Life Sciences Trainers & Educators Network (LTEN)’s Post

A New Era For Patient Recruitment & Retention In Rare Disease? Pharma and biotech companies continue to prioritize diversifying their pipelines to address high and rare unmet needs, but rare diseases present challenges in R&D, especially patient recruitment and retention. This article looks at the challenges and promising changes to come... #raredisease #patientrecruitment

The American Association for the Study of Liver Diseases (AASLD) #TLM24 may be over, but ProSciento’s dedication to advancing metabolic disease research continues. If you missed us at the conference, let’s connect! As the leading CRO specializing in steatotic liver diseases like MASLD and MASH, ProSciento brings unmatched expertise to complex SLD clinical trials. Our global network and proven science-driven strategies streamline study execution, expand patient access, and enhance site capabilities to improve patient care and advance therapeutics. Reach out to learn how we can support your metabolic continuum program: https://bit.ly/3KnHEGw. #AASLD2024 #AASLD #TheLiverMeeting #LiverResearch #MetabolicDisease #MetabolicContinuum #ClinicalTrials #SLDResearch #SteatoticLiverDisease #LiverDisease #ScienceDriven #PatientFocused #MissionCentered

Bayer unveils pivotal FINEARTS-HF trial insights at AHA 2024, highlighting finerenone's impact on heart failure and kidney disease management. #ClinicalResearches #AHA2024 #HealthcareInnovation #PharmaceuticalCompanies #Pharma #MarketAccessToday #MarketAccess

Drug shortages can be life-threatening for patients, causing treatment delays, missed doses, and reliance on less-effective treatments. With this urgent problem in mind, US Pharmacopeia and the American Cancer Society Cancer Action Network (ACS CAN) are pleased to launch a task force to identify solutions to mitigate and prevent drug shortages. This coalition of diverse organizations representing patients, practitioners, and caregivers, calls on policymakers to address root causes, risks, and vulnerabilities in the medicines supply chain. Together, we can ensure that patients have stable, consistent access to the treatments they need. We encourage you to join us by taking these actions. ⤵️ 1️⃣ Visit https://ow.ly/6WZQ50QJuRn to learn more about our call to action, recommendations, and upcoming ways to get involved. 2️⃣ Help spread the word with a re-post or tagging your network to join in a comment below. 3️⃣ Follow and engage with our task force members: Alliance for Aging Research Angels for Change American Pharmacists Association Arthritis Foundation ASHP American Society of Clinical Oncology (ASCO) Cancer Support Community Friends of Cancer Research Generics Access Project Hemophilia Federation of America Howard University-College of Pharmacy Susan G. Komen National Consumers League (NCL) National Psoriasis Foundation #DrugShortages #SupplyChain #PatientAccess

Patient centricity has become the norm in biopharma, at least it should be, right? 🤷♀️ Well.. a report on HCPs attitudes to biopharma companies only 45% of them feel they embody patient centricity at a high level. #Patientcentricity is now more than just patient research and usability testing. Its: 1. engaging the community as contributors in the drug development  2. co-creation in the design of end to end clinical trials 3. leveraging the communities that surround patients to understand the true patient experience AND A LOT MORE! Avalere Health have developed an article touching on how rare disease teams may approach the ultimate goal of patient co-creation. Why not check it out? https://bit.ly/3SRVGDA Other helpful information on Rare Disease patient co-creation: - National Organization for Rare Disorders (NORD) at NCSU have developed on Education Series to Advance Patient Involvement in Rare Disease Drug Development - Rare Disease Moonshot- an organisation working with patient groups to optimize translational research, modernize clinical trials, and develop the infrastructure to shorten the path to diagnosis and treatment. #raredisease #patientcentricty #biopharma #patientexperience #drugdevelopment #thoughtleadership

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

We are live at the World Orphan Drug Congress USA! Come meet with us at booth #413 and talk about how we can tackle rare disease challenges together. We can help find people living with rare disease that are still undiagnosed, often many years before they might eventually be diagnosed following standard clinical diagnostic pathways. And help understand who are most at risk of rapidly progressing towards poor outcomes, thus helping HCPs triage and accelerate care pathways for better outcomes for patients. If you have missed our pre-conference workshop yesterday, the slides and, importantly, survey are now uploaded to the Terrapinn World Orphan Drug Congress USA conference app. See link in comments. And if you are at the event, come listen to our speaker session "How you can deliver consequential projects for patients, using AI in multi-year deployments.". See session link in comments. #inTrigue #inAdvance #inFlow #rarediseaseawareness #rarediseaseresearch With Volv Global SA 💭 Christopher Rudolf and Mike Musson at the Terrapinn World Orphan Drug Congress USA with Justin Franks, Alexandria Edleman , Giovanna Ronzetti and many others.

The FDA just announced a significant step forward in the fight against rare diseases with the launch of the Rare Disease Innovation Hub. With over 10,000 rare diseases affecting more than 30 million people in the U.S., around half of whom are children, the need for innovative treatments has never been more critical. Key goals of the hub include: •Serving as a central point for engagement with patient groups, scientific organizations, and industry stakeholders.  •Enhancing collaboration across FDA centers to tackle scientific, clinical, and policy challenges. •Advancing regulatory science through innovative approaches in trial design, biomarker development, and more. •This collaborative model will integrate efforts from existing FDA programs, such as the CDER Accelerating Rare disease Cures (ARC) program and the CBER Rare Disease Program, and will be supported by a newly created director of strategic coalitions, who will ensure community input shapes priorities effectively. A public meeting is planned for this fall to provide more information and gather stakeholder feedback. It is refreshing to see the rare disease patient community having such a prominent seat at the table, which will no doubt be a boon for the development of new therapeutic options. I’m optimistic about the positive impacts ahead. Read the full announcement: https://lnkd.in/dhjUtjuh #RareDisease #PatientEngagement #HealthcareInnovation

Many of you have inquired about the recent FDA meeting focused on the Rare Disease Innovation Hub. The meeting covered four essential topics: Cross-Cutting Rare Disease Issues: The need for a more flexible regulatory framework and clearer guidelines, particularly concerning natural history studies. Rare Disease-Specific Issues: Ethical considerations surrounding the use of placebos in pediatric trials. Joint Activities by CDER or CBER: A call to streamline processes and reduce redundancy in data collection. Engagement Approaches: Emphasizing patient and community engagement as a fundamental strategy. The urgency for change was palpable, and many participants left the meeting feeling hopeful. It’s clear that transformative policies are necessary for progress, and I am optimistic that the insights shared will drive meaningful advancements in the drug development landscape for rare diseases. For more key takeaways, be sure to check out my latest blog post. #RareDiseases #FDA #PatientAdvocacy #InnovationHub #DrugDevelopment EveryLife Foundation for Rare Diseases

💡Did you know, in the past 5 years, we have supported >350 rare disease clinical trials & consulting projects? Download our fact sheet to learn more about how we can help you advance development and approval of rare/ultra-rare disease therapies 📩 https://bit.ly/3IY5yaj #RareDisease #UltraRareDiseases