IHI RealiseD Project Launches to Change the Paradigm for Clinical Trials in Ultra-Rare Diseases
The new European public-private partnership aims to optimise and accelerate the development of treatments for rare and ultra-rare conditions and ultimately improve the lives of millions of patients across Europe and beyond.
The Innovative Health Initiative (IHI) RealiseD project (compRehensive mEthodological and operational Approach to cLinical trIalS in ultra rarE Diseases) has officially launched, bringing together nearly 40 public and private partners to revolutionise clinical trials for rare and ultra-rare diseases. The initiative, under the leadership of Sigmund Freud Private University and AstraZeneca, aims to optimise and accelerate the development of treatments for over 30 million people living with rare diseases across Europe, the majority of whom suffer from ultra-rare conditions with no available treatment options.
With a total budget of 17 million euros, the RealiseD project seeks to break down the barriers hindering progress in rare disease research. By collaborating with clinicians, methodologists, pharmaceutical researchers, patient organisations, regulatory bodies, and HTA (Health Technology Assessment) agencies, the project will develop new operational and methodological tools that will drive forward treatment evaluations and accelerate therapeutic development.
Over the next five years, the RealiseD consortium will focus on addressing key challenges in clinical trials, including limited patient populations, high symptom variability, and fragmented regulatory frameworks. The project will leverage advancements from previous public-private partnerships and incorporate new tools such as Real World Evidence (RWE) and digital health technologies to improve clinical trial designs. This collaborative effort aims to create easy-to-use playbooks and digital tools for planning and running clinical trials, ensuring efficient, patient-centred innovations.
Ralf-Dieter Hilgers, Professor at SFU and RealiseD coordinator, explains, "RealiseD will develop and evaluate innovative clinical trial designs and analysis tools that can accelerate drug development pipelines, while minimising patients' burden to participate in trials."
The European Medicines Agency (EMA), IQWiG, and various European Reference Networks (ERNs) are key partners in this initiative, ensuring that the project adheres to regulatory frameworks while prioritising patient-centred outcomes.
The project also aims to foster collaboration with European Reference Networks (ERNs) particularly (e.g., ERN ERKNet, ERN Epicare, ERN Bond, MetabERN, ERN-EuroBloodNet and ERN-EYE). They will help establish a network of top-notch clinical trial sites across Europe and streamline patient recruitment, particularly for cutting-edge therapies like Advanced Therapy Medicinal Products (ATMPs).
Dr. Solange Corriol-Rohou, Senior Regulatory Affairs & Global Policy Director at AstraZeneca, highlights, "RealiseD showcases the value of public-private partnerships in addressing the needs of those with rare and ultra-rare diseases in Europe and beyond."
ERN-EuroBloodNet Scientific Coordinator and ENROL registry Coordinator María del Mar Mañú Pereira is involved in the EU referral frame for Rare Diseases patients to be enrolled in Clinical Trials.
RealiseD will contribute to initiatives like ERDERA and ACT-EU, aiming to enhance the European clinical trials ecosystem and promote health equity across the EU. The project's methodologies may have broader applications in paediatric research and other complex disease areas, paving the way for a new era in evidence generation.
Read the full announcement here.