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Transcript of Ricerca medica indipendente Nicoletta Dentico Health Innovation in Practice (HIP) La Sapienza, Roma,...
Ricerca medica indipendente
Nicoletta DenticoHealth 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 auspicabile-necessaria 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
compoundsMigliaia 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
Launch & utilization
CHALLENGE 3
CHALLENGE 4
Specific
ActivitiesOrganizations
TDR
Unspecified
GSK
WRAIR
La ricerca per le malattie dimenticate nel 2000: il deserto
Screening
Lead identification
Lead optimization
In vivo efficacy
Regulatory toxicity & safety
Phase 1
Phase 2
Phase 3
Registration
CutaneousLeishmaniasis
CHALLENGE 1
CHALLENGE 2
VisceralLeishmaniasis
HAT Chagas
CHALLENGE 3
Launch &utilization
CHALLENGE 4
2007 Drug R&D Landscape for NTDs
Screening
Lead identification
Lead optimization
In vivo efficacy
Regulatory toxicity & safety
Phase 1
Phase 2
Phase 3
Registration
Launch & utilization
HAT Chagas VisceralLeishmaniasis
CutaneousLeishmaniasis
Dengue Malaria TB
CHALLENGE 1
Sandler Center
IOWHMMVGSK
DD@D
CDNDTB Alliance
DNDi
Specific
ActivitiesOrganizations
TDR
NITD – PC dengue, TB
CHALLENGE 2
CHALLENGE 3
CHALLENGE 4
Unspecified
BuruliUlcer
Intestinal helminth
WRAIR
BONO
MSF
250 PPPH WEFPHA
World Bank
http://www.dndi.org/index.phphttp://www.dndi.org/index.php
Ensure Sustainability
Critical Challenges for Product Development Partnerships
Generate Leads
Optimise Leads
Clinical Trial Capacity to Test New Drugs or Regimens
Pragmatic ID and
selection of
candidates from
existing compounds
Deliveringproducts to neglected population
s
Challenge 1
Clinical AVAILABLEto patients
ScreeningLead
selection
DiscoveryPre-Clinical
Lead optimisation
Challenge 3 Challenge 4Challenge 2
Challenge 5
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 PDPsPDPs 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
JS Dr. Jannin, WHO
GPTB
• Access to Sites
• Status of Infrastructure
• Staff Limitations
Dr. Jannin, WHO
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”
Big Pharma
Pro duct Development Partnerships
(PDPs)
FDA, EMA, Swissmedic, etc
Public R&D institutions and academia
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 setting
Translational research and developoment
Knowledge generation:
FundingPhilanthropic and
International development organizations
Corporate & Private
“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/2012report/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 two-thirds 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-of-open-source/
Big Pharma PDPs
NTD Product access & use in the context
of Endemic Country Health Systems
Reshaping of NTD Product R&D and delivery architecture
Product R&D based on ENDEMIC COUNTRY
Frameworks for: Priority setting, Knowledge generation
and access, Research collaboration, Medicines Regulation & Financing
agreements