The US Health and Human Services Department’s communications freeze and President Trump’s threat to halt federal grant funding have stressed the rare disease community.
Rare Disease Community Stressed By US Funding Threats, Communications Freeze
There has been “a lot of fear and anxiety” resulting from the Trump Administration’s early actions aimed at freezing grants and FDA communications, NORD’s Pamela Gavin says.
More from Rare Diseases
• By
Not all companies will be able to access joint scientific consultations under the EU Health Technology Assessment Regulation, but success is still possible for those that engage with national agencies early on, says EUCOPE’s Alexander Natz.
• By
Payers and health technology assessment bodies in the Netherlands, Germany and Italy are either unwilling to use real-world data in assessments or cannot due to their existing frameworks, say representatives from Gilead Sciences and Autolus Therapeutics.
• By
Companies have until 17 March to respond to an EU consultation on a new strategy that will seek to simplify the regulatory framework and make it easier for innovative small and medium-sized enterprises to “access the capital they need” to scale up in the bloc.
• By
Center for Biologics Evaluation and Research Director Peter Marks expects no fundamental changes in support for FDA initiatives and said the Rare Disease Innovation Hub, which promotes cross-center collaboration, is “consistent with what we're hearing in the current environment.”
More from Geography
• By
The European Medicines Agency has recommended five drugs for EU-wide approval , including Averoa’s Xoanacyl for concomitant hyperphosphatemia. Two companies have withdrawn their marketing authorization applications.
• By
Eli Lilly’ will request a re-examination after the European Medicines Agency declined to recommend its Alzheimer’s disease drug Kisunla for EU approval.
• By
Recent and upcoming US FDA advisory committee meetings and a summary of the topics covered.