WELCOME TO i4KIDS-4RARE:

A dedicated accelerator programme to support the creation and advancement of innovative solutions specifically tailored for paediatric patients affected by rare diseases.

THE PROJECT

i4KIDS-4RARE is a 24-month EU-funded project that aims to provide a structured framework, essential resources, mentorship and financial support to aspiring entrepreneurs, researchers and healthcare professionals who are passionate about making a significant impact in the field of paediatric rare diseases through an accelerator programme focused on three specific use cases developing innovative solutions in the areas of cardiology, neurology and rehabilitation.

The i4KIDS-4RARE project is designed to address the critical medical, research and social needs associated with orphan medical devices for rare diseases:

Overall, i4KIDS-4RARE not only addresses urgent medical needs, but also creates a positive social impact and supports ongoing research that is changing the landscape of rare disease management.

Medical needs

The programme will accelerate the development of innovative medical devices to ensure that children with rare diseases have access to advanced treatments and technologies tailored to their conditions.

Research needs

i4KIDS-4RARE drives research advances by fostering collaboration between researchers, healthcare professionals and technology experts. The programme generates valuable data and insights that contribute to ongoing research efforts and the discovery of new treatments.

Social needs

By providing timely access to advanced medical devices, the programme improves the quality of life for patients and their families. It promotes social inclusion, empowers patients and fosters a supportive community network.

i4KIDS-4RARE Accelerator Programme

This accelerator will build on two main programmes from the original i4KIDS initiative, which will run in parallel and focus on three use cases:

• Challenge-based Clinical 4RARE Programme (with clinical and industry focus):

Use Case #1: paediatric epilepsy (neurology)

• Validation & Valorisation 4RARE programme:

Use Case #2: Congenital Heart Disease (Cardiology)

Use Case #3: Spinal Muscular Atrophy (SMA) (Rehabilitation)

The objectives include the provision of mentorship, where projects will receive guidance from rare disease experts to help them overcome the unique challenges of the sector. In addition, there will be collaboration with industry partners, in particular companies with experience in paediatric rare diseases, to foster innovation and drive progress.

In detail, the three use cases to be supported by i4KIDS-4RARE are as follows:

Use case #1 – Paediatric Epilepsy (Neurology)

Paediatric epilepsy includes various disorders affecting neurodevelopment, quality of life, and mortality. In Europe, over 6 million people are affected, with nearly 5 in 10,000 suffering from rare, complex epilepsy syndromes. While 65% of patients respond to treatments, many require further interventions, such as surgery. Childhood epilepsy presents unique challenges due to its impact on development, cognitive function, and behaviour. Early diagnosis and appropriate treatment are essential. To address unmet needs, i4KIDS-4RARE will implement the “Challenge-based Clinical 4RARE” Programme. This initiative will unite experts, patients, and families to find solutions, supported by the EpiCARE network and led by Sant Joan de Déu Barcelona Children’s Hospital. This collaborative approach will enhance epilepsy care, focusing on clinical, innovation, and regulatory requirements.

Use case #2 – Congenital Heart Disease (Cardiology)

Congenital heart disease (CHD) is the most common congenital malformation, affecting 8 in every 1,000 live births. Although 85% of children with CHD now reach adulthood, many face residual defects that impact their quality of life. CHD is a complex condition that often requires multiple surgeries and ongoing personalised care. A key challenge in CHD management is the close relationship between cardiovascular and brain systems, particularly during surgery, where maintaining brain function is critical. Current tools, like brain MRI and neuromonitoring devices, provide essential data but lack real-time, bedside integration. This gap led to the creation of ArcoeRIS (Advanced Real-Time Cerebral Electronic Risk Score for Children with Congenital Heart Disease), a project aimed at improving perioperative care. ArcoeRIS combines data from various sources, including noninvasive neuromonitoring, bedside monitors, and lab biomarkers, into a real-time Brain Damage Score (BDS) to assess cerebral well-being during surgery. Using AI, this platform helps predict and prevent brain damage, allowing immediate interventions. The i4KIDS team has identified the need for further market access, regulatory planning, and business development to advance ArcoeRIS into clinical practice. As part of the Acceleration-4RARE programme, the next steps include developing a clinical investigational plan, regulatory roadmap, patentability assessment, and early market access study to benefit CHD patients and healthcare professionals.

Use case #3 – Spinal Muscular Atrophy (SMA) (Rehabilitation)

Spinal muscular atrophy (SMA) is a group of inherited neuromuscular disorders caused by mutations in the SMN1 gene, leading to progressive muscle weakness and atrophy, particularly in muscles close to the body such as the shoulders, hips, and back. Affecting approximately 1 in 10,000 live births, SMA impacts both males and females equally. Early diagnosis through newborn screening allows for timely treatment, which can significantly improve outcomes. While new therapies have emerged since 2016 to modify the disease’s progression, rehabilitation remains a key part of treatment, aiming to prevent joint deformities, improve posture, maintain mobility, reduce respiratory complications, and enhance quality of life.

Rehabilitation for non-ambulatory SMA patients can be repetitive and difficult, leading to challenges in maintaining motivation and adherence. Travel to rehabilitation centres can also be burdensome. To address these issues, the Sant Joan de Déu Barcelona Children’s Hospital rehabilitation and innovation teams developed a virtual reality (VR) video game to support home rehabilitation for SMA-II and III patients. This innovative approach allows for monitored rehabilitation exercises, tracking progress, and enhancing treatment adherence. The project has been selected to enter the validation phase of the Acceleration-4RARE programme, focusing on technical proof of concept and market access strategies. If successful, the i4KIDS-4RARE team will work on the regulatory roadmap to move towards broader implementation.