1. An agency of the European Union 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 2

3. 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: Silver Spring, October 2012

4. 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. 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. 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. 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 Silver Spring, October 2012

8. 8 Fee Deferral & Conditional Fee Exemption Post-authorisation Marketing Authorisation Application Granting of the Marketing 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

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

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

11. 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 Silver Spring, October 2012 11

12. RDCVF € 2,6 M TREATRUSH € 6M EURO-PADnet € 3 M MEFOPA € 5,8 M Natural history Pathophysiolo gy Preclinical dvpt ODs Clinical dvpt ODs BIO-NMD € 5,6 M Neurology Immunology Pemphigus € 3 M EFACTS € 6 M EUROTRAPS € 3 M E-Rare-2 € 2 M RareDiseasePlatform € 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 Silver Spring, October 2012 12

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 13

14. Silver Spring, October 2012 14 IRDIRC The International Rare Diseases Research Consortium (IRDiRC) launched in April 2011launched 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

15. 15 Support to SMEs 2005-2011 Scientific Advice & Regulatory assistance: 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 16

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 17

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

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 19

20. Average amount of funding asked per proposal JTC 2007 vs. 2009 vs. 2011 Average (€) Silver Spring, October 2012 20

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) - Plans for sustainability of the E-Rare network (WP6) Silver Spring, October 2012 21

22. 22 Thank you for your attention Links for further information: on SMEs - smeoffice@ema.europa.eusmeoffice@ema.europa.eu - http://www.ema.europa.euhttp://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/ http://ec.europa.eu/research/enquiries/ E-rare: http://www.e-rare.eu