Personalized Medicine: The Changing Landscape of Health Care

Transcription

1 Personalized Medicine: The Changing Landscape of Health Care The 2 nd Biomarker Meeting in Personalized Reproductive Medicine Valencia, Spain 11 April, 2014 Edward Abrahams, Ph.D. President Personalized Medicine Coalition

2 Ancient Precedent It s far more important to know what person the disease has than what disease the person has. Hippocrates (ca. 400 BCE) 1

3 Moving from Art to Science If it were not for the great variability among individuals, medicine might as well be a science, not an art. Sir William Osler, Physician (1892) 2

4 Defining Personalized Medicine Current Practice Personalized Medicine One size fits all Trial and Error Trial and error The right treatment for the right person at the right time 3

5 PCAST Definition of Personalized Medicine Personalized medicine refers to the tailoring of medical treatment to the individual characteristics of each patient to classify individuals into subpopulations that differ in their susceptibility to a particular disease or their response to a specific treatment. Preventive or therapeutic interventions can then be concentrated on those who will benefit, sparing expense and side effects for those who will not. President s Council of Advisors on Science and Technology (PCAST), Priorities for Personalized Medicine, September,

6 Revised Definition of Personalized Medicine Personalized medicine is an evolving field in which physicians use molecular diagnostic tests to determine which medical treatments will work best for the patients. By combining the data from those tests with an individual s medical history and circumstances, health care providers and patients can develop targeted treatment and prevention plans. 5

7 Reactions to Definitions of Personalized Medicine Reactions to PCAST and revised definitions of Personalized Medicine were dramatic. People who saw the new definition responded positively, saying it was clear, easy to understand, and consistent with the term. 6

8 Preferred Name for Personalized Medicine By far, the two favorite names were individualized medicine and personalized medicine. The word, targeted was also a favorite. Precision medicine was more negative than positive; and stratified medicine was disliked most of all. 7

9 Hope and Fears for Personalized Medicine The majority of those surveyed feel excited about personalized medicine. The biggest concerns were regarding cost and access to treatments. The most exciting ideas were around better more informed tailored treatment options 8

10 What is Driving Personalized Medicine? Safer, More Effective Drugs Faster Time to a Cure Cost-Effective Healthcare 9

11 The Benefits of Personalized Medicine Diagnose disease more accurately Select optimal therapies and target medicines and dosages more precisely Increase safety, reduce adverse drug reactions Detect onset of disease at the earliest moments Shift emphasis in medicine from reaction to prevention Increase the efficiency of the health system by improving quality, accessibility and affordability 10

12 Major Drugs Ineffective for Many Hypertension Drugs 10-30% ACE Inhibitors Heart Failure Drugs 15-25% Beta Blockers Anti Depressants 20-50% SSRIs Cholesterol Drugs 30-70% Statins Asthma Drugs 40-70% Beta-2-agonists Source: Spear B, Heath-Chiozzi M, Huff J Clinical Trends Molecular Medicine 2001; 7(5):

13 Ineffective Therapies Waste Money Major Drug Hypertension Drugs Ace Inhibitors Cost of Ineffectiveness to Healthcare System $390 million $1.2 billion Heart Failure Drugs Beta Blockers $345 million $575 million Anti Depressants SSRIs $2.3 billion $5.8 billion Cholesterol Drugs Statins $3.8 billion $8.8 billion Asthma Drugs Beta-2-agonists $560 million $1.0 billion 12

14 Ineffective Therapies Can Cause Harm Adverse Events Estimated 100,000 deaths per year (in 1994; Lazarou et al 1998) 6 th leading cause of death in the US Experienced by approximately 7% of patients (2.2 million) per year Medication-related health problems account for an estimated 3% to 7% of hospital admissions (Pirmohamed M, et al 2004) During their hospital stay, 15% of patients experienced adverse drug reactions (Davies, et al 2009) Increased patient non-compliance 13

15 Disease Severity The Old Paradigm: Treatment of Disease Reactive Medical Care Switch Drug Again Diagnosis Switch Drug Select Drug Time Diagnose Disease; Treat Symptoms; Costly, Trial and Error Treatment 14

16 Disease Severity Effective, Efficient Health Management Efficient Medical Care Right Drug Monitoring Diagnosis/Prognosis Predisposition Screening Time Health Management; Molecular Screening; Early Detection; Rapid Effective Treatment; Improved Quality of Care 15

17 Personalized Medicine: Impacts Care 60 Years Ago 5 Year Survival 50 Years Ago "Disease of the Blood" Leukemia or Lymphoma ~0% 40 Years Ago Chronic Leukemia Acute Leukemia Preleukemia Indolent Lymphoma Aggressive Lymphoma Today ~38 Leukemia types identified: Acute myeloid leukemia (~12 types) Acute lymphoblastic leukemia (2 types) Acute promyelocytic leukemia (2 types) Acute monocytic leukemia (2 types) Acute erythroid leukemia (2 types) Acute megakaryoblastic leukemia Acute myelomonocytic leukemia (2 types) Chronic myeloid leukemia Chronic myeloproliferative disorders (5 types) Myelodysplastic syndromes (6 types) Mixed myeloproliferative/myelodysplastic syndromes (3 types) ~51 Lymphomas identified: Mature B-cell lymphomas (~14 types) Mature T-cell lymphomas (15 types) Plasma cell neoplasm (3 types) Immature (precursor) lymphomas (2 types) Hodgkin's lymphoma (5 types) Immunodeficiency associated lymphomas (~5 types) Other hematolymphoid neoplasms (~7 types) 70% Ries LAG, Eisner MP, Kosary CL, Hankey BF, Miller BA, Clegg L, Mariotto A, Feuer EJ, Edwards BK (eds). SEER Cancer Statistics Review, , National Cancer Institute. Bethesda, MD, based on November 2004 SEER data submission, posted to the SEER web site Source: Mara G. Aspinall, former President, Genzyme Genetics 16

18 The Blockbuster Model is Broken The power in tailored therapeutics is for us to say more clearly to payers, providers, and patients -- this drug is not for everyone but it is for you. John C. Lechleiter, Ph.D. Chief Executive Officer Eli Lilly and Company 17

19 Trends in R&D Costs and Drug Approvals R&D Expenditures ($ Billions) 60 New Drug Approvals (3 year moving average)

20 October 2,

21 Decreasing Cost of Genome Sequencing 20

22 Personalized Medicine in R&D Safer, More Effective Drugs Identify disease targets, speed clinical trials, and advance more drugs that are safe and effective for specific populations Faster path to disease targets using genetic data Speed trials by testing on patients selected for likely high response and safety Target Identification Target Validation Lead Development Preclinical Clinical Market Knowledge of biological pathways helps eliminate poor candidates Target optimal population by combining drug with molecular diagnostic test Over 400 drugs currently under review at FDA include biomarkers 100 in late stage oncology clinical trials. 21

23 Tackling Tumors Source: Wall Street Journal Copyright 2011 by DOW JONES & COMPANY, INC. Reproduced with permission of DOW JONES & COMPANY, INC. 22

24 As Science Advances, Oncology Drug Development Accelerates Years From Discovery of Target to Approved Treatment BCR-ABL 2001 EGFR 2003 BRAF 2011 ALK 2011 Target and Year of Approved Treatment Adapted from Gerber and Minna Cancer Cell: 18: 548,

25 Many Biopharma Companies have Launched Therapeutics Informed by CDx Tests Launch timeline of targeted therapeutics with required or recommended CDx Source: L.E.K. 24

26 Marketed Therapeutics with an Associated CDx Test Generate $12B Note: * Branded drug sales; does not include sales of Zelboraf (~35M in 2011) and Xalkori (sales unkown), both approved Aug 2011 ** Other includes: Iressa (AstraZeneca), Camptosar (Pfizer), Selzentry (Pfizer), Tegretol (various), Ziagen (GSK), Imuran (GSK) *** Other includes infectious disease and CNS Source: L.E.K. 25

27 Significant Growth for Commercial Personalized Medicine Diagnostics Note: * Includes non-unique tests Source: L.E.K. 26

28 Personalized Medicine: How Will It Affect Healthcare? 27

29 Changing Role of the Patient Greater knowledge of one s genetic risks Actionable lifestyle prescription for reducing risk of disease Greater control and patientcentered access to medical records Treatment decisions will be improved by patient education 28

30 Changing Role of the Health Care Provider Health care providers as manager, rather than repository of medical knowledge Greater reliance on HIT for decision support Improved care through use of aggregate patient data Highly networked, team-based care New ethical and legal issues/quandaries 29

31 New Business Strategies for Pharma Uncertain economics of drug development and commercialization Regulatory mandates could disrupt development budgets and market plans Personalization of drugs will require partnering with diagnostics companies 30

32 New Demands On and From Payers Greater emphasis on clinical validity and utility of diagnostic tests Increased need to demonstrate cost efficiencies Proactive strategies to limit reimbursement Increased pressure to change paradigm towards preventive medicine 31

33 Personalized Medicine Will Become Fundamental to Our Healthcare Systems Our current healthcare practices cannot continue and still be affordable. Pharmaceutical companies need to increase their probability of technical success to maintain profitability. The public is genuinely interested in genetic information and in having personal choices. We have a regulatory framework that is being designed to meet our future needs. 32

34 Making the Case for Personalized Medicine We face significant challenges in accelerating growth in this field scientific, business, regulatory and policy challenges. Together we must break down the barriers and move personalized medicine forward. John Castellani President and Chief Executive Officer Pharmaceutical Research and Manufacturers of America (PhRMA) 33

35 Policy Issues In Personalized Medicine Reimbursement Regulation Health care Provider Education and Adoption Patient Education Personalized Medicine Comparative Effectiveness Research Privacy / Ethics R&D Incentives Intellectual Property 34

36 Barriers Implementing Personalized Medicine There is nothing more difficult... than to take the lead in the introduction of a new order of things. For the reformer has enemies in all those who profit by the old order, and only lukewarm defenders in all those who would profit by the new order. This indifference arises partly from fear of their adversaries... and partly from the incredulity of mankind, who do not truly believe in anything new until they have had actual experience of it. Niccolo Machiavelli 35

37 The Personalized Medicine Coalition, representing innovators, scientists, patients, providers and payers, promotes the understanding and adoption of personalized medicine concepts, services and products to benefit patients and the health system. 36

38 1710 Rhode Island Ave., NW Suite 700 Washington, DC