Ricerca e sviluppo per le malattie dimenticate - e-learning

Report
Ricerca medica indipendente
Nicoletta Dentico
Health Innovation in Practice (HIP)
La Sapienza, Roma, 4 aprile 2014
Accesso ai Farmaci Essenziali
OMS, 1977: il TRS 615
• TRS (Technical Report Series) 615, destinato
a fare storia come best seller dell’OMS, ben
presto diventa uno degli snodi esemplari del
rapporto tra salute e sanità, pubblico e
privato, economia e diritto: IL PROGRAMMA
DEI FARMACI ESSENZIALI
• Il significato della parola ESSENZIALE, che
rimanda, senza specificare, a radici, progetti,
obiettivi da raggiungere
la rivoluzione pacifica dell’OMS
Sono essenziali quei farmaci che sono di più grande
importanza, di base, indispensabili, necessari pe ri
bisogni di salute della maggioranza delle popolazioni
dei diversi Paesi, a costi, e con condizioni di
approvvigionamento, che siano compatibili con
un’accessibilità corrispondente a quella auspicabilenecessaria per identificare, definire e farsi carico dei
problemi-bisogni per I quali I farmaci hanno
un’indicazione adeguatamente documentata
 La Lista dei Farmaci Essenziali (1978)
Il 10/90 GAP
• The 1990 Commission on Health Research for
Development made the first estimates of
worldwide spending on health research and
development (US$30.0 billion) and in analysing
the flows of resources described what became
known as the “10/90 gap” – capturing the
inequality revealed in their estimate that: less
than 10 per cent of the global budget for health
R&D was being spent on 90 per cent of the
world’s health problems
Non solo malattie infettive
Le morti nel mondo per cancro
Decenni di enormi progressi nel campo
della scienza e della tecnologia
>50 y of parasitology:
targets, inhibitors,
cytotoxic compounds
Migliaia di
pubblicazioni, ma
solo pochi farmaci
contro le malattie
tropicali
Quali farmaci per le malattie tropicali
dimenticate?
• Non efficaci
(resistenze)
• Tossici
• Costosi
• Di dolorosa
somministrazione
• Difficili da
monitorare
• Non adatti ai sistemi
sanitari dei paesi
poveri
• Non registrati nelle
regioni endemiche
• Restrizioni dovute
alla protezione
brevettuale
La ricerca per le malattie
dimenticate nel 2000: il
deserto
Organizations
Activities
Specific
TDR
Unspecified
GSK
WRAIR
CHALLENGE 4
CHALLENGE 4
Launch &
Launch &
utilization
utilization
CHALLENGE 3
CHALLENGE 3
Registration
Phase 3
Phase 2
Phase 1
CHALLENGE 2
Regulatory toxicity
& safety
In vivo efficacy
Lead optimization
CHALLENGE 1
Lead identification
Screening
Visceral
Leishmaniasis
Cutaneous
Leishmaniasis
HAT
Chagas
2007 Drug R&D Landscape
for NTDs
Organizations
Activities
TDR
Specific
CDND
TB Alliance
NITD – PC dengue, TB
IOWH
MMV
Unspecified
CHALLENGE 4
Launch &
utilization
GSK
DNDi
[email protected]
Sandler Center
WRAIR
CHALLENGE 3
Registration
Phase 3
Phase 2
HAT
Chagas
Visceral
Leishmaniasis
Cutaneous
Leishmaniasis
Dengue
Malaria
TB
Buruli
Ulcer
Intestinal
helminth
Phase 1
CHALLENGE 2
Regulatory toxicity
& safety
In vivo efficacy
Lead optimization
CHALLENGE 1
Lead identification
Screening
MSF
http://www.dndi.org/index.php
BONO
250 PPPH
WEF
PHA
World Bank
Critical Challenges
for Product Development Partnerships
Discovery
Lead
Lead
Screening selection optimisation
Pre-Clinical
Challenge 2
AVAILABLE
to patients
Clinical
Challenge 3
Challenge 5
Challenge 4
Challenge 1
Optimise
Leads
Generate
Leads
Pragmatic ID
and selection
of candidates
from existing
compounds
Clinical Trial
Capacity to Test
New Drugs or
Regimens
Delivering
products to
neglected
populations
Ensure
Sustainability
The benefit of using global networks: creative
harnessing of fragmented knowledge
DNDi
Sharing of experiences between
South-South LEAP scientits
CIPIH Report http://www.who. int/intellectualproperty/en/
Diagnosi e raccomandazioni dell’OMS per garantire la
ricerca medica essenziale
“Innovation is pointless in
the absence of favourable
conditions for poor people
in developing countries to
access existing, as well as
new, products”
“The market is not a
determinant of value “
Il valore aggiunto delle Product
Development Partnerships (PDPs)
PDPs possono fungere da motore, in termini di leadership
scientifica;
PDPS hanno un ruolo determinante nella identificazione
delle piste di R&D da seguire, e per natura possono più
facilmente superare eventuali strozzature nel processo di
favorire l’accesso ai farmaci ai pazienti che ne hanno bisogno
PDPs reppresentano una nuova opportunità affinché big
pharma re-focalizzi la sua ricerca verso i bisogni dei paesi in
via di sviluppo”
PPPs sono una nuova ed efficace strategia per portare avanti
la ricerca che serve ai paesi impoveriti, sfruttando la
diversità degi attori che riescono ad agggregare.
Il valore aggiunto delle PDPs
PDPs can provide the drive and the scientific
leadership + have a crucial role in identifying R&D
pathways and overcome bottlenecks to get
products to those in need
PPPs represent a new opportunity for big pharma to
re-focus their research for developing countries
PPPs a new, effective and important means to
pursue R&D relevant to developing countries,
making use of a diversity of new players
After 15 years (and close to 1 billion US$):
• Combined PDP pipeline including 122 candidates
– “Ripple effect”: R&D Capacity building, Developing country markets less opaque,
Incentive for local industry, Regulatory networks
Challenges to build innovation and research
capacities in Africa
• Public leadership to define R&D priorities
• Significant investment with sustainable
funding
• Ensure better access to knowledge and
promote innovative IP management policies
• Enable more adapted regulatory environment
• Transfer technology and strengthen research
capacities in developing countries
Challenge to conduct clinical trials
in very difficult settings
• Access to Sites
• Status of Infrastructure
TB
GP
• Staff Limitations
JS
Dr. Jannin,
Jannin, WHO
WHO
Dr.
Key question: How much is funding source determining R&D agenda / priority
setting? Role of endemic countries?
How much are developing countries in
the driving seat?
•Notion that PDPs are global organizations with equal representation from all
regions is FALSE in most cases
•Mision & Vision of PDPs is set in north with marginal input from south
•“Nature of work, scope and budget decided by head offices not African researchers:
executive decision-making remains outside Africa”
•“....the current PPPO paradigm is fundamentally neocolonial in structure and
operation and this needs to be revisited and addressed”
At the end of the 90s a new “North Focused” architecture for R&D for
health products for Neglected Diseases disproportionately affecting
developing countries begins to be shaped
Regulatory approval and standard
FDA, EMA, Swissmedic, etc
setting
Translational
research and
developoment
Funding
Knowledge
generation:
Big Pharma
Corporate & Private
Pro duct
Development
Partnerships
(PDPs)
Philanthropic and
International
development
organizations
Public R&D institutions and academia
“London Declaration on Neglected
Tropical Diseases: Ending the Neglect &
Reaching 2020 goals” (30 Jan.2012)
Promoters:
Abbot, AstraZeneca, Bayer, Becton Dickinson,
Bill & Melinda Foundation, Bristol-Myers
Squibb, DNDi, Esai, Gilead, GlaxoSmithKline,
Johnson & Johnson, Merck KGaA, MSD, Mundo
Sano, Novartis, Pfizer, Sanofi, UKaid, USAID,
World Bank
http://unitingtocombatntds.org/
E l’OMS?
• Accelerating work to overcome the global
impact of neglected tropical diseases. A
roadmap for implementation,
Geneva, WHO, March 2012
• Sustaining the Drive to Overcome the Global
Impact of Neglected Tropical Diseases: Second
WHO Report on Neglected Tropical Diseases,
Geneva, January 2013,
http://www.who.int/neglected_diseases/2012rep
ort/en/
Quale impatto?
• Overall, the number of drugs shipped to endemic
countries has increased nearly thirty-six percent over
2011 (995 million) with the amazing donation of
nearly 1.35 billion in 2013;
• In the past two years, 74 countries — roughly twothirds of all NTD-endemic countries — have
developed integrated, comprehensive national
NTD plans.
Fondo AIFA per la ricerca indipendente
Quale la situazione degli studi clinici non-commerciali in
Italia?
I dati dell’ultimo Rapporto dell’AIFA segnalano che la
percentuale di ricerca no-profit sul totale della ricerca sui
farmaci in Italia, è passata dal 41,4% del 2008 al 35,7% nel
2010, fino a raggiungere un 32,3% nel 2012. E così dai 364
studi registrati nel 2008 si è arrivati a 225 nel 2012. Circa il
38% in meno.
http://www.agenziafarmaco.gov.it/it/content/la-ricerca-indipendente-sui-farmaci-0
Open Source Drug Discovery (OSDD), India:
democratising research against Tuberculosis
OSDD(www.osdd.net ) : a global initiative driven by the
Indian Council of Scientific and Industrial Research (CSIR)
to provide affordable healthcare to the developing world.
Set up in September 2008 as a platform for collaborative
research, with the idea to offer a scientific venue where
the best minds – scientists, doctors, research
professionals, research organizations, software
professionals, pharmaceutical enterprises and others with
diverse expertise - can collectively endeavour to solve the
complex problems associated with discovering novel
therapies bacterial pathologies like TB
Open Source Drug Discovery (OSDD), India:
democratising research against Tuberculosis (2)
• the financial commitment of $ 38 million granted by the
Indian government, OSDD is one of the few public-funded
open source initiatives dedicated to finding new affordable
treatments for tuberculosis.
• The underlying vision of the OSDD platform was steered by
the urgent need to replicate and test in the field of
healthcare and medical science the success of open source
models used in information technology and
biotechnology, by initiating a similar open source model
for drug discovery as a potentially competitive alternative
to traditional closed propriety approach . See in this regard
http://www.scribemedia.org/2010/10/11/the-ethos-ofopen-source/
Reshaping of NTD Product R&D and delivery
architecture
NTD Product
access & use in the context
of Endemic Country Health Systems
Big
Pharma
Product R&D based on
ENDEMIC COUNTRY
Frameworks for: Priority
setting, Knowledge generation
and access, Research
collaboration, Medicines
Regulation & Financing
agreements
PDPs

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