Page 23 - SRPSKO DRUŠTVO ISTRAŽIVAČA RAKA

SDIRSACR Oncology Insights

questions, share doubts and concerns, or simply express worry and sadness. It is deeply meaningful to feel that
someone is there to listen - that you are seen and heard. As simple as it may sound, a parent’s identity often gets
lost in the daily struggle for their child’s recovery and we become a support system by the bedside for our child or
family member, and less of a spouse, sibling, or daughter - putting all other relationships on hold until we get the
answers: a diagnosis, a therapy, or recovery. What I realized while staying in the hospital with my child is that not
all of us have the same capacity to face fear and questions that arise - especially when a family member needs help
and there is little we can do to speed up the healing process. What we can do is create conditions for treatment to
proceed smoothly, convey love and support, and walk the path together.
ͳ Community support: Only about 5% of rare diseases have an available therapy, and our child was among that 5%.
According to Global Genes , around 30% of children diagnosed with rare diseases or paediatric cancer do not live
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to see their fifth birthday, which is why all these diagnoses are categorized as rare. What we didn’t know at the
beginning of our son’s treatment was that for 95% of rare diseases, there is no available therapy. Not only in Serbia
but globally, for the rest of 95% of rare diseases. That is why communication with the rare disease community is
so valuable. Our attempts to meet other patients with the same diagnosis led us to events, meetings, and spaces
where we met courageous parents and rare diseases patient advocates who had overcome their challenges and
turned them into solution proposals.
ͳ Our motivation is to offer a space for others - a place where people can ask questions, propose solutions, and
highlight the everyday challenges they face. In September 2022, we launched the podcast Rare and Share , which
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created a platform for sharing personal experiences of patients when dealing with diagnoses, as well as parents
who “moved mountains” for their children - whether by advocating for legal changes, introducing therapies, or
introducing new diagnoses in newborn screening programs, as it was the case with the inclusion of Spinal Muscular
Atrophy in September 2022.

2.1. Informal Education Toward Patient Advocacy

Learning is a continuous process, but it is the only way to gain the arguments that allow you to seek new solutions to
the challenges faced by patients with rare diseases and paediatric cancers. That is why, after completing my formal
education, I have focused on informal learning when it comes to patient advocacy.
In order to better understand the complexities of clinical trials and scientific innovation, I attended the Winter School
of Scientific Innovation and Translational Research in 2023, organized by EURORDIS – the European Organisation for
Rare Diseases.
Continuing this educational journey on the topic of regulatory procedures and the terminology by which drugs and
medical devices obtain agencies permits, I completed a training program for patient representatives organized by the
European Capacity Building for Patients (EUCAPA).
This education gave me the skills, tools, insights, and expertise I needed to take part in joint clinical assessments and
scientific consultations, especially in the context of the EU HTA (Health Technology Assessment) regulation.
After that, I began training to become a Patient Expert through the European Patients’ Academy on Therapeutic
Innovation (EUPATI). I earned my certification after one year and 28 lessons covering six different modules. Additional
benefit of this journey was the network of collaborators I met, coming from various parts of the world - people who
can help connect and empower patient advocates working to improve the quality of life for patients with paediatric
cancers and rare diseases.
The goal of creating European Reference Networks (ERNs) is to ensure that information travels instead of the patient,
thereby reducing the costs associated with transport and hospital treatments. On the other hand, the exchange of
experiences and expertise among specialists can significantly improve the quality of care provided at expert centers.
This is where I see the role of patient advocates: to connect individual patients, doctors, and rare disease treatment
centers by sharing critical information when, maybe, those centers may lack the capacity to share these information in
language that is comprehensible to patients.
After one year and obtaining a certificate from this prestigious European organization, I was invited to join the
Patient Expert Training Committee that prepares the curriculum for the next generation of patient advocacy experts,
contributing with insights from my own experience and sharing the information I gained working with organizations
supporting patients with cancer and rare diseases.

1 Source: Global Genes, https://globalgenes.org/rare-disease-facts/?gad_source=1&gad_campaignid=2080237801&gbraid=0AAAAACthKWbQJp7qiFKZg38Uds-
Gl8bWKp&gclid=Cj0KCQjwvajDBhCNARIsAEE29WqmaqbutmkipmNXpAvXzkaD2jkWteoZZi7qK9vDaBM3aRMx7cl9u4gaAsRYEALw_wcB, date of access July 13
th
2025.
2 Podcast Rare and Share: www.rareandshare.net
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