Post on 24-Dec-2015
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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
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
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Philanthropy (funding, fund
raising ….)
Philanthropy (funding, fund
raising ….)
Public sector (early research, funding…)Public sector (early research, funding…)
Private sector (compound
libraries, facilities,
expertise …)
Private sector (compound
libraries, facilities,
expertise …)PDPs
(portfolio mngt, scientific due diligence, create links
across academia /industry; incentivize …)
PDPs(portfolio mngt, scientific due diligence, create links
across academia /industry; incentivize …)
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, 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 $530.0m
• This represented 16.6% of global funding, 22.5% of global grant funding for neglected diseases
6 2010 G-FINDER report
Treatments: Activities of PDPs104 biopharmaceutical candidates in development
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
59Pre Clinical
15Phase I
12Phase II
10Phase III
2Registration
6Launched
Drugs
Vaccines
Microbicides
# candidates
10%
12%
14%
57%
6%
2%
Slides source from:
Diagnostics and vector control: Activities of PDPs39 candidates
7Feasibility
7Test
Development6Evaluation
1Demonstration
6Country
Adoption
CD4FIND
IDRI
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
4%
22%
26%
22%
Early Stage
In
Development
IVCC
# candidates
Diagnostics Vector control
26%
Slides source from:&
Medicines for Malaria Venture: PDP to discover, develop and deliver effective & affordable drugs
• Established in 1999 by WHO, World Bank, Governments, the Pharma 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 disease-endemic countries by discovering, developing and facilitating delivery of new, effective and affordable anti-malaria 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
• 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
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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
RegistrationPreclinicalResearch Translational Development
Lead Opt Phase IIaPhase ILead Gen Phase IVPhase IIb/III
Novartisminiportfolio
Novartis2 Project
MK 4815(Merck)
IV artesunateGuilin
DHA-Piperaquine sigma-tau
Coartem®-DNovartis
GSKminiportfolio
Broad/Genzymeminiportfolio
AntimalarialsSt Jude/Rutgers
Other Projects12 Projects
sanofi aventisOrthologue screen
KinasesMonash
PfizerScreening
GSK2 Project
NITD609Novartis
OZ 439(Monash/UNMC/
STI)
AZCQPfizer
Pyramax®Shin Poong/University
of Iowa
ASAQ Winthropsanofi aventis/DNDi
AminoindoleBroad/Genzyme
AminopyridineUCT
QuinolonesUSF/ VAMC
PyrazolesDrexel
AstraZenecaScreening
GNF156Novartis
DHODHUTSW/UW/Monash
AN3661Anacor
TafenoquineGSK
MMV Access and delivery
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R&D
SRA1 RegistrationCountry of origin
WHO prequalification
National registration
Scale-upAcceptance
Outcome
Pricing (contractual)
Distribution
Pharmacovigilance
PolicyEssential Medicines List
WHO treatment guidelines(scientific)
Local guidelines / national policy1. Stringent Regulatory Authority
Results from MMV partnerships
• 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, non-pharmaceuticals corporations, government …..