Your CAR T therapy has the green light?
How strong is your market access
strategy?
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VITALSOURCE are trademarks or registered trademarks of Cardinal Health. All other marks are the property of their respective owners.
CAR T-Cell landscape
Recent study of lymphoblastic
leukemia patients
90%
achieved remission
after relapsing or failing to respond
to standard therapies
Analyst forecasts:
Expected costs of
$300,000
per patient for treatment
Will payers be prepared
to fund CAR-T treatment
at these levels?
Source: https://www.lls.org/sites/default/files/National/USA/Pdf/Publications/FS27_CAR%20T-Cell_FS_5_16_FINAL.pdf
http://files.massbio.org/file/yj75EkR.vs0CW5hfj6G.2.pdf
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VITALSOURCE are trademarks or registered trademarks of Cardinal Health. All other marks are the property of their respective owners.
A complex ecosystem
Today’s challenges
Manufacturer
capacity
Risk mitigation
strategies
Accounts
receivable
collection
Financial approval
for therapy
reimbursement
Certification and
database maintenance
Coordination and
psychosocial needs
Biopharma
Providers
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Payers
Patients
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VITALSOURCE are trademarks or registered trademarks of Cardinal Health. All other marks are the property of their respective owners.
Manufacturer planning
Considerations for program design
and regulatory requirements.
Cryopreservation
qualification
Qualified
facilities
REMS
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FDA
regulations
Regulatory
guidelines
Coordination
of facility
validations
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VITALSOURCE are trademarks or registered trademarks of Cardinal Health. All other marks are the property of their respective owners.
Payer approval challenges
Talk to payers!
What evidence is required for a positive reimbursement decision?
The Centers for Medicare &
Medicaid Services (CMS)
Most important considerations to payers
Review of Provenge:
• Safety and efficacy
• Price of treatment
One year before CMS agreed to
provide national coverage with a
temporary code
Most common reasons for denial:
Two years before permanent code
• Considered experimental
• Considered not medically necessary
Source: http://gabi-journal.net/barriers-to-market-uptake-of-biosimilars-in-the-us.html
http://www.bmtinfonet.org/before/financesinsurance
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VITALSOURCE are trademarks or registered trademarks of Cardinal Health. All other marks are the property of their respective owners.
Lifetime leasing (NICE)
If there is a combination of great uncertainty but potentially substantial patient
benefit, innovative payment methodologies are needed to manage/share risk
Example: CAR T-cell therapy specific to antigen CD19 for treating relapsed or
refractory B-ALL in children and young adults:
•
Lifetime leasing method payment viewed with considerable interest
•
Lifetime leasing method: monthly fee is paid for the duration
of treatment benefits (until death) = found to greatly reduce
decision uncertainty
Note: In the context of lifetime leasing considered here, the asset in
question is the health gain delivered rather than the CAR T-cell therapy
itself.
Source: Exploring the assessment and appraisal of regenerative medicines and cell therapy products. Centre for Health Technology Evaluation,
National Institute for Health and Care Excellence (NICE). March 2016.
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VITALSOURCE are trademarks or registered trademarks of Cardinal Health. All other marks are the property of their respective owners.
Challenges to acceptance
• Proof that it works is critical!
• Unanswered question: How long will it last?
• Concerns with cytokine release syndrome
Stem cell therapy
Stem cell therapy
Average cost in 2010
Reimbursement
$512,000
per person
Apheresis: 4-6%
Bone marrow transplant: 4%
Cryopreserve stem cells: 2.5%
Insurance + access ≠ healthcare
Source: http://www.wsj.com/articles/new-costly-cancer-treatments-face-hurdles-getting-to-patients-1412627150
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2259445/
http://californiastemcellreport.blogspot.com/2013/06/cost-of-stem-cell-therapy-estimated.html
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© 2016 Cardinal Health. All Rights Reserved. CARDINAL HEALTH, the Cardinal Health LOGO, ESSENTIAL TO CARE, and
VITALSOURCE are trademarks or registered trademarks of Cardinal Health. All other marks are the property of their respective owners.
Reimbursement
Considerations
• How long will it take to receive payment from
the payer?
• What if prior authorization is required?
• Will the payer even provide coverage?
• Will there be copay assistance?
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VITALSOURCE are trademarks or registered trademarks of Cardinal Health. All other marks are the property of their respective owners.
Process modules
Patient case tracking
Ordering
Apheresis
center
Pre-cryo
storage
Manufacturing
Registry / certification
Coordination / patient services
Transportation
Reimbursement
Reporting / access / notification
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VITALSOURCE are trademarks or registered trademarks of Cardinal Health. All other marks are the property of their respective owners.
Post-cryo
storage
Infusion
Psychosocial needs
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Anxiety
Depression
Financial
problems
Caregiver
coordination
Family
responsibilities
Transportation
School / work
pressure
Spiritual
beliefs
Lack of
information
© 2016 Cardinal Health. All Rights Reserved. CARDINAL HEALTH, the Cardinal Health LOGO, ESSENTIAL TO CARE, and
VITALSOURCE are trademarks or registered trademarks of Cardinal Health. All other marks are the property of their respective owners.
Manufacturer wrap around services
•
•
•
•
•
•
•
•
Psychosocial needs
Travel/transportation
Meals
Caregiver/housing assistance
Education
Foundation support
Patient assistance programs?
Copay
Will you provide services to support these patient needs?
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VITALSOURCE are trademarks or registered trademarks of Cardinal Health. All other marks are the property of their respective owners.
Education and marketing
Patient
First to market:
Caregivers
•
•
Advocacy groups
•
Hospital
Medical staff
Physicians
Payers
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•
Doing all of the educating
Educating on all of the
sciences
Consider different tiers of
education
Coordination/scheduling
Second to market:
•
•
Meeting or exceeding
expectations set by first
Coordination/scheduling
© 2016 Cardinal Health. All Rights Reserved. CARDINAL HEALTH, the Cardinal Health LOGO, ESSENTIAL TO CARE, and
VITALSOURCE are trademarks or registered trademarks of Cardinal Health. All other marks are the property of their respective owners.
Adverse events
Mild to
severe
Cytokine release
syndrome:
Challenge to manage
symptoms without
compromising efficacy
Need
qualified
medical
staff
Occur in
More
treatment
time
60%
of patients
Need
certified
hospitals
Source: http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4119809/
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VITALSOURCE are trademarks or registered trademarks of Cardinal Health. All other marks are the property of their respective owners.
More
expenses
Data and information
What will be your data collection
process?
What will be included in your
enrollment form?
How will you comply with the FDA’s
mandated follow up 15 years later?
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VITALSOURCE are trademarks or registered trademarks of Cardinal Health. All other marks are the property of their respective owners.
Distribution
State licensing
• Can take up to 1 year once commercialized
• Review time and fees vary state-by-state
Accounts receivable
• When/where will this happen?
Will you outsource or build internally?
• If you outsource, how will you qualify your vendors?
Title Model
Third Party Logistics
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VITALSOURCE are trademarks or registered trademarks of Cardinal Health. All other marks are the property of their respective owners.
Issues to mitigate
• Apheresis chair
• Quality of apheresis specimen
• Coordination of specimen to manufacturer – damaged? late?
• Staff availability to do induction
• Certified medical staff
• Cryopreservation storage
• Transportation delays
• Caregiver / patient support (family member crisis, financial duress)
• Patient expired or unstable and unable to accept medication
• Denial of claim
• Medication delivery not logged according to standard operating
procedures
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© 2016 Cardinal Health. All Rights Reserved. CARDINAL HEALTH, the Cardinal Health LOGO, ESSENTIAL TO CARE, and
VITALSOURCE are trademarks or registered trademarks of Cardinal Health. All other marks are the property of their respective owners.
Thank You!
Jan Nielsen
Division President, Sonexus™ Access & Patient Support
jan.nielsen02@sonexushealth.com
© 2016 Cardinal Health. All Rights Reserved. CARDINAL HEALTH, the Cardinal Health LOGO, ESSENTIAL TO CARE, and
VITALSOURCE are trademarks or registered trademarks of Cardinal Health. All other marks are the property of their respective owners.