‘We need truly collaborative initiatives like this’
Begonya Nafria Escalera of Sant Joan de Déu Children’s Hospital in Barcelona has both a professional and personal interest in ensuring that patients’ needs are met by personalized medicine
Tell me about yourself and your organization
Begonya Nafria Escalera – Sant Joan de Déu Research Foundation
I’m working at Sant Joan de Déu Children’s Hospital as Patient Engagement in Research Coordinator. My area of responsibility is to ensure that the patients and families have an active role, as experts, in any of the research and innovation projects in which our institution is involved. Sant Joan de Déu Children’s Hospital is the largest paediatric hospital in Spain. My background is in social sciences and working in the defence of the patients’ rights for more than 10 years. Also I have a personal story linked with my area of expertise, as I’m caregiver of an adult with cerebral palsy, my brother. In recent years I have also been volunteering with several patient organizations.
What does personalized medicine mean to you?
For me it means the opportunity to offer a treatment for a huge group of complex conditions, the genetic diseases. Most of these conditions affect paediatric patients and there is no cure for them, only in some cases palliative treatments. PM from the patient side means hope but also uncertainty. At this moment, few conditions have the opportunity of these innovative treatments, this means that we need to inform and educate the patients/families about this type of treatment, how it works and what it means to be involved in a clinical trial.
Why do you believe this topic is important?
Because research in new treatments is moving to this direction: try to provide the best and most accurate treatment for the patients. Society and specifically the patients/caregivers need also to move in this direction, knowing what a therapy means, when it works and also the status of research according to the different genetic conditions.
Why is your organization interested in participating in this multi-stakeholder collaboration?
We are increasing our activity in the field of clinical trials in paediatric conditions, as most of these diseases are genetic. For our patients this really can be an opportunity for treatment in the future, considering that probably at the present we don’t have therapeutic options to offer to them. In parallel, in our institution the research model is based on a patient-centric approach, ensuring that in any project in which our institution will be involved patients/families can take part as advisors and/or part of the research team. We have a young person’s advisory group helping in many projects, a board of parents, and according to the needs of every initiative we set up a specific group of patients/parents to help in the design and development of the project. We don’t envision clinical research without the involvement of patients and parents.
What makes this collaboration unique?
For me, it’s the 360 degree approach with a pillar being patient involvement and the issues that need to be addressed to ensure the best quality of life, disease management and empowerment. The activities around health literacy are essential to ensure that any initiative about personalized medicine will be patient-centric, providing patients with the right information to make decisions about their health and treatments.
What are your ultimate expectations from the project?
I am happy to contribute to a global initiative in which patient engagement will be an essential part of the framework, while increasing awareness about health literacy in this field and providing the expertise of working with paediatric patients. As clinical trials in children and young people are global, we need truly collaborative initiatives involving different stakeholders in order to provide the best treatments for the patients, always considering the unmet and specific needs of the most vulnerable groups of patients.