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