cracking the code on gene therapy - amsus

Cracking the Code On Gene TherapySteve Miller, MDChief Medical OfficerNov. 27, 2018

AMSUSTHE SOCIETY OF FEDERAL HEALTH PROFESS IONALS

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

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

© 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

23© 2018 Express Scripts. All Rights Reserved.

CE/CME Credit

If you would like to receive continuing education credit for this activity, please visit:http://amsus.cds.pesgce.com

Hurry, CE Certificates will only be available for 30 DAYS after this event!