Ultragenyx’s Post

Unlocking Access to Life-Changing Therapies: Discover how orphan drugs are transforming the lives of those affected by rare diseases. Swipe through to learn about the challenges, strategies, and our commitment at BioXplore to making these vital treatments accessible to all. 💊✨ #RareDiseaseAwareness #OrphanDrugs #HealthTechnology #PatientAccess #PharmaInnovation #HealthcareStrategy #BioXplore #ValueAssessment #PatientAdvocacy #LifeChangingTherapies #MedicalBreakthroughs #DrugDevelopment #HealthcareSolutions #MarketAccess #PharmaceuticalIndustry

Interesting article on the challenges of RWE studies for reimbursement decision-making in rare diseases, and suggestions for addressing them #RWE #HTA #Outcomes https://lnkd.in/dm_UpZia

Today is Rare Disease Day, and in honor of it, the House Energy and Commerce Committee will hold a hearing to discuss more than a dozen bills that offer support to patients. Among those bills are the ORPHAN Cures Act and the BENEFIT Act. While both are good first steps, more must be done. CAHC's latest blog examines how Congress could create real change by amending the Inflation Reduction Act's unfair treatment of orphan drugs, leading to more treatments for rare diseases. Learn more: https://lnkd.in/ec-rF_vQ #rarediseaseday #rarediseaseweek #RareDC2024 #orphandrugs #inflationreductionact #congress #legislation

With high development costs and little ROI, many rare diseases were neglected – until the Orphan Drug Act of 1983. Recap it with us and see how we can support your program: https://bit.ly/3Op3PPi #RareDiseases #OrphanDrugs #OrphanDrugDesignation

Today, February 29th, is Rare Disease Day, raising awareness for the hundreds of million people living with a rare disease. Read our latest blog, 'Rare Diseases, Pharma’s Perennial Hitmaker' here: https://lnkd.in/eHaRfiRP   Over its 20 year history, Deallus has supporting the clinical development, market access, commercialisation and advancement of 100+ rare diseases. We recognise that there is still more to be done with only about 5% of the 10,000 rare diseases recognised by the NIH’s Genetic and Rare Disease (GARD) Information Centre having approved medicines.   Hormoz M., one of our Rare Diseases experts, has written a blog reflecting on how rare diseases drug development will be commonplace within the industry, and how Deallus is supporting pharma organisations and committed to supporting drug development in this area. You can read the blog here: https://lnkd.in/eHaRfiRP #rarediseaseday2024 #rarediseases #healthcareinequality #thoughtleadership #blog #expertise #orphandrugs #fdaapproval #commitmenttochange #drugdevelopment

According to the European Organization for Rare Diseases (EURORDIS) there are over 300 million people living with one or more of over 6,000 identified rare diseases around the world. This is equivalent to the population of the world’s third largest country. With Rare Disease day this year falling on 29th February, the rarest day of the year, there is an opportunity to reflect on what still needs to be done to ensure equity in access to diagnostics and treatments for rare diseases. In this blog, my colleague & Deallus Rare Disease expert Hormoz M. reflects on the great advances we have seen over the past 20 years in driving new treatment options within rare diseases. #rarediseaseday #rarediseaseawareness #healthequity

#ICYMI: Nazim Kanji, Executive Director of Pediatric Services, speaks on the challenges of rare disease development in this article with Manufacturing Chemist. Read here: https://bit.ly/4950uvQ

FDA establishes a Rare Disease Innovation Hub to enhance treatment development, promote collaboration, and advance #regulatory science for over 30 million people in the US with #rarediseases. Read at The Evidence Base® #heor #healtheconomics #outcomesresearch #regulation #regulatoryaffairs #healthdata #healthequity #healthpolicy

📃: 𝗠𝗲𝗱𝗶𝗰𝗶𝗻𝗲𝘀 𝗳𝗼𝗿 𝗿𝗮𝗿𝗲 𝗱𝗶𝘀𝗲𝗮𝘀𝗲𝘀 𝗺𝗮𝗸𝗲 𝘂𝗽 𝗼𝘃𝗲𝗿 𝗵𝗮𝗹𝗳 𝗼𝗳 𝗻𝗲𝘄 𝗱𝗿𝘂𝗴 𝗮𝗽𝗽𝗿𝗼𝘃𝗮𝗹𝘀 𝗶𝗻 𝘁𝗵𝗲 𝗨𝗦 𝗮𝗻𝗱 𝗘𝘂𝗿𝗼𝗽𝗲. 🧬💊 𝐷𝑒𝑠𝑝𝑖𝑡𝑒 𝑠𝑖𝑔𝑛𝑖𝑓𝑖𝑐𝑎𝑛𝑡 𝑖𝑛𝑛𝑜𝑣𝑎𝑡𝑖𝑜𝑛𝑠, 95% 𝑜𝑓 𝑡ℎ𝑒 7,000+ 𝑟𝑎𝑟𝑒 𝑑𝑖𝑠𝑒𝑎𝑠𝑒𝑠 𝑠𝑡𝑖𝑙𝑙 𝑙𝑎𝑐𝑘 𝑡𝑟𝑒𝑎𝑡𝑚𝑒𝑛𝑡𝑠. 𝑇ℎ𝑒 𝑒𝑐𝑜𝑛𝑜𝑚𝑖𝑐 𝑏𝑢𝑟𝑑𝑒𝑛? 𝐴 𝑠𝑡𝑎𝑔𝑔𝑒𝑟𝑖𝑛𝑔 $997 𝑏𝑖𝑙𝑙𝑖𝑜𝑛 𝑖𝑛 𝑡ℎ𝑒 𝑈.𝑆. alone. 💸 🔎 Discover how we can navigate the complexities of launching these critical therapies in an increasingly challenging healthcare landscape. How do you see the future of rare disease treatments evolving? 🤔 Hit that comment button and share your insights! 🚀🗨️ Tag a colleague who's passionate about rare diseases. 🌐✨ #RareDiseases #meetingtheunmet #TreatmentAccess #pharmaindustry #pharmabusiness

As demonstrated in Vol. 6 of our Millennium Health Signals Report™ The “Fourth Wave”: The Rise of Stimulants and the Evolution of Polysubstance Use in America’s Fentanyl Crisis, urine drug testing data tightly correlate with trends in fentanyl-involved overdose mortality and closely follow the evolution of America’s fentanyl crisis. https://ow.ly/K2Fg50QPeLV