cracking the code on gene therapy - amsus
TRANSCRIPT
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Cracking the Code On Gene TherapySteve Miller, MDChief Medical OfficerNov. 27, 2018
AMSUSTHE SOCIETY OF FEDERAL HEALTH PROFESS IONALS
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2© 2018 Express Scripts. All Rights Reserved.
• Presenter(s) has the following interest to disclose:• Full-time employee of Express Scripts• Board of Directors, Medibeacon• Chairman of the Board, SureScripts
ACE/PESG and AMSUS staff have no interest to disclose.
This continuing education activity is managed and accredited by AffinityCE/Professional Education Services Group in cooperation with AMSUS. ACE/PESG, AMSUS, and all accrediting organizations do not support or endorse any product or service mentioned in this activity.
Disclosures
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3© 2018 Express Scripts. All Rights Reserved.
Learning objectives
Participants will understand the developing gene therapy market; which diseases, market size and implications for providers and plan sponsors.
1
Participant will be introduced to innovative approaches to fund treatments for small populations with very expensive, often one-time therapies.
2
Participant will understand the actions they need to take today to be prepared for the future.3
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Our costliest conflict
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Which was more deadly?
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A ‘magic bullet’
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Drug spend and specialty share are growing
63%
37%48%52%2016 2019
Note: Data is actual for 2016, projected for 2019Source: Express Scripts research
Specialty Traditional
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0
5
10
15
20
25
30
35
40
SpecialtyTraditional
Novel FDA approvals continue to increase
Source: U.S. Food & Drug Administration
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New scientific approaches will leadto more novel, but costly medications
529
202
173
188
Pipeline Products Using Gene-based Mechanisms
Cell Therapy (CAR-T)
Conjugated Monoclonal Antibody
DNA & RNA Therapeutics
Gene Therapy
Sources: Analysis Group, PhRMA
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Growth of rare disease FDA approvals (%) Average cost to treat a patient annually
54% increase in the cost of treatment over 4 yearsSource: Express Scripts data
Rare disease therapies are much more costly
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RARE CONDIT ION PREVALENCE
More common than you think
7,000 known rare conditions1
with more identifiedall the time
1. Global Genes: Rare Disease Impact Report2. American Diabetes Association
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Gene augmentation therapy
Cell with non-functioning
gene
Functioning gene
Cell functioning normally
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Gene inhibition therapy
Cell containing faulty gene
Blocking gene
Cell functioning normally
X X
New gene product blocks
faulty gene
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Transfection: The process of inserting genes
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Gene therapy poses unique challenges• 4,000 diseases linked to gene disorders
• High cost: $375k-$1.5M per patient
• Single administration
• Very small patient populations
• Durability periods vary
American healthcare system is ill equipped for this model
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$0
$200,000
$400,000
$600,000
$800,000
$1,000,000
$1,200,000
$1,400,000
$1,600,000Glybera
<10 patients
Luxturna600 patients Strimvelis
<20 patients
Kymriah300 patients
Yescarta7,500 patients
Estim
ated
cos
t of t
hera
py
Fewer patients lead to higher prices
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Critical questionsneed to be addressed
• Clinical Utilization/Eligibility –Who will manage?
• Centers of Excellence –Where will patients go for treatment?
• Follow up –How will patients be monitored?
• Risk management –Who will collect and manage outcomes?
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• Lethal blood and bone-marrow cancer
• Affects children and young adults
Cost: $475,000Treats lymphoblastic leukemia
• Aggressive non-Hodgkin lymphoma
• Indicated after other treatments fail
Cost: $373,000Treats large B-cell lymphoma
First U.S. CAR-T therapies approved for cancers
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• Outcomes-based rebates• Innovative contracting model• Payments over time
LuxturnaTM: Therapy with novel payer programsbrings hope for inherited retinal dystrophies
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Specialized patient care enables risk sharing and improves health outcomes
IN-HOME NURSING SERVICES
ONE-ON-ONE PATIENT SUPPORT
SAFE, APPROPRIATE MEDICATION USE
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New payment models under consideration
Lump Sum
Pooled Risk
Pooled Risk With Performance Guarantees
Periodic Payments
The right solution will enable collaboration among manufacturers, payers, patients and policymakers
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© 2018 Express Scripts. All Rights Reserved.
Learning objectives
Participants will understand the developing gene therapy market; which diseases, market size and implications for providers and plan sponsors.
1
Participant will be introduced to innovative approaches to fund treatments for small populations with very expensive, often one-time therapies.
2
Participant will understand the actions they need to take today to be prepared for the future.3
![Page 23: Cracking the Code On Gene Therapy - AMSUS](https://reader034.vdocuments.site/reader034/viewer/2022051801/6281713ce1203f0ef0742b39/html5/thumbnails/23.jpg)
23© 2018 Express Scripts. All Rights Reserved.
CE/CME Credit
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