Save Sight Now Europe’s Post

Excited for #WODC Europe next week? Join us for two dynamic panels with Dolon's Elena Nicod alongside industry leaders and policy experts, where they’ll tackle critical challenges in the rare disease and orphan product space. Panel 1: Wednesday, 23rd October, 4:40pm Topic: Evidence Assessment in Rare Diseases: Why We Need a Paradigm Shift. The work of HTAi RDIG Join Elena Nicod with Farzana Malik Ph.D, Alicia Granados, MD, PhD PH, Monica Ferrie, and Sheela Upadhyaya as they explore the evolving role of HTA in rare disease evidence and the groundbreaking work of the HTAi Rare Diseases Interest Group. Panel 2: Thursday, 24th October, 2:40pm Topic: Access and HTA Pathways for Orphan Medicinal Products (OMPs): Are They Really Driving Innovation? Delve into access and incentive structures with Lisa Marsden, Nick Meade, David Kolář, and Elena as they discuss whether current HTA pathways are effectively improving access for OMPs and fostering innovation in this crucial field. Don’t miss these insightful sessions and make sure to add them to your agenda and join the conversation! #WODCEU #RareDiseases #MarketAccess #Dolon World Orphan Drug Congress Europe

News from the other side of the globe! 🇦🇺 #LaunchR is a new network of Western Australian, national, and international rare disease experts dedicated to developing, providing access to, and manufacturing of, precision medicines for rare disease. #LaunchR also stimulates the pathways to translate these to impact for common diseases. The network leverages WA's expertise in rare disease precision medicine, particularly, but not only, in antisense oligonucleotides to pioneer personalised therapies for rare diseases. #LaunchR brings together initiatives for integrated pipelines for diagnosis, drug discovery, trials and treatment access; and new local (drug) manufacturing industry. As part of LaunchR’s initiatives, through the #RareCareCentre and a network of public-private partnerships, an expanding paediatric rare diseases clinical trials and treatment ecosystem, called TrialR, to administer these cutting-edge medicines, is in deployment. Additionally, LaunchR will prioritise equity by advancing access to treatments and clinical trials for underrepresented populations, including First Nations Australians, for example through Lyfe Languages (the Universal Indigenous Medical Translator); and for those living in remote regions, through teletrials partnerships. #LaunchR is fuelled by the Rare Diseases Moonshot-Australia (RDMA). With linkages to local, national and international teams, #LaunchR represents a crucial step forward in addressing the unmet needs of people living with rare diseases and their families, a staggering of 95% of rare diseases lack a specific and prognosis altering therapy. Gareth Baynam International Rare Diseases Research Consortium (IRDiRC)

NextCell Pharma AB’s CSO Lindsay Davies is in Portugal for #ATE2024 with CEO Mathias Svahn. Lindsay will present the company’s long term clinical data for #t1d and the company’s vision moving forward. She is also presenting in the #supplychain session on #patientaccess and how ProTrans has been developed to ensure its #offtheshelf accessibility to #patients irrespective of geographical location or the need for specialist clinics. Putting the patient first, the ethos of our #development #strategy at NextCell. #cgt #celltherapy #cellandgenetherapy #atmps #europe #portugal #commercialization #type1diabetes #autoimmune

Andrew attended World Orphan Drug Congress USA last week and shares his key take aways. "The conference, as ever, was a great way to connect with friends and peers in the rare disease community. Key themes in the access track were naturally, changes that are coming in Europe and the US, although the conference was not dominated by these. During the final day plenary sessions 2 of our current client companies took to the stage to discuss planning for access. Both talked about the need to plan early, resource your access and HEOR, and consider all options. Sage advice, especially given the complexity we see in are and ultra rare. Both talked about the need for choosing the right partners, which you can imagine gives a sense of pride, knowing that we are their partners. In other tracks we heard from patient advocates talking about the need for patients to be heard and incorporated into access decision making, along with great success stories where this is happening right now. It was also good to hear that investors are back at the table for biotech developers given the hiccups in ’22 and ’23 for longer term investment decisions. Patients living with rare conditions will ultimately benefit from these decisions." #raredisease #WODC #networking #marketaccess #heor

𝐓𝐡𝐞 𝐫𝐞𝐜𝐨𝐫𝐝𝐢𝐧𝐠 𝐚𝐧𝐝 𝐊𝐞𝐲 𝐓𝐚𝐤𝐞𝐚𝐰𝐚𝐲𝐬 𝐟𝐫𝐨𝐦 𝐭𝐡𝐞 𝐰𝐞𝐛𝐢𝐧𝐚𝐫 𝐂𝐥𝐢𝐧𝐢𝐜𝐚𝐥 𝐓𝐫𝐢𝐚𝐥𝐬 𝐢𝐧 𝐔𝐤𝐫𝐚𝐢𝐧𝐞  Over 142 industry professionals tuned in live. If you couldn't join the webinar, here's an opportunity to watch the full recording here: https://lnkd.in/exA53syy 𝐊𝐞𝐲 𝐓𝐚𝐤𝐞𝐚𝐰𝐚𝐲𝐬: As of April 2024, 290 clinical trials were active across various therapeutic areas in Ukraine. Importantly, 75 new clinical trials were approved by Ukrainian regulatory authorities in 2023-2024 (through September), including studies from top sponsors such as AstraZeneca, MSD, Sanofi, Teva, and Janssen Pharmaceutica NV, as well as other middle and small-sized sponsors. The country offers faster study start-up times compared to many European countries, with regulatory review timelines reduced to 30 days for new trial applications and 25 days for substantial amendments. Ukraine consistently ranks as a top-enrolling country in global trials, often exceeding recruitment targets. Over 95% of surveyed sites report full operational capacity, with adequate investigational medicinal product supply and logistics for biosample shipments. While acknowledging ongoing conflict, the webinar emphasized that the majority of Ukraine's research infrastructure in western and central regions remains fully operational. Sponsors are encouraged to focus on these low-risk areas to maximize trial success while mitigating potential challenges.

🌟 It was an honour to participate in the Medtech Malta 2024 panel discussion, "From Lab Bench to Bedside Care: What Does It Take?" As someone deeply involved in the commercialization of cancer diagnostics, I had the opportunity to share insights into some of the most complex challenges in transforming research into real-world solutions. Our discussion focused on several key obstacles: 📝 Regulatory Documentation - Navigating the intricate documentation requirements, from initial concept through to prototype. 🔐 IP and Development Agreements - Setting up solid agreements with IP holders is critical to avoid roadblocks during product development. 💰 Funding for Validation Studies - Securing resources for essential validation work to build credibility and confidence. Above all, building trust among stakeholders is essential to smooth the path to commercialization. It was a pleasure to discuss these points alongside Dr Laurent Pacheco, MD PhD MBA, venture partner at Solas BioVentures, and Ing. Michelle Cortis, RTTP, head of corporate research and technology transfer at the University of Malta. Our conversation was expertly moderated by Gabriella Mifsud. Thank you to Dylan Attard MD, MRCSI, MEnt. for bringing together such a knowledgeable group to address these vital issues. Looking forward to seeing how these discussions will help advance the field! #MedtechMalta2024 #Commercialization #CancerDiagnostics #HealthcareInnovation @ MedTech World @Lifeblood @Malta Enterprise @VisitMalta Incentives & Meetings, @Malta Digital Innovation Authority. @Malta Medicines Authority

Topas’ Cristina de Min, MD, Chief Medical Officer, Hugo Fry, Chief Executive Officer, and Alex Marshall, Chief Business Officer, will be attending BIO-Europe 2024 from November 4-6 in Stockholm, Sweden. Following the initial review of the data from our Phase 2a clinical trial announced earlier this week, we look forward to connecting with fellow industry innovators and investors. Cristina, Hugo, and Alex will be available to discuss the programs in our pipeline and our proprietary platform, which targets the underlying causes of a range of autoimmune and immune-mediated diseases, such as celiac disease and type 1 diabetes, with the goal of delivering long-lasting, disease-modifying therapies. If you’re interested in discussing the data in more detail, make sure to set up a meeting with the Topas team through the PartneringONE platform. #BIOEurope24 #BIOEurope2024 #BIOEurope

Earlier this week, I had the privilege of joining other life science industry representatives in an important discussion with Sweden's ministers for Healthcare, Social Affairs and Public Health, Business and Industry, and the state secretary for Education. Our focus was on how to enhance clinical trials, better implement innovations, and increase Sweden's competitiveness as a leading life science nation. Sweden has seen a decline in industry-sponsored clinical trials, and urgent action is needed to reverse this trend. We must remain at the forefront of clinical research and continue to develop breakthrough medicines for patients in Sweden and beyond. Previous government inquiries have suggested concrete solutions, such as a national partnership platform for clinical trials, based on successful models like Trial Nation in Denmark. It's time to turn the good dialogue and ideas into concrete actions. At Novartis, we're excited to be a partner on this journey and help put Sweden back where it belongs, as a research-based, leading life science nation. Thank you to Acko Ankarberg Johansson, Jakob Forssmed, Ebba Busch, and @Maria Nilsson for the invitation and for the open and constructive dialogue! #lifescience #clinicaltrials #innovation #Sweden #Novartis

🌟 It was an honour to participate in the Medtech Malta 2024 panel discussion, "From Lab Bench to Bedside Care: What Does It Take?" As someone deeply involved in the commercialization of cancer diagnostics, I had the opportunity to share insights into some of the most complex challenges in transforming research into real-world solutions. Our discussion focused on several key challenges: 📝 Regulatory Documentation - Navigating the intricate documentation requirements, from initial concept through to prototype. 🔐 IP and Development Agreements - Setting up solid agreements with IP holders is critical to avoid roadblocks during product development. 💰 Funding for Validation Studies - Securing resources for essential validation work to build credibility and confidence. Above all, building trust among stakeholders is essential to smooth the path to commercialization. It was a pleasure to discuss these points alongside Dr Laurent Pacheco, MD PhD MBA, venture partner at Solas BioVentures, and Ing. Michelle Cortis, RTTP, head of corporate research and technology transfer at the University of Malta. Our conversation was expertly moderated by Gabriella Mifsud. Thank you to Dylan Attard MD, MRCSI, MEnt. for bringing together such a knowledgeable group to address these vital issues. Looking forward to seeing how these discussions will help advance the field! #MedtechMalta2024 #Commercialization #CancerDiagnostics #HealthcareInnovation @MedTech World @Lifeblood @Malta Enterprise @VisitMalta Incentives & Meetings, @Malta Digital Innovation Authority. @Malta Medicines Authority

🌟🌟 We are thrilled to welcome Ilias Pyrnokokis, Head of Value, Access & External Affairs, Chiesi Hellas, a distinguished expert in healthcare access at the upcoming International Conference on Rare Diseases and Orphan Drugs. 🌈🤝He will join the most awaited panel discussion on "Access and Reimbursement Strategies for Orphan Drugs in Europe" on [Friday 1st of March 🕒 14:30 PM - 15:15 PM] 🗓️ Digital. Along with our esteemed panelists he will delve into the challenges, opportunities, and innovative strategies shaping the future of rare disease treatments. 🌍 📌Ilias brings a wealth of knowledge and experience to our discussion on reshaping access and reimbursement strategies for orphan drugs in Europe. Join us as he shares invaluable insights, tackling the challenges and envisioning innovative solutions that can transform the landscape for rare disease patients. 💊 Don't miss this unique opportunity to gain valuable insights and network with industry experts. 🌐 Secure your spot! Registations are still open: https://bit.ly/3HkkWxA 🌍💙Spread the Word: Tag your network, colleagues, and anyone passionate about making a difference in the rare disease community. Let's amplify the impact of this panel discussion on a global scale! #ICORD2023 #rarediseasesconference24 #OrphanDrugs #RareDiseases #HealthcareAccess #PatientAdvocacy #IliasPyrnokokis