy innovators, the challenges sound similar to those
faced by all manufacturers: Gain coverage for products to drive access and
ensure a clear path to treatment.
But how do you ensure a path when
one does not yet exist?
What will it take to create new
access models for altogether new
classes of treatments?
The Challenges
2
1

4.
The Survey
Xcenda surveyed 46 executives from managed care organizations, pharmacy benefit managers and similar
organizations to discover how they are evaluating the cell and gene therapy space.
The insights show a market that’s not yet fully aligned around
how it will approach coverage for cell and gene therapy
products—which presents ample opportunities for cell and
gene therapy innovators to shape payers’ thoughts and
decision-making in this burgeoning therapeutic class.

5.
Rethink: Payer Readiness
Findings
When it comes to traditional therapies—and even specialized therapies for rare and orphan products—payers have
established processes for evaluation. Not so with cell and gene therapies, despite the fact that new products are already
beginning to enter the market.
Is a separate process needed to evaluate cell and gene therapies?
say yes, but they have
not yet started on it63%
have completed
the process0%

6.
“First off, start early. Because of the complexity of cell and gene
therapies, the type of committees responsible for assessing the
therapies may vary. It is essential then to engage in discussions
with payers as much as 2-3 years prior to anticipated launch to
glean insights into the possible evaluation pathways, associated
steps and evidence packages required.”
Rethink: Payer Readiness
Xcenda’s Perspective

7.
Rethink: Coverage Barriers
Findings
In something of a chicken-or-egg
conundrum, how do cell and gene
therapy innovators demonstrate
long-term outcomes for therapies that
are just now receiving approval? And
how do the product manufacturers
drive utilization that would generate
the data without coverage that would
enable utilization?
Cost vs. value is cast into stark focus
as payers consider cell and gene
therapy coverage.
When asked what factors would significantly
impede cell and gene therapy coverage…
say long-term outcomes
data may be lacking74%
say total cost
of therapy72%

8.
“We’re talking about transformative therapies that might cost
$1 million or more. So payers are likely to ask for data beyond
the clinical package to increase their comfort level with the
up-front cost burden. Manufacturers need to think about market
research, advisory boards or other collaborations with payers to
gain a clearer understanding of what will be required. Creating
registries to capture utilization and burden of illness with the
existing standard of care may be important to have a baseline for
demonstrating the economic impact—not just the clinical impact.”
Rethink: Coverage Barriers
Xcenda’s Perspective

9.
Rethink: Reimbursement Strategies
Findings
Even as cell and gene therapies are reshaping paradigms for how diseases are treated and the long-term prognoses for
many patients, they’re similarly pushing payers to innovate their thinking on how the products are paid for, when
payments are made and upon what criteria those payments are based.
How are payers thinking outside the box for payment models?
consider outcomes-based agreements
that have financial contingencies tied
to long-term outcomes
70% would consider an annuity-based
model for reimbursement
80%

10.
“Cell and gene therapies will push the boundaries of innovation
and creativity, not just medically, but also financially. Payers will
have to consider the procedural complexity, evaluating the merits
of all payment models—from value-based payments to
fee-for-service models, split payments, as well as combined and
bundled payments.”
Rethink: Reimbursement Strategies
Xcenda’s Perspective

11.
Rethink: Payer Resources
Findings
Long-term outcomes
monitoring is highlighted
as a lynchpin component in
coverage and reimbursement
decisions, yet payers
surveyed are overwhelmingly
under-resourced for carrying
out this crucial function.
What does managed care preparedness
look like for outcomes monitoring?
have all the resources needed
to monitor long-term outcomes7%
say monitoring capabilities are lacking or non-existent,
or they are unsure of their ability to cover93%

12.
“What all this tells us is that payer readiness may lag significantly
behind medical innovation in the cell and gene therapy space.
The companies who will succeed initially in this new frontier are
the ones who work proactively and methodically to shape payer
thinking, evaluation and payment models in this space. In that
sense, these companies will be pioneers in medical innovation
and in how that innovation ultimately reaches the patients who
need it most.”
Rethink: Payer Resources
Xcenda’s Perspective

13.
EBV_Slide_
Access more detailed findings from our survey by viewing our scientific poster here, which
was presented at the 2017 Academy of Managed Care Pharmacy Nexus conference.
Talk to us about how to design a market access strategy that includes an effective clinical
and health economic message, along with critical coding, coverage and reimbursement
considerations that drive awareness and action from the payer community.
Tina Chiang, Director
tina.chiang@xcenda.com
Jay Jackson, Senior Vice President
jay.jackson@xcenda.com
Learn More