Maria Cavaller (Patient Engagement Manager) and Gulcin Gumus (Research and Policy Project Manager) are from patient organisation EURORDIS-Rare Diseases Europe, which is a c4c project partner. We talked to them about how patient organizations can take part in the c4c project, the importance of engaging young patients in research and having patients as equal partners in medicines development.

‘Patient engagement is a key component of the c4c project. Young patients and their families who are involved in several activities of the project make a substantial impact in paediatric drug development’

What do you think when you read the words patient engagement?

What comes to my mind is the active collaboration between all stakeholders involved in the process of drug development and in the decision-making process. More specifically, thinking of patients as active collaborators that add value and take part throughout the medicine’s lifecycle. This means driving research to co-create protocols and being part of the regulatory, pricing and reimbursement activities.


How will c4c improve the engagement of young patients in regulatory activities?

c4c has already been integrating the voice of young patients in various activities. So far, we have developed educational materials, 2-day online workshop for the patient community (including a session on regulatory). By training the patient community and raising awareness on how young patients can be involved in different activities we can ensure meaningful engagement. For instance, the European Medicines Agency (EMA) has a specific framework for engaging with young patients and how they can be involved in the regulatory process.


What would you like to see in the future?

Around 70% of rare diseases have a childhood onset, meaning that involving young patients is vital for the development of paediatric drugs. Therefore, we aim to have a common framework that enables structured, effective, meaningful, ethical, and sustainable patient engagement, specifically for young patients, like the framework that already exists for adults.


How can we achieve that?

By adapting existing materials (such as PARADIGM toolbox, EJP-RD Guidelines on how to involve patients in research projects, EMA training resources) for the use of young patients; training and increasing awareness for all the stakeholders involved in drug development (i.e. researchers, clinicians and other health care providers, industry, policy makers, regulators, etc.) on the importance of involving young patients. c4c will also develop specific materials that will complement and ensure that young patients voices are embedded into paediatric drug development.