EU publicly funded research programs

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

3

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:


4

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


5

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


6

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)


7

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


8

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)


Outline





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


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


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


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



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%


Outline





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


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


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 (€)


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


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

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EU publicly funded research programs