Download IASC Geneva Dec 2005 - Inter

They need new drugs,
vaccines and diagnosis now:
reality of neglected diseases
Bernard Pecoul
Executive Director, DNDi
Geneva
7 December 2005
Global causes of death
Respiratory
7%
Injuries
9%
Infectious and parasitic
diseases 19%
Infectious and Perinatal
4.3%
Parasitic diseases
33%
Maternal
1%
Other noncommunicable
diseases
16.9%
Cardiovascular
29.3%
Source: WHO Health Report 2004
Cancers
12.5%
Defining neglected diseases
Global Diseases
Most Neglected
Diseases
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
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The needs remain huge
Arsenical
Anti-cancer drug
Leishmaniasis
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An estimated 12 million people affected
 Different forms: visceral, (muco)cutaeous, PKDL
350 million people at risk in 88 countries
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
Buruli ulcer
Source: WHO, WHO/CDS/CPE/GBUI/2001.1
AIDS is 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
Treatments do not exist or are
inadequate and inaccessible
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Toxic
Expensive
Painful to deliver
Difficult to follow up
Not adapted to
patient’s needs
• Not registered in
endemic regions
• Restricted by
patents
Analysing the problems:
Fatal imbalance
Developing countries have a tiny
share of the pharma market
World Pharmaceutical Market, 2004: Total $518 billion
Japan $58bn
(11.1%)
Asia, Africa and
Australia $40bn
(7.7%)
Latin America
$19bn (3.8%)
Rest of Europe
$9bn (1.8%)
EU $144bn
(27.8%)
Source: IMS Health
North America
$248bn (47.8%)
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
Spending on health R&D has
increased
•
World-wide spending on health R&D was never so high
– Estimated at US$106bn for 2004 (GFHR, 2004)
•
Since 90’s: private sector has become biggest investor
Bn US$
US-spending on health R&D:
(>2/3rd total)
35.0
Government
30.0
Industry
25.0
20.0
15.0
10.0
5.0
Sources:
For government: National Science Foundation 2004,
http://www.nsf.gov/sbe/srs/nsf04329/pdf/nsf04329.pdf
For Industry: PhRMA 2004,
http://www.nsf.gov/sbe/srs/nsf04329/pdf/nsf04329.pdf
0.0
1980
1985
1990
1995
2000
2005
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)
GAP1
Discovery
mainly public sector
Pre
Clinical
Development
GAP2
Validated candidate drugs
do not enter clinical
development because of
strategic company
choices.
Availability
to patients
GAP3
New or existing drugs do not
reach patients: registration
problems, lack of production,
high prices, or not adapted to
the local conditions of use
…due to failure of the market and
public policy
Market failure
 Drug development
largely confined to
the R&D-based
pharmaceutical
industry operating
for profit
 Poorer patients are
thus neglected
Public policy failure
 Public policy does
not redress this
imbalance
DNDi’s created in 2003: vision
•
Use an alternative model to develop new drugs for
neglected diseases - leishmaniasis, sleeping
sickness and malaria
•
Ensure equitable access of needs-driven products
•
Strengthen existing capacity in disease-endemic
countries
•
Build public responsibility and leadership in
addressing needs of these patients
•
Bring together the international community, public
sector and pharmaceutical industry
DNDi’s Founding Partners
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
Discovery
Nitroimidazoles project for trypanosomiasis
Pharma
• sanofi-aventis, France-Germany
• Roche, CH
• Chiron, USA
• Novartis (NITD), CH Singapore
• Romark, USA
• Alkem, India
Academics
OBJECTIVE: To identify new drug candidates amongst old
and new nitroimidazoles for trypanosomiasis
• Swiss Tropical Institute
• Fiocruz, Brazil
• U of Sao Paolo, Brazil
• U of Tehran, Iran
• U of Bern, CH
• Silesian University, Poland
• Roma University, Italy
other
+ contacts Japan, USA
• TB alliance
• Dr Nagarajan , India
DNDi
Pre-clinical development
Ravuconazole project for Chagas disease
Pharma companies
Academic groups
•Federal Univ of
Ouro Preto, Brazil
•Instituto Venezolano
de Investigaciones
Científicas, Venezuela
DNDi
Eisai, JAPAN
OBJECTIVE:
To investigate the
activity and toxicity of
ravuconazole in
preclinical disease
models for acute and
chronic Chagas disease
Clinical development
Leishmaniasis East Africa Platform
A group of scientists
•University of Khartoum
(LEAP)
•Federal Ministry of Health
•MSF- Holland
SUDAN
•Addis Ababa University
•DACA
•Ministry of Health
ETHIOPIA
DNDi
and institutions
working on
developing clinical
trial capacity to
bring new
treatments to
patients
IOWH- India
IDA
WHO/TDR
•Ministry of Health
•KEMRI
KENYA
Agreement between DNDi and
sanofi-aventis
Governments should tackle this
imbalance
• The response should not be purely
philanthropic
• Governments should increase
public responsibility towards R&D of
drugs for neglected diseases
– More political leadership
– Sustained financial support
– New rules to stimulate drug R&D
Increased public responsibility:
1. More public leadership
• Make global health and
medicines a strategic priority
•
Set R&D agenda according to
the needs of patients
Increased public responsibility:
2. Sustained financial support
Governments need to
• 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
2. Sustained financial support
This funding should be focused on:
 A needs-driven R&D agenda for safe,
effective, affordable and field-adapted
treatments
 Encouraging scientific community to do
basic research on neglected diseases
 Translation of basic research to new medical
applications, e.g. by supporting PDPs
 Encouraging R&D capacity strengthening in
disease-endemic countries
 Securing the market
Increased public responsibility:
3. New rules to stimulate drug R&D
• Regulatory standards
• Streamline regulatory approval processes to
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rapidly deliver essential medicines to patients
Analyse risks and benefits of each drug or
vaccine in relation to the needs of patients,
severity of the disease and lack of alternative
solutions
Regulatory authorities (FDA and EMEA)
should provide support and transfer knowhow to authorities in developing countries
3: New rules to stimulate drug R&D
Intellectual Property: develop drugs as public
goods
• Guarantee that the public sector develops
open access to information (recent Wellcome
Trust policy) => open source (Human
Genome Project) => freedom to operate
• Ensure that industry provides sustainable
access to knowledge, chemical compounds
and tools
• Make technology transfer happen to diseaseendemic countries
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