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Comprehensive methodological and operational approach to clinical trials in rare and ultra-rare diseases

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.

Coordinator

SIGMUND FREUD PRIVATUNIVERSITAT WIEN GMBH
Net EU contribution
€ 699 291,25
Address
FREUDPLATZ 1
1020 Wien
Austria

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Region
Ostösterreich Wien Wien
Activity type
Higher or Secondary Education Establishments
Links
Total cost
€ 1 086 791,25

Participants (38)

Partners (13)