Pediatric clinical trials: US, EU, UK requirements and differences

New research in Clinical Pharmacology & Therapeutics (ASCPT Journal Family) by Ameet Sarpatwari and colleagues explores patient and caregiver experiences with FDA-mandated Risk Evaluation and Mitigation Strategy (REMS) programs to ensure safe use of medications. Through interviews with 135 participants, the authors found that most patients were well-informed about REMS-related drug risks and requirements, but many faced challenges finding prescribers and navigating REMS-related administrative burdens, especially for drugs requiring frequent blood or pregnancy testing. Satisfaction was higher when care coordination support was available, and participants expressed a desire for similar communications about risks not covered in REMS materials. The authors recommend better integration of REMS into clinical workflows and enhanced communication about both REMS and non-REMS risks to improve patient access and safety. Read the full analysis via the link in the comments! Su Been Lee Heidi Zakoul, MPH Winta Tekle, MPP Ariel Freedman Shruti Belitkar Gita A. Toyserkani Cynthia LaCivita Esther Zhou Kate Heinrich Oswell Aaron S. Kesselheim, M.D., J.D., M.P.H.

Proud to share our new study in Clinical Pharmacology & Therapeutics on patient and caregiver experiences with REMS programs. We often evaluate these programs after implementation, but what if we brought patient and provider perspectives into REMS design from the start? Early engagement could transform REMS from a compliance exercise into a truly patient-centered safety system, aligned with the future of individualized medicine. https://lnkd.in/ervBxw_m #REMS #PatientCentricity #DrugSafety #RegulatoryScience

📢 Lupin launches Rivaroxaban Oral Suspension (1 mg/mL) in the U.S. Market! We are excited to introduce the generic equivalent of Janssen’s Xarelto® Oral Suspension, expanding access to vital pediatric anticoagulant therapy. 👉 Indications include: Treatment and reduction of recurrent venous thromboembolism (VTE) in pediatric patients Thromboprophylaxis for children with congenital heart disease post-Fontan procedure With estimated U.S. sales of USD 11 million (IQVIA MAT July 2025), this launch underscores Lupin’s growing presence in the high-value U.S. generics market. Together, we continue to deliver affordable, high-quality healthcare solutions worldwide. 🌍💊 #Lupin #HealthcareInnovation #Generics #USMarket #Pharmaceuticals #PediatricCare #GlobalHealthcare https://lnkd.in/gNfcVT39

At Therakann Pharmaceuticals, we’re exploring new frontiers in pediatric epilepsy care. 🧠 A recent review of cannabidiol (CBD) in Dravet Syndrome highlights its potential to significantly reduce seizures with an acceptable safety profile. While more research is needed, CBD is emerging as a promising adjunct therapy for children with challenging epilepsy. (Read the study here: https://lnkd.in/dXX4tgde) These insights inspire our mission and hint at exciting developments ahead in our pediatric epilepsy program designed to bring safer, more effective options to children and families in need. #TherakannPharmaceuticals #PediatricEpilepsy #InnovationInHealthcare #DravetSyndrome

#OnlineFirst w/ #PMT #JPharmTechnol this week, Thomas & colleagues with East Tennessee State University Bill Gatton College of Pharmacy assess use of proton pump inhibitors in outpatients with cirrhosis, identifying frequent undocumented use and potential overuse of PPIs. Free PMC Full-Text: PMC12373648 #PPIs #pharmacy #pharmacist #medicationstewardship #medicationmanagement https://lnkd.in/eu-Em-rc

Edoxaban’s Journey Shows How Targeted Pharmacology and Real-World Data Converge - Abedalrahman Maen Addassi Daiichi Sankyo | FDA https://lnkd.in/eKdxFSpE #Anticoagulation #Bleeding #DaiichiSankyo #DeepVeinThrombosis #DVT #Edoxaban #FactorXaInhibitor #Health #Hemorrhage #HemostasisToday #Medicine #OralAnticoagulant #PE #PulmonaryEmbolism #Savaysa

The recent success of Eli Lilly's Mounjaro in a Phase 3 trial for pediatric patients with Type 2 diabetes signals a major shift in the market. The trial's positive results for controlling blood sugar and reducing BMI are not just a win for patients but also a strategic victory for the company. Here's a breakdown of the market implications: • Significant Market Expansion: Gaining regulatory approval for this younger demographic (ages 10-17) opens up a new, rapidly growing patient population for Mounjaro. This directly expands the drug's total addressable market and revenue potential. • Competitive Advantage: While other GLP-1 drugs are available, Mounjaro's unique dual-action mechanism (GLP-1/GIP) provides a powerful new tool in a competitive landscape dominated by players like Novo Nordisk. This new indication could give Lilly a crucial edge in a high-demand segment. • Strengthening Market Share: The trial's strong results—including a 2.2% average A1C reduction—position Mounjaro to capture significant market share in pediatric endocrinology. The approval process, which is now underway, is a critical step toward cementing the drug’s status as a market leader. This development reinforces Lilly's position at the forefront of diabetes and weight management and demonstrates its commitment to driving innovation in high-value, unmet needs. For smart insights on market moves and how to grow your skills in today's fast world, click here for a free 15-minute call: https://www.rfr.bz/l0ad56d

FDA Clears Simponi (Golimumab) for Children with Ulcerative Colitis The U.S. Food and Drug Administration (FDA) has approved Simponi (Golimumab) for the treatment of children aged two years and older with moderately to severely active ulcerative colitis (UC) who weigh at least 15 kg. https://lnkd.in/d44Ec-3T #Simponi #Golimumab #UlcerativeColitis #PediatricUC #FDAApproval #JohnsonAndJohnson #InflammatoryBowelDisease #IBD #PediatricGastroenterology #BiologicTherapy #TNFInhibitor #ClinicalResearch #DrugApproval #MedicalAdvancements #Pharmacally #TheraSpace #Gastroenterology #AutoimmuneDisease #ClinicalTrials #FDA2025

On this day in science Thalidomide was marketed. This notorious drug was marketed as a mild sleeping pill that was safe even for pregnant women. It wasn’t until 1962 that the severe side effects were revealed, where it had caused the development of malformed limbs in babies. During early testing, researchers at the company found that it was virtually impossible to give test animals a lethal dose of the drug (based on the LD50 test). Largely based on this, the drug was deemed to be harmless to humans. Thalidomide was licensed in July 1956 for over-the-counter sale (no doctor’s prescription was needed) in Germany Thalidomide exists in two mirror-image forms: it is a racemic mixture of (R)- and (S)-enantiomers. The (R)-enantiomer, shown in the figure, has sedative effects, whereas the (S)-isomer is teratogenic. Under biological conditions, the isomers interconvert, so separating the isomers before use is ineffective. It was not until 2010, H. Handa and colleagues at the Tokyo Institute of Technology showed that its biological target is cereblon, a component of an E3 ubiquitin ligase complex. More recently, thalidomide has proven useful for treating cancer and leprosy and is approved for these uses. #science #scienceandtechnology #chemistry #thalidomide #medicalsciences #pregnancy #medicalcommunications #pharma

THALIDOMIDE: FROM TRAGEDY TO THE STRATEGIC ROLE OF PHARMACIVIGILANCE The tragedy of #thalidomide, a drug sold as a sedative and anti-nausea drug in the 1950s and 1960s, led to the discovery and creation of modern #pharmacovigilance regulations. #Thalidomide use by pregnant women caused severe congenital limb defects in newborns, and its subsequent analysis and withdrawal from the market highlighted the need for constant post-marketing monitoring of drugs to ensure their safety.