THE VALUE OF
MEDICAL INNOVATION
IN IMMUNE DISORDERS
2
“
Just as energy is the basis of life itself,
and ideas the source of innovation, so is
innovation the vital spark of all human
change, improvement and progress.
Theodore Levitt
American Economist
”
3
4
TABLE OF CONTENTS
INTRODUCTION
Chapter 1: Virtuous Cycle of Medical
Innovation
Chapter 2: Immune Disorders: Prevalence
and Complexity
Chapter 3: The Burden of Immune
Disorders
Chapter 4: Unmet Need Driving Innovation
Chapter 5: Accelerating Innovation in
Immune Disorders
INTRODUCTION
Medical innovation is one of the greatest sources of
longer life and economic prosperity. In the 21st century,
medical innovation will dramatically improve health
outcomes, reduce the overall cost of healthcare and
stimulate the growth of the global economy, producing a
world where people can lead more healthy and in some
disease areas, potentially disease-free lives. In turn, this
cycle of innovation stimulates investment in biomedical
research to further improve health and create economic
value throughout the world.
In recent years, medical innovation has significantly
improved quality of life for patients living with chronic,
life-long immune disorders. However, more needs to be
done to address unmet needs. This sourcebook will take
a close look at the unmet need in immune disorders,
what is driving innovation and how to ensure the
virtuous cycle of innovation continues.
5
6
1
VIRTUOUS CYCLE
OF MEDICAL INNOVATION
MEDICAL INNOVATION REQUIRES COMMITMENT,
COLLABORATION AND INVESTMENT
Medical innovation has added enormous benefit to individuals and
society. In fact, it can be argued that, among the technological
advances of the past 100 years, medical innovation has contributed
more to our ability to live longer, healthier and more prosperous
lives than any other development.
We will demonstrate that medical innovation brings about a virtuous
cycle of better health, longer life and greater prosperity, which in
turn, stimulates additional investment in innovation for preventing
and treating disease.
1
VIRTUOUS
CYCLE
In many chronic disease areas, medical innovation success means
fewer hospitalizations, lower disability rates and increased wellbeing, among other factors. Medical innovation is increasing in the
area of immune disorders. To accelerate innovation, the
pharmaceutical industry, academia, medical societies, government,
advocacy and others must work together collaboratively to stimulate
investment in research as well as generate greater public awareness
and patient access.
7
8
“
Over the last half century, improvements
in health have been as valuable as all
other sources of economic growth
combined.
”
Kevin Murphy, Ph.D. and Robert Topel, Ph.D.*
University of Chicago Economists
1
VIRTUOUS
*Adapted
from William D. Nordhaus
CYCLE
9
1
10
VIRTUOUS
CYCLE
Innovation Is a Virtuous Cycle Requiring Commitment
Improvements in healthcare are an important source of gains in health, longevity and
productivity
Innovation results from continuous
investment of time and resources
by biopharmaceutical companies
such as Celgene
Access and reimbursement
for current innovative
therapies fund investment
in future innovation
Virtuous
Cycle of
Innovation
Celgene has a
proven track record
of delivering better
healthcare through
innovation
Innovative Medicines Have Greatly Increased Life
Expectancy
>96%
Decrease
Deaths per
100,000 People
500
450
400
350
300
250
200
150
100
50
0
475
78
80
75
70
65
60
55
48
50
20
1900
1920
1940
1921
Diphtheria and TB
Vaccines
1900
1920
1908
First Large-Scale
Adoption of
Water
Chlorination
1960
1940s
Penicillin Used as
a Treatment
1940
1930s
Pertussis Vaccine
1935
Sulfa Drugs
(first antibiotic)
1960
1950s
Methotrexate
1955
Polio Vaccine
1980
1963
Hepatitis A Vaccine
1980
1974
Life
Expectancy
45
40
2000
1995
Measles
Vaccine
>62%
Increase
1998
Infliximab
2000
1985
Meningococcal
Influenza Vaccine
Disease Vaccine 1987
Azidothymidine
(first HIV treatment)
2005
Targeted
Cancer
Treatments
1936
Yellow Fever Vaccine
Source 1: The Hamilton Project at the Brookings Institution. Available at http://www.hamiltonproject.org/multimedia/charts/deaths_from_major_infectious_disease/. Accessed April, 2015.
Source 2: Crow, J. Psoriasis Uncovered. Nature Outlook. 2012; 492(20/27): S50-S51.
Source 3: U.S. Food and Drug Administration (FDA). Infliximab Product Approval Information - Licensing Action. Available at http://www.fda.gov/Drugs/Development
ApprovalProcess/HowDrugsareDevelopedandApproved/ApprovalApplications/TherapeuticBiologicApplications/ucm093327.htm. Accessed April, 2015.
Source 4: CenterWatch. Available at https://www.centerwatch.com/drug-information/fda-approved-drugs/year/2005. Accessed April, 2015.
1
VIRTUOUS
CYCLE
11
1
12
VIRTUOUS
CYCLE
Investment in Healthcare R&D Has Risen While the
Mortality Rate Has Fallen
Millions of
Dollars in R&D
Death per
100,000
1,100
60,000
50,000
40,000
$47,903.10
$39,857.90
$50,709.80
$47,383.10
$42,973.50
$46,441.60
$49,587.60
$51,613.60
$48,645.00
1039.1
938.7
900
800
30,000
10,000
0
869.0
815.0
775.3
774.9 747.0
732.8
791.8
$26,030.80
R&D
Investment*
20,000
Mortality Rate
(All Causes)
1,000
$8,420.30
$1,976.70
700
749.6
741.3
731.9
600
500
*R&D spending in 2000 U.S. dollars
Source 5: National Vital Statistics Reports. Detailed Tables for “Deaths: Final Data for 2013.” Table 1: Number of Deaths, Death Rates, and Age-Adjusted Death Rates, by
Race and Sex: United States, 1940, 1950, 1960, 1970, and 1980-2013. Available at http://www.cdc.gov/nchs/data/nvsr/nvsr64/nvsr64_02.pdf. Accessed June, 2015.
Source 6: Pharmaceutical Research and Manufacturers of America (PhRMA). 2015 Profile: Biopharmaceutical Research Industry. Table 1: Domestic R&D and R&D Abroad,
PhRMA Member Companies: 1980-2014. Available at http://www.phrma.org/sites/default/files/pdf/2014_PhRMA_PROFILE.pdf. Accessed June, 2015.
12
HIV Is a Clear Example of Medical Innovation
Delivering at its Best
3 Million
Life Years Saved
$1.3
Trillion
Economic
Value
Since the late 1980s
Source 7: Walensky, R., Freedberg, K., Weinstein, M., et al. Cost-Effectiveness of HIV Testing and Treatment in the United States. Clinical Infectious Diseases. 2007; 45(4): S248-S254.
Source 8: Philipson, T., Jena, A. Who Benefits from New Medical Technologies? Estimates of Consumer and Producer Surpluses for HIV/AIDS Drugs. National Bureau of Economic Research,
Working Paper No. 11810. December 2005. Available at http://www.nber.org/papers/w11810. Accessed April, 2015.
1
VIRTUOUS
CYCLE
13
1
14
VIRTUOUS
CYCLE
Medical Innovation Reduces Healthcare Spending While
Increasing Patient Health and Survival
For every dollar spent on innovative medicines,
total healthcare spending is reduced by $6.20
Total Healthcare Spending
Innovative Medicines
$1.00
SPENT
$6.20
SAVED
REDUCTIONS
DECREASE
REDUCTIONS
MEDICAL
SPENDING
HOSPITAL
EXPENDITURE
PHYSICIAN
OFFICE-VISIT
EXPENDITURES
Source 9: Lichtenberg, F. Benefits and Costs of Newer Drugs: An Update. National Bureau of Economic Research, Working Paper No. 8996. June 2007. Available at
http://www.nber.org/papers/w8996. Accessed April, 2015.
Drug Development Costs Continue to Climb
The average cost to develop one new approved therapy
more than tripled between the late 1990s and 2014
Drug Development Costs
$800m
$140m
$1.2b
$2.6b
$320m
Source 10: Pharmaceutical Research and Manufacturers of America (PhRMA). 2013 Profile: Biopharmaceutical Research Industry. Available at
http://www.phrma.org/sites/default/files/pdf/PhRMA%20Profile%202013.pdf. Accessed April, 2015.
Source 11: Paul, S., Mytelka, D., Dunwiddie, C., et al. How to Improve R&D Productivity: the Pharmaceutical Industry’s Grand Challenge. Drug Discovery. 2010; 9(3): 203-14.
Source 12: Tufts Center for the Study of Drug Development. Cost to Develop and Win Marketing Approval for a New Drug is $2.6 Billion. Available at
http://csdd.tufts.edu/news/complete_story/pr_tufts_csdd_2014_cost_study. Accessed April, 2015.
1
VIRTUOUS
CYCLE
15
1
16
VIRTUOUS
CYCLE
Society Benefits from New Treatments and,
Ultimately, Lower Costs Forever
Developing a new medicine takes an average of 10–15 years and the Congressional Budget
Office reports that “relatively few drugs survive the clinical trial process.” Innovative therapies
have a limited time in their lifecycle to recapture investment and fund future innovation.
DISCOVERY AND
DEVELOPMENT
APPROXIMATELY
5,000
to
10,000
COMPOUNDS
3-6 YEARS
Preclinical
FDA
REVIEW
GENERIC
1
5
COMPOUNDS
6-7 YEARS
Clinical Trials
INNOVATOR
EXCLUSIVITY
0.5-2 YEARS
FDA Review
10-12 YEARS
FDA Approved Therapy
FOREVER
$2.6 BILLION
Source 12: Tufts Center for the Study of Drug Development. Cost to Develop and Win Marketing Approval for a New Drug is $2.6 Billion. Available at
http://csdd.tufts.edu/news/complete_story/pr_tufts_csdd_2014_cost_study. Accessed April, 2015.
Source 13: Drug Discovery and Development: Understanding the R&D Process. Available at www.innovation.org. Accessed April, 2015.
Source 14: Congressional Budget Office, Research and Development in the Pharmaceutical Industry, 2006. PhRMA. 2013 Profile Biopharmaceutical Research Industry. Available
at http://www.phrma.org/sites/default/files/pdf/PhRMA%20Profile%202013.pdf. Accessed April, 2015.
Results of the Virtuous Cycle of Medical Innovation
Percent Share of Prescriptions
100%
90%
80%
70%
60%
50%
40%
30%
20%
10%
0%
54%
46%
2003
57%
43%
2004
60%
40%
2005
63%
37%
2006
67%
33%
2007
72%
28%
2008
74%
26%
2009
Innovator Brand
78%
22%
2010
80%
20%
2011
84%
86%
87%
16%
14%
13%
2012
2013
2017
Generics
Chart Notes: Includes all prescriptions dispensed through retail pharmacies, including independent and chain drug stores, food store pharmacies and mail order as well as long-term care facilities.
Generics include branded and unbranded generic medicines. Prescription counts are not adjusted for length of therapy. 90-day and 30-day prescriptions are both counted as one prescription.
Source 15: IMS Institute for Health Informatics. Declining Medicine Use and Costs: For Better or Worse? A Review of the Use of Medicines in the United States in 2012. May 2013. Available at
http://static.correofarmaceutico.com/docs/2013/05/20/usareport.pdf. Accessed April, 2014
Source 16: IMS Institute for Health Informatics. Medicine Use and Shifting Costs of Healthcare: A Review of the Use of Medicines in the United States in 2013. April 2014. Available at
http://www.imshealth.com/deployedfiles/imshealth/Global/Content/Corporate/IMS%20Health%20Institute/Reports/Secure/IIHI_US_Use_of_Meds_for_2013.pdf. Accessed April, 2015.
1
VIRTUOUS
CYCLE
17
18
18
2
IMMUNE DISORDERS:
PREVALENCE AND COMPLEXITY
THE NUMBER OF PEOPLE DIAGNOSED WITH
THESE COMPLEX DISORDERS IS INCREASING
There are more than 100 types of immune disorders, a cluster
of conditions in which the immune system attacks its own host’s
body or specific organs.
Immune disorders can manifest at an early age, often go
undiagnosed for many years, are life-long and greatly impact
the lives of patients.
We will examine the prevalence and delay in diagnosis
associated with immune disorders. This sourcebook will focus
on several immune disorders including psoriasis, psoriatic
arthritis, rheumatoid arthritis and Crohn’s disease.
As the prevalence of immune disorders continues to rise,
greater awareness and research is needed to help understand
the complexity of these disorders.
2
PREVALENCE AND
COMPLEXITY
19
20
“
Since cures are not yet available for most
autoimmune diseases, patients face a
lifetime of illness and treatment. They often
endure debilitating symptoms, loss of
organ function, reduced productivity at
work, and high medical expenses.
Elias A. Zerhouni, M.D.
Former Director, National Institutes of Health
”
21
2
PREVALENCE AND
COMPLEXITY
22
Immune Disorders May Be Chronic and Disabling
Immune disorders may be chronic, disabling disorders in which underlying defects
in the immune response lead the body to attack its own organs and tissues
Organ-specific
immune
diseases
are localized to
a single organ
or tissue
Non-organ-specific
diseases are
characterized
by immune
reactions against
many different organs
and tissues resulting
in widespread injury
Source 1: U.S. Department of Health and Human Services. Autoimmune Diseases Coordinating Committee: Autoimmune Disease Research Plan. Available at
http://www.niaid.nih.gov/topics/autoimmune/Documents/adccreport.pdf. Accessed April, 2015.
Source 2: American Autoimmune Related Diseases Association (AARDA). Autoimmune Statistics. Available at http://www.aarda.org/autoimmuneinformation/autoimmune-statistics/. Accessed April, 2015.
22
There Are More Than 100 Immune Disorders
Source 3: American Autoimmune Related Diseases Association (AARDA). The Cost Burden of Autoimmune Disease: The Latest Front in the War on Healthcare Spending. Available at
http://www.diabetesed.net/page/_files/autoimmune-diseases.pdf. Accessed April, 2015.
Source 4: Autoimmune and Autoimmune-Related Diseases. List of Diseases. Available at http://www.aarda.org/autoimmune-information/list-of-diseases/. Accessed April, 2015.
Source 5: Mayo Clinic. Disease and Conditions: Hashimoto’s Disease. Available at http://www.mayoclinic.org/diseases-conditions/hashimotos-disease/basics/definition/con20030293?p=1. Accessed April, 2015.
2
PREVALENCE AND
COMPLEXITY
23
2
24
PREVALENCE AND
COMPLEXITY
Nearly 236 Million People Worldwide Live with at Least One
of These Four Immune Disorders
125
Psoriasis
72
Rheumatoid Arthritis
38
Psoriatic Arthritis
0.9
Crohn's Disease*
0
50
100
150
Millions
*Includes people living in the United States, France, Germany, Italy, Spain, United Kingdom and Japan
Source 6: National Psoriasis Foundation. Media Kit. Available at http://www.psoriasis.org/file/communications---all-documents/MediaKit.pdf. Accessed April, 2015.
Source 7: Gibofsky, A. Overview of Epidemiology, Pathophysiology, and Diagnosis of Rheumatoid Arthritis. The American Journal of Managed Care. 2012; 18: S295-S302.
Source 8: United States Census Bureau. U.S. and World Population Clock. Available at https://www.census.gov/popclock/. Accessed April, 2015.
Source 9: Datamonitor Healthcare. Inflammatory Bowel Disease: Treatment. 2014. Available at https://service.datamonitorhealthcare.com/hkc/disease/immunology-andinflammation/gastroenterology/inflammatory-bowel-disease/treatment/article126968.ece. Accessed April, 2015.
More Than 10 Million People in the U.S. Live with at
Least One of These Four Immune Disorders
7.5
Psoriasis*
1.3
Rheumatoid Arthritis
.75
Psoriatic Arthritis
.55
Crohn's Disease
0
1
2
3
4
Millions
5
6
7
8
*1.9 million people are living with moderate to severe psoriasis
Source 6: National Psoriasis Foundation. Media Kit. Available at http://www.psoriasis.org/file/communications---all-documents/MediaKit.pdf. Accessed April, 2015.
Source 9: Datamonitor Healthcare. Inflammatory Bowel Disease: Treatment. 2014. Available at https://service.datamonitorhealthcare.com/hkc/disease/immunology-andinflammation/gastroenterology/inflammatory-bowel-disease/treatment/article126968.ece. Accessed April, 2015.
Source 10: American College of Rheumatology. Prevalence Statistics. Available at http://www.rheumatology.org/ACR/about/newsroom/prevalence/prevalence-two.pdf. Accessed April, 2015.
Source 11: Decision Resources. Psoriatic Arthritis: Pharmacor Edition. 2010. Data on file.
Source 12: National Psoriasis Foundation. Statistics. Available at http://www.psoriasis.org/research/science-of-psoriasis/statistics. Accessed April, 2015.
2
PREVALENCE AND
COMPLEXITY
25
2
PREVALENCE AND
COMPLEXITY
Symptom Onset of Immune Disorders May Occur Early in
Life and Are Often Life-Long
*According to the American Academy of Dermatology, psoriasis can happen at any age. Most people get psoriasis between 15 and 30 years of age. About 75% of
people who will get psoriasis will have it by age 40. Another time when symptoms appear is between 50-60 years of age.
Source 6: National Psoriasis Foundation. Media Kit. Available at http://www.psoriasis.org/file/communications---all-documents/MediaKit.pdf. Accessed April, 2015.
Source 13: American Academy of Dermatology. Psoriasis: Who Gets and Causes. Available at http://www.aad.org/dermatology-a-to-z/diseases-and-treatments/m---p/psoriasis/who-getscauses. Accessed April, 2015.
Source 14: Arthritis Foundation. What is Rheumatoid Arthritis? Available at http://www.arthritis.org/about-arthritis/types/rheumatoid-arthritis/what-is-rheumatoid-arthritis.php. Accessed June,
2015.
Source 15: National Institute of Diabetes and Digestive and Kidney Diseases. What I Need to Know About Crohn's Disease. Available at http://www.niddk.nih.gov/health-information/
health-topics/digestive-diseases/crohns-disease/Pages/ez.aspx. Accessed April, 2015.
26
Significant Delay in Diagnoses for Immune Disorders
AVERAGE DELAY (YEARS) IN DIAGNOSIS AFTER SYMPTOM ONSET
Source 16: Lebwohl, M., Bachelez, H., Barker, J., et al. Patient Perspectives in the Management of Psoriasis: Results from the Population-Based Multinational Assessment of Psoriasis and Psoriatic
Arthritis. Journal of the American Academy of Dermatology. 2014; 70(5): 871-881.e30.
Source 17: Chan, K., Felson, D., Yood, R., et al. The Lag Time Between Onset of Symptoms and Diagnosis of Rheumatoid Arthritis. Arthritis & Rheumatology. 1994; 37(6): 814-20.
Source 18: Schoepfer, A., Dehlavi, M., Fournierr, N., et al. Diagnostic Delay in Crohn's Disease is Associated With a Complicated Disease Course and Increased Operation Rate. The American
Journal of Gastroenterology. 2013; 108(11): 1744-53.
2
PREVALENCE AND
COMPLEXITY
27
28
3
THE BURDEN OF IMMUNE
DISORDERS
IMMUNE DISORDERS ARE LIFE-ALTERING AND
ASSOCIATED WITH SIGNIFICANT
COMORBIDITIES
The impact of immune disorders is significant but greatly
underappreciated. We will examine the physical and emotional
burden associated with immune disorders, and how emerging
research is linking these disorders with comorbidities such as
cardiovascular disease, diabetes, cancer and infection,
sometimes manifesting as a combination of these comorbidities.
Immune disorders can lead to increased work-related disability
rates, loss of productivity in the workplace and unemployment.
These diseases place a substantial economic toll on patients,
employers, the healthcare system and the economy.
3
BURDEN OF
IMMUNE DISORDERS
29
30
“
Although the systemic nature of psoriasis
often remains unrecognized, the inflammatory
processes involved may be associated with
the development of comorbidities, which
themselves, have a significant impact on the
patient's health and quality of life.
Kristian Reich, MD
Dermatologikum Hamburg, Germany
”
31
3
BURDEN OF
IMMUNE DISORDERS
Psoriasis, Psoriatic Arthritis, Rheumatoid Arthritis and
Crohn’s Disease Impose Heavy Burdens
32
Psoriasis Comes in Several Distinct Forms
Patients usually display a single form at any one time, although forms can
coexist, and one form can be followed by another
Numbers represent the percentage of patients with these types of psoriasis
Source 1: Crow, J. Psoriasis Uncovered. Nature Outlook. 2012; 492(20/27): S50-S51.
3
BURDEN OF
IMMUNE DISORDERS
33
3
34
BURDEN OF
IMMUNE DISORDERS
People with Both Psoriasis and Psoriatic Arthritis
Experience a Variety of Discomforting Symptoms
50
45
Percent of Patients
40
35
43
40
30
25
Psoriasis
23
20
20
15
16
Psoriatic Arthritis
19
16
10
15
10
5
9
5
0
Itching
Scales
Flaking
Redness
Pain
4
Burning
3
3
Bleeding
Source 2: Lebwohl, M., Bachelez, H., Barker, J., et al. Patient Perspectives in the Management of Psoriasis: Results from the Population-Based Multinational Assessment of
Psoriasis and Psoriatic Arthritis. Journal of the American Academy of Dermatology. 2014; 70(5): 871-881.e30.
34
Patients with Psoriatic Arthritis Experience
Manifestations of the Disease that Are Difficult
to Treat
Source 3: Husted, J., Tom, B., Farewell, V., et al. A Longitudinal Study of the Effect of Disease Activity and Clinical Damage on Physical Function over the Course of Psoriatic Arthritis. Arthritis &
Rheumatology. 2007; 56(3): 840‐849.
Source 4: Kane, D., Stafford, L., Bresnihan, B., et al. A Prospective, Clinical and Radiological Study of Early Psoriatic Arthritis: an Early Synovitis Clinic Experience. Rheumatology. 2003; 42: 1460‐1468.
Source 5: Brockbank, J., Stein, M., Schentag, C., et al. Dactylitis in Psoriatic Arthritis: A Marker for Disease Severity? Annals of the Rheumatic Diseases. 2005; 64: 188‐190.
3
BURDEN OF
IMMUNE DISORDERS
35
3
BURDEN OF
IMMUNE DISORDERS
36
People with Psoriasis Can Go On to Develop
Psoriatic Arthritis
36
Source 6: National Psoriasis Foundation. Media Kit. Available at http://www.psoriasis.org/file/communications---all-documents/MediaKit.pdf. Accessed April, 2015.
People with Rheumatoid Arthritis Report
Experiencing a Range of Challenges
*Patients reported that they sometimes have these symptoms
Source 7: Rheumatoid Patient Foundation. Unmasking Rheumatoid Disease: The Patient Experience of Rheumatoid Arthritis. Available at http://rheum4us.org/wpcontent/uploads/2013/04/Unmasking-Rheumatoid-Disease-The-Patient-Experience-of-Rheumatoid-Arthritis-White-Paper.pdf. Accessed April, 2015.
3
BURDEN OF
IMMUNE DISORDERS
37
3
BURDEN OF
IMMUNE DISORDERS
Patients with Crohn’s Disease May Experience
Extreme Symptoms During a Flare-Up
Source 8: Crohn’s and Colitis UK. Taking the IBD Standards Forward in Scotland. Available at
http://www.crohnsandcolitis.org.uk/Resources/CrohnsAndColitisUK/Research/survey-report-draft-final.pdf. Accessed April, 2015.
38
People with Immune Disorders Are at a
Greater Risk of Cardiovascular Events
80
70
70
60
Percent Increased
Risk Compared
to General Population
50
30 30
29
20
19
10
*Includes patients with Crohn's
disease and ulcerative colitis
Heart Attack/Ischemic
Heart Disease
39
30
0
50 50
50
40
Cardiovascular
Mortality
60
56
0
12
18
0
Mild Psoriasis Severe Psoriasis Psoriatic Arthritis
Rheumatoid
Arthritis
Stroke/Cerebrovascular
Disease
Inflammatory
Bowel Disease*
Source 9: Han, C., Robinson, D., Hackett, M., et al. Cardiovascular Disease and Risk Factors in Patients with Rheumatoid Arthritis, Psoriatic Arthritis and Ankylosing Spondylitis. The Journal of
Rheumatology. 2006; 33(11): 2167-72.
Source 10: Armstrong, E., Harskamp, C., Armstrong, A. Psoriasis and Major Adverse Cardiovascular Events: A Systematic Review and Meta-Analysis of Observational Studies. Journal of the American
Heart Association. 2013; 2: e000062.
Source 11: Aviña-Zubieta, J., Choi, H., Sadatsafavi, M., et al. Risk of Cardiovascular Mortality in Patients with Rheumatoid Arthritis: a Meta-Analysis of Observational Studies. Arthritis &
39
Rheumatology. 2008; 59(12): 1690-7.
Source 12: Singh, S., Singh, H., Loftus, E., et al. Risk of Cerebrovascular Accidents and Ischemic Heart Disease in Patients with Inflammatory Bowel Disease: a Systematic Review and Meta-Analysis.
Clinical Gastroenterology and Hepatology. 2014; 2(3): 382-93.
Source 13: Bewtra, M., Kaiser, L., TenHave., T., et al. Crohn’s Disease and Ulcerative Colitis are Associated with Elevated Standardized Mortality Ratios: A Meta-Analysis. Inflammatory Bowel
Diseases. 2013; 19(3): 599-613.
Source 14: Jamnitski, A., Symmons, D., Peters, M., et al. Cardiovascular Comorbidities in Patients with Psoriatic Arthritis: a Systematic Review. Annals of the Rheumatic Diseases. 2013; 72: 211216.
3
BURDEN OF
IMMUNE DISORDERS
39
3
BURDEN OF
IMMUNE DISORDERS
Significant Comorbidities* Are Associated with
Immune Disorders
*Not an exhaustive list
Source 15: The Arthritis Foundation. The Heavy Burden of Arthritis in the U.S. Available at http://www.arthritis.org/media/newsroom/Arthritis_Prevalence_Fact_Sheet_5-3111.pdf. Accessed April, 2015.
Source 16: Centers for Disease Control and Prevention. Comorbidities. Available at http://www.cdc.gov/arthritis/data_statistics/comorbidities.htm. Accessed April, 2015.
Source 17: Centers for Disease Control and Prevention. Rheumatoid Arthritis. Available at http://www.cdc.gov/arthritis/basics/rheumatoid.htm. Accessed April, 2015.
Source 18: Husted, J., Thavaneswaran, A., Chandran, V., et al. Incremental Effects of Comorbidity on Quality of Life in Patients with Psoriatic Arthritis. The Journal of
Rheumatology. 2013; 40(8): 1349-1356.
Source 19: Aurangabadkar, S. Comorbidities in Psoriasis. Indian Journal of Dermatology, Venereology and Leprology. 2013; 79(7): 10-17.
Source 20: Cucino, C., Sonnenberg, A., The Comorbid Occurrence of Other Diagnoses in Patients with Ulcerative Colitis and Crohn's Disease. The American Journal of
Gastroenterology. 2001; 96(7): 2107-12.
40
People with Immune Disorders Are More
Likely to Die Early
*For people with disease onset in the early 1990s
Source 21: Lassere, M., Rappo, J., Portek, I., et al. How Many Life Years are Lost in Patients with Rheumatoid Arthritis? Secular Cause‐Specific and All-Cause Mortality in Rheumatoid Arthritis, and
Their Predictors in a Long‐Term Australian Cohort Study. Journal of Internal Medicine. 2013; 43(1): 66‐72.
Source 22: Gelfand, J., Troxel, A., Lewis, J., et al. The Risk of Mortality in Patients with Psoriasis: Results From a Population‐Based Study. Archives of Dermatology. 2007; 143(12): 1493‐1499.
Source 23: Ali, Y., Tom, B., Schentag, C., et al. Improved Survival in Psoriatic Arthritis with Calendar Time. Arthritis & Rheumatology. 2007; 56(8): 2708‐2714.
3
BURDEN OF
IMMUNE DISORDERS
41
“
“
“
42
I tend to put off going to the hairdresser. Some of the
girls who wash hair wear gloves when I come in;
people think it’s contagious.
– Psoriasis Patient
”
Crohn's disease has affected every aspect of my body and life.
Beyond the gastrointestinal pain, it has wreaked havoc on my skin,
eyes, hair and joints and destroyed my energy level. It has been a
driving force behind many family and career decisions. Crohn's has
impacted my life in ways many people could never understand.
– Crohn’s Disease Patient
”
It was hard for me to open a marker to write on a white
board, hard to answer the phone or type, because I
couldn't make a fist, I couldn't grip anything.
– Psoriatic Arthritis Patient
Source 24: Anonymous Psoriasis, Crohn’s Disease and Psoriatic Arthritis Patients
”
Psoriasis Significantly Impacts Emotional
Well-Being
Percent of people* with psoriasis who reported:
Emotional Components
Self-consciousness
89 %
Anger or Frustration
89 %
Helplessness
87 %
Embarrassment
87 %
Concealed Physical
Manifestation of their Disease
83 %
0
20
40
60
80
100
Percent of Patients
*Patients may or may not have psoriatic arthritis
Source 25: Armstrong, A., Schupp, C., Wu, J., et al. Quality of Life and Work Productivity Impairment Among Psoriasis Patients: Findings from the National Psoriasis
Foundation Survey Data 2003‐2011. PLoS One. 2012; 7(12): e52935.
3
BURDEN OF
IMMUNE DISORDERS
43
3
BURDEN OF
IMMUNE DISORDERS
44
Global Survey: People with Psoriasis Often Shunned
by Others
Source 26: BBC News. People with Psoriasis ‘Shunned'. Updated October 27, 2004. Available at http://news.bbc.co.uk/2/hi/health/3958617.stm. Accessed April, 2015. Survey
was conducted by Taylor Nelson Sofres, a Global Market Research Company.
Psoriatic Arthritis Patients Have Problems
with Daily Activities
Healthy Controls
Mean SF-36 Score
(Relative Health Status)
100
Psoriatic Arthritis
90
80
70
60
50
40
30
20
10
0
Social
Functioning
Role Limitations Emotional Problems
Mental
Health
Physical
Functioning
Psoriatic arthritis patients have problems with daily activities due to emotional and physical
problems and have difficulty performing normal social activities
Source 27: Picchianti-Diamanti, A., Rosado, M., Scarsella, M., et al. Health-Related Quality of Life and Disability in Patients with Rheumatoid, Early Rheumatoid and
Early Psoriatic Arthritis Treated with Etanercept. Quality of Life Research. 2010; 19(6): 821-826.
3
BURDEN OF
IMMUNE DISORDERS
45
3
BURDEN OF
IMMUNE DISORDERS
Crohn’s Disease and Ulcerative Colitis
Substantially Impact Overall Well-Being
Source 28: European Federation of Crohn’s and Ulcerative Colitis Associations (EFCCA). The True Impact of IBD: A European Crohn’s and Ulcerative Colitis Patient Life: IMPACT
Survey 2010-2011. Available at http://efcca-solutions.net/media/jointhefight/ImpactReport.pdf. Accessed April, 2015.
46
Immune Disorders Have a Significant Impact
on Work-Related Disability
Source 29: Verstappen, S., Watson, K., Lunt, M., et al. Working Status in Patients with Rheumatoid Arthritis, Ankylosing Spondylitis and Psoriatic Arthritis: Results from the British Society for
Rheumatology Biologics Register. Rheumatology. 2010; 49(8): 1570‐1577.
Source 30: Tillett, W., de‐Vries, C., McHugh, N. Work Disability in Psoriatic Arthritis: A Systematic Review. Rheumatology. 2012; 51(2): 275‐283.
3
BURDEN OF
IMMUNE DISORDERS
47
3
BURDEN OF
IMMUNE DISORDERS
The Impact of Crohn’s Disease on WorkRelated Disability
Source 31: Lichtiger, S., Binion, D., Wolf, D., et al. The CHOICE Trial: Adalimumab Demonstrates Safety, Fistula Healing, Improved Quality of Life and Increased Work Productivity
in Patients with Crohn's Disease who Failed Prior Infliximab Therapy. Alimentary Pharmacology & Therapeutics. 2010; 32(10): 1228-1239.
48
Significant Work Days Are Lost by Patients
with Psoriasis and Psoriatic Arthritis
Source 25: Armstrong, A., Schupp, C., Wu, J., et al. Quality of Life and Work Productivity Impairment Among Psoriasis Patients: Findings from the National Psoriasis Foundation Survey
Data 2003‐2011. PLoS One. 2012; 7(12): e52935.
3
BURDEN OF
IMMUNE DISORDERS
49
3
BURDEN OF
IMMUNE DISORDERS
Significant Work Days Are Lost by Inflammatory
Bowel Disease* and Rheumatoid Arthritis Patients
*Includes patients with Crohn's disease and ulcerative colitis
Source 28: European Federation of Crohn’s and Ulcerative Colitis Associations (EFCCA). The True Impact of IBD: A European Crohn’s and Ulcerative Colitis Patient Life: IMPACT Survey
2010-2011. Available at http://efcca-solutions.net/media/jointhefight/ImpactReport.pdf. Accessed April, 2015.
Source 32: Moral, R., Rillo, O., Casalla, L., et al. Work Productivity in Rheumatoid Arthritis: Relationship with Clinical and Radiological Features. Arthritis. 2012; Article ID 137635.
50
Psoriatic Arthritis Decreases Work Productivity
Source 33: Kennedy, M., Papneja A., Thavaneswaran A., et al. Prevalence and Predictors of Reduced Work Productivity in Patients with Psoriatic Arthritis. Clinical and
Experimental Rheumatology. 2014; 32(3): 342-8.
3
BURDEN OF
IMMUNE DISORDERS
51
3
BURDEN OF
IMMUNE DISORDERS
52
The Impact of Psoriasis on Unemployment
*Patients may or may not have psoriatic arthritis
Source 25: Armstrong, A., Schupp, C., Wu, J., et al. Quality of Life and Work Productivity Impairment Among Psoriasis Patients: Findings from the National Psoriasis
Foundation Survey Data 2003‐2011. PLoS One. 2012; 7(12): e52935.
52
Estimated Economic Burden of These Four
Immune Disorders Is Substantial in the U.S.
*Medical and productivity costs of psoriatic arthritis, psoriasis, rheumatoid arthritis and Crohn’s disease combined
Source 34: American Autoimmune Related Diseases Association (AARDA). The Cost Burden of Autoimmune Disease: The Latest Front in the War on Healthcare Spending. Available at
http://www.diabetesed.net/page/_files/autoimmune-diseases.pdf. Accessed April, 2015.
Source 35: Cortesi, P., Scalone, L., D’Angiolella, L., et al. Systematic Literature Review on Economic Implications and Pharmacoeconomic Issues of Psoriatic Arthritis. Clinical and Experimental
Rheumatology. 2012; 30(73): S126-S131.
Source 36: United States Census Bureau. U.S. and World Population Clock. Available at https://www.census.gov/popclock/. Accessed April, 2015.
3
BURDEN OF
IMMUNE DISORDERS
53
54
“
Public health efforts exist for other chronic
diseases like heart disease and diabetes, yet
psoriasis and psoriatic arthritis have not had
similar attention, despite their prevalence and
profound burden on health care costs,
quality of life and employment.
Randy Beranek
”
President and CEO, National Psoriasis Foundation
55
56
4
UNMET NEED DRIVING
INNOVATION
CONTINUED MEDICAL INNOVATION CAN
HELP PATIENTS LIVE FULLER LIVES
This section will demonstrate that while progress has been
made in treating immune disorders, particularly in the past
20 years, we are continuing to make advances to help
patients live fuller, healthier and more productive lives.
Not everyone is benefitting from existing therapies, while
others can’t continue on these therapies for extended
periods of time. Although medical innovation has enhanced
patient well-being, the need exists to improve the health
and productivity of millions who are diagnosed with
immune disorders. Fortunately, there are a number of
molecules being explored and research on new therapies
for immune disorders is increasing.
4
UNMET NEED
DRIVING INNOVATION
57
58
“
At the time I entered this field, there was a lot to
be learned about the basics of how the immune
system worked. Now a lot of the large questions
have been answered. The most important
challenge today is how to best use that
knowledge to benefit mankind.
Howard Grey, M.D.
”
Professor, La Jolla Institute for Allergy and Immunology
59
4
60
UNMET NEED
DRIVING INNOVATION
Treatments for Immune Disorders* Have Accelerated Over
the Past Decade
TREATMENTS
Topical
Systemic small molecule
(DMARDs**)
Phototherapy
1950s
>100
YEARS AGO
METHOTREXATE
100 YEARS AGO
1970s
2010 and
Beyond
1996
ACITRETIN
UVB LIGHT
IMMUNE MEDIATORS,
e.g. PDE4 and
JAK inhibitors, IL-17
PSORALEN AND UVA
LIGHT (PUVA)
CYCLOSPORINE
COAL TAR
1920
1925
GOECKERMAN
THERAPY
1940
1960
1951
CORTICOSTEROIDS
*Psoriasis, psoriatic arthritis, rheumatoid arthritis and Crohn’s disease
**Disease-modifying antirheumatic drugs
Immune
mediators
Systemic biologic
1980
2000
1993
VITAMIN D3
ANALOGUES
2020
2002
ADALIMUMAB
1998
2009
USTEKINUMAB
INFLIXIMAB
ETANERCEPT
Source 1: Crow, J. Psoriasis Uncovered. Nature Outlook. 2012; 492(20/27): S50-S51.
Source 2: ReportsnReports. Therapeutic Class Overview: Psoriasis – Plaque Psoriasis & Psoriatic Arthritis: Novel Oral Drugs and Biologics to Change Future Treatment Paradigm. Available at http://www.
reportsnreports.com/reports/275192-therapeutic-class-overview-psoriasis-plaque-psoriasis-psoriatic-arthritis-novel-oral-drugs-and-biologics-to-change-future-treatment-paradigm.html. Accessed April, 2015.
Source 3: Zuckerman, A. Cyclosporine: A Review. Journal of Transplantation. 2012. doi:10.1155/2012/230386.
Source 4: The American College of Rheumatology. Infliximab Approved for Use in Rheumatoid Arthritis. Available at https://www.rheumatology.org/publications.hotline/0200infliximab.asp. Accessed April, 2015.
Source 5: FDA Arthritis Advisory Committee. ENBREL. Available at http://www.fda.gov/ohrms/dockets/ac/01/briefing/3779b2_02_immunex.pdf. Accessed April, 2015.
Source 6: CenterWatch. Humira New FDA Drug Approval. Available at https://www.centerwatch.com/drug-information/fda-approved-drugs/drug/814/humira-adalimumab. Accessed April, 2015.
Rheumatoid Arthritis: The Evolution of Clinical Value
for Patients
“The clinical improvements produced by novel treatment options for
rheumatoid arthritis have been far above what could have been anticipated
or achieved at the time of initial introduction of the first treatment options.”
Biologic
DMARDs**
Future
New Approvals
Synthetic
DMARDs**
NSAIDs*/
Corticosteroids
1980s
1990s
Use in other indications
2000s
Earlier use in disease progression
2010 and Beyond
Use in combination with other agents
*Nonsteroidal anti-inflammatory drugs
**Disease-modifying antirheumatic drugs
Source 7: Augustyn, C., Walker, B., Goss, T. Recognizing the Value of Innovation in the Treatment of Rheumatoid Arthritis: White Paper. Boston Healthcare. March 2013.
4
UNMET NEED
DRIVING INNOVATION
61
4
UNMET NEED
DRIVING INNOVATION
Introduction of Biologics in Rheumatoid Arthritis Have
Impacted Productivity
Source 8: Chaudhari, P. The Impact of Rheumatoid Arthritis And Biologics on Employers and Payers. Biotechnology Healthcare. 2008; 5(2): 37-44.
62
A Large Percentage of Patients Are Not Receiving
Treatment or Only Using Topical Therapy
100%
Current Treatments
Biologic ± topical
14%
8%
Oral medications* + biologic
Oral medications* ± topical
Topical only
% Patients
19%
31%
No treatment
59%
of psoriatic arthritis patients +
psoriasis patients are receiving no
therapy or topical therapy only
28%
0%
(n=712)
*E.g., cyclosporine, methotrexate, acitretin or fumaric acid esters
Source 9: Lebwohl, M., Bachelez, H., Barker, J., et al. Patient Perspectives in the Management of Psoriasis: Results from the Population-Based Multinational Assessment of
Psoriasis and Psoriatic Arthritis. Journal of the American Academy of Dermatology. 2014; 70(5): 871-881.e30.
4
UNMET NEED
DRIVING INNOVATION
63
4
UNMET NEED
DRIVING INNOVATION
Safety/Tolerability Issues and Lack or Loss of Efficacy
Are the Top Reasons Patients Discontinue Medicine
Source 9: Lebwohl, M., Bachelez, H., Barker, J., et al. Patient Perspectives in the Management of Psoriasis: Results from the Population-Based Multinational Assessment of
Psoriasis and Psoriatic Arthritis. Journal of the American Academy of Dermatology. 2014; 70(5): 871-881.e30.
64
Many Patients Are Not Fully Benefiting from Existing
Therapies
Note: Percentages do not add up to 100% as patients could name more than one reason for discontinuing therapy
Source 10: Strand, V., Williams, S., Miller, P., et al. Discontinuation of Biologic Therapy in Rheumatoid Arthritis (RA): Analysis From the Consortium of Rheumatology Researchers of
North America (Corrona) Database. Annals of the Rheumatic Diseases. 2013; 72(3): 71.
Source 11: Krupa, L., Kennedy, H., Jamieson, C., et al. The Reasons for Discontinuation of Infliximab Treatment in Patients with Crohn's Disease: A Review of Practice at NHS
Teaching Hospital. ISRN Gastroenterology. Article ID 672017.
4
UNMET NEED
DRIVING INNOVATION
65
4
66
UNMET NEED
DRIVING INNOVATION
Patients Surveyed Identified Concerns with Available
Therapies
Oral DMARDs*
Adverse effects/abnormal
laboratory tests
30%
Inconvenient
14%
Laboratory monitoring
Biologics
Fear, anxiety or
inconvenience of injections
26%
Adverse effects/abnormal
laboratory tests
15%
Inconvenient
15%
12%
Pain/discomfort
Lack/loss of
effectiveness
2%
Cost/insurance
issues
1%
Psoriatic Arthritis Patients ±
Psoriasis % (n=820)
Fear of adverse effects
Lack/loss of effectiveness
7%
5%
2%
Psoriatic Arthritis Patients ±
Psoriasis % (n=389)
*Disease-modifying antirheumatic drugs
Source 9: Lebwohl, M., Bachelez, H., Barker, J., et al. Patient Perspectives in the Management of Psoriasis: Results from the Population-Based Multinational Assessment of
Psoriasis and Psoriatic Arthritis. Journal of the American Academy of Dermatology. 2014; 70(5): 871-881.e30.
Safety Considerations May Limit the Use of Certain
Therapies
Safety Considerations*
Oral DMARDs**
• Severe infections
• Severe liver or kidney disorders
• Alcohol abuse
• Acute peptic ulcer
• History of or current malignancy
• Significant anemia, leukopenia or
thrombocytopenia
Biologics
• History of or currently active infections (including
tuberculosis and chronic hepatitis B)
• Systemic malignancy
• Congestive heart failure
• Moderate to severe heart failure
• Sepsis
*Not
an exhaustive list
**Disease-modifying antirheumatic drugs
Source 12: Pathirana, A. European S3-Guidelines on the Systemic Treatment of Psoriasis Vulgaris. JEADV. 2009; 23(2): 1-69.
Source 13: Kalb, R. Methotrexate and Psoriasis: 2009 National Psoriasis Foundation Consensus Conference. Journal of American Academy of Dermatology. 2008; 60(5): 824-837.
Source 14: Chung, E., Packer, M., Hung Lo, K., et al. Randomized, Double-Blind, Placebo-Controlled, Pilot Trial of Infliximab, a Chimeric Monoclonal Antibody to Tumor Necrosis
Factor Alpha, in Patients With Moderate-to-Severe Heart Failure. Circulation. 2003; 107: 3133-3140.
Source 15: Kling, A., Mjörndal, T., Rantapää-Dahlqvist, S. Sepsis as a Possible Adverse Drug Reaction in Patients With Rheumatoid Arthritis Treated With TNF Alpha Antagonists.
Journal of Clinical Rheumatology. 2004; 10(3): 119-122.
4
UNMET NEED
DRIVING INNOVATION
67
4
UNMET NEED
DRIVING INNOVATION
68
Vast Majority of Psoriasis and Psoriatic Arthritis Patients
Believe There Is a Need for Additional Innovative Therapies
Source 9: Lebwohl, M., Bachelez, H., Barker, J., et al. Patient Perspectives in the Management of Psoriasis: Results from the Population-Based Multinational Assessment of
Psoriasis and Psoriatic Arthritis. Journal of the American Academy of Dermatology. 2014; 70(5): 871-881.e30.
69
69
70
5
ACCELERATING INNOVATION
IN IMMUNE DISORDERS
WHAT IS REQUIRED TO CONTINUE
INNOVATION?
This section will highlight the need to accelerate medical innovation
throughout the world. The alternative would lead to the negative
impact of halting medical progress and innovation in immune
disorders, and its significant impact on quality of life, prosperity and
progress.
Although the pace of progress is accelerating, scientists are just
beginning to understand immune disorders. Additional research is
needed to unravel the complexities of immune disorders, including
better understanding of epidemiological data, exploration of gene
and biomarker identification, discovering new cellular pathways and
leveraging new genomic technologies. Greater public awareness
about the burden of these conditions is also essential.
Life science companies, academic institutions, government, medical
societies, patient advocacy groups and the media need to work as a
collaborative ecosystem to foster greater understanding about
immune disorders and stimulate investment in the virtuous cycle of
medical innovation.
5
ACCELERATING
INNOVATION
71
72
“
Despite our progress, we recognize that more
needs to be done so that we may close the
gaps in our knowledge and achieve our overall
goal of reducing the rising toll of autoimmune
disease.
”
Elias A. Zerhouni, M.D.
Former Director, National Institutes of Health
73
74
5
ACCELERATING
INNOVATION
Pace of Medical Progress in Immune Disorders
Is Accelerating
Genomic and
proteomic technology
The development of genomic and
proteomic technologies provides an
ability to identify novel bio-signatures
to diagnose, classify and guide
therapeutic decision making in
patients with immune disorders.
Identification of genes
that cause the
diseases
Biomarker
identification and
development
Researchers are identifying genes that
predispose individuals to develop
immune disorders and studying how
these genes initiate disease process or
exacerbate symptoms, including the
impact of environmental factors on
genes.
Identification of biomarkers (i.e.,
clinical signs or lab tests) will allow
for earlier and more accurate
diagnosis, better prediction of disease
flare-ups and improved monitoring of
disease progression and response to
treatment. Identification of many
biomarkers is needed as some
biomarkers may be common in a
variety of immune disorders and
some unique and disease-specific.
Identification of
cellular pathways
and new drug
targets)
Researchers have been working to
explore intracellular pathways that
decrease the production of antiinflammatory mediators, as well as
identify novel drug targets (e.g.,
PDE4, TNF-α, IL-17, IL-23) in an
effort to discover and develop
innovative treatments.
Maximizing the Promise of Science:
5,000+ Medicines in Development in 2011
2,000
1,800
1,795
1,586
1,600
Number of Medicines
1,400
1,247
1,200
1,000
788
800
600
731
650
454
400
412
204
200
69
0
Rare
Diseases
Infectious
Diseases
Neurological
Disorders
Cancers
Immunological Cardiovascular Musculoskeletal
Conditions
Disorders
Diseases
Diabetes
Mellitus
HIV/AIDS
& Related
Conditions
Liver Disease
& Related
Conditions
Reflects compounds in all phases of development, including having been filed with the Food and Drug Administration (FDA) or approved by
the FDA, but not yet on the market in the U.S. as of December 2011. Medicines with multiple indications may appear in more than one
category, but in the total number (5,000+ medicines), only the initial indication is counted.
Source 1: Long, G., Works, J. Analysis Group, Inc. Innovation in the Biopharmaceutical Pipeline: A Multi‐Dimensional View. January 2013. Available at
http://www.phrma.org/sites/default/files/pdf/2013innovationinthebiopharmaceuticalpipeline‐analysisgroupfinal.pdf. Accessed April, 2015.
5
ACCELERATING
INNOVATION
75
76
5
ACCELERATING
INNOVATION
Active Pipeline with Numerous Treatments* in Development
for These Four Immune Disorders
50
Rheumatoid Arthritis
40
Crohn's Disease
32
Psoriasis**
6
Psoriatic Arthritis
0
10
20
30
40
50
Number of Treatments in Development
*Some of the treatments are being studied in multiple indications
**Includes topical treatments
Source 2: National Psoriasis Foundation. Drug Pipeline. Available at http://services.psoriasis.org/drug-pipeline/index.php. Accessed April, 2015.
Source 3: Pharmaceutical Research and Manufacturers of America (PhRMA). Medicines in Development: Arthritis. 2014.
Available at http://www.phrma.org/sites/default/files/pdf/2014-meds-in-dev-arthritis.pdf. Accessed April, 2015.
Source 4: Datamonitor Healthcare. Pipeline: Inflammatory Bowel Disease (IBD). 2015. Available at https://service.datamonitorhealthcare.com/
disease/immunology-and-inflammation/gastroenterology/inflammatory-bowel-disease/pipeline/?articleId=72704&page=tableau. Accessed April, 2015.
60
A Collaborative Ecosystem Is Necessary to Accelerate
Innovation in Immune Disorders
LIFE SCIENCES
COMPANIES
Continued commitment to R&D, collaboration with
academic researchers, public awareness efforts,
unmet needs assessment
PATIENT ADVOCACY
ORGANIZATIONS
Academic
Institutio
ns
Awareness and unmet
needs clarification
ACADEMIC
INSTITUTIONS
Increased focus on
collaboration to identify
gene markers, new
cellular pathways, etc.
MEDICAL
INNOVATION
MEDICAL
SOCIETIES
Best practice
sharing
GOVERNMENT
RESEARCH
Governmen
t
Research
Medical
Societies
MEDIA
Greater public awareness
and recognition
Greater allocation of
funding for research
Media
5
ACCELERATING
INNOVATION
77
78
5
ACCELERATING
INNOVATION
Promise and Progress in Immune Disorders
We’re making progress but much more needs to be done to accelerate innovation in immune
disorders. Today’s investments in healthcare and R&D can create a better, more healthy and
potentially disease-free world in some conditions for our children and our children’s children.
Gene
therapy
Gene
mapping
Immune modifiers
Cellular
therapies
Personalized
medicine
OFFER HOPE
78
“
This is the most exciting time in the history
of medicine. If we can make some radical
changes to accommodate the enormous
opportunities, there will be better health at
lower costs for many generations to come.
Eric Topol, M.D.
The Creative Destruction of Medicine Author
”
79
80
“
The best way to predict
the future is to invent it.
Alan Kay
American Computer Scientist
”
81
Celgene Corporation
86 Morris Avenue
Summit, NJ 07901
+1-908-673-9000
www.Celgene.com
For inquiries, contact:
Zeba M. Khan, RPh, PhD
Vice President
[email protected]
Summer 2015