Transcript Financing and R&D for neglected diseases impact & role of product development partnerships - Andrea Lucard, Medicines for Malaria Venture (MMV) ppt, 2.04Mb

Financing and R&D for neglected diseases
Role of Product Development Partnerships
First meeting of Consultative Expert Working Group on Research
and Development: Financing and Coordination –
Open Forum 6 April 2011
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Contents
• Product development partnership (PDP) model
• Example of a PDP: Medicines for Malaria Venture (MMV)
PDP model
• Leverages public and philanthropic funds
• Engages the pharmaceutical industry and academic
research institutions
• Undertaking R&D for diseases of the developing world
that they would normally be unable or unwilling to
pursue independently, without additional incentives.
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PDPs leverage the strengths of global partners to
develop new tools for neglected diseases
Public sector (early
research, funding…)
PDPs
(portfolio mngt, scientific
due diligence, create links
across academia
/industry; incentivize …)
Philanthropy
(funding, fund
raising ….)
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Private sector
(compound
libraries,
facilities,
expertise …)
The PDP model is vital to the R&D process for
neglected diseases
• Access to best science, high tech facilities, skills compound
libraries from Academia, Pharma and Biotech
– e.g. 6 million compounds screened for malaria and 25,000 new starting
points identified
• Innovative and cost-effective
• Portfolio management allows flexibility to reallocate resources
• Apply rigorous industry style practices to progress projects
through pipeline
• Shares the risk of R&D investment
• Uses funding to leverage further private sector assets,
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creating a virtuous circle
Champions role in approaching unaddressed public health
problems
Trusted “broker” working across sectors and within countries
PDPs received less than 17% of global funding for
neglected diseases in 2009
• Funding to PDPs in 2009 was
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2010 G-FINDER report
$530.0m
This represented 16.6% of global
funding, 22.5% of global grant
funding for neglected diseases
Treatments: Activities of PDPs
104 biopharmaceutical candidates in development
59
Pre Clinical
15 14%
Phase I
12
12%
Phase II
10
10%
Phase III
2%
2
Registration
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Drugs
Vaccines
Microbicides
6%
Launched
0
20
40
60
# candidates
Notes: Includes products not funded by Gates Foundation.
Biopharmaceutical candidates in development Include: IAVI, IPM, IVI, GATB, Aeras, MMV, MVI, MVP, PVS, DNDi, iOWH, PDVI, HHVI.
Source: PDPs
Slides source from:
57%
Diagnostics and vector control:
Activities of PDPs
39 candidates
Diagnostics
Vector control
Feasibility
7
Test
Development
7
Evaluation
6
Demonstration
1
26%
Early Stage
26%
In
Development
22%
4%
Country
Adoption
6 22%
0
2
4
6
5
7
0
CD4
FIND
IDRI
2
4
# candidates
8
Notes: Includes products not funded by Gates Foundation.
Biopharmaceutical candidates in development Include: IAVI, IPM, IVI, GATB, Aeras, MMV, MVI, MVP, PVS, DNDi, iOWH, PDVI, HHVI.
Source: PDPs
Slides source from:
&
IVCC
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Medicines for Malaria Venture: PDP to discover,
develop and deliver effective & affordable drugs
• Established in 1999 by WHO, World Bank, Governments, the Pharma
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Industry; subsequent funding from the Gates Foundation
Swiss Foundation based in Geneva
Largest ever antimalarial drug portfolio
Launched 1 product (with Novartis); two under regulatory review by
European Medicines Agency (with sigma-tau and Shin Poong)
Supported prequalification of IV Artesunate for severe malaria (Guilin)
Partners in ca. 130 countries
Medicines for Malaria Venture – Our Mission
MMV’s mission is to reduce the
burden of malaria in diseaseendemic countries by
discovering, developing and
facilitating delivery of new,
effective and affordable antimalaria drugs.
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Managing the MMV portfolio
• R&D priorities and Target Product Profiles evaluated and
agreed by Expert Scientific Advisory Committee (ESAC)
and published on MMV website
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Call for proposals published annually
Assessment of proposals by MMV Science Team and ESAC
Grants awarded
Projects managed on a daily basis in collaboration with
MMV scientific expert and assessed annually by ESAC
MMV Governance structures
MMV Board of Directors
The highest policy and decision-making body of MMV which ensures that MMV’s
objectives are efficiently executed by the management
MMV North America Inc. Board
Supports MMV’s Corporate Development activities in the USA
Expert Scientific Advisory Committee (ESAC)
Helps identify the best projects worthy of inclusion in the MMV portfolio and
continues to monitor progress through an annual review of all projects
Access and Delivery Advisory Committee (ADAC)
Advises MMV on its global access activities to ensure timely and effective delivery
of new antimalarial drugs in malaria endemic countries
Global Safety Board (GSB)
The Global Safety Board ensures adherence to scientific and ethical principles
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MMV global priorities
Malaria
• Increase the range of affordable quality ACTs to reduce morbidity and mortality
• Approval of quality treatment for severe malaria
Children
• Development of age-appropriate formulations to safely and effectively dose the
youngest patients
Women and pregnancy
• Intermittent Preventative Treatment during Pregnancy
• Drug-drug interaction studies to support safe use with oral contraceptives
Resistance
• New drugs with better pharmacokinetic and safety profiles and activity against
artemisinin-resistant parasites
Eradication: Transmission, relapse, long term protection
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Composition of the MMV portfolio 2011
Research
Lead Gen
Translational
Lead Opt
Preclinical
Novartis
Novartis
MK 4815
miniportfolio
2 Project
(Merck)
GSK
GSK
GNF156
miniportfolio
2 Project
Novartis
Broad/Genzyme
Aminoindole
miniportfolio
Broad/Genzyme
AN3661
Anacor
Pfizer
Aminopyridine
Screening
UCT
sanofi aventis
Pyrazoles
Orthologue screen
Drexel
AstraZeneca
Quinolones
Screening
USF/ VAMC
Kinases
DHODH
Monash
UTSW/UW/Monash
Antimalarials
St Jude/Rutgers
Other Projects
12 Projects
Phase I
NITD609
Novartis
Tafenoquine
GSK
Development
Phase IIa
OZ 439
(Monash/UNMC/
STI)
Phase IIb/III
Registration
AZCQ
DHA-Piperaquine
sigma-tau
Pfizer
Pyramax®
Shin Poong/University
of Iowa
Phase IV
Coartem®-D
Novartis
ASAQ Winthrop
sanofi aventis/DNDi
IV artesunate
Guilin
MMV Access and delivery
R&D
Scale-up
Acceptance
Outcome
Pricing (contractual)
Distribution
Pharmacovigilance
SRA1 Registration
Country of origin
WHO prequalification
National registration
Policy
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Essential Medicines List
WHO treatment guidelines(scientific)
Local guidelines / national policy
1. Stringent Regulatory Authority
Results from MMV partnerships
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55 million courses of paediatric Coartem distributed in Africa
(MMV/Novartis)
IV Artesunate WHO pre-qualified and registered in 23 countries
through (MMV/Guilin)
2 new ACTs under regulatory review; paediatric formulations in
advanced development (MMV/sigma-tau; MMV/Shin Poong)
Studies ongoing to reduce burden of malaria in pregnant women
(MMV/Pfizer)
Programmes underway to improve understanding of malaria market
dynamics (MMV/IMS/Vodaphone and Vodacom/Novartis)
Portfolio of fifty projects including potential single-dose cure, drugs
with activity against artemisinin-resistant parasites, transmission
blocking, relapse and long-acting drugs
Partners include R&D-based Pharma, Generic manufacturers, academia, nonpharmaceuticals corporations, government …..