The Healthonauts’ Post

🌍 The Rare Disease Treatment Market: Opportunities Amid Challenges! The rare disease treatment market is set to soar from USD 216.65 billion in 2024 to an impressive USD 380.62 billion by 2029, with a CAGR of 11.93%. 🚀 This growth is driven by increasing rare disease cases, innovative therapies, and government initiatives like the US FDA’s Accelerating Rare Disease Cures (ARC) Program. 🔬 Why is rare disease research so challenging?  With over 6,000 rare diseases affecting more than 300 million people globally and its crucial to recognise that, collectively these conditions are not rare. Research is often slowed by small patient populations, lack of data, and limited funding. Each rare disease presents unique complexities, making clinical trials harder to design, and drug development is often riskier and more expensive. At Biorizon Consulting, we have extensive experience overcoming these barriers. Our team offers deep expertise in preclinical trial design, strategic market analysis, and regulatory navigation, ensuring your research and development programs are designed to succeed in this challenging field. Whether it’s tailored strategies for rare diseases or helping you leverage the latest scientific advancements, we provide the support needed to turn these complexities into actionable insights. Let’s accelerate your rare disease research together—contact Biorizon Consulting  for expert guidance in navigating these hurdles!

I’m delighted to hear that the FDA is set to launch a Rare Disease Innovation Hub which will help enhance collaboration and accelerate the development and approval of treatments for rare diseases. The Hub will bring together various FDA Centres to leverage existing programmes and create a unified strategy for better patient outcomes. It primarily aims to: 🔹 Connect with the rare disease community  🔹 Enhance intercentre collaboration  🔹 Advance regulatory science In a similar vein, LifeArc is also working to improve infrastructure and collaboration within the rare disease ecosystem through our Translational Centres for Rare Diseases. We are investing £40 million in these Centres, which were created to address fragmented expertise, dispersed patient populations, and limited funding, all of which stall rare disease research. Each Centre focuses on unmet needs in different fields including rare respiratory, kidney, and mitochondrial diseases, and a Centre that aims to boost the capacity and efficiency of rare disease trials across the UK. It’s great to see this same collaboration and infrastructure-building happening across the pond, as we all work to unlock new diagnostic tests, treatments, and cures. Read more: https://lnkd.in/eKag_6E9 

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

Today marks Rare Disease Day. Globally, approximately 6,000 to 7,000 rare diseases have been identified, with new ones continually emerging. Despite each rare disease affecting only a small number of individuals, the cumulative impact is significant, touching about 7% of the population. Obtaining a confirmed diagnosis for a rare disease is often a formidable challenge. Developing drugs for these conditions is even more difficult, given the limited number of patients. This not only hampers research into disease mechanisms but also complicates clinical studies due to slow patient recruitment and achieving statistical significance. In 2017, Joern Klinger and I founded biotx.ai with the mission of providing algorithmic solutions for analyzing high-dimensional data—characterized by many features and a small sample size. We are particularly pleased to witness the integration of these methods into the planning of clinical trials, allowing for the stratification of patients based on biomarkers and the definition of improved clinical outcomes. All with the overall goal to make clinical trials in rare diseases smaller and more successful. An additional milestone that brings us immense satisfaction is our recent appointment to the scientific advisory board of Simbec-Orion, a full-service contract research organization specializing in oncology and rare diseases. We are humbled and grateful. Every step forward, whether in refining analytical approaches or contributing to the scientific advisory board, reinforces our dedication to advancing solutions for those affected by rare diseases. Together, let's ensure that accessibility to effective treatments becomes a reality for everyone. Drugs for rare diseases must not be a luxury. #RareDiseaseDay #BiotxAI #ResearchInnovation

𝐓𝐡𝐞 𝐧𝐞𝐰 "𝐀𝐧𝐭𝐢 𝐂𝐑𝐎" 𝐥𝐨𝐨𝐤𝐢𝐧𝐠 𝐭𝐨 𝐝𝐢𝐬𝐫𝐮𝐩𝐭 𝐭𝐡𝐞 𝐢𝐧𝐝𝐮𝐬𝐭𝐫𝐲 Lindus Health, known as the "anti-CRO," has launched a comprehensive CRO tailored for sponsors aiming to bring diagnostic products to market. With a track record of enrolling over 30,000 patients in diagnostic trials across the US, UK, and Europe, Lindus Health offers expertise in various therapeutic areas like oncology, infectious disease, respiratory, and women's health. Their new offering, the "All-in-One Diagnostics CRO," aims to streamline diagnostics clinical trials by leveraging their experience, network, and proprietary technologies. Lindus Health addresses the unique challenges of diagnostics studies, such as large-scale recruitment requirements, by offering specialized recruitment tactics and expert regulatory guidance. #cro #disruption

🔬 Exciting Research Alert! 🔬 A groundbreaking study aims to identify biomarkers for Inflammatory Bowel Disease (IBD), a chronic condition affecting over 540,000 people in the UK. This research is a significant step towards personalized medicine, aiming to develop more targeted and effective treatments for IBD patients. Understanding specific biomarkers associated with IBD will pave the way for tailored therapies, improving patient outcomes and quality of life. Stay tuned for more updates on this innovative research! For more details, check out the full article here: https://lnkd.in/gWxp28zD #IBD #PersonalizedMedicine #Healthcare #MedicalResearch #Biomarkers #Innovation #PatientCare #ChronicIllness #HealthScience

https://lnkd.in/g_jzxcqR Article title: Lipid nanoparticulate drug delivery system for the treatment of hepatic fibrosis Author(s): Swarupananda Mukherjee; Ayon Dutta; Dipanjana Ash Journal: Archives of Hepatitis Research Journal ISSN: 2641-2977 Abstract: Background: Irreversible hepatic fibrosis, an excessive production and accumulation of extra cellular matrix by hepatic stellate cells in the liver, becomes a remarkable economic burden in global health care system.Low therapeutic efficacy and undesirable systemic effect of conventional therapies limit their clinical applications to targethepatic stellate cells. Method: Surface engineered lipid nano-particle becomes a potential candidate to deliver anti-fibrotic nutrients or Small interfering RNA (siRNA) of fibrogenic genes for treating hepatic disorders. #Chemokines #Interferon #Hepatic #Transforming #Receptor #AcuteHepatitis #AlcoholicHepatitis #AutoimmuneHepatitis #ChronicHepatitis #ChronicHepatitisB #ChronicHepatitisC #DeltaHepatitisTreatment #DifferentialDiagnostics #GiantCellHepatitis #Hepatitis #HepatitisTreatmentAndManagement #HepatitisB #HepatitisBVaccination #HepatitisBVaccine #HepatitisBVirus #HepatitisC #HepatitisCTreatment #HepatitisE #HepatitisEpidemiology #HepatitisPrevalence #HepatitisTenofovir #HepatitisThalassemia #HepatitisTherapy #Peertechz #PeertechzPublications #OpenAccess #ScientificJournals #PeerReviewedJournals #OpenAccessPublishers #HepatitisThrombocytopenia #HepatitisTransmission #HepatitisV #IschemicHepatitis #Mechanism #MolecularGeneticsOfHepatitis #MolecularPathologyOfHepatitis #OccultHepatitis #ToxicAndDrugInducedHepatitis #ViralHepatitis

#Chronickidneydisease is a progressive condition that affects >10% of the general population worldwide. Current #clinicalbiomarkers prove effective at advanced stages of #renalimpairment, limiting the early use of potentially successful #therapeuticinterventions. There is an unmet need for more sensitive and specific biomarkers to detect #CKD earlier to improve patients' outcomes. Learn more: https://buff.ly/3wFB2yV BIOMEDICA offers #assays to reliably detect biomarkers in #blood and #urine #samples of #patients with kidney diseases. • Endostatin, Vanin-1, Periostin, FGF23, IL-6 and more.. Reliable and efficient #ELISA kits for your #research! #biomedica #biomedicaimmunoassays #kidneydisease #acutekidneyinjury #renalfibrosis #biomarker #prognosis

💡 ICYMI, one of our clinical collaborators Dr Emma Saunsbury, presented her team's crucial research on #HepatitisC at #EASL2024 in Milan earlier this month. Here's a quick summary: 🎯 Defining the data requirement to achieve the hepatitis C elimination in the United Kingdom: the Somerset liver improvement project Despite efforts to meet the WHO's 2030 target for hepatitis C (HCV) elimination, many UK patients remain untreated. To efficiently identify these patients and assess the impact of deprivation on them, Dr Saunsbury and her team used our liver disease case-finding tool to scan through existing blood test data. They found: 💡 67% of the study's 14,525 patients with abnormal liver function tests didn't receive HCV antibody tests. 💡 1,001 patients were HCV antibody positive, predominantly from deprived areas. 💡 Higher early mortality was noted among HCV-positive patients in deprived areas. This research highlights the urgent need for a systematic approach to diagnosing hepatitis C. It is crucial that we ensure all patients with abnormal LFTs have HCV antibody and PCR tests, integrate data across geographies, and regularly refresh data to address new untreated populations. We're incredibly proud to have contributed to this work and look forward to continued cooperation with Dr Saunsbury and other leading hepatologists across the UK and the world. To learn more about the study and how you can collaborate with us for your liver disease studies, don't hesitate to reach out! Speak to our team 👉 https://lnkd.in/eKs4-Mjn  Read the abstract 👉 https://lnkd.in/eVU7h-Xz #LiverDisease #PublicHealth #MedicalTechnology #Hepatology