Jacqueline Keith, J.D. New York Pharma Forum October 16, 2013 J - PowerPoint PPT Presentation

Jacqueline Keith, J.D. New York Pharma Forum October 16, 2013 J Keith Consulting Corp.

 Global Environment  Vaccine Milestones  Public-Private Partnerships  Innovative Financing  Economic Context  Evolving Landscape 2 J Keith Consulting Corp.

 Under-5 mortality has declined by ½ over the past 50 years  But in least developed countries (LDC’s), 1,500 people die every hour from infectious disease (ID); ½ of them children  LDC’s bear 90% of the burden of global infectious disease – but ID represents only 10% of global R&D  New approaches are needed to foster R&D and timely delivery for these “neglected tropical diseases” (NTDs )  The solution lies in inn nnovative ative scien ience, ce, partners tnerships hips, , and d finan nancing cing 3 J Keith Consulting Corp.

◦ The past 10 years have seen the introduction of new generation vaccines against the two of the leading vaccine-preventable causes of death in the developing countries -- pneumococcal and rotavirus disease ◦ Of 374 neglected tropical disease (NTD) projects currently underway, 201 (54%) are vaccines targeted at 16 diseases affecting the poorest countries* ◦ Many are the result of collaboration between large pharma companies, small biotech enterprises, academia, and public partners * Source: Bio Venures for Global Health 2012 4 J Keith Consulting Corp.

Product Development Partnerships (PDPs)  Meningitis Vaccine Project: Gates/NIH/Serum Institute of India  Aeras: R&D for tuberculosis vaccines  International AIDS Vaccine Initiative (IAVI)  Dengue Vaccine Initiative (DVI)  WIPO* Re:Search Consortium (offering shared access to intellectual property (e.g. compound libraries, regulatory dossiers, and platform technologies) to support research into neglected diseases.) * World Intellectual Property Organization 5 J Keith Consulting Corp.

1970’s : WHO Expanded Program on Immunizations (EPI) • Objective: universal access to 6 routine pediatric vaccines • Challenge: only 5% coverage of children in some LDCs • Price: <$1.00 (all 6 vaccines) – differential pricing 1980’s/l990’s: Children’s Vaccines initiative (CVI) • Objective: enhance immunization coverage in LDCs • Coverage of EPI vaccines grew to 80% 1990’s: New Generation Vaccines • Complex new technologies/more expensive • Time lag between introduction in high-and low-income countries(15 years) 6 J Keith Consulting Corp.

2000: Global Alliance for Vaccines and Immunization (GAVI) • Objective: accelerate access to new vaccines in the LDCs • Public-private partnership • Immunized 288 million children, saving est. 5.5 million lives (to date) 2010: Decade of Vaccines (DOV) • Announced in 2010: $10 billion (Gates Foundation) • Collaboration between Gates Foundation, WHO, UNICEF, NIAID • By 2020, save 6.4 million lives/avert $151 billion in treatment costs/lost productivity – via increased vaccines R&D and access • Global Vaccine Action Plan approved at 2012 World Health Assembly 7 J Keith Consulting Corp.

2011-15: GAVI Replenishment Cycle • Concluded on June 13, 2011 in London • US$ 4.3 billion was pledged • Multi-year predictable funding to immunize a quarter of a billion children in developing countries 2012-2020: Global Vaccine Action Plan (GVAP) • Implementation of DOV mission • 6 strategic immunization objectives • Monitoring and Evaluation Framework (indicators) • Annual progress reports: country/regional • World Immunization Week (2013) 8 J Keith Consulting Corp.

 Launched 2000  Public Private Partnership  72 GAVI-eligible countries (<$1,550 GNI per capita)  Vaccines: ◦ Phase 1 (2000-2006): Hep B, Hib, Yellow Fever ◦ Phase 2 (2006-2015): Pneumococcal, Rotavirus ◦ Phase 3 (2015-2020): malaria, cholera, yellow fever, rabies* * Subject to funding availabliity 9

10 Source: GAVI Alliance

◦ GAVI I Alli lian ance: Gates Foundation/donor countries (US $7.6 billion: 2010-2015) ◦ Inter ternati national al Fina nance nce Facility acility for Immu mmunizat izatio ion (IFF FFIm Im)  Launched 2006  Raises funds by issuing bonds in the capital markets – backed by long-term government pledges (UK, France, Italy, Spain, Sweden, Norway, South Africa)  US$ 5.3 billion over 20 years (triple-A rating) ◦ Advan vance e Market ket Comm ommit itmen ent t (AMC) MC)  Sustainable markets for needed new vaccines (“pull”)  Demand “guarantee”  AMC price/tail price  Pneumococcal pilot launched 2009 (UK, Canada, Italy, Norway, Russia Gates Foundation):  US $1.5 billion/ 18 countries (2012) 11 J Keith Consulting Corp.

 Vaccines as “Public Good”  Public vs. private market  Prevention vs. Treatment  Cost-Benefit Ratios/QALYs  Tiered Pricing/Access in LDCs  Lower-Middle Income Countries (LMICs)  GAVI “graduating” countries  Price Referencing/Transparency 12 J Keith Consulting Corp.

Tiered Pricing – the charging of lower prices in poorer countries, taking into  account their economic status – has been a fundamental aspect of current global vaccine procurement, and has contributed to enhanced vaccine access. The launch of initiatives such as GAVI, are providing -- for the first time --  sufficient funding to support introduction of new vaccines in the least developed countries (LDCs) By targeting these LDCs, these initiatives have had the unintended  consequence of the MICs/LMICs falling behind in introduction of new vaccines In recognition of this disparity, the international community is debating how to  address the lack of vaccine uptake in MICs This evolving situation – combined with rising costs of new vaccines – is raising  challenges to traditional concepts of tiered pricing. It is also prompting calls for the speedy market entry of emerging country  producers through technology transfer, voluntary licensing, and patent pools. 13 J Keith Consulting Corp.

GAVI can prevent four million future death deaths by immunizing 243 million children from 2011- 2015.
 
 - See more at: http://www.gavialliance.org/about/#sthash.bpD7j2s5.dpuf 14

New York Pharma Forum Building a Global Vaccine Business Rajeev Venkayya, MD Head, Vaccines Business Division Takeda Pharmaceuticals International, Inc.

Vaccination is high-impact, focused on prevention, and driven by innovation “The most successful and cost -effective health care intervention, second only to clean drinking water” “Vaccines prevent 3 - 4M deaths/ year” “New vaccines / better global use of existing vaccines could prevent another 4- 7M deaths” “The single most important tool to reach the 2000 UN Millennium Development Goals” “We must make this the Decade of Vaccines” Quotes from Gro Brundland, Jeffrey Sachs, Kofi Annan, Julio Frenk, Bill Gates and others 16

Vaccination is one of the greatest medical advances in human history Annual Morbidity Percent Disease in the US 2006 Decrease (20 th Century) Smallpox 48,164 0 100% Diphtheria 175,885 0 100% Polio (paralytic) 16,316 0 100% Rubella 47,745 11 >99% Measles 503,282 55 >99% Hib 20,000 208 99% Mumps 152,209 6,584 96% Pertussis 147,271 15,632 89% Hep A 117,333 3,579 87% Varicella 4,085,120 48,445 85% Hep B 66,232 4,713 80% Sources: CDC, MMWR April 2, 1999. 48:242-264 and August 24, 2007/56(33);851-863 17

Vaccination is a major contributor to child health Global childhood mortality declined by half over the past 50 years . The global mortality for children has declined by half over the past 50 years More than 100 million infants are immunized each year with over 2 billion doses of vaccines Source: UNICEF and Johns Hopkins University. Novel vaccines and advances in delivery mortality can be cut in half again by 2025 18

Vaccination remains one of the most cost- effective public health interventions Costs prevented or saved by routine Reduction in mortality for diseases prevented childhood vaccination programs, US or treated with innovative medicines (vaccines, pharmaceuticals; 1965-1999) VACCINATION 24,930 5,874 Infectious ≥97% Diseases 4,890 (polio, measles, Hib, HBV, Hib etc) Costs ($M) 3,545 THERAPEUTICS Rheumatic fever and 2,689 - 75% rheumatic heart disease Hypertensive heart - 67% 1,456 disease 1,121 993 380 - 61% Peptic ulcer disease Ischemic heart - 41% disease Source: EFPIA 1999 – 2002 Source: Zhou F et al. Arch Pediatr Adolesc Med 2005; 159:1136-44. 19

Why is Takeda pursuing a global vaccine business?  Extends Takeda’s commitment to global public health  The vaccine market is expanding rapidly, and has higher R&D success rates than pharmaceuticals • Better long term investment than pharma • No threat of rapid decline in sales post patent expiry  Significant barriers to entry limits competition • Complex intellectual property issues • Long development timelines • Complex manufacturing • Large scale investments needed to become Source: EvaluatePharma, Extracted April 2012; Sanofi Aventis 2008 FY Consolidated Income Statement for Vaccines OpInc 29.8% on Net Sales of EURO 2,861 million; established in the industry quote from Q12010 Conference Call - CFO Julian Hislop ; industry PTS survey  Takeda has the ambition, capability and commitment to overcome these barriers 20