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EU publicly funded research programs

EU publicly funded research programs. FDA/EMA Orphan Designation and Grant Workshop (12 Oct 2012). Presented by: Jordi Llinares Head of orphan medicines. Outline. Small and Medium sized Enterprises at EMA The European Framework programme A collaborative example.

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EU publicly funded research programs

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  1. EU publicly funded research programs FDA/EMA Orphan Designation and Grant Workshop (12 Oct 2012) Presented by: Jordi Llinares Head of orphan medicines

  2. Outline • Small and Medium sized Enterprises at EMA • The European Framework programme • A collaborative example Silver Spring, October 2012

  3. Tailoring assistance to SMEs To promote innovation and development of new medicines by SMEs • A single interface • SME assignment, public SME register • Fee incentives, regulatory assistance, translations • Facilitate communication • News bulletins, SME User Guide, • Workshops SME Office: Silver Spring, October 2012

  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 Silver Spring, October 2012

  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 Silver Spring, October 2012

  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) Silver Spring, October 2012

  7. SME fee incentives for centralised MAA Fee deferral for SMEs, plus for 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 Orphan medicines: 100% waiver for SMEs - waiver of 1st year post-authorisation fees Silver Spring, October 2012

  8. Fee Deferral & Conditional Fee Exemption Granting of the Marketing Authorisation Marketing Authorisation Application Post-authorisation TIME Payment deferred until the end of the procedure IF scientific advice used: => Payment only in case of success (Marketing Authorisation Granted) Silver Spring, October 2012

  9. Outline • Small and Medium sized Enterprises at EMA • The European Framework programme • A collaborative example Silver Spring, October 2012

  10. Focus of Rare DiseasesResearch in FP7 • Europe-wide studies of their natural history, pathophysiology • Development of preventative, diagnostic and therapeutic interventions. • This sector includes rare Mendelian phenotypes of common diseases. Silver Spring, October 2012

  11. Basic principles • annual calls for proposals • eligibility (scope, partners, funding limits, deadline) • evaluation by panels of independent experts • overseen by independentobservers • 3 criteria: • Science & Technology excellence • Implementation & Management • Potential Impact Silver Spring, October 2012

  12. Clinical dvpt ODs Natural history Preclinical dvpt ODs Pathophysiology Coord. BIO-NMD € 5,6 M TREATRUSH € 6M PADDINGTON € 5,8 M EFACTS € 6 M RDCVF € 2,6 M Neurology LeukoTreat € 6 M MEFOPA € 5,8 M FIGHT-MG € 6 M NIMBL € 5,4 M Pemphigus € 3 M EUROTRAPS € 3 M Immunology E-Rare-2 € 2 M MABSOT € 6 M RareDiseasePlatform € 0.95 M EURO-PADnet € 3 M Euradrenal € 3 M PRATH € 1,8 M Cure HLH € 3 M ALPHAMAN € 5,9M Metabolism EUCLYD € 3 M AIPgene € 3,3 M EUCILIA € 3 M Disorders of urogenital tract EUNEFRON € 3 M EuroDSD € 3 M Pneumology eurIPFnet € 3 M IMPACTT € 5,4M Silver Spring, October 2012 €110 M, 27 projects Dermatology GENEGRAFT € 4,9M

  13. 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 Silver Spring, October 2012

  14. 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 Research More than 28 partners from 12 countries IRDiRC gathers organisations sharing common goals and principles and have agreed to work in coordination and collaboration Silver Spring, October 2012

  15. Support to SMEs 2005-2011 • Scientific Advice & Regulatoryassistance: Applications for marketing authorisation (MAA): - 75 submitted MAAs (human & vet medicines) • - provision of translations for 33 SMEs Silver Spring, October 2012

  16. Outline • Small and Medium sized Enterprises at EMA • The European Framework programme • A collaborative example Silver Spring, October 2012

  17. 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 diseases • To Develop common research policy on rare diseases • To Implement transnational research funding activities (Joint Calls) Silver Spring, October 2012

  18. The E-Rare-2 Consortium in 2012 12 European (or Associated) Member States 16 Research (funding) Agencies/Ministries ZonMw BMBF; PT-DLR NCBiR FNRS ANR FWF UNIPECS UEFISCDI ISS FCT; DGS ISCIII TUBITAK GSRT: Keelpno CSO/MOH Silver Spring, October 2012

  19. 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 Silver Spring, October 2012

  20. Average(€) Average amountoffundingasked per proposalJTC 2007 vs. 2009 vs. 2011 Silver Spring, October 2012

  21. 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) • - Plansforsustainability of the E-Rare network (WP6) Silver Spring, October 2012

  22. Thank you for your attention • Links for further information: on SMEs • smeoffice@ema.europa.eu • 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 Silver Spring, October 2012

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