Orsini - Rare Disease Pharmacy Solutions’ Post

Partnering with Sciensus reduces risk and adds value to your European expansion. We believe in making sure every family has access to rare disease medicines and the care they need. No matter where a patient lives or the regulatory governance of that country, we can safely launch and efficiently deliver vital medication and clinical services for families that need it most. Find out how Sciensus can support you launch your orphan drug: https://lnkd.in/e5PtQYet #RareDiseases #Biotechs #MarketAccess #OrphanDrugs

READ NOW: NAVLIN Last Week in Review https://ow.ly/KW7150SsPgf This week from NAVLIN by EVERSANA's “Last Week in Review,” recapping the latest regulatory changes, pricing & reimbursement updates, drug approvals and more from across the world: Policy: Learn how new criteria could impact market access and treatments like Gilead’s Trodelvy. Pricing & Reimbursement Discover how Vertex’s long-term agreement with NHS England marks a new era for cystic fibrosis treatment availability. HTA: Learn how CSL Behring’s Hemgenix is set to transform the treatment landscape for adults with severe hemophilia B, following NICE’s final draft guidance. Other: Discover how the EMA and HMA are addressing shortages of key GLP-1 receptor agonists like Ozempic, Saxenda, Trulicity, and Victoza. And more... #NAVLIN #CysticFibrosis #HemophiliaBTreatment #MarketAccess #DrugEvaluation

When the EMA Orphan Drug Designation (ODD) designation is awarded, it provides a sponsor additional support and financial benefits to incentivize the advancement of treatments in the rare disease space that is not generally available to all sponsors. In this whitepaper, Pace® experts explain the value of pursuing this designation and provide insights into the application process. Request the whitepaper here: https://ow.ly/oucB50SAB9K

Just about a week to go and counting! I am excited to see everyone in San Diego at #DIA2024. What a treat to discuss "Sustainability of Rare Disease Drug Development" with an esteemed panel of FDA experts including Julie Tierney, Patroula Smpokou, MD, and Katherine Donigan - of course expertly moderated by Darcy Frear, PhD. Current incentives for #raredisease #drugdevelopment including the #orphandrugact #ODA have changed to world for many #raredisease patients and families. We went from fewer than 40 FDA approved therapies in 1983 before the #ODA to #orphandrugs consistently making up more than 50% of new drug approvals (#NMEs) at #FDA. But more than 95% of rare diseases still lack any FDA-approved therapies. So how do we sustainably improve rare disease drug development and bring life-changing therapies to those communities that have for too long been left behind? There are so many promising approaches, pilots, and policy priorities to make this dream a reality, and I can't wait to discuss them with this esteemed panel of experts!

Our new #BrainHealthTimeMatters is now available: https://lnkd.in/eDavWAg So many have made this work possible. Our thanks go to Alexion Pharma Germany GmbH and Merck Healthcare for sponsoring the launch of this report and helping bring this work to all; to Roche, MS Society and Horizon, now part of Amgen for their critical support towards the development of the report; to the panel of authors Helmut Butzkueven, Gavin Giovannoni, Sofia Arkelsten, Giancarlo Comi, Kathy Costello, Michael Devlin, Jelena Drulovic, Emma Gray, Jodi Haartsen, Anne Helme, Jana Hlaváčová, Elisabeth Kasilingam, Yaou Liu, Thomas Mathew, Saul Reyes, Jérôme de Sèze and Mitzi Joi Williams M.D., FAAN and to the many members of the Working Groups named in the report who brought expertise and evidence to shape and support the recommendations. We thank you and look forward the next steps - collaborating with you and further stakeholders to support the implementation of the recommendations. #BrainHealthTM #BrainHealth #MSTimeMatters, #NMOSDTimeMatters, #MOGADTimeMatters, #MSBH #OHPF, The Sumaira Foundation, Sumaira Ahmed, EMSP - European Multiple Sclerosis Platform, MS International Federation (MSIF), Can Do MS

It’s published 👏 a collaboration with over 60 experts across geography and disciplines. Delve in and see what it can mean for you and how you can support the implementation of recommendations for #MS, #NMOSD, #MOGAD and related disorders. MS Brain Health Oxford Health Policy Forum #MSBH #BrainHealthTM #NMOSDTimeMatters #MOGADTimeMatters

Out now: the updated 'MS Brain Health – time matters' report was launched at ECTRIMS yesterday! It highlights the importance of early diagnosis, timely treatment and holistic, person-centred care, along with the policy changes needed to improve long-term outcomes for those living with MS, NMOSD and MOGAD. #BrainHealthTM #BrainHealth #MSTimeMatters

#raredisease #drugdevelopment is really hard and inherently risky - and it can be tremendously rewarding! When else do you have a chance to bring the first treatment - and a glimmer of hope - to a community affected by really serious conditions, with incredible unmet medical need, and an almost unprecedented resolve to change the world. Rare disease drug development is a big priority for FDA. Great primer and summary of many of the efforts #CDER is undertaking to help spur rare disease drug development published this week: https://lnkd.in/eZRs3Dsv

The GARDIAN Registry has released data on the first 26 patients: 96% with Gaucher disease Type 3 (GD3) and 4% with Type 2 (GD2). GARDIAN (Gaucher Registry for Development, Innovation, and Analysis of Neuronopathic Disease) is a global initiative by the International Gaucher Alliance (IGA). It aims to improve understanding of GD2 and GD3 by collecting personal experiences from patients and families. At Prevail Therapeutics, we celebrate the remarkable work of the IGA as we recognize the critical need to include patient and caregiver voices throughout the drug development process. By collaborating with the IGA to help support GARDIAN, we hope to develop a more authentic understanding of the patient experience that will improve current drug development efforts. Discover more about the results here: https://bit.ly/4eipC5w #GARDIANRegistry #GaucherDisease #InternationalGaucherAlliance #PatientDrivenResearch #InnovationInHealthcare

FDA Oncologic Drugs Advisory Committee (ODAC) meeting voted in favor of J&J's #Carvykti (11-0) and BMS & 2seventy bio's #Abecma (8-3)—recommending both CAR T therapies for the treatment of #RRMM patients. This is something to start a conversation about: https://lnkd.in/eY--MxYT