E-Rare and IRDiRC: Future collaboration perspectives? Daria Julkowska E-Rare Coordinator 5th IRDiRC Executive Committee Meeting 23 September 2013, Miami

E-Rare Consortium (2010-2014) ERA-Net for Research Programmes on Rare Diseases 13 European (or Associated) Member States 17 Research (funding) Agencies/Ministries

E-Rare Joint Transnational Calls JTC 2007 X X X X X X JTC 2009 X X X X X X X X X X JTC 2011 X/X X X X X X X X X JTC 2012 (YI) X X X X X X X X X X JTC 2013 X/X X X X X X X X X X X/X X X/X X E-Rare-1 E-Rare-2 JTC 2007: 6 call partners from 6 countries Funded Non Funded Teams Consortia JTC 2013: 17 call partners from 14 countries Since January 2008 (JTC 2007, JTC 2009, JTC 2011 & JTC 2012): 37,5 Mio. € to support 249 research groups in 53 research consortia The global success rate of E-RARE proposals is 11%

Recruitment of call partners: E-Rare Joint Transnational Calls: PREPARATION Jan Feb Mar Apr May Jun Jul Aug Sep Oct Nov Dec Definition of theme Recruitment of call partners: E-Rare members New funding bodies (National agencies/ministries/private companies/private foundations or patients’ associations) Preparation of call documents Funding commitment MoU signature Call pre-announcement Call launch VIRTUAL COMMON POT Each agency funds only ITS national research teams

+ E-Rare Joint Transnational Calls: CALL SCHEDULE Dec Jan Feb Mar Apr May Jun Jul Aug Sep Oct Nov Call launch Pre- proposals submission Eligibility check 1st SEC meeting Full Evaluation by experts Rebuttal step Each proposal is allocated to : 2-3 external peer reviewers 1 member of the SEC as “rapporteur” 2 members of the SEC as readers 2nd SEC + CSC Meeting (funding bodies) - Ranking of proposals based on evaluation criteria - Set up of the list of proposals proposed for funding - Selection and final decision for funding FUNDING Negotiation

E-Rare Joint Transnational Call 2014 Theme: Development of innovative therapeutic approaches for rare diseases Up to the pre-clinical proof-of-concept in cell or animal models Includes: cell therapy, gene therapy, pharmacological compounds (repurposing, molecules screening in iPS cell models, exon skipping, etc) Collaborations with SMEs and pharma are encouraged

E-Rare-3 in Horizon 2020 ERA-Net for research programmes on rare diseases with specific focus on IRDiRC objectives Mostly dedicated to joint calls’ activity Topics established in respect to IRDiRC roadmap Possibility of first joint call with EC top up funding Close collaboration with European Infrastructures Possible collaboration with IRDiRC members: As an E-Rare full member? As a call partner? Bilateral agreements (with E-Rare consortium, with specific country)?

E-Rare as a tool for IRDiRC Transnational Calls?

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