Since joining MSUK last year, I have been gradually deepening my understanding of the field of inherited metabolic disorders to maximally support the team and our work. Recognizing that there was still much to learn about research and development in rare diseases, I decided to attend the EURORDIS Open Academy School on Medicines Research and Development. This Open Academy presented a unique opportunity to delve into the area, offering a brilliant chance to learn about the latest developments and engagement opportunities in rare disease research. 

 The Open Academy began with an online pre-training phase, which consisted of completing online learning e-modules and attending webinars that covered topics such as clinical trials methodology, clinical research, ethics in medicines development, regulatory affairs, health technology assessment, and marketing authorization. 

The long-awaited second part took the form of an intensive one-week training in Barcelona, held from June 3rd to 7th. The week was packed with presentations and workshops by experts in rare diseases, covering topics such as the use of AI and other technologies to drive progress and facilitate everyday life, involvement opportunities within the EMA, health technology assessments, and the compassionate use of medicines. We also experienced what it’s like to be a member of an ethics committee overseeing a clinical trial, designed our own clinical trial, and workshopped the importance of individual steps in drug repurposing. 

Among the highlights of the week’s program were two on-site visits. We first visited the Sant Joan de Déu Hospital, where cutting-edge technologies and a patient-centered approach contribute to excellent care. Two days later, we explored the research and lab facilities at Vall d’Hebron Hospital, specializing in neurology and cancer research, where we learned about the development of promising new therapies. 

This learning experience allowed me to bridge theoretical knowledge with hands-on experience, both through workshops and on-site hospital visits, while connecting with advocates and researchers from Europe and North America. I found this particularly useful given that the high complexity of medicines research and development calls for a comprehensive understanding of its individual aspects. 

Given that my role as a Project Assistant involves working on a variety of diverse projects with an overlap to medicines research and development, this comprehensive training has enabled me to better adapt, contribute to, and switch between them, helping me better support our team and the inherited metabolic disorder community. 

Lastly, I would like to thank the entire MSUK team for their support and the opportunity to attend the Open Academy training, and of course EURORDIS for their excellent work. 

Pavel joined the team at MSUK in 2023 whilst completing his BA Internatinal Relations at the University of Essex. We’re excited to let you know that he has since graduated and will be staying with us throughout 2024 to continue supporting the inherited metabolic disorder community while completing his MSc in Health and International Development at the London School of Economics and Political Science.