Project R-15561

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

RealiseD implements a collaborative and compRehensive mEthodological and operational Approach to cLinical trIalS in rarE Diseases enabling timely drug development and approval centered on patients' needs with high level of evidence reachable in a limited environment. The core of RealiseD operational approaches include 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 ERNs. RealiseD methodological teams will optimize innovative statistical 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. The operational and methodological approaches will be co-created by regulators, HTA, Pharma, patients, clinicians, and biostatisticans 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 this innovative approach, RealiseD will capitalize on an international stakeholders effort as the problem in ultra-rare disease drug development cannot be solved in restricted geographical area. The development of playbooks arising from the cocreated 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.

01 January 2025 - 31 December 2029