Aude Clement’s Post

The work on digital biomarkers never sits still, and every conference is expanding on experiences from case studies, methodologies, etc. Great summary from Aude Clement on the key takeaways from this event.

🌍 PFMD is excited to attend the World Orphan Drug Congress Europe 2024 in Barcelona! 🎙 Nicholas Brooke, Executive Director PFMD, will present tomorrow in the workshop "Holistic Approaches to Rare Disease Drug Development: Integrating ESG, Patient Engagement, and Commercial Strategy for Improved EU Access." Key insights from the workshop will cover: ➡️ Tackling unique challenges in rare disease drug development ➡️ ESG in rare disease: Exploring the role of environmental, social, and governance factors ➡️ The critical importance of patient engagement in shaping access strategy, clinical trials, and regulatory decisions ➡️ Ethical considerations and social impact on underserved populations Join us as we dive into these critical topics and showcase strategies for shaping the future of rare disease drug development through patient engagement! https://lnkd.in/esU7EFd #WODC #RareDisease #PatientEngagement #ESG #PFMD #HealthcareInnovation #RegulatoryAffairs

🚨Happening tomorrow! 🚨 Join us at 11 a.m. ET for our FREE monthly Journal Club on “Net financial benefits of digital endpoints publication in Clinical and Translational Science”. Digital measures offer the clinical trials community unparalleled insights into people’s real-life experiences, enhancing our understanding of numerous conditions. So, why haven’t we implemented digital endpoints on a larger scale? Findings from research conducted by Digital Medicine Society (DiMe), Tufts Center for the Study of Drug Development, Johnson & Johnson, Roche, Genentech, UCB, Bayer, Takeda, and MindMed, now published in the Journal of Clinical & Translational Science, showcase evidence of significant cost reductions and financial benefits tied to leveraging digital endpoints in trials. Key findings include: ▪️ Digital endpoints significantly shorten trial phases and permit smaller trial enrollment sizes ▪️ For phase 2 trials, the increase in eNPV varied from $2.1 million to $3.3 million, with ROIs between 30% to 48% per indication ▪️ The net benefits were substantially higher for phase 3 trials, with the increase in eNPV varying from $24 million to $40 million, with ROIs that were four to six times the investment 🔗 Haven’t signed up yet? There’s still time! Register here and join the conversation tomorrow! https://bit.ly/3zsEhM8

💊 📱What an incredible PDURS webinar we had! 📱 💊 Missed it? Don’t worry—you can catch the recording now! Here’s what our expert panel discussed: Marty Culjat, SVP, Global Head of Digital Medicine & Regulatory Innovation at EVERSANA, helped us to get to know PDURS. Jessica J. Federer, Board Member and former Bayer Chief Digital Officer, explained how PDURS can revolutionize data collection to improve patient outcomes.  Amir Zur, former Head of Digital Health Products at Takeda & Teva, explored how AI could be harnessed in the context of PDURS. Michal Tsur, Co-Founder & Co-CEO of Remepy, moderated the panel and discussed how PDURS could drive the integration of digital therapeutics and digital tools with drugs. Many other fascinating subjects came up as we tried to understand how PDURS would promote innovation in pharma.  Ready to dive in? The link is in the first comment. 

It’s published 👏 a collaboration with over 60 experts across geography and disciplines. Delve in and see what it can mean for you and how you can support the implementation of recommendations for #MS, #NMOSD, #MOGAD and related disorders. MS Brain Health Oxford Health Policy Forum #MSBH #BrainHealthTM #NMOSDTimeMatters #MOGADTimeMatters

We would like to extend our sincere thanks to the sponsors who were instrumental in making the 2nd Congress on Rare Diseases for Latin America and the Caribbean possible. Your support enabled key discussions, including the need for clear public policies, the importance of international collaboration, and innovative models like Casa dos Raros in Brazil. Panels explored the role of regulatory agencies, health technology assessments, and innovative financing strategies for rare diseases. Advocacy leaders and patient testimonials reinforced the importance of continued stakeholder engagement and a unified approach to improving care for those living with rare diseases. With over 1,500 participants, the congress focused on advancing critical discussions around rare diseases and identifying practical solutions to improve diagnostics, access to treatment, and patient care in the region. A special thanks to our Platinum sponsors, Amgen and AstraZeneca, for their ongoing commitment to rare disease care. We also thank our Gold sponsors, Chiesi Group, Takeda, Johnson & Johnson, Valentech Pharma, and MSD, for their valuable contributions to advancing rare disease research. In addition, we appreciate the support of our Silver sponsors, Boehringer Ingelheim, Sanofi, ARCHIMEDlife Medical Laboratories, Illumina, JCR Pharmaceuticals, and Multicare Pharma, and our Allies, Optimal Therapies, Farmacon Global, and RARASCRO. We look forward to continuing our collaboration to drive progress in rare disease care across Latin America and the Caribbean. Learn more about AHF and our work: https://lnkd.in/drNVhhwU #RareDiseases #Collaboration #Innovation #PublicPolicy #Healthcare #AccessToCare #Congress #ThankYou #LatinAmerica

IMPORTANT & RELEVANT INDUSTRY DISCUSSION @ MOBILE IN CLINICAL TRIALS - I am so honored to be part of this important panel focused on the value & ROI benefit of digital technologies in clinical trials. Now, in the post-COVID environment, in a world where there are currently over 7.2 Billion digital smartphones worldwide, where ~90% of all cellular phones are (digital) smartphones and the majority of the world’s population currently owns one, it sure seems there have been retractions in the value and adoption of digital technologies in clinical trials for traditional legacy solutions. This panel is focused on realities, value propositions and ROIs that begs the question, "What are we doing and why, or, . . . . why not?"

The journey of #ALS and #FTD drug development is one of hope and resilience. With QurAlis and Eli Lilly and Company’s collaboration targeting UNC13A, Trace Neuroscience’s $101M funding, and progress in TDP-43 biomarkers, it’s inspiring to see advancements in the field. But recent failures to be even more topical - the Denali Therapeutics & Calico Life Sciences late-stage trial failures - remind us how much more work is needed for patients and their families. That’s why we're honored to announce the launch of the 𝟒𝐭𝐡 𝐀𝐋𝐒 𝐃𝐫𝐮𝐠 𝐃𝐞𝐯𝐞𝐥𝐨𝐩𝐦𝐞𝐧𝐭 𝐌𝐞𝐞𝐭𝐢𝐧𝐠 (📅 𝐌𝐚𝐲, 𝐁𝐨𝐬𝐭𝐨𝐧). Sophie Evans has done outstanding work in bringing together the entire community—#biotechs, #pharma, #clinicians, #researchers, #patientadvocates, and more—to share breakthroughs, tackle challenges, and focus on patient-centered solutions. Expert Speakers from Eli Lilly and Company, Trace Neuroscience, Barrow Neurological Institute, Regeneron, Biogen, and more. Collaboration across disciplines is key to ensuring that every insight brings us closer to effective treatments—and one day, a cure. If this resonates with you or someone in your network, and you believe you can make a meaningful contribution to this industry-led meeting, we offer Partnership opportunities to help your organisation share its story. ❓𝐈𝐧𝐭𝐞𝐫𝐞𝐬𝐭𝐞𝐝? ⚠️ 𝐃𝐨𝐰𝐧𝐥𝐨𝐚𝐝 𝐀𝐠𝐞𝐧𝐝𝐚: https://ter.li/oc7oph #ALS #FTD

We need to call on policymakers to drive regional, national, EU, and international policies that can help to improve the lives of people living with the disease, define a post diagnostic pathway through sufficient guidelines and funds allocated for optimal implementation, which will also improve alignment and harmonisation of the pathways across the countries.

📢 On the occasion of the #WorldAlzheimersMonth, we are proud to launch, in collaboration with EFPIA - European Federation of Pharmaceutical Industries and Associations, the Perspective Paper "Rethinking Alzheimer's Disease Pathway: From Diagnosis to Care." The aim of this report is not merely to document the status quo, but to challenge it—to rethink how we approach Alzheimer’s treatment, care and management. By offering a comprehensive review of current practices, identifying challenges, gaps and barriers, and proposing policy recommendations, this report seeks to reshape the Alzheimer’s disease healthcare pathway in ways that enhance outcomes and quality of life for all involved, and reduce the impact on society. 🔎 Read more here: https://lnkd.in/e_iSYZwN