Estelle Haenel’s Post

// Rare disease clinical research // 👨⚖️ What is the specific position of regulatory agencies in rare diseases clinical trials? 🙌 How does engaging with patient organizations improve rare disease drug development? ✅ How important do Patient Reported Outcomes need to become to improve the success of rare disease clinical trials? 👉 Read the full article: https://bit.ly/3KFLMRu #kayentis #eCOA #clinicaltrials #raredisease

Check out the latest article we created for our partner PCM TRIALS - Quality Mobile Research on our Clinical Trials Arena website - Mobile visits are critical for the decentralization of rare disease research As rare disease drug development gains momentum, the growing movement towards decentralized clinical trials could not be more well-timed. In this article, we discuss the important synergy between the two trends and look at data around the increasing use of mobile visits in rare disease protocols. - https://lnkd.in/d3Jqugg7 For information about our award winning content and ABM solution, please request a demo here: https://lnkd.in/e8wNB3cj #decentralizedclinicaltrials #dct #decentralizedtrials #clinicaltrials #accountbasedmarketing #contentmarketing #healthcaremarketing

🧬 3 challenges and opportunities in rare disease drug development 🇪🇺 In Europe alone, approximately 36 million people are suffering from rare diseases, which is approximately equivalent to half the population of France. 🚨 There is no cure for most rare diseases, and the field suffers from gaps in medical and scientific understanding. Nevertheless, impressive progress has already been made in recent years and appropriate treatments and medical care have proven to enhance the quality of life and extend the lifespan of those affected. Our team explored three challenges and three opportunities for the current R&D of rare diseases: 🔴 Challenge 1: small patient populations exacerbate clinical trials 🔴 Challenge 2: limited knowledge of rare diseases impacts research strategies  🔴 Challenge 3: development of therapies for rare diseases isn’t financially feasible Despite these challenges, there are promising opportunities that could pave the way for the development of new therapies for rare diseases: 🟢 Opportunity 1: innovative technologies and designs in clinical trials 🟢 Opportunity 2: potential of multinational research collaborations  🟢 Opportunity 3: chances of personalized treatment approaches 💡 Want to learn more about the challenges and opportunities that could transform the landscape of rare disease research? Read our latest article here: https://lnkd.in/eNDuMZWU #healthcare #pharmaceuticals #pharmaceuticalindustry #raredisease #drugdevelopment

Today is #RareDiseaseDay, and Norstella joins the global community in raising awareness for those affected by rare diseases. Our mission echoes this sentiment: to help bring life-saving therapies to market quicker—and help patients in need. Rare disease is one therapeutic area in which large pharma companies have seen significant investment in recent years, though many challenges remain. Norstella experts share their insights on the major trends and challenges currently impacting the rare disease landscape on our blog. Click to read it here: https://ow.ly/4eXt50QJ7wJ #Pipeline2Patient #RareDiseases #RareDiseaseAwareness #Pharma

Did you know that over 30 million Americans, or 1 in 10 people, struggle with rare diseases? Despite this, the national economic burden of 379 rare diseases reached nearly $1 trillion in 2019. Patients often wait an average of 6.3 years before receiving a confirmed diagnosis, causing significant uncertainty and anxiety. Furthermore, 93–95% of the 10,000+ recognized rare diseases lack FDA-approved therapies. What can potentially be done to reduce this burden and increase rare disease awareness and research? ➡️ Increased funding, collaborative research initiatives, and utilization of advanced technologies. Leveraging advancements in genomics, precision medicine, and digital health technologies can enhance our understanding of rare diseases and facilitate the development of targeted therapies tailored to individual patients. ➡️ Encouraging collaboration among researchers, healthcare providers, patient advocacy groups, and pharmaceutical companies can accelerate the pace of rare disease research. ➡️ Sharing data, resources, and expertise can lead to a more comprehensive understanding and faster development of therapies. ➡️ Regulatory agencies like the FDA can implement expedited review processes for rare disease therapies, such as accelerated approval pathways and orphan drug designations. ➡️ Most importantly, incorporating patient perspectives and experiences into the research and drug development process is crucial. These mechanisms can also incentivize pharmaceutical companies to invest in rare disease research and bring treatments to market more swiftly. Check out the attached reference article from #BIO for a well-crafted summary on rare disease advocacy and awareness. #RareDiseaseWeek2024 #PatientAdvocacy #rarediseaseawareness #rarediseaseresearch

The article below is free to view and includes comments/analysis from myself along with several of my colleagues about trends and challenges in the rare disease ecosystem. #rarediseaseday #rarediseases #orphandrugs

Partner Content - PCM TRIALS - Quality Mobile Research explains why mobile visits are critical for the decentralization of rare disease research As rare disease drug development gains momentum, the growing movement towards decentralized clinical trials could not be more well-timed. In this article, we discuss the important synergy between the two trends and look at data around the increasing use of mobile visits in rare disease protocols. - https://lnkd.in/d3Jqugg7 #dct #decentralizedclinicaltrials #digitaltrials #decentralizedtrials #clinicaltrials #raredisease

https://lnkd.in/gdzB39HE There is a remarkably high level of activity in liver disease diagnostics and drug development. Great insights shared by Tim Jobson in this article about the growing challenge of conducting clinical trials in metabolic associated steatohepatitis (#MASH). “Currently, there are 417 MASH-related clinical trials that are either active or in the recruitment stage”. At Livivos, Inc, we emphasize the urgent need for accurate non-invasive testing (#NIT) in point-of-care settings to support liver disease clinical trials and treatment monitoring. To address this need, we have developed the #LiverScope, which aims to deliver MRI-level accuracy in point-of-care environments. www.livivos.com #clinicaltrials #liverdisease #NASH #PDFF #pointofcare #medicaldevice #steatosis #fattyliver

The FDA’s Center for Drug Evaluation and Research (CDER) is making strides in addressing rare diseases through various initiatives. With over 7,000 rare diseases affecting millions, CDER's Accelerating Rare disease Cures (ARC) Program is central to these efforts. Key initiatives include enhancing collaboration across FDA centers, developing the Rare Disease Innovation Hub, and implementing expedited review processes. These efforts aim to overcome the challenges of drug development for rare diseases, ensuring faster access to effective therapies for patients. #raredisease #FDA #CBER #healthcare #innovation Explore the full article: https://lnkd.in/dTacjaRM.

Today, on #RareDiseaseDay, Citeline acknowledges this significant day dedicated to raising awareness and driving change for the 300 million people worldwide who live with rare diseases and their families and caregivers. Rare disease is one therapeutic area that has seen significant investment in recent years by large pharma companies, though many challenges remain. In this article Citeline and Norstella experts share their insights on how the life sciences industry is shaping up for a new era of patient recruitment and retention: https://ow.ly/Ji3550QJfSu. #Pipeline2Patient #RareDiseases #RareDiseaseAwareness #Pharma