Project description
Timely drug development for ultra-rare diseases
Timely drug development and approval are essential for addressing rare diseases. The EU-funded RealiseD project seeks to revolutionise clinical trials for rare and ultra-rare diseases through a collaborative, patient-centred approach. The project will establish systematic procedures for patient referrals, certify clinical sites, and address gaps in clinical trial development, focusing on four diseases within four European research networks. A public-private partnership, including regulatory and health technology assessment representatives, will co-create operational and methodological solutions through multi-stakeholder agreements. By reducing uncertainties in drug development for rare and ultra-rare diseases, the project leverages international collaboration to address challenges that extend beyond geographical boundaries.
Objective
RealiseD implements a collaborative and compRehensive mEthodological and operational Approach to cLinical trIalS in rarE Diseases
enabling timely drug development and approval centred on patients’ needs with high level of evidence reachable in a limited
environment. The core of RealiseD operational approaches includes the systematic procedures for patients’ referral, the certification of
clinical sites and filling the gaps for successful development of rare and ultra rare diseases[(U)RD]’ clinical trials using 4 (U)RD diseases
from 4 European Research Networks (ERNs). RealiseD methodological teams will optimise innovative statistical and quantitative
approaches for design and analysis of (U)RD-CTs developed in the past 10 years and refined in the last years to facilitate the regulatory
pathway for innovative drug development. RealiseD is implemented in a public private partnership consortium including regulators
and HTA bodies representatives to co-create operational and methodological approaches in an iterative procedure using a
multistakeholders’ agreement process. RealiseD will increase the incentives and motivation for the pharmaceutical industry by
reducing many uncertainties on the path of drug development in (U)RD that will follow widely accepted rules. To achieve these
innovative approaches, RealiseD will capitalise on an international multistakeholders effort as the problem in (U)RD drug
development cannot be solved in a restricted geographical area. With the development and deployment of playbooks arising from the
co-created developments, RealiseD will ascertain visibility and readiness of the innovations. The overall ambition of RealiseD is to
change the paradigm of CT design for U(RD) by enlarging the spectrum of methodological and operational approaches and to
establish a sustainable, innovative, and optimised CT paradigm for U(RD) medicinal product development programs, while
maximizing the acceptance by all stakeholders.
Programme(s)
- HORIZON.2.1 - Health Main Programme
- HORIZON.2.1.7 - Innovative Health Initiative
Call for proposal
(opens in new window) HORIZON-JU-IHI-2023-04-two-stage
See other projects for this callFunding Scheme
HORIZON-JU-RIA - HORIZON JU Research and Innovation ActionsCoordinator
1020 Wien
Austria
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Participants (38)
3500 Hasselt
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1081 HZ Amsterdam
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Legal entity other than a subcontractor which is affiliated or legally linked to a participant. The entity carries out work under the conditions laid down in the Grant Agreement, supplies goods or provides services for the action, but did not sign the Grant Agreement. A third party abides by the rules applicable to its related participant under the Grant Agreement with regard to eligibility of costs and control of expenditure.
0313 Oslo
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08007 Barcelona
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The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.
75012 Paris
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75014 Paris
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75654 Paris
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69120 Heidelberg
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67091 Strasbourg Cedex
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40136 Bologna
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33100 Udine
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08035 Barcelona
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1090 Wien
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6525 GA Nijmegen
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1083 HN Amsterdam
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The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.
08950 Esplugues De Llobregat
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Legal entity other than a subcontractor which is affiliated or legally linked to a participant. The entity carries out work under the conditions laid down in the Grant Agreement, supplies goods or provides services for the action, but did not sign the Grant Agreement. A third party abides by the rules applicable to its related participant under the Grant Agreement with regard to eligibility of costs and control of expenditure.
08950 Esplugues De Llobregat
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20090 Pieve Emanuele
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9000 Gent
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39106 Magdeburg
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3511 HG UTRECHT
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44801 Bochum
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37075 Goettingen
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5020 Salzburg
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1341 OTTIGNIES-LOUVAIN-LA-NEUVE
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The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.
10587 Berlin
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1083 HS Amsterdam
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CB2 0AA Cambridge
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94250 Gentilly
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91190 GIF-SUR-YVETTE
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2340 Beerse
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55218 Ingelheim
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1070 Bruxelles / Brussel
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4056 Basel
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51373 Leverkusen
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91940 LES ULIS
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4070 Basel
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2880 Bagsvaerd
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Partners (13)
Partner organisations contribute to the implementation of the action, but do not sign the Grant Agreement.
02210 Boston
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65929 Frankfurt Am Main
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1831 Diegem
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94250 Gentilly
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0214 Cambridge, Massachusetts
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20158 Milano
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02142 Cambridge
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08019 Barcelona
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The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.
Partner organisations contribute to the implementation of the action, but do not sign the Grant Agreement.
1640 Martinez
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02210-2453 BOSTON
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NJ 08869 Raritan
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Partner organisations contribute to the implementation of the action, but do not sign the Grant Agreement.
55218 Ingelheim
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Partner organisations contribute to the implementation of the action, but do not sign the Grant Agreement.
RG14 1JA Newbury
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