The vast majority of healthcare research in the UK is investigator-led. While national progress in patient and public involvement (PPI) increasingly mandates patient consultation, research questions and outcomes still frequently misalign with patient priorities. This is particularly important in rare disease research, as more than 95% of 11 000 conditions have no effective or curative treatment, and around 20% are not clinically defined, making them difficult to diagnose and manage. The unmet physical, mental and emotional needs of people living with rare diseases are immense. Extensive guidance and toolkits exist to support investigators with PPI, but none target patient communities attempting to promote their own priorities, initiate or co-lead research.

This communication article introduces the newly established patient-led Rare Disease Research Network (RDRN).

Launched in November 2024, the RDRN is an open-access collaborative platform designed to support patient-driven and co-produced research, connecting patient and professional partners with similar research interests. Originally conceived by an ultra-rare patient group, the network was co-produced with the rare disease community, including individuals living with rare conditions, parents, carers and charity advocates, whose lived experience and priorities shaped every aspect of its design. Supported by academic and research networks, its collaborative development ensures RDRN removes barriers to participation while complementing existing initiatives. RDRN is a novel approach to driving new impactful research by aligning investigator priorities with real-world needs and building capacity from patients outward. Rare disease communities bring lived expertise, creativity and motivation. Yet without a structured route to collaborate, their insights are often lost. RDRN offers an inclusive space, fostering new partnerships and supporting upstream collaboration. The approach enables patients to become ‘research ready’ and empowers them to have an active role in generating ideas and delivering research from inception, leading to innovative research and driving meaningful change in patients’ lives. With further development, RDRN could present a lasting, scalable and unified model for co-designed rare disease research. By enabling trust, capacity and shared purpose, it can drive discovery, improve outcomes and build a more resilient and self-sustaining research ecosystem, underpinning key pillars of the 2021 UK Rare Diseases Framework.

An increasing number of teenagers and young adults (TYA) with chronic conditions and complex needs are transitioning from paediatric to adult services, including admission to intensive care units (ICUs). As these services are often ill-equipped to care for TYA, there is a risk of compromised care. Despite recent guidelines from the UK Paediatric Critical Care and Intensive Care Societies highlighting the importance and urgency of improving ICU transition, current recommendations are not evidence-based and established pathways for ICU transition remain limited.

This mixed-methods research study aims to generate evidence to underpin national policy on transition from paediatric to adult ICUs that will improve clinical care and patient experience. To do this, we will: (1) link and analyse UK national data (years 2017–2024) on paediatric and adult ICU admissions, hospital inpatient, outpatient and emergency care visits and survival status, to determine the clinical characteristics and healthcare resource utilisation from teenage years to early adulthood of people admitted to an ICU as a young person (admission aged 14 and 15), and how these relate to ICU admissions after age 16; (2) conduct semistructured interviews, online forums and surveys with TYA patients, carers and health professionals to understand their experience of transition in ICU services; and (3) synthesise these strands of evidence and use a structured process of stakeholder engagement to propose potential targeted improvements as appropriate.

This study was approved by the East of England - Cambridge South Research Ethics Committee on 1 August 2024 (research ethics committee number 24/EE/0108), and the Health Research Authority Confidentiality Advisory Group (CAG) on 7 October 2024 (CAG number 24/CAG/0068). Study results will be actively disseminated through peer-reviewed journals, conference presentations and accessible lay texts and graphic summaries for the use of charities and patients including those with learning disabilities and neurodevelopmental disorders.