EURORDIS-Rare Diseases Europe

10 Minutes With Rhiannon Walls: Parenting & rare diseases Becoming a new parent is challenging at the best of times. But, what does this look like when you’re living with a rare disease? In this month’s episode of 10 Minutes With, Rhiannon W. will be joined by Professor Lara Bloom, CNE, CEO of The Ehlers-Danlos Society, rare disease advocate, and new mother. Mark your calendars and make sure to tune in! 🗓 Tuesday 8 October, 2024 ⏰ 5 pm CEST

The latest International Rare Diseases Research Consortium (IRDiRC) publication, "Leaving no patient behind! Expert recommendation in the use of innovative technologies for diagnosing rare diseases", is now live on Orphanet Journal of Rare Diseases with open access! This paper offers key insights and expert guidance on how cutting-edge technologies can revolutionise rare disease diagnostics. A valuable resource for anyone invested in advancing rare disease diagnosis and care. 🔗 https://lnkd.in/eMWbjtHC #RareDiseases #InnovativeDiagnosis #TechForGood #OpenAccess #IRDiRC

Start planning your #RareDiseaseDay 2025 with pledge cards, a poster, social media templates, and more - all available in multiple languages! Join us in leveraging these resources to raise awareness of rare diseases, amplify the voices of the your community, and advocate for equity! By standing together and calling for change, we can drive action to ensure that the unique needs of people living with rare diseases are recognised and addressed! Check them out! 👉 https://lnkd.in/efRbDevB #RareDiseases #RaisingAwareness #campaign #community

Today, European policymakers gathered at the European Parliament for a plenary debate recognising #WorldMentalHealthDay, calling for comprehensive European action on mental health. #WMHD2024 In his opening statement, European Commission Vice-President Margaritis Schinas emphasised the need to support vulnerable groups, ensure equitable access to mental health care, and break the stigma. 🗣️“Vulnerable people… are more affected than others, and that is where we need more help,” said Schinas. Contributions from MEPs, including Ondrej Knotek, Tilly Metz, Kateřina Konečná, and Tomislav Sokol, followed, warning about the dangers of cutting EU health funding. They stressed that mental health challenges are worsening for vulnerable groups and called for a holistic, comprehensive strategy that includes prevention. 🗣️ “Mental health cannot be separated from physical health, and we must make it a priority,” stated MEP Tomislav Sokol. In addition to the MEPs' interventions, Commissioner Stella Kyriakides, in her statement for World Mental Health Day, highlighted the significant toll on mental health from the COVID-19 pandemic, the Russian invasion of Ukraine, and economic crises. She reaffirmed the EU's commitment to prioritising mental health, with dedicated actions and initiatives. Learn more about the mental wellbeing challenges facing our community: ➡️ https://lnkd.in/ehpmZ7Ep #MentalHealth #MentalWellbeing #RareDiseases #Europe

This #WorldMentalHealthDay, we are focusing on the importance of good mental health in the workplace. 🧑💼💻 Living with a rare disease can have a significant and accumulating impact on mental health and wellbeing. This is especially true for those juggling the responsibilities of care - whether for themselves or a loved one - alongside the need to earn a living. The rare disease community has identified, as a top priority, the need to look beyond the physiological symptoms of rare diseases and take concrete action to address the psychological impact associated with these complex conditions. Proper, 360° holistic care, encompassing healthy, supportive, and accessible working environments, is essential for the rare disease community to thrive and reach their full potential. Learn more about the mental health challenges facing our community! ➡️ https://lnkd.in/ehpmZ7Ep #MentalHealth #MentalWellbeing #RareDiseases

Today, at the 38th Workshop of the EURORDIS Roundtable of Companies (ERTC), we’re discussing the upcoming EU Regulation on Health Technology Assessment (HTA), set to come into effect in January 2025. This regulation will introduce a collaborative framework for EU Member States, including new guidelines for joint clinical assessments of medicines and medical devices. EURORDIS has identified six conditions for successful Joint Clinical Assessment (JCA): 🧑🤝🧑 Well-resourced National HTA Bodies Member States must ensure their national HTA bodies are properly staffed and prepared to integrate joint reports into their workflows, avoiding duplication of efforts. 📑 Reasonable Scope of Assessment (PICO) A balanced number of PICO (Population, Intervention, Comparator, Outcome) requests should be made. We recommend limiting this to three for efficient assessments. ⏱️ Adequate Time for Developers Sufficient time must be given to developers to respond to PICO requests. 🤝 Ensure Transparency Fostering a culture of transparency over exclusion is essential to protect research integrity and foster innovation. 🙋 Meaningful Involvement of Patients and Healthcare Providers Patients should play an active role, rather than just reviewing documents. Their involvement, alongside healthcare providers, is critical to a patient-centred assessment process. ✅ JCA Must Demonstrate Value The joint clinical assessments must provide clear benefits and avoid unnecessary hurdles that could undermine trust in the process. To emphasise the importance of this last condition, today’s sessions featured perspectives from a range of stakeholder groups on their role in the clinical assessment of medicines. Breakout sessions covered topics including methodological guidelines and timeframes in joint clinical assessments and novel approaches to assess real-world medical data using new technologies. Want to learn more about #HTA? 👉 https://lnkd.in/eJYh_Q9g Transparent public-private relationships between patient advocates, industry, academia and decision makers are essential to shape policies that best serve the interests of our community. Learn more about the ERTC on our website. #collaboration #industry #relationships #workshop #cooperation #meeting

📣 Calling all PhD researchers and those up to 2 years post-PhD! Do you want to expand the impact of your work on rare disease medicines research and development? Now is your chance! The comprehensive and interactive #OpenAcademy2025 programme is tailored to the specific needs and challenges of the rare disease field and offers unique insights into clinical trial methodology, regulatory pathways, and health technology assessment. The knowledge base provided enables researchers to focus their own research efforts on evolving patient needs, which will in turn help to minimise the risks of failure. Researchers leave our training programme with new ideas applicable to their research, extended multi-stakeholder networks for future research collaborations, and the knowledge to enhance their influence in the rare disease scientific community! Apply now! 👉 https://lnkd.in/ejSRQhkF #training #RareDiseases #research #advocacy #MedicinesDevelopment

Join us for the next in our mental health and wellbeing webinar series, How to Deliver a Diagnosis. This webinar will explore the critical moment of delivering a rare disease diagnosis for families and individuals impacted by rare conditions. Experts from European Reference Networks will share real cases and best practices, followed by a panel discussion with leading specialists about best practices in delivering a rare disease diagnosis. 💬 Mental health open webinar: How to deliver a diagnosis 🗓️ 23 October 2024 ⏰ 2:00 pm to 3:30 pm CEST ➡️ https://lnkd.in/eY5rXk3M #RareDiseases #Diagnosis #webinar #MentalHealth #MentalWellbeing

The news of Sammy Basso’s passing deeply saddens us. More than an advocate and former Black Pearl Awardee, Sammy used his experience with Hutchinson-Gilford to become a powerful voice for change. At just nine, he founded l’Associazione Italiana Progeria Sammy Basso. Later on, he became a molecular biologist and researcher, and succeeded in uniting people in raising awareness and supporting research on his condition and rare diseases in general. His love for life shone through his travels, as seen in the documentary Il viaggio di Sammy, and his warm connections with everyone he met, including many at EURORDIS. We are holding his family, friends, and all who were inspired by his remarkable life in our hearts. 🕊️

Don’t miss out on our next episode of 10 Minutes With Rhiannon W.! 🗓 Tuesday 8 October, 2024 ⏰ 5 pm CEST This month, Rhiannon will be joined by Professor Lara Bloom, CNE, CEO of The Ehlers-Danlos Society, to discuss her rare disease journey. From her former career, through her diagnosis journey, to the hectic joy of becoming a first-time mother, it promises to be an exciting and varied conversation! Make sure to register to attend for notifications when we go live! 🔴 https://lnkd.in/eyTHzjEb