Alexion Pharmaceuticals, Inc.’s Post

💡Today, Jo De Cock, thought leader of RWE4Decisions, chaired the session “Innovative Therapies, Unequal Access: Bridging the Gap for Rare Disease Treatments" at the European Conference on Rare Diseases hosted by EURORDIS-Rare Diseases Europe.   🎯 The session focused on the pivotal role of Pricing & Reimbursement to enable patient access to innovative therapies.   📋 Different solutions for better access were discussed, as well as the importance of international and regional collaborations linking with national plans, trust-building amongst stakeholders and transparency.   🤝 RWE4Decisions welcomes these issues remaining high on the agenda for patient access to innovative therapies in Europe, and looks forward to outcomes from the work of the WHO Novel Medicines Platform.    Donatello Crocetta MD MBA Celeste Scotti MD, PhD Mariangela Pellegrini Christine Leopold Daniel de Vicente #RWE #RealWorldEvidence #RWD #RealWorldData #RareDiseases #ECRD2024 #HTA #HealthData #EHDS

Recap from #ISCT2024 Roundtable Discussion with Donald B. Kohn and Steve Grossman “Rare Diseases and Patient Access – The Mission and The Challenges”. Top 3 Takeaways: 1. A global repository for data ,gained through research and clinical trials that are not advancing, is ideal to ensure that efforts for rare disease research and drug development are able to be mined for gold. 2. Reclaiming an asset that is not take forward by a partner puts the follow-up burden back on the original IP holder as they seek to partner and further finance trials. 3. Could re-insurance be a solution for broader access of rare disease therapies?

📅 Today is a day to honor the strength of patients, caregivers, and researchers who face unique challenges every day. A disease is considered rare when it affects fewer than 1 in 2,000 people. Yet, rare diseases impact around 30 million people across the EU and over 1 million individuals in the Nordics alone. The landscape is evolving: for instance, the European Commission has already authorized more than 200 orphan medicines, offering new treatment options for those suffering from rare diseases. At MedEngine, we’re inspired by this progress and are dedicated to contributing to this field. Our expertise in strategic planning, regulatory support, and market access is more relevant than ever for navigating the complexities of rare disease treatment development. Let’s continue to advocate for research, innovation, and policies that will bring new treatments to those in need. Together, we can make a difference in the lives of those affected by rare diseases! 📲 https://lnkd.in/gk6hbAF #MedEngine #TheValueIsInTheLinks #RareDiseaseDay #RareDiseases #OrphanDrugs #PrecisionMedicine

Take a look at our webinar library   As we approach the end of the year, here’s a look at some of our webinars in 2024, which brought together representatives from industry, patient groups and experts from the MAP Group team: - Breaking Down Barriers To Access In Europe – Getting It Right First Time For Rare Diseases! - The Value Of Delphi Panels In Accelerating Patient Access - Navigating EU HTA – The Future Of Supranational HTA Initiatives; Lessons Learnt So Far… - Amplifying The Rare Disease Patient Voice In The HTA Process In UK And Ireland In total, we've curated a library of over 20 on-demand webinar recordings to ensure you stay ahead in the market access landscape. Our webinars are full of strategies to empower your team, covering some of the most critical topics such as patient access to rare disease treatments and the EU HTA.   Access these and more valuable resources, exclusively available to biopharma professionals. Request your recordings here: https://lnkd.in/exewTNnb   #Webinar #MarketAccess #Events #EUHTA

Today, EUCOPE co-hosted the Rare Disease Forum 2024, discussing how to secure #EU competitiveness in R&D for people living with #RareDiseases. 🗣“The EU #regulatory and #incentive frameworks allowed smaller companies to develop several #OMPs and flourish in the rare disease space. We need to ensure a predictable ecosystem to remain globally competitive”, said Alexander Natz, EUCOPE’s Secretary General. ⚡An insightful panel with Daria Julkowska, Caroline Ven and Stefan Joris highlighted some key elements for the the future of EU #Competitiveness in rare diseases: fostering basic #Research; collaborating to improve #Access; leveraging #ERNs, public-private partnerships and joint initiatives; directly involving #Patients; developing EU and national Rare Disease Action Plans.

New White Paper! Rare diseases are an area of huge unmet patient need, both because of the thousands of rare diseases for which there are no currently available pharmacotherapeutics, and because even where there are #Orphanmedicines, they often do not reach all patients effectively because of challenges to market access, health system readiness and other issues. Whilst policy to encourage the development of Orphan medicines has been in many ways highly successful, it is clear both that there is some way to go, and that Orphan medicines, which proved resilient in the immediate Pandemic era, are now entering a new era with a tougher market access environment, greater challenges to bring Orphan medicines to rare disease patients in still fragile healthcare systems, and more complex stakeholder engagement, where partnering with patients, their carers and patient advocacy groups becomes a more committed and multi-faceted relationship. https://lnkd.in/eJhZ7c5s

🔍 Sandra Schüle's Insights on Rare Disease Launch Excellence 🔍 Having delved into the latest Whitepaper on Mastering Rare Disease Launch Excellence by IQVIA EMEA, I've uncovered critical findings that could redefine your launch strategies. Here are the highlights: Instant Real-World Evidence (RWE) with AI Solutions: Advanced AI technologies now enable real-time generation of RWE, prioritizing patients' voices and unveiling their unmet needs instantly, rather than relying on historic data. Bespoke AI Solutions for Multilingual Patient Insights: With over a decade of training in medical language, bespoke AI solutions unveil meaningful patient needs and journeys across 65+ languages, ensuring a comprehensive understanding. The findings from the Whitepaper underscore the significance of three key pillars for achieving excellence in launching rare disease treatments: 🚀 Preparedness of Health Systems 🚀 Engagement with Stakeholders 🚀 Presentation of Value Evidence To elevate launch excellence in rare diseases, it's essential to harness the power of LLM AI solutions and AI tools for social listening. Key strategies to be addressed by bespoke AI solutions are: 1. Understanding the patient journey within the health system 2. Early identification of patients and caregivers for informed decision-making 3. Utilizing RWE for patient identification and educating stakeholders 4. Building strong relationships with key stakeholders for deep understanding 5. Collaborating with patients and advocacy groups in trial recruitment and tool design 6. Early evidence planning and leveraging RWE for informed decisions 7. For rare disease launches, every patient matters, and it's essential to inform and supplement clinical trials effectively. These insights are crucial for optimizing rare disease launches and ensuring patient-centricity. To learn more about how Accellara Consult's solutions can elevate your launch strategies, feel free to reach out to me and schedule an appointment via LinkedIn booking request. #DigitalHealthInnovations #AI #PatientVoice #PatientExperience #PatientJourney #PatientEngagement #RareDiseaseCommunity #NLP #NLU #FemaleTransformersinHealthcare #BusinessStrategy

Ending the diagnostic odyssey in rare disease is an ambitious undertaking—and one that cannot be accomplished by any single discipline or health care sector. I’m pleased to have contributed to the new framework aimed to accelerate diagnosis developed by the Global Commission to End the Diagnostic Odyssey for Children with a Rare Disease, co-chaired by Takeda, EURORDIS-Rare Diseases Europe and Sanofi. To achieve the Global Commission’s vision of a clear path to a timely, accurate diagnosis for all children we must learn from each other what IS working -- then share those best practices to inspire change. You can find out more in the Global Commission’s first iteration of the 2024 Framework for Action report linked below. I hope you find this framework useful for your efforts and encourage you to share your feedback with the Global Commission. https://lnkd.in/eu2mzMtC #raredisease #TeamTakeda

Under the theme “Navigating the Uncharted: Advancements, Collaborations, and Hope in Rare Diseases”, the International Congress on Rare Diseases and Orphan Drugs aspires to stimulate dialogue and cooperation across all stakeholders – patient representatives, policymakers, clinicians, researchers, industry, payers and regulators – towards advancing a comprehensive rare disease strategy that promotes sustainability and equity within healthcare ecosystems. It is a great honour for me to participate in such an important event as a keynote speaker. Utilizing the experience and knowledge of international partners, the conference will open the dialogue with the current political developments in Europe and the drawing up of a National Strategy for Rare Diseases with the ultimate goal of creating and implementing the National Action Plan for Rare Diseases within 2024. #rarediseaseconference2024

Just under one month to go until our next webinar 🔔 Join our expert panel as they look at the value of Delphi panels in accelerating patient access to innovative treatments. This webinar aims to provide a comprehensive overview of the Delphi panel methodology, its application in supporting reimbursement decisions and practical considerations for effectively implementing this approach as part of your evidence generation strategy. By attending this webinar, you will: - understand the Delphi panel methodology, including its origins and structured approach for generating expert consensus - discover the value of Delphi Panels in supporting reimbursement decisions - explore practical considerations for implementation in your evidence generation strategy This session is particularly relevant for Market Access teams looking to leverage robust expert opinion and consensus to strengthen their evidence packages and value propositions for reimbursement submissions, particularly for rare disease submissions where clinical data may be limited and expert perspectives are crucial to contextualise evidence for local settings. Click the link to register your free place now: https://lnkd.in/egEWJ999 #webinar #delphipanels #marketaccess #reimbursement