New York Bio Conference 2016. Mark J. Alles Chief Executive Officer

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New York Bio Conference 2016 Mark J. Alles Chief Executive Officer

Great Progress AGE 72 World Life Expectancy 2005-2014* 17 new drugs for rare diseases 71.5 71 Two new MS drugs First drug to target root cause of cystic fibrosis 2015: 4 new drugs for multiple myeloma 70.5 70 69.5 First vaccine for the prevention of cervical cancer The first drug for Huntington s disease New class of medicines to treat high blood pressure First treatment for PTCL First therapeutic cancer vaccine First new lupus drug in 50 years 2 new personalized medicines to treat skin cancer Oral treatments for Hep C with upwards of 90% cure rates In 2014, 1 in 5 FDA approvals are personalized medicines 69 2005 2006 2007 2008 2009 2010 2011 2012 2013 2014 2015 2 * The World Bank Data, accessed May 4, 2016, Life Expectancy at birth, total years http://data.worldbank.org/indicator/sp.dyn.le00.in/countries/1w?display=graph

3 Great Opportunities

4 Great Challenges

5 Great Challenges & Great Opportunities

6 The Politics of Pricing vs. The Value of Medical Innovation

We Have Been Asking the Same Question for Decades 7 LIFE Magazine, May 1992 TIME Magazine, April 2013

Jimmy Carter s Cancer Treatment Inspires New Law May 3, 2016 ATLANTA (AP) - Georgia's governor has signed a bill inspired by former President Jimmy Carter's cancer treatment. The measure signed Tuesday by Republican Gov. Nathan Deal prevents insurance companies from limiting coverage of drugs for stage four cancer patients. Supporters of the bill say patients sometimes cannot get certain drugs unless they first try other treatment options. Carter, now 91, announced in August that he had been diagnosed with skin cancer that had spread to his brain and would begin receiving doses of Keytruda. The newly approved drug helps the immune system seek out cancer cells appearing in a patient's body. Carter said in March that he had stopped treatment after several scans found no cancer in his body. 8

The Cost of Pharmaceuticals is a Worldwide Focus Restrictive policies for NICE and the Cancer Drugs Fund in the UK France is calling for the addition of drug pricing to the agenda for the G-7 summit in May Proposals for drug pricing transparency in the EU The United Nations Secretary General has convened a highlevel panel on access to medicines Countries in Asia are looking to NICE Cost Effectiveness Assessments as a model for reimbursement decisions Latin American countries are considering joint negotiations China CFDA proposal to require lowest prices in Asia for new drug applications In Japan, the government is considering moving to annual price reductions

National Health Expenditures (in Billions) The Growing Aging Population Drives Healthcare Spending Increase NATIONAL HEALTH EXPENDITURES IN BILLIONS, 1980-2024 $5,000 $4,000 $3,000 $2,000 Baby Boomers Entering Medicare $1,000 $- 1980 2004 2009 2014 2019 2024 1. Centers for Medicare & Medicaid Services. National Health Expenditure Projections 2014-2024. July 2015. Available at: http://www.cms.gov/research-statistics-data-and-systems/statistics-trends-andreports/nationalhealthexpenddata/nationalhealthaccountsprojected.html

Unless We Find New Solutions, The Cost of Conditions Like Alzheimer s, Diabetes and Obesity Will Be Crippling $1.1 Trillion Proportion of Americans Age 65 and Older Living with Alzheimer s by Disease Stage, 2015-2050 $48B - $66B 30% 27% 0 43% 28% 23% 48% Severe Mild Moderate $390B - $580B 2015 Current 5.1 million 2050 Trajectory 13.5 million Sources: Changing the Trajectory of Alzheimer s Disease: How a Treatment by 2025 Saves Lives and Dollars, Alzheimer s Association; The State of Obesity: Better Policies for a Healthier America: 2014, Robert Wood Johnson Foundation

Far Too Much Cost In Healthcare Is Driven by Waste HUNDREDS OF BILLIONS CAN BE SAVED ANNUALLY WITHOUT ADVERSELY IMP ACTING PATIENT CARE Sources: 1. http://healthaffairs.org/healthpolicybriefs/brief_pdfs/healthpolicybrief_72.pdf 2. http://www.ncbi.nlm.nih.gov/pmc/articles/pmc3934668/ 3.http://www.cdc.gov/tobacco/data_statistics/fact_sheets/fast_facts/ 4. http://www.ncsl.org/portals/1/documents/health/medicalmalreform-2011.pdf 5. 0 Agency for Healthcare Research and Quality. Statistical Brief #172, April 2014 Available from: http://www.hcup-us.ahrq.gov/reports/statbriefs/sb172-conditions- 12 Readmissions-Payer.pdf (Accessed December 9, 2014). 6. IMS Institute for Healthcare Informatics. Avoidable costs in US healthcare: the $200 billion opportunity from using medicines more responsibly. June 2013. http://www.imshealth.com/deployedfiles/imshealth/global/content/corporate/ims%20institute/ruom-2013/ihii_responsible_use_medicines_2013.pdf. Accessed March 2015.

13 Empowering Patients & Digital Medicine: People Will Be More Connected Than Ever

14 Big Data & Personalized Medicine: More Advances Expected Over the Next 5 Years

How Employers are Reducing Healthcare Costs Value-Based Insurance Design Disease Management Programs Employer Health & Wellness Programs Eliminated or Reduced Copays Fully Covers Preventive Care Reduced Premiums if Participated in Disease Management Program Linked Pharmacists & Employees to Fight Chronic Conditions Smoking Cessation Programs Obesity & Wellness Education and Programs Who s Done it? Who s Done it? Who s Done it? 15

Partnering with Government to Advance Solutions AMP (Accelerating Medicines Partnership) Developing new diagnostics and targets in Alzheimer s, type 2 diabetes, rheumatoid arthritis, and lupus The Partners: biopharmaceutical companies, NIH, patient and disease organizations BIOMARKERS CONSORTIUM Combining expertise and resources to rapidly identify, develop, and qualify biomarkers, which will then advance new therapies and guide improvements in regulatory and clinical decision making The Partners: biopharmaceutical companies, NIH, CMS, FDA, patient and disease organizations LUNG-MAP (Lung Cancer Master Protocol) Using comprehensive genetic screening to identify mutations in lung cancer patients in order to direct them to a specific investigational treatment, all operating under a single clinical trial protocol The Partners: biopharmaceutical companies, NIH, FDA, patient and disease organizations CLINICAL TRIALS TRANSFORMATION INITIATIVE (CTTI) CTTI engages all stakeholders as equal partners to analyze existing research impediments and recommend consensus-driven, actionable solutions that will lead to a more sustainable and effective clinical trial system. The Partners: biopharmaceutical and device companies, NIH, CMS, professional societies, academic institutions, patient and disease organizations REAGAN UDALL FOUNDATION FOR THE FDA Independent 501(c)(3) not-for-profit organization created by Congress to advance the mission of the FDA The Partners: biopharmaceutical and device companies, NIH, CMS, professional societies, academic institutions, patient and disease organizations 16

Part D One of the Most Successful Partnering Examples Gaining Part D coverage resulted in an 8% decrease in hospital admissions for seniors overall PHARMACEUTICAL INDUSTRY SUPPORT FOR THE AFFORDABLE CARE ACT ESTIMATED TO TOTAL APPROXIMATELY $90B BY 2020, WITH APPROXIMATELY $30B IN EXPENSES FROM 2011-2020 RELATING TO ELIMINATING PART D COVERAGE GAP 17 Sources: PhRMA Medicines in development for Older Americans Reports, 2003 and 2013; Kaestner R, Long C, Alexander C. Effects of prescription drug insurance on hospitalization and mortality: evidence from Medicare Part D. The National Bureau of Economic Research. Published February 2014; http://www.businesswire.com/news/home/20120430005168/en/2020-pharmaceutical-industrys-support-u.s.-affordable-care

Retail Spending on Prescription Medicines Is a Small Share of Total US Health Care Spending Prescription medicines today account for about 10% of health care spending in America, the same percentage as in 1960. *Other includes dental, home health, and other professional services as well as durable medical equipment costs. PhRMA analysis of CMS data. National health expenditures by type of service and source of funds, CY 1960-2013. Baltimore, Md.: CMS; 2013. Accessed February 2015. Profile Figure 4 Accessed at http://chartpack.phrma.org/biophrma-chartpack/spending-and-costs/retail-spending-on-prescription-medicines-is-a-small-share-of-total-us-health-care-spending.

One Driver of Healthcare Spending Guaranteed to Go Down in Cost: Prices of Current Best-In-Class Therapies, Due To Limited Exclusivity Periods Cost Per Day ($) Jan-06 Jul-06 Jan-07 Jul-07 Jan-08 Jul-08 Jan-09 Jul-09 Jan-10 Jul-10 Jan-11 Jul-11 Jan-12 Jul-12 Jan-13 Jul-13 Jan-14 Jul-14 Jan-15 Jul-15 Jan-16 Jul-16 Jan-17 Jul-17 Medicine Year Brand Price Then Generic Price Now % Change DIOVAN HCT 2010 $87 $15-85% Daily Cost of Top-10* Therapeutic Classes Most Commonly Used by Part D Enrollees Has Declined and is Estimated to Continue to Decline 1.60 1.40 1.20 Actual Estimated LIPITOR 2010 $85 $5-95% PLAVIX 2011 $166 $8-97% SEROQUEL 2010 $87 $7-97% ZYPREXA 2010 $393 $12-98% Figures represent the average annual price for 30 pills of the most commonly dispensed form and strength. Then price represents the average price in the year prior to generic entry. Now price represents the average price in CY 2016. 1.00 0.80 0.60 0.40 0.20 0.00 * From 2006 19 Sources: IMS Analysis of PhRMA, May 2015; Kleinrock M. Daily cost of Medicare Part D. December 2013 Update. December 2013. IMS Institute for Healthcare Informatics.

Solutions Will Come From Us Source: Sampat B, Lichtenberg F. What are the respective roles of the public and private sectors in pharmaceutical innovation? Health Affairs. 2011; 30(2):332-339. 20

Over 7,000 New Medicines in Development CANCERS 1,813 CARDIOVASCULAR DISORDERS 599 DIABETES 475 HIV/AIDS 159 IMMUNOLOGICAL DISORDERS 1,120 INFECTIOUS DISEASES 1,256 MENTAL HEALTH DISORDERS 511 NEUROLOGICAL DISORDERS 1,329 Source: Adis R&D Insight Database. Accessed May 2015. 21

22 Investment Is Coming From Our Industry

Celgene Has Championed a Mutually Beneficial Collaborative Model CELGENE PLATFORMS JNK1, New Targets, Novel phenotypic screens GDF Family PKCq, BTKi, Novel Targets Fibrosis Immunology Payload Delivery, Next Gen Enhanced Activities Next Generation Biologics Novel IMiDs / CRBN & Other Ubiquitin Ligase Targets Protein Homeostasis New Targets, Epigenetic Priming & Convergence w/ Metabolic Targets Epigenetics Immuno- Oncology Complementary Approaches to I/O, Breaking Tumor Tolerance Cancer Stem Cells/ Resistance Unique Validation / Testing Capabilities from Tumor Tissue 23

Future Medical Breakthroughs Will Come from Innovative & Entrepreneurial Solutions Patients Are Waiting

New York Bio Conference 2016 Mark J. Alles Chief Executive Officer