Cracking the EMA Code: Your Roadmap to European Rare Disease Drug Approval

Watch the next chapter in our Orphan Drug Designation (ODD) webinar series, featuring our principal, Margaret Jenkins, alongside experts from Lumis International GmbH and Alithia Life Sciences.   Building on the foundations from our previous webinar, this session will guide you through key considerations in ODD planning, focusing on how to navigate the regulatory landscape, strategically position your product and helping you maximize the potential of your rare disease therapies.   Watch the third part of our ODD series below, and be sure to follow us on LinkedIn for more insights!   If you missed the earlier sessions, catch up here: Part 1: https://lnkd.in/gcsP9tDc Part 2: https://lnkd.in/g5wnQN32     Heike Schoen Liam Spencer John Oakley A/Prof Tina Soulis ALITHIA LIFE SCIENCES (CRO)     #OrphanDrugDesignation #RareDiseases #RegulatoryStrategy #ClinicalTrials #RegulatoryAffairs #Biotech #Pharma #Healthcare #LumisInternational #AlithiaLifeSciences #GlobalPharmaSolutions

In case you missed it at ISPOR Europe last year, the team presented our research on the use of patient and carer perspectives and real-world evidence in orphan disease health technology assessment submissions. In this video, my colleague, Stephanie Swift (Senior Consultant – Systematic Review, Mtech Access), introduces the key findings from the research. If you’re preparing an evidence dossier for an orphan disease treatment, our experts can help you synthesise real-world and clinical trial evidence to ensure a robust submission for health technology assessment. Get in touch today at [email protected] 👉 To request a copy of the research poster visit: https://lnkd.in/e6UVf-wD #realworldevidence #RWE #HTA #orphandisease #raredisease #pharma #pharmaceuticals #marketaccess #ISPOR

In case you missed it at ISPOR Europe last year, the team presented our research on the use of patient and carer perspectives and real-world evidence in orphan disease health technology assessment submissions. In this video, my colleague, Stephanie Swift (Senior Consultant – Systematic Review, Mtech Access), introduces the key findings from the research. If you’re preparing an evidence dossier for an orphan disease treatment, our experts can help you synthesise real-world and clinical trial evidence to ensure a robust submission for health technology assessment. Get in touch today at [email protected] 👉 To request a copy of the research poster visit: https://lnkd.in/en3K5UZs #realworldevidence #RWE #HTA #orphandisease #raredisease #pharma #pharmaceuticals #marketaccess #ISPOR

This white paper is a good reminder of the clinical and regulatory incentives associated with Orphan drug/biologic development. As Mike Eging states, companies can be caught off guard at the speed to approval and launch if the data are compelling. Commercial strategies including distribution, market, and patient access leads to a greater likelihood of success if considered early.

3D-PharmXchange is proud to be a Bronze Sponsor of RExPo24! As one of the key partners of the REPO4EU project, a Horizon Europe initiative launched in 2022, 3D-PharmXchange provides clinical expert support both strategically and operationally. REPO4EU aims to create a connected European platform for drug repurposing, with the ultimate goal of developing an industry-level online platform for validated precision drug repurposing with global reach. This initiative focuses on understanding and targeting the mechanisms behind diseases, recognizing that combining therapies is essential as most diseases cannot be addressed through a single target. Interested in reading more on how drug repurposing can make a difference in your drug development pipeline? Catch up with Bianca Pauly, Bridget Peterson, Bert Jan Haijema, and Frank Ruwe for more insights into drug repurposing and how it can accelerate your drug development program. Also check out Bianca’s publication in DrugRxiv -- https://lnkd.in/eS5yfCpi For more information on Repo4EU visit: https://repo4.eu/ #RexPo24 #REPO4EU #DrugRepurposing #drugdevelopment #drugrepositioning

In case you missed it at ISPOR Europe last year, the team presented our research on the use of patient and carer perspectives and real-world evidence in orphan disease health technology assessment submissions. In this video, my colleague, Stephanie Swift (Senior Consultant – Systematic Review, Mtech Access), introduces the key findings from the research. If you’re preparing an evidence dossier for an orphan disease treatment, our experts can help you synthesise real-world and clinical trial evidence to ensure a robust submission for health technology assessment. Get in touch today at [email protected] 👉 To request a copy of the research poster visit: https://lnkd.in/ekYCn96F #realworldevidence #RWE #HTA #orphandisease #raredisease #pharma #pharmaceuticals #marketaccess #ISPOR

Here it is, folks: The healthcare industry's equivalent of the script for The Empire Strikes Back has officially launched. And I wrote it! (And I designed it!) Go grab yourself a copy through the link in the post below from Ambrose Healthcare. And if you missed the previous release (ostensibly the healthcare industry's equivalent of the script for A New Hope—I also wrote and designed it), you can catch it here: https://lnkd.in/eBmnumK4 Both white papers examine the current landscape for companies developing drugs for the treatment of rare diseases throughout the world, such as: - Nations leading the drive to invest in rare disease drug development - Government incentives for pharma companies - Recent case studies demonstrating the benefits of leveraging incentives - Technological resources to streamline clinical trials - Strategies for fostering integrated communities of researchers, practitioners, and patients

Rare Disease Leaders in US & APAC: A dual #FDA and #EMA submission strategy can accelerate time-to-market & expand your reach. Learn how in Part 2 of our "Don't Sleep on the EU" series, The Global Ambition, now available over at Partner Rare. Next week's final article, "Hurdles to Harmonization," explores how to leverage EU success for a triumphant return to the FDA after experiencing a setback. Stay tuned. #biotech #pharma #drugdevelopment #clinicaltrials #marketaccess

US & APAC Rare Disease Biotech: FDA setback? Use it to your advantage! Don't let regulatory hurdles derail your global aspirations. Turn them into a strategic opportunity. The European Medicines Agency (EMA) often provides more flexible pathways for Rare Disease therapies, offering a second chance for your innovation to shine. In Article 3, the final instalment of our "Don't Sleep on the EU!" series, we break down: 💚 Why FDA and EMA approvals often diverge 💚 How to leverage EU insights to overcome FDA setbacks 💚 Partner Rare's expert support to navigate the EMA landscape Missed the earlier articles? Catch up now on the link below. Article 1: EU as a Launchpad: Explore how the EU can be a strategic starting point for global Rare Disease therapy approvals. Article 2: The Global Ambition: Streamline #FDA and #EMA approvals with efficient dual-submission strategies. Read Article 3 now! https://lnkd.in/e2PZnDJ9 Turn your challenges into a launchpad for global Rare Disease success! 💚 #RareDisease #Biotech #Pharma #RegulatoryStrategy #GlobalExpansion #PartnerRare

At the end of December, the FDA released draft guidance on “Rare Diseases: Considerations for the Development of Drugs And Biological Products.” The draft provides comprehensive regulatory considerations for the successful clinical development of small molecule- and biologics-based drug products for rare diseases. In a guest column for Clinical Leader, Raj Bandaru, PhD, Associate Director at Accenture Applied Life Sciences Solutions, breaks down the draft section by section. Since orphan drug development is so complicated, it’s worth checking out, particularly if you work in the rare disease space. Source: https://hubs.li/Q02mXtjz0 #rarediseases #FDA #drugdevelopment #orphandrugs #naturalhistorystudies #nonclinicalstudies #qualityconsiderations