Industry's Commitment to R&D for Diseases of the Developing World

1. Alicia D. Greenidge, Director General, IFPMAWHO Expert Working Group, Geneva, 12 Jan 2009 IFPMA Presentation to WHO Expert Working Group on Funding R&D for Diseases of the Developing World (DDW)

2. Tackling DDWs: a Combined Effort… Access (incl. Delivery) Basic Research Research & Development Manufacture Academia Government Agencies R&D Industry R&D Industry Product DevelopmentPartnerships (PDPs) Governments Foundations R&D Industry Generic Industry … to benefit patients in developing countries

3. Industry is Committing Resources to DDW R&D through multiple mechanisms… Independent Efforts Industry Dedicated DDW R&D Centers Partnerships (Current industry involvement & partnerships - illustrative only - not comprehensive)

4. R&D for a New Medicine: 10+ years, $1 bn+ … a big challenge for addressing both developed and developing world diseases RegulatoryReview Post-MarketingSurveillance Scale-Up to Manufacture Discovery Preclinical Clinical Trials PhaseIII PhaseI Phase II ~ 5,000 – 10,000 250 5 1Approved NewMedicine Compounds Pre-Discovery IND Submitted NDA Submitted Number Of Patients / Subjects 20 – 100 100 – 500 1,000 – 5,000 Indefinite 3 – 6 Years 6 – 7 Years 0.5 – 2 Years $335 million (1) $467 million (1) Sources: Drug Discovery and Development: Understanding the R&D Process, www.innovation.org; CBO, Research and Development in the Pharmaceutical Industry, 2006(1) incl. cost of capital – from The price of innovation: new estimates of drug development costs, DiMasi et al, Tufts Center for the Study of Drug Development, 2002

5. Industry R&D for DDW is Growing # R&D Projects by IFPMA Companies, with Product Development Partnerships or alone Medicine R&D projects Vaccine R&D projects TDR 10 Priority Diseases - Chagas- Dengue- Human African Trypanosomiasis- Leishmaniasis- Leprosy- Lymphatic Filariasis- Malaria- Onchocerciasis- Schistosomiasis- Tuberculosis Number of Active R&D Projects 1 project = 1 compound in development OR 1 screening program for 1 disease Sources: 2005 - Moran et Al “New Landscape…” 2006, 2007, 2008 – IFPMA Status Reports

6. Industry DDW R&D is Increasingly Collaborative # Medicine & Vaccine R&D Projects by IFPMA Companies, with PDPs or alone 67 58 49 Companies working alone 32 Companies working with PDPs Sources: 2005 - Moran et Al “New Landscape…” 2006, 2007, 2008 – IFPMA Status Reports

7. Current Industry DDW R&D is Mostly Early Stage Non-industry R&D for DDW is also predominantly preclinical (or earlier)

8. Factors for Increased DDW R&D Product Development Partnerships Large Scale Donor Funding for Access Increased Not-for-Profit R&D Work

9. Basic Research Organizations Bill & Melinda Gates PDP Since Focus Clinton Foundation 2003 TB vaccines CIDA 2003 HAT, VL, Chagas & malaria medicines Carter Center 2000 TB medicines DFID Doris Duke 2000 VL, malaria, diarrhoea& STH medicines Industry players GAVI 2003 Dengue vaccines Global Fund 2000 Malaria medicines Grand Challenges PEPFAR 1999 Malaria vaccines PMI 1977 Malaria, rotavirus, JE, pneumococcal & meningitis vaccines Rockefeller Independent PDP-driven R&D USAID 2003 Pneumonia vaccines Wellcome Trust 2003 Rotavirus vaccines World Bank 2000 Hookworm vaccines 1998 TB treatments Central Role of PDPs in DDW R&D Bringing partners together over the last decade… Funders Product Development Partnerships R&D Execution (Funders, PDPs, Basic Research Organizations & Industry Players - illustrative only - not comprehensive)

10. Some DDWs are More Neglected than Others Some “More Neglected Diseases” need R&D more urgently than others and should be prioritized 4 Approved Meds: ALL intravenous or intramuscular, 3 are v. old 0 Approved Meds 2 Approved Meds: 1) intravenous & side effects 2) v. expensive 2 Approved Meds: BOTH less effective against fatal late stage 1 Approved Med: Effective, large donation program. Resistance? 3 Approved Meds: Effective, large donation program 3 Approved Meds: Effective, large donation program 1 Approved Med: Effective, large donation program (HAT = Human African Trypanosomiasis or “Sleeping Sickness”) Source: 2008 IFPMA Status Report

11. Current DDW R&D Funding will be Insufficient as Pipelines Advance Dalberg Study for IFPMA (2007) • Total Funding for PDPs to 2007 = USD 0.5 billion • Est. funding to take current PDP pipelines to approval AND to augment inadequate pipelines for Most Neglected Diseases = USD 8.3 billion Source: Dalberg for IFPMA

12. Study Also Looked at Sustainable Funding Structure Diseases Treatments Stages of the R&D value chain Intermediaries & recipients Portfolio management Sustainable Funding Funding Prioritization Malaria & TB Medicines Clinical trials focus – Malaria and TB(3) Pre-selection of intermediaries / recipients Full Portfolio management with Fund Push financing – spot commitments Picking winners TDR list(1) - excluding those w/o PDPs Medicines + Diagnostics All stages of the value chain – VL, Chagas, HAT(3) All intermediaries - All recipients Partial Portfolio management with Fund Push financing – long term commitments Spreading TDR list - excluding Malaria and TB Medicines + diagnostics + vaccines Discovery only – smaller diseases(3) Push financing – IFF mechanism Impact based sequence TDR list Success based sequence Firm Recommendation Initial recommendation TDR list + 7 diseases from NTD list(2) Options Notes: (1) TDR: WHO Special Programme for Research and Training in Tropical Diseases (2) NTD: WHO Control of Neglected Tropical Disease programme (3) Clinical trials only for Malaria and TB, discovery only for other diseases without existing PDPs, all stages of the value chain for other diseases with existing PDPs Source: Dalberg for IFPMA

13. DDW R&D Trends that will Impact Financing • Maturing Pipeline • More projects entering expensive later stages (clinical trials) • Need to invest in trials infrastructure & regulatory frameworks • Regulatory delays can be significant (e.g. ~4 years in S. Africa) • Some Avenues of Research Prove Fruitless • From Development, return to Basic Research (e.g. Dengue) • Implications for total funding and for overall timeframe • New Funding Sources / Incentives • Advanced Market Commitment (AMC) • Priority Review Voucher (PRV) • Recognition of Importance of DDW R&D

14. Enhanced Industry Contribution: Potential Next Steps • Consult with WHO & other partners to prioritize DDW R&D needs • Explore working with African Network for Drugs Discovery & Innovation (ANDI) • IFPMA working on a Developing Country Young Researcher Award • IFPMA exploring ways to facilitate increased availability of industry resources • IFPMA Members looking at additional activities in partnership with DNDi et al • Consulting on other possible collaborations

15. Conclusions • DDW R&D has increased substantially • Increased activity by Product Development Partnerships • Increased in-kind & not-for-profit contribution by industry • Current DDW R&D is mainly in less expensive earlier stages • Funding requirements will increase as more projects move into clinical trials • Industry is working to help expand and improve current DDW effort • Current volume of DDW R&D inadequate to deliver enough new medicines for all the most needy diseases • Shortfall is significant – funding to date perhaps 10 % of what is needed • Additional funding critical to achieve adequate range of new medicines & vaccines for Most Neglected Diseases

16. Combined Effort Needed to Advance Existing & Future Pipeline for DDWs Access (incl. Delivery) Basic Research Research & Development Manufacture Academia Government Agencies R&D Industry R&D Industry Product DevelopmentPartnerships (PDPs) Governments Foundations R&D Industry Generic Industry … to benefit patients in developing countries

17. Thank You!