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New Drugs for Neglected Diseases New Hope for Forgotten Patients Jana Armstrong Executive Director, DNDi North America Partnering for Cures 2009 Thursday, Dec 3, 2010
Overview Drugs for Neglected Diseases  initiative ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],DNDi:  an alternative model for drug development “ an experiment borne out of extreme frustration about health care for the poorest”
Disease Landscape Neglected Tropical Diseases (NTDs) ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],Visceral Leishmaniasis (VL) Sleeping Sickness (HAT) Chagas Disease Malaria
Disease Landscape Current Treatment Limitations Patients and Healthcare Workers Need Safe, Effective, Easy-to-Use Drugs Melarsoprol (arsenic-based IV drug)  “Fire in the Veins” Benznidazole: adult tablets fractioned or macerated for pediatric use ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object]
Disease Landscape Human African Trypanosomiasis (HAT)  or Sleeping Sickness ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object]
Disease Landscape Visceral Leishmaniasis (VL)  ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object]
Disease Landscape Chagas Disease: A Silent Killer ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object]
Market Landscape A Fatal Imbalance ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],*Source: IMS Health, 26.2.2008 Chart Data Source: Chirac P, Torreele E.  Lancet . 2006 May 12; 1560-1561.
Market Landscape Global R&D funding in 2007 Source: Moran et al., G-Finder report, 2009 Neglected Diseases   $2.5 billion (US) Kinetoplastids $125 million (US)
DNDi “Business” Plan & Organization ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],7 worldwide offices ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],Brazil India Kenya Malaysia USA DRC Japan Geneva Coordination Team + consultants
Strategy New compounds Screening of existing libraries >6 years Therapeutic switch “ Rediscovered” compounds 3-6 years New formulations (FDC) Geographical extensions Co-administration   3 years R&D Strategy DNDi  Portfolio-Building Model  Long-Term projects Medium-term projects Short-term projects
Exploratory Alternative formulations Amphotericin B  (VL) 6 to 8 new treatments 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  (Chagas) Exploratory ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],Combination therapy   (VL in Latin America)  –  in preparation NECT Nifurtimox - Eflornithine  Co-Administration Stage 2 HAT ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],Reference  screening centres: LSHTM, Swiss Tropical Institute, University of Antwerp Exploratory  ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],R&D Strategy Robust Portfolio – November 2009 a robust pipeline
Accomplishments DNDi 6-Year Results ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object]
ASAQ (Malaria) Fixed-Dose Artesunate/ Amodiaquine NECT  (HAT) Nifurtimox - Eflornithine  Co-Administration ASMQ (Malaria) Fixed-Dose Artesunate/ Mefloquine Accomplishments 3 New Treatments Delivered Farmanguinhos (Brazil) Cipla  (India) sanofi-aventis (France) National Control Programs MSF WHO 2007 2008 2009 Partners ,[object Object],[object Object],[object Object],[object Object]
Budget $187M of $410M  Secured (2003-2014)   ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object]
Budget ~$223M Still Needed for 3-5 More Treatments 2004-2014 Projected (€):
www.dndi.org By working together in a creative way,  we can bring innovation to neglected patients!

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DNDi: New Drugs for Neglected Diseases; New Hope for Forgotten Patients

  • 1. New Drugs for Neglected Diseases New Hope for Forgotten Patients Jana Armstrong Executive Director, DNDi North America Partnering for Cures 2009 Thursday, Dec 3, 2010
  • 2.
  • 3.
  • 4.
  • 5.
  • 6.
  • 7.
  • 8.
  • 9. Market Landscape Global R&D funding in 2007 Source: Moran et al., G-Finder report, 2009 Neglected Diseases $2.5 billion (US) Kinetoplastids $125 million (US)
  • 10.
  • 11. Strategy New compounds Screening of existing libraries >6 years Therapeutic switch “ Rediscovered” compounds 3-6 years New formulations (FDC) Geographical extensions Co-administration  3 years R&D Strategy DNDi Portfolio-Building Model Long-Term projects Medium-term projects Short-term projects
  • 12.
  • 13.
  • 14.
  • 15.
  • 16. Budget ~$223M Still Needed for 3-5 More Treatments 2004-2014 Projected (€):
  • 17. www.dndi.org By working together in a creative way, we can bring innovation to neglected patients!

Notas do Editor

  1. Primeiramente gostaria de parabenizar os organizadores pelo evento e agradecer a inclusão no programa de mini-conferências dando fala a novas iniciativas no âmbito da Doença de Chagas. Como já discutido na abertura por Tânia Araújo Jorge, Paulo Gadelha, assim como Prof. João Carlos Pinto Dias, devemos sim celebrar o centenário deste marco na histõria da ciência e da saúde brasileira que foi a descoberta da Doença de Chagas e comunicação ao mundo científico em 1909, assim como os progressos alcançados nos últimos anos, em particular o sucesso do controle vetorial. No entanto, é preciso seguir discutindo e agindo para preencher as lacunas existentes no conhecimento da Doença de Chagas, assim como a necessidade ampla utilização das ferramentas hoje disponíveis para seu diagnóstico e tratamento, assim como a necessidade de desenvolvimento de novas ferramentas para diagnóstico e tratamento do Mal de Chagas. Os gargalos existentes na Doença de Chagas, são comuns a várias das doenças negligenciadas, em especial as doenças de Tipo III, e resultam de falhas de mercado quanto de políticas públicas. Entre falhas de mercado, temos o d esenvolvimento de medicamentos enormemente concentrado na indústria farmacêutica com P&D direcionada por lucros, onde os mais pobres s ã o negligenciados. Em paralelo, temos políticas públicas que não corrigem este desequilíbrio. Vivemos um momento de mudança no entanto, com aumento no investimento em P&D para estas doenças, principalmente de países como o Brasil, discussão de novos mecanismos de financiamento e incentivo para pesquisa e desenvolvimento e criação de novas iniciativas, como parcerias para desenvolvimento de produtos. Como uma das iniciativas para lidar com as limitações existentes no diagnóstico e tratamento e com gargalos existentes na P&D para doenças negligenciadas, a DNDi foi criada em 2003.