Managed Healthcare Executive’s Post

Informing a national #raredisease registry strategy in #Australia: a mixed methods study The conclusions documented in this study released today seem to be universal from my view point, but are great to see documented to learn from... "The main challenges proved to be cost/funding of data collection, data completeness, and patient consent"...."Conclusion Findings from this study highlighted significant dataset heterogeneity based on the individual disease, and current lack of interoperability and coordination between different existing Rare Disease Registries". If you are building a national or global rare disease registry or just need data on a rare disease come talk to us. We can put you on a path to success and learn from the findings cited above. Join over 75 rare disease groups that are leveraging our innovative platform to collect data from 113 countries. #acrosshealthcare #acrossmatrix #JoinTheMatrix Website: https://lnkd.in/gKJeq7hk https://lnkd.in/d4FcRbj2

In honor of #RareDiseaseDay, we’re sharing an interview with @RareGenomics, covering the importance of patient connection and community. Read the full conversation here: https://hubs.ly/Q02mJFq20

Natural history of disease studies are foundational elements of integrated evidence generation plans, providing critical insights into disease progression, variability, and patient outcomes. Integrating natural history of disease studies into broader evidence generation strategies allows for a more comprehensive and nuanced understanding of how new therapies can improve patient outcomes, particularly in the context of rare and complex diseases. #iegp #ieps #clinicalresearch #evidencegeneration #naturalhistorydisease #clinicalevidence

Last week, HealthLumen attended Rare Diseases Forum 2024 to learn about how Europe can secure competitiveness in the R&D space for people living with rare diseases. Bringing together a range of key stakeholders, discussions focused on how a collective European Action Plan for rare diseases can be mobilised – with calls for changes to the legislations, regulations, and data standards impacting rare disease patients. Take a look at our most recent blog post to read more about the key takeaways from the event. Global Counsel #rarediseases #orphandrugs #ResearchandDevelopment 

In a survey of PCPs and GI-Hep providers across 5 US safety-net health systems, knowledge gaps and perceived barriers to HCC surveillance were identified, which can targeted with educational interventions to improve HCC surveillance in cirrhosis patients. https://ja.ma/3UOs9f4

🔊New podcast klaxon🔊 The steatotic liver disease nomenclature was published last year to much fanfare, but what prognostic information does it convey? How do clinical outcomes differ between #MASLD, #MetALD, and #ALD subclasses? For this episode I spoke to Mads Israelsen, first author on a new paper exploring the nomenclature in a Danish cohort of patients with a history of excessive alcohol intake. We cover👇 - Why the need for a new nomenclature replacing #NAFLD? - Study design and participants - Key results, including the stepwise increase in the risk of hepatic decompensation and overall mortality from MASLD, through MetALD, to ALD - What the findings mean for patients, clinicians, and triallists as we enter a new era of therapeutics Listen here or search on your preferred platform: https://lnkd.in/eWZkRZ5Y Read the full paper: https://lnkd.in/ewc-kJPM And the brilliant linked comment by Fredrik Åberg: https://lnkd.in/egKEcQkp Aleksander Krag Nikolaj Torp Camilla Dalby Hansen Katrine Thorhauge Johanne Kragh Hansen Charlotte Wernberg Peter Andersen Katrine Prier Lindvig Emmanuel Tsochatzis Maja Thiele Maru/Mary Rinella

Navigating rare disease treatment challenges is tough. Real-world evidence (RWE) is a game-changer, accelerating innovation outside traditional trials. RWE becomes the catalyst, propelling breakthroughs by providing insights into treatment efficacy and safety in real-world scenarios. Uncover how real-world evidence is rewriting the rules, accelerating breakthroughs outside traditional trials in our latest blog. #MarksManHealthcare #BlogPost #RareDiseases #RealWorldEvidence #Innovation #PatientCentricity

Optimizing Real-World Evidence through Disease Registries: Key Insights from the Experts 📊🔍 *The recent webinar on "Partnering with Disease Registries for Real-World Evidence Generation" provided an in-depth look at the critical role of disease registries in the landscape of healthcare and pharmaceutical research. Here are the key insights and takeaways from the discussion: ☑ Historical Evolution and Significance: Disease registries, now more commonly referred to as clinical registries, have evolved from basic surveillance tools to sophisticated systems supporting a broad range of research activities. Their evolution reflects the growing complexity and specificity of healthcare needs and research questions. 📜➡️🔬 ☑ Multifaceted Applications: Today’s clinical registries serve multiple purposes: from supporting epidemiological surveillance and patient-centric outcomes research to informing clinical trial designs and regulatory submissions. Their versatility makes them invaluable across various stages of pharmaceutical and medical product lifecycles. 🌐👥 ☑ Stakeholder Collaboration: Effective registry utilization requires collaboration among diverse stakeholders, including academia, government agencies, and industry. This multi-stakeholder approach ensures that registries address comprehensive healthcare questions and contribute to meaningful health outcomes. 🤝💡 ☑ Technological Advancements: The integration of advanced technology with registry operations has transformed data collection and analysis. This shift from manual, CRF-based data entry to automated, real-time data capture enhances the quality and speed of evidence generation. 💻🚀 ☑ Regulatory and Ethical Considerations: Navigating the regulatory landscape and ensuring ethical management of registry data are pivotal. As registries evolve, so do the regulatory frameworks governing them, necessitating continuous adaptation and compliance. ⚖️📏 ☑ Future Directions: The future of clinical registries lies in their ability to adapt and integrate with emerging technologies and healthcare paradigms. This will likely include greater use of artificial intelligence, machine learning, and enhanced patient engagement through digital platforms. 📈🤖 *This discussion underscores the importance of disease registries not only as data repositories but as dynamic tools capable of driving forward medical research and healthcare improvements. As we continue to face complex health challenges, the strategic use of registries will be crucial in developing effective and targeted therapies. Special shout out to key contributors: Javier Cid Nahila Justo, PhD MBA Delphine Saragoussi, MD, MScPH #HealthcareInnovation #RealWorldEvidence #ClinicalResearch #DataScience #MedicalAffairs

I recently had the pleasure of meeting Vera Mucaj, who recently published an insightful piece on Medium. In her article, Vera discusses the crucial role of AI and large datasets in the fight against rare diseases. She highlights the need for innovative approaches to accelerate diagnoses and therapy discoveries. This is a must-read for anyone passionate about advancing healthcare innovation and improving the lives of those affected by rare diseases. Check it out! #HealthcareInnovation #RareDiseases #AI #DataScience

I'm excited to announce my recent engagement with a new publisher to discuss comprehensive management and treatment strategies for Chronic Hepatitis B. Chronic Hepatitis B poses a significant global health challenge, emphasizing the need to develop and disseminate effective management strategies. Our discussions focused on innovative approaches, patient education, and the value of multidisciplinary care. Our goal is to equip healthcare professionals with the knowledge and resources necessary to enhance patient outcomes and improve the quality of life for those impacted by this disease. Stay tuned for more updates as we collaborate to share valuable insights and research in this critical field! 🌍💡 #HepatitisB #PublicHealth #HealthcareInnovation #ChronicDiseaseManagement #PatientCare Link to the discussion: https://lnkd.in/gVHzGC8z