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Robert J. Hariri, MD, PhD Founder & Chairman Celgene Cellular Therapeutics Understanding the Value of Medical Innovation

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Page 1: Understanding the Value of Medical Innovationknowledgecenter.csg.org/kc/system/files/Dr. Hariri.pdfVaccines 1930s Pertussis Vaccine 1936 Yellow Fever Vaccine 1935 Sulfa Drugs (first

Robert J. Hariri, MD, PhD Founder & Chairman Celgene Cellular Therapeutics

Understanding the Value of Medical Innovation

Page 2: Understanding the Value of Medical Innovationknowledgecenter.csg.org/kc/system/files/Dr. Hariri.pdfVaccines 1930s Pertussis Vaccine 1936 Yellow Fever Vaccine 1935 Sulfa Drugs (first

Longer Life Through Better Medicines 1900-2010

2

1908 First Large-Scale

Adoption of Water Chlorination

1921 Diphtheria and TB

Vaccines

1930s Pertussis Vaccine

1936 Yellow Fever

Vaccine

1935 Sulfa Drugs

(first antibiotic)

1940s Penicillin First Used

as a Treatment

1955 Polio

Vaccine

1963 Measles Vaccine

1974 Meningococcal

Disease Vaccine

1985 Influenza Vaccine 1995

Hepatitis A Vaccine

1987 Azidothymidine

(first HIV treatment)

Deaths per 100,000 people

48

78

20

475 Life Expectancy

Source: The Hamilton Project at the Brookings Institution. Available at http://www.hamiltonproject.org/multimedia/charts/deaths_from_major_infectious_disease/. Accessed on 11/1/12.

>96% Decrease

>62% Increase

Page 3: Understanding the Value of Medical Innovationknowledgecenter.csg.org/kc/system/files/Dr. Hariri.pdfVaccines 1930s Pertussis Vaccine 1936 Yellow Fever Vaccine 1935 Sulfa Drugs (first

Life Expectancy Growth

49 U.S.

31 WW

68

49

79

67

85yrs

72yrs

New drugs are no small part of this medical miracle. Mark McClellan, FDA “ ”

The Gift of Life

3

3 Source: United Nations Development Program. U.S. Food and Drug Administration. Centers for Disease Control and Prevention and National Center for Health Statistics. Health, United States, 2011: With Special Feature on Socioeconomic Status and Health. Available at http://www.cdc.gov/nchs/index.htm. Accessed 11/12/12.

Page 4: Understanding the Value of Medical Innovationknowledgecenter.csg.org/kc/system/files/Dr. Hariri.pdfVaccines 1930s Pertussis Vaccine 1936 Yellow Fever Vaccine 1935 Sulfa Drugs (first

New Therapies are the Greatest Contributor to Increased Life Expectancy

4

New therapies account for 45% of the increase in life expectancy

New therapies account for 73% of the increase in life expectancy

45% 73%

4

Source: Lichtenberg FR. NBER Working Paper No. 18235. Pharmaceutical innovation and longevity growth in 30 developing and high-income countries, 2000-2009.

Page 5: Understanding the Value of Medical Innovationknowledgecenter.csg.org/kc/system/files/Dr. Hariri.pdfVaccines 1930s Pertussis Vaccine 1936 Yellow Fever Vaccine 1935 Sulfa Drugs (first

Impact of New Therapies on Death Rates

5

Millions of Dollars

100

300

500

700

900

1,100

1,300

1,500 80,000

70,000

60,000

50,000

40,000

30,000

20,000

10,000

0

Deaths per 100,000

R&D in Millions

Mortality Rate (All Causes)

Source: Centers for Disease and Prevention’s National Center for Health Statistics. Health, United States, 2011: With Special Feature on Socioeconomic Status and Health. Available at http://www.cdc.gov/nchs/. Accessed 11/16/12. PhRMA Annual Reports 1960-2010. Available at www.phrma.org. Accessed 11/16/12.

Page 6: Understanding the Value of Medical Innovationknowledgecenter.csg.org/kc/system/files/Dr. Hariri.pdfVaccines 1930s Pertussis Vaccine 1936 Yellow Fever Vaccine 1935 Sulfa Drugs (first

Cancer Innovation Tells the Story

6

Annual Percent

Change in Death Rate

from Cancer

0.0

0.5

1.0

-1.5

-1.0

-0.5

-2.0

U.S. War on

Cancer – 1972

Source: Kort EJ, Paneth N, Vande Woude GF. Cancer Res. 2009;69:6500-6505.

Page 7: Understanding the Value of Medical Innovationknowledgecenter.csg.org/kc/system/files/Dr. Hariri.pdfVaccines 1930s Pertussis Vaccine 1936 Yellow Fever Vaccine 1935 Sulfa Drugs (first

Decline in Death Rates from Leading Causes 1960-2011

7

2011

1960

Deaths Per 100,000

Source: Centers for Disease Control and Prevention and National Center for Health Statistics. National Vital Statistics System. 2011. Available at http://www.cdc.gov/nchs/data/nvsr/nvsr61/nvsr61_06.pdf#page1. Accessed 11/12/12.

Page 8: Understanding the Value of Medical Innovationknowledgecenter.csg.org/kc/system/files/Dr. Hariri.pdfVaccines 1930s Pertussis Vaccine 1936 Yellow Fever Vaccine 1935 Sulfa Drugs (first

Survival Rate For Myeloma Patients Soars With New Innovative Therapies

8

5-year relative survival rate, myeloma

Year of Diagnosis

SurvivalRate %

50% INCREASE 1992-2008

Source: National Cancer Institute, Surveillance Epidemiology and End Results (SEER). Available at http://seer.cancer.gov/csr/1975_2009_pops09/browse_csr.php?section=18&page=sect_18_table09.html. Accessed 10/31/12.

Page 9: Understanding the Value of Medical Innovationknowledgecenter.csg.org/kc/system/files/Dr. Hariri.pdfVaccines 1930s Pertussis Vaccine 1936 Yellow Fever Vaccine 1935 Sulfa Drugs (first

Myeloma Survivorship Rapidly Increasing

9

81,089

46,856 Survivors

Survivors

2000

2009

73% Increase in

Myeloma Survivors

Source: National Cancer Institute, Surveillance Epidemiology and End Results (SEER). Cancer Statistics Review: 1975-2009 Estimates of complete prevalence. Available at http://seer.cancer.gov/csr/1975_2009_pops09/. Accessed 11/16/12.

Page 10: Understanding the Value of Medical Innovationknowledgecenter.csg.org/kc/system/files/Dr. Hariri.pdfVaccines 1930s Pertussis Vaccine 1936 Yellow Fever Vaccine 1935 Sulfa Drugs (first

Five-Year Survival from Myelodysplastic Syndromes (MDS) Increasing

10

+60%

38% 24%

Source: Smout R, Horn S, Goldberg R. Age Period Cohort Analysis of Cancer Survival In SEER 18 Registry. Center for Medicine in the Public Interest.

Page 11: Understanding the Value of Medical Innovationknowledgecenter.csg.org/kc/system/files/Dr. Hariri.pdfVaccines 1930s Pertussis Vaccine 1936 Yellow Fever Vaccine 1935 Sulfa Drugs (first

11

The Virtuous Cycle

Page 12: Understanding the Value of Medical Innovationknowledgecenter.csg.org/kc/system/files/Dr. Hariri.pdfVaccines 1930s Pertussis Vaccine 1936 Yellow Fever Vaccine 1935 Sulfa Drugs (first

Innovation Celgene has a proven track record of delivering better outcomes with better

healthcare through innovation.

Innovation is a Virtuous Cycle. Requiring Commitment. Investment. Vision.

12

Virtuous Cycle of Innovation

Improvements in healthcare are an important source of gains in health, longevity

and productivity globally.

Access Access and reimbursement

for current innovative therapies funds investment in future innovation.

Investment Innovation results from continuous

investment of time and resources by biopharmaceutical companies such

as Celgene.

Commitment The unprecedented survival results reported with Celgene’s innovative therapies are direct

results of the company’s commitment to improving the lives of patients worldwide.

Page 13: Understanding the Value of Medical Innovationknowledgecenter.csg.org/kc/system/files/Dr. Hariri.pdfVaccines 1930s Pertussis Vaccine 1936 Yellow Fever Vaccine 1935 Sulfa Drugs (first

Maximizing the Promise of Science: 5,000+ Medicines in Development in 2011

13

Reflects number of compounds in Phase I, II, III, or having been filed with the FDA, or approved by the FDA, but not yet on the market in the U.S (as of January 2013). Medicines with multiple indications may appear in more than one category but are counted only once for total (3,091).

454 Musculoskeletal Diseases

1,247 Neurological

Disorders

650 Cardiovascular

Disorders 412

Diabetes Mellitus

204 HIV/AIDS

and Related Conditions

69 Liver Disease and Related Conditions

1,586 Infectious Diseases

1,795 Rare

Diseases

731 Immunological

Conditions

95 Breast Cancer

63 Colorectal Cancer

142 Lung Cancer

383 Blood Cancers

105 Skin Cancer

13

Source: PhRMA. The Biopharmaceutical Pipeline: Evolving Science, Hope for Patients. Available at http://phrma.org/sites/default/files/2435/phrmapipelinereportfinal11713.pdf. Accessed 1/18/13.

3,436 Cancer

Page 14: Understanding the Value of Medical Innovationknowledgecenter.csg.org/kc/system/files/Dr. Hariri.pdfVaccines 1930s Pertussis Vaccine 1936 Yellow Fever Vaccine 1935 Sulfa Drugs (first

Living Longer, Better and Healthier Benefits Society

14

$500 Billion In Societal Value

A 1%

REDUCTION

In CANCER- related DEATHS

in the U.S.

Stimulates economy

Improved quality of life

Maximized life expectancy

*Extended survival contributes to economic stimulus by affording people more time to purchase and enjoy leisure activities

Source: Murphy KM and Topel RH. The Value of Health and Longevity. J Political Econ 2006: 114; 5; 871-904.

Page 15: Understanding the Value of Medical Innovationknowledgecenter.csg.org/kc/system/files/Dr. Hariri.pdfVaccines 1930s Pertussis Vaccine 1936 Yellow Fever Vaccine 1935 Sulfa Drugs (first

Greatest Source of Jobs in 21st Century

15

TOTAL JOBS SUPPORTED

4 million total U.S. jobs supported by the biopharmaceutical sector

More than 650,000 jobs in the U.S. biopharmaceutical sector

OPEN

Each direct biopharmaceutical job

supports 5 additional jobs in other sectors

BIOPHARMA JOBS

Source: Battelle Technology Partnership Practice. The U.S. Biopharmaceuticals Sector: Economic Contribution to the Nation. July 2011. Available at http://www.phrma.org/sites/default/files/159/2011_battelle_report_on_economic_impact.pdf. Accessed 11/1/12.

Page 16: Understanding the Value of Medical Innovationknowledgecenter.csg.org/kc/system/files/Dr. Hariri.pdfVaccines 1930s Pertussis Vaccine 1936 Yellow Fever Vaccine 1935 Sulfa Drugs (first

Estimated Value of Additional Life Years Gained From Innovative Cancer Treatments 1988-2010

16

42 Million

Life Years $3.5

Trillion GDP

Source: Estimate derived from the National Bureau of Economic Research. An Economic Evaluation of the War on Cancer. NBER Working Paper No. 15574. Issued in December 2009. Available at http://www.nber.org/papers/w15574. Accessed 11/16/12.

Page 17: Understanding the Value of Medical Innovationknowledgecenter.csg.org/kc/system/files/Dr. Hariri.pdfVaccines 1930s Pertussis Vaccine 1936 Yellow Fever Vaccine 1935 Sulfa Drugs (first

History of Celgene

17

2011 2013 2009 2007 2005 2000 1992 1986

2014 2010 2008 2006 2003 1998 1987 2012

Celgene spins off

as independent

biopharmaceutical

Company from Celanese

Corporation

Celgene licenses Thalomid

(thalidomide)

Celgene acquires Signal

Pharmaceuticals Inc.

FDA approves Revlimid

(lenalidomide) for treatment

of patients with

transfusion-dependent

anemia due to Low- or

Intermediate-1-risk MDS

(associated with deletion

5q cytogenetic abnormality

with or without additional

cytogenetic abnormalities)

EMA (European Medicines

Agency) approves Revilimid,

in combination with

dexamethasone, for treatment

of multiple myeloma

National Institute for Health

and Clinical Excellence

(NICE) recommends

Revlimid in combination

with dexamethasone, for

treatment of patients with

multiple myeloma who

have received two or more

prior therapies (in U.K.

and Wales)

Celgene inducted into

NASDAQ

FDA grants accelerated

approval of Istodax for

patients with peripheral t-cell

lymphoma who have received

at least one prior therapy

FDA approves POMALYST for

multiple myeloma patients

FDA approves REVLIMID for

relapsed or refractory mantle

cell lymphoma

EMA approves REVILIMID for

deletion 5q myelodysplastic

syndromes

Celgene completes

initial public offering

Food and Drug

Administration (FDA)

approves Thalomid for acute

treatment of cutaneous

manifestations of moderate

to severe erythema

nodosum leprosum

Celgene works with FDA to

develop pioneering System

for Thalidomide Education

and Prescribing Safety

(S.T.E.P.S.®) program

Celgene acquires

Anthrogenesis

Corporation (now

Celgene Cellular

Therapeutics)

FDA approves Thalomid

plus dexamethasone for

treatment of multiple

myeloma

FDA approves Revlimid

plus dexamethasone for

the treatment of multiple

myeloma

Celgene beings distribution

of Revlimid through

RevAssist® program

Celgene inducted into

S&P 500

EMA approves Thalomid

Celgene acquires Pharmine

EMA approves Vidaza for

treatment of myelodysplastic

syndromes (MDS) (Pharmion

received FDA approval for

Vidaza in 2004)

Celgene Cellular Threapeutics

announces first ever

human patient treated with

combination of cord blood and

placental-derived stem cells

Celgene acquires Glouster

Pharmaceuticals

Ministry of Health, Labour

and Welfare (MHLW)

approves Revlimid for

the treatment of multiple

myeloma and MDS

associated with a deltetion

5q chromosomal

abnormality in Japan

Celgene acquires Abraxis

Pharmaceuticals

Celgene acquires Avila

Therapeutics

Celgene earns

placement

on Fortune 500 list

FDA approves

Abraxane (paciitaxel

protein-bound) in

combination with the

drug carboplatin to treat

advanced or spreading

non-small cell lung

cancer

EMA approves IMNOVID in

combination with low-dose

dexamethasone for relapsed

and refractory multiple

myeloma

FDA approves ABRAXANE

in combination with

gemcitabine for metastatic

pancreatic cancer

Celgene Then and Now

FDA approves OTEZLA for

Psoriatic Arthritis and

Psoriasis

Page 18: Understanding the Value of Medical Innovationknowledgecenter.csg.org/kc/system/files/Dr. Hariri.pdfVaccines 1930s Pertussis Vaccine 1936 Yellow Fever Vaccine 1935 Sulfa Drugs (first

Sustaining Growth through Innovation

18

• Advanced partnered programs into clinical trials

• Accelerated Ph II trials to critical decision points

• Initiated broad program with novel-novel combinations

• Filed 4 Investigational New Drug (IND) applications

• Initiated new and broadened existing strategic collaborations

Sustaining innovation and long-term growth Major Accomplishments

Page 19: Understanding the Value of Medical Innovationknowledgecenter.csg.org/kc/system/files/Dr. Hariri.pdfVaccines 1930s Pertussis Vaccine 1936 Yellow Fever Vaccine 1935 Sulfa Drugs (first

the industry average

A Commitment to Research and Development Unmatched in the Industry

19

Celgene's commitment to R&D remains strong as we continue to develop innovative therapies

compounds in our library, several of which are

being studied in hundreds of clinical trials

30% More than

reinvestment

into R&D

in the past 2 years

Innovative Discoveries in

Page 20: Understanding the Value of Medical Innovationknowledgecenter.csg.org/kc/system/files/Dr. Hariri.pdfVaccines 1930s Pertussis Vaccine 1936 Yellow Fever Vaccine 1935 Sulfa Drugs (first

Our Compounds Are Carefully Studied in Clinical Trials

20

Even though new treatments can take 12+ years to reach approval status — Celgene remains dedicated to clinical development of innovative therapies

pivotal/phase III programs underway

clinical trials

at major

medical centers

(as of 2014)

Compounds are being studied for • incurable hematological cancers

• Solid tumor cancers

• Serious inflammatory diseases

Continued investment in

Page 21: Understanding the Value of Medical Innovationknowledgecenter.csg.org/kc/system/files/Dr. Hariri.pdfVaccines 1930s Pertussis Vaccine 1936 Yellow Fever Vaccine 1935 Sulfa Drugs (first

Immunology & Inflammation

Solid Tumor Cancers

Hematological Cancers

Leveraging Our Global Infrastructure to Build a Portfolio of Innovative Therapies

21

…while focusing on rare diseases and unmet medical needs

Myelodysplastic syndromes (MDS)

Metastatic breast cancer (MBC)

Squamous cell carcinoma

Large cell carcinoma

Adenocarcinoma

Non-small cell lung cancer (NSCLC):

mPAC (pancreatic cancer)

Multiple myeloma (MM)

Mantle cell lymphoma (MCL)

Cutaneous T-cell lymphoma (CTCL)

Peripheral T-cell lymphoma (PTCL)

Non-Hodgkin lymphoma:

Psoriatic arthritis

Psoriasis

Page 22: Understanding the Value of Medical Innovationknowledgecenter.csg.org/kc/system/files/Dr. Hariri.pdfVaccines 1930s Pertussis Vaccine 1936 Yellow Fever Vaccine 1935 Sulfa Drugs (first

Complementing Our Internal Strengths with Selected External Partnerships

22

Rational Combinations

Cancer Stem Cells/ Resistance

Immuno- therapy

Epigenetics

Next Generation

Biologics

Protein Homeostasis

Apremilast+ Combinations

Novel Targets Fit

for Purpose Fibrosis

Novel IMiDs® / CRBN & Other

Ubiquitin Ligase Targets

New Targets, Epigenetic Priming &

Convergence with Metabolic

Targets

+PDE4 Complementation

+PKCq

GDF Family PKCq,

BTKi, TYK2,

Novel Targets

JNK1, New Targets,

Novel phenotypic

screens

Payload Delivery, Next Gen Enhanced Activities

Novel and Complementary Approaches to

Immuno- therapy,

Breaking Tumor Tolerance

Unique Validation /

Testing Capabilities from

Breaking Tumor

Tolerance

Pathway Convergent

Mechanisms, Synthetic Lethal Combinations

PLATFORMS

Page 23: Understanding the Value of Medical Innovationknowledgecenter.csg.org/kc/system/files/Dr. Hariri.pdfVaccines 1930s Pertussis Vaccine 1936 Yellow Fever Vaccine 1935 Sulfa Drugs (first

23

Net Product Sales ($B)

EPS2 ($)

Notes: 1) CAGR calculated using 2017 midpoint 2) Adjusted EPS reflects 2014 stock split

Accelerating Growth Outlook Through 2017

2013 2015E 2017E 2013 2015E 2017E

$13-$14

$6.4

$8.5-$9.5

21% CAGR1

~$7.50

$2.98

$4.50-$4.75

26% CAGR

We will invest about $2B in R&D in 2015!

Page 24: Understanding the Value of Medical Innovationknowledgecenter.csg.org/kc/system/files/Dr. Hariri.pdfVaccines 1930s Pertussis Vaccine 1936 Yellow Fever Vaccine 1935 Sulfa Drugs (first

Powerful Business Model, Driving Sustainable Growth

24

Leveraging Powerful Model

Strong Growth to 2017

Sustainable Trajectory Beyond 2017

Leveraging model – all metrics

improving

More diversified – lower risk

4 blockbuster products by 2017

Adjusted EPS expected to grow at 26% CAGR

22 programs

>50 indications

100+ clinical trials

2013 2015E 2017E

5-10 years

26% CAGR

Adjusted EPS

Page 25: Understanding the Value of Medical Innovationknowledgecenter.csg.org/kc/system/files/Dr. Hariri.pdfVaccines 1930s Pertussis Vaccine 1936 Yellow Fever Vaccine 1935 Sulfa Drugs (first

2013 2014 2015 2016 2017 2018 2019 2020 2021 2022 2023 2024 2025 2026 2027 2028

25

Current Portfolio

Leverage Internal Pipeline

Building for the Long-Term Building for the Long-Term

Page 26: Understanding the Value of Medical Innovationknowledgecenter.csg.org/kc/system/files/Dr. Hariri.pdfVaccines 1930s Pertussis Vaccine 1936 Yellow Fever Vaccine 1935 Sulfa Drugs (first

Doing Well by Doing Good Our Commitment to Making a Difference Worldwide

26

Hundreds of millions of dollars contributed

annually to third party co-pay foundations to help

patients access medicines

Page 27: Understanding the Value of Medical Innovationknowledgecenter.csg.org/kc/system/files/Dr. Hariri.pdfVaccines 1930s Pertussis Vaccine 1936 Yellow Fever Vaccine 1935 Sulfa Drugs (first

The Promise of Medical Innovation = Fewer Lives Lost

27

160 Million

96 Million

Life Years Lost to Cancer

40% REDUCTION

Source: Soerjomataram I, et al Global burden of cancer in 2008: a systematic analysis of disability-adjusted life-years in 12 world regions. Lancet 2012; 10.1016/S0140-6736(12)60919-2.

Page 28: Understanding the Value of Medical Innovationknowledgecenter.csg.org/kc/system/files/Dr. Hariri.pdfVaccines 1930s Pertussis Vaccine 1936 Yellow Fever Vaccine 1935 Sulfa Drugs (first

Investment in Medical Innovation is the Most Powerful Force for Human Progress

28

If cancer were prevented in 2020, we could save:

$158 Billion a year in medical costs

1.9 Million lives

$2.2 Trillion in value

$158

a year

Save

Source: National Cancer Institute. Cancer costs projected to reach at least $158 billion in 2020. Available at http://www.cancer.gov/newscenter/newsfromnci/2011/CostCancer2020. Estimates from International Agency for Research on Cancer. GLOBOCAN 2008. Available at http://globocan.iarc.fr/. Accessed 11/15/12.

Billion

Page 29: Understanding the Value of Medical Innovationknowledgecenter.csg.org/kc/system/files/Dr. Hariri.pdfVaccines 1930s Pertussis Vaccine 1936 Yellow Fever Vaccine 1935 Sulfa Drugs (first

29 29

We are at Critical Crossroads

Page 30: Understanding the Value of Medical Innovationknowledgecenter.csg.org/kc/system/files/Dr. Hariri.pdfVaccines 1930s Pertussis Vaccine 1936 Yellow Fever Vaccine 1935 Sulfa Drugs (first

Medical Innovation Freeing the World from Disease

30

Pneumonia or influenza

202.2

Tuberculosis

194.4

Gastrointestinal Infections

142.7

Heart Disease 137.4

Cerebrovascular Disease 106.9

Nephropathies 88.6

Cancer 64.0

Senility 50.2

Heart Disease 192.9.2

Cancer 185.9

Noninfectious Airway Diseases 44.6

Cerebrovascular Disease 41.8 Alzheimer’s Disease 27.0 Diabetes 22.3 Nephropathies 16.3 Pneumonia or Influenza 16.2

Nu

mb

er

of

De

ath

s p

er

10

00

,00

0

Diphtheria 40.3

Source: Centers for Disease Control and Prevention and National Center for Health Statistics. National Vital Statistics System. 2011. Available at http://www.cdc.gov/nchs/nvss.htm. Accessed 11/12/12.

Page 31: Understanding the Value of Medical Innovationknowledgecenter.csg.org/kc/system/files/Dr. Hariri.pdfVaccines 1930s Pertussis Vaccine 1936 Yellow Fever Vaccine 1935 Sulfa Drugs (first

The Value of Medical Innovation: HIV

31

90% decline in

people dying from HIV

4 HIV Therapies

Source: Francis DR. New Drugs and HIV Mortality. National Bureau of Economic Research. Based on NBER Working Paper No. 11109. Issued in February 2005. Available at http://www.nber.org/digest/apr05/w11109.html. Accessed 10/31/12.

Page 32: Understanding the Value of Medical Innovationknowledgecenter.csg.org/kc/system/files/Dr. Hariri.pdfVaccines 1930s Pertussis Vaccine 1936 Yellow Fever Vaccine 1935 Sulfa Drugs (first

Medical Innovation Delivered on Its Promise for Patients with HIV

32

3M life

years saved 3 Million

Life Years Saved

$1.3

Trillion Economic

Value

32 Source: Walensky RP, Freedberg KA, Weinstein MC. Cost-Effectiveness of HIV Testing and Treatment in the United States. Clin Infect Dis. 2007; 45, Supplement 4: S248-S254. Available at http://cid.oxfordjournals.org/content/45/Supplement_4/S248.full. Accessed 11/16/12.

Page 33: Understanding the Value of Medical Innovationknowledgecenter.csg.org/kc/system/files/Dr. Hariri.pdfVaccines 1930s Pertussis Vaccine 1936 Yellow Fever Vaccine 1935 Sulfa Drugs (first

Potential Cost of Increased Reimbursement Regulation on Innovation

33

Between 2010-2040:

Decline in R&D for cancer and rare diseases

75 Billion

Fewer life years

80 Million

Lost economic growth potential

4.5 Trillion

33 Source: Vernon J and Goldberg R. Comparative Effectiveness Research: Effect on Pharmaceutical Innovation, Value of Health and Longevity. Center for Medicine in the Public Interest. 2011. Available at http://www.cmpi.org/uploads/File/Comparative-2.pdf. Accessed 11/15/12.

Page 34: Understanding the Value of Medical Innovationknowledgecenter.csg.org/kc/system/files/Dr. Hariri.pdfVaccines 1930s Pertussis Vaccine 1936 Yellow Fever Vaccine 1935 Sulfa Drugs (first

We Are at a Critical Crossroads for Medical Innovation

34

R&D Investment Longer, Better, Healthier Lives

A

MAJOR PARADOX The potential of science is

greater than ever …

But the outlook for

investment has

never been more

uncertain

34

Page 35: Understanding the Value of Medical Innovationknowledgecenter.csg.org/kc/system/files/Dr. Hariri.pdfVaccines 1930s Pertussis Vaccine 1936 Yellow Fever Vaccine 1935 Sulfa Drugs (first

What’s at Stake …

35

Today’s investments in healthcare and R&D can create a world free from cancer for our children and our children’s children.

Gene therapy

Personalized medicine

OFFER HOPE

Immunomodulators

Epigenetics

Nanotechnology

Gene mapping

Cellular therapies