Sciensus - Rare Disease Services’ Post

🌐 Join us for Avance Clinical's upcoming webinar to uncover the strategic advantages of early-phase #oncology clinical development across Australia and the USA! 🇦🇺🇺🇸 Our expert speakers, Gabriel Kremmidiotis, Alex Kavros, PhD, and John Zalcberg, will explore how Australia's streamlined regulatory pathways, leading research centers, and significant R&D tax incentives make it an ideal starting point for oncology trials. They will also discuss how transitioning to the USA for later-stage trials aligns with FDA requirements and offers access to a diverse patient population, which is essential for successful oncology research. Discover how an integrated approach can ensure a seamless transition, maintain trial consistency, and maximize R&D tax benefits. 🔗 REGISTER NOW: https://lnkd.in/g-6WaG93 #ClinicalTrials #OncologyResearch #DrugDevelopment #Pharma #CRO #Biotech #Australia #USA #R&D #FDA #ClinicalResearch #Biotechnology #HealthcareInnovation #CancerResearch #ClinicalDevelopment #Webinar #PharmaInnovation #LifeSciences #ResearchAndDevelopment #Healthcare

NMD Pharma Initiates Phase 2 Study in Patients with Charcot-Marie-Tooth Disease 💊🚀 NMD Pharma, a spinout from Aarhus Universitet and a clinical-stage biotech company dedicated to developing novel and improved treatments for patients living with neuromuscular diseases, announces that it has dosed the first Charcot-Marie-Tooth (CMT) disease patient in its Phase 2a clinical trial of NMD670 following FDA IND clearance in June 2024. Thomas Holm Pedersen, CEO of NMD Pharma, states: “As a highly debilitating disease with no currently approved medicines or cure, there is an urgent need for new therapeutic approaches to help improve the quality of life of patients with CMT and their families. Read the press release here https://lnkd.in/dkybnuwJ #lifescience #biotech #wheresciencemeetslife Morten Holmager Health Innovation Aarhus INCUBA A/S

Adoption of legislation for joint EU HTA Assessment has initiated a new era, with the Joint Clinical Assessment (JCA) being implemented for cancer and Advanced Therapy Medicinal Products (ATMPs) from January 2025. Our MD Anand Jha is moderating a session on this topic on 7th October, 2024 at the Pharma Marcon conference in Berlin, with fellow panelists Sandeep Kiri Stephanie Dr. med. Rosenfeld Inka Heikkinen Thomas Grub to look at the challenges that the companies will face for access planning and how to address them. The discussion will cover multiple aspects that the industry and multiple stakeholder groups are grappling with... Anastasia Trubnikova #euhta #healthpolicy #patientaccess #eunethta

Innovation opens opportunities for patients and caregivers, especially with cell and gene therapies offering the chance to tackle intractable diseases…but what happens if we cannot match innovation in science with innovation in our healthcare systems? Let’s discuss at the Pharma MARCON.

Massive Bio is introducing our latest innovation, the Drug Utilization Optimizer (#DUO), at the ASCO Annual Meeting 2024 in Chicago! Building on the success of our SYNERGY-AI platform, DUO is set to transform how pharmaceutical commercial teams target and engage physicians. This innovative analytics and real-time notification platform provides unparalleled insights and efficiencies, allowing commercial teams to analyze the competitive landscape with up-to-date and accurate data. DUO connects with the right physicians, identifies relevant patients quickly and efficiently, increases brand revenues, and reduces the cost of sales. Unlike traditional real-world data companies, we improve operational efficiency and facilitate patient access to medicines by providing real-time and detailed insights. We recognize the challenges in cancer clinical trials and continue to work rapidly to bring more hope. Visit our booth at #ASCO24 to learn more about DUO and see a demo and click on the link below. 👉🏻 https://bit.ly/3yL8eGo Booth: #29137 Selin Kurnaz, PhD, Arturo LoAIza-Bonilla MD, Çağatay M. Çulcuoğlu, Toygun Onaran, Oz Huner, Aslan Aslanağı, Eren Caskurlu, Hakki Isik, PMP, Mert Sarı, Ertuğrul T., Toykan Dogan, MCogSc, #MassiveBio #ClinicalTrials #Innovation #Oncology #AIinHealthcare

Biotech and pharma companies - How do we determine the most efficient regulatory path for codeveloped companion diagnostic tests (CDx)? A CDx can significantly maximize return on investment in clinical trials, but transitioning from a research-grade to a clinical-grade assay can present resource management challenges, especially for emerging companies. Regulatory experts Mwango Kashoki and Sinan Sarac share how to minimize time and resources in our New Medicines, Novel Insights Oncology report: https://lnkd.in/eYHpRUxA #PrecisionOncology #Regulatory #NovelInsights

Biotech and pharma companies - How do we determine the most efficient regulatory path for codeveloped companion diagnostic tests (CDx)? A CDx can significantly maximize return on investment in clinical trials, but transitioning from a research-grade to a clinical-grade assay can present resource management challenges, especially for emerging companies. Regulatory experts Mwango Kashoki and Sinan Sarac share how to minimize time and resources in our New Medicines, Novel Insights Oncology report: https://lnkd.in/eNiy26EP #PrecisionOncology #Regulatory #NovelInsights

Biotech and pharma companies - How do we determine the most efficient regulatory path for codeveloped companion diagnostic tests (CDx)? A CDx can significantly maximize return on investment in clinical trials, but transitioning from a research-grade to a clinical-grade assay can present resource management challenges, especially for emerging companies. Regulatory experts Mwango Kashoki and Sinan Sarac share how to minimize time and resources in our New Medicines, Novel Insights Oncology report: https://lnkd.in/eQxy2MXC #PrecisionOncology #Regulatory #NovelInsights

Biotech and pharma companies - How do we determine the most efficient regulatory path for codeveloped companion diagnostic tests (CDx)? A CDx can significantly maximize return on investment in clinical trials, but transitioning from a research-grade to a clinical-grade assay can present resource management challenges, especially for emerging companies. Regulatory experts Mwango Kashoki and Sinan Sarac share how to minimize time and resources in our New Medicines, Novel Insights Oncology report: https://lnkd.in/eDJ7B7Nx #PrecisionOncology #Regulatory #NovelInsights

C-Path is excited to announce the launch of the Critical Path for Alpha-1 Antitrypsin Deficiency Consortium. The CPA-1 consortium aims to accelerate drug development for alpha-1 antitrypsin deficiency, a rare disease that affects individuals and families worldwide. This will be achieved by integrating data through C-Path’s Rare Diseases Cures Accelerator–Data Analytics Platform and leveraging those data for CPA-1 to collaboratively develop regulatory-grade solutions to continuously address unmet needs in drug development for this condition, including: ▶ Generating evidence to support the use and regulatory acceptance of novel drug development tools, such as biomarkers, for AATD liver and lung disease clinical trials and; ▶ Identifying how to incorporate and utilize patients’ lived experiences to optimize clinical trial design and outcome measures that capture how patients feel, function and thrive. Inhibrx, Inc., Takeda and global biotherapeutics leader CSL Ltd have joined as founding members of the consortium signaling the launch of this collaborative effort. Full details, here: https://lnkd.in/gJhdX3bV Obi Umeh, M.D., MSc. Lars Groenke, Celia Witten, Jeffrey Siegel, Gina Smith FDA Center For Drug Evaluation and Research #CPath #CDER #CBER #FDA #CPA1 #raredisease #RDCADAP #drugdevelopment #datasharing #collaboration #globalhealth