AxTalis

𝐒𝐌𝐄𝐬 𝐚𝐧𝐝 𝐭𝐡𝐞 𝐄𝐌𝐀: 𝐏𝐫𝐨𝐦𝐨𝐭𝐢𝐧𝐠 𝐢𝐧𝐧𝐨𝐯𝐚𝐭𝐢𝐨𝐧 𝐚𝐧𝐝 𝐭𝐡𝐞 𝐝𝐞𝐯𝐞𝐥𝐨𝐩𝐦𝐞𝐧𝐭 𝐨𝐟 𝐧𝐞𝐰 𝐦𝐞𝐝𝐢𝐜𝐢𝐧𝐞𝐬 A couple of weeks ago, our new Regulatory Affairs Manager Sam Bryant took part in the EMA Small and Medium Enterprise (SME) Information Day. He says: “In a previous role as Regulatory Affairs Manager at a small Biotech I have first-hand experience of the benefits that SME status can bring. It is pleasing to see that the EMA continues to recognise SMEs as important actors in innovation and the development of new medicinal products. Incentives are provided to help SMEs overcome some of the financial and administrative hurdles associated with bringing a product to market.”  To find out more about how your company could benefit from SME status, please read our latest blog: https://lnkd.in/eivjkndV #RegulatoryAffairs #SMEs #EMA

𝐊𝐞𝐲 𝐭𝐚𝐤𝐞𝐚𝐰𝐚𝐲𝐬 𝐟𝐫𝐨𝐦 𝐭𝐡𝐞 𝐖𝐨𝐫𝐥𝐝 𝐎𝐫𝐩𝐡𝐚𝐧 𝐃𝐫𝐮𝐠 𝐂𝐨𝐧𝐠𝐫𝐞𝐬𝐬 𝐄𝐮𝐫𝐨𝐩𝐞 🌍   As the World Orphan Drug Congress in Barcelona concludes, Sven Tops and Frank Vanderdonck reflect on some key takeaways from the conference. Attending the event was a great opportunity to listen to and contribute to discussions on evidence needs and standards for rare and ultra-rare diseases:   - Flexibility in evidence standards: Health Technology Assessment (HTA) bodies often have rigorous evidence thresholds, yet randomised controlled trials (RCTs) are often challenging or unfeasible for many rare or ultra-rare diseases. Pragmatic evidence approaches—such as case reports, registries, and natural disease history studies—should be recognised as viable alternatives.   - Adapting payment models : Linking pragmatic evidence generation directly to payment models could ensure faster access for patients who urgently need new and highly specialised treatments. This would support great access and timely care without compromising the evidence base.   - Incorporating patient and caregiver voices: It's essential that the patient and caregiver perspectives are actively included in evidence generation and data presentation, especially within HTA evaluations. Their insights are invaluable in ensuring a holistic understanding of disease impact. While data can show the efficacy of a treatment, patients and caregivers provide an important insight into how that treatment will impact daily life, whether it improves their quality of life or addresses unmet needs.   - Championing a European Action Plan for rare diseases: The keynote panel highlighted the need for political commitment and shared accountability across the EU to drive the plan’s success and ensure meaningful impact. There was a focus on the need to align existing regional, national, and supranational initiatives in order to promote continued innovation and access to new treatments.   We are looking forward to seeing how these perspectives can help shape the future of rare disease treatment in Europe, and greater patient access.     #WorldOrphanDrugCongress2024 #WODC #RareDiseases #HTA

New EU Regulation Alert Last week, the second Implementing Act of the new EU HTA Regulation was adopted. The Implementing Regulation (EU) 2024/2699 details procedural rules for cooperation between the HTA coordination group and the European Medicines Agency (EMA).  Key highlights include: - Information exchange: streamlined processes for sharing data on medicinal products and medical devices.  - Expert involvement: clear guidelines for identifying and engaging clinical and technical experts.  - Confidentiality: robust measures to protect sensitive information.  - IT integration: secure IT platform for efficient data exchange. This implementing act aims to enhance collaboration and ensure high standards in health technology assessments across the EU. 🌍💊🔬 You can find a more detailed summary of the Implementing Act below. If you would like to discuss any of these points, please reach out to our team directly. #EUHTA #ImplementingRegulation #EU

𝐖𝐞𝐥𝐜𝐨𝐦𝐢𝐧𝐠 𝐃𝐚𝐧𝐢𝐞𝐥 𝐂𝐚𝐩𝐫𝐚 𝐭𝐨 𝐭𝐡𝐞 𝐈𝐓 𝐓𝐞𝐚𝐦 👋   We are excited to welcome Daniel C., our new IT Support Associate at MAP x AxTalis! With a passion for all things tech, Daniel brings expertise in application development and IT infrastructure. Before transitioning to the tech world, Daniel worked as a motion designer in the Design & Animation industry, collaborating with high-profile brands like Saturday Night Live and YouTube Music. Now, he's on a mission to enhance and expand our IT systems, employing his skills to drive innovation and accessibility.   Daniel says: "I love pushing technology to its limits and making it more accessible to everyone. Whether it's building apps, automating tasks, or managing web servers, I find joy in using technology to improve people's lives."   Outside of work, you can find him exploring the virtual worlds of video games, taking long walks and enjoying his favourite music. A warm welcome to Daniel and we are looking forward to seeing your growth in this role!   #technology #welcometotheteam #innovation #IT

𝐖𝐞𝐥𝐜𝐨𝐦𝐞 𝐭𝐨 𝐒𝐚𝐦 𝐁𝐫𝐲𝐚𝐧𝐭, 𝐨𝐮𝐫 𝐧𝐞𝐰 𝐑𝐞𝐠𝐮𝐥𝐚𝐭𝐨𝐫𝐲 𝐀𝐟𝐟𝐚𝐢𝐫𝐬 𝐌𝐚𝐧𝐚𝐠𝐞𝐫 👋 With extensive experience in Regulatory Affairs and Clinical Development, Sam Bryant has worked in SMEs, and on behalf of biotech and pharma at a top-5 CRO. He has successfully led regulatory interactions in support of clinical development of innovative products, including most recently for a cell therapy ATMP. At MAP x AxTalis, Sam will be helping our clients advance their products through the complex regulatory landscape.   He says: “I am passionate about bringing live-saving and life-changing therapies to patients; for me Regulatory Affairs is key to transforming fascinating science and exciting ideas into approved treatments that can transform patients' lives.” When Sam is not busy understanding new regulations, he likes to spend as much time as possible outside; exploring French rivers by canoe, roads by bike, and trails on foot, or simply enjoying the garden with his family. A very warm welcome to the team, Sam - it's great to have you on board! #regulatoryaffairs #welcometotheteam

𝐌𝐚𝐫𝐤 𝐲𝐨𝐮𝐫 𝐜𝐚𝐥𝐞𝐧𝐝𝐚𝐫𝐬 𝐟𝐨𝐫 𝐈𝐒𝐏𝐎𝐑 𝐄𝐮𝐫𝐨𝐩𝐞 𝐢𝐧 𝐁𝐚𝐫𝐜𝐞𝐥𝐨𝐧𝐚! 🗓️   In 5 weeks' time our team will attend ISPOR—The Professional Society for Health Economics and Outcomes Research Europe. We are preparing to join the global HEOR community, and are looking forward to presenting some new research posters. Attending will be: - Sven Tops, Chief Market Access Officer - Melanie van Riemsdijk, Director Market Access Benelux - Jo Wallis, Vice President, Early Strategic Advice and Market Access UK/IRE - Maciej Maruszczak, Health Economics, Operational & Data Insights Lead - Monica Ricci, Ph.D., Senior Medical & Market Access Manager - Ben Hendry, Associate Director, Market Access - Praptee Ghimire, Consultant, Value Communication - Xingyao Liu, Consultant, Health Economics and Outcomes Research - Daniel Harrop, Senior Consultant, Health Economics and Outcomes Research - Kristof Theys, Health Economics and Data Science Manager   If you would like to meet up with our team, please feel free to reach out to them directly via LinkedIn direct message. 🤝   #ISPOR #ISPOREurope #HealthEconomics

Dear colleagues tomorrow, October 15th, I will be pitching at the Advanced Therapies in Belgium event in Namur. I will give an update on the new EU HTA regulation roling out for ATMPs and Oncology treatments, as from January 12th 2025. Happy to take any questions you might have.

Wim Neckebroeck shares his reflections on last week's ATMP Symposium by EMA 👇

The European Commission (EC) has announced a consultation on Joint Scientific Consultations (JSC) by 29 October 2024. JSCs are a core part of the new EU Health Technology Assessment Regulation (EU HTAR) , enabling early engagement between health technology developers and the EU HTA as well as the European Medicines Agency (EMA) to streamline the preparation for joint clinical assessments (JCAs). MAP x AxTalis will be responding to the consultation and inviting our members to provide their feedback and insights on the proposed JSC process. #JSC #EUHTA #JCA 

𝐄𝐔 𝐇𝐓𝐀 𝐢𝐬 𝐜𝐨𝐦𝐢𝐧𝐠 Oncology and ATMP products will be required to go through the EU HTA from January 2025. With 3 months to go, we are continuing to advise our clients on steps they can take to prepare. An important first step is to familiarise yourself with the key milestones of the EU JCA process ⬇ If you would like to discuss any of these points in more detail with our consultancy teams, please do reach out 📞   #EUHTA #JCA #HTA