The Rare Disease Clinical Trial Network, which is hosted by UCD, funded by the HRB and co-led by Professor Rachel Crowley and Professor Cormac McCarthy, St Vincent’s University Hospital and UCD, held its inaugural conference on the 29 February and 1 March in the Grand Hotel Malahide. Over 100 people attended the conference which brought together global experts, researchers, and advocates to explore the forefront of rare disease clinical research and how it can transform the lives of people living with a rare condition.
Rare diseases are diseases which affect a small number of people compared to the general population (less than 1 person per 2000). However, while these diseases are rare, around 300,000 people in Ireland are living with a rare disease and this can have a big impact on people’s health and the health service.
At the conference, world-leading researchers shared their experience of conducting research and clinical trials across a range of rare conditions including those affecting the lungs, eyes and nervous system. The meeting also heard from global experts in rare disease education and training, clinical trial design and methodology and orphan drug approval.
A common thread throughout all the talks was the importance of incorporating patient and public involvement as a central plank of rare disease research and clinical trial development. This was explored in depth from the perspective of both patient organisations and patient advocates as principal partners in driving clinical research studies and in shaping the broader clinical research landscape. Challenges in rare disease research due to low patient numbers, geographic dispersal and lack of resources were discussed. Speakers outlined how these can be addressed through improving infrastructure, adapting methodologies and streamlining regulatory and legal requirements. An over-riding theme of the conference was the power of collaboration across stakeholder groups, disease specialties, disciplines, institutions and borders.
The conference featured the announcement of the RDCTN Seed Funding Award 2024. This was presented to Dr Laura Williams, Consultant Neurologist, St Vincent’s University Hospital for her project to pilot an inexpensive wearable device that measures dystonia severity to assess if it can offer a reliable and simple means to measure the effectiveness of treatments in clinical trials and in clinical practice.
The award for best poster was presented to Dr Marissa O’Callaghan, St Vincent’s University Hospital, Dublin.
Congratulations to all involved.
Photo right: Presentation of the RDCTN Seed funding Award to Dr Laura Williams by Prof Cormac McCarthy and Prof Rachel Crowley
Photo left: Presentation of the Award for Best Poster to Dr Marissa O'Callaghan by Prof Rachel Crowley
About the Rare Disease Clinical Trial Network (RDCTN)
The RDCTN is funded by the Health Research Board Ireland and hosted by University College Dublin. The co-leads are Prof Rachel Crowley, consultant endocrinologist and Prof Cormac McCarthy, consultant respiratory physician. The aims of the network are:
1) to facilitate and support academic investigators from within and outside Ireland in conducting clinical trials in rare diseases and to establish and augment partnerships with industry to increase rare disease trial conduct in Ireland
2) to provide infrastructural support
3) to expand the involvement of patients, their families and members of the public in the design and conduct of trials in rare disease
4) to educate and train researchers and investigators in rare disease trial conduct and explore/research methodological approaches to rare disease trials in order to optimise recruitment, retention and relevance of research outcomes to patient priorities.
The RDCTN collaborates with Irish, US, UK and European partners. These include patients and patient advocates, scientists, health care professionals and industry representatives. New members are welcome and interested stakeholders can sign up to our mailing list.
More information is available at www.rarediseaseresearch.ie and @rare_trial