Rare diseases present a formidable global health challenge, affecting over 300 million individuals worldwide. With approximately 7,000 identified conditions, Rare Diseases significantly impact global communities, including 410,000 individuals on the Island of Ireland. 

1 in 17 people will experience living with a rare disease at some point in their lives, and about 70% of Rare Diseases afflict children, young people, and their families, with a significant portion being chronic conditions. 35% of these conditions are life-limiting or life-threatening, often leading to premature death. 

Living with a rare disease is an ongoing learning journey for patients and families, often accompanied by adverse impacts. A shortage of specialist expertise necessitates patients and families becoming experts in their conditions. Challenges in accessing essential services for daily needs, including self-management, health service transition, educational/vocational support and integrated care, further exacerbate the situation.

Rare Disease Day 2024 marks a significant milestone in rare disease research: the official launch of the All-Ireland Children and Young Adults Research Advisory Group for Rare Diseases (RAIN CRAG), the first of its kind on the Isle of Ireland.

Spearheaded by the all-Island interdisciplinary rare disease research network (RAiN), this ground-breaking initiative addresses the unique needs of children and young people affected by Rare Diseases. By actively engaging with children and young people aged 6-17 years, the initiative will ensure that their voices are heard and valued at every stage, helping to facilitate collaborative research and advocacy and drive positive change in the rare disease landscape, to ultimately improve outcomes for affected individuals and their families. 

The Ombudsman for Children, Dr Niall Muldoon, launched the RAiN CRAG and children, young people, families and Rare Disease advocates united for this remarkable occasion to officially forge the beginning of this exciting initiative. Dr Muldoon said, “It is really important to bring children and young people to the table on this with the launch of the Children and Young Adults Research Advisory Group. For us in the Ombudsman for Children’s Office, listening to children and young people and ensuring their voices are heard on issues that impact them is fundamental to the work we do. It is really great to see the RAIN Network look to incorporate the lived views and experiences into their work through this Children and Young Adults Research Advisory Group.”  

Lucy Gallagher, RAiN CRAG member aged 11 from Donegal, said, “The RAiN CRAG gives me an exciting opportunity to tell everyone about my experience with rare disease; at times, it can feel like I’m the only person living with a rare disease. CRAG has shown me that there are so many young people living with rare diseases. We love having our voices and ideas heard. As a CRAG member, I am proud to be an ambassador for rare diseases as I want to raise awareness. Together we don’t feel rare.”

Associate Professor Suja Somanadhan, co-lead of the RAIN initiative at UCD School of Nursing, Midwifery and Health Systems, said, "Despite facing numerous challenges of living with rare diseases, children and young people exhibit remarkable resilience. The RAIN CRAG inclusive platform amplifies their voices, highlighting their diverse needs and the support required. Empowering them with knowledge and advocacy skills sparks a transformative journey towards enhanced peer-to-peer support, self-management, and resilience. We look to dismantle barriers and amplify their voices through training and mentoring. This collaborative effort fosters greater understanding and support within communities and institutions alike, generating innovative approaches with unwavering determination, making them powerful advocates for driving change and paving the way for an inclusive future for all impacted by rare diseases."

Professor Amy Jayne McKnight, Co-lead of the RAIN initiative at Queens University Belfast, said, “The establishment of the RAIN CRAG is a testament to the collaborative spirit of RAiN. This ground-breaking All Ireland initiative brings together young people, advocates, and rare disease experts to drive forward research, raise awareness, and improve outcomes, empowering children and young people living with rare diseases to influence positive change.” 

In addition to the CRAG, RAiN has launched 'Cup O’ Collaboration,' fostering collaboration and amplifying the voices of those living with rare diseases and parents/caregivers of those with a rare disease. Also, the RAiN hosts an early career researchers' platform, connecting our next generation of researchers and pioneers in rare disease research.  

Outputs from the research network will be disseminated through open-access publications, regular presentations, and the network's website in easily accessible formats. RAiN looks forward to hosting interdisciplinary networking events, bringing together researchers, practitioners, policymakers, patients, and families across the Island of Ireland.

In an effort of global solidarity, you are invited to light or decorate your home with the Rare Disease Day colours at 7 PM your local time on 29 February 2024. Use garlands, social media filters, candles, disco lamps, colourful decorations… Let your creativity shine! Take a picture of yourself with the illuminations or decorations and publish it on your social media using our “Global Chain of Light” filters and #rarediseaseday, #RAiNAllIreland, #RAiN, #RareDiseaseNI

Image 1: CRAG Members, including Lucy Gallagher, and patients advocates for rare diseases with Dr Niall Muldoon (Ombudsman for Children), Dr Ciara Martin, National Clinical Advisor and Group lead for Children and Young People (HSE), Dr Suja Somanadhan (Co-Lead RAiN, UCD), Dr Lynne Lohfeld (QUB), Melissa Kinch (RAiN network Co-ordinator, UCD) Dr Harry Shier (Children’s rights activist and facilitator), Laura Egan and Louise O’Keefe (Rare Ireland), Bernadette Gilroy (Munster Technological University (MTU), Dr Carleen Walsh (Ulster University) 

Image 2: Dr Niall Muldoon, the Ombudsman for Children, engaging with CRAG members

Image 3: CRAG member leaving a meaningful quote on the wall at the Office of the Ombudsman in recognition of Rare Disease Day

For further information, contact Melissa Kinch, RAiN network Co-ordinator at @RAINALLireland@ucd.ie or visit Rainrareresearch.org