Together for Rare Diseases

⚡ A new Intergroup on Cancer and Rare Diseases   As of this week, there is a dedicated group of MEPs working to address the unmet needs of people living with cancer and rare diseases in the EU.   Led by our MEP Champion Stine Bosse, alongside MEPs Vlad Voiculescu, Tilly Metz, ALESSANDRA MORETTI and Tomislav Sokol, the intergroup will work with civil society to develop and propose policy recommendations, collaborate on policy initiatives and advocate for the highest standards of healthcare, social care and research for people living with rare diseases.   💯 Congratulations to our partners EURORDIS-Rare Diseases Europe, as well as the European Cancer Organisation and SIOP Europe, the European Society for Paediatric Oncology (SIOPE) for this initiative.   https://lnkd.in/e6ymCDrV.

ERNs are a powerhouse of information and knowledge. The opportunities the evidence they collect in registries is one example of how important they are to partner with when considering research in rare diseases. Thank you ERKNet for inviting me to speak at this meeting about Together for Rare Diseases where we are looking to advance opportunities for ERN and Industry collaboration. Collective efforts, breaking down silos and perceived barriers will allow these partnerships to flourish enabling the rare disease research landscape to thrive. I urge industry stakeholders, policymakers and researchers to look for more collaboration opportunities so we can continue to advance knowledge that will benefit living with rare disease. #raredisease #collaboration

The Rare Disease Moonshot and Together for Rare Diseases coalitions are advocating for a transformative vision to reimagine the European Reference Networks (ERNs) as research powerhouses, with public-private partnerships (PPPs) at their core. While we believe that the ERNs’ original (and current) focus of supporting cross-border healthcare remains a critical aspect of the European Rare Disease collaboration, we also consider that ERNs have the potential to become research powerhouses with PPPs at their core. The following actions would ensure ERNs become key partners in the rare disease research continuum: 👉 Provide the resources (human and funding) to support their increase research activities and become ‘research-ready’ networks to be further integrated in the European Rare Diseases Research Alliance (ERDERA). 👉 Scale up the ambition on research activities within ERNs, making them a core component of their mission and evaluation criteria. 👉 Develop a framework for meaningful PPPs that leverage both network and industry expertise in scientific, regulatory, and operational domains. 👉 Create incentives for ERNs to engage in innovative research initiatives, including preclinical research, natural history studies and clinical trials. 👉 Establish clear metrics for assessing ERNs' research output and impact, including collaborative projects with industry partners. 👉 Integrate ERNs more deeply and consistently across the network of centres into the broader rare disease research ecosystem, fostering connections with fundamental research institutions, pharmaceutical and biotech companies. 👉 Support the empowerment of patient organisations and ePAGs to engage effectively and proactively in clinical research. By leveraging the combined expertise of healthcare providers and industry partners, we can accelerate scientific breakthroughs and bring hope to millions affected by rare diseases. EURORDIS-Rare Diseases Europe EFPIA - European Federation of Pharmaceutical Industries and Associations EUCOPE - European Confederation of Pharmaceutical Entrepreneurs EuropaBio - the European Association for Bioindustries EATRIS BBMRI-ERIC ECRIN (European Clinical Research Infrastructure Network) Critical Path Institute (C-Path) ERDERA Magda Chlebus Anton Ussi Daria Julkowska Mathieu Boudes, PhD Cécile Ollivier Roseline Favresse Victor Maertens Stefania Alessi Adrien Samson Marta del Álamo Manuela Pausan International Rare Diseases Research Consortium (IRDiRC) Sheela Upadhyaya Clara Romero Hélène Dollfus Maurizio Scarpa Franz Schaefer Alexis Arzimanoglou Ines Hernando Graham Slater Ana Rath Orphanet Rima NABBOUT-TARANTINO Toon Digneffe Anne-Sophie Chalandon Morgane Cuisenier Estelle Michael Matteo Scarabelli Victoria Hedley https://lnkd.in/dN6KvMsv

✍ The European Health Data Space was adopted last week, which could give a 𝐡𝐮𝐠𝐞 𝐛𝐨𝐨𝐬𝐭 𝐭𝐨 𝐫𝐞𝐬𝐞𝐚𝐫𝐜𝐡 𝐚𝐧𝐝 𝐭𝐡𝐞𝐫𝐚𝐩𝐢𝐞𝐬 𝐟𝐨𝐫 𝐫𝐚𝐫𝐞 𝐝𝐢𝐬𝐞𝐚𝐬𝐞𝐬. Here’s why:   🔓 Registries: data registries carry significant potential for better and understanding and meeting needs of people living with rare diseases, as long as data is fully exploited with a streamlined and collaborative approach. The EHDS will create a strong system of data governance and rules for data quality, providing a template for industry and ERNs to follow. 🇪🇺 🌍 Cross-border data sharing: many people living with a rare disease have to travel to another country to receive treatment. The EHDS should encourage data sharing (in a secure manner), enhance research opportunities, and speed up diagnosis. 🤝 Common rules for data sharing: there are different rules and standards for how this is done across Europe. Having one clear system will help patients, researchers and healthcare professionals understand how and where data can be used.   ➡️ Read more on this from EURORDIS-Rare Diseases Europe: https://lnkd.in/eQGgzP6e 

While several partnerships between ERNs and industry have launched in recent years, the value that pharmaceutical companies bring to rare disease research is not yet fully understood. Our 2nd webinar with #ERICA brought together 40+ researchers, industry, diagnostics, patient representatives and policymakers to highlight 𝙬𝙝𝙖𝙩 𝙞𝙣𝙙𝙪𝙨𝙩𝙧𝙮 𝙘𝙤𝙣𝙩𝙧𝙞𝙗𝙪𝙩𝙚𝙨 𝙩𝙤 𝙧𝙖𝙧𝙚 𝙙𝙞𝙨𝙚𝙖𝙨𝙚 𝙧𝙚𝙨𝙚𝙖𝙧𝙘𝙝 𝙗𝙚𝙮𝙤𝙣𝙙 𝙛𝙪𝙣𝙙𝙞𝙣𝙜. Vinciane Pirard of Sanofi outlined why industry knowledge and expertise is crucial to scaling up rare disease research, given their: 🔹 Scientific and regulatory expertise 🔹 Access to infrastructure and assets 🔹 Global operational capabilities and capacity 🔹 Innovation-driven approach Luca Sangiorgi delved into the successful partnership on 𝘰𝘴𝘵𝘦𝘰𝘨𝘦𝘯𝘦𝘴𝘪𝘴 𝘪𝘮𝘱𝘦𝘳𝘧𝘦𝘤𝘵𝘢 forged between ERN BOND - European Reference Network on Rare Bone Diseases and Sanofi, the steps needed to kick off the project, and the growing and mutual understanding of the 'what's in it for me.' Anton Ussi of EATRIS, the European infrastructure for translational medicine, explained that industry's experience in meeting regulatory standards could help researchers improve regulatory-grade outcomes in academic research. 💬 Vinciane Pirard: "𝙋𝙋𝙋𝙨 𝙖𝙧𝙚 𝙖𝙣 𝙚𝙘𝙤𝙨𝙮𝙨𝙩𝙚𝙢 - 𝙖 𝙘𝙤𝙢𝙢𝙪𝙣𝙞𝙩𝙮 𝙤𝙛 𝙗𝙧𝙖𝙞𝙣𝙨 where everyone brings knowledge, perspectives and resources. Mutual understanding and a 𝙜𝙧𝙤𝙬𝙞𝙣𝙜 𝙩𝙧𝙪𝙨𝙩 𝙖𝙣𝙙 𝙧𝙚𝙨𝙥𝙚𝙘𝙩 𝙤𝙫𝙚𝙧 𝙩𝙞𝙢𝙚 𝙖𝙧𝙚 𝙘𝙧𝙞𝙩𝙞𝙘𝙖𝙡 to the success of these partnerships" 💬 Luca Sangiorgi: "It was 𝙖 𝙧𝙚𝙖𝙡 𝙘𝙤-𝙘𝙧𝙚𝙖𝙩𝙞𝙤𝙣 𝙚𝙭𝙥𝙚𝙧𝙞𝙚𝙣𝙘𝙚 that brought benefits to both sides" 💬 Anton Ussi: “Industry helps 𝙙𝙚𝙥𝙡𝙤𝙮 𝙧𝙚𝙨𝙚𝙖𝙧𝙘𝙝 𝙞𝙣𝙩𝙤 𝙧𝙚𝙖𝙡 𝙬𝙤𝙧𝙡𝙙 𝙨𝙚𝙩𝙩𝙞𝙣𝙜𝙨. Being able to work with companies that understand industrial processes helps bring in a multidisciplinary mindset to 𝙚𝙣𝙨𝙪𝙧𝙚 𝙮𝙤𝙪𝙧 𝙥𝙪𝙗𝙡𝙞𝙘𝙖𝙩𝙞𝙤𝙣 𝙞𝙨 𝙖 𝙗𝙚𝙜𝙞𝙣𝙣𝙞𝙣𝙜 𝙧𝙖𝙩𝙝𝙚𝙧 𝙩𝙝𝙖𝙣 𝙖𝙣 𝙚𝙣𝙙” ➡️ Read more about webinar here: https://lnkd.in/eZx3rtCW 💡 Interested in a forum for collaboration between ERNs and industry? Get in touch at secretariat@together4rd.eu!

📢 𝑪𝒂𝒍𝒍𝒊𝒏𝒈 𝒂𝒍𝒍 𝑬𝑹𝑵𝒔 - find out more about what industry can bring to rare disease research   Public-private partnerships (PPPs) hold the 🔑 key to accelerating rare disease research. Beyond funding, what does industry provide to PPPs?   Join us on Thursday for our 2nd webinar with #ERICA, where Luca Sangiorgi, Vinciane Pirard and Anton Ussi will join our chair, Sheela Upadhyaya, to highlight how specialised expertise, cutting-edge technologies and global infrastructure can complement academic strengths to create synergies that drive innovation.   📅 Thursday 16 January 🕔 5 - 5:45pm CET 🔗 https://lnkd.in/ehRhNTD4 ERN BOND - European Reference Network on Rare Bone Diseases | Endo- ERN | ERKNet | ERN CRANIO | ERN EpiCARE - Rare and Complex Epilepsies | ERN eUROGEN | EURACAN ERN-EuroBloodNet (European Reference Network on Rare Hematological Diseases) | EURO-NMD European Reference Network for Rare Neuromuscular Diseases | ERN-EYE | ERN GUARD-Heart | ERN GENTURIS | ERNICA | ERN ITHACA | ERN-LUNG | ERN RARE-LIVER | ERN ReCONNET | ERN-RITA | ERN-RND, European Reference Network for Rare Neurological Diseases | Ern-Skin | ERN TransplantChild | VASCERN, European Reference Network on Rare Multisystemic Vascular Diseases

💡 2025 will be an important year for Together For Rare Diseases, as we continue to build bridges in the European rare disease research community. 🤝 Our 2nd webinar with #ERICA will take place next Thursday, 16 January, outlining how collaboration with industry can accelerate rare disease research and giving examples of how this is materialising in a partnership between ERN BOND - European Reference Network on Rare Bone Diseases and Sanofi. 🔗 Register here or scan the QR code below: https://lnkd.in/ehRhNTD4 Sheela Upadhyaya Luca Sangiorgi Vinciane Pirard Anton Ussi Sanofi Novo Nordisk UCB Takeda EFPIA - European Federation of Pharmaceutical Industries and Associations EUCOPE - European Confederation of Pharmaceutical Entrepreneurs ERDERA EURORDIS-Rare Diseases Europe Rare Disease Moonshot Toon Digneffe Anne-Sophie Chalandon Morgane Cuisenier Gabriella Almberg Matteo Scarabelli Stefania Alessi Hélène Dollfus Isabella Anna Vacchi Maurizio Scarpa Franz Schaefer Alexis Arzimanoglou Matt Bolz-Johnson Ines Hernando Daria Julkowska Ana Rath Yanis Mimouni Rima NABBOUT-TARANTINO

🎞️ 𝐏𝐫𝐞𝐬𝐞𝐧𝐭𝐢𝐧𝐠 𝐨𝐮𝐫 𝐚𝐜𝐡𝐢𝐞𝐯𝐞𝐦𝐞𝐧𝐭𝐬 𝐢𝐧 2024🌟   None of these would be possible without the support and cooperation we have received from the rare disease research community across Europe.   Thank you to: 🔹 T4RD's sponsors, Toon Digneffe, Anne-Sophie Chalandon, Morgane Cuisenier, Gabriella Almberg, Matteo Scarabelli and Stefania Alessi from Takeda, Sanofi, Novo Nordisk, UCB, EFPIA - European Federation of Pharmaceutical Industries and Associations and EUCOPE - European Confederation of Pharmaceutical Entrepreneurs 🔹Our Steering Group members, Hélène Dollfus, Isabella Anna Vacchi, Maurizio Scarpa, Franz Schaefer, Alexis Arzimanoglou, Matt Bolz-Johnson, Ines Hernando, Graham Slater, Daria Julkowska, Ana Rath, Anton Ussi, Yanis Mimouni and Rima NABBOUT-TARANTINO 🔹 Our MEP Champion Stine Bosse and her office 🔹 The Board of Member States of ERNs, and their Chair Birute Tumiene 🔹 The European Commission's DG Research and Innovation, in particular Hélène Le Borgne and Christina Kyriakopoulou 🔹 ERDERA and Rare Disease Moonshot 🔹 And to everyone who participated in our workshops, webinars and events in 2024!

💬 "Registries provide multiple opportunities to collaborate with industry"   Speaking in Udine this morning, our Steering Group member and ERKNet coordinator Franz Schaefer encouraged ERNs to explore possibilities to work with industry partners through registries.   Registries carry significant potential for better understanding and meeting the needs of people living with a rare disease, and our pilot projects between ERNs and industry have demonstrated the progress that can be delivered when registry data is fully exploited with a streamlined and collaborative approach.   Sheela Upadhyaya also presented Together4RD's proposal for a Forum for Exchange of Information, a collaborative platform that facilitates exchanges between ERNs and industry, promoting mutual understanding and fostering joint projects.

Together 4 Rare’s new MEP Champion is Stine Bosse! 🌟   The Danish Vice-Chair of the SANT Subcommittee shares our commitment to addressing the unmet needs of over 3️⃣0️⃣ 𝗺𝗶𝗹𝗹𝗶𝗼𝗻 𝗽𝗲𝗼𝗽𝗹𝗲 𝗶𝗻 𝗘𝘂𝗿𝗼𝗽𝗲 𝘄𝗵𝗼 𝗹𝗶𝘃𝗲 𝘄𝗶𝘁𝗵 𝗮 𝗿𝗮𝗿𝗲 𝗱𝗶𝘀𝗲𝗮𝘀𝗲, and has endorsed our mission to boost ERN-industry cooperation.   MEP Bosse has called for an European Action Plan on Rare Diseases – a call we fully echo and hope the new European Commission and new members of the European Parliament will pick up on in the new mandate.    While significant progress was made in the last 5 years to promote cross-border research and action to support people living with rare diseases, there are still significant gaps in research, funding and collaboration that we, alongside MEP Bosse, will strive to fill.