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


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


Our costliest conflict


Which was more deadly?


A ‘magic bullet’


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


0

5

10

15

20

25

30

35

40

SpecialtyTraditional

Novel FDA approvals continue to increase

Source: U.S. Food & Drug Administration


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


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


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


Gene augmentation therapy

Cell with non-functioning

gene

Functioning gene

Cell functioning normally


Gene inhibition therapy

Cell containing faulty gene

Blocking gene

Cell functioning normally

X X

New gene product blocks

faulty gene


Transfection: The process of inserting genes


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

© 2018 Express Scripts. All Rights Reserved.

$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


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?


• 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


• Outcomes-based rebates• Innovative contracting model• Payments over time

LuxturnaTM: Therapy with novel payer programsbrings hope for inherited retinal dystrophies


Specialized patient care enables risk sharing and improves health outcomes

IN-HOME NURSING SERVICES

ONE-ON-ONE PATIENT SUPPORT

SAFE, APPROPRIATE MEDICATION USE


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


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


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!

http://amsus.cds.pesgce.com/

cracking the code on gene therapy - amsus

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