Global Clinical Trials (GCT)’s Post

The FDA has announced the release of a draft guidance for industry titled “Pediatric Inflammatory Bowel Disease: Developing Drugs for Treatment.” Developed by the Division of Gastroenterology within the Center for Drug Evaluation and Research, this guidance aims to assist sponsors in the clinical development of treatments for pediatric ulcerative colitis and Crohn’s disease. It outlines key considerations for study population, design, efficacy, and safety assessments necessary for developing effective treatments for these conditions in children. This initiative underscores FDA’s commitment to improving therapeutic options and outcomes for pediatric patients with inflammatory bowel disease. Know more - https://lnkd.in/e45GyWEj #Pharmacovigilance #ClinicalResearch #FDA #Pediatrics #InflammatoryBowelDisease #DrugDevelopment

Congress is in discussion of reauthorizing the FDA's Pediatric Rare Disease Priority Review Voucher (PRV) Program. This program has been crucial in accelerating the development and approval of treatments for rare pediatric conditions, enabling life-saving therapies to reach children in need more swiftly. The PRV program incentivizes pharmaceutical companies to prioritize rare diseases, a critical area of healthcare. With its expiration imminent, prompt congressional action is necessary to sustain this vital program. Reauthorizing the PRV program is essential for maintaining progress in the fight against rare pediatric diseases and improving patient outcomes. #HealthcareInnovation #PediatricCare #RareDiseases #FDA

In this #blog, Nazim Kanji, Executive Director of Pediatric Services, discusses how strong sponsor and CRO/CDMO partnerships help streamline orphan/rare and #pediatric drug development. He also details a case study where #QuotientSciences worked on a high level of customization of a drug product to support an extensive Phase II/III clinical program. Read more: https://bit.ly/4cv54G9

Advancing therapies intended to treat pediatric patient populations is highly complex with numerous confounding challenges. With this, trial design and conduct come with heightened scrutiny for regulatory, ethical, and safety standards for children as patients. 🆕Watch our latest video today to discover how Veristat can help design and run your next pediatric clinical development studies📽️ https://bit.ly/3Us4BMK #Pediatric #PediatricClinicalDevelopment

𝐄𝐧𝐡𝐚𝐧𝐜𝐢𝐧𝐠 𝐏𝐞𝐝𝐢𝐚𝐭𝐫𝐢𝐜 𝐃𝐫𝐮𝐠 𝐃𝐞𝐯𝐞𝐥𝐨𝐩𝐦𝐞𝐧𝐭: 𝐈𝐂𝐇 𝐄𝟏𝟏 𝐆𝐮𝐢𝐝𝐞𝐥𝐢𝐧𝐞 𝐚𝐧𝐝 𝐌𝐨𝐝𝐞𝐥𝐢𝐧𝐠 & 𝐒𝐢𝐦𝐮𝐥𝐚𝐭𝐢𝐨𝐧 👶💊 While pediatric extrapolation has been used, past guidelines provided limited harmonized approaches across regions. This led to potential delays in #pediatric access and unnecessary #clinicaltrials exposing children unnecessarily. 𝗧𝗵𝗲 𝗡𝗲𝗲𝗱? Reducing likelihood of differences between regions through harmonized #guidelines on extrapolation can: ⏬ Reduce pediatric exposure in unnecessary trials  🌍 Facilitate timely global access to pediatric medicines The International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) has developed a harmonized guideline, ICH E11, 𝘁𝗼 𝗳𝗮𝗰𝗶𝗹𝗶𝘁𝗮𝘁𝗲 𝘁𝗵𝗲 𝘁𝗶𝗺𝗲𝗹𝘆 𝗱𝗲𝘃𝗲𝗹𝗼𝗽𝗺𝗲𝗻𝘁 𝗼𝗳 𝘀𝗮𝗳𝗲 𝗮𝗻𝗱 𝗲𝗳𝗳𝗲𝗰𝘁𝗶𝘃𝗲 𝗺𝗲𝗱𝗶𝗰𝗶𝗻𝗲𝘀 𝗳𝗼𝗿 𝗰𝗵𝗶𝗹𝗱𝗿𝗲𝗻. This guideline outlines an approach to the ethical study of medicinal products in the pediatric population. 𝗤𝘂𝗮𝗻𝘁𝗶𝘁𝗮𝘁𝗶𝘃𝗲 𝗺𝗼𝗱𝗲𝗹𝗶𝗻𝗴 𝗮𝗻𝗱 𝘀𝗶𝗺𝘂𝗹𝗮𝘁𝗶𝗼𝗻 (𝗠&𝗦) 𝗺𝗲𝘁𝗵𝗼𝗱𝘀 𝗶𝗹𝗹𝘂𝗺𝗶𝗻𝗮𝘁𝗲 𝗮𝗻 𝗶𝗻𝘀𝗶𝗴𝗵𝘁𝗳𝘂𝗹 𝗽𝗮𝘁𝗵 𝗳𝗼𝗿𝘄𝗮𝗿𝗱 𝗶𝗻 𝗽𝗲𝗱𝗶𝗮𝘁𝗿𝗶𝗰 𝗱𝗿𝘂𝗴 𝗱𝗲𝘃𝗲𝗹𝗼𝗽𝗺𝗲𝗻𝘁 𝗯𝘆 𝘀𝘆𝗻𝗲𝗿𝗴𝗶𝘀𝘁𝗶𝗰𝗮𝗹𝗹𝘆 𝗵𝗮𝗿𝗻𝗲𝘀𝘀𝗶𝗻𝗴 𝗲𝘅𝗶𝘀𝘁𝗶𝗻𝗴 𝗱𝗮𝘁𝗮 𝗿𝗲𝘀𝗲𝗿𝘃𝗼𝗶𝗿𝘀 𝘁𝗼 𝗯𝗿𝗶𝗱𝗴𝗲 𝗸𝗻𝗼𝘄𝗹𝗲𝗱𝗴𝗲 𝗴𝗮𝗽𝘀, 𝗴𝘂𝗶𝗱𝗲 𝗼𝗽𝘁𝗶𝗺𝗮𝗹 𝘀𝘁𝘂𝗱𝘆 𝗱𝗲𝘀𝗶𝗴𝗻𝘀, 𝗮𝗻𝗱 𝗽𝗿𝗼𝗴𝗻𝗼𝘀𝘁𝗶𝗰𝗮𝘁𝗲 𝗽𝗵𝗮𝗿𝗺𝗮𝗰𝗼𝗸𝗶𝗻𝗲𝘁𝗶𝗰, 𝗽𝗵𝗮𝗿𝗺𝗮𝗰𝗼𝗱𝘆𝗻𝗮𝗺𝗶𝗰, 𝗮𝗻𝗱 𝘀𝗮𝗳𝗲𝘁𝘆 𝗼𝘂𝘁𝗰𝗼𝗺𝗲𝘀 - 𝗽𝗼𝘁𝗲𝗻𝘁𝗶𝗮𝗹𝗹𝘆 𝗺𝗶𝗻𝗶𝗺𝗶𝘇𝗶𝗻𝗴 𝘁𝗵𝗲 𝗻𝘂𝗺𝗯𝗲𝗿 𝗼𝗳 𝗽𝗲𝗱𝗶𝗮𝘁𝗿𝗶𝗰 𝗽𝗮𝘁𝗶𝗲𝗻𝘁𝘀 𝗿𝗲𝗾𝘂𝗶𝗿𝗲𝗱 𝗳𝗼𝗿 𝗰𝗹𝗶𝗻𝗶𝗰𝗮𝗹 𝘁𝗿𝗶𝗮𝗹𝘀. By embracing M&S, drug developers can enhance the #efficiency, #safety, and #ethical conduct of pediatric clinical trials, ultimately #accelerating access to essential #medicines for children worldwide. 🌍 ✨ Want to dive deeper into the world of ICH guidelines? Check out our comprehensive blog post on 🔗 https://lnkd.in/gjdrvUYa for a detailed exploration of this game-changing initiative.    InSilicoTrials #PediatricDrugDevelopment #ICH #ModelingAndSimulation #ClinicalTrials #Extrapolation #Pediatrics 

Advancing therapies intended to treat pediatric patient populations is highly complex with numerous confounding challenges. With this, trial design and conduct come with heightened scrutiny for regulatory, ethical, and safety standards for children as patients. 📽️ Watch our video to learn how we can help you design and run your next pediatric clinical development studies 👇 https://hubs.li/Q02DNj3h0 #Pediatric #RareDiseases

The study highlights the clinical implications of inaccurate potassium determination in hemolyzed pediatric blood specimens. It notes that hemolysis, common during blood acquisition from newborns and infants, often goes undetected in laboratory assessments of whole blood. Consequently, potassium levels in whole blood may be erroneous by up to 4 mmol/L in 20% of specimens, leading to errors and delays in patient care. The findings underscore the need for better tools to detect pre-analytic hemolysis in whole blood, thereby improving the accuracy of potassium determination in pediatric patients. #Pediatrics #Hemolysis #PotassiumDetermination #BloodSpecimens #PatientCare #ClinicalImplications #LaboratoryAssessment #PreAnalyticErrors

The clinical application of paediatric serum troponin T testing remains a topic of debate. While serum troponin testing can aid in diagnosing suspected cardiac ischaemia in adults, its utility in paediatric patients with chest pain is limited due to the rarity of cardiac ischaemia in this population. Despite its widespread use in adult populations, current literature suggests caution in relying on serum troponin testing alone for diagnosing chest pain in children, given its multifactorial nature. Further research is needed to determine the optimal diagnostic approach for paediatric patients presenting with chest pain. #Pediatrics #CardiacIschaemia #TroponinTesting 🩺👶❤️

🚀 𝑭𝑫𝑨 𝑭𝒂𝒔𝒕 𝑻𝒓𝒂𝒄𝒌𝒔 𝑱𝒐𝒉𝒏𝒔𝒐𝒏 & 𝑱𝒐𝒉𝒏𝒔𝒐𝒏’𝒔 𝑵𝒊𝒑𝒐𝒄𝒂𝒍𝒊𝒎𝒂𝒃 𝒇𝒐𝒓 𝑭𝒆𝒕𝒂𝒍 𝑵𝒆𝒐𝒏𝒂𝒕𝒂𝒍 𝑨𝒍𝒍𝒐𝒊𝒎𝒎𝒖𝒏𝒆 𝑻𝒉𝒓𝒐𝒎𝒃𝒐𝒄𝒚𝒕𝒐𝒑𝒆𝒏𝒊𝒂 🚀 FNAIT is a rare disease in which the immune system of a pregnant individual attacks platelets in the developing fetus, which can affect clotting and bleeding, causing a significant risk of severe bleeding complications to the fetus or newborn. Currently, there are no approved target therapies to manage FNAIT, making the Fast Track designation for nipocalimab a significant milestone in addressing this unmet medical need. The FDA's Fast Track designation expedites the development and review of drugs intended to treat severe conditions with an unmet medical need. This designation comes after promising preclinical and early clinical data for nipocalimab, demonstrating its potential to effectively lower the risk of FNAIT in pregnant patients. Learn more about this potentially life-saving treatment option for neonates. https://lnkd.in/ejvpbX6k #unmeetneeds #pediatric #rarediesease #FDAFasttrack #Childrenshealth #JohnsonandJohnson

AJOG Expert Review in Labor: Clinical chorioamnionitis at term: definition, pathogenesis, microbiology, diagnosis, and treatment - A needle in the epidural space, optical imaging, and mechanisms of fever A, Fiberscope of the epidural space. During pregnancy, the density of the epidural vessels is higher than that of nonpregnant patients. It has been proposed that during pregnancy the inferior vena cava becomes compressed by the pregnant uterus and that this causes the epidural veins to engorge and the volume of the epidural space (asterisk, blue) to decrease. Thus, a similar volume of local anesthetic agents can spread more extensively in the pregnant than in the nonpregnant state. Modified from Eltzschig et al359 and Igarashi et al.360 B, Local anesthetics such as ropivacaine used during epidural analgesia can induce maternal systemic inflammation. Ropivacaine can directly cause endothelial cell apoptosis, and this induces the release of damage-associated molecular patterns (DAMPs), also known as alarmins, that trigger the production of pyrogenic cytokines (eg, IL-1β, IL-6, IL-8), which may be responsible for fever in pregnant women following epidural analgesia. https://ow.ly/sYb750R0hXY