EURORDIS-Rare Diseases Europe

We’ve just concluded an insightful #CNAMeeting with representatives from the Council of National Alliances and the Council of European Federations! Today’s sessions covered critical areas for the rare disease community, including mental health, healthcare competitiveness, data-driven advocacy using Rare Barometer surveys, newborn screening, and HTA (Health Technology Assessment). 🧠 We highlighted the critical need for better mental health integration in rare disease care. Advocates are calling for holistic care models and greater EU support to address the mental health challenges faced by people with rare conditions. 🔬On EU competitiveness, we explored the ongoing revision of pharmaceutical legislation to ensure a balance between fostering innovation and ensuring affordable access to treatments. Joint procurement could be a viable solution to reduce costs for rare disease therapies. We also stressed the need for a comprehensive EU Action Plan on rare diseases to address disparities in healthcare. 🧑🤝🧑 Rare Barometer surveys, which engage 20,000 people across 92 countries, revealed the lengthy diagnostic journeys many rare disease patients face. The data calls for faster, more equitable access to diagnosis and treatment. 👶 In newborn screening, while many European countries have expanded their programmes, significant disparitiesremain. A harmonised approach is essential to ensure early diagnosis for all newborns. 👶 Regarding HTA, the discussion focused on the importance of patient involvement in joint clinical assessments. Simplifying complex documents and improving educational programmes for patient advocates will be vital for their meaningful participation in decision-making. Through collective advocacy, knowledge exchange, and collaboration, we will continue to push for better mental health support, innovative treatments, and equitable access to healthcare for all people living with a rare disease and their families! Thank you to everyone involved for a productive couple of days! #CNA #RareDiseases #Advocacy #DisabilityRights Latvian Alliance of Rare Diseases, Alliance maladies rares, ACHSE e.V. Allianz Chronischer Seltener Erkrankungen e.V., RD-Portugal, ALAN - MALADIES RARES LUXEMBOURG, FEDER ONG, Genetic Alliance UK, NORBS - National Organisation for Rare Diseases of Serbia, Pro Rare Austria, VSOP

We’re thrilled today to have welcomed representatives of rare disease National Alliances and European Federations to Paris to share best practices, strategic updates, and strengthen our collective networks to better serve the rare disease community across Europe! We tackled critical topics including: ✊ Reflections on the direction of the European Institutions to ensure a European Action Plan on rare diseases remains a priority. 🤝 Updates on the progress of rare disease national plans, reflecting on the importance of collaboration at national and European levels. 👥 Discussions around National Mirror Groups and their role in aligning national needs with European rare disease strategies. We saw how these groups are expanding across Europe to strengthen connections and share best practices. ✅ Important social policy discussions, focusing on disability rights and the need for better integration with rare disease advocacy. The intersection of rare diseases and disability continues to be a key priority. 🌏 A preview of Rare Disease Day 2025! With the slogan 'More Than You Can Imagine,' the campaign will include adaptable posters, social media materials, and videos to amplify awareness globally. We’re excited to see how everyone will engage at the national level. Thanks to all participants for their input! #CNA #RareDiseaseDay2025 #RareDiseases #Advocacy #DisabilityRights Latvian Alliance of Rare Diseases, Alliance maladies rares, ACHSE e.V. Allianz Chronischer Seltener Erkrankungen e.V., RD-Portugal, ALAN - MALADIES RARES LUXEMBOURG, FEDER ONG, Genetic Alliance UK, NORBS - National Organisation for Rare Diseases of Serbia, Pro Rare Austria, VSOP

Waiting to hear back about your #OpenAcademy2025 application? In the meantime, why not apply for the REMEDi4ALL Drug Repurposing Bootcamp! Held back-to-back with Open Academy, in the same location, this training will cover key aspects of drug repurposing, including regulatory pathways, clinical trials, and patient engagement strategies. Role the next steps of your patient advocacy journey into one trip and apply for both training programmes today! ➡️ https://lnkd.in/ejSRQhkF #training #RareDiseases #research #advocacy #MedicinesDevelopment

What does “access to health and care” mean for people with rare diseases? In her article for The Lancet Global Health, Maria Cavaller Bellaubi, EURORDIS Patient Engagement & Therapeutic Development Director, explores how access goes far beyond health services and medicines. She examines its many dimensions - from research to regulation, geography, economy, education, and care - showing how these elements combine to support people with rare diseases. From diagnosis and treatment to overcoming social and financial barriers, true access empowers the rare disease community to live a full life. ☕ Read the full article now: https://lnkd.in/edKuHxq6 #SundayReads #RareDiseases #access #RareDiseases #health #publication

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

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