HI-Bio: A Biogen Company’s Post

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

Exciting news from Amgen as Uplizna proves its worth in another rare disease setting! Learn more about the regulatory filings in the US and EU. #RareDisease #Amgen #Uplizna

Exciting news from Amgen as Uplizna proves its worth in another rare disease setting! Learn more about the regulatory filings in the US and EU. #RareDisease #Amgen #Uplizna

Exciting news from Amgen as Uplizna proves its worth in another rare disease setting! Learn more about the regulatory filings in the US and EU. #RareDisease #Amgen #Uplizna

Exciting news from Amgen as Uplizna proves its worth in another rare disease setting! Learn more about the regulatory filings in the US and EU. #RareDisease #Amgen #Uplizna

Exciting news from Amgen as Uplizna proves its worth in another rare disease setting! Learn more about the regulatory filings in the US and EU. #RareDisease #Amgen #Uplizna

🚀 Exciting news for the rare disease community! 🚀 The FDA has launched the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) Pilot Program, aimed at accelerating the development of therapies for rare diseases. As part of START, participants will receive invaluable guidance and support from FDA experts, addressing key challenges in clinical trial design and patient population identification. However, challenges still persist. Many in the industry have raised concerns about the Inflation Reduction Act (IRA), which some view as a barrier to orphan drug developers. While orphan drugs are exempt from IRA negotiations, this exemption only applies to drugs approved for one specific indication. Looking ahead, there's hope for positive change. Advocates are urging governmental regulators to enhance incentives for rare disease drug development in the coming year, paving the way for more treatments to reach the market. Let's work together to bring hope and healing to those affected by rare diseases. Together, we can make a difference. 💪 #RareDisease #FDA #ClinicalTrials #OrphanDrugs #HealthcareInnovation #citeline

Exciting news from Amgen as Uplizna proves its worth in another rare disease setting! Learn more about the regulatory filings in the US and EU. #RareDisease #Amgen #Uplizna

For rheumatoid arthritis patients with persistent remission or low disease activity, recommendations support tapering biological DMARDs. However, this trial-and-error approach is associated with an increased risk of flare-ups. In this study published in Annals of Rheumatic Diseases, a therapeutic drug monitoring-based tapering strategy showed promising results in both efficacy and cost reduction. ➡ https://hubs.ly/Q02Jh3nC0 | #TherapeuticDrugMonitoring #PrecisionMedicine #ClinicalTrials

Exciting news from Amgen as Uplizna proves its worth in another rare disease setting! Learn more about the regulatory filings in the US and EU. #RareDisease #Amgen #Uplizna