By Vijay Tailor-Hamblin,
Hello, I am Leo, along with my brothers Tyler and Ryker, we are part of the Eye-YPAG group that went to the ERDERA conference in Athens. If there are any words you possibly don’t understand, we have a glossary at the bottom of the page. The words there are the ones in bold like this.
We woke up, had breakfast and headed downstairs and started our first day in Athens. One of the people, Donato Bonifazi, introduced himself and told us how all of these YPAG groups help researchers and health programs. At the meeting there were participants from Portugal, Greece, Italy, Albania, Ireland and of course, the UK!
Then, each of the YPAG groups told us where they were from and some of their achievements. Links to some of the groups are below the glossary.
After all of the presentations,we learnt about advocacy and patient organisations. These are basically organisations that help people with rare diseases with information and support their aim is to help patients,their families and the doctors who work with them. Demetrios Anasthasiou, an advocate for rare diseases and Duchenne muscular dystrophy in particular, said ‘in advocacy, use your worst traits for good!’
That interesting talk lead to a discussion where persistence and determination, a stubborn desire to see something that you believe through could be an asset for an advocate because are we not advocates for ourselves after all? Then we had a break.
Once we finished our break, we listened to another presentation by Rima Nabbot, MD, PHD. This one was about an overview of rare and genetic diseases in paediatric patients. The interesting information about this, around 70 % of these rare diseases start in childhood and most of them don’t have an effective treatment. Most of them are genetic and these conditions can be very difficult for every family, especially those with financial problems.
For the next 30 minutes we talked about challenges in diagnosis, research and care. These can include the entire process being long and expensive which delays proper treatments.
After that, we had a break and then talked about legal and ethical considerations in paediatric research. In simple terms this means to protect children’s rights and well-being during research studies. Examples of this include consent, which means letting the parents or guardians decide if they want their child to undergo the research project. In addition, the child can also decline doing the research if they don’t want to. Legal and ethical considerations include letting children stop doing the research whenever they want to. Researchers also give chances and questions if the child wants to stop the research in case they are shy.
Next we learnt about the role of young patients in matters related to their health. Some of the key points in this included how children who are sixteen or higher are allowed to decide what is best for them in projects due to their maturity and understanding.
After that session, we came to the biggest break in our day, lunch! After some delicious food we went back to work and talked about how unmet needs and prioritization in health and research decision-making meanscurrent treatments or interventions don’t fully address the needs of patients or researchers. This can impact many people and can have severe consequences.
We then had an interactive session with scenarios on how we could handle a situation where a survey is getting boring and patients want a less repetitive test. We discussed who we could side with, the patients or the researchers and ways to make it fair on both sides.
It was a pretty full day and a lot of information. We all took a trip to see Syntagma square and found a good place to eat souvlaki!
At the beginning of our last day of the conference, we talked about involvement in CTs and innovative studies. These benefit patients as they have access to new treatments and contribute to the scientific community. We also learnt about PREMs and PROMSs. PROMSs are Patient-Reported Outcome Measures which focus on health-related outcomes, while PREMs focus on the patient’s experience with the healthcare system.
After we finished the PREMs and PROMs, we focused on consent/assent, clinical study protocol, and lay summaries. Clinical study protocols are detailed, step-by-step plan for a research study, outlining the study’s design, procedures, data collection methods, and analysis plans.it’s kind of like a clinical studies blueprint.
After another break, we talked about the availability and access to orphan drugs in the EU. A medicine is called an orphan drug when it is intended for the diagnosis, prevention, or treatment of a life-threatening or seriously debilitating condition that affects no more than 5 in 10,000 people in the EU.
We then focused on pharmacovigilance and off-label use in paediatric settings. Pharmacovigilance plays a crucial role in ensuring patient safety. Off-label use refers to using a medicine for a purpose, in a way, or in a population not approved by the regulatory body. because of factors like difficulties in developing age-appropriate formulations and conducting clinical trials in children, many medicines are prescribed off-label. Off-label use can increase the risk of adverse drug reactions (ADRs) That’s when medication has other side effects that are not wanted.
Again, after our final break, we had our closing session. I would like to thank all of the doctors, researchers, advocates and YPAG groups from around the EU. This was an amazing opportunity and I hope we can do this again next time. I would also like to thank my parents and brothers for support while writing this article. Bye!
EyeYPAG: the YPAG group that is situated at Moorfields Eye Hospital in London.
ERDERA: European Rare Diseases Research Alliance is part of the Horizon Europe program. It is a major European initiative launched to advance research on Rare Diseases and provide real support to the approximately 30 million people affected across Europe. For more information, visit the ERDERA website.
Advocacy: Public support for or recommendation of a particular cause or policy
Patient organisations:non-profit groups focused on representing the interests and needs of patients, their families, and caregivers.
Diagnosis: The identification of the nature of an illness or other problem by examination of the symptoms.
Consent: A legally binding agreement provided by an adult.
Assent: An agreement given by a minor or someone lacking the legal capacity to consent
Orphan drugs: Medications developed to treat rare diseases, which affect a small proportion of the population.
Pharmacovigilance: The science and activities focused on detecting, assessing, understanding, and preventing adverse effects of medicines.
Paediatric settings: A wide range of healthcare environments dedicated to the medical and developmental needs of infants, children, and adolescents.
https://www.cvbf.net/ypag-kids-bari/
https://www.teddynetwork.net/2017/09/25/kids-albania-the-first-ypag-in-albania/
https://www.rainrareresearch.org/rain-crag
Today was my first meetup with the Young People’s Advisory Group for eye research, it was the best time. I got to relate to kids my age, the group made me feel welcome, this allowed me to feel confident when I was speaking. The topics we discussed will have an impact on the future and […]
In this video from the inaugural Generation R meeting in 2013, YPAG members lead a discussion of why medical research is important.
These scenarios have been developed and tested by Generation R YPAGs.
