Critical Path Institute (C-Path)’s Post

Very insightful publication on multilevel network meta-regression (ML-NMR) and its potential in health technology assessments. This extension of the standard network meta-analysis framework addresses the challenges of existing indirect treatment methods, allowing for comprehensive treatment comparisons. #HealthTechnologyAssessment #IndirectTreatmentComparison #MetaAnalysis #HEOR

We are thrilled to share that C-Path has entered into a strategic data-sharing partnership with Vivli, an independent nonprofit dedicated to advancing global clinical trials data sharing. This collaboration marks a significant step forward in breaking silos and making rare disease data more accessible to researchers and stakeholders worldwide. The partnership will see data sets from Biogen, including the phase II PASSPORT trial in progressive supranuclear palsy and two choroideremia datasets, available on both C-Path's Rare Disease Cures Accelerator Data and Analytics Platform and Vivli's Clinical Trials Data Platform. This alignment reinforces our shared commitment to the FAIR principles of data sharing, ensuring that valuable research data is findable, accessible, interoperable, and reusable. “We are thrilled to announce that Vivli, RDCA-DAP®, and Biogen collaborated to integrate a Progressive Supranuclear Palsy clinical trial dataset into both partnering platforms, as well as two datasets in choroideremia, our first data in rare eye disorders,” shared Alexandre Bétourné, PhD, PharmD, PMP, Executive Director for RDCA-DAP at C-Path. Together, we're accelerating the development of treatments for rare diseases by fostering collaboration and data sharing among academic researchers, industry leaders, healthcare organizations, and patient advocacy groups. Julie Wood Janet Krause #CPath #Vivli #DataSharing #RareDiseases #Collaboration #ClinicalTrials #RDCADAP #DrugDevelopment

🎉 Exciting news from Tavo Biotherapeutics! They have been awarded a generous $1,066,178 grant from the U.S. Department of Health and Human Services (HHS), specifically the National Institutes of Health and the NIH National Eye Institute. This funding will support their groundbreaking work in developing a first-in-class extended-release IOP-lowering formulation for glaucoma, a leading cause of irreversible blindness worldwide. Their innovative approach aims to address major limitations of current treatments, including poor patient compliance and side effects. #SBIR #STTR #Innovation 🔬 This Phase II SBIR grant will enable Tavo Biotherapeutics to perform the necessary IND-enabling studies required by the FDA, paving the way for their first-in-human Phase IIA IND application. The research, which falls under the category of late-stage pre-clinical development, will span 24 months, starting from September 1, 2024, and ending on August 31, 2026. Kudos to Tavo Biotherapeutics for their dedication to improving glaucoma treatment and patient outcomes! #Healthcare #Biotech #Research

Next week, CEO Dominique Demolle and CCO Krinx Kong will be at the ISCTM 2024 Autumn Conference in San Diego, California – participating in Friday's Working Group Session: Diversity in CNS Clinical Trials. In this working group, we're discussing how diversity can be addressed at key stages of CNS preclinical and clinical development, including: 💡Engagement of diverse participants in study design considerations 💡Data-driven methodological considerations for site selection 💡Operational trial conduct 💡Considerations for eligibility criteria and generalizability of endpoints We're excited to be part of this important discussion and offer our unique perspective on how technology can support diverse clinical trials. Specifically, Cognivia's mission is to minimize data variability caused by patient differences. Our tools help trials confidently meet Diversity Action Plan goals while maintaining study power and generalizable endpoints. #ClinicalTrials #ISCTM #CNS #DiverseClinicalTrials #ClinicalTrialDiversity

Milestone Achieved: 8th Clinical Trial Emerges from Target ALS funded consortia QurAlis has completed dosing the first participant cohort in a Phase 1 clinical trial for QRL-101, a first-in-class precision therapy targeting hyperexcitability in ALS. This breakthrough treatment has the potential to benefit both familial and sporadic ALS patients by addressing motor neuron degeneration caused by hyperexcitability—affecting nearly 50% of all cases. Target ALS is proud to have supported the foundational research that led to this clinical trial, as well as providing additional grants, marking a significant step toward effective treatments for ALS. Read the full press release here: https://prn.to/47KVTiX #ALSResearch #ClinicalTrial #EveryoneLives #MilestoneAchieved

Last night, was one of those nights where I did something I rarely do. I watched a "soapy" just to switch off from my work day. Which one? Grey's Anatomy. I don't think I've ever seen a full episode of this before and it was pretty torrid but what I found interesting was that one of patients in this episode had cystic fibrosis. He was very pragmatic and said that he had lived a lot longer than anyone expected him to, so he felt lucky he'd made it to 26 years old. Any my brain went - wait! What??? That's not right..... And then I realised, this episode first aired in 2005 when the median survival for CF patients was less than 30 years. It would be another 7 years before Vertex' first product, Kalydeco, would be approved by the FDA and a few months later, in 2013, I would submit this registration application to the TGA in Australia. The median survival for cystic fibrosis patients has now almost doubled thanks to new treatments for this disease. We are all very focused on finding the next drug target, starting the next clinical trial, submitting the next registration application, and rightly so. But sometimes, it's good to reflect on how far we've come in the orphan drug space in recent years and the impact this has had on patients and their families and friends. Next week I'll be attending the ARCS Conference in Sydney and will be part of the new BioBeacon sessions. Companies will be presenting on their new products in development and panels of experts will provide opinions on some of the pitfalls they may face, questions to ask etc. I very much look forward to being a member of this panel and hearing about what may come to market, from the Australian biotech ecosystem, in another 10-20 years time. #ARCS #biotech #innovation Adjutor Healthcare Group

Another clip from my interview with Cameron Santoro of AJMC - The American Journal of Managed Care is live! In this video, I shared my favorite thing from #AMCP2024: how much I loved interacting with the next generation of researchers. I was energized by conversations with students and early career researchers about their passions and professional aspirations.   Check out the interview below for conference highlights from me, Adam Colborn and Jennifer L. Mathieu of AMCP, Jenny Craven, PharmD, BCPS of UC Davis Health, Andy Killpack of Prime Therapeutics, and Leslie Fish of IPD Analytics!

By integrating a clinical-grade Knowledgebase with the Clinical Genomics Workspace (CGW), you can quickly make informed decisions regarding patient treatment. The Velsera Knowledgebase offers current information on clinical trials, medical literature, drug labels, and additional relevant data. Utilizing this information, CGW facilitates the automatic assessment of variant significance, enabling the creation of reports that present the most relevant therapeutic options to the treating clinician. This integration not only saves your team time but also ensures the delivery of high-quality clinical results, ultimately leading to the best possible treatment for your patients. Learn more: https://lnkd.in/guXky8s7 #PrecisionMedicine #NGS #Genomics #EraOfVelsera

🚀 Breakthrough in scleroderma research! The @Scleroderma Research Foundation (SRF) has launched CONQUEST, a cutting-edge clinical trial designed to accelerate the development of new therapies for #systemicsclerosis. 🩺 This platform trial is the first of its kind in rheumatology to test multiple investigational treatments simultaneously in order to achieve more efficient results. 🌍 This worldwide project involves over 150 sites in more than 30 countries, bringing together global expertise. Thanks to the continuous approach, new treatments can be added over time, continuously advancing research. This milestone brings us closer to a breakthrough that could change the lives of those affected by #scleroderma. Let’s keep pushing for progress! 💙 #SclerodermaAwareness #ChronicIllnessResearch #HealthInnovation

Sponsors are turning to technology to improve efficiency and streamline workflows. While technology is having a positive impact on trials (accelerating the collection of higher quality data, for example), its introduction into clinical trial settings has resulted in unforeseen burdens on sites. In a recent article for ACRP - Association of Clinical Research Professionals, our Chief Product Officer Elisa Cascade shares findings from Advarra's 2023 Study Activation Survey Report, which revealed that a typical oncology study now uses about 22 (yes, 22!) different systems. Sites are already dealing with substantial administrative burdens, and with nearly two-thirds of respondents in Advarra’s survey reporting that the burden caused by technology is far greater than it was just five years ago, dealing with training, onboarding, and management on 22 different systems just isn't feasible. Elisa shares her insights around what we can do to navigate this challenge here: https://lnkd.in/gn7xbsBY #ClinicalResearch #ClinicalTrials #HealthcareTechnology #HealthcareInnovation