IHI RealiseD

RealiseD is a public-private partnership supported by the Innovative Health Initiative Joint Undertaking (IHI JU) under grant agreement N° 101165912 that brings together 50 public and private partners from academia, public institutions, patient organizations, clinical research institutes and hospitals, and pharmaceutical companies to optimise and accelerate the development of treatments for rare/ultra-rare diseases and ultimately improve the lives of millions of patients across Europe and beyond; this will also benefit small populations such as children with cancer.

The aim of RealiseD, is to change the paradigm for clinical trials in rare/ultra-rare diseases. By bringing together all the relevant experts, RealiseD will catalyse the development and acceptance of innovative approaches for designing trials. RealiseD will initiate its tools and systems via four use cases from the fields of paediatric epilepsy, bone disorders, eye disease, and haematology and will be tested within ERDERA, the European rare disease partnership. Between them, these use cases cover all age groups and a range of specific challenges in ultra-rare disease research. These resources will be incorporated into easy-to-use and co-created playbooks and digital tools designed to facilitate the set-up and conduct of innovative trials focusing on white spots (conditions for which there is no approved treatment option and where development is not currently commercially viable).

The project will also generate patient referral strategies to boost enrolment in future rare/ultra-rare disease trials and will set up a certification system to identify clinical trial sites capable of running studies with patients suffering from rare/ultra-rare diseases.

RealiseD will disseminate and integrate its solutions in playbooks to reach the broader rare disease ecosystem through partnerships like ERDERA, the European Rare Disease Partnership, and will contribute to the ACT-EU (Accelerating Clinical Trials in European Union) initiative. This will boost the European 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.

As for any other PPP, the RealiseD consortium who drafted their final project proposal which was endorsed by both IMI and the European Commission, will have to work in line with the deliverables identified in their workplan and according to their objectives. RealiseD will implement a collaborative and compRehensive mEthodological Approach to cLinical trIalS in (ultra-)rarE Diseases, enabling faster therapeutic development while balancing the need for timely development and regulatory compliance with the level of evidence realistically attainable in a constrained environment.

To achieve their goals, the RealiseD consortium has formulated objectives, supported by one or more of the WPs, and fully aligned with the expectations cited in the IHI Call 4, Topic 4: Establishing novel approaches to improve clinical trials. Clinicians, methodologists, pharmaceutical industry researchers, representatives from patient organisations, regulatory agencies and HTA bodies involved in the consortium will collaborate in the next five years to establish new gold standards for clinical trials in rare and ultra-rare diseases and to develop cutting edge operational and methodological tools and resources to dramatically advance treatment evaluation.

The RealiseD consortium includes 50 public and private partners with EMA and IQWIG on board as formal public partners. There may be opportunity to welcome new partners to join the consortium, knowing this is something that has already happened with previous IMI/IHI projects. This PPP project which is funded by IHI (Innovative Health Initiative) and EFPIA (European Federation of Pharmaceutical Industries and Associations) was officially launched in January 2025 while initially proposed by EFPIA as part of the Rare Disease Moonshot Initiative in 2022. When IHI launched the project in July 2023, this was the opportunity to attract public partners that are now involved in the RealiseD consortium.

This will be the opportunity to work work closely with some European Reference Networks (e.g., ERKNet or MetabERN) who are involved in ERICA (the European Rare Disease Research Coordination and Support Action) and to build on advancements and outputs from prior and ongoing PPPs like EU-PEARL, Screen4Care, DREAMS or ERA  MET and other relevant initiatives such as ERDERA, IRDIRC, REMEDI4ALL or Together4RD).

Over the next 5 years, by enabling clinical trials in the large number of white spot rare diseases, RealiseD will contribute to improving the care and quality of life of people living with rare diseases. RealiseD’s findings could also benefit other groups facing similar challenges, such as paediatric cancers.

IHI RealiseD website: https://realised-ihi.eu/

Link to the IHI RealiseD factsheet: https://www.ihi.europa.eu/projects-results/project-factsheets/realised