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

Neglected patients need new drugs, vaccines and diagnostics NOW

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

Sleeping sickness is a most neglected disease 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 Source: WHO 2001

The needs remain huge Arsenical Anti-cancer drug

Leishmaniasis: 350 million at risk An estimated 12 million people affected in 88 countries  Different forms: visceral, (muco)cutaneous, PKDL Per year: 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

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

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

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 18 new drugs for AIDS

The fatal imbalance

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

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

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) Insignificant progress towards new health tools for the poor

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 Validated candidate drugs do not enter clinical development because of strategic company choices. New or existing drugs do not reach patients: registration problems, lack of production, high prices, or not adapted to the local conditions of use mainly public sector mainly industry (in North) Availability to patients Development Pre Clinical Discovery GAP2GAP3GAP1

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

DND i 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

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

18 projects in DND i’ s portfolio 2005

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 2 new malaria drugs in 2006 e.g. DNDi/sanofi-aventis agree to deliver AS/AQ anti- malarial fixed dose combination

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 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 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 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 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 Neglected Diseases Appeal

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

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