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
England’s health technology assessment institute, NICE, is looking to “reimagine” its evaluation process with the help of AI, rather than just using this technology to speed up its existing processes.
• 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.
More from Pink Sheet
• By
Most, if not all, adcomm support staff in the drug and biologic centers who handled meeting-specific tasks have been laid off, although sponsors still are preparing for the public reviews. Meanwhile, some cut FDA employees are being called back to work for "continuity of operations activities."
• By
Product reviews for drugs, biologics, devices, tobacco and foods could be consolidated into one office at the FDA, as part of a restructuring plan circulating that was obtained by Pink Sheet.
• By
Pink Sheet reporter and editors discuss how the massive layoffs will reshape the FDA and its impact on the drug and biologics industries.