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

RealiseD implements a collaborative and compRehensive mEthodological and operational Approach to cLinical trIalS in rarE Diseasesenabling timely drug development and approval centred on patients’ needs with high level of evidence reachable in a limitedenvironment. The core of RealiseD operational approaches includes the systematic procedures for patients’ referral, the certification ofclinical sites and filling the gaps for successful development of rare and ultra rare diseases[(U)RD]’ clinical trials using 4 (U)RD diseasesfrom 4 European Research Networks (ERNs). RealiseD methodological teams will optimise innovative statistical and quantitativeapproaches for design and analysis of (U)RD-CTs developed in the past 10 years and refined in the last years to facilitate the regulatorypathway for innovative drug development. RealiseD is implemented in a public private partnership consortium including regulatorsand HTA bodies representatives to co-create operational and methodological approaches in an iterative procedure using amultistakeholders’ agreement process. RealiseD will increase the incentives and motivation for the pharmaceutical industry byreducing many uncertainties on the path of drug development in (U)RD that will follow widely accepted rules. To achieve theseinnovative approaches, RealiseD will capitalise on an international multistakeholders effort as the problem in (U)RD drugdevelopment cannot be solved in a restricted geographical area. With the development and deployment of playbooks arising from theco-created developments, RealiseD will ascertain visibility and readiness of the innovations. The overall ambition of RealiseD is tochange the paradigm of CT design for U(RD) by enlarging the spectrum of methodological and operational approaches and toestablish a sustainable, innovative, and optimised CT paradigm for U(RD) medicinal product development programs, whilemaximizing the acceptance by all stakeholders.