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Pfizer Rare Disease here to listen, learn and make a difference Michael Duchenne Muscular Dystrophy Amelia Growth Hormone Deficiency Kyle Sickle Cell Disease Ian Growth Hormone Deficiency Kyle Friedreich’s Ataxia Carolyn Acromegaly Curt Hemophilia B Amiya Sickle Cell Disease Gary TTR-FAP Emily Cystic Fibrosis Amy Growth Hormone Deficiency Michael Duchenne Muscular Dystrophy Amelia Growth Hormone Deficiency Kyle Sickle Cell Disease Susan Acromegaly Tiahna Sickle Cell Disease Ian Growth Hormone Deficiency Kyle Friedreich’s Ataxia Cyndi Gaucher Disease Carolyn Acromegaly Matt Friedreich’s Ataxia Curt Hemophilia B Gabe Hemophilia A Amiya Sickle Cell Disease Gary TTR-FAP David TTR-FAP Confidential. Internal Use Only. Amy Growth Hormone Deficiency Michael Duchenne Muscular Dystrophy Amelia Growth Hormone Deficiency Kyle Sickle Cell Disease Susan Acromegaly Tiahna Sickle Cell Disease Ian Growth Hormone Deficiency Kyle Friedreich’s Ataxia Cyndi Gaucher Disease Carolyn Acromegaly Matt Friedreich’s Ataxia Curt Hemophilia B Gabe Hemophilia A Amiya Sickle Cell Disease Gary TTR-FAP David TTR-FAP 1 Sonal Bhatia, MD Vice President North America Rare Disease Medical Affairs Lead February 13, 2018

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Page 1: IanIan Ian Kyle Friedreich’s Ataxia Pfizer Rare Disease ... · Pfizer Rare Disease is committed to transforming the lives of the rare disease community through life-changing innovations,

Pfizer Rare Disease here to listen, learn and make a difference

Michael

Duchenne Muscular Dystrophy

Amelia

Growth Hormone Deficiency Kyle

Sickle Cell Disease

Ian

Growth Hormone Deficiency Kyle

Friedreich’s Ataxia

Carolyn

Acromegaly

Curt

Hemophilia B

Amiya

Sickle Cell Disease

Gary

TTR-FAP

Emily

Cystic Fibrosis

Amy

Growth Hormone Deficiency Michael

Duchenne Muscular Dystrophy

Amelia

Growth Hormone Deficiency Kyle

Sickle Cell Disease

Susan

Acromegaly

Tiahna

Sickle Cell Disease

Ian

Growth Hormone Deficiency Kyle

Friedreich’s Ataxia

Cyndi

Gaucher Disease

Carolyn

Acromegaly

Matt

Friedreich’s Ataxia

Curt

Hemophilia B

Gabe

Hemophilia A Amiya

Sickle Cell Disease

Gary

TTR-FAP

David

TTR-FAP

Confidential. Internal Use Only.

Amy

Growth Hormone Deficiency Michael

Duchenne Muscular Dystrophy

Amelia

Growth Hormone Deficiency Kyle

Sickle Cell Disease

Susan

Acromegaly

Tiahna

Sickle Cell Disease

Ian

Growth Hormone Deficiency Kyle

Friedreich’s Ataxia

Cyndi

Gaucher Disease

Carolyn

Acromegaly

Matt

Friedreich’s Ataxia

Curt

Hemophilia B

Gabe

Hemophilia A Amiya

Sickle Cell Disease

Gary

TTR-FAP

David

TTR-FAP

1

Sonal Bhatia, MD

Vice President

North America Rare Disease Medical Affairs Lead

February 13, 2018

Page 2: IanIan Ian Kyle Friedreich’s Ataxia Pfizer Rare Disease ... · Pfizer Rare Disease is committed to transforming the lives of the rare disease community through life-changing innovations,

• Global footprint & investment

• Around 70 scientists working to find

treatments

• Over 20 compounds in pre-clinical and

clinical development

• Late-stage pipeline opportunities

• Investigational one time gene therapy

treatments

Our Commitment

Michael | DMD Amiya | SCD

Scientists | Pfizer

2

Page 3: IanIan Ian Kyle Friedreich’s Ataxia Pfizer Rare Disease ... · Pfizer Rare Disease is committed to transforming the lives of the rare disease community through life-changing innovations,

3

Pfizer’s Global Rare Disease Portfolio*

Page 4: IanIan Ian Kyle Friedreich’s Ataxia Pfizer Rare Disease ... · Pfizer Rare Disease is committed to transforming the lives of the rare disease community through life-changing innovations,

Approximately 300,000 infants worldwide are born each year with sickle cell disease

1. Piel FB, et al. NEJM. 2017;376:1561-1573. 2. Hassell KL. Am J Prev Med. 2010;38(4S):S512-S521. 3. SicklecellNA.org.2017

Sickle Cell Disease (SCD): The Global Presence

4

~100,000 Americans have SCD and 2% to 10% are Hispanic (~2,500–10,000)

~2 million Americans are carriers of the sick cell trait

Page 5: IanIan Ian Kyle Friedreich’s Ataxia Pfizer Rare Disease ... · Pfizer Rare Disease is committed to transforming the lives of the rare disease community through life-changing innovations,

5

SCD patient in VOC

presents in ED Subject receives supportive

care (IV hydration, opioids)

from ER assessment to

discharge

Pivotal Phase 3 study in patients with SCD experiencing Vaso-Occlusive Crisis (VOC)

• N=350, randomized, double blind, placebo controlled; approximately 70 centers US & Canada

• Study population: 6 years of age; VOC requiring hospitalization

• Primary Endpoint: Readiness for discharge

• Secondary Endpoints: Time to stopping IV opioids; cumulative IV opioid consumption

Open-label extension study

• N=150

• Study population: Subjects who complete the double-blind study and meet other qualification criteria

• Objective: Evaluate the safety of rivipansel when used to treat >1 episode of VOC.

Safety follow-

up 35-day

post last dose

Placebo

Rivipansel DIS

CH

AR

GE

DE

CIS

ION

TO

AD

MIT

1:1

RA

ND

OM

IZA

TIO

N

Open Label

Study

Phase 3 Program

Page 6: IanIan Ian Kyle Friedreich’s Ataxia Pfizer Rare Disease ... · Pfizer Rare Disease is committed to transforming the lives of the rare disease community through life-changing innovations,

Empowers Patients to Self Advocate thereby Elevating the Care Paradigm

rareCourage Social Wall

Integrating vetted disease content into members’ conversations

Curation Working Group Guides curation of credible content to address knowledge gaps

Community Managers Engages patients & advocates to utilize oneSCDvoice by providing tips & timely connections to content

rareCurate Knowledge

Access to vetted disease content

Engagement Working Group Drives awareness & connectivity to patients engaging local advocacy networks

Platform Developed With The Community and For The Community

rareClinical trials

Education for drug development and diversity in clinical trials

6

Harmonizing and Empowering a Fragmented Patient Community

Page 7: IanIan Ian Kyle Friedreich’s Ataxia Pfizer Rare Disease ... · Pfizer Rare Disease is committed to transforming the lives of the rare disease community through life-changing innovations,

7

Partner Organizations

Pfizer collaborates with many groups and organizations both through and beyond

“Council For Change”

Page 8: IanIan Ian Kyle Friedreich’s Ataxia Pfizer Rare Disease ... · Pfizer Rare Disease is committed to transforming the lives of the rare disease community through life-changing innovations,

Pfizer Rare Disease here to listen, learn and make a difference

Michael

Duchenne Muscular Dystrophy

Amelia

Growth Hormone Deficiency Kyle

Sickle Cell Disease

Ian

Growth Hormone Deficiency Kyle

Friedreich’s Ataxia

Carolyn

Acromegaly

Curt

Hemophilia B

Amiya

Sickle Cell Disease

Gary

TTR-FAP

Emily

Cystic Fibrosis

Amy

Growth Hormone Deficiency Michael

Duchenne Muscular Dystrophy

Amelia

Growth Hormone Deficiency Kyle

Sickle Cell Disease

Susan

Acromegaly

Tiahna

Sickle Cell Disease

Ian

Growth Hormone Deficiency Kyle

Friedreich’s Ataxia

Cyndi

Gaucher Disease

Carolyn

Acromegaly

Matt

Friedreich’s Ataxia

Curt

Hemophilia B

Gabe

Hemophilia A Amiya

Sickle Cell Disease

Gary

TTR-FAP

David

TTR-FAP

Confidential. Internal Use Only.

Amy

Growth Hormone Deficiency Michael

Duchenne Muscular Dystrophy

Amelia

Growth Hormone Deficiency Kyle

Sickle Cell Disease

Susan

Acromegaly

Tiahna

Sickle Cell Disease

Ian

Growth Hormone Deficiency Kyle

Friedreich’s Ataxia

Cyndi

Gaucher Disease

Carolyn

Acromegaly

Matt

Friedreich’s Ataxia

Curt

Hemophilia B

Gabe

Hemophilia A Amiya

Sickle Cell Disease

Gary

TTR-FAP

David

TTR-FAP

Pfizer Rare Disease is committed to transforming the lives of the

rare disease community through life-changing innovations,

trusted partnerships, and relentless passion. 8

Page 9: IanIan Ian Kyle Friedreich’s Ataxia Pfizer Rare Disease ... · Pfizer Rare Disease is committed to transforming the lives of the rare disease community through life-changing innovations,

Pfizer Rare Disease Time to seize the moment! We have been here, we are

here and we will be here for

the Rare Disease community.


100,000 Genomes Project Rare Disease Programme · 100,000 Genomes Project Rare Disease Programme Dr Richard Scott Clinical Lead for Rare Disease, Genomics England ... Report QA Test

Rare Disease

Citizen Science and Rare Disease Research

Rare Disease Scotland Progress Report 2016 - 2018 · 2018. 8. 12. · a rare disease. It’s Not Rare to Have a Rare Disease was produced following the publication of the the UK Strategy

Rare Disease Day 2017 · Rare Disease Day 2017 March 30 - 31, 2017 Sheraton Vancouver Wall Centre ... • The Strategy was acknowledged by Parliament on Rare Disease Day 2016 and

Rare Disease Day Conference 2016 · 2016-03-09 · Rare Disease Day Conference 2016 Canada’s Rare Disease Strategy: Building on Success March 9 – 10, 2016 Sheraton Ottawa Hotel

Understanding Rare Disease Registries - Global Genesglobalgenes.org/.../Webinar_Understanding-Rare-Disease-Registries... · Understanding Rare Disease Registries Part 1 July 31, 2013

Case Report Rare Manifestation of a Rare Disease, Acute

WHAT IS A RARE DISEASE? - EURORDIS - The Voice of Rare ... · WHAT IS A RARE DISEASE? CHARACTERISTICS OF RARE DISEASES • Rare diseases are often chronic, progressive,degenerative,

BOOSTINGTHE DEVELOPMENT OF RARE DISEASE THERAPIES …

Rare Disease Caregiving in America · Rare Disease Caregiving in America 1 Acknowledgments The National Alliance for Caregiving (NAC) is proud to present Rare Disease Caregiving in

RARE DISEASE FACTS NORD - Rare Action Network | Home...RARE DISEASE FACTS NORD® RARE Learn more at rarediseases.org INSIGHTS TM There are approximately 7,000 RARE DISEASES 2 MANY

NIH Rare Disease Day Poster

Applied Therapeutics Rare Disease Forum

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Rare Voices Australia Ltd - Microsoft...Rare Voices Australia (RVA) is Australia’s peak body for rare disease, advocating for Australians living with rare disease. We provide a strong,

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Genetic Alliance UK/ Rare Disease UK

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CAMBRIDGE RARE DISEASE SUMMIT...CAMBRIDGE RARE ÉO DISEASE SUMMIT Judge Business School Trumpington St ... 08:55 - 09:10 Welcome from Cambridge Rare Disease Network 09:10 - 09:30 Opening