can a global framework on research & development (r&d ... · the cipla-mrc collaboration...
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Can a Global Framework on Research & Development (R&D) overcome existing challenges for access to
treatment?
Access to Treatment: An Integrated Response
Geneva 28 May 2013
P. Boulet, Head of Policy Affairs
Brazil
India
Kenya
Malaysia
USA
DRC
Japan
Geneva Headquarters
7 worldwide offices
Patient Needs-Driven & Innovative R&D Model
Founding Partners• Indian Council for Medical
Research (ICMR)
• Kenya Medical Research Institute (KEMRI)
• Malaysian MOH
• Oswaldo Cruz Foundation, Brazil
• Médecins Sans Frontières (MSF)
• Institut Pasteur France
• TDR (permanent observer)
Malaria Leishmaniasis
Sleeping Sickness (HAT) Chagas Disease
Paediatric HIV
Helminth infections
Responding to the Needs of Patients Suffering from Neglected Diseases…
Neglected Diseases: Current Treatment Limitations
We Need Safe, Effective, Easy-to-Use Drugs
Melarsoprol Eflornithine
Ineffective (resistance) Toxic Expensive Painful when delivered Difficult to use Not registered in
endemic regions Restricted by patents
Vision:
A collaborative, patients’ needs-driven, virtual, non-profit drug R&D organisation to develop new treatments against the most neglected communicable diseases
Vision & Objectives
Objectives: Deliver 11 to 13 new treatments by 2018 for sleeping
sickness, Chagas disease, leishmaniasis, malaria, paediatric HIV and specific helminth infections
Establish a robust pipeline for future needs Use and strengthen existing research capacity in disease-
endemic countries
≅ 30
≅ 40
≅ 300
≅ 100
≅ 90
≅ 55
≅ 15
Dedicated Teams WorldwideOver 630 People Committed to DNDi’s Vision
A Global Network to Leverage ResourcesMore Than 100 R&D Partners
Balance of public and private partnerships worldwide
Oct. 2012
Easy to Use Affordable Field-Adapted Non-Patented
6 New Treatments Developed Since 2007
Discovery Pre-clinical Clinical Implementation
Nitroimidazole backupNitroimidazole backup
Oxaborole backupOxaborole backup
Scr
een
ing
&
Lead
Op
tim
isati
on
VL-2098 (VL)VL-2098 (VL)
Nitroimidazole backup (VL)
Nitroimidazole backup (VL)
Fenarimol seriesFenarimol series
K777K777
Flubendazole -MacrofilaricideFlubendazole -Macrofilaricide
Improved PI for 1st-lineImproved PI for 1st-line
Fexinidazole Phase 2/3Fexinidazole Phase 2/3
Oxaborole SCYX-7158Oxaborole SCYX-7158
New VL treatments – Bangladesh
New VL treatments – Bangladesh
New VL treatments – Africa AmBisome® - MiltefosineNew VL treatments – Africa AmBisome® - Miltefosine
New VL treatments – (Latin America)
New VL treatments – (Latin America)
HIV / VL HIV / VL
Azoles E1224 & BiomarkersAzoles E1224 & Biomarkers
Benznidazole Paed. Dosage form
Benznidazole Paed. Dosage form
NECT (Stage 2 HAT) Nifurtimox - Eflornithine
Co-administration
NECT (Stage 2 HAT) Nifurtimox - Eflornithine
Co-administration
SSG&PM co-administration
VL in Africa
SSG&PM co-administration
VL in Africa
New VL treatments in Asia
(SD AmBisome®,3 drug combinations)
New VL treatments in Asia
(SD AmBisome®,3 drug combinations)
✓ : NCE
✓✓
✓
✓
✓✓
✓
✓HATHAT
Leish.Leish.
ChagasChagas
HelminthsHelminths
Paed. HIVPaed. HIV
ASMQ Fixed-Dose Artesunate/ Mefloquine
ASMQ Fixed-Dose Artesunate/ Mefloquine
ASAQ Fixed-Dose Artesunate/ Amodiaquine
ASAQ Fixed-Dose Artesunate/ AmodiaquineMalariaMalaria
Anfoleish (CL)Anfoleish (CL)
NitroimidazoleNitroimidazole
✓
‘Superboosting’ – TB/HIV‘Superboosting’ – TB/HIV
PI sprinkles (CHAPAS-2)PI sprinkles (CHAPAS-2)
✓
Fexinidazole (VL)Fexinidazole (VL)✓
✓
DNDi Portfolio: A Mix of Existing Drugs & NCEs
Dec. 2012
✓
Paediatric HIV: Most Urgent Treatment Needs (TPP)
Formulations/regimens that are simple, easy to administer, and more tolerable (once daily or less, heat-stable, dispersible/sprinkles, tolerable taste)
Durable (forgiving and minimal requirement for repeated immunological or virological testing; minimal risk for developing resistance)
Suitable for infants (< 2 mos - 3 yrs) TB treatment compatible Affordable
Innovative PI Formulation:The Cipla-MRC Collaboration
LPV/r sprinkles by Cipla*
CHAPAS-2: Pharmacokinetics and acceptability of
sprinkle formulation compared with syrup/tablets**
Sprinkles preferred: better to swallow, store, transport;
important advantage for caregivers 71% (<1 y.o.) chose to continue sprinkles over syrup after study
Inspired DNDi, leading to the concept of “4-in-1” sachet
* http://www.retroconference.org/2012b/PDFs/982.pdf** http://www.controlled-trials.com/isrctn/pf/01946535; 4th Pediatric HIV Workshop, 2012 DC
Bring a “4-in-1” Sachet to Patients: DNDi-Cipla Collaboration on Product Development & Access
For illustration only
• Address the need for a PI-based first-line ARV FDC• Adaptable for use in treating TB co-infected children
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
All patients in need shall have access to the drug at the lowest sustainable price in all endemic countries
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
Negotiating & managing IP to ensure access to patients & access to knowledge
13
Delinking R&D costs from product price to ensure access: DNDi practice
Target price in ‘target product profile’ (TPP) e.g. ideally < 50 USD/patient/year (consistent with adult ART),
acceptable if lower than current price of products used separately
Contractual commitment of manufacturer to make final product available at cost, plus a minimal margin, in all endemic countries
No patent or non-exclusive licenses to enable competition, technology transfer and sharing of knowledge
No intent to recoup R&D investments
But many challenges remain
No major scientific breakthrough (NCE) Funding gap for development of pipeline
candidates R&D priorities set by few donors Major regulatory and access challenges Lack of incentive for private sector (beyond CSR) Limited leadership and involvement of endemic
countries Lack of overall coordination – WHO role?
A dynamized critical mass of
Neglected Disease players
WHO Big
Pharma
& Biotechs
Generics & Pharma
New Funding Mechanisms PPPs & PDPs
Philanthropy
Public institutions in endemic countries
Governments
From individual successes to sustainable change
Global framework for R&D Define priorities for medical innovation New mechanisms for sustainable funding New incentives for neglected diseases R&D Promote & incentivize open innovation Ensure affordable & equitable access Coordination mechanisms Strengthening capacity & promoting technology transfer
With leadership and spearheading of endemic countries
Thank You to All Our Partners & Donors
www.dndi.org
via the 4th Sector Health Project implemented by Abt Associates, Inc.
by
www.connect2fightneglect.org