an agency of the european union eu publicly funded research programs fda/ema orphan designation and...
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An agency of the European Union
EU publicly funded research programsFDA/EMA Orphan Designation and Grant Workshop FDA/EMA Orphan Designation and Grant Workshop
(12 Oct 2012)(12 Oct 2012)
Presented by: Jordi LlinaresHead of orphan medicines
Outline
• Small and Medium sized Enterprises at EMA
• The European Framework programme
• A collaborative example
Silver Spring, October 20122
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Tailoring assistance to SMEs
•A single interface
•SME assignment, public SME register
•Fee incentives, regulatory assistance, translations
•Facilitate communication
•News bulletins, SME User Guide,
Workshops
To promote innovation and development of new medicines by SMEs
SME Office:
Silver Spring, October 2012
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Assignment of SME Status
Applicant must be established in the EEA
Meet SME criteria defined in Recommendation 2003/361/EC:
Headcount < 250 and annual turnover not more than € 50 mil or balance sheet not more than € 43 mil
Submit information to show comply with criteria
http://www.ema.europa.eu/SME/SMEoverview.htm
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Incentives for SMEs
• Administrative and procedural assistance
• Fee reductions and deferrals
• Certification of Quality / Non-clinical data for advanced therapy medicinal products*
• Translation of product information
* Medicines based on genes / cells / tissues
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Fee Reductions for SMEs
90% reduction on :- scientific advice
- inspections
- scientific services
- maximum residue limits (veterinary medicines)
100% ‘waiver’ on administrative services(except for parallel distribution)
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SME fee incentives for centralised MAA
Fee deferral for SMEs, plus for
Orphan medicines:
100% waiver for SMEs
- waiver of 1st year post-authorisation fees
Advanced Therapies:
50% reduction for SME/hospitals
- centralised MAA where public health interest in EU until end 2012 for tissue engineered products
- reduction of 1st year post-authorisation fees
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Fee Deferral & Conditional Fee Exemption
Post-authorisationMarketing Authorisation Application
Granting of theMarketing Authorisation
Payment deferred until the end of the procedure
TIME
IF scientific advice used:
=> Payment only in case of success(Marketing Authorisation Granted)
Silver Spring, October 2012
Outline
• Small and Medium sized Enterprises at EMA
• The European Framework programme
• A collaborative example
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Europe-wide studies of their natural history, pathophysiology
Development of preventative, diagnostic and therapeutic interventions.
This sector includes rare Mendelian phenotypes of common diseases.
Focus of Rare Diseases Research in FP7
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annual calls for proposals
eligibility (scope, partners, funding limits, deadline)
evaluation by panels of independent experts
overseen by independent observers
3 criteria: Science & Technology excellence Implementation & Management Potential Impact
Basic principles
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RDCVF € 2,6 M
TREATRUSH € 6M
EURO-PADnet € 3 M
MEFOPA € 5,8 M
Natural history
Pathophysiology
Preclinical dvpt ODs
Clinical dvptODs
BIO-NMD € 5,6 M
Neurology
Immunology
Pemphigus € 3 M
EFACTS € 6 M
EUROTRAPS € 3 M
E-R
are
-2
€ 2
M
Rare
Dis
easeP
latf
orm
€ 0
.95
M
PADDINGTON € 5,8 M
MABSOT € 6 M
IMPACTT € 5,4M
Metabolism ALPHAMAN €
5,9M
Cure HLH € 3 M
FIGHT-MG € 6 M
Disorders of urogenital tract
EUCILIA € 3 M
EUCLYD € 3 M
LeukoTreat € 6 M
PRATH € 1,8 M
AIPgene € 3,3 M
Dermatology
GENEGRAFT € 4,9M
EuroDSD € 3 M
eurIPFnet € 3 M
EUNEFRON € 3 M
NIMBL € 5,4 M
Pneumology
Coord.
€110 M, 27 projects
Euradrenal € 3 M
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Rare diseases:
strengthening EU/US/international collaboration
Recognised need for more international cooperation in research on rare diseases
• to align taxonomy, diagnosis and treatment options• to optimise scattered and scarce resources (patients,
experts, budgets)• with a view to accelerate the development of new
diagnostic and therapeutic options
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IRDIRC
The International Rare Diseases Research Consortium (IRDiRC) launched in April 2011 to foster international collaboration in rare diseases research.
Initiated by NIH and DG ResearchMore than 28 partners from 12 countries
IRDiRC gathers organisations sharing common goals and principles and have agreed to work in coordination and collaboration
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Support to SMEs 2005-2011
Scientific Advice & Regulatory assistance:
020406080
100120140160180
2006 2007 2008 2009 2010 2011
Scientic advice Regulatory assistance
Applications for marketing authorisation (MAA):
- 75 submitted MAAs (human & vet medicines)
- provision of translations for 33 SMEs
Clinical47%
Quality21%
Pre-clinical32%
Silver Spring, October 2012
Outline
• Small and Medium sized Enterprises at EMA
• The European Framework programme
• A collaborative example
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E-Rare-1 (2006-2010) and E-Rare-2 (2010-2014) - funded ERA-Net
Partners: National Research Funding Agencies / Ministries
Objectives: Coordinate national / regional research programmes on RDs and develop joint and strategic activities:
To Harmonise and develop synergies between national research programmes on rare diseasesTo Develop common research policy on rare diseasesTo Implement transnational research funding activities (Joint Calls)Silver Spring, October 201217
The E-Rare-2 Consortium in 2012The E-Rare-2 Consortium in 2012
12 European (or Associated) Member States16 Research (funding) Agencies/Ministries
ISCIII
FCT;
DG
S
ANR
BMBF; PT-DLRFNRS
ZonMw
ISS
TUBITAKGSRT:Keelpno
CS
O/M
OH
NCBiR
FWFUNIPECS
UEFISCDI
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E-RARE, lessons learnt Success of the E-Rare Calls for research projects on RDs reflects
the needs of the European RD research community for funding transnational collaboration
The transnational calls have managed to leverage funds from agencies which do not have a national research programme on RDs
The linking of the responsible research funding organisations and ministries have increased the awareness of the funding agencies on rare disease research
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Average amount of funding asked per proposal Average amount of funding asked per proposal JTC 2007 vs. 2009 vs. 2011JTC 2007 vs. 2009 vs. 2011
Average (€)
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Aims and objectives of E-Rare (2010-2014) Aim:
- Settle a wide Transnational Research Programme on Rare Diseases
Objectives: - Enlarge E-Rare consortium to increase European/International
cooperation
- Establish E-Rare as a portal for transnational funding of collaborative and multidisciplinary research on RDs
Specific Objectives:
- European (and beyond) landscape of RD research programmes (WP2)
- External communication / dissemination of E-Rare results (WP1&WP7)
- Future Common Strategic Programme for RD research (WP6)
- Plans for sustainability of the E-Rare network (WP6)
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Thank you for your attentionThank you for your attention
Links for further information: on SMEs - [email protected] - http://www.ema.europa.eu
Silver Spring, October 2012
EU research: http://ec.europa.eu/research
Seventh Framework Programme: http://ec.europa.eu/research/fp7
Information on research programmes and projects: http://cordis.europa.eu/
http://ec.europa.eu/research/research-eu/
Information requests: http://ec.europa.eu/research/enquiries/
E-rare: http://www.e-rare.eu