Efforts to create an ultra-rare disease designation and related incentives seem to have hit a roadblock at the US Food and Drug Administration.
Push To Create Ultra-Rare Orphan Drug Designation Finds Resistance At US FDA
Agency officials don’t think a new definition or incentives will fix the issues related to development in rare diseases with only a handful of patients, as advocates continue pushing the idea.
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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.
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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.
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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.
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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.”
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The Brazilian medicines regulator will also offer more clarity on the requirements for radiopharmaceuticals that are exempt from registration.
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South Korea announces planned revisions to a government scheme to designate "innovative" biopharma companies amid allegations of "discrimination" against foreign firms.
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Experts say PBMs should become familiar with transparency rules and prepare to release data, although more legal wrangling could occur.