The importance of educating patients and families
about about the science of rare diseases
On the 28th February, we commemorated Rare Disease day. The majority of rare diseases affect children, with an estimated 70% having a Paediatric onset (source www.eurordis.org). There is a huge, unmet need to develop effective drugs for children with rare diseases. Currently, only 5% of rare diseases have an effective treatment option. c4c is committed to improving the clinical trial infrastructure to create better medicines for all children and young people, including those with a rare disease.
To mark Rare Disease day, we wanted to highlight the importance of education in empowering patients and families to become involved in research. This article presents the role that education in basic scientific concepts plays to facilitate patients, families and caregivers to make well-informed decisions about their health, therapeutic options and participating in research, using the example of the Rare Disease project Share4Rare.
Science shapes our daily lives — see for example to what extent the COVID-19 pandemic outbreak has affected us —, influencing many of the decisions we make as individuals, which in the end have an impact on society. The way we manage our health, the way we eat, the way we take care of the environment. Knowing how things in the scientific sphere actually work will lead us to make better decisions. If this happens at a society level, what happens if we focus on the community of patients and caregivers?
In general terms, patient education improves adherence to therapeutic regimens and helps patients adopt behaviours that could prevent disease. Educating patients on the science behind their disease and the ins and outs of the research process is also empowering. It helps them understand the disease mechanisms and learn about the potential therapeutic options and, therefore, they will more likely make well-informed decisions, get involved in advocacy and awareness, participate in basic research projects or enroll in a clinical trial.
Share4Rare educates patients and families to support their participation in research. In this context, Share4Rare works with a wide-ranging group of experts on biomedical sciences and communication to create educational contents that will help the rare disease community improve their scientific knowledge. All contents are free of charge and open source. From medical books containing exhaustive and updated information on different rare diseases to blogs portraying scientific breakthroughs in drug development or disease models and educational webinars hosted by researchers or science communicators explaining the natural history of a specific disease, basic concepts of genetics or disease diagnosis or how to tackle pseudoscience and find trustworthy medical information. We always rely on highly qualified professionals to help us create these contents and host these events, considering whether the focus is appropriate and ensuring the implication of patients and families in the revision process.