Call to governments: Boost innovation for neglected diseases

1. Call to governments: Boost innovation for neglected diseases Bernard Pécoul Executive Director MSF meeting 8 June 2005

2. Neglected patients need new drugs, vaccines and diagnostics NOW

3. Neglected diseases desperately need new therapies Global Diseases Most Neglected Diseases Neglected Diseases World pharmaceutical market> $518 bn in 2004

4. Sleeping sickness is a most neglected disease Source: WHO 2001 • An estimated 300,000 infected • 55 million at risk in sub-Saharan Africa • Difficult to diagnose • Fatal if untreated • Existing drugs: old - toxic - resistance - difficult to use - expensive

5. The needs remain huge Arsenical Anti-cancer drug

6. Leishmaniasis: 350 million at risk • An estimated 12 million people affected in 88 countries • Different forms: visceral, (muco)cutaneous, PKDL • Per year: 1-1.5 million new cases of CL/MCL • 500,000 cases of VL • VL is fatal if left untreated • Existing drugs: old - toxic - resistance - difficult to use - expensive

7. Chagas disease: 18 million infected • Endemic in Latin America • Threatens 100 million people in 21 countries • In about one-third of the acute cases, chronic forms develop 10 – 20 years after infection • Severe chronic disease leads to death  • Only two drugs for this disease – not effective for chronic patients

8. No drugs at all for Buruli ulcer Source: WHO, WHO/CDS/CPE/GBUI/2001.1

9. 18 new drugs for AIDS But it is still a neglected disease for adults and children living in developing countries • Drugs not adapted to health systems of endemic countries • No treatment adapted to children • Limited tools for diagnosis and follow up • No field-adapted preventive tools

10. The fatal imbalance

11. Developing countries have a tiny share of the pharma market World Pharmaceutical Market, 2004:Total $518 billion Source: IMS Health

12. Tropical diseases: 13 Tuberculosis: 3 Only 1% of new drugs developed are for neglected diseases • Approx. 1-2% is spent on R&D for neglected diseases • 10/90 gap in health research spending • 1975-1999: 1,393 new chemical entities marketed

13. Insignificant progress towards new health tools for the poor In spite of • Huge increase in global funding for health research - from $30 bn in 1986 to US$106bn for 2004 (Monitoring Financial Flows for Health Research, Global Forum for Health Research, 2004) • Higher levels of intellectual property protection have not resulted in increased drug R&D for global health needs (UK Commission on Intellectual Property Rights, 2002)

14. Pre Clinical Development Availability to patients Discovery GAP2 GAP3 GAP1 Gaps exist in the R&D process for neglected diseases New knowledge on drug targets and lead compounds is published but pre-clinical research does not begin mainly industry (in North) mainly public sector New or existing drugs do not reach patients: registration problems, lack of production, high prices, or not adapted to the local conditions of use Validated candidate drugs do not enter clinical development because of strategic company choices.

15. In recent years… • Increased awareness from the global community • Developing countries strengthening their R&D capacity • New not-for-profit initiatives established

16. DNDi is an alternative model • To develop new drugs for neglected diseases • Ensure equitable access to needs-driven products • Strengthen existing capacity in disease-endemic countries • Build public responsibility and leadership • Bring together the public sector and pharmaceutical industry

17. DNDi was created in 2003 Medecins Sans Frontieres (MSF) WHO/TDR (permanent observer) Institut Pasteur, France Malaysian Ministry of Health Oswaldo Cruz Foundation, Brazil Indian Council for Medical Research (ICMR) Kenya Medical Research Institute (KEMRI)

18. 18 projects in DNDi’s portfolio 2005

19. 2 new malaria drugs in 2006 e.g. DNDi/sanofi-aventis agree to deliver AS/AQ anti- malarial fixed dose combination • Easy to use: fewer tablets in treatment regimen • Affordable: Target price for public sector, <$1 for adult and $0.5 for children • Available: 2006, non exclusive rights

20. Neglected Diseases Appeal 1. More public leadership • Make global health and medicines a strategic priority • Set R&D agenda according to the needs of patients • More than just philanthropy Sign up at www.researchappeal.com

21. Neglected diseases Appeal 2. Sustained financial support • Raise current levels of funding for neglected diseases by 3 billion euros per year to start to correct the 10/90 gap • Put in place new, sustainable funding mechanisms (IFF, Global tax, …) Sign up at www.researchappeal.com

22. Neglected Diseases Appeal 2. Sustained financial support focused on: • A needs-driven R&D agenda • Maintaining basic scientific research • Translation of basic research into new medicines • Strengthening R&D capacity in DEC • Securing the market Sign up at www.researchappeal.com

23. Neglected Diseases Appeal 3. New rules to stimulate drug R&D Regulatory standards • Streamline approval processes • Analyse risks and benefits of each drug or vaccine • Build regulatory capacities in developing countries (support from EMEA and FDA ) Sign up at www.researchappeal.com

24. Neglected Diseases Appeal 3: New rules to stimulate drug R&D Intellectual Property: develop drugs as public goods • Ensure that public sector develops open access to information • Ensure that industry provides sustainable access to knowledge, chemical compounds and tools • Freedom to operate in R&D for ND • Make technology transfer to disease-endemic countries happen Sign up at www.researchappeal.com

25. Global Appeal Without bold new steps disease will continue to ravage the developing world, with global consequences. Governments should act NOW

27. Neglected Diseases Appeal Sign up at www.researchappeal.org