BridgeBio’s Post

Did you know almost 10 years ago the European Medicines Agency established the Patient Registries Initiative to enhance the use of registries in regulatory processes? At Cisiv Ltd, we are continually advocating for the use of patient registries. There is an untapped potential of patient registries in regulatory decision-making for medicinal products. Setting up a registry means you can systematically collect critical data on patients with specific diseases or conditions over time, offering valuable insights for regulatory decision-making, especially for rare diseases. A super interesting paper we recommend, that can be found below, written by Patricia McGettigan, Carla Alonso, Kelly Brown-Plueschke, Mireia Castillón Melero, Daniel Nogueras Zondag, Priya Bahri and Xavier Kurz discusses and highlights the potential and current underutilisation of patient registries in regulatory assessments of medicinal products. If you want to learn more about how to set up a registry or discuss your own registry, get in touch through https://lnkd.in/eH-gkw4w or at email us at info@cisiv.com #realworldevidence #patientregistries #EMA #healthcare

Comparing the EMA and FDA on Statistical Significance in Pivotal Studies When conducting pivotal clinical trials, both the EMA and FDA share the requirement for statistical significance (p-value < 0.05), but their approaches diverge in several ways. While the FDA is more flexible—especially in cases of unmet medical needs, rare diseases, or innovative trial designs—the EMA often demands a higher level of statistical rigor and robust evidence to support clinical relevance. 1) P-value Threshold: Both agencies require p-values below 0.05 for significance, but the EMA tends to emphasize clinical relevance more stringently. 2) Multiplicity Adjustments: Both agencies require adjustments when multiple endpoints are tested, but the EMA applies stricter scrutiny to ensure robustness in single pivotal studies. 3) Confidence Intervals: The EMA typically demands narrower confidence intervals to ensure the treatment effect is precisely estimated, while the FDA may balance precision with the urgency of providing treatment. 4) Real-World Evidence: The FDA has been more open to accepting real-world evidence and innovative trial designs, while the EMA remains cautious, often requiring more validation through traditional methods. Both regulatory bodies ensure that statistically significant results translate into meaningful improvements for patients, but their paths to approval reflect differing levels of flexibility. References: European Medicines Agency. Guideline on the Investigation of Subgroups in Confirmatory Clinical Trials. Available at: https://lnkd.in/gWfpx3y8 U.S. Food and Drug Administration. Various Statistical Considerations for Clinical Trials. Available at: https://lnkd.in/gMg3acuM Also, please don't forget to peruse this insightful journal; Wang, X., & Ji, X. (2020). Sample size estimation in clinical research: from randomized controlled trials to observational studies. Chest, 158(1), S12-S20. #ClinicalTrials #CRO #StatisticalAnalysis #Power #Type1Error #Type2Error #CNRResearch

🎉 Good news! The European Medicines Agency’s Committee for Medicinal Products for Human Use has recommended granting a marketing authorisation in the European Union for the IMI-supported treatment EMBLAVEO® (a fixed dose combination of two active substances - aztreonam and avibactam). 👉 The treatment is indicated for complicated intra-abdominal and urinary tract infections, hospital-acquired pneumonia and infections caused by Gram-negative bacteria. 👉 The IMI COMBACTE-CARE project supported two clinical trials for EMBLAVEO® - REJUVENATE and REVISIT. 👉 Niklas Blomberg, our Executive Director, says: “This excellent result from the COMBACTE-CARE project is a testament to the cooperative approach of the public and private partners. And it demonstrates their joint commitment to finding innovative solutions to address the urgent public health need of anti-microbial resistance." 👉 Read our press release here: https://europa.eu/!mrHN8F #IHITransformingHealth

#LAC24 is kicked off in Dublin today🍀 Key high level panellists are on stage for the first panel debate form Muiris O’Connor from the Irish Dept. of Health, the European Patients Forum, and General Counsel from Fresenius Kabi and Sandoz. Discussions highlighted the need to improve the objectives of the European Commission #pharma reform: accessibility, availability, and affordability of medicines to ensure timely generic and biosimilar competition at IP expiry. This can be delivered by: - Regulatory modulation of incentives for innovation that keeps 11 years cap (data & market) while ensuring #Access to all member states - Harmonised #Bolar exemption across all MS - Incentivising innovation on #AMR through fairer and more effective alternatives to the transferable voucher Bird & Bird European Patients' Forum Fresenius Kabi Sandoz Department of Health - Ireland

The European Medicines Agency (EMA) has launched a new pilot program aimed at supporting the development of orphan medical devices - technologies designed to diagnose, prevent, or treat rare diseases affecting fewer than 12,000 people annually in the EU. The pilot program, running until the end of 2025, offers free expert guidance to manufacturers and notified bodies at every stage—from concept development to market approval. Key Highlights: ➡ The pilot program will prioritize devices intended for children, those addressing unmet medical needs, and novel devices that promise significant clinical benefits. ➡ This initiative is part of EMA's broader regulatory support following the introduction of new EU legislation, which aims to enhance the clinical evaluation process for these devices. ➡ The guidance provided will help manufacturers and notified bodies navigate the clinical evidence requirements more effectively. #OrphanMedicalDevices #HealthcareInnovation #EMA #MedicalDevices #PatientCare https://lnkd.in/eTHYUFZC

The European Medicines Agency has published a Clinical Trials Information System newsflash.  - Key updates include the latest KPI Report with data from March 2024 on the implementation of the Clinical Trials Regulation has been published on the ACT EU website. To discuss this newsflash with a member of the team, get in touch now www.scendea.com/contact - #guidanceupdate #regulatoryaffairs #drugdevelopment #CTISnewsflash

Discover the latest EMA guidance now!

The FDA has announced the Patient Engagement Advisory Committee (PEAC) meeting will be held virtually on October 30, 2024. During this meeting, PEAC will discuss and make recommendations on patient-centered informed consent in clinical study of FDA-regulated medical products. The topics for discussion include: 1.The informed consent process, and 2.Factors to consider when communicating informed consent to clinical study participants to increase the likelihood of participants understanding the key elements of research.

#portfolionews I Our portfolio company Xeltis has received Investigational Device Exemption (IDE) approval from FDA - La Administración de Alimentos y Medicamentos for initiation of US pivotal #trial for aXessTM, its restorative vascular access conduit. 👏 👉 Full details here https://bit.ly/4c3uSsf 🎯 aXess is a restorative conduit that enables the creation of a new, permanent, living vessel for #hemodialysis vascular access, combining the #safety and patency of an arteriovenous fistula (AVF), with the speed to treatment of an arteriovenous graft (AVG). The aXess vascular access conduit offers an improved dialysis patient experience and avoids the frequent reinterventions and complications, such as infections, faced by many renal disease patients. 🔬 The #US-based pivotal trial follows strong 12-month data from the first-in-human trial in Europe in comparison to hemodialysis vascular access solutions. aXess is also undergoing an EU pivotal trial, recruiting up to 110 patients in nine #EU countries. #FDA #VascularAccess #IDE #hemodialysis #renal #vc #innovation #MedicalInnovation #Healthcare #RenalCare #FDAApproval #ClinicalTrials 

Read Scendea's breakdown of the latest CTR guidance published by the EMA!