About us
RealiseD unites nearly 40 partners from academia, regulatory bodies, clinical research institutes and hospitals, patient organisations, pharmaceutical companies or European Research Infrastructures to establish new gold standards for clinical trials in rare and ultra-rare diseases. Sigmund Freud Private University and AstraZeneca lead this project, which is funded by the Innovative Health Initiative (IHI) and will run until 2029.
Innovative methodologies and tools
RealiseD builds on advancements and outputs from prior and ongoing public private partnerships like EU-PEARL or Screen4Care and other relevant initiatives, such as ERICA, IDeA or ERDERA to co-create tools, methodologies and standards, to enhance innovative clinical trial designs. These will incorporate new methods like Real World Evidence and digital health technologies. Solutions will result in easy-to-use playbooks and digital tools for planning and running clinical trials.
Dieter Hilgers – Professor at SFU and RealiseD coordinator
“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 tools will result in a paradigm shift of treatment evaluation. This is especially important in diseases with extremely low prevalence and incidence, where therapeutic intervention options are limited or non-existent”.
Crucial collaborations to shape innovative solutions
The European Medicines Agency (EMA), the German Institute for Quality and Efficiency in Health Care (IQWIG) and clinical partners from the European Reference Networks (ERNs) for rare diseases or EURORDIS are amongst the organisations that participate in the co-creation of these innovative methodologies and tools designed for limited patient groups and clusters of diseases.
Their role will be critical to pave the way for acceptance of innovative paradigms generated by the project and making sure outcomes relevant to patients are considered in decision-making, while adhering to a regulatory framework.
Dr. Thomas Kaiser, IQWiG Director
“People with ultra-rare diseases have the right to high-quality evidence on new therapies compared to the current standard of care. Only then they can make an informed decision for or against a new treatment option. However, conducting high-quality trials in ultra-rare diseases is challenging. In this project, we aim to overcome these obstacles. I am convinced that by enhancing the quality of comparative studies, we can significantly improve the lives of those affected now and in the future.”
Dr Alicia Granados, Head of Global Medical Scientific Advocacy & Insights for Rare Diseases at Sanofi
“RealiseD’s approaches may also have broader applications in other complex areas, including pediatric research, signaling a new era for evidence generation, interpretation, and use for decision making, where the concept of totality of evidence will be better understood and accepted, therefore creating a new framework for scientific dialogues between developers, regulators and HTAs“
Building on the European Reference Networks
European Reference Networks (ERNs) (e.g., ERN ERKNet, ERN Epicare, ERN Bond, MetabERN, ERN-EuroBloodNet and ERN-EYE) are helping to establish a network of top-notch clinical trial sites across Europe and create an efficient referral system for patients. This system that brings together European hospital centres of expertise and reference to tackle rare, low prevalence and complex diseases and conditions requiring highly specialised healthcare will help streamline the process of finding and enrolling willing patients in appropriate clinical trials, especially for cutting-edge treatments like Advanced Therapy Medicinal Products (ATMPs).
Rima Nabbout – Professor of Pediatric Neurology, director of the National Centre for Rare Epilepsies at Necker-Enfants Malades Hospital
“The active participation of physicians’ experts in rare diseases in this project witnesses the major unmet needs that they face in their everyday practice with patients and families. RealiseD will accelerate meaningful trials and the use of multisource data in rare and ultra-rare diseases in order to accelerate the development of therapies with no patients left behind”.
Solutions guided by impact
RealiseD will disseminate and integrate its solutions in playbooks to reach the broader rare disease ecosystem through partnerships like ERDERA and will contribute to the Accelerating Clinical Trials in EU (ACT-EU) initiative. This will boost the EU industry, make Europe more appealing for clinical trials and, ultimately, will promote health equity across EU borders, by ensuring that patients with rare and ultra-rare diseases receive the attention they need.
Kathy Williams – Executive Director, Global Regulatory Affairs, AstraZeneca, and Realise-D project lead
“RealiseD unites the right experts and partners to reimagine how treatments for rare diseases are developed. Together, we are co-creating innovative approaches that can speed progress, elevate what truly matters to patients, and inspire broader acceptance of new paradigms—all while honoring a strong regulatory framework that protects trust and safety”.
