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

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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: 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

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

Rest of Europe $9bn (1.8%)

Japan $58bn (11.1%)

Latin America $19bn (3.8%)

Asia, Africa and Australia $40bn

(7.7%)

North America $248bn (47.8%)

EU $144bn (27.8%)

Developing countries have a tiny share of the pharma marketWorld 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

• 1975-1999: 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)

Availabilityto patients

DevelopmentPre

ClinicalDiscovery

GAP2 GAP3

GAP1

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 disease-

endemic countries

• Build public responsibility and leadership

• Bring together the public sector and

pharmaceutical industry

DNDi 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 DNDi’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

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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, …)

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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

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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 )

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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

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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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