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

🎯 Challenges in rare disease drug development include small patient populations, limited disease knowledge and financial constraints. 💡 Innovative designs in clinical trial, potential multinational research collaborations and personalized treatments offer promising opportunities. 👀 In this article, #Alcimed explores these challenges and opportunities, emphasizing the need for continuous research and interdisciplinary collaboration to enhance the lives of those affected by rare diseases. #RareDiseases #Healthcare #ResearchAndDevelopment #Pharma #Innovation   https://lnkd.in/dKuhYKVt

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

Over 10,000 rare diseases have been identified, affecting approximately 1 in 10 Americans. Unfortunately, over 90% of these conditions lack a cure or disease-modifying treatment. Clinical trials serve as a beacon of hope, driving research forward and potentially uncovering effective therapies. Patients with rare diseases willingly participate in trials to slow disease progression or alleviate common adverse events like fatigue. However, rare disease drug development presents significant challenges. These include small patient populations, geographic dispersion, treatment heterogeneity, study reproducibility issues, and ethical considerations. Conducting adequately powered clinical trials remains difficult due to the scarcity of patients. The traditional approach of testing one drug for one indication proves inefficient in this context. To overcome these hurdles, novel statistical approaches have emerged. These methods allow for information sharing across disease subtypes with similar characteristics, such as shared genetic alterations. Examples include master protocols, Bayesian hierarchical models and meta-analytic predictive models. However, applying these approaches to randomized controlled trials, often necessary for regulatory approval, remains a challenge. Enter Complex Innovative Trial Designs (CIDs). CIDs hold promise in clinical trials by enhancing efficiency and reducing drug development costs. The US 21st Century Cures Act and the Prescription Drug User Fee Act (PDUFA) VI actively encourage their use. The FDA’s CID Paired Meeting Program fosters discussions on alternative trial designs. In 2024, the FDA launched the Center for Clinical Trial Innovation (C3II) to promote innovative practices. While there is no fixed definition of CIDs, simulations—rather than mathematical formulas—play a crucial role in estimating trial operating characteristics. For further insights, explore our just-published white paper! https://lnkd.in/erZipKqb #SyneosHealth #ComplexInnovativeDesigns #RareDiseaseDrugDevelopment #Acceleration

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

🚀 Exciting news for the rare disease community! 🚀 The FDA has launched the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) Pilot Program, aimed at accelerating the development of therapies for rare diseases. As part of START, participants will receive invaluable guidance and support from FDA experts, addressing key challenges in clinical trial design and patient population identification. However, challenges still persist. Many in the industry have raised concerns about the Inflation Reduction Act (IRA), which some view as a barrier to orphan drug developers. While orphan drugs are exempt from IRA negotiations, this exemption only applies to drugs approved for one specific indication. Looking ahead, there's hope for positive change. Advocates are urging governmental regulators to enhance incentives for rare disease drug development in the coming year, paving the way for more treatments to reach the market. Let's work together to bring hope and healing to those affected by rare diseases. Together, we can make a difference. 💪 #RareDisease #FDA #ClinicalTrials #OrphanDrugs #HealthcareInnovation #citeline