Valid Insight (part of the Bioscript Group)’s Post

📢 Last week the FDA updated its progress on the establishment of the Rare Disease Innovation Hub, a cross-center initiative aimed at accelerating rare disease treatment by fostering collaboration across the agency. Community feedback was gathered at the 'Advancing Rare Disease Therapies' event last month and is being used to inform the Hub’s direction. Amy Comstock Rick, J.D., was announced as the Director of Strategic Coalitions. With collaboration across CBER, CDER, and other FDA centers, the Hub will drive regulatory science for rare diseases through a united approach. Over 7,000 rare diseases affect more than 30 million people in the United States. Since the Orphan Drug Act came into effect in 1983, the FDA has approved hundreds of drugs for rare diseases. However, many rare conditions remain life-threatening, and most still lack treatments. Read the announcement from the FDA here: https://lnkd.in/gHyX-jUx #RareDiseaseHub #FDAInnovation #RareDisease

Rare diseases, such as sickle cell disease and thalassemia, affect up to 30 million people in the United States and at least 300 million across the globe. Our new report provides recommendations for enhancing and promoting rare disease drug development in the U.S. and the European Union by improving engagement with people affected by a rare disease, advancing regulatory science, and fostering collaboration between the FDA and the European Medicines Agency. "While rare diseases by definition have small patient populations, together, they afflict hundreds of millions of people — meaning very large numbers of people across the world have no drug designed to treat their condition," said Jeffrey Kahn, chair of the committee that wrote the report. Learn more at https://ow.ly/6ME850Tm7wx. #RareDisease #RareDiseases #DrugDevelopment #DrugDiscovery

For #WorldSanfilippoAwarenessDay tomorrow, we recognize the importance of spreading awareness and sparking conversations globally about #SanfilippoSyndrome, including what it is and how it impacts families. This year, one of the ways that the Cure Sanfilippo Foundation is helping to spread awareness is through its #HandsofHope viral challenge.   At JCR Pharmaceuticals, we maximize our cutting-edge J-Brain Cargo® technology to develop treatment options for devastating diseases like #SanfilippoSyndrome, and we hold out our hands to stand with the Sanfilippo community to honor the children around the world living with #SanfilippoSyndrome. Our knowledge about #SanfilippoSyndrome has benefited greatly from our conversations with the community and with the Cure Sanfilippo Foundation.   You can learn more how you can participate in the #WorldSanfilippoAwarenessDay here: https://bit.ly/4hi11PQ.   #WorldSanfilippoAwarenessDay #CureSanfilippo #HandsOfHope #SanfilippoSyndrome #RareDisease #JCRPharma #LysosomalStorageDisorders #BloodBrainBarrier #MPSIII

🌍 Join us at the World Orphan Drug Congress 2024! 🌍 We’re excited to announce that Gerdi Strydom, Stephen H., Sarah Holland, and Inês Rodrigues de Oliveira will be representing Valid Insight (part of the Bioscript Group) at the World Orphan Drug Congress in Barcelona, 22-25 October. Come visit us at Booth 111 or schedule a meeting in advance by contacting us at discover@bioscriptgroup.com. Let’s discuss how we can help you navigate the challenges of market access, rare diseases, and orphan drugs. #WorldOrphanDrugCongress, #OrphanDrugs, #RareDisease, #PatientAdvocacy, #MarketAccess

𝐈𝐧𝐯𝐨𝐥𝐯𝐞𝐝 𝐢𝐧 𝐎𝐫𝐩𝐡𝐚𝐧 𝐌𝐞𝐝𝐢𝐜𝐢𝐧𝐞𝐬 𝐃𝐞𝐯𝐞𝐥𝐨𝐩𝐦𝐞𝐧𝐭? Explore the recently published Q&A document by the European Medicines Agency (EMA)! This document follows the interactive webinar held on Rare Disease Day and provides valuable insights for developers of products targeting rare diseases. 🔍 𝘞𝘩𝘢𝘵 𝘵𝘰 𝘌𝘹𝘱𝘦𝘤𝘵 𝘪𝘯 𝘵𝘩𝘦 𝘘&𝘈 𝘋𝘰𝘤𝘶𝘮𝘦𝘯𝘵: - Answers to frequently asked questions about orphan medicines and their development. - Information on orphan drug status, incentives and many more. 𝘍𝘶𝘭𝘭 𝘙𝘦𝘤𝘰𝘳𝘥𝘪𝘯𝘨 𝘈𝘷𝘢𝘪𝘭𝘢𝘣𝘭𝘦: In addition to the document, you can find the full recording of the session online. It’s a unique opportunity to delve deeper into this critical topic! 𝘘𝘶𝘦𝘴𝘵𝘪𝘰𝘯𝘴? Contact AxTalis if you have any queries related to orphan medicines development. #AxTalis #RegulatoryAffairs #EMA #orphandrug https://lnkd.in/eZfJnf-5

On this #RareDiseaseDay, we are raising awareness of the remarkable strength and resilience of individuals and families affected by rare diseases around the world. With STADA's Specialty Pharma segment, we deliver medicines to improve the lives and clinical outcomes of patients worldwide with chronic, complex or rare diseases. #CaringForPeoplesHealth #RareDiseaseDay #RareDiseaseDay2024

Are you an Alpha-1 patient relying on plasma-derived medicines (PDMPs)? Rare diseases like Alpha-1 antitrypsin deficiency can be complex and often life-threatening condition. Without reliable access to PDMPs, patients face serious health risks, loss of independence, mental health challenges, and a reduced quality of life. 🩸💔 At #IPAW2024, we call for better patient access policies for the 300.000 rare disease patient across the EU. 🌍💪 📢 Learn more and share your story at www.plasmaweek.org Plasma Protein Therapeutics Association (PPTA) PPTA Europe 🤝 Alpha1 Österreich-Verein 🇦🇹 Alpha-1 plus-1-plus 🇧🇪 Alpha-1 Deutschland 🇩🇪 Alpha-1 Foundation Ireland 🇮🇪 Associazione Nazionale Alfa 1 AT ODV 🇮🇹 Longfonds 🇳🇱 Alfa-1 Norden 🇩🇰 🇳🇴 🇸🇪 LHL 🇳🇴 Associação Alfa1 de Portugal 🇵🇹 Asociatia Alfasim 🇷🇴 Alfa-1 España 🇪🇸 Lovexair Foundation 🇪🇸 Alpha 1 Verein Schweiz 🇨🇭 Alpha1 UK 🇬🇧

This post is the third installment in #CRALifeSciences’s infographic series exploring pricing and market access trends for rare disease treatments in France. Each post highlights a specific PMA trend by leveraging data from RADAR, CRA’s orphan drug repository, which covers all orphan drugs approved by the EMA between July 2013 and September 2023. In this edition, we focus on the price achieved for a first indication in relation to its ASMR rating. To explore more PMA trends or revisit previous articles on reimbursement timelines and the duration of price negotiations, click here: https://crai.news/wqf

Tune in as our Regulatory Strategists Alexis Northcutt and Ellen Truitt, discuss Orphan Drug Designations (ODD) and Orphan Subsets for the development of treatments for rare diseases. Learn about the classification and the strategic use of subsets in study design. 🎧 Listen to the podcast now 👇 https://bit.ly/3wkcA5W #RareDiseases #RareDiseaseMonth #ODD

🚀 Exciting News from DDW 2024! 🚀 Johnson & Johnson recently presented the maintenance results of the GALAXI 2 & 3 trials for Tremfya (guselkumab) in Crohn's Disease 🔹 Tremfya demonstrated superior efficacy of both SC maintenance doses (200 mg [q4w] and 100 mg [q8w]) compared to ustekinumab (UST) and placebo (pbo) 🔹 Data showed that both maintenance doses of Tremfya met the composite co-primary endpoints compared to placebo in each study 🔹 JNJ submitted marketing applications of guselkumab in the EU and expects to submit for regulatory applications in the US in 2024 for Crohn's Disease 🎯Insights🎯 💡 Guselkumab's EU approval is expected in Q2 2025, aligning with mirikizumab's approval for CD and US approval is anticipated in early Q2/Q3 2025 💡 These approvals could challenge Skyrizi and reshape the IL-23 inhibitor market Link: https://lnkd.in/g4-EjAiH Johnson & Johnson #DDW2024 #Tremfya #CrohnsDisease #ClinicalResearch #Gastroenterology #MedicalInnovation #Inflammatoryboweldisease