Rare disease advocates want Congress to initiate a process that could result in a formal definition for an ultra-rare disease and additional incentives for drug development in the space.
Ultra-Rare Orphan Drugs: Advocates Begin Laying Groundwork For New Incentive, Special Pathway
Rare disease advocates push for National Academies of Sciences, Engineering and Medicine study on how or whether ultra-rare should be defined, which could create legislative pressure. In an interview, Ultragenyx CEO Emil Kakkis suggests patient thresholds, accelerated approval triggers.
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 Pink Sheet
• By
In a historic move for Africa, five products have been backed for approval via a pilot that tested a continental listing process.
• By
CMED, which sets medicine prices in Brazil, has published the maximum price adjustment index for 2025.
• By
Amid ongoing generic drug supply problems, Japan's government is to set up a new fund to support corporate investments geared towards manufacturing and consolidation.