Call to governments Boost innovation for neglected diseases

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

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

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 Ø 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

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

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

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 • 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 Tropical diseases: 13 Tuberculosis: 3

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$106 bn 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)

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) GAP 1 Discovery mainly public sector Pre Clinical Development GAP 2 Validated candidate drugs do not enter clinical development because of strategic company choices. Availability to patients GAP 3 New or existing drugs do not reach patients: registration problems, lack of production, high prices, or not adapted to the local conditions of use

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

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

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

18 projects in DNDi’s portfolio 2005

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

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

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

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

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

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