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Financing and Coordination of R&D for neglected diseases: Challenges and opportunities Consultative Expert Working Group on Research and Development Open.

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Presentation on theme: "Financing and Coordination of R&D for neglected diseases: Challenges and opportunities Consultative Expert Working Group on Research and Development Open."— Presentation transcript:

1 Financing and Coordination of R&D for neglected diseases: Challenges and opportunities Consultative Expert Working Group on Research and Development Open Forum 6 April 2011 WHO, Geneva Dr. Bernard Pécoul Executive Director, DNDi

2 Best Science for the Most Neglected Source: Chirac P, Torreele E. Lancet May 12; A Fatal Imbalance Tropical diseases: 18 new drugs (incl. 8 for malaria) Tuberculosis: 3 new drugs 1.3% 21 new drugs for neglected diseases 98.7% 1,535 new drugs for other diseases ( ) Tropical diseases (including malaria) and tuberculosis account for: 12% of the global disease burden Only 1.3% of new drugs developed

3 Best Science for the Most Neglected 7Feasibility 7 Test DevelopmentTest DevelopmentTest DevelopmentTest Development 6Evaluation 1Demonstration 6 Country AdoptionCountry AdoptionCountry AdoptionCountry Adoption CD4 FIND 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% 104 biopharmaceutical candidates in development and 39 diagnostic & vector control candidates 59Pre Clinical 15Phase I 12Phase II 10Phase III 2 Registration 6Launched Drugs Vaccines Microbicides # candidates 10% 12% 14% 57% 6% 2% Early Stage In DevelopmentIn DevelopmentIn DevelopmentIn Development IVCC # candidates Diagnostics Vector control 26% Source from: & Pipeline now begins to be filled 143 candidates

4 Best Science for the Most Neglected Brazil India Kenya Malaysia USA DRC Japan Geneva Coordination Team + consultants 7 Founding Partners Indian Council for Medical Research (ICMR) Kenya Medical Research Institute (KEMRI) Malaysian MOH Oswaldo Cruz Foundation Brazil Medecins Sans Frontieres (MSF) Institut Pasteur France WHO/TDR (permanent observer) 7 worldwide offices DNDi A patient needs driven & innovative R&D model Deliver new treatments by 2014 for sleeping sickness, Chagas disease, leishmaniasis and malaria Establish a robust pipeline for future needs Use and strengthen existing capacity in disease-endemic countries

5 Best Science for the Most Neglected DNDi portfolio: 100m spent since 2003 Exploratory Alternative formulations of Amphotericin B (VL) Drug combination (Chagas) Oxaborole (HAT) Nitroimidazole backup (HAT) Fexinidazole (HAT) ASAQ (Malaria) Fixed-Dose Artesunate/ Amodiaquine ASMQ (Malaria) Fixed-Dose Artesunate/ Mefloquine Combination therapy (VL in Asia) Paediatric benznidazole (Chagas) Azoles E1224 & Biomarker (Chagas) Exploratory Combination therapy (VL in Africa) AmBisome® Miltefosine Combination therapy (VL in Latin America) NECT (Stage 2 HAT) Nifurtimox - Eflornithine Co- Administration HAT LO Consortium - Scynexis - Pace Univ. Chagas LO Consortium - CDCO - Epichem - Murdoch Univ. - FUOP VL LO Consortium - Advinus - CDRI Major Collaborators: - Sources for hit and lead compounds: GSK, Anacor, Merck, Pfizer, Novartis (GNF, NITD), TB Alliance,… - Screening Resources: Eskitis, Institut Pasteur Korea, Univ. Dundee,… - Reference screening centres: LSHTM, Swiss Tropical & Public Health, University of Antwerp Discovery Activities: - Compound mining - Chemical classes - Target-based - Screening SSG&PM Combination therapy (VL in Africa ) K777 (Chagas) Nitroimidazole (VL) a robust pipeline 6 to 8 new treatments by 2014

6 Best Science for the Most Neglected Average Cost to Develop One Drug – The Pharmaceutical Industry Data (in $ Million) Based on this model, DNDi would have to raise billions to accomplish its goals. Through effective partnerships, we are able to bring the costs down. *Source: PhRMA Pharmaceutical Industry Profiles 2007

7 Best Science for the Most Neglected How much will R&D cost for neglected diseases? + a robust pipeline 1 billion 1 drug 100 million Pharma = SSG&PM Sodium Stibogluconate & Paromomycin Combination Therapy VL in Africa NECT Nifurtimox - Eflornithine Co-Administration Stage 2 HAT ASAQ (Malaria) Fixed-Dose Artesunate/ Amodiaquine ASMQ (Malaria) Fixed-Dose Artesunate/ Mefloquine =

8 Best Science for the Most Neglected Main policy challenges Challenge 1: IP and open innovation Challenge 2: Overcoming regulatory barriers Challenge 3: sustainable financing and new incentives for R&D

9 Best Science for the Most Neglected DNDi agreements with pharmas, biotechs, PDPs: Merck Pfizer GSK sanofi-aventis Anacor TB Alliance Others in negotiation…. Quality compounds sourcing Access to focused knowledge and data => Accessing proprietary compounds to jumpstart discovery Access to compound librairies Challenge 1

10 Best Science for the Most Neglected Need for more open innovation and sharing of knowledge Nitroimidazole compounds developed by TB Alliance showed great promise for leishmaniasis treatment –Grant DNDi royalty free license to develop new compounds –sharing of scientific expertise and specific knowledge Synergy between two PDPs – collaboration to benefit patients –avoid duplication –saving costs –speeding up R&D process –stimulate innovation Challenge 1

11 Best Science for the Most Neglected Negotiating freedom to operate… paving the way for equitable access Royalty-free sub-licensable licenses Licenses for R&D and manufacture: world-wide Licenses for distribution and sale: all endemic regions, without exclusion Sales on the public sector: at cost plus (lowest sustainable price) Sales on the private sector: possible margins but linked to partners financial contribution Limited confidentiality: make freely available all information generated about the product during its development (publications, databases, etc.) Challenge 1

12 Best Science for the Most Neglected Innovative partnership with sanofi-aventis DNDi formulation out-licensed to s-a WHO prequalified Registered in 28 sub-Saharan countries + India Public price: at cost < US$1 for adult, US$0.50 for children Over 80 million treatments distributed in Africa Next step : Transfer of technology to an additional African industrial partner © MSF ASAQ An innovative unpatented anti-malarial FDC

13 Best Science for the Most Neglected Affordable treatment and equitable access to patients in need Delinking the costs of R&D from the price of products DNDi activities not financed by IP revenues No partnership without overcoming IP barrier Develop drugs as public goods, when possible Disseminate the results of DNDi work Encourage open publication of research data and technology transfer Decisions regarding ownership of patents and licensing terms made on a case-by-case basis IP & open innovation: DNDi vision Challenge 1

14 Best Science for the Most Neglected JS Dr. Jannin, WHO Overcoming regulatory barriers Majority of treatments submitted for approval in Africa: -first approved by EMA/US FDA, or -generic drugs New Chemical Entities (NCEs), vaccines, combination treatments now being developed to respond to the specific needs in endemic countries African regulatory agencies will have to perfom regulatory assessment of new treatments never evaluated before How can this be achieved the most efficiently? Challenge 2

15 Best Science for the Most Neglected Need for new pathways for registering innovative drugs for Africa Increased participation of endemic countries within existing mechanisms Regional centres of excellence to support strengthening of African regulatory agencies Challenge 2

16 Best Science for the Most Neglected Sustainable financing & incentive for R&D Combined PDP pipeline include 143 Candidates BUT Sustainable funding not secured for expensive clinical trials New incentives needed to replenish pipelines with new compounds Global framework needed to ensure public health & access oriented R&D Challenge 3

17 Best Science for the Most Neglected PUSH and PULL mechanisms for stimulating R&D on neglected diseases

18 Best Science for the Most Neglected Sustainable funding for product development and access Funding needed for large efficacy trials, manufacturing scale-up, registration, delivery, and access Model: UNITAID airline ticket tax Other indirect tax proposals: European tax on financial transactions Digital tax, mobile phone tax, etc Possible benefits New sources of funds Ensures predictability required for long-term planning clinical development Stable & subsidized market through interaction with international financing organizations Faster registration, adoption, and delivery through interaction with WHO & international procurement agencies Challenge 3

19 Best Science for the Most Neglected Pilot milestone prizes Would pay a substantial reward ( 5-20M) for specific steps in the discovery process or for clinical drug candidates that meet specific criteria Possible benefits Replenish pipeline by motivating new actors such as biotechs Vehicle to engage endemic countries as partners Pay only for success (unlike conventional push funding) Incentive to collaborate with PDPs IP management to ensure access Source of funds Usual donors: OECD governments, Foundations Endemic country governments Challenge 3

20 Best Science for the Most Neglected Need for a global framework for R&D coordination Central role of WHO defining priorities treatment recommendations Extension of Prequalification to NTD Endemic countries involvement R&D partners new funders Identifying needs Access oriented IP management delinking R&D costs from final price Technology transfer to strengthen capacity New products as Public goods


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