pharma.be’s Post

Yesterday, I attended this insightful event to discuss the burden of rare diseases. It's clear that their impact is profound, often overshadowing more common illnesses due to their complexity and the unique challenges they present to people. One of the most significant takeaways for me is the need for unity in creating an EU regulatory environment that not only encourages research but also accelerates approvals. Countries must collaborate to harmonize Health Technology Assessments (HTA) across Europe, ensuring fair access to treatment and cure. There is definitely a lot of work ahead of us, but this kind of conversation is exactly what we need to make change happen. #rarediseases #lifescience

Diseases know no borders. And neither does our commitment to fight them together. Had a very useful day of meetings yesterday in Brussels, organized by EFPIA. With colleagues from various European countries, we talked about important topics. Such as: ✅ the clinical research ecosystem in Europe; ✅ revision of the European pharmaceutical legislation; ✅ a competitive strategy for Life Sciences & Health. Europe risks 'missing the boat' in drug development, compared to the United States and China. Less clinical research on our continent, for example, does not only affect European patients, who therefore have later access to innovative drugs. It is also bad for the level of knowledge of our doctors. Mario Draghi recently wrote a penetrating report on Europe's strategic position: 'The future of European competitiveness'. We also talked about ways to responsibly and effectively face the challenges in European healthcare together with other stakeholders - such as politicians, scientists, patient organizations, doctors and journalists. Let no one be under the illusion that we can solve major challenges like #Alzheimer's, #cancer and #obesity from a silo. Together we are so much stronger! #wewontrest #clinicaltrials #eupharmalegislation EFPIA - European Federation of Pharmaceutical Industries and Associations Andy Powrie-Smith Lavinia Meloni Maryna Bokorna Jack Neill-Hall Espen Snipstad Veronika Bendere Stephanie Prate

Great article on this #RareDiseaseDay. RLM Consulting

🌟 RWE4Decisions is taking part in #HTAi2024Seville - one panel today and another panel tomorrow. Today, RWE4Decisions Steering Group member Francois Meyer moderated a very successful session on “Can We Agree When an RCT Is Not Possible and the Implications for Comparative Effectiveness?”. Many thanks to our brilliant speakers and to the very interactive audience! Antoine Vanier presented the findings on non-randomised studies in Haute Autorité de Santé's paper on 'Rapid access to innovative medicinal products while ensuring relevant HTA': https://lnkd.in/dSdp4rfs. Anne-Pierre Pickaert from France Lymphome Espoir provided the patient perspective on participation in clinical or single arm trials and the balance between ethics and the need for evaluating comparative effectiveness. James Ryan from AstraZeneca delved into the European Medicines Agency's conditional approval system and the cases when single arm trials might provide the most reliable information. 💡 Let us know in the comments if you participated in today's session and any reflections on the topic. 👇 🔜 We look forward to welcoming you tomorrow at 17:15, in room Andalucia 1&2, for our second panel: “Post-Launch Evidence Generation: Can We Anticipate When and What We Need?”. #RealWorldEvidence #RCT #HTA Health Technology Assessment international (HTAi)

The Fourth Annual Nordic Innovation Day (NID) returns to Valby, Denmark on 26 September 2024. Will you join us? Hosted by @Lundbeck and @Medidata Solutions, NID brings together thought leaders and innovators from the region, so that they can gain inspiration from each other, and examine the opportunities, latest advancements and challenges in clinical trials. The aim of the NID is to spur innovation and knowledge-sharing. The event provides a forum where attendees exchange ideas and ask questions. Above all, NID offers a great opportunity to network with peers from across the Nordics, who are working on similar challenges and explore solutions. There is very limited availability at the event. For the agenda and more details, visit the NID website https://lnkd.in/etJx92am #NIDDenmark #Medidata #Lundbeck #WeAre3DS #FerringPharmaceuticals #AscendisPharma #DiamydMedical #NovoNordisk #LifeSciences #Nordic #LifeSciences #digitaltransformation #ClinicalTrials #Medidata #artificialintelligence #AIinHealthcare #ClinicalDevelopment #Pharma #DigitalHealth #AI #productivity #PatientCentricity #datamanager #VirtualTwin #clinicalinnovation #regulatory #regulatorylandscape #regulatorychange #clinicaldatamanagement #modellingandsimulation #digitalhealth #digitalhealthtechnologies #generativeai #largelanguagemodels #medicalimaging 

Our in-depth full report from #EPAConf is here! Our report consolidates key plenaries and tracks, and includes main takeaway points from Graham Foxon's talk on #EUHTA. 📌 If our summary is helpful, take a moment to repost it to your network ♻ #healthcare #marketaccess #pharma #rarediseases #realworldevidence #digitalhealth #digitaltransformation

⚠️ EVENTO DA NON PERDERE ⚠️ 𝐓𝐫𝐚𝐧𝐬𝐥𝐚𝐭𝐢𝐧𝐠 𝐈𝐧𝐧𝐨𝐯𝐚𝐭𝐢𝐨𝐧 𝐢𝐧𝐭𝐨 𝐚𝐜𝐜𝐞𝐬𝐬 𝐟𝐨𝐫 𝐀𝐓𝐌𝐏𝐬  𝟑𝐫𝐝 𝐄𝐔-𝐈𝐧𝐧𝐨𝐯𝐚𝐭𝐢𝐨𝐧 𝐍𝐞𝐭𝐰𝐨𝐫𝐤 𝐦𝐮𝐥𝐭𝐢-𝐬𝐭𝐚𝐤𝐞𝐡𝐨𝐥𝐝𝐞𝐫 𝐦𝐞𝐞𝐭𝐢𝐧𝐠  📅 15 Novembre 2024   📍 Piazza della Pilotta, 4 - 00187 Roma AIFA Agenzia Italiana del farmaco and the EU-Innovation Network (EU-IN) are organising a multi-stakeholder meeting in Rome to be held on 15 November 2024. The meeting focuses on how innovation in the development of Advanced Therapy Medicinal Products #ATMPs can be translated into effective and safe therapies at the point of care in Europe. The objectives are to: ▪️ seek feedback from developers on the main challenges and barriers currently encountered from early development to access, ▪️ provide insights on scientific and regulatory advice tools available to innovative developers at European and national level, ▪️ discuss opportunities for promoting mutual exchange and interactions between stakeholders with the aim of strengthening the European innovation ecosystem. The meeting will provide an open forum for discussion with a view to raising awareness of existing initiatives, as well as identifying further actions that could help to foster collaboration between stakeholders and ultimately facilitate patient access to safe and effective ATMPs. Virtual participation to the event is open to all stakeholders. Registration 𝐰𝐢𝐥𝐥 𝐛𝐞 𝐨𝐩𝐞𝐧 𝐮𝐧𝐭𝐢𝐥 𝟓 𝐍𝐨𝐯𝐞𝐦𝐛𝐞𝐫 𝟐𝟎𝟐𝟒. ⬇️ Learn more and go to registration form ⬇️

Clinical Trial Diversification: What do Hong Kong & the Greater Bay Area Have to Offer? Hong Kong has grand plans to become one of Asia’s leading clinical trial hubs. Important regulatory upgrades and increased integration with mainland China via the Greater Bay Area initiative – including a new dedicated GBA Clinical Trial Institute at the Hong Kong/mainland border – mean that this is not just a pipe dream. However, as an illuminating panel discussion hosted by PharmaBoardroom at last month’s BIOHK 2024 conference showed, several challenges still need to be ironed out to fully realise Hong Kong’s clinical trial hub potential. Featuring: Henry Yau of Clinical Trials Centre, The University of Hong Kong, Charlie Chen of Nuance Pharma, and Xia Jin of Immuno Cure Read more at: https://lnkd.in/dfYcR--R

The recent overhaul of the EU's General Pharmaceutical Legislation (GPL) marks a pivotal moment in our quest to modernize healthcare. Why am I excited about it? 🔍 Innovation at its core The proposed amendments to streamline processes at the European Medicines Agency are a step in the right direction, promising a predictable and efficient regulatory environment. 📉 Addressing a concerning trend A study by Dolon warns of a significant decrease in incentives for companies to invest in breakthrough medicines in Europe. Addressing it, would power Europe's global R&D investment share. This is not just about numbers; it's about the millions of lives at stake and the years of life lost due to delayed or inaccessible treatments. 🚀 The way forward As we engage with the European Union Institutions on these proposals, let's champion policies that are pro-patient and pro-innovation. This will foster an environment conducive to investment, particularly for small and medium biotech enterprises. #pharmainnovation #EUpharma #LegislativeChange #PatientCare

🚀  Access to orphan drugs in emerging markets At the World Orphan Drug Congress USA 2024 that took place 2 weeks ago, challenges related to accessing orphan drugs in low- and middle-income countries (LMICs) were discussed, including: 📢 Awareness: A lack of disease awareness, unclear treatment protocols, and limited knowledge of the benefits of new therapies. 🌐 Accessibility: Disorganized healthcare systems and inadequate transportation networks leading to delayed diagnoses and insufficient care. 💊 Availability: A stringent Pricing & Market Access (P&MA) environment often resulting in orphan drugs being excluded from public reimbursement. 💰 Affordability: High drug costs, and constrained healthcare budgets At Inbeeo, we provide strategic insights to accelerate patient access to orphan drugs in emerging markets, supporting them in: ·      Product and market prioritization ·      Access pathway prioritization ·      Commercialization strategy ·      Developing pricing strategies 💬 Let’s Chat! 💬 Reach out for an informal chat, and let’s explore how we can be your partner in devising access strategies for orphan drugs in emerging markets #MarketAccess #PatientAccess #OrphanDrugs #EmergingMarkets