Transcript Novartis’ Commitment to Neglected Diseases – NITD

Funding Full Development
of the Emerging Pipeline in Tb
Paul L Herrling
Head of Corporate Research
Novartis International AG
Geneva, 11 April 2008 MSF
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THE DRUG DISCOVERY PROCESS: phases
target validation
D0
D1
D2
D3
D4
PhI
PhII-IV
O
D0: basic science
target discovery
D4: final preparation for human
testing, PK,ADME, tox,
D1: assay development
HTS: high throughput screening
PhI: Proof of concept/mechanism
in man; tolerance in human
D2: HTS ligand finding
proprietary libraries,
commercial libraries
PhII-III: dose finding, efficacy,
registration studies
PhIV: post marketing studies
D3: lead optimization
medicinal chemistry
animal models
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Historic funding has built a portfolio that is moving towards
clinical trials
…has created the pipeline of today
Historic funding . . .
R&D Funding Received – PDPs (USD M)
447
Private
Chagas
450
HAT
Entities
VL
3%
400
Gov
21%
PDP Compounds by Disease – including industry **
12
12
Malaria
11
TB
10
VL
5
350
9
TB
300
HAT
9
Chagas
8
8
Clinical
funding
needs
7
250
2
6
200
Private
Non Profits
150
5
6
4
5
5
76%
2
4
3
Malaria
3
100
Other
4
4
2
2
1
4
1
2
3
50
1
*
0
1999-2007
- by
disease
1996-2005
1
3
1
2
2
1
1
1
1
1
0
Early Lead id Lead Preclini Phase 1Phase 2Phase 3 Reg Phase 4
stage
op
cal
D0/1
D2
D3
D4
*Chagas, 17
HATJuly
& VL are
sourced from DNDi through 2006; Does not include IOWH **Some projects have not been included in graph due to uncertainty of stage in pipeline
2015
Sources: DNDi Business Plan, "Independent Review MMV" DIFID, MMV Annual Report 2006, Focus on Finances MMV.org; The New Landscape of Neglected Disease Drug
Development, Wellcome Trust (2005); "TB DRUGS: Where We Are Today, The Vision For Tomorrow" CEO GATB (Jan 2007); WHO Draft “Global plan of action on public 3
health innovation and intellectual property” (August 2007)
THE DRUG DISCOVERY PROCESS: phases
target validation
D0
D1
D2
D3
D4
PhI
PhII-IV
O
Existing pipeline, already financed
not financed
EXPENSIVE
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Funding needs – drugs - Underestimate
100%
90%
80%
70%
Funding needs for Neglected diseases R&D drugs – 2008-2017
USD B
4.4
5.9
0.9
0.2
Total
10.3
1.1
Other diseases
2.0
0.4
HAT/ VL/ Chagas
0.6
1.3
Malaria
1.7
3.1
TB
6.0
0.4
60%
50%
40%
30%
2.9
20%
10%
0%
Discovery
Development
Sources: Global plan to stop TB 2006-2015; Tuberculosis research and development: a critical analysis, Treatment Action Group; MMV financial plan; DNDi
Business plan 2007-2014; The new landscape of neglected disease drug development, Wellcome trust; “How much does it cost to develop a new drug?” 5
Biomedical Industry advisory group; Dalberg interviews and analysis
Portfolios are fragmented across players – even in key
diseases
100%
39
54
5
16
12
Novartis
Public
90%
GSK 1
80%
70%
Sanofi-aventis 5
Public
Public
TDR 1
GSK 1
Public
2
Public
24
Pfizer 1
TDR 1
60%
Sanofi-aventis
1
Astrazeneca 3
50%
40%
TDR 2
Pfizer
1
DNDi 11 DNDi 6
Other 12
DNDi 3
20%
TDR 1
10%
GATB 11
IOWH
1
DNDi 2
IOWH 1
Malaria
IOWH 1
TB
• Fragmentation
between Industry
Public, and
PDPs for
malaria and TB
• Fragmentation
between public
and PDPs for
smaller diseases
MMV 24
30%
0%
3 3 Public
Chagas
HAT
VL
Other
Dengue
Public Only
Industry Only
PDPs
Notes: Number of public projects is estimated based on interviews and research; Surface area does not depict size of projects, which vary significantly by
stage of discovery and development
Source: The New Landscape of Neglected Disease Drug Development, Wellcome Trust (2005); TB Alliance Portfolio.ppt (October 2007);
(www.ifpma.org/clinicaltrials); Industry R&D for Diseases Primarily Affecting Developing Countries (company response to IFPMA survey);
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Working Group on New TB Drugs (Oct 2006)
Improved management of the neglected disease
portfolio a key challenge
The emerging portfolio of neglected disease compounds is
fragmented across PDP, industry and public entities – with limited or
no coordination and oversight
Improved approaches to portfolio management will be key to making
the case for the expanded financing. The techniques and experience
of pharmaceutical companies will be key in this respect
Prioritization of funding across diseases is going to be necessary,
even in a scenario with sufficient funding. This is politically sensitive
area, some donors are hesitant to delegate this responsibility
The portfolio management challenges are still less well recognized
17 July 2015
among the product development partners. However, there is a broad
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FUND FOR NEGLECTED DISEASES R+D
Board [strategy, diseases, tools]
Funders: current + new
$
ie BMGF, WT etc plus Developed countries governments royalties,
as well as Developing world governments
milestones
$$$
Portfolio management Team
Medical, Scientific, Technical Professionals
(fund only to next go/no go decision)
projects and
exclusive licenses to
neglected disease
IP
Academia
PPPs
money for development
to next stage
Industry
In case of profits made using data paid by fund:
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Alternative model – Neglected Diseases Fund
1. Create a major pool of funds by mechanisms similar to Global Fund, GAVI,
International Finance Facility fed by Governments, both of developed and
developing world (latter must contribute as they are responsible for the health of
their citizens ), charities (Welcome, BMGF, others)
- Major problem might not be finding money but how to make it efficiently
available to entities discovering and developing medicines for neglected
diseases
2. Establish a portfolio management committee nominated by funders but fulfilling
stringent professional criteria. Their task would be to evaluate submitted projects
and fund the selected ones to the next decision point in development (phase
transitions), monitor the progress and make funding decisions for next phases as
well decide development strategy
- This is identical to the process currently used in Pharma, the same decision
criteria (scientific, technical feasibility, medical need, target product profile etc.)
should be used with the exception of commercial viability.
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3. Companies alone or in conjunction with PPPs who want to access these
funds would protect their IP (see why below) BUT would legally and
bindingly commit to make their products available at cost for poor patients
and where no markets exist as a condition to obtain funds from the pool
(exclusive license to fund)
4. Wherever markets allowing returns for these products emerge as is
eventually to be expected, WHOEVER sells the products in these markets
will commit to pay royalties on those sales back into the pool for
refinancing if they used Fund financed data. The IP protection mentioned
above is needed to allow this mechanism as it incentivises and protects
the innovators' investment (including the pool entity)
This mechanism might address the gray zones where the current returns
are insufficient today for big Pharma to invest, but that do need big Pharma
contributions
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Neglected Diseases Fund Model
This draft of a model might be sufficiently attractive to Pharma companies
as some of them have already shown their interest to contribute to the
access to medicines problem. It does not threaten the IP protection
essential for innovation. This model would remove a major hurdle to their
investment in the R&D of neglected diseases, short term they would gain in
reputation by contributing, long term (very) new markets may emerge
This model might be combined with others such as advanced market
commitments, for late stage developments e.g a vaccine /medicines in late
Phase 3 clinical trials
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