The Therapies Scientific Committee (TSC) is establishinga Working Group on the Engagement of Young People Living with Rare Diseases in Therapy Development.
The aim of the working group is to develop a landscape view of the field and create practical tools such a decision tree to help inform stakeholders involved in pediatric rare diseases research and therapies development.
Engaging young people in research is essential for advancing therapies for rare diseases by involving them in the design, execution and real-world application of studies. Despite global initiatives supporting youth involvement, there is a lack of clear and harmonized guidance on how to effectively engage young people in rare disease research.
The main objective is to understand and map out the different patient engagement activities and tools in therapy development categorized by specific age groups (e.g., early childhood, middle childhood, adolescents and young people that have transitioned to adult care); and identify opportunities to advance the field, address existing gaps, and propose solutions to improve patient outcomes.
IRDiRC is looking for members to populate this Working Group with the below expertise in one or more of the following areas:
- Patient/patient representatives, who have previously been involved in patient engagement activities/methodologies for young people. Ideally young patients themselves (from different age groups (18-35 years)). The focus will be on increasing representation from younger voices and those with varied backgrounds or experiences.
- Professionals such aspediatric or rare disease advanced practice nurses, psychologists and experts in developing pediatric research tools are particularly sought. Health care professionals with experience working with pediatric populations, especially those who have expertise in psychosocial care, health psychology, or child development. Professionals such as pediatric or rare disease specialist nurses, psychologists, and experts in developing pediatric research tools are particularly sought. Additionally, representatives from ethical committees, pediatric networks (e.g., young people advisory groups (YPAGs)), and other global initiatives in the field of pediatrics.
- Regulatory and HTA experts, as well as payers, with experience in engaging young patients in the decision-making processes related to pediatric therapies. Specific emphasis will be placed on those familiar with adapting regulations or health technology assessments for pediatric populations.
- Funders and company representatives with experience in global patient advocacy or developing pediatric engagement frameworks. Experts who can bring industry perspectives to inform practical, scalable solutions are particularly valued.
- Researchers and therapy developers working with young people, preferably pediatrics, who can contribute specifically to the objectives of the call by offering innovative approaches to engagement or leveraging data-driven insights to inform best practices.
- Educators or advocates from rare disease organizations who have supported young patients in understanding and participating in research processes. Their expertise in bridging communication gaps between young participants and researchers will strengthen the working group.
The usual time commitment includes monthly 1-hour teleconferences, one digital workshop (0.5 days), regular email correspondence and collaborative working, with the opportunity to co-author peer reviewed article(s).
If you are interested in taking part in this activity please send a CV, biosketch and letter of motivation clearly describing your expertise to the Scientific Secretariat scientific.secretariat[at]irdirc.org before the 9h February, 2025.
Please add in the subject of your email the reference for this call Ref: WG-YPE.
Only selected candidates will be contacted.